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    Clinical Trial Results:
    A Phase 2 Dose Ranging, Randomized, Double Blind, Placebo-controlled Study Evaluating the Safety, Tolerability, Pharmacokinetics and Efficacy of EDP-305 in Subjects with Primary Biliary Cholangitis (PBC) with or without an Inadequate Response to Ursodeoxycholic Acid (UDCA)

    Summary
    EudraCT number
    2017-003528-62
    Trial protocol
    DE   GB   BE   ES   AT   NL  
    Global end of trial date
    16 Jan 2020

    Results information
    Results version number
    v1(current)
    This version publication date
    07 Feb 2021
    First version publication date
    07 Feb 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    EDP 305-201
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03394924
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Enanta Pharmaceuticals, Inc.
    Sponsor organisation address
    500 Arsenal St., Watertown, United States, MA 02472
    Public contact
    Nathalie Adda, Enanta Pharmaceuticals, Inc., +1 6176070705, nadda@enanta.com
    Scientific contact
    Nathalie Adda, Enanta Pharmaceuticals, Inc., +1 6176070705, nadda@enanta.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    16 Jan 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    16 Jan 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to evaluate the effect of EDP-305 on alkaline phosphatase (ALP) levels.
    Protection of trial subjects
    This study was conducted in accordance with the protocol, the guideline for Good Clinical Practice E6(R2), the Declaration of Helsinki, and all applicable local laws and national regulations governing clinical studies.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    13 Dec 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 30
    Country: Number of subjects enrolled
    Canada: 3
    Country: Number of subjects enrolled
    Australia: 5
    Country: Number of subjects enrolled
    Spain: 7
    Country: Number of subjects enrolled
    United Kingdom: 13
    Country: Number of subjects enrolled
    Austria: 2
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    France: 1
    Country: Number of subjects enrolled
    Germany: 4
    Country: Number of subjects enrolled
    Netherlands: 2
    Worldwide total number of subjects
    68
    EEA total number of subjects
    30
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    51
    From 65 to 84 years
    17
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The trial included 68 participants from 43 sites in Australia, Canada, Europe and the United states from December 2017 to January 2020.

    Pre-assignment
    Screening details
    132 participants were screened, 64 of which were screen failures. The remaining 68 were enrolled and received trial treatment.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    EDP-305 1 mg
    Arm description
    Participants took EDP-305 1 mg as an oral tablet once daily for 12 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    EDP-305
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet

    Arm title
    EDP-305 2.5 mg
    Arm description
    Participants took EDP-305 2.5 mg as an oral tablet once daily for 12 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    EDP-305
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet

    Arm title
    Placebo
    Arm description
    Participants received an oral placebo matching EDP-305 once daily for 12 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Oral tablet

    Number of subjects in period 1
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Started
    31
    28
    9
    Completed
    28
    22
    9
    Not completed
    3
    6
    0
         Consent withdrawn by subject
    2
    -
    -
         Did not meet all inclusion criteria
    1
    -
    -
         Adverse event, non-fatal
    -
    5
    -
         Lost to follow-up
    -
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    EDP-305 1 mg
    Reporting group description
    Participants took EDP-305 1 mg as an oral tablet once daily for 12 weeks.

    Reporting group title
    EDP-305 2.5 mg
    Reporting group description
    Participants took EDP-305 2.5 mg as an oral tablet once daily for 12 weeks.

    Reporting group title
    Placebo
    Reporting group description
    Participants received an oral placebo matching EDP-305 once daily for 12 weeks.

    Reporting group values
    EDP-305 1 mg EDP-305 2.5 mg Placebo Total
    Number of subjects
    31 28 9 68
    Age categorical
    Units: Subjects
        In utero
    0
        Preterm newborn infants (gestational age < 37 wks)
    0
        Newborns (0-27 days)
    0
        Infants and toddlers (28 days-23 months)
    0
        Children (2-11 years)
    0
        Adolescents (12-17 years)
    0
        Adults (18-64 years)
    0
        From 65-84 years
    0
        85 years and over
    0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    57.4 ± 8.61 54.9 ± 10.92 56.9 ± 8.49 -
    Gender categorical
    Units: Subjects
        Female
    31 27 9 67
        Male
    0 1 0 1
    Race
    Units: Subjects
        White
    28 27 9 64
        All other races
    3 1 0 4
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    3 2 0 5
        Not Hispanic or Latino
    27 25 9 61
        Not Reported
    1 1 0 2
        Unknown
    0 0 0 0

    End points

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    End points reporting groups
    Reporting group title
    EDP-305 1 mg
    Reporting group description
    Participants took EDP-305 1 mg as an oral tablet once daily for 12 weeks.

    Reporting group title
    EDP-305 2.5 mg
    Reporting group description
    Participants took EDP-305 2.5 mg as an oral tablet once daily for 12 weeks.

    Reporting group title
    Placebo
    Reporting group description
    Participants received an oral placebo matching EDP-305 once daily for 12 weeks.

    Subject analysis set title
    EDP-305 1 mg - Pharmacokinetic Substudy
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Blood samples for Pharmacokinetic (PK) analysis were collected from a subset of study sites.

    Subject analysis set title
    EDP-305 2.5 mg - Pharmacokinetic Substudy
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Blood samples for Pharmacokinetic (PK) analysis were collected from a subset of study sites.

    Primary: Percentage of Participants with At Least a 20% Reduction in Alkaline Phosphatase (ALP) or Normalization of ALP at Week 12 Compared to Baseline

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    End point title
    Percentage of Participants with At Least a 20% Reduction in Alkaline Phosphatase (ALP) or Normalization of ALP at Week 12 Compared to Baseline
    End point description
    End point type
    Primary
    End point timeframe
    Baseline and Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31
    28
    9
    Units: Percentage of participants
        number (not applicable)
    45.2
    46.4
    11.1
    Statistical analysis title
    Primary Analysis for EDP-305 1 mg
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.106
    Method
    Cochran-Mantel-Haenszel
    Parameter type
    Odds ratio (OR)
    Point estimate
    5.6
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.6
         upper limit
    52
    Statistical analysis title
    Primary Analysis for EDP-305 2.5 mg
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.063
    Method
    Cochran-Mantel-Haenszel
    Parameter type
    Odds ratio (OR)
    Point estimate
    7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.75
         upper limit
    65.22

    Secondary: Percentage of Participants With a Treatment-Emergent Adverse Event (TEAE) during On-Treatment Period

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    End point title
    Percentage of Participants With a Treatment-Emergent Adverse Event (TEAE) during On-Treatment Period
    End point description
    An adverse event (AE) was defined as any event, side effect, or untoward medical occurrence in a subject enrolled in a clinical trial whether or not it is considered to have a causal relationship to the study drug. A TEAE was an AE that first occurred or began previous to and worsened on or after the first dose date and before the last dose date +7 days.
    End point type
    Secondary
    End point timeframe
    Up to approximately Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31
    28
    9
    Units: Percentage of participants
        number (not applicable)
    71.0
    89.3
    88.9
    No statistical analyses for this end point

    Secondary: Percentage of Participants With a Treatment-Emergent Serious Adverse Event (SAE) during On-Treatment Period

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    End point title
    Percentage of Participants With a Treatment-Emergent Serious Adverse Event (SAE) during On-Treatment Period
    End point description
    A SAE is any untoward medical occurrence at any dose that results in death, is a life-threatening event, requires inpatient hospitalization or prolonged hospitalization of an existing hospitalization, results in permanent or prolonged disability or incapacity, is a congenital anomaly or birth defect in the offspring of a study subjects, or is a medically important event.
    End point type
    Secondary
    End point timeframe
    Up to approximately Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31
    28
    9
    Units: Percentage of participants
        number (not applicable)
    3.2
    7.1
    0
    No statistical analyses for this end point

    Secondary: Percentage of Participants Who Stopped Study Treatment Due to a Treatment-Emergent Adverse Event (TEAE) during On-Treatment Period

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    End point title
    Percentage of Participants Who Stopped Study Treatment Due to a Treatment-Emergent Adverse Event (TEAE) during On-Treatment Period
    End point description
    An adverse event (AE) was defined as any event, side effect, or untoward medical occurrence in a subject enrolled in a clinical trial whether or not it is considered to have a causal relationship to the study drug. A TEAE was an AE that first occurred or began previous to and worsened on or after the first dose date and before the last dose date +7 days.
    End point type
    Secondary
    End point timeframe
    Up to approximately Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31
    28
    9
    Units: Percentage of participants
        number (not applicable)
    3.2
    17.9
    0
    No statistical analyses for this end point

    Secondary: Change from Baseline to Week 12 in Total, Conjugated and Unconjugated Bilirubin

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    End point title
    Change from Baseline to Week 12 in Total, Conjugated and Unconjugated Bilirubin
    End point description
    The data presented below was measured using least square mean change from baseline.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [1]
    28 [2]
    9
    Units: µmol/L
    least squares mean (confidence interval 95%)
        Total
    -0.04 (-0.93 to 0.86)
    -0.31 (-1.35 to 0.74)
    -0.50 (-2.13 to 1.12)
        Conjugated
    -0.55 (-1.03 to -0.06)
    -0.51 (-1.08 to 0.06)
    0.13 (-0.75 to 1.00)
        Unconjugated
    0.71 (-0.02 to 1.44)
    0.22 (-0.61 to 1.06)
    -0.50 (-1.81 to 0.81)
    Notes
    [1] - Analysed participants: 28
    [2] - Analysed participants: 21
    Statistical analysis title
    Analysis for EDP-305 1 mg Total Bilirubin
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.616
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.47
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.39
         upper limit
    2.32
    Statistical analysis title
    Analysis for EDP-305 2.5 mg Total Bilirubin
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.844
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.19
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.78
         upper limit
    2.16
    Statistical analysis title
    Analysis for EDP-305 1 mg Conjugated Bilirubin
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.18
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.67
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.67
         upper limit
    0.32
    Statistical analysis title
    Analysis for EDP-305 2.5 mg Conjugated Bilirubin
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.239
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.64
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.71
         upper limit
    0.44
    Statistical analysis title
    Analysis for EDP-305 1 mg Unconjugated Bilirubin
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.116
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    1.21
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.31
         upper limit
    2.73
    Statistical analysis title
    Analysis for EDP-305 2.5 mg Unconjugated Bilirubin
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.36
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.72
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.84
         upper limit
    2.28

    Secondary: Change from Baseline to Week 12 in alanine aminotransferase (ALT), aspartate aminotransferase (AST), and gamma glutamyl transferase (GGT)

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    End point title
    Change from Baseline to Week 12 in alanine aminotransferase (ALT), aspartate aminotransferase (AST), and gamma glutamyl transferase (GGT)
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [3]
    28 [4]
    9
    Units: U/L
    least squares mean (confidence interval 95%)
        ALT
    -17.35 (-24.45 to -10.25)
    -13.14 (-21.58 to -4.71)
    8.20 (-4.61 to 21.02)
        AST
    -12.08 (-17.49 to -6.68)
    -11.51 (-17.91 to -5.10)
    9.33 (-0.43 to 19.09)
        GGT
    -95.91 (-114.12 to -77.70)
    -124.55 (-146.00 to -103.11)
    -9.42 (-42.19 to 23.35)
    Notes
    [3] - Analysed participants: 28
    [4] - Analysed participants: 21
    Statistical analysis title
    Analysis for EDP-305 1 mg ALT
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.001
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -25.55
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -40.07
         upper limit
    -11.04
    Statistical analysis title
    Analysis for EDP-305 2.5 mg ALT
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.009
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -21.35
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -37.1
         upper limit
    -5.6
    Statistical analysis title
    Analysis for EDP-305 1 mg AST
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -21.42
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -32.48
         upper limit
    -10.35
    Statistical analysis title
    Analysis for EDP-305 2.5 mg AST
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.001
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -20.84
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -32.8
         upper limit
    -8.87
    Statistical analysis title
    Analysis for EDP-305 1 mg GGT
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -86.49
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -123.78
         upper limit
    -49.21
    Statistical analysis title
    Analysis for EDP-305 2.5 mg GGT
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -115.13
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -155.04
         upper limit
    -75.22

    Secondary: Change from Baseline to Week 12 in Noninvasive Liver Fibrosis Markers: Enhanced Liver Fibrosis (ELF) panel and N-terminal Type III Collagen Propeptide (PRO C3)

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    End point title
    Change from Baseline to Week 12 in Noninvasive Liver Fibrosis Markers: Enhanced Liver Fibrosis (ELF) panel and N-terminal Type III Collagen Propeptide (PRO C3)
    End point description
    The ELF panel included hyaluronic acid (HA), procollagen III amino terminal peptide (PIIINP), and tissue inhibitor of metalloproteinase 1 (TIMP 1). This endpoint also presents PRO C3 results.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [5]
    28 [6]
    9 [7]
    Units: µg/L
    least squares mean (confidence interval 95%)
        HA
    1.17 (-16.69 to 19.03)
    -1.16 (-22.20 to 19.87)
    27.83 (-4.20 to 59.86)
        PIIINP
    -0.08 (-1.28 to 1.11)
    -0.77 (-2.20 to 0.65)
    3.01 (0.72 to 5.30)
        TIMP 1
    -16.29 (-32.31 to -0.27)
    -20.90 (-39.58 to -2.22)
    25.66 (-3.64 to 54.97)
        PRO C3
    -0.67 (-4.97 to 3.63)
    2.33 (-2.31 to 6.96)
    9.06 (2.16 to 15.95)
    Notes
    [5] - Analysed participants: 28 for HA, PIIINP and TIMP 1; 19 for PRO C3.
    [6] - Analysed participants: 21 for HA, PIIINP and TIMP 1; 18 for PRO C3.
    [7] - Analysed participants: 9 for HA, PIIINP and TIMP 1; 8 for PRO C3.
    Statistical analysis title
    Analysis for EDP-305 1 mg HA
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.148
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -26.66
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -63.1
         upper limit
    9.79
    Statistical analysis title
    Analysis for EDP-305 2.5 mg HA
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.142
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -28.99
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -68.02
         upper limit
    10.05
    Statistical analysis title
    Analysis for EDP-305 1 mg PIIINP
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.02
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -3.09
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.68
         upper limit
    -0.51
    Statistical analysis title
    Analysis for EDP-305 2.5 mg PIIINP
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.009
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -3.79
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.6
         upper limit
    -0.97
    Statistical analysis title
    Analysis for EDP-305 1 mg TIMP 1
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.015
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -41.96
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -75.47
         upper limit
    -8.44
    Statistical analysis title
    Analysis for EDP-305 2.5 mg TIMP 1
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.011
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -46.56
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -81.91
         upper limit
    -11.21
    Statistical analysis title
    Analysis for EDP-305 1 mg PRO C3
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 27.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.018
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -9.73
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -17.7
         upper limit
    -1.75
    Statistical analysis title
    Analysis for EDP-305 2.5 mg PRO C3
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 26.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.125
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -6.73
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -15.41
         upper limit
    1.95

    Secondary: Change from Baseline to Week 12 in Noninvasive Liver Fibrosis Markers: AST to Platelet Ratio Index (APRI) Score

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    End point title
    Change from Baseline to Week 12 in Noninvasive Liver Fibrosis Markers: AST to Platelet Ratio Index (APRI) Score
    End point description
    APRI was calculated as ([AST IU/L/AST ULN]/[Platelet count 1^09/L])×100. AST is aspartate aminotransferase. ALT is alanine aminotransferase. IU is International Units. ULN is Upper Limit of Normal range for test.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [8]
    28 [9]
    9 [10]
    Units: AST to Platelet Ratio Index
        least squares mean (confidence interval 95%)
    -0.16 (-0.25 to 0.06)
    -0.12 (-0.23 to 0.01)
    0.22 (0.05 to 0.38)
    Notes
    [8] - Analysed participants: 24
    [9] - Analysed participants: 20
    [10] - Analysed participants: 8
    Statistical analysis title
    Analysis for EDP-305 1 mg APRI
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 32.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.37
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.56
         upper limit
    -0.18
    Statistical analysis title
    Analysis for EDP-305 2.5 mg APRI
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 28.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.002
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.33
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.54
         upper limit
    -0.13

    Secondary: Change from Baseline to Week 12 in Noninvasive Liver Fibrosis Markers: Fibrosis-4 (FIB-4) Score

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    End point title
    Change from Baseline to Week 12 in Noninvasive Liver Fibrosis Markers: Fibrosis-4 (FIB-4) Score
    End point description
    FIB-4 score was calculated as (Age [years]×AST [IU/L])/(Platelet count [10^9/L]×(sqrt ALT [IU/L])). AST is aspartate aminotransferase. ALT is alanine aminotransferase. IU is International Units. ULN is Upper Limit of Normal range for test.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [11]
    28 [12]
    9 [13]
    Units: Fibrosis 4 Index
        least squares mean (confidence interval 95%)
    -0.14 (-0.29 to 0.01)
    -0.05 (-0.22 to 0.11)
    0.21 (-0.06 to 0.47)
    Notes
    [11] - Analysed participants: 24
    [12] - Analysed participants: 20
    [13] - Analysed participants: 8
    Statistical analysis title
    Analysis for EDP-305 1 mg FIB-4
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 32.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.026
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.35
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.65
         upper limit
    -0.04
    Statistical analysis title
    Analysis for EDP-305 2.5 mg FIB-4
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 28.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.104
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.26
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.57
         upper limit
    0.05

    Secondary: Change from Baseline to Week 12 in Fibrinogen and C Reactive Protein (CRP) Levels

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    End point title
    Change from Baseline to Week 12 in Fibrinogen and C Reactive Protein (CRP) Levels
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [14]
    28 [15]
    9 [16]
    Units: mg/dL
    least squares mean (confidence interval 95%)
        Fibrinogen
    16.28 (-5.39 to 37.95)
    41.25 (15.56 to 66.93)
    9.47 (-28.82 to 47.76)
        CRP
    -0.57 (-1.62 to 0.49)
    -2.69 (-3.89 to -1.50)
    0.41 (-1.53 to 2.34)
    Notes
    [14] - Analysed participants: 28 for fibrinogen; 27 for CRP.
    [15] - Analysed participants: 20 for fibrinogen; 21 for CRP.
    [16] - Analysed participants: 9 for fibrinogen; 8 for CRP.
    Statistical analysis title
    Analysis for EDP-305 1 mg Fibrinogen
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.757
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    6.81
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -37.17
         upper limit
    50.78
    Statistical analysis title
    Analysis for EDP-305 2.5 mg Fibrinogen
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 29.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.174
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    31.77
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -14.42
         upper limit
    77.97
    Statistical analysis title
    Analysis for EDP-305 1 mg CRP
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 35.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.378
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.98
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.18
         upper limit
    1.23
    Statistical analysis title
    Analysis for EDP-305 2.5 mg CRP
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 29.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.008
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -3.1
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.38
         upper limit
    -0.83

    Secondary: Change from Baseline to Week 12 in Interleukin (IL) and Tumor Necrosis Factor (TNF) Levels

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    End point title
    Change from Baseline to Week 12 in Interleukin (IL) and Tumor Necrosis Factor (TNF) Levels
    End point description
    For IL, both IL6 and IL1β variants were analysed. For TNF, both TNF α and TNF β (also known as lymphotoxin alpha) variants were analyzed. Values of 99999 indicate that data could not be calculated.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [17]
    28 [18]
    9 [19]
    Units: ng/L
    least squares mean (confidence interval 95%)
        IL6
    0.73 (-0.97 to 2.43)
    -2.10 (-4.03 to -0.17)
    0.39 (-2.57 to 3.34)
        IL1β
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    99999 (99999 to 99999)
        TNF α
    -0.15 (-0.38 to 0.09)
    -0.01 (-0.28 to 0.25)
    0.39 (-0.03 to 0.81)
        TNF β
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    Notes
    [17] - Analysed participants: 28 for TNF α and IL1β; 27 for IL6; 19 for TNF β.
    [18] - Analysed participants: 21 for TNF α and IL1β; 21 for IL6; 18 for TNF β.
    [19] - Analysed participants: 9 for TNF α, IL1β and IL6; 8 for TNF β.
    Statistical analysis title
    Analysis for EDP-305 1 mg IL6
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 36.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.841
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.34
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.07
         upper limit
    3.76
    Statistical analysis title
    Analysis for EDP-305 2.5 mg IL6
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.163
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -2.49
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.01
         upper limit
    1.04
    Statistical analysis title
    Analysis for EDP-305 1 mg TNF α
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.03
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.53
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.02
         upper limit
    -0.05
    Statistical analysis title
    Analysis for EDP-305 2.5 mg TNF α
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.11
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.9
         upper limit
    0.09

    Secondary: Change from Baseline to Week 12 in Haptoglobin and Alpha2 Macroglobulin Levels

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    End point title
    Change from Baseline to Week 12 in Haptoglobin and Alpha2 Macroglobulin Levels
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [20]
    28 [21]
    9
    Units: g/L
    least squares mean (confidence interval 95%)
        Haptoglobin
    -0.14 (-0.27 to -0.01)
    -0.18 (-0.33 to -0.03)
    -0.15 (-0.38 to 0.08)
        Alpha2 Macroglobulin
    -0.02 (-0.09 to 0.05)
    -0.00 (-0.08 to 0.08)
    0.02 (-0.11 to 0.15)
    Notes
    [20] - Analysed participants: 28
    [21] - Analysed participants: 21
    Statistical analysis title
    Analysis for EDP-305 1 mg Haptoglobin
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.944
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.01
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.25
         upper limit
    0.27
    Statistical analysis title
    Analysis for EDP-305 2.5 mg Haptoglobin
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.83
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.03
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.3
         upper limit
    0.24
    Statistical analysis title
    Analysis for EDP-305 1 mg Alpha2 Macroglobulin
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.546
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.04
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.19
         upper limit
    0.1
    Statistical analysis title
    Analysis for EDP-305 2.5 mg Alpha2 Macroglobulin
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.785
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.02
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.18
         upper limit
    0.13

    Secondary: Change from Baseline to Week 12 in Triglycerides (TG), Total Cholesterol (TC), High Density Lipoprotein Cholesterol (HDL-C), Low Density Lipoprotein Cholesterol (LDL-C)

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    End point title
    Change from Baseline to Week 12 in Triglycerides (TG), Total Cholesterol (TC), High Density Lipoprotein Cholesterol (HDL-C), Low Density Lipoprotein Cholesterol (LDL-C)
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [22]
    28 [23]
    9
    Units: mmol/L
    least squares mean (confidence interval 95%)
        TG
    -0.13 (-0.25 to -0.00)
    0.01 (-0.13 to 0.16)
    0.05 (-0.17 to 0.27)
        TC
    -0.47 (-0.80 to -0.14)
    -0.46 (-0.85 to -0.08)
    0.17 (-0.42 to 0.76)
        HDL-C
    -0.16 (-0.31 to -0.02)
    -0.46 (-0.62 to -0.30)
    -0.24 (-0.49 to 0.01)
        LDL-C
    -0.21 (-0.47 to 0.05)
    -0.01 (-0.31 to 0.29)
    0.29 (-0.19 to 0.76)
    Notes
    [22] - Analysed participants: 28
    [23] - Analysed participants: 21
    Statistical analysis title
    Analysis for EDP-305 1 mg TG
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.176
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.17
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.43
         upper limit
    0.08
    Statistical analysis title
    Analysis for EDP-305 2.5 mg TG
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.783
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.04
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.3
         upper limit
    0.23
    Statistical analysis title
    Analysis for EDP-305 1 mg TC
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.061
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.64
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.31
         upper limit
    0.03
    Statistical analysis title
    Analysis for EDP-305 2.5 mg TC
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.08
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.63
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.34
         upper limit
    0.08
    Statistical analysis title
    Analysis for EDP-305 1 mg HDL-C
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.584
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.08
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.21
         upper limit
    0.37
    Statistical analysis title
    Analysis for EDP-305 2.5 mg HDL-C
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.148
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.22
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.51
         upper limit
    0.08
    Statistical analysis title
    Analysis for EDP-305 1 mg LDL-C
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.074
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.04
         upper limit
    0.05
    Statistical analysis title
    Analysis for EDP-305 2.5 mg LDL-C
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.304
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.87
         upper limit
    0.28

    Secondary: Change from Baseline to Week 12 in Domain and Total Scores on the 5D-Itch Scale

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    End point title
    Change from Baseline to Week 12 in Domain and Total Scores on the 5D-Itch Scale
    End point description
    The 5D-Itch scale is a multidimensional questionnaire completed by participants to quantify the magnitude of pruritus, assessed considering the past 2 weeks. Scale range is 1 to 5 covering five dimensions: duration (1=Less than 6 hrs/day to 5=All day), degree (1=Not present to 5=Unbearable), direction (1=Completely resolved to 5=Getting worse), disability (for Sleep rated as 1=Never affects sleep to 5=Delays falling asleep and frequently wakes me up at night; for Leisure/Social, Housework/Errands and Work/School rated as 1=Never affects activity to 5=Always affects activity), and distribution (assess if itching is present in 16 body locations, scored as 1=present at 0-2 locations to 5=present at 14-16 locations). Total scores (including highest disability score obtained from any of the daily activities) ranged between 5 and 25 where higher scores indicated more severe itching. Negative change scores indicate improvement from the baseline score.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [24]
    28 [25]
    9 [26]
    Units: Scores on a scale
    least squares mean (confidence interval 95%)
        Duration
    0.01 (-0.27 to 0.29)
    0.41 (0.11 to 0.71)
    -0.24 (-0.73 to 0.25)
        Degree
    0.00 (-0.25 to 0.26)
    0.65 (0.37 to 0.93)
    -0.53 (-0.96 to 0.10)
        Direction
    -0.63 (-1.12 to -0.15)
    0.36 (-0.17 to 0.90)
    -0.65 (-1.46 to 0.16)
        Disability
    -0.24 (-0.63 to 0.15)
    0.85 (0.42 to 1.28)
    -0.61 (-1.28 to 0.05)
        Distribution
    0.07 (-0.32 to 0.45)
    0.81 (0.38 to 1.24)
    -0.42 (-1.08 to 0.24)
        Total
    -0.95 (-2.29 to 0.39)
    3.19 (1.74 to 4.64)
    -3.16 (-5.50 to -0.82)
    Notes
    [24] - Analysed: 24 (Duration and Total); 25 (Direction); 26 (Degree, Disability and Distribution)
    [25] - Analysed: 21
    [26] - Analysed: 8 (Duration and Total); 9 (Direction, Degree, Disability and Distribution)
    Statistical analysis title
    Analysis for EDP-305 1 mg - Duration
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 32.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.378
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.25
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.32
         upper limit
    0.82
    Statistical analysis title
    Analysis for EDP-305 2.5 mg - Duration
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 29.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.03
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.65
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.07
         upper limit
    1.23
    Statistical analysis title
    Analysis for EDP-305 1 mg - Degree
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 35.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.036
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.53
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.04
         upper limit
    1.03
    Statistical analysis title
    Analysis for EDP-305 2.5 mg - Degree
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    1.18
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.67
         upper limit
    1.69
    Statistical analysis title
    Analysis for EDP-305 1 mg - Direction
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 34.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.971
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.02
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.92
         upper limit
    0.96
    Statistical analysis title
    Analysis for EDP-305 2.5 mg - Direction
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.042
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    1.01
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.04
         upper limit
    1.99
    Statistical analysis title
    Analysis for EDP-305 1 mg - Disability
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 35.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.336
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.37
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.4
         upper limit
    1.14
    Statistical analysis title
    Analysis for EDP-305 2.5 mg - Disability
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    1.46
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.67
         upper limit
    2.26
    Statistical analysis title
    Analysis for EDP-305 1 mg - Distribution
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 35.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.214
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.48
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.29
         upper limit
    1.25
    Statistical analysis title
    Analysis for EDP-305 2.5 mg - Distribution
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.003
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    1.23
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.44
         upper limit
    2.02
    Statistical analysis title
    Analysis for EDP-305 1 mg - Total
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 32.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.103
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    2.21
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.47
         upper limit
    4.9
    Statistical analysis title
    Analysis for EDP-305 2.5 mg - Total
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 29.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    6.35
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    3.57
         upper limit
    9.13

    Secondary: Change from Baseline to Week 12 in Visual Analog Score (VAS) for Itching

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    End point title
    Change from Baseline to Week 12 in Visual Analog Score (VAS) for Itching
    End point description
    An itch VAS (0-100mm) was used to record the intensity of the event. Participants drew a line on a scale corresponding to the maximum intensity of itch. Lines drawn towards the right of the line indicated greater itching and higher scores indicated more severe itching. Negative change scores indicate that participants’ scores decreased from the baseline score.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [27]
    28 [28]
    9
    Units: Scores on a scale
        least squares mean (confidence interval 95%)
    0.55 (-8.35 to 9.44)
    13.64 (4.00 to 23.29)
    -11.93 (-27.05 to 3.18)
    Notes
    [27] - Analysed participants: 26
    [28] - Analysed participants: 22
    Statistical analysis title
    Analysis for EDP-305 1 mg VAS Scale
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 35.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.16
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    12.48
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.09
         upper limit
    30.06
    Statistical analysis title
    Analysis for EDP-305 2.5 mg VAS Scale
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 31.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.006
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    25.58
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    7.67
         upper limit
    43.48

    Secondary: Change from Baseline to Week 12 in Domain Scores on the Primary Biliary Cholangitis-40 (PBC-40) Quality of Life (QoL) Assessment

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    End point title
    Change from Baseline to Week 12 in Domain Scores on the Primary Biliary Cholangitis-40 (PBC-40) Quality of Life (QoL) Assessment
    End point description
    The PBC-40 is a survey measuring health related quality of life in participants with PBC. The 40 questions from the PBC-40 questionnaire are scored from 1-5, with 5 representing the highest impact and 1 the lowest impact of PBC on the quality of life. Six domains were computed from the 40 questions: symptoms (score range 7-35), itch (0-15), fatigue (11-55), cognition (6-30), social (8-50) and emotional (1-15). Higher scores indicate worse quality of life and negative change scores indicate improvement from the baseline score.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [29]
    28 [30]
    9
    Units: Scores on a scale
    least squares mean (confidence interval 95%)
        Symptoms
    -0.21 (-1.47 to 1.06)
    -1.46 (-2.93 to 0.00)
    -0.06 (-2.30 to 2.18)
        Itch
    0.18 (-0.72 to 1.09)
    1.74 (0.69 to 2.78)
    -1.73 (-3.33 to -0.13)
        Fatigue
    -0.36 (-2.58 to 1.86)
    -0.22 (-2.79 to 2.34)
    0.10 (-3.82 to 4.01)
        Cognition
    -0.21 (-1.46 to 1.04)
    0.82 (-0.63 to 2.27)
    -0.48 (-2.66 to 1.71)
        Social
    -0.38 (-2.22 to 1.46)
    0.96 (-1.17 to 3.08)
    1.73 (-1.52 to 4.99)
        Emotional
    -0.77 (-1.54 to 0.01)
    0.23 (-0.68 to 1.13)
    -0.15 (-1.52 to 1.22)
    Notes
    [29] - Analysed participants: 28
    [30] - Analysed participants: 21
    Statistical analysis title
    Analysis for EDP-305 1 mg Symptoms
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.908
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.15
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.72
         upper limit
    2.43
    Statistical analysis title
    Analysis for EDP-305 2.5 mg Symptoms
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.298
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -1.41
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.09
         upper limit
    1.27
    Statistical analysis title
    Analysis for EDP-305 1 mg Itch
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.042
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    1.91
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.07
         upper limit
    3.76
    Statistical analysis title
    Analysis for EDP-305 2.5 mg Itch
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    Placebo v EDP-305 2.5 mg
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.001
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    3.47
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1.55
         upper limit
    5.38
    Statistical analysis title
    Analysis for EDP-305 1 mg Fatigue
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.839
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.46
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.96
         upper limit
    4.04
    Statistical analysis title
    Analysis for EDP-305 2.5 mg Fatigue
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    Placebo v EDP-305 2.5 mg
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.891
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.32
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5
         upper limit
    4.36
    Statistical analysis title
    Analysis for EDP-305 1 mg Cognition
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.834
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.26
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.25
         upper limit
    2.77
    Statistical analysis title
    Analysis for EDP-305 2.5 mg Cognition
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    Placebo v EDP-305 2.5 mg
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.327
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    1.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.33
         upper limit
    3.92
    Statistical analysis title
    Analysis for EDP-305 1 mg Social
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.262
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -2.12
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.86
         upper limit
    1.62
    Statistical analysis title
    Analysis for EDP-305 2.5 mg Social
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    Placebo v EDP-305 2.5 mg
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.69
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.78
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.68
         upper limit
    3.12
    Statistical analysis title
    Analysis for EDP-305 1 mg Emotional
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 37.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.433
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -0.62
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.19
         upper limit
    0.95
    Statistical analysis title
    Analysis for EDP-305 2.5 mg Emotional
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    Placebo v EDP-305 2.5 mg
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.653
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    0.37
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.28
         upper limit
    2.03

    Secondary: Maximum Plasma Concentration (Cmax) of EDP-305 and its Metabolites

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    End point title
    Maximum Plasma Concentration (Cmax) of EDP-305 and its Metabolites
    End point description
    Metabolites of EDP-305 are EP-022571, EP-022572, and EP-022679.
    End point type
    Secondary
    End point timeframe
    Day 1 and Week 12: Pre-dose and 2, 6 and 8 hours post-dose
    End point values
    EDP-305 1 mg - Pharmacokinetic Substudy EDP-305 2.5 mg - Pharmacokinetic Substudy
    Number of subjects analysed
    5 [31]
    6 [32]
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        EDP-305 Day 1
    15.4 ± 44.30
    27.9 ± 54.19
        EDP-305 Week 12
    10.8 ± 67.77
    50.4 ± 53.20
        EP-022571 Day 1
    0.5 ± 44.58
    0.6 ± 61.81
        EP-022571 Week 12
    0.2 ± 59.57
    1.3 ± 160.28
        EP-022572 Day 1
    0.5 ± 41.69
    0.8 ± 38.27
        EP-022572 Week 12
    0.2 ± 41.74
    1.6 ± 138.55
        EP-022679 Day 1
    0.8 ± 113.24
    1.7 ± 115.17
        EP-022679 Week 12
    0.6 ± 147.81
    10.9 ± 352.53
    Notes
    [31] - Analysed participants: 5 at Day 1 and 4 at Week 12.
    [32] - Analysed participants: 5 at Day 1 and 4 at Week 12.
    No statistical analyses for this end point

    Secondary: Time to Maximum Plasma Concentration (Tmax) of EDP-305 and its Metabolites

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    End point title
    Time to Maximum Plasma Concentration (Tmax) of EDP-305 and its Metabolites
    End point description
    Metabolites of EDP-305 are EP-022571, EP-022572, and EP-022679.
    End point type
    Secondary
    End point timeframe
    Day 1 and Week 12: Pre-dose and 2, 6 and 8 hours post-dose
    End point values
    EDP-305 1 mg - Pharmacokinetic Substudy EDP-305 2.5 mg - Pharmacokinetic Substudy
    Number of subjects analysed
    5 [33]
    6 [34]
    Units: hours
    median (full range (min-max))
        EDP-305 Day 1
    6.00 (2.02 to 6.03)
    6.02 (2.00 to 8.00)
        EDP-305 Week 12
    7.01 (6.00 to 8.02)
    6.01 (2.05 to 8.02)
        EP-022571 Day 1
    2.0 (2.0 to 6.0)
    6.0 (2.0 to 6.1)
        EP-022571 Week 12
    6.0 (2.0 to 6.0)
    4.0 (2.0 to 6.1)
        EP-022572 Day 1
    2.0 (2.0 to 8.0)
    6.0 (2.0 to 6.1)
        EP-022572 Week 12
    6.0 (6.0 to 8.0)
    4.0 (2.0 to 6.0)
        EP-022679 Day 1
    6.0 (6.0 to 8.0)
    6.1 (2.0 to 8.0)
        EP-022679 Week 12
    6.0 (6.0 to 8.0)
    6.0 (6.0 to 8.0)
    Notes
    [33] - Analysed participants: 5 at Day 1 and 4 at Week 12.
    [34] - Analysed participants: 5 at Day 1 and 4 at Week 12.
    No statistical analyses for this end point

    Secondary: Area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration (AUClast) of EDP-305 and its Metabolites

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    End point title
    Area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration (AUClast) of EDP-305 and its Metabolites
    End point description
    Metabolites of EDP-305 are EP-022571, EP-022572, and EP-022679.
    End point type
    Secondary
    End point timeframe
    Day 1 and Week 12: Pre-dose and 2, 6 and 8 hours post-dose
    End point values
    EDP-305 1 mg - Pharmacokinetic Substudy EDP-305 2.5 mg - Pharmacokinetic Substudy
    Number of subjects analysed
    5 [35]
    6 [36]
    Units: h*ng/mL
    geometric mean (geometric coefficient of variation)
        EDP-305 Day 1
    85.5 ± 51.57
    95.3 ± 141.72
        EDP-305 Week 12
    67.6 ± 75.47
    316.2 ± 42.34
        EP-022571 Day 1
    2.3 ± 37.32
    2.0 ± 132.47
        EP-022571 Week 12
    0.9 ± 78.75
    7.3 ± 154.62
        EP-022572 Day 1
    2.5 ± 32.37
    2.9 ± 96.70
        EP-022572 Week 12
    1.2 ± 53.12
    10.1 ± 148.55
        EP-022679 Day 1
    3.7 ± 109.30
    5.0 ± 305.62
        EP-022679 Week 12
    3.0 ± 203.04
    57.0 ± 343.47
    Notes
    [35] - Analysed participants: 5 at Day 1 and 4 at Week 12.
    [36] - Analysed participants: 5 at Day 1 and 4 at Week 12.
    No statistical analyses for this end point

    Secondary: Percentage Change from Baseline to Week 12 in Fibroblast Growth Factor 19 (FGF19), 7α-OH-4-cholesten-3-one (C4) and Bile Acid (BA) Concentrations

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    End point title
    Percentage Change from Baseline to Week 12 in Fibroblast Growth Factor 19 (FGF19), 7α-OH-4-cholesten-3-one (C4) and Bile Acid (BA) Concentrations
    End point description
    FGF19 was measured in plasma. BA was measured in serum. C4 was measured in serum.
    End point type
    Secondary
    End point timeframe
    Day 1 and Week 12: Pre-dose and 2, 6 and 8 hours post-dose.
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [37]
    28 [38]
    9 [39]
    Units: Percentage change from baseline
    arithmetic mean (standard deviation)
        FGF19
    28.10 ± 94.526
    46.90 ± 76.667
    39.83 ± 100.579
        C4
    -18.066 ± 114.9959
    -61.960 ± 42.8603
    39.839 ± 163.6265
        BA
    -21.79 ± 74.581
    -15.79 ± 112.934
    3.17 ± 48.656
    Notes
    [37] - Analysed participants: 26 for FGF19 and C4; 23 for BA.
    [38] - Analysed participants: 20 for FGF19 and C4; 14 for BA.
    [39] - Analysed participants: 7
    Statistical analysis title
    Analysis for EDP-305 1 mg FGF19
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 33.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.971
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    1.34
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -71.51
         upper limit
    74.18
    Statistical analysis title
    Analysis for EDP-305 2.5 mg FGF19
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 27.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.882
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    5.54
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -68.86
         upper limit
    79.95
    Statistical analysis title
    Analysis for EDP-305 1 mg C4
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 33.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.242
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -51.66
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -139.27
         upper limit
    35.96
    Statistical analysis title
    Analysis for EDP-305 2.5 mg C4
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 27.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.042
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -94.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -184.85
         upper limit
    -3.75
    Statistical analysis title
    Analysis for EDP-305 1 mg BA
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 30.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.52
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -24.57
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -101.03
         upper limit
    51.89
    Statistical analysis title
    Analysis for EDP-305 2.5 mg BA
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 21.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.672
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -17.96
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -103.07
         upper limit
    67.16

    Secondary: Percentage Change from Baseline to Week 12 in AUC0-8 and AUC2-8 of Fibroblast Growth Factor 19 (FGF19), 7α-OH-4-cholesten-3-one (C4) and Bile Acid (BA)

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    End point title
    Percentage Change from Baseline to Week 12 in AUC0-8 and AUC2-8 of Fibroblast Growth Factor 19 (FGF19), 7α-OH-4-cholesten-3-one (C4) and Bile Acid (BA)
    End point description
    AUC0-8 is area under the biomarker concentration-time curve from time zero to 8 hours. AUC2-8 is area under the biomarker concentration-time curve from 2 hours to 8 hours. FGF19 was measured in plasma. BA was measured in serum. C4 was measured in serum. Values of 99999 indicate that data could not be calculated.
    End point type
    Secondary
    End point timeframe
    Day 1 and Week 12: Pre-dose and 2, 6 and 8 hours post-dose
    End point values
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Number of subjects analysed
    31 [40]
    28 [41]
    9 [42]
    Units: Percentage change from baseline
    arithmetic mean (standard deviation)
        FGF19 AUC0-8
    24.9 ± 68.90
    25.0 ± 71.72
    -25.9 ± 99999
        FGF19 AUC 2-8
    7.8 ± 86.25
    18.9 ± 66.35
    -25.3 ± 99999
        C4 AUC0-8
    3.7 ± 51.74
    100.0 ± 0.0
    138.2 ± 99999
        C4 AUC2-8
    -9.2 ± 66.30
    -100.0 ± 0.0
    122.5 ± 99999
        BA AUC0-8
    -20.7 ± 74.80
    -51.5 ± 41.06
    -26.1 ± 99999
        BA AUC2-8
    -33.6 ± 76.26
    -42.8 ± 56.77
    -24.4 ± 99999
    Notes
    [40] - Analysed participants: 4 for AUC0-8 FGF19, C4 and BA; 5 for AUC2-8 FGF19, C4 and BA.
    [41] - Analysed participants: 2 for AUC0-8 and AUC0-8 FGF19, and BA; 1 for AUC0-8 and AUC2-8 C4.
    [42] - Analysed participants: 1
    Statistical analysis title
    Analysis for EDP-305 1 mg FGF19 AUC0-8
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 5.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.611
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    44.54
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -205.97
         upper limit
    295.06
    Statistical analysis title
    Analysis for EDP-305 2.5 mg FGF19 AUC0-8
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 3.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.819
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -29.98
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -411.28
         upper limit
    351.31
    Statistical analysis title
    Analysis for EDP-305 1 mg FGF19 AUC2-8
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 6.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.818
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    24.41
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -251.92
         upper limit
    300.74
    Statistical analysis title
    Analysis for EDP-305 2.5 mg FGF19 AUC2-8
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 3.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.948
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -10.53
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -435.33
         upper limit
    414.27
    Statistical analysis title
    Analysis for EDP-305 1 mg C4 AUC0-8
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 5.
    Comparison groups
    Placebo v EDP-305 1 mg
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.412
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -91.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -475.92
         upper limit
    292.51
    Statistical analysis title
    Analysis for EDP-305 2.5 mg C4 AUC0-8
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 2.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.094
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -250.18
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -605.33
         upper limit
    104.98
    Statistical analysis title
    Analysis for EDP-305 1 mg C4 AUC2-8
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 6.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.266
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -83.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -279.15
         upper limit
    111.75
    Statistical analysis title
    Analysis for EDP-305 2.5 mg C4 AUC2-8
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 2.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.047
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -238.19
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -470.22
         upper limit
    -6.15
    Statistical analysis title
    Analysis for EDP-305 1 mg BA AUC0-8
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 5.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.694
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    28.41
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -180.2
         upper limit
    237.02
    Statistical analysis title
    Analysis for EDP-305 2.5 mg BA AUC0-8
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 3.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.615
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -39.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -264.32
         upper limit
    185.32
    Statistical analysis title
    Analysis for EDP-305 1 mg BA AUC2-8
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 6.
    Comparison groups
    EDP-305 1 mg v Placebo
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.974
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    2.93
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -229.6
         upper limit
    235.46
    Statistical analysis title
    Analysis for EDP-305 2.5 mg BA AUC2-8
    Statistical analysis description
    Number of subjects included in analysis presented is automatically calculated by the system based on overall summary table “Number of subjects analysed.” Actual number included in this statistical comparison is 3.
    Comparison groups
    EDP-305 2.5 mg v Placebo
    Number of subjects included in analysis
    37
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.793
    Method
    ANCOVA
    Parameter type
    Least squares mean difference
    Point estimate
    -26.05
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -283.24
         upper limit
    231.15

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 16 Weeks
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    20.1
    Reporting groups
    Reporting group title
    EDP-305 1 mg
    Reporting group description
    -

    Reporting group title
    EDP-305 2.5 mg
    Reporting group description
    -

    Reporting group title
    Placebo
    Reporting group description
    -

    Serious adverse events
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 31 (3.23%)
    2 / 28 (7.14%)
    0 / 9 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 28 (3.57%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 28 (3.57%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 28 (3.57%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 28 (3.57%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urticaria
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 28 (3.57%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Escherichia urinary tract infection
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 28 (3.57%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Bacterial infection
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 28 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    EDP-305 1 mg EDP-305 2.5 mg Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    23 / 31 (74.19%)
    24 / 28 (85.71%)
    8 / 9 (88.89%)
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    2 / 31 (6.45%)
    0 / 28 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    3
    0
    0
    Contusion
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 31 (9.68%)
    5 / 28 (17.86%)
    3 / 9 (33.33%)
         occurrences all number
    3
    8
    3
    Paraesthesia
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    1
    0
    1
    Amnesia
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Hyperaesthesia
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    2 / 31 (6.45%)
    1 / 28 (3.57%)
    1 / 9 (11.11%)
         occurrences all number
    2
    1
    1
    Influenza like illness
         subjects affected / exposed
    0 / 31 (0.00%)
    2 / 28 (7.14%)
    0 / 9 (0.00%)
         occurrences all number
    0
    2
    0
    Swelling
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Eye disorders
    Dry eye
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    4 / 31 (12.90%)
    1 / 28 (3.57%)
    0 / 9 (0.00%)
         occurrences all number
    5
    1
    0
    Diarrhoea
         subjects affected / exposed
    1 / 31 (3.23%)
    3 / 28 (10.71%)
    0 / 9 (0.00%)
         occurrences all number
    1
    3
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 31 (3.23%)
    3 / 28 (10.71%)
    0 / 9 (0.00%)
         occurrences all number
    1
    6
    0
    Nausea
         subjects affected / exposed
    2 / 31 (6.45%)
    3 / 28 (10.71%)
    1 / 9 (11.11%)
         occurrences all number
    2
    3
    1
    Abdominal distension
         subjects affected / exposed
    2 / 31 (6.45%)
    1 / 28 (3.57%)
    0 / 9 (0.00%)
         occurrences all number
    2
    1
    0
    Dry mouth
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 28 (3.57%)
    2 / 9 (22.22%)
         occurrences all number
    0
    1
    2
    Dyspepsia
         subjects affected / exposed
    0 / 31 (0.00%)
    2 / 28 (7.14%)
    0 / 9 (0.00%)
         occurrences all number
    0
    3
    0
    Toothache
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 28 (3.57%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Vomiting
         subjects affected / exposed
    2 / 31 (6.45%)
    0 / 28 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    0
    Reproductive system and breast disorders
    Menorrhagia
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 28 (3.57%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Oropharyngeal pain
         subjects affected / exposed
    0 / 31 (0.00%)
    2 / 28 (7.14%)
    0 / 9 (0.00%)
         occurrences all number
    0
    2
    0
    Epistaxis
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Sinus congestion
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    11 / 31 (35.48%)
    16 / 28 (57.14%)
    3 / 9 (33.33%)
         occurrences all number
    27
    59
    7
    Pruritus generalised
         subjects affected / exposed
    5 / 31 (16.13%)
    9 / 28 (32.14%)
    0 / 9 (0.00%)
         occurrences all number
    5
    13
    0
    Alopecia
         subjects affected / exposed
    1 / 31 (3.23%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    1
    0
    1
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 31 (0.00%)
    3 / 28 (10.71%)
    0 / 9 (0.00%)
         occurrences all number
    0
    3
    0
    Anxiety
         subjects affected / exposed
    2 / 31 (6.45%)
    0 / 28 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 28 (3.57%)
    2 / 9 (22.22%)
         occurrences all number
    0
    1
    2
    Arthralgia
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 28 (3.57%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Bursitis
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Myalgia
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    2 / 31 (6.45%)
    2 / 28 (7.14%)
    0 / 9 (0.00%)
         occurrences all number
    2
    2
    0
    Nasopharyngitis
         subjects affected / exposed
    1 / 31 (3.23%)
    2 / 28 (7.14%)
    1 / 9 (11.11%)
         occurrences all number
    1
    2
    1
    Upper respiratory tract infection
         subjects affected / exposed
    3 / 31 (9.68%)
    0 / 28 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    3
    0
    0
    Sinusitis
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 28 (3.57%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Bronchitis
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Cytomegalovirus infection
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 28 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 31 (3.23%)
    2 / 28 (7.14%)
    0 / 9 (0.00%)
         occurrences all number
    1
    2
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Apr 2018
    • Various changes in the inclusion and exclusion criteria to better define the target subject population. • Additional details were included for the management of liver enzymes: the original protocol was incomplete as it did not address close observation of subjects for whom repeat assessment showed persistent elevations of transaminases, but who did not meet drug discontinuation criteria. For these subjects, the “close observation” guidelines were to be followed as noted in the FDA Guidance for Industry – Drug-Induced Liver Injury: Premarketing Clinical Evaluation.

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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