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    Clinical Trial Results:
    A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Explore the Efficacy and Safety of BIO89-100 in Subjects With Severe Hypertriglyceridemia

    Summary
    EudraCT number
    		 2020-000641-13
    Trial protocol
    		 HU   CZ   PL  
    Global end of trial date
    31 Mar 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 Dec 2024
    First version publication date
    28 Dec 2024
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    BIO89-100-221
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04541186
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    89bio, Inc.
    Sponsor organisation address
    655 Montgomery Street, Suite 1500, San Francisco, CA, United States, 94111
    Public contact
    Entrigue Study Team, 89bio, Inc., 1 4154329270, Ct.gov_SHTG@89bio.com
    Scientific contact
    Entrigue Study Team , 89bio, Inc., 1 4154329270, Ct.gov_SHTG@89bio.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
    18 Jan 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    19 May 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Mar 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    This study was designed to assess the efficacy, safety, and tolerability of different doses and dose regimens (once weekly [QW] or every 2 weeks [Q2W]), subcutaneous (SC) dosing of BIO89-100 (pegozafermin) compared to placebo in participants with severe hypertriglyceridemia (SHTG).
    Protection of trial subjects
    This study was conducted according to the ethical principles derived from the Declaration of Helsinki and Council for International Organizations of Medical Sciences (CIOMS) International Ethical Guidelines, International Council for Harmonisation (ICH) guidelines for Good Clinical Practice (GCP), and applicable laws and regulations.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Sep 2020
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 58
    Country: Number of subjects enrolled
    Hungary: 10
    Country: Number of subjects enrolled
    Poland: 14
    Country: Number of subjects enrolled
    Czechia: 4
    Worldwide total number of subjects
    86
    EEA total number of subjects
    28
    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)
    70
    From 65 to 84 years
    16
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants in the Main Study cohort were randomized in a 1:1:1:1:1 ratio to 1 of 4 doses of pegozafermin (9 mg QW, 18 mg QW, 27 mg QW, or 36 mg Q2W) or matching placebo. Participants in the Fibrate Expansion cohort were randomized in a 1:1 ratio to pegozafermin 27 mg QW or matching placebo.

    Pre-assignment
    Screening details
    		 Four participants randomized to 9 mg received 18 mg instead. One participant was randomized but did not receive treatment. Full Analysis set analyzed participants as per randomized assignment. Safety Analysis set analyzed participants as per treatment received.

    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
    		 Pegozafermin 9 mg QW
    Arm description
    		 Pegozafermin 9 mg QW was administered as a SC injection.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Pegozafermin
    Investigational medicinal product code
    Other name
    BIO89-100
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received pegozafermin 9 mg QW as an SC injection for 8 weeks in the Main Study cohort.

    Arm title
    		 Pegozafermin 18 mg QW
    Arm description
    		 Pegozafermin 18 mg QW was administered as an SC injection.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Pegozafermin
    Investigational medicinal product code
    Other name
    BIO89-100
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received pegozafermin 18 mg QW as an SC injection for 8 weeks in the Main Study cohort.

    Arm title
    		 Pegozafermin 27 mg QW
    Arm description
    		 Pegozafermin 27 mg QW was administered as an SC injection. The Main Study cohort and Fibrate Expansion cohorts for pegozafermin 27 mg were pooled together due to low sample size in the expansion cohort.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Pegozafermin
    Investigational medicinal product code
    Other name
    BIO89-100
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received pegozafermin 27 QW as an SC injection for 8 weeks in the Main Study cohort or Fibrate Expansion cohort.

    Arm title
    		 Pegozafermin 36 mg Q2W
    Arm description
    		 Pegozafermin 36 mg Q2W was administered as an SC injection.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Pegozafermin
    Investigational medicinal product code
    Other name
    BIO89-100
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received pegozafermin 36 mg Q2W as an SC injection for 8 weeks in the Main Study cohort.

    Arm title
    		 Placebo
    Arm description
    		 Matching placebo was injected at matching frequency per assigned cohort. The Main Study cohort and Fibrate Expansion cohorts for placebo were pooled together due to low sample size in the expansion cohort.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Matching placebo was injected at matching frequency per assigned cohort for 8 weeks in the Main Study cohort or Fibrate Expansion cohort.

    Number of subjects in period 1
    		Pegozafermin 9 mg QW Pegozafermin 18 mg QW Pegozafermin 27 mg QW Pegozafermin 36 mg Q2W Placebo
    Started
    16
    17
    19
    16
    18
    Full Analysis Set (FAS)
    16
    17
    16
    16
    17
    Received at Least 1 Dose of Study Drug
    16
    17
    18
    16
    18
    Completed
    15
    16
    13
    15
    16
    Not completed
    1
    1
    6
    1
    2
         Consent withdrawn by subject
    1
    1
    -
    -
    -
         Adverse event, non-fatal
    -
    -
    4
    -
    -
         COVID-19-related Dose Interruption
    -
    -
    -
    -
    1
         Randomized in Error Not Treated
    -
    -
    1
    -
    -
         Other than Specified
    -
    -
    -
    -
    1
         Investigator Decision
    -
    -
    1
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Pegozafermin 9 mg QW
    Reporting group description
    		 Pegozafermin 9 mg QW was administered as a SC injection.

    Reporting group title
    Pegozafermin 18 mg QW
    Reporting group description
    		 Pegozafermin 18 mg QW was administered as an SC injection.

    Reporting group title
    Pegozafermin 27 mg QW
    Reporting group description
    		 Pegozafermin 27 mg QW was administered as an SC injection. The Main Study cohort and Fibrate Expansion cohorts for pegozafermin 27 mg were pooled together due to low sample size in the expansion cohort.

    Reporting group title
    Pegozafermin 36 mg Q2W
    Reporting group description
    		 Pegozafermin 36 mg Q2W was administered as an SC injection.

    Reporting group title
    Placebo
    Reporting group description
    		 Matching placebo was injected at matching frequency per assigned cohort. The Main Study cohort and Fibrate Expansion cohorts for placebo were pooled together due to low sample size in the expansion cohort.

    Reporting group values
    		 Pegozafermin 9 mg QW Pegozafermin 18 mg QW Pegozafermin 27 mg QW Pegozafermin 36 mg Q2W Placebo Total
    Number of subjects
    		 16 17 19 16 18 86
    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)
    		 54.6 ( 12.71 ) 49.2 ( 10.85 ) 54.5 ( 7.43 ) 53.1 ( 12.36 ) 57.5 ( 10.30 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 5 3 5 2 6 21
        Male
    		 11 14 14 14 12 65

    End points

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    End points reporting groups
    Reporting group title
    Pegozafermin 9 mg QW
    Reporting group description
    		 Pegozafermin 9 mg QW was administered as a SC injection.

    Reporting group title
    Pegozafermin 18 mg QW
    Reporting group description
    		 Pegozafermin 18 mg QW was administered as an SC injection.

    Reporting group title
    Pegozafermin 27 mg QW
    Reporting group description
    		 Pegozafermin 27 mg QW was administered as an SC injection. The Main Study cohort and Fibrate Expansion cohorts for pegozafermin 27 mg were pooled together due to low sample size in the expansion cohort.

    Reporting group title
    Pegozafermin 36 mg Q2W
    Reporting group description
    		 Pegozafermin 36 mg Q2W was administered as an SC injection.

    Reporting group title
    Placebo
    Reporting group description
    		 Matching placebo was injected at matching frequency per assigned cohort. The Main Study cohort and Fibrate Expansion cohorts for placebo were pooled together due to low sample size in the expansion cohort.

    Subject analysis set title
    Pegozafermin
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    FAS: All randomized participants who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline triglyceride (TG) measurement not including end of study (EOS) visit. Main study and fibrate expansion cohorts were combined due to the low number of participants in fibrate expansion cohort. All dose groups for pegozafermin were pooled to evaluate the overall effectiveness of pegozafermin. Here, ‘Overall number of participants analyzed’ = participants evaluable for this outcome measure.

    Subject analysis set title
    Placebo
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    FAS: All randomized participants who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline TG measurement not including EOS visit. Here, ‘Overall number of participants analyzed’ = participants evaluable for this outcome measure. Main study and fibrate expansion cohorts were combined due to the low number of participants in fibrate expansion cohort. All dose groups for placebo groups were pooled.

    Subject analysis set title
    Pegozafermin 9 mg QW (Safety)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Pegozafermin 9 mg QW was administered as an SC injection. Participants were summarized by treatment received.

    Subject analysis set title
    Pegozafermin 18 mg QW (Safety)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Pegozafermin 18 mg QW was administered as an SC injection. Participants were summarized by treatment received.

    Subject analysis set title
    Pegozafermin 27 mg QW (Safety)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Pegozafermin 27 mg QW was administered as an SC injection. Participants were summarized by treatment received. The Main Study cohort and Fibrate Expansion cohort for pegozafermin 27 mg were pooled together due to low sample size in the expansion cohort.

    Subject analysis set title
    Pegozafermin 36 mg Q2W (Safety)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Pegozafermin 36 mg Q2W was administered as an SC injection. Participants were summarized by treatment received.

    Subject analysis set title
    Placebo (Safety)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Placebo was administered as an SC injection. Participants were summarized by treatment received. The Main Study cohort and Fibrate Expansion cohort for placebo were pooled together due to low sample size in the expansion cohort.

    Primary: Percent Change from Baseline to Week 8 in Serum TG

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    End point title
    		 Percent Change from Baseline to Week 8 in Serum TG
    End point description
    FAS: All randomized participants who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline TG measurement not including EOS visit. Here, ‘Overall number of participants analyzed’ = participants evaluable for this outcome measure. As pre-specified in the SAP, the sample size in each arm (by dose group) was insufficient (under powered) to effectively evaluate differences in efficacy by dose, therefore, efficacy analysis was performed using total pegozafermin and placebo groups.
    End point type
    Primary
    End point timeframe
    Baseline, Week 8
    End point values
    		 Pegozafermin Placebo
    Number of subjects analysed
    64
    17
    Units: percent change from Baseline
        median (inter-quartile range (Q1-Q3))
    -57.33 (-69.07 to -32.31)
    -11.85 (-23.22 to 7.88)
    Statistical analysis title
    		 Percent Change From Baseline to Week 8 in Serum TG
    Comparison groups
    Pegozafermin v Placebo
    Number of subjects included in analysis
    81
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [1]
    P-value
    		 < 0.001 [2]
    Method
    		 van Elteren test
    Parameter type
    		 Median Percent Change Difference
    Point estimate
    -43.73
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -57.08
         upper limit
    		 -30.31
    Notes
    [1] - Nonparametric analysis stratified by baseline TG level and background lipid therapy to test the treatment difference using pooled data. The location shift and Hodges-Lehmann 95% confidence interval were based on Hodges-Lehman estimation. Placebo group is the reference group, and the comparison was performed in pooled pegozafermin treatment group vs. placebo pooled.
    [2] - Significant level = 0.05

    Secondary: Percentage of Participants Who Achieved TG Level <500 mg/dL at Week 8

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    End point title
    		 Percentage of Participants Who Achieved TG Level <500 mg/dL at Week 8
    End point description
    FAS: All randomized participants who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline TG measurement not including EOS visit. Here, ‘Overall number of participants analyzed’ = participants evaluable for this outcome measure. As pre-specified in the SAP, the sample size in each arm (by dose group) was insufficient (under powered) to effectively evaluate differences in efficacy by dose, therefore, efficacy analysis was performed using total pegozafermin and placebo groups.
    End point type
    Secondary
    End point timeframe
    Week 8
    End point values
    		 Pegozafermin Placebo
    Number of subjects analysed
    64
    17
    Units: percentage
        number (not applicable)
    79.69
    29.41
    Statistical analysis title
    		 Achieved TG Level <500 mg/dL at Week 8
    Statistical analysis description
    Cochran-Mantel-Haenszel test stratified by baseline TG level and lipid modifying therapy use.
    Comparison groups
    Placebo v Pegozafermin
    Number of subjects included in analysis
    81
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.001 [3]
    Method
    		 Cochran-Mantel-Haenszel
    Confidence interval
    Notes
    [3] - Significant level = 0.05

    Secondary: Percent Change From Baseline to Week 8 in Non-high-density Lipoprotein Cholesterol (Non-HDL-C), Apolipoprotein B100 (ApoB), Low-density Lipoprotein Cholesterol (LDL-C), and High-density Lipoprotein Cholesterol (HDL-C)

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    End point title
    		 Percent Change From Baseline to Week 8 in Non-high-density Lipoprotein Cholesterol (Non-HDL-C), Apolipoprotein B100 (ApoB), Low-density Lipoprotein Cholesterol (LDL-C), and High-density Lipoprotein Cholesterol (HDL-C)
    End point description
    Least Squares Mean was calculated using Mixed model with repeated measure (MMRM). FAS: All randomized participants who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline TG measurement not including EOS visit. Here, ‘Overall number of participants analyzed’ = participants evaluable for this outcome measure. As pre-specified in the SAP, the sample size in each arm (by dose group) was insufficient (under powered) to effectively evaluate differences in efficacy by dose, therefore, efficacy analysis was performed using total pegozafermin and placebo groups.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 8
    End point values
    		 Pegozafermin Placebo
    Number of subjects analysed
    63
    17
    Units: percent change
    least squares mean (standard error)
        Non-HDL-C (n=63 and 17)
    -18.29 ( 2.943 )
    -0.57 ( 5.586 )
        ApoB (n=63 and 17)
    -10.51 ( 2.238 )
    1.07 ( 4.309 )
        LDL-C (n=62 and 15)
    10.44 ( 4.654 )
    8.72 ( 9.018 )
        HDL-C (n=63 and 17)
    24.65 ( 3.752 )
    9.67 ( 7.045 )
    Statistical analysis title
    		 Percent Change From Baseline to Week 8: non-HDL-C
    Statistical analysis description
    MMRM analysis of non-HDL-C comparison between pegozafermin pooled versus placebo pooled group.
    Comparison groups
    Pegozafermin v Placebo
    Number of subjects included in analysis
    80
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.007 [4]
    Method
    		 MMRM
    Parameter type
    		 Least Squares Means Difference
    Point estimate
    -17.87
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -30.67
         upper limit
    		 -5.07
    Variability estimate
    Standard error of the mean
    Dispersion value
    6.425
    Notes
    [4] - Significant level = 0.05
    Statistical analysis title
    		 Percent Change From Baseline to Week 8 in ApoB
    Statistical analysis description
    MMRM analysis of ApoB comparison between pegozafermin pooled versus placebo pooled group.
    Comparison groups
    Pegozafermin v Placebo
    Number of subjects included in analysis
    80
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.019 [5]
    Method
    		 MMRM
    Parameter type
    		 Least Squares Means Difference
    Point estimate
    -11.75
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -21.48
         upper limit
    		 -2.01
    Variability estimate
    Standard error of the mean
    Dispersion value
    4.888
    Notes
    [5] - Significant level of 0.05
    Statistical analysis title
    		 Percent Change From Baseline to Week 8 in LDL-C
    Statistical analysis description
    MMRM analysis of LDL-C comparison between pegozafermin pooled versus placebo pooled group.
    Comparison groups
    Pegozafermin v Placebo
    Number of subjects included in analysis
    80
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.87 [6]
    Method
    		 MMRM
    Parameter type
    		 Least Squares Means Difference
    Point estimate
    1.73
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -19.21
         upper limit
    		 22.68
    Variability estimate
    Standard error of the mean
    Dispersion value
    10.519
    Notes
    [6] - Significant level of 0.05
    Statistical analysis title
    		 Percent Change From Baseline to Week 8 in HDL-C
    Statistical analysis description
    MMRM analysis of HDL-C comparison between pegozafermin pooled versus placebo pooled group.
    Comparison groups
    Pegozafermin v Placebo
    Number of subjects included in analysis
    80
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.064 [7]
    Method
    		 MMRM
    Parameter type
    		 Least Squares Means Difference
    Point estimate
    15.41
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.89
         upper limit
    		 31.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    8.182
    Notes
    [7] - Significant level of 0.05

    Secondary: Percent Change From Baseline to Week 8 in Very Low-density Lipoprotein Cholesterol (VLDL-C) and VLDL-TG

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    End point title
    		 Percent Change From Baseline to Week 8 in Very Low-density Lipoprotein Cholesterol (VLDL-C) and VLDL-TG
    End point description
    FAS: All randomized participants who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline TG measurement not including EOS visit. Here, ‘Overall number of participants analyzed’ = participants evaluable for this outcome measure. As pre-specified in the SAP, the sample size in each arm (by dose group) was insufficient (under powered) to effectively evaluate differences in efficacy by dose, therefore, efficacy analysis was performed using total pegozafermin and placebo groups.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 8
    End point values
    		 Pegozafermin Placebo
    Number of subjects analysed
    62
    15
    Units: percent change
    median (inter-quartile range (Q1-Q3))
        VLDC-C (n=62 and 15)
    -47.96 (-63.14 to -16.67)
    -0.41 (-30.38 to 10.98)
        VLDL-TG (n= 61 and 15)
    -58.50 (-72.53 to -32.80)
    -0.85 (-27.72 to 14.25)
    Statistical analysis title
    		 Percent Change From Baseline to Week 8 in VLDL-TG
    Statistical analysis description
    Nonparametric analysis of VLDL-TG comparison between pegozafermin pooled versus placebo pooled group.
    Comparison groups
    Pegozafermin v Placebo
    Number of subjects included in analysis
    77
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.002 [8]
    Method
    		 van Elteren Test
    Confidence interval
    Notes
    [8] - Significant level of 0.05
    Statistical analysis title
    		 Percent Change From Baseline to Week 8 in VLDL-C
    Statistical analysis description
    Nonparametric analysis of VLDL-C comparison between pegozafermin pooled versus placebo pooled group.
    Comparison groups
    Pegozafermin v Placebo
    Number of subjects included in analysis
    77
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.006 [9]
    Method
    		 van Elteren Test
    Confidence interval
    Notes
    [9] - Significant level of 0.05

    Secondary: Percent Change in Baseline to Week 8 in Fasting Plasma Glucose, Adiponectin, and Body Weight

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    End point title
    		 Percent Change in Baseline to Week 8 in Fasting Plasma Glucose, Adiponectin, and Body Weight
    End point description
    Least Squares Mean was calculated using MMRM. FAS: All randomized participants who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline TG measurement not including EOS visit. Here, ‘Overall number of participants analyzed’ = participants evaluable for this outcome measure. As pre-specified in the SAP, the sample size in each arm (by dose group) was insufficient (under powered) to effectively evaluate differences in efficacy by dose, therefore, efficacy analysis was performed using total pegozafermin and placebo groups.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 8
    End point values
    		 Pegozafermin Placebo
    Number of subjects analysed
    63
    17
    Units: percent change
    least squares mean (standard error)
        Fasting Plasma Glucose
    -3.90 ( 2.681 )
    -2.67 ( 5.230 )
        Adiponectin
    69.53 ( 7.383 )
    5.70 ( 14.556 )
        Body Weight
    -0.15 ( 0.339 )
    -0.14 ( 0.654 )
    Statistical analysis title
    		 Percent Change in Fasting Plasma Glucose
    Statistical analysis description
    MMRM analysis of the percent change in Baseline to Week 8 in fasting plasma glucose comparison between pegozafermin pooled versus placebo pooled group.
    Comparison groups
    Pegozafermin v Placebo
    Number of subjects included in analysis
    80
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.809 [10]
    Method
    		 MMRM
    Confidence interval
    Notes
    [10] - Significant level = 0.05
    Statistical analysis title
    		 Percent Change in Adiponectin
    Statistical analysis description
    MMRM analysis of the percent change in Baseline to Week 8 in adiponectin comparison between pegozafermin pooled versus placebo pooled group.
    Comparison groups
    Pegozafermin v Placebo
    Number of subjects included in analysis
    80
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.001 [11]
    Method
    		 MMRM
    Confidence interval
    Notes
    [11] - Significant level = 0.05
    Statistical analysis title
    		 Percent Change in Body Weight
    Statistical analysis description
    MMRM analysis of the percent change in Baseline to Week 8 in body weight comparison between pegozafermin pooled versus placebo pooled group.
    Comparison groups
    Pegozafermin v Placebo
    Number of subjects included in analysis
    80
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.973 [12]
    Method
    		 MMRM
    Confidence interval
    Notes
    [12] - Significant level = 0.05

    Secondary: Percent Change From Baseline to Week 8 in High-sensitivity C-reactive Protein (hsCRP)

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    End point title
    		 Percent Change From Baseline to Week 8 in High-sensitivity C-reactive Protein (hsCRP)
    End point description
    FAS: All randomized participants who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline TG measurement not including EOS visit. Here, ‘Overall number of participants analyzed’ = participants evaluable for this outcome measure. As pre-specified in the SAP, the sample size in each arm (by dose group) was insufficient (under powered) to effectively evaluate differences in efficacy by dose, therefore, efficacy analysis was performed using total pegozafermin and placebo groups.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 8
    End point values
    		 Pegozafermin Placebo
    Number of subjects analysed
    63
    17
    Units: percent change
        median (inter-quartile range (Q1-Q3))
    -21.43 (-43.30 to 42.37)
    -1.10 (-50.41 to 18.84)
    No statistical analyses for this end point

    Secondary: Percent Change From Baseline to Week 8 in Liver Fat as Assessed by Magnetic Resonance Imaging - Whole Liver Proton Density Fat Fraction (MRI-PDFF)

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    End point title
    		 Percent Change From Baseline to Week 8 in Liver Fat as Assessed by Magnetic Resonance Imaging - Whole Liver Proton Density Fat Fraction (MRI-PDFF)
    End point description
    Least Squares Mean was calculated using analysis of covariance (ANCOVA). FAS: All randomized participants who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline TG measurement not including EOS visit. Here, ‘Overall number of participants analyzed’ = participants evaluable for this outcome measure. As pre-specified in the SAP, the sample size in each arm (by dose group) was insufficient (under powered) to effectively evaluate differences in efficacy by dose, therefore, efficacy analysis was performed using total pegozafermin and placebo groups.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 8
    End point values
    		 Pegozafermin Placebo
    Number of subjects analysed
    17
    6
    Units: percent change
        least squares mean (standard error)
    -42.18 ( 6.207 )
    -8.26 ( 11.214 )
    Statistical analysis title
    		 Percent Change From Baseline to Week 8: Liver Fat
    Comparison groups
    Pegozafermin v Placebo
    Number of subjects included in analysis
    23
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.012 [13]
    Method
    		 ANCOVA
    Confidence interval
    Notes
    [13] - Significant level = 0.05

    Secondary: Percent Change From Baseline to Week 8 in Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST)

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    End point title
    		 Percent Change From Baseline to Week 8 in Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST)
    End point description
    Least Squares Mean was calculated using MMRM. FAS: All randomized participants who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline TG measurement not including EOS visit. Here, ‘Overall number of participants analyzed’ = participants evaluable for this outcome measure. As pre-specified in the SAP, the sample size in each arm (by dose group) was insufficient (under powered) to effectively evaluate differences in efficacy by dose, therefore, efficacy analysis was performed using total pegozafermin and placebo groups.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 8
    End point values
    		 Pegozafermin Placebo
    Number of subjects analysed
    63
    17
    Units: percent change
    least squares mean (standard error)
        ALT
    -4.28 ( 4.549 )
    0.43 ( 8.881 )
        AST
    -11.50 ( 3.341 )
    0.51 ( 6.597 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 (after dosing) up to 12 weeks
    Adverse event reporting additional description
    As per SAP, participants in the pegozafermin 27 mg QW fibrate cohort were pooled with the pegozafermin 27 mg QW main cohort. Similarly, placebo groups in main and fibrate cohorts were pooled. Safety data analysis was by treatment group received. 4 participants randomized to receive 9 mg QW received 18 mg QW instead.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    Pegozafermin 9 mg QW (Safety)
    Reporting group description
    		 Pegozafermin 9 mg QW was administered as a SC injection. Participants were summarized by treatment received.

    Reporting group title
    Pegozafermin 18 mg QW (Safety)
    Reporting group description
    		 Pegozafermin 18 mg QW was administered as an SC injection. Participants were summarized by treatment received.

    Reporting group title
    Placebo (Safety)
    Reporting group description
    		 Matching placebo was injected at matching frequency per assigned cohort. Participants were summarized by treatment received. Main Study cohort and Fibrate Expansion cohort for placebo were pooled together due to low sample size in the expansion cohort.

    Reporting group title
    Pegozafermin 36 mg Q2W (Safety)
    Reporting group description
    		 Pegozafermin 36 mg Q2W was administered as an SC injection. Participants were summarized by treatment received.

    Reporting group title
    Pegozafermin 27 mg QW (Safety)
    Reporting group description
    		 Pegozafermin 27 mg QW was administered as an SC injection. The Main Study cohort and Fibrate Expansion cohort for pegozafermin 27 mg were pooled together due to low sample size in the expansion cohort.

    Serious adverse events
    		 Pegozafermin 9 mg QW (Safety) Pegozafermin 18 mg QW (Safety) Placebo (Safety) Pegozafermin 36 mg Q2W (Safety) Pegozafermin 27 mg QW (Safety)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Pegozafermin 9 mg QW (Safety) Pegozafermin 18 mg QW (Safety) Placebo (Safety) Pegozafermin 36 mg Q2W (Safety) Pegozafermin 27 mg QW (Safety)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 12 (58.33%)
    13 / 21 (61.90%)
    9 / 18 (50.00%)
    7 / 16 (43.75%)
    13 / 18 (72.22%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Lymphoedema
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    General disorders and administration site conditions
    Injection site erythema
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 21 (4.76%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    2 / 18 (11.11%)
         occurrences all number
    0
    2
    0
    3
    10
    Injection site reaction
         subjects affected / exposed
    1 / 12 (8.33%)
    2 / 21 (9.52%)
    0 / 18 (0.00%)
    2 / 16 (12.50%)
    1 / 18 (5.56%)
         occurrences all number
    1
    3
    0
    2
    2
    Injection site pruritus
         subjects affected / exposed
    1 / 12 (8.33%)
    2 / 21 (9.52%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    1
    3
    0
    0
    2
    Injection site pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    Injection site induration
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Injection site bruising
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    Fatigue
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    2 / 18 (11.11%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    2
    0
    1
    Pyrexia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Wheezing
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 21 (4.76%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    0
    0
    1
    Investigations
    Blood thyroid stimulating hormone increased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    C-reactive protein increased
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Injury, poisoning and procedural complications
    Skin laceration
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Foot fracture
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    0
    1
    Corneal abrasion
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Contusion
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Cardiac disorders
    Atrioventricular block first degree
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    0
    1
    Supraventricular extrasystoles
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Nervous system disorders
    Taste disorder
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Paraesthesia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Dysgeusia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Dizziness
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    0
    1
    Headache
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    2 / 18 (11.11%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    0
    1
    Iron deficiency anaemia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    0
    1
    Lymphadenitis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    0
    0
    Gastrointestinal disorders
    Inguinal hernia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Vomiting
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    0
    2
    Nausea
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 21 (4.76%)
    0 / 18 (0.00%)
    2 / 16 (12.50%)
    5 / 18 (27.78%)
         occurrences all number
    1
    1
    0
    2
    8
    Diverticulum
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Abdominal pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    2 / 18 (11.11%)
         occurrences all number
    0
    0
    0
    3
    3
    Diarrhoea
         subjects affected / exposed
    2 / 12 (16.67%)
    1 / 21 (4.76%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    4 / 18 (22.22%)
         occurrences all number
    2
    1
    1
    0
    4
    Frequent bowel movements
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 21 (4.76%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    0 / 18 (0.00%)
         occurrences all number
    0
    1
    0
    1
    0
    Hepatobiliary disorders
    Hepatic steatosis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Dermatitis contact
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Ecchymosis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Pigmentation disorder
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Endocrine disorders
    Hyperthyroidism
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Myalgia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    1
    0
    1
    Back pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    2 / 18 (11.11%)
         occurrences all number
    0
    0
    0
    0
    2
    Pain in extremity
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    0 / 12 (0.00%)
    3 / 21 (14.29%)
    3 / 18 (16.67%)
    1 / 16 (6.25%)
    0 / 18 (0.00%)
         occurrences all number
    0
    3
    3
    1
    0
    Respiratory tract infection viral
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    0
    1
    Otitis media
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 21 (4.76%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    0
    0
    1
    Gastroenteritis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    1
    1
    Sinusitis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    1 / 18 (5.56%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    Urinary tract infection
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    1
    0
    0
    0
    1
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    0 / 18 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    Metabolism and nutrition disorders
    Hyperkalaemia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    0
    1
    Hypoglycaemia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    1 / 16 (6.25%)
    0 / 18 (0.00%)
         occurrences all number
    0
    0
    0
    1
    0
    Increased appetite
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 21 (4.76%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    1
    0
    0
    1
    Hypomagnesaemia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 21 (0.00%)
    0 / 18 (0.00%)
    0 / 16 (0.00%)
    1 / 18 (5.56%)
         occurrences all number
    0
    0
    0
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    27 May 2020
    - Specific laboratory assessments were added or amended. - Updated eligibility criteria - Added stopping role of “External circumstances that do not enable the study to be properly conducted under the existing protocol. - Clarified that only central labs and protocol defined assessments may be used for screening and at baseline.
    23 Dec 2020
    - Updated study population - Expanded description of re-screening - Updated secondary endpoints - Clarified clinical laboratory assessments - Risk assessment section updated based on currently available data - Updated eligibility criteria
    03 May 2021
    - Updated clinical laboratory assessments - Updated eligibility criteria

    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.
    The study did not separately evaluate the response to pegozafermin in participants who were on and who were not on concurrent fibrate therapy due to small sample size in the Fibrate Expansion cohort.

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/37355760
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