KZR-616-003E

Kezar Life Sciences, Inc.

4000 Shoreline Court, Suite 300, South San Francisco, United States, 94080

Regulatory Affairs, Kezar Life Sciences, Inc., 001 6508225600, PRESIDIO@kezarbio.com

Clinical Science, Kezar Life Sciences, Inc., 001 6508225600, PRESIDIO@kezarbio.com

No

No

No

Final

27 Jul 2023

Yes

10 Mar 2023

Yes

12 Jun 2023

No

Efficacy: To evaluate the long-term efficacy of zetomipzomib (KZR-616) in patients with PM or DM. Safety: To evaluate the long-term safety and tolerability of zetomipzomib (KZR-616) in patients with PM or DM.

Investigators and all parties involved in this study conducted the study in adherence to the ethical principles based on the Declaration of Helsinki, ICH guidelines for cGCP, and the applicable laws and regulatory requirements. IRB/IEC approval of the study and relevant study information (e.g. protocol, informed consent form (ICF), patient-facing materials) was obtained before initiation of study sites or releasing study drug to sites. Extensions/renewals of the approval were obtained as necessary. Written informed consent (signed and dated) was obtained before any study-related procedures were performed. Patients were given every opportunity to ask for clarification and were given ample time to consider the study. Patients may refuse to enter the study or to withdraw from the study at any time, without consequences for their further care or penalty or loss of benefits to which the patient is otherwise entitled. All Investigators promptly reported any new information that may have adversely affected patient safety or the study conduct and submitted study status summaries to the IRB/IEC as required. Patients were informed about new information available that was relevant to their willingness to continue participation in the study and were reconsented to the IRB/IEC/regulatory authorities currently approved ICF. Patients’ identity remained confidential in any presentations or publications of the study results. All personal data collected and processed for the purposes of this study were managed with adequate precautions to ensure confidentiality of data, and in accordance with the applicable laws and regulations on personal data protection. A study-specific Data Monitoring Committee met to review accumulating safety data, study conduct and progress and to make recommendations about the study progress on a regular basis. Each voting member provided their recommendation at the conclusion of each meeting.

04 Nov 2020

No

No

United States: 17

Czechia: 1

18

1

0

0

0

0

0

0

15

3

0

Open Label Extension (overall period)

Not applicable

zetomipzomib

KZR-616

Powder for solution for injection

Subcutaneous use

All patients received a SC injection of 30 mg KZR-616 at Visit 1 (Day 1), followed by weekly SC injections of 45 mg KZR-616 up to a maximum of 96 weeks. Study drug administration ended for all patients in Study KZR-616-003E when the last patient enrolled completed 48 weeks of dosing.

Open Label Extension

KZR-616 45 mg + Standard Therapy (Open-label)

KZR-616 45 mg + Standard Therapy (Open-label)

All patients received SC injections of KZR-616 QW at doses of 30 mg on day 1 and 45 mg thereafter for up to 96 weeks. Study drug administration ended when the last enrolled patient completed 48 weeks of dosing in the OLE. Patients had an end of study visit 12 weeks after the last dose of KZR-616. KZR-616: zetomipzomib subcutaneous injection

The mean Total Improvement Score (TIS) at OLE Week 48, which ranges from 0 to 100 [low of 0 to high of 100, where higher scores are better]. The timeframe of 48 weeks was selected because it represented the maximum timeframe of dosing for the last patient enrolled as the study drug administration ended when the last patient enrolled completed 48 weeks of dosing.

Primary

48 weeks

Up to 108 weeks

25.1

KZR-616 45 mg + Standard Therapy (Open-label)