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KZR-616

Phase 2

Polymyositis | Small molecule | Immunology |Kezar Life Sciences, Inc.|Last Updated: Nov 19, 2025

Success Probability

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials2
Total Enrollment43

FDA Designations

No designations recorded

Clinical trial landscape

KZR-616 · 3 trials · 4 indications

Phase 2 2Phase 1 1
NCT04628936Open-label Extension to the Phase 2 Crossover Study (PRESIDIO) Evaluating KZR-616 in Patients With PM and DM.Polymyositis
COMPLETED18 Analytics
NCT04033926A Phase 2 Study of KZR-616 to Evaluate Safety and Efficacy in Patients With Active Polymyositis or DermatomyositisPolymyositis
COMPLETED25 Analytics
PHASE2COMPLETED
Open-label Extension to the Phase 2 Crossover Study (PRESIDIO) Evaluating KZR-616 in Patients With PM and DM.
PolymyositisUnlock trial analytics
PHASE2COMPLETED
A Phase 2 Study of KZR-616 to Evaluate Safety and Efficacy in Patients With Active Polymyositis or Dermatomyositis
PolymyositisUnlock trial analytics

Study Endpoints

Primary Endpoints

Mean Total Improvement Score (TIS) at OLE Week 48
48 weeks

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.

Mean Change in the Total Improvement Score (TIS) From Start to End of Zetomipzomib (KZR-616) Treatment Period
16 weeks in each Treatment Period (32 weeks total)

The primary efficacy endpoint was mean change from start to end of zetomipzomib (KZR-616) Treatment Periods in the Total Improvement Score (TIS), which ranges from 0 to 100 \[low of 0 to high of 100, where higher scores are better\]. Mean change in TIS was calculated by comparing the Baseline and post Baseline observations for patients in both KZR-616 treatment periods combined. Note: TIS scores for placebo treatment periods are presented in this outcome measure but were not included in the primary outcome measure analysis.

Phase 1b: Number of Patients Who Experienced at Least One Treatment-Related Treatment-Emergent Adverse Event
25 weeks

The safety and tolerability of zetomipzomib (KZR-616) when administered as a subcutaneous injection weekly for 13 weeks in adult patients with systemic lupus erythematous (SLE) with and without nephritis, as assessed by number of patients who experienced at least one treatment-related treatment-emergent adverse event. For additional information about the safety and tolerability of KZR-616, please reference the adverse events section of this posting.

Phase 2: Number of Patients With Lupus Nephritis With a 50% Reduction in UPCR
24 weeks

To assess the number of patients with lupus nephritis with a 50% reduction in UPCR after 24 weeks of weekly SC injections with KZR-616 when compared to baseline.

Secondary Endpoints

Proportion of Patients With TIS Response
16 weeks in each Treatment Period (32 weeks total)
Number of Patients Meeting the International Myositis Assessment and Clinical Studies Group (IMACS) Definition of Improvement (DOI)
16 weeks in each Treatment Period (32 weeks total)
Mean Percent Change From Baseline From Start to End of Treatment in the IMACS Individual CSAMs
16 weeks in each Treatment Period (32 weeks total)
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
KZR-616 45 mg + standard therapy (open-label)EXPERIMENTALAll 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.
Arm AOTHER* Treatment Period 1: KZR-616 30 mg SC weekly for 2 weeks, then 45 mg SC weekly for 14 weeks * Treatment Period 2: Placebo SC weekly for 16 weeks
Arm BOTHER* Treatment Period 1: Placebo SC weekly for 16 weeks * Treatment Period 2: KZR-616 30 mg SC weekly for 2 weeks, then 45 mg SC weekly for 14 weeks
KZR-616 45 mg + standard of care therapy (Phase 1b)EXPERIMENTALDose escalation cohort of patients with SLE with and without nephritis to receive 45 mg dose level of KZR-616 in combination with standard of care therapy. Two Phase 1b cohorts received 45 mg at some point during the study. Cohort 1 received 45 mg zetomipzomib frozen maleate weekly for 13 weeks. Cohort 2a followed a step-up dosing procedure. Patients received zetomipzomib frozen maleate, 30 mg weekly for 2 weeks, followed by 45 mg weekly for 2 weeks then followed by 60 mg weekly for 9 weeks. KZR-616 was administered as a SC injection.
KZR-616 60 mg + standard of care therapy (Phase 1b)EXPERIMENTALDose escalation cohort of patients with SLE with and without nephritis to receive 60 mg dose level of KZR-616 in combination with standard of care therapy. Four Phase 1b cohorts received 60 mg at some point during the study. Cohort 2 received 60 mg zetomipzomib frozen maleate weekly for 13 weeks. Cohorts 2a, 2b, and 2c all followed a step-up dosing procedure. Patients in Cohort 2a received zetomipzomib frozen maleate, 30 mg weekly for 2 weeks, followed by 45 mg weekly for 2 weeks then followed by 60 mg weekly for 9 weeks. Patients in Cohort 2b received zetomipzomib lyophile, 30 mg weekly for 1 week, followed by 60 mg weekly for 12 weeks. Patients in Cohort 2c (tolerability strategies cohort) received zetomipzomib lyophile, 30 mg weekly for 1 week, followed by 60 mg weekly for 12 weeks. KZR-616 was administered as a SC injection.
KZR-616 75 mg + standard of care therapy (Phase 1b)EXPERIMENTALDose escalation cohort of patients with SLE with and without nephritis to receive 75 mg dose level of KZR-616 in combination with standard of care therapy. One Phase 1b cohort received 75 mg at some point during the study. Cohort 3 followed a step-up dosing procedure. Patients in Cohort 3 received zetomipzomib lyophile, 30 mg weekly for 1 week, followed by 75 mg weekly for 12 weeks. KZR-616 was administered as a SC injection.
KZR-616 60 mg + standard therapy (Phase 2)EXPERIMENTAL60 mg dose level of KZR-616 selected based on data from the phase 1b dose escalation and administered to patients with active lupus nephritis in combination with standard therapy including at least one immunosuppressive agent. KZR-616 was administered as a SC injection weekly at a dose of 60 mg for 24 weeks (including a step-up from an initial Week 1 dose of 30 mg). \*\* See Limitations/Caveats for additional information

Interventions

NameTypeDescription
KZR-616DRUGSubcutaneous 30 mg for 1 week, then 45 mg weekly
PlaceboDRUGSubcutaneous injection for 16 weeks
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: * Must have successfully completed Study KZR-616-003 through Week 32, including the Week 32 Visit assessments * Women of childbearing potential (WOCBP) must have a negative urine or serum pregnancy test prior to the first dose of KZR-616 in KZR-616-003E, and must agree to contin...

Countries:United StatesCzechiaGermanyAustraliaColombiaMexicoPeruPolandRussiaUkraine
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Frequently asked questions about KZR-616

What is KZR-616 used for?

KZR-616 is an investigational small molecule being developed for lupus nephritis and polymyositis. It is also being studied in dermatomyositis and systemic lupus erythematosus. The drug is in clinical development and has not been approved by the FDA.

Who makes KZR-616?

KZR-616 is developed by Kezar Life Sciences, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker KZR. The company is conducting clinical trials to evaluate the drug's safety and efficacy in autoimmune conditions.

What phase is KZR-616 in?

KZR-616 has completed Phase 1 and Phase 2 clinical trials. The Phase 1 study evaluated the drug in patients with systemic lupus erythematosus with and without lupus nephritis, while Phase 2 studies assessed it in polymyositis and dermatomyositis. It remains investigational.

What clinical trials is KZR-616 in?

KZR-616 has been studied in three completed trials: NCT03393013, a Phase 1 study in lupus nephritis and systemic lupus erythematosus; NCT04033926, a Phase 2 study in polymyositis and dermatomyositis; and NCT04628936, an open-label extension of the Phase 2 study.

Is KZR-616 the same as any other drug?

KZR-616 is the primary name for this investigational drug. No alternative names have been reported in the clinical trial data. It is a small molecule being developed by Kezar Life Sciences for autoimmune indications.