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zetomipzomib

Phase 2

Autoimmune Hepatitis | Small molecule | Immunology |Kezar Life Sciences, Inc.|Last Updated: Jan 13, 2026

Success Probability

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment24

FDA Designations

No designations recorded

Clinical trial landscape

zetomipzomib · 1 trial · 1 indication

Phase 2 1
NCT05569759A Study of Zetomipzomib (KZR-616) in Patients With Autoimmune Hepatitis (PORTOLA)Autoimmune Hepatitis
COMPLETED24 Analytics
PHASE2COMPLETED
A Study of Zetomipzomib (KZR-616) in Patients With Autoimmune Hepatitis (PORTOLA)
Autoimmune HepatitisUnlock trial analytics

Study Endpoints

Primary Endpoints

Patients Who Achieved Complete Biochemical Response
Week 12, Week 16, Week 20, and Week 24

The number of patients who achieve complete biochemical response (CR), defined as normal ALT, AST, and IgG values (if IgG level is elevated at Baseline) with glucocorticoid dose not higher than starting dose (at Baseline), by Week 24 of the Double-Blind Treatment Period. Analyses were also conducted at Week 12, Week 16, and Week 20.

The Safety and Tolerability of Zetomipzomib
Baseline through end of study visit (DBTP, Week 28 and OLE, Up to Week 24)

Proportion of participants who experience AEs (adverse events) and SAEs (serious adverse events) during the double-blind treatment period (DBTP) and the open-label extension (OLE).

Proportion of Participants Experiencing a Disease Flare Among the Participants Who Achieved a CR During the Double-blind Treatment Period
Start of open-label extension (OLE) period through End of Study (EOS) up to OLE Week 25

Proportion of participants experiencing a disease flare among the participants who achieved a complete biochemical response (CR) during the double-blind treatment period.

Secondary Endpoints

Alanine Aminotransferase (ALT)
Weeks 12, 16, 20, and 24
Partial Response
Weeks 12, 16, 20, and 24
Time to Complete Response
Baseline through Week 24
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
zetomipzomib + standard-of-care (glucocorticoids)EXPERIMENTALInitial 30 mg dose of zetomipzomib, followed by weekly 60 mg doses of zetomipzomib, for the remaining 23 weeks of the treatment period.
placebo + standard-of-care (glucocorticoids)PLACEBO_COMPARATORInitial 30 mg dose of placebo (sterile water for injection), followed by weekly 60 mg doses of placebo, for the remaining 23 weeks of the treatment period.
zetomipzomib + standard-of care (glucocorticoids) open-label extension periodEXPERIMENTALInitial 30 mg dose of zetomipzomib at the open-label extension (OLE) Week 1 visit, followed by weekly doses of 60 mg of zetomipzomib, for up to a total of 24 additional weeks of treatment.

Interventions

NameTypeDescription
zetomipzomibDRUGSubcutaneous injection of zetomipzomib with a target dose of 60 mg weekly
placeboDRUGSubcutaneous injection of placebo
zetomipzomib in open-label extensionDRUGSubcutaneous injection of zetomipzomib with a target dose of 60 mg weekly
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites24

Key Inclusion Criteria for the Double-blind Treatment Period: * Must be aged ≥18 years. * Must have a clinical diagnosis of AIH and signs of active disease despite standard-of-care therapy for ≥3 months or disease flare after experiencing complete remission induced by standard-of-care treatment,...

Countries:United States
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Frequently asked questions about zetomipzomib

What is zetomipzomib used for?

Zetomipzomib is an investigational small molecule being studied for the treatment of autoimmune hepatitis, a condition where the immune system attacks the liver. It is being developed by Kezar Life Sciences, Inc. (NASDAQ: KZR) and is currently in Phase 2 clinical development.

What does zetomipzomib target?

Zetomipzomib is a small molecule that targets the immunoproteasome, a protein complex involved in antigen processing and immune regulation. By inhibiting this complex, it aims to modulate the immune response in autoimmune hepatitis. This mechanism is under investigation in clinical trials.

Who makes zetomipzomib?

Zetomipzomib is being developed by Kezar Life Sciences, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol KZR. The company is conducting clinical research to evaluate the drug's safety and efficacy in patients with autoimmune hepatitis.

What phase is zetomipzomib in?

Zetomipzomib is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. A Phase 2 study has been completed to assess its effects in patients with autoimmune hepatitis.

What clinical trials is zetomipzomib in?

Zetomipzomib has been studied in a Phase 2 clinical trial with the identifier NCT05569759, titled 'A Study of Zetomipzomib (KZR-616) in Patients With Autoimmune Hepatitis (PORTOLA)'. This trial enrolled 24 participants in the United States and has been completed.

Is zetomipzomib the same as KZR-616?

Yes, zetomipzomib is also known as KZR-616. The clinical trial NCT05569759 refers to the drug as both zetomipzomib and KZR-616, confirming that these names refer to the same investigational compound being developed for autoimmune hepatitis.