Kezar Announces Top Results from PRESIDIO Trial of Zetomipzomib for the Treatment of Dermatomyositis and Polymyositis | national company

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–May 3, 2022–

Kezar Life Sciences, Inc.., (Nasdaq: KZR ), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncology disorders, today announced top results from the PRESIDIO Phase 2 clinical trial of zetomipzomib (KZR-616) in patients with dermatomyositis (DM) and polymyositis (PM).

In PRESIDIO, 25 patients with DM (n=13) or PM (n=12) with active disease despite the best available treatments, and 20 patients completed treatment until the end of treatment. During the 32-week study period, all patients received 16 weeks of treatment with zetomipzomib: patients received either 45 mg zetomipzomib or placebo subcutaneously (SC) once weekly for 16 weeks on top of standard treatment, followed by a switch to the other arm of placebo or zetomipzomib, respectively, for an additional 16 weeks. Patients continued their background therapy, but were able to taper medications as clinically indicated. The primary endpoint of PRESIDIO was the mean change in the total improvement score (TIS).

Early results from the PRESIDIO trial showed that most DM and PM patients saw clinically meaningful improvements in TIS, but zetomipzomib demonstrated no significant differentiation from placebo. At week 16, the group receiving zetomipzomib 45 mg s.c. achieved an average TIS of 25.5 compared to an average TIS of 25 for the control group. the control group has an average TIS of 31.3.

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Zetomipzomib was well tolerated in the PRESIDIO trial. Adverse events were generally mild to moderate (grade 1 or 2) and the most common treatment-emergent adverse events (TEAEs) were injection site reactions, which were transient and manageable. One subject withdrew from the study due to an injection site reaction at week 9. There were three Grade 3 serious adverse events in the zetomipzomib arms, all considered unrelated to zetomipzomib, which occurred occurred in two patients and did not lead to study discontinuation or dose change. One patient had a mechanical fall and syncope, and another patient had retinal detachment. There was one grade 3 adverse event in a placebo arm identified as rash worsening. No opportunistic infections or cytopenias were observed. Safety data is summarized in the table below.

Safety and tolerability of zetomipzomib for the 32-week PRESIDIO trial

Patients with Adverse Events: N (%)

Zetomipzomib

N=25

Placebo

N=22*

Any adverse event

22 (88.0)

16 (72.7)

At least 1 TEAE

22 (88.0)

16 (72.7)

Nausea

3 (12.0)

3 (13.6)

Vomiting

1 (4.0)

1 (4.5)

Most common TEAEs

Injection site reaction

18 (72.0)

8 (36.4)

TEAE = Grade 3 (all unrelated)

2 (8.0)

1 (4.5)

Infectious TEAE ≥ Grade 3

0

0

Infectious TEAE, all grades

7 (28.0)

6 (27.3)

Opportunistic infections

0

0

Serious TEAE (all unrelated)

2 (8.0)

0

*Three patients withdrew in Period 1 before receiving placebo in Period 2

Open-label extension study

KZR-616-003E is an open-label extension study (OLE) available for patients who have completed 32 weeks in the PRESIDIO trial. After completion of PRESIDIO, 18 out of 20 patients enrolled in OLE. For the first time, patients have the ability to self-administer zetomipzomib in the OLE. Current active participation in OLE ranges from 2 to 77 weeks. 6 patients discontinued their participation in OLE for reasons unrelated to zetomipzomib. No additional safety or tolerability issues were observed, and mean TIS scores improved from scores observed at the end of 32 weeks of PRESIDIO.

“I would like to thank all of the investigators, site staff, Kezar employees and especially the patients for their commitment to this study in the face of the headwinds associated with the pandemic. Although we are disappointed with the results of this trial, we are encouraged by the favorable safety data and we maintain our strong belief in the promise of zetomipzomib in lupus nephritis and our commitment to the development of this new agent in autoimmune diseases. immune,” said John Fowler. , CEO. “The high response rates we have seen to date in MISSION – using objective endpoints in LN patients – inform this belief, and we look forward to sharing key results from this trial in June. Additionally, our strong financial position provides a broad avenue for our zetomipzomib and protein secretion programs, including our Phase 1 trial of KZR-261 in solid tumors. »

Kezar’s unaudited cash position is approximately $253 million, including cash, cash equivalents and marketable securities as of April 30, 2022. The company plans to release MISSION Phase 2 data on the lupus nephritis (LN) at a Kezar corporate event in June 2022.

About zetomipzomib (KZR-616)

Zetomipzomib (KZR-616) is a novel, first-in-class selective immunoproteasome inhibitor with broad therapeutic potential in multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition elicits a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1 clinical trials provide evidence that zetomipzomib has a favorable safety and tolerability profile for development in severe chronic autoimmune diseases.

In addition to PRESIDIO, Kezar is conducting the Phase 2 MISSION trial in patients with lupus nephritis. Interim data from this trial reported in November 2021 showed that of the five patients who completed the full 24 weeks of weekly treatment with 60 mg doses of zetomipzomib, two achieved partial renal responses (PRRs) and two achieved complete renal responses (CRR).

About dermatomyositis and polymyositis

Dermatomyositis and polymyositis are two of five types of autoimmune myositis. Both are chronic, debilitating, inflammatory autoimmune myopathies characterized by inflammation of the muscles as well as the skin (in DM). Approximately 30,000 to 120,000 people in the United States live with these severe and progressive inflammatory myopathies which are characterized by marked morbidity and associated mortality. While debilitating muscle weakness is the hallmark of these myopathies, including weakened respiratory muscles, other internal organ system dysfunctions can be just as debilitating. The goal of treatment for these diseases is to suppress inflammation, increase muscle strength, and prevent long-term damage to muscles and extramuscular organs; however, treatment options are limited for DM and there are currently no approved treatments for MP.

About Kezar Life Sciences

Kezar Life Sciences is a clinical-stage biopharmaceutical company discovering and developing breakthrough treatments for immune-mediated and oncology disorders. The company is a pioneer in the field of small molecule therapies that harness master regulators of cellular function to inhibit multiple disease factors through single and potent targets. Zetomipzomib, its lead development asset, is a selective immunoproteasome inhibitor being evaluated in phase 2 clinical trials in lupus nephritis, dermatomyositis and polymyositis. This product candidate also has the potential to treat multiple immune-mediated chronic diseases. KZR-261 is the company’s first clinical cancer candidate targeting the Sec61 translocon and protein secretory pathway. A Phase 1, open-label, dose-escalation clinical trial of KZR-261 to assess safety, tolerability, and preliminary tumor activity in solid tumors is ongoing. For more information, visit www.kezarlifesciences.com.

Caution Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “may”, “will”, “should”, “expect”, “believe”, “promise” , “potential”, “anticipate”, “plan” and similar expressions (as well as other words or expressions referring to future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Kezar’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause Kezar’s future clinical development programs, results or performance to differ materially from those expressed or implied by the forward-looking statements. Forward-looking statements in this press release include, but are not limited to, statements regarding the design, progress, timing, scope and results of clinical trials, Kezar’s capital adequacy and other resources, the anticipated clinical and regulatory development of Kezar product candidates, the anticipated nonproprietary name approval of KZR-616, the preliminary nature of interim and primary data, the likelihood that the data will support future development and therapeutic potential, the association of the data with treatment outcomes and the likelihood of obtaining regulatory approval for Kezar’s product candidates. Many factors could cause current expectations to differ from actual results, including the availability of additional data, confirmation of interim and primary data resulting from audit and trial verification procedures, unexpected safety data or efficacy observed during clinical studies, impacts of the COVID-19 19 pandemic and other global events on the Company’s business and clinical trials, changes in planned or existing competition, changes in the regulatory environment, uncertainties and timing of the regulatory approval process; and unexpected litigation or other disputes. Other factors that could cause actual results to differ from those expressed or implied by the forward-looking statements in this press release are discussed in Kezar’s filings with the United States Securities and Exchange Commission, including “risk factors” contained therein. Except as required by law, Kezar undertakes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even when new information becomes available.

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KEYWORD: CALIFORNIA UNITED STATES NORTH AMERICA

INDUSTRY KEYWORD: ONCOLOGY HEALTH CLINICAL TRIALS RESEARCH SCIENCE PHARMACEUTICALS BIOTECHNOLOGY

SOURCE: Kezar Life Sciences, Inc.

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PUBLISHED: 03/05/2022 16:01 / DISK: 03/05/2022 16:02

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Elna M. Lemons