Kezar Life Sciences, Inc.

Plus, news about Kezar Life Sciences and Concentra Biosciences: Viatris, Lexicon Pharmaceuticals sign licensing deal for cardio drug: Per the agreement , Viatris will pay $25 million upfront to Lexicon in exchange for the rights to sotagliflozin in all markets outside of the US and Europe. Lexicon may also receive regulatory- and sales-based milestones and tiered royalties. The FDA last year approved the drug to reduce the risk of cardiovascular death, hospitalization for heart failure, and urgent heart failure. — Katherine Lewin Capricor Therapeutics prices $75M public stock offering: The rare disease biotech is selling 4.4 million of its shares at $17.00 apiece. The funds are set to go toward the development and manufacturing of its investigational drugs, as well as “working capital and general corporate purposes.” Capricor’s lead candidate, deramiocel, is a cell therapy currently in Phase 3 development for the treatment of Duchenne muscular dystrophy. Earlier this month, Capricor announced positive longer-term data from the ongoing HOPE-2 trial and that it had filed for approval with the FDA. — Katherine Lewin Kezar Life Sciences rejects Concentra’s unsolicited offer: The Bay Area biotech’s board said “no” to Kevin Tang’s Concentra Biosciences, which had offered $1.10 per share $KZR and 80% of potential asset sale proceeds to buy the company. Concentra made the offer last week after Kezar stopped a Phase 2b in lupus nephritis. On Thursday, Kezar also reiterated that it expects to have Phase 2a data in autoimmune hepatitis in the first half of next year. Kezar’s stock was down about 10% on Thursday morning. — Kyle LaHucik

PORTOLA Phase 2a clinical trial in patients with autoimmune hepatitis (AIH) to continue without modification following review by Independent Data Monitoring Committee (IDMC); reiterating guidance of topline data in first half 2025 PALIZADE Phase 2b clinical trial in patients with active lupus nephritis (LN) will be discontinued; focusing resources on clinical development of zetomipzomib in AIH SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company developing novel small molecule therapeutics to treat unmet needs in immune-mediated diseases, today announced key clinical development updates. The IDMC overseeing the PORTOLA Phase 2a clinical trial of zetomipzomib in patients with AIH recommended that the trial may proceed without modification following its third scheduled meeting. The IDMC examined safety data from all patients enrolled in the trial, including data from patients who completed the blinded treatment period and continued to the open-label extension portion of the trial which includes an additional 24 weeks of treatment. Three members of the IDMC, including the committee chair, are also members of the IDMC overseeing the PALIZADE study of zetomipzomib in LN. To date, no Grade 4 or 5 serious adverse events (SAEs) have been observed in the PORTOLA trial which is being conducted at clinical sites in the United States. Kezar has completed enrollment of PORTOLA and plans to report topline data in the first half of 2025. Kezar has also made the strategic decision to terminate the PALIZADE Phase 2b clinical trial in patients with active LN and focus clinical development efforts of zetomipzomib in AIH, a rare disease with a significant unmet medical need. PALIZADE was recently placed on clinical hold following the recommendation of the IDMC after its assessment of four Grade 5 (fatal) SAEs that occurred in patients enrolled in the Philippines and Argentina (including one patient on placebo). Kezar will unblind the trial and perform a full investigation into all safety events from the study. As of termination, 84 patients were enrolled, and Kezar will report available data from PALIZADE at a later date. “The IDMC recommendation to continue the PORTOLA trial without modification strengthens our confidence in the potential for zetomipzomib to be a meaningful treatment for patients living with autoimmune hepatitis,” said Chris Kirk, PhD, Kezar’s Chief Executive Officer. “While we are disappointed to discontinue our development program in LN, we would like to thank the investigators, patients and their families for their participation in the PALIZADE trial. This decision was difficult given the favorable safety pro clinical activity data we have presented from the MISSION study and rapid enrollment to PALIZADE. However, a focused development effort in AIH extends our cash runway and provides flexibility as we work to bring zetomipzomib forward as a treatment for patients living with this life-threatening disease.” Kezar’s unaudited cash position is approximately $148 million, including cash, cash equivalents and marketable securities, as of September 30, 2024. This cash estimate is a preliminary estimate and based on information currently available to management, and this estimate could change. About PORTOLA PORTOLA is a placebo-controlled, randomized, double-blind Phase 2a clinical trial evaluating the efficacy and safety of zetomipzomib in patients with AIH that are insufficiently responding to standard of care or have relapsed. The study has completed enrollment of 24 patients, randomized (2:1) to receive 60 mg of zetomipzomib or placebo in addition to background therapy for 24 weeks, with a protocol-suggested steroid taper. The primary efficacy endpoint will measure the proportion of patients who achieve a complete biochemical response by Week 24 measured as normalization of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and Immunoglobulin G (IgG) values (if elevated at baseline), with steroid dose levels not higher than baseline. About Autoimmune Hepatitis Autoimmune hepatitis (AIH) is a rare chronic disease in which the immune system attacks the liver and causes inflammation and tissue damage, severely impacting patients’ physical health and quality of life. Lifelong maintenance therapy is required to avoid relapse and burdensome adverse effects. If left untreated, AIH can lead to cirrhosis, liver failure and hepatocellular carcinoma. In the United States, AIH affects approximately 140,000 individuals, with incidence rates increasing. The cause of this condition remains unclear, with females affected four times as often as males. Currently, standard of care treatment for AIH is chronic, immunosuppressive treatment with corticosteroids that frequently cause life-altering side effects, including diabetes, osteoporotic fractures and cataracts. There is a significant need for treatment regimens that reduce or remove the need for chronic immunosuppression from using corticosteroids. About Kezar Life Sciences Kezar Life Sciences is a clinical-stage biopharmaceutical company developing novel small molecule therapeutics to treat unmet needs in immune-mediated diseases. For more information, visit , and follow us on LinkedIn , Facebook , Twitter and Instagram . Cautionary Note on 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,” “can,” “should,” “expect,” “believe,” “plans,” “potential,” “anticipate” and similar expressions (as well as other words or expressions referencing 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 results or performance to differ materially from those expressed or implied by the forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the nature, frequency and severity of adverse events; the design, progress and outcome of Kezar’s clinical trials; Kezar’s ability to report data from its clinical trials on expected timelines, if at all; and Kezar’s ability to extend its cash runway. Many factors may cause differences between current expectations and actual results, including those factors that are discussed in Kezar’s filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” contained therein. Except as required by law, Kezar assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. Contacts Investor and Media Contact: Gitanjali Jain Senior Vice President, Investor Relations and External Affairs Kezar Life Sciences, Inc. gjain@kezarbio.com

Earlier this month, Kezar Life Sciences announced that the mid-stage test of zetomipzomib in lupus nephritis had been placed on an FDA clinical hold. Now, that program is being terminated. Kezar Life Sciences is discontinuing a lupus program after four patient deaths occurred during a Phase IIb trial and will focus on developing the same drug in autoimmune hepatitis. Earlier this month, Kezar announced that the mid-stage study of zetomipzomib in lupus nephritis had been placed on an FDA clinical hold. The regulatory action came after the company reported four patient deaths in the Phase IIb PALIZADE trial. Other serious adverse events were reported as well that “showed a similar proximity to dosing.” Kezar voluntarily suspended enrollment and began looking at mitigation strategies for the drug. Now, an independent data monitoring committee that had been examining the PALIZADE trial and the Phase IIa PORTOLA trial of zetomipzomib in hepatitis has recommended that the lupus program be suspended. Kezar will proceed with the hepatitis study and expects a readout in the first half of 2025, according to a Thursday press release. Kezar noted that one of the deaths that occurred in the PALIZADE study was in a patient who had received a placebo. The trial was conducted in Argentina and the Philippines. The trial will be unblinded and a full investigation will be conducted into the safety events, the company said. At the time the study was terminated, 84 patients were enrolled. Kezar promised to release available data at a later time. CEO Chris Kirk said Kezar’s decision was a difficult one, as the company had seen a favorable safety pro clinical activity in an earlier trial called MISSION. The PALIZADE trial also enrolled very quickly. “However, a focused development effort in AIH [autoimmune hepatitis] extends our cash runway and provides flexibility as we work to bring zetomipzomib forward as a treatment for patients living with this life-threatening disease,” Kirk said. The data committee recommended that the PORTOLA trial proceed without modification after examining all safety data from the patients enrolled in the trial. No serious adverse events rated grade 4 or higher, which would indicate a death, have occurred in the PORTOLA trial which is being conducted in the U.S. In other news, Kezar has rejected an unsolicited bid from Concentra Biosciences to acquire the biotech. The offer had been to buy all outstanding shares for $1.10 apiece, plus a contingent value right. Kezar has decided that the value significantly undervalues the company and zetomipzomib. Concentra, meanwhile, has moved to acquire 9.9% of Kezar’s common stock. Kezar has adopted a stockholder rights plan, also known as a “poison pill” defense, to protect against any party buying a controlling stake through the open market without paying stockholders their due. The rights plan does not block a more traditional buyout, even from Concentra, if the board deems it in the best interests of shareholders, Kezar noted. Kezar’s shares were trading at 80 cents on the Nasdaq Capital Market Thursday morning, down more than 9% from the prior close. Concentra is owned by Tang Capital, a life sciences-focused investment firm. The company has made many attempts over the years to buy out struggling biotechs, most recently succeeding in acquiring Theseus Pharmaceuticals. Similar buyouts offered to LianBio, Rain Oncology and Atea Pharmaceuticals were unsuccessful.