Zetomipzomib

Plus, news about Nippon Shinyaku, Atsena, Orna Therapeutics, Ovid Therapeutics, Graviton Bioscience, Eupraxia, Entero Therapeutics, Journey Therapeutics, Theravance Biopharma, Alpha Cognition and Travere Therapeutics: 89bio’s $125M offering: The liver and cardiometabolic biotech is selling shares $ETNB at $8.50 apiece, or about 55 cents below Tuesday’s closing price. Its stock slid nearly 10% on Tuesday. — Kyle LaHucik FDA places partial hold on Kezar study: The company is testing an immunoproteasome inhibitor called zetomipzomib in autoimmune hepatitis. The currently enrolled patients will be allowed to complete the trial, but four patients who are still in the blinded portion cannot proceed to the open-label extension. It’s the second hold for the drug since the start of October when another trial was placed on full clinical hold after four patients died. Topline data are expected in the first half of 2025. — Max Gelman Nippon Shinyaku licenses Atsena’s gene therapy in the US and Japan: Financial details of the deal were not disclosed, though Nippon Shinyaku will reimburse Atsena for continued development work for the rare eye disease treatment, including an expected pivotal study. Atsena also gets to keep the rights to a priority review voucher if it receives one. Nippon Shinyaku plans to market the therapy in the US via its subsidiary, NS Pharma. — Lei Lei Wu Orna cuts more staffers: The circular RNA-focused biotech told Endpoints News in a statement that it “restructured its team” as part of a broader pipeline prioritization, but did not disclose the number of cuts. The drugmaker previously laid off less than 25% of its staff in November 2023 in what it described as a “defensive reduction.” — Ayisha Sharma Ovid, Graviton pause starting Phase 2 trial: The companies are holding back from initiating their study of OV888 in people with cerebral cavernous malformations (CCM) to “evaluate clinical design learnings emerging from competitor” readouts. In September, Recursion Therapeutics’ CCM drug showed strong safety but mixed efficacy in a Phase 2 trial. — Ayisha Sharma Eupraxia touts additional Phase 1b/2a data: The company said that data from two cohorts of the trial, which is investigating EP-104GI as a treatment for eosinophilic esophagitis, showed reductions in the severity, symptoms and extent of the disease. In one cohort, a patient achieved complete histological remission. Eupraxia CEO James Helliwell said that “higher dosing levels are yielding improved patient responses.” — Katherine Lewin Entero’s reverse merger: The company is handing its Nasdaq spot to ADC biotech Journey Therapeutics three months after letting go of nearly all its employees and seeking strategic alternatives. Journey will get 99% of Entero’s equity, and its CEO Henry Ji will lead the combined company. — Kyle LaHucik Theravance forms strategic review committee: In its third-quarter financial results , the company said it would “assess all strategic alternatives,” including those related to its COPD drugs Yupelri and Trelegy. Theravance pulled in $16.9 million in revenue this quarter, but it all came from the Viatris collaboration for Yupelri. Along with announcing the review, Theravance separated the roles of board chair and CEO, naming Susannah Gray as chair while Rick Winningham will continue as CEO. The company made staff and program cuts last year. Its stock $TBPH was up about 11% on Wednesday morning. — Katherine Lewin Alpha Cognition prices $50M upsized public offering: The neuro biotech received a notice of allowance for its patent application covering its Alzheimer’s drug, called Zunveyl, through 2044. The funds from the public offering are expected to go toward the commercialization and launch of Zunveyl, R&D and other commercial and general corporate purposes. Zunveyl scored FDA approval in July. — Katherine Lewin Travere upsized its public offering . It raised a total of $143.8 million. — Jaimy Lee

Topline data from PORTOLA Phase 2a clinical trial evaluating zetomipzomib in patients with autoimmune hepatitis (AIH) expected in first half 2025 Cash, cash equivalents and marketable securities totaled $148 million as of September 30, 2024 SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Nov. 12, 2024-- 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 reported financial results for the third quarter ended September 30, 2024, and provided a business update. “The team at Kezar has made great progress towards completing the double-blind portion of the PORTOLA trial as we prepare for a data release in first half of 2025,” said Chris Kirk, PhD, Kezar’s Chief Executive Officer. “There are currently no approved drugs for the treatment of autoimmune hepatitis, and we are focused on bringing zetomipzomib to patients living with this life-threatening disease. In addition, we are working to understand the safety events that occurred in the PALIZADE trial in lupus nephritis, including deaths that occurred in both the placebo and drug arms, so that we can provide patients and physicians appropriate guidance during our ongoing and future clinical trials.” Zetomipzomib: Selective Immunoproteasome Inhibitor PORTOLA – Phase 2a clinical trial of zetomipzomib in patients with AIH (ClinicalTrials.gov: NCT05569759) 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. Kezar plans to report topline data in the first half of 2025. In October, the Independent Data Monitoring Committee (IDMC) recommended that the PORTOLA trial proceed without modification. The IDMC examined safety data from all patients enrolled in the trial, including data from patients who completed the 24-week double-blind treatment period (DBTP) and continued to the open-label extension (OLE) portion of the trial that includes an additional 24 weeks of treatment. To date, no Grade 4 or 5 serious adverse events (SAEs) have been observed in this trial, which is being conducted at clinical trial sites in the United States. This recommendation occurred following the FDA’s clinical hold on the PALIZADE trial, as described below. Following the recommendation made by the IDMC, the FDA notified Kezar that it is allowing enrolled patients to complete the DBTP of the PORTOLA trial without modification. However, the FDA has placed a partial clinical hold on PORTOLA requiring that the four remaining patients currently in the DBTP should not continue to the OLE portion of the trial. Patients who are currently participating in the OLE may continue treatment on zetomipzomib, but their prednisone dosage may not be tapered below 5 mg/day, and any patients who tapered below this amount will raise their prednisone back to 5 mg/day. PALIZADE – Phase 2b clinical trial of zetomipzomib in patients with active lupus nephritis (LN) (ClinicalTrials.gov: NCT05781750) In October, Kezar made the strategic decision to terminate the PALIZADE Phase 2b clinical trial in patients with active LN and focus clinical development efforts on zetomipzomib in AIH. PALIZADE was placed on full clinical hold following the recommendation of the PALIZADE 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 is unblinding the trial and will perform a full investigation into all safety events from the study. 84 patients were enrolled in PALIZADE as of termination, and Kezar expects to report available data from PALIZADE at a later date. MISSION – Kezar will present results from the open-label Phase 1b/2 MISSION trial in patients with systemic lupus erythematosus (SLE) with or without LN showing zetomipzomib demonstrated improvements in SLE/LN disease measures and biomarkers in patients with highly active SLE or nephrotic range proteinuria at the upcoming American College of Rheumatology (ACR) Convergence 2024, which is taking place November 14 – 19, 2024, in Washington, D.C. Business Updates In October, Kezar effected a one-for-ten reverse stock split of its outstanding shares of common stock (Reverse Stock Split) to regain compliance with the minimum bid price requirement of $1.00 per share required to maintain continued listing on The Nasdaq Capital Market. The Reverse Stock Split reduced the number of shares of Kezar’s outstanding common stock from 72,962,220 shares to 7,296,222 shares, subject to adjustment due to the issuance of full shares in lieu of fractional shares. Financial Results Cash, cash equivalents and marketable securities totaled $148.4 million as of September 30, 2024, compared to $201.4 million as of December 31, 2023. The decrease was primarily attributable to cash used in operations to advance clinical-stage programs. Research and development (R&D) expenses for the third quarter of 2024 decreased by $7.5 million to $16.2 million, compared to $23.7 million in the third quarter of 2023. This decrease was primarily due to the Company’s strategic restructuring in October 2023 to prioritize its clinical-stage programs, reducing personnel-related costs and spending in its early-stage research activities. The decrease was partially offset by the increased clinical trial costs related to the PALIZADE and PORTOLA trials. General and administrative (G&A) expenses for the third quarter of 2024 decreased by $3.1 million to $5.7 million compared to $8.8 million in the third quarter of 2023. The decrease was primarily due to a decrease in legal and professional service expenses and non-cash stock-based compensation. Net loss for the third quarter of 2024 was $20.3 million, or $2.78 per basic and diluted common share, compared to a net loss of $23.1 million, or $3.18 per basic and diluted common share, for the third quarter of 2023. The weighted-average shares used to compute net loss per basic and diluted common share have been retroactively adjusted to reflect the one-for-ten reverse stock split completed on October 29, 2024. Total shares of common stock outstanding were 7.3 million shares as of September 30, 2024, after taking into effect the retroactive application of the one-for-ten reverse stock split completed on October 29, 2024. 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. Zetomipzomib, a selective immunoproteasome inhibitor, is currently being evaluated in a Phase 2a clinical trial for autoimmune hepatitis. This product candidate also has the potential to address multiple chronic immune-mediated diseases. For more information, visit www.kezarlifesciences.com, 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,” “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 clinical development programs, 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 design, initiation, progress, timing, scope and results of clinical trials, the enrollment and expected timing of reporting topline data from our clinical trials, the likelihood that data will support future development and therapeutic potential, the association of data with treatment outcomes and the likelihood of obtaining regulatory approval of Kezar’s product candidates. Many factors may cause differences between current expectations and actual results, including unexpected safety or efficacy data observed during clinical studies, difficulties enrolling and conducting our clinical trials, changes in expected or existing competition, changes in the regulatory environment, the uncertainties and timing of the regulatory approval process, and unexpected litigation or other disputes. Other factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release 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. KEZAR LIFE SCIENCES, INC. Selected Balance Sheets Data (In thousands) September 30, 2024 December 31, 2023 (unaudited) Cash, cash equivalents and marketable securities $ 148,388 $ 201,372 Total assets 164,086 221,235 Total current liabilities 20,429 17,744 Total noncurrent liabilities 9,608 15,921 Total stockholders' equity 134,049 187,570 Three Months Ended Nine Months Ended September 30 September 30 2024 2023 2024 2023 (unaudited) (unaudited) Collaboration revenue $ - $ 7,000 $ - $ 7,000 Operating expenses: Research and development 16,242 23,738 49,712 63,055 General and administrative 5,706 8,789 17,848 20,780 Impairment charge - - 1,482 - Total operating expenses 21,948 32,527 69,042 83,835 Loss from operations (21,948 ) (25,527 ) (69,042 ) (76,835 ) Interest income 2,038 2,820 6,728 8,376 Interest expense (403 ) (396 ) (1,204 ) (1,151 ) Net loss $ (20,313 ) $ (23,103 ) $ (63,518 ) $ (69,610 ) Net loss per common share, basic and diluted $ (2.78 ) $ (3.18 ) $ (8.72 ) $ (9.60 ) Weighted-average shares used to compute net loss per common share, basic and diluted (1) 7,296,222 7,268,165 7,286,967 7,249,188 (1) Shares outstanding have been retroactively adjusted to reflect the one-for-ten reverse stock split that completed on October 29, 2024, subject to adjustment due to the issuance of full shares in lieu of fractional shares. View source version on businesswire.com: https://www.businesswire.com/news/home/20241112979408/en/ Investor and Media Contact: Gitanjali Jain Senior Vice President, Investor Relations and External Affairs Kezar Life Sciences, Inc. gjain@kezarbio.com Source: Kezar Life Sciences, Inc.