Acute myeloid leukaemia with 11q23 abnormality

– New PDUFA action date of December 26, 2024 allows FDA additional time to complete their review – WALTHAM, Mass., July 29, 2024 /PRNewswire/ -- Syndax Pharmaceuticals (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) action date for the New Drug Application (NDA) for revumenib for the treatment of adults and pediatric patients with relapsed or refractory (R/R) KMT2Ar acute leukemia. The FDA notified Syndax on July 26, 2024 that they required additional time to conduct a full review of supplemental information provided to the FDA in response to their requests. The submission of additional information to the FDA was determined to constitute a Major Amendment to the NDA and resulted in a standard three-month extension to the original PDUFA action date of September 26, 2024. No additional trials or manufacturing information have been requested by the FDA. "Revumenib, upon approval, will be the first drug indicated to treat patients with KMT2A-rearranged acute leukemia, a population with significant unmet need," said Michael A. Metzger, Chief Executive Officer. "We are confident that the data from the AUGMENT-101 trial, as well as the additional information provided to the FDA, support approval and continue to demonstrate the meaningful benefit revumenib brings to patients with this devastating disease. We look forward to continuing our engagement with the FDA as they complete their review of the NDA by December 26, 2024." The NDA for revumenib was granted Priority Review and is being reviewed under the FDA's Real-Time Oncology Review (RTOR) program. The FDA previously granted Breakthrough Therapy, Fast Track and Orphan Drug designations for revumenib. About Revumenib Revumenib is a potent, selective, small molecule inhibitor of the menin-KMT2A binding interaction that is being developed for the treatment of KMT2A-rearranged (KMT2Ar), also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including ALL and AML, and mutant nucleophosmin (mNPM1) AML. Positive topline results from the Phase 2 AUGMENT-101 trial in R/R KMT2Ar acute leukemia showing the trial met its primary endpoint were presented at the 65th American Society of Hematology Annual Meeting, and data from the Phase 1 portion of AUGMENT-101 in acute leukemia was published in Nature. Revumenib was granted Orphan Drug Designation for the treatment of AML and ALL by the FDA and for the treatment of AML by the European Commission, and Fast Track designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation. Revumenib was granted Breakthrough Therapy Designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement. About the Real-Time Oncology Review Program (RTOR) RTOR provides a more efficient review process for oncology drugs to ensure that safe and effective treatments are available to patients as early as possible, while improving review quality and engaging in early iterative communication with the applicant. Specifically, it allows for close engagement between the sponsor and the FDA throughout the submission process and it enables the FDA to review individual sections of modules of a drug application rather than requiring the submission of complete modules or a complete application prior to initiating review. Additional information about RTOR can be found at: About Syndax Syndax Pharmaceuticals is a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Highlights of the Company's pipeline include revumenib, a highly selective inhibitor of the menin–KMT2A binding interaction, and axatilimab, a monoclonal antibody that blocks the CSF-1 receptor. For more information, please visit or follow the Company on X (formerly Twitter) and LinkedIn. 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 "anticipate," "believe," "could," "estimate," "expects," "intend," "may," "plan," "potential," "predict," "project," "should," "will," "would" or the negative or plural of those terms, 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 Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials, the reporting of clinical data for Syndax's product candidates, and the potential use of its product candidates to treat various cancer indications and fibrotic diseases. Many factors may cause differences between current expectations and actual results, including: unexpected safety or efficacy data observed during preclinical or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; changes in expected or existing competition; changes in the regulatory environment; failure of Syndax's collaborators to support or advance collaborations or product candidates; and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. Syndax Contact Sharon Klahre Syndax Pharmaceuticals, Inc. [email protected] Tel 781.684.9827 SNDX-G SOURCE Syndax Pharmaceuticals, Inc.

– Company enrolls 85 patients with R/R NPM1-mutant AML in fewer than 16 months – – Topline data expected in early 2025 – – Breakthrough Therapy Designation to enable expedited review by FDA – SAN DIEGO, May 14, 2024 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today announced that it has completed enrollment of 85 patients in the Phase 2 portion of KOMET-001, a registration-directed clinical trial of the Company’s menin inhibitor, ziftomenib (KO-539), in patients with relapsed or refractory (R/R) NPM1-mutant acute myeloid leukemia (AML). The Company expects to report topline data from the trial in early 2025. “We are thrilled to announce this critical milestone, which brings us one step closer to delivering ziftomenib as a potentially best-in-class treatment for patients with genetically defined acute leukemias,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “Our confidence is supported by our recently announced Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA), which recognizes ziftomenib’s potential as an innovative medicine for patients with R/R NPM1-mutant AML and is intended to expedite review as we prepare for submission of a New Drug Application. We are grateful for the KOMET-001 investigators, patients and their families, and we look forward to sharing topline data from this pivotal study early next year.” Kura announced the first patients dosed in the Phase 2 portion of KOMET-001 in February 2023. The registration-directed study is designed to assess evidence of clinical activity, safety and tolerability of ziftomenib in patients with R/R NPM1-mutant AML, with a primary endpoint of complete response. The study has completed enrollment of the 85 patients necessary to support the primary endpoint analysis. “The rapid enrollment of this study reflects the urgent need for more effective treatment options in AML as well as the potential for ziftomenib to address this need,” said Eunice Wang, M.D., Chief of the Leukemia Service at Roswell Park Comprehensive Cancer Center and principal investigator of the trial. “NPM1-mutant AML represents approximately 30% of new AML cases annually and is a disease of significant unmet need for which there is no approved targeted therapy. The favorable safety pro encouraging clinical activity demonstrated by ziftomenib to date offer the potential to transform the standard of care for these AML patients.” About NPM1-mutant AML AML is the most common acute leukemia in adults and begins when the bone marrow makes abnormal myeloblasts (white blood cells), red blood cells or platelets. Despite the many available treatments for AML, prognosis for patients remains poor and a high unmet need remains. The menin pathway is considered a driver for multiple genetic alterations of the disease, of which NPM1 mutations are among the most common, representing approximately 30% of AML cases. While patients with NPM1-m AML have high response rates to frontline therapy, relapse rates are high and survival outcomes are poor, with only 30% overall survival at 12 months in the R/R setting. Additionally, NPM1 mutations frequently occur with co-mutations in other disease-associated genes, including FLT3, DNMT3A and IDH1/2, with prognosis heavily influenced by the occurrence of co-occurring mutations. Adult patients with NPM1-m AML and select co-mutations and/or R/R disease have a poor prognosis, with median overall survival of only approximately 7.8 months in 2nd line, 5.3 months in 3rd line and 3.5 months following the 4th line.1 There are currently no FDA-approved therapies targeting NPM1-m AML. About Ziftomenib Ziftomenib is a novel, once-daily, oral investigational drug candidate targeting the menin-KMT2A/MLL protein-protein interaction for treatment of genetically defined AML patients with high unmet need. In the KOMET-001 Phase 1 study, ziftomenib demonstrated an encouraging safety pro tolerability with reported events most often consistent with features and manifestations of underlying disease. Clinical activity of ziftomenib as a monotherapy was optimal at the 600 mg daily dose and a 35% complete remission rate was observed in 20 patients with NPM1-mutant AML treated at the recommended Phase 2 dose (600 mg). Ziftomenib has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration for the treatment of R/R NPM1-mutant AML. Additional information about clinical trials for ziftomenib can be found at kuraoncology.com/clinical-trials/#ziftomenib. About Kura Oncology Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer. The Company’s pipeline consists of small molecule drug candidates that target cancer signaling pathways. Ziftomenib, a once-daily, oral drug candidate targeting the menin-KMT2A protein-protein interaction, has received Breakthrough Therapy Designation for the treatment of R/R NPM1-mutant AML. Kura has completed enrollment in a Phase 2 registration-directed trial of ziftomenib in NPM1-mutant R/R AML (KOMET-001) and expects to report topline data in early 2025. The Company is also conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in NPM1-mutant and KMT2A-rearranged newly diagnosed and R/R AML. Tipifarnib, a potent and selective farnesyl transferase inhibitor (FTI), is currently in a Phase 1/2 trial in combination with alpelisib for patients with PIK3CA-dependent head and neck squamous cell carcinoma (KURRENT-HN). Kura is also evaluating KO-2806, a next-generation FTI, in a Phase 1 dose-escalation trial as a monotherapy and in combination with targeted therapies (FIT-001). For additional information, please visit Kura’s website at and follow us on X and LinkedIn. Forward-Looking Statements This news release contains certain forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, the efficacy, safety and therapeutic potential of ziftomenib, potential benefits of combining ziftomenib with appropriate standards of care, and progress and expected timing of the ziftomenib program and clinical trials. Factors that may cause actual results to differ materially include the risk that compounds that appeared promising in early research or clinical trials do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials, the risk that Kura may not obtain approval to market its product candidates, uncertainties associated with performing clinical trials, regulatory filings, applications and other interactions with regulatory bodies, risks associated with reliance on third parties to successfully conduct clinical trials, the risks associated with reliance on outside financing to meet capital requirements, and other risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. You are urged to consider statements that include the words “may,” “will,” “would,” “could,” “should,” “believes,” “estimates,” “projects,” “promise,” “potential,” “expects,” “plans,” “anticipates,” “intends,” “continues,” “designed,” “goal,” or the negative of those words or other comparable words to be uncertain and forward-looking. For a further list and description of the risks and uncertainties the Company faces, please refer to the Company's periodic and other filings with the Securities and Exchange Commission, which are available at . Such forward-looking statements are current only as of the date they are made, and Kura assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. Contacts Investors: Pete De Spain Executive Vice President, Investor Relations & Corporate Communications (858) 500-8833 pete@kuraoncology.com Media: Alexandra Weingarten Associate Director, Corporate Communications & Investor Relations (858) 500-8822 alexandra@kuraoncology.com 1 Issa G, et al. Blood Adv 2023;7(6):933-42.

SAN DIEGO, May 08, 2024 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today announced its participation in Bank of America Securities 2024 Healthcare Conference. Troy Wilson, Ph.D., J.D., President and Chief Executive Officer, is scheduled to participate in a virtual fireside chat at 1:40 p.m. ET / 10:40 a.m. PT on May 15, 2024. A live audio webcast of the fireside chat will be available in the Investors section of Kura’s website at www.kuraoncology.com, with an archived replay following the event. About Kura Oncology Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer. The Company’s pipeline consists of small molecule drug candidates that target cancer signaling pathways. Ziftomenib, a once-daily, oral drug candidate targeting the menin-KMT2A protein-protein interaction, has received Breakthrough Therapy Designation for the treatment of relapsed/refractory (R/R) NPM1-mutant acute myeloid leukemia (AML). Kura is currently enrolling patients in a Phase 2 registration-directed trial of ziftomenib in NPM1-mutant R/R AML (KOMET-001). The Company is also conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in NPM1-mutant and KMT2A-rearranged newly diagnosed and R/R AML. Tipifarnib, a potent and selective farnesyl transferase inhibitor (FTI), is currently in a Phase 1/2 trial in combination with alpelisib for patients with PIK3CA-dependent head and neck squamous cell carcinoma (KURRENT-HN). Kura is also evaluating KO-2806, a next-generation FTI, in a Phase 1 dose-escalation trial as a monotherapy and in combination with targeted therapies (FIT-001). For additional information, please visit Kura’s website at www.kuraoncology.com and follow us on X and LinkedIn. Contacts Investors:Pete De SpainExecutive Vice President, Investor Relations &Corporate Communications(858) 500-8833pete@kuraoncology.com Media:Alexandra WeingartenAssociate Director, Corporate Communications & Investor Relations(858) 500-8822alexandra@kuraoncology.com