Syndax Announces Completion of Enrollment in AUGMENT-101 Pivotal Trial Cohort of Patients with Relapsed/Refractory mNPM1 Acute Myeloid Leukemia
- Revumenib targets a $2 billion market opportunity in mutant nucleophosmin acute myeloid leukemia.
- Syndax Pharmaceuticals aims to expand therapeutic reach in genetically defined acute leukemias.
- Company positioned well with potential launches of revumenib and axatilimab in 2024.
- NDA under FDA review for revumenib in R/R KMT2Ar acute leukemia with PDUFA action date in September 2024.
- None.
Insights
The enrollment completion of Syndax Pharmaceuticals' AUGMENT-101 pivotal trial marks a significant milestone in the development of revumenib for treating relapsed/refractory acute myeloid leukemia with mutant nucleophosmin. The focus on mNPM1 mutations is noteworthy as these are associated with distinct pathogenesis and prognosis in AML, representing a subgroup where targeted therapies can potentially offer a substantial clinical benefit.
From a medical standpoint, the anticipation of topline data and the subsequent sNDA filing highlight the accelerated pathway that novel therapies can undergo when addressing unmet medical needs. The primary endpoint of complete remission rates, combined with secondary endpoints like duration of response and overall survival, will be critical in evaluating revumenib's potential impact on patient outcomes. This is especially relevant in the context of a disease like AML, where treatment-resistant forms pose a significant challenge.
The projected market opportunity for revumenib in the U.S. is substantial, with an estimated value of $2 billion for the mNPM1 and KMT2Ar relapsed/refractory acute leukemia segment. Syndax's strategic positioning with revumenib, potentially as a first-and-best-in-class drug, could allow them to capture a significant share of this market, assuming successful trial outcomes and regulatory approvals.
The company's pipeline and the planned launch of revumenib and axatilimab in 2024 suggest a proactive approach to growth and diversification within the oncology space. Investors will be closely monitoring the PDUFA action date and the potential market penetration strategies post-approval. The long-term value for shareholders will hinge on the drug's efficacy, safety profile and the ability to secure reimbursement and adoption within clinical practice.
The completion of enrollment for the AUGMENT-101 trial is a critical event that can influence Syndax Pharmaceuticals' financial trajectory. The anticipation surrounding the topline data release in 4Q24 and the potential sNDA filing in 1H25 are likely to be catalysts for the company's stock performance in the near term. Positive outcomes could lead to substantial appreciation in stock value due to the high stakes associated with the drug's addressable market.
In assessing the potential financial implications, it's essential to consider the costs associated with the drug's development, the pricing strategy in a competitive market and the potential for insurance coverage. Moreover, the company's ability to manage the commercialization process effectively will be important for realizing the projected revenues and ensuring a return on investment for stakeholders.
– Topline data expected in 4Q24 could support sNDA filing in 1H25 –
– Revumenib has the potential to address
U.S. market opportunity –
"We are thrilled to announce that we are one step closer to potentially expanding the therapeutic reach of revumenib in genetically defined acute leukemias," said Neil Gallagher, M.D., Ph.D., President, Head of Research and Development at Syndax. "We look forward to reporting topline data for this pivotal cohort in the fourth quarter of this year, which will follow closely behind a potential first approval of KMT2A acute leukemia in the third quarter."
Michael A. Metzger, Chief Executive Officer, added, "With revumenib and axatilimab, two first-and best-in-class drugs, expected to launch in 2024 and the potential to expand beyond first approvals and into additional indications, Syndax is well positioned to deliver on its mission to improve the lives of cancer patients and create meaningful long-term value for shareholders."
Sixty-four (64) adult and up to 20 pediatric patients with mNPM1 AML have been enrolled into the pivotal portion of AUGMENT-101, a pivotal trial designed to evaluate the safety, tolerability, pharmacokinetics, and efficacy of orally administered revumenib. The primary endpoint for the trial is efficacy as measured by complete remission (CR) or a CR with partial hematological recovery (CRh) rate (CR + CRh) per protocol, with secondary endpoints including duration of response (DOR) and overall survival (OS).
A new drug application (NDA) is under review by the
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, also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including ALL and AML, and NPM1-mutant 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 by the FDA and European Commission for the treatment of patients with AML, 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 (BTD) by the FDA for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.
About NPM1-Mutant Acute Myeloid Leukemia
NPM1-mutant AML, which is distinguished by mutations in the NPM1 gene that drive the leukemic phenotype, is the most common type of cytogenetically normal adult AML and represents approximately
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 colony stimulating factor 1 (CSF-1) receptor. For more information, please visit. For more information, please visit www.syndax.com 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.
sklahre@syndax.com
Tel 781.684.9827
SNDX-G
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SOURCE Syndax Pharmaceuticals, Inc.
FAQ
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