Syndax Announces PDUFA Action Date Extension for Revumenib NDA for Relapsed or Refractory KMT2Ar Acute Leukemia

Rhea-AI Summary

Syndax Pharmaceuticals (NASDAQ: SNDX) announced that the FDA has extended the PDUFA action date for the New Drug Application (NDA) of revumenib to December 26, 2024. This three-month extension from the original date of September 26, 2024, allows the FDA additional time to review supplemental information provided by Syndax in response to their requests. The submission was deemed a Major Amendment to the NDA.

Revumenib is being developed for the treatment of adults and pediatric patients with relapsed or refractory KMT2Ar acute leukemia. Upon approval, it would be the first drug indicated for this specific condition. The NDA has been granted Priority Review and is being reviewed under the FDA's Real-Time Oncology Review program. Revumenib has also received Breakthrough Therapy, Fast Track, and Orphan Drug designations.

Positive

• Revumenib, if approved, will be the first drug indicated for KMT2A-rearranged acute leukemia
• NDA granted Priority Review and being reviewed under FDA's Real-Time Oncology Review program
• Revumenib has received Breakthrough Therapy, Fast Track, and Orphan Drug designations

Negative

• PDUFA action date extended by three months to December 26, 2024
• Delay in potential market entry for revumenib

Oncology Research Analyst

The FDA's extension of the PDUFA action date for Syndax's revumenib NDA is a significant development in the oncology drug pipeline. This three-month delay to December 26, 2024, while not ideal for Syndax, is not uncommon in the regulatory process, especially for novel therapies.

Key points to consider:

• The extension is due to the FDA requiring more time to review supplemental information, which constitutes a Major Amendment. This suggests the agency is thoroughly examining the data, which could be seen as a positive sign of their engagement with the application.
• No additional trials or manufacturing information have been requested, indicating that the core data package is likely sufficient.
• Revumenib's potential as the first drug for KMT2A-rearranged acute leukemia underscores its importance. This rare subtype of leukemia has a poor prognosis and new targeted therapies are desperately needed.
• The drug's multiple FDA designations (Priority Review, Breakthrough Therapy, Fast Track and Orphan Drug) highlight its potential significance in addressing an unmet medical need.

For investors, while this delay might cause short-term volatility in Syndax's stock price, it's important to focus on the long-term potential of revumenib. The additional review time could potentially lead to a more robust label if approved, which could benefit commercialization efforts.

Biotech Financial Analyst

From a financial perspective, the PDUFA date extension for revumenib presents a mixed scenario for Syndax Pharmaceuticals:

• Short-term impact: The three-month delay could potentially affect Syndax's cash runway and push back revenue projections. Investors should closely monitor the company's cash position and burn rate.
• Market reaction: We might see some volatility in SNDX stock as the market digests this news. However, given that no additional trials were requested, any negative reaction might be
• Long-term outlook: If approved, revumenib could still be first-to-market in its indication, which is important for capturing market share in the orphan drug space.
• Competitive landscape: The delay gives competitors more time to advance their pipelines, but revumenib's unique positioning in KMT2A-rearranged acute leukemia maintains its potential as a breakthrough therapy.

Investors should consider that while delays are not ideal, they're not uncommon in the biotech sector, especially for novel therapies. The key will be Syndax's ability to navigate this extended review period without significant additional costs or delays. The company's communication strategy and transparency during this period will be important for maintaining investor confidence.

– 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: https://www.fda.gov/about-fda/oncology-center-excellence/real-time-oncology-review-pilot-program

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 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

What is the new PDUFA action date for Syndax's revumenib NDA?

The new PDUFA action date for Syndax's (SNDX) revumenib NDA is December 26, 2024.

Why did the FDA extend the PDUFA action date for revumenib?

The FDA extended the PDUFA action date for revumenib to allow additional time to review supplemental information provided by Syndax in response to their requests, which was deemed a Major Amendment to the NDA.

What condition is revumenib being developed to treat?

Revumenib is being developed to treat adults and pediatric patients with relapsed or refractory KMT2Ar acute leukemia.

What designations has revumenib received from the FDA?

Revumenib has received Breakthrough Therapy, Fast Track, and Orphan Drug designations from the FDA.