Syndax Announces FDA Priority Review of NDA for Revumenib for the Treatment of Relapsed/Refractory KMT2Ar Acute Leukemia
- Priority Review granted for Syndax's NDA for revumenib, a menin inhibitor for KMT2Ar acute leukemia.
- Efficient review process under FDA's RTOR program for potential earlier approval.
- Positive data from AUGMENT-101 trial supports NDA submission for revumenib.
- 23% CR/CRh rate among efficacy evaluable patients in KMT2Ar acute leukemia population.
- 70% of patients achieving CR/CRh were MRD negative.
- 63% overall response rate, with 39% undergoing HSCT and 50% restarting revumenib post-transplant.
- None.
Insights
The FDA's decision to grant Priority Review for revumenib, a menin inhibitor aimed at treating KMT2A-rearranged acute leukemia, marks a critical advancement in oncology therapeutics. Menin inhibitors represent a novel class of drugs that target leukemogenic proteins involved in the pathogenesis of certain leukemias. This is particularly pertinent for patients with relapsed or refractory acute leukemia, where treatment options are limited and prognosis is poor.
The AUGMENT-101 trial results, showing a complete remission rate of 23% and a high percentage of MRD-negative responses among those in remission, demonstrate a promising therapeutic potential for revumenib. The ability to achieve MRD negativity is a strong prognostic factor for improved survival outcomes in leukemia patients. Furthermore, the fact that a significant portion of responders could proceed to hematopoietic stem cell transplant, a potentially curative treatment, underscores the drug's potential impact on patient management.
While these results are encouraging, it's important to consider the long-term effects and safety profile of revumenib, as well as the impact of post-transplant maintenance therapy on survival and quality of life. The RTOR program's expedited review process could bring this drug to market more quickly, offering hope to patients with limited treatment options.
The FDA's Real-Time Oncology Review Program is designed to expedite the availability of cancer treatments that may offer significant benefits. Syndax Pharmaceuticals' transition to a commercial-stage company is indicative of the industry trend where biopharmaceutical companies are rapidly advancing from research and development to commercialization of therapies. This reflects positively on the company's capability to innovate and execute on clinical development plans.
Investors and stakeholders should note the potential market impact of revumenib. The high MRD-negative response rate is a key factor, as it may lead to a competitive advantage in the market, considering the importance of MRD status in treatment decisions and prognosis. The success of this drug could also pave the way for further development of menin inhibitors, potentially opening new revenue streams for Syndax.
However, it is important to monitor the competitive landscape, as other companies may also be developing similar therapies. The long-term commercial success of revumenib will depend on its efficacy and safety profile compared to existing and future treatments, pricing and insurance coverage policies.
The assignment of a PDUFA date is a significant event for Syndax Pharmaceuticals and its investors. It serves as a catalyst for potential stock price movements as the market anticipates the FDA's decision. The progress under the RTOR program and the Priority Review status are likely to be viewed favorably by investors, as they suggest a higher probability of approval and a potentially expedited time to market.
From a financial perspective, the successful commercialization of revumenib could lead to substantial revenue growth for Syndax. Given the drug's 'first-in-class' status and its application in a high-need area, the pricing power could be strong, assuming the clinical benefits are confirmed upon further review. The company's focus on commercial preparations indicates a strategic foresight into the necessary infrastructure and marketing efforts required for a successful launch.
It is important for investors to consider the implications of the upcoming PDUFA date on Syndax's financials, including potential increases in operational expenses related to the launch and marketing of the drug, as well as the impact on cash flow and earnings. Additionally, the outcome of the FDA's decision will likely have a significant influence on the company's valuation.
– PDUFA action date set for September 26, 2024 –
– NDA being reviewed under FDA's RTOR program –
"The receipt of Priority Review for the revumenib NDA filing is a significant milestone as we transition to a leading commercial-stage oncology company with the planned launches of two first- and best-in class drugs in 2024," said Michael A. Metzger, Chief Executive Officer. "With two regulatory filings now under FDA Priority Review, our team is focused on commercial preparations to enable Syndax's continued success as we enter this next stage of growth."
The NDA submission is supported by positive data from the pivotal AUGMENT-101 trial of revumenib in adult and pediatric patients with KMT2Ar acute myeloid leukemia (AML) and acute lymphoid leukemia (ALL). As previously reported, the trial met its primary endpoint at the protocol-defined interim analysis with a complete remission (CR) or a CR with partial hematological recovery (CRh) rate of
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 Real-Time Oncology Review (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 colony stimulating factor 1 (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
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