Syndax Pharmaceuticals (NASDAQ: SNDX) has received FDA approval for Revuforj (revumenib) for the treatment of acute leukemia in both adult and pediatric patients (one year and older) with a KMT2A translocation. This approval marks a significant milestone as Revuforj is the first and only FDA-approved menin inhibitor for this patient population.
The FDA's decision, which came after the market closed on Friday, was based on data from the Phase 1/2 AUGMENT-101 trial. The agency had previously granted Revuforj Breakthrough Therapy Designation, recognizing its potential as a substantial improvement over existing treatments. The approval was expedited through the FDA's Real-Time Oncology Review program, ahead of the expected Prescription Drug User Fee Act (PDUFA) deadline of December 26, 2024.
Clinical Trial Data
The AUGMENT-101 trial included an analysis of 104 patients with relapsed or refractory acute leukemia exhibiting a KMT2A translocation. Results from the trial demonstrated a complete response rate of 21%. The median duration of response was 6.4 months, with a median time to achieve a response of 1.9 months. Notably, 23% of the patients were able to proceed to hematopoietic stem cell transplantation following treatment with Revuforj.
Analyst Outlook
H.C. Wainwright's analyst cited the FDA approval and the drug's breakthrough status as key reasons for reiterating the Buy rating and raising the price target for Syndax's shares from $49.00 to $51.00. The firm's positive outlook reflects the potential market impact of Revuforj as a new treatment option for patients with this challenging form of leukemia.
Additional Developments
Syndax Pharmaceuticals recently announced positive results from its AUGMENT-101 trial for revumenib in acute myeloid leukemia (AML), where the trial met its primary endpoint with a 23% complete response rate among evaluable adults. In financial news, Syndax announced a $350 million royalty agreement with Royalty Pharma for Niktimvo during its Q3 2024 earnings call. The company reported $399.6 million in cash as of September 30, with Q3 operating expenses at $102.1 million.
Market Volatility
Despite the positive news, Syndax Pharmaceuticals' stock has experienced significant volatility. InvestingPro data indicates a 1-week price total return of -27.32%. However, the company holds more cash than debt on its balance sheet, providing financial flexibility for the commercialization of Revuforj. Analysts anticipate that Syndax will not be profitable this year, which is not uncommon for biotech companies in the early stages of bringing a drug to market. The market cap stands at $1.35 billion, reflecting investor expectations for future growth.
