WALTHAM, Mass. - Syndax Pharmaceuticals (NASDAQ:SNDX) announced positive results from a Phase 2 trial of revumenib for treating relapsed or refractory mutant NPM1 (mNPM1) acute myeloid leukemia (AML). The AUGMENT-101 trial met its primary endpoint, reporting a complete remission (CR) and CR with partial hematological recovery (CRh) rate of 23% among evaluable adults. The trial also demonstrated a 47% overall response rate in a heavily pre-treated patient group, which included 75% with prior exposure to venetoclax, a common AML treatment.
The study included 64 adult patients, with a median age of 65, who had received multiple prior lines of therapy. The safety profile was favorable, with only 5% of patients discontinuing due to treatment-related adverse events. The most common severe adverse events were QTc prolongation, anemia, febrile neutropenia, differentiation syndrome, and decreased platelet count.
Syndax's CEO, Michael A. Metzger, expressed optimism about revumenib's potential, particularly given the expected FDA approval for a related leukemia treatment in the fourth quarter of 2024. With this positive data, Syndax is preparing for a supplemental New Drug Application (sNDA) filing in the first half of 2025.
Dr. Eytan M. Stein from Memorial Sloan Kettering Cancer Center highlighted the significance of these results for a patient population with limited treatment options and poor prognosis. The findings are especially encouraging given the number of participants who had previously failed venetoclax treatment.
The company plans to present further data at the American Society of Hematology Annual Meeting in December 2024 and to initiate additional clinical trials by the end of the year. These developments are part of Syndax's broader efforts to explore the clinical benefits of revumenib for various acute leukemias.
This news is based on a press release statement from Syndax Pharmaceuticals and does not include any independent verification of the claims. The information reflects the company's current expectations and may change as further data becomes available.
In other recent news, Syndax Pharmaceuticals underscored its financial and clinical progress in its Q3 2024 earnings call. The company announced a substantial $350 million royalty agreement with Royalty Pharma for Niktimvo, aiming to strengthen its financial position and aid in the commercialization of Niktimvo and revumenib. The FDA's approval of Niktimvo for chronic graft-versus-host disease and the anticipated approval of revumenib for acute leukemia were underlined as notable achievements.
Syndax reported $399.6 million in cash as of September 30, with operating expenses for Q3 standing at $102.1 million. The 2024 guidance was adjusted to $365 million to $370 million. The company is preparing for the commercial launch of Niktimvo in early Q1 2025, targeting a market worth $1.5 billion to $2 billion for third-line cGVHD treatment.
Revumenib's clinical data shows promise, with plans to present at an upcoming conference and initiate a pivotal Phase 3 trial. Despite a lack of a specific timeline for the conclusion of the HOVON trial data, Syndax is optimistic about the commercial prospects of Niktimvo and revumenib. These recent developments reflect Syndax's commitment to aggressive clinical development and commercialization strategies.
InvestingPro Insights
Syndax Pharmaceuticals' positive Phase 2 trial results for revumenib have sparked investor interest, as reflected in the company's recent stock performance. According to InvestingPro data, SNDX has shown a strong 10.61% return over the last week and an impressive 60.74% return over the past year, indicating growing market confidence in the company's pipeline.
Despite these encouraging clinical results, it's important to note that Syndax is not yet profitable. An InvestingPro Tip highlights that the company is not expected to be profitable this year, which is common for biotech firms in the development stage. However, another InvestingPro Tip reveals that Syndax holds more cash than debt on its balance sheet, suggesting a solid financial position to support ongoing research and development efforts.
The company's market capitalization stands at $1.86 billion, reflecting investor optimism about its potential. However, with a price-to-book ratio of 5.07, the stock may be considered relatively expensive compared to its book value. This valuation could be justified by the promising clinical results and the potential market for revumenib in AML treatment.
For investors seeking a more comprehensive analysis, InvestingPro offers 12 additional tips for Syndax Pharmaceuticals, providing a deeper understanding of the company's financial health and market position.
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