Syndax Reports Strong Q2 2025 Results with Revuforj Revenue Growth and Pending FDA Approval for NPM1 AML
核心洞察
Syndax Pharmaceuticals reported $28.6 million in Revuforj net revenue for Q2 2025, representing a 43% increase over the first quarter despite approximately one-third of patients pausing treatment for stem cell transplants.
The FDA granted Priority Review to Syndax's supplemental New Drug Application for Revuforj in relapsed/refractory mutant NPM1 (搜索) acute myeloid leukemia (搜索), with a PDUFA action date of October 25, 2025.
Niktimvo generated $36.2 million in net revenue during its first full quarter of launch, with Syndax recording $9.4 million in collaboration revenue from its 50% profit-sharing agreement with Incyte.
Syndax Pharmaceuticals reported robust second quarter 2025 financial results, driven by strong commercial performance of its two FDA-approved cancer therapies and significant regulatory progress toward expanding Revuforj's approved indications.
The commercial-stage biopharmaceutical company achieved $28.6 million in net revenue from Revuforj (revumenib) during the second quarter, representing a 43% increase over the first quarter of 2025. This growth occurred despite approximately one-third of patients temporarily discontinuing treatment to receive stem cell transplants, according to CEO Michael A. Metzger.
FDA Priority Review for NPM1 AML Indication
The U.S. FDA granted Priority Review to Syndax's supplemental New Drug Application (sNDA) seeking approval of Revuforj for treating relapsed or refractory mutant NPM1 (搜索) (mNPM1) acute myeloid leukemia (搜索). The application is being reviewed under the FDA's Real-Time Oncology Review program and has been assigned a Prescription Drug User Fee Act target action date of October 25, 2025.
The sNDA is supported by pivotal data from the AUGMENT-101 trial, which demonstrated an overall response rate of 48% (37/77) in efficacy-evaluable patients with relapsed/refractory mNPM1 AML. The complete remission plus complete remission with partial hematologic recovery rate was 26% (20/77), with a median duration of response of 4.7 months. Among patients achieving complete remission who were assessed for measurable residual disease, 63% (12/19) were MRD negative.
Subpopulation analysis revealed a median overall survival of 23.3 months (95% CI: 8.4-NR) among the 37 patients who achieved an overall response, based on Kaplan-Meier estimates from the single-arm trial.
Niktimvo Launch Success
Niktimvo (axatilimab-csfr) generated $36.2 million in net revenue during the second quarter, its first full quarter following the U.S. launch in late January 2025. This represented significant growth compared to $13.6 million in net revenue during the first quarter. Under the co-commercialization agreement with Incyte, Syndax records 50% of Niktimvo's net commercial profit, resulting in $9.4 million in collaboration revenue for the quarter.
"Niktimvo is off to a very fast start generating $50 million in net revenue in just the first five months of the launch," Metzger stated. "It is already profitable to Syndax and we expect increasing profitability margins as revenues continue to grow."
Expanding Clinical Development Programs
Multiple trials evaluating revumenib across the treatment landscape are ongoing, including several combination studies in newly diagnosed patients:
The EVOLVE-2 trial, a pivotal Phase 3 study, is evaluating revumenib combined with venetoclax and azacitidine in newly diagnosed mNPM1 AML patients unfit for intensive chemotherapy. The trial is being conducted in collaboration with the HOVON network.
The Beat AML Phase 1 trial showed promising results in newly diagnosed older adults, with updated data demonstrating a 67% (29/43) complete remission rate and 100% (37/37) flow-MRD negativity rate among evaluable responders. These results were published in the Journal of Clinical Oncology and presented at the European Hematology Association Annual Congress Meeting in June 2025.
The SAVE trial, evaluating an all-oral combination of revumenib with venetoclax and decitabine/cedazuridine, reported an overall response rate of 82% (27/33) and a complete remission/complete remission with partial hematologic recovery rate of 48% (16/33) in patients with relapsed/refractory disease.
Financial Position and Outlook
As of June 30, 2025, Syndax maintained cash, cash equivalents, and short and long-term investments of $517.9 million. Total revenue for the second quarter reached $38.0 million, consisting of $28.6 million in Revuforj net product revenue and $9.4 million in Niktimvo collaboration revenue.
Research and development expenses increased to $62.2 million from $48.7 million in the comparable prior year period, primarily due to expanded revumenib-related costs across multiple trials, a milestone payment to AbbVie (搜索) upon sNDA submission, and increased personnel costs supporting commercialization.
The company reported a net loss of $71.8 million, or $0.83 per share, compared to a net loss of $68.1 million, or $0.80 per share, in the prior year period.
For the third quarter of 2025, Syndax expects total research and development plus selling, general and administrative expenses of $95 to $100 million, excluding non-cash stock compensation. Full-year 2025 guidance remains unchanged at $370 to $390 million for the same expense categories.
Management expects the company's cash position, combined with anticipated product revenue and interest income, will enable Syndax to reach profitability. "With strong Revuforj and Niktimvo sales momentum along with our expectation for stable expenses over the next few years and more than $500 million in cash, we are well on our way to becoming a profitable company with two blockbuster products," Metzger concluded.