Syndax Announces Q3 2024 Financial Results and Business Update

Syndax Pharmaceuticals, a prominent biopharmaceutical company based in Waltham, Massachusetts, has announced significant progress in its pipeline of cancer therapies and shared its financial results for the quarter ending September 30, 2024. The company, now at a commercial stage, highlighted several key milestones and anticipated future developments.

Syndax reported the approval of Niktimvo™ by the U.S. FDA for the treatment of chronic graft-versus-host disease (GVHD) in patients who have not responded to at least two prior lines of systemic therapy. This approval marks a pivotal moment for the company, enabling it to transition into a commercial-stage entity. Niktimvo, which will be co-commercialized by Syndax and Incyte in the U.S., is expected to be launched by early 2025.

The company also shared updates on revumenib, an oral menin inhibitor for treating relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar) acute leukemia. The New Drug Application (NDA) for revumenib has been granted Priority Review by the U.S. FDA and is under evaluation through the Real-Time Oncology Review (RTOR) program. The Prescription Drug User Fee Act (PDUFA) target action date is set for December 26, 2024. Syndax anticipates topline data from the AUGMENT-101 pivotal trial cohort for patients with R/R mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML) by the end of 2024. Positive outcomes could support a supplemental NDA filing in the first half of 2025.

Moreover, Syndax announced that data from the pivotal Phase 2 portion of the AUGMENT-101 trial would be presented at the 66th American Society of Hematology (ASH) Annual Meeting. The study includes a larger population of 97 patients, showing durable responses and robust rates of overall response, minimal residual disease (MRD) negativity, and hematopoietic stem cell transplantation (HSTC). The median duration of complete response/complete response with partial hematologic recovery (CR/CRh) extended to 13 months among the CR/CRh responders.

Syndax is conducting several trials to expand revumenib’s use in the treatment landscape for mNPM1 and KMT2Ar acute leukemia. These include the BEAT AML trial, which evaluates revumenib in combination with venetoclax and azacitidine, showing a 96% composite complete remission rate. The SAVE trial assesses an all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in R/R AML or mixed phenotype acute leukemias, exhibiting an 88% overall response rate.

Additionally, the INTERCEPT trial explores novel therapies, including revumenib, targeting MRD and early relapse in AML, with preliminary results to be presented at the ASH Annual Meeting. Another trial evaluates revumenib with intensive chemotherapy followed by maintenance treatment in newly diagnosed patients with mNPM1 or KMT2Ar acute leukemias. A Phase 2 trial by Break Through Cancer investigates whether revumenib combined with venetoclax can eliminate MRD in AML patients and extend progression-free survival.

Syndax is also evaluating revumenib in patients with R/R metastatic microsatellite stable (MSS) colorectal cancer (CRC) and enrolling participants in a Phase 1/2 proof-of-concept trial.

Financially, Syndax reported having $399.6 million in cash, cash equivalents, and investments as of September 30, 2024. Research and development expenses for the third quarter increased to $71.0 million, up from $39.1 million the previous year, driven by higher clinical development and pre-commercial manufacturing costs. Selling, general, and administrative expenses rose to $31.1 million from $17.3 million, reflecting enhanced commercial readiness activities.

The company forecasts full-year research and development expenses at $245 to $250 million and total operating expenses at $365 to $370 million. Syndax anticipates reaching profitability with its current cash reserves, the $350 million from the Niktimvo royalty sale, and expected revenue and interest income.

Overall, Syndax Pharmaceuticals is well-positioned to achieve significant milestones in its innovative cancer therapy pipeline, with the potential for substantial market impact in the near future.