Syndax Pharmaceuticals Q1 2024 Financial Results and Business Update

Syndax Pharmaceuticals, a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, announced its financial results for the first quarter ending March 31, 2024, along with a significant business update. The company, which focuses on developing innovative cancer therapies, is poised for a transformative year with potential approvals for its lead candidates, revumenib and axatilimab.

Revumenib, a selective menin inhibitor aimed at treating relapsed or refractory KMT2A-rearranged acute leukemia, has seen notable progress. In March 2024, the FDA granted Priority Review status for its New Drug Application (NDA) under the Real-Time Oncology Review Program (RTOR), setting a Prescription Drug User Fee Act (PDUFA) target action date for September 26, 2024. The RTOR program facilitates a more efficient review process, potentially leading to earlier approvals. Additionally, enrollment for the pivotal AUGMENT-101 trial in patients with relapsed or refractory mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML) was completed, with topline data expected in the fourth quarter of 2024. These findings could support a supplemental NDA (sNDA) filing in the first half of 2025.

Further advancements include several Phase 1 combination trials of revumenib, such as BEAT AML, SAVE, and AUGMENT-102, focusing on different combination treatments for mNPM1 and KMT2Ar acute leukemias. A Phase 1 trial combining revumenib with standard chemotherapy for newly diagnosed patients was also initiated. The company plans to start a pivotal trial combining revumenib with venetoclax and azacitidine for newly diagnosed patients by the end of 2024.

Axatilimab, an anti-CSF-1R antibody targeting chronic graft-versus-host disease (GVHD), also marked a significant milestone. The FDA accepted its Biologics License Application (BLA) and granted it Priority Review with a PDUFA action date of August 28, 2024. Axatilimab is being evaluated in a Phase 2 trial for idiopathic pulmonary fibrosis (IPF) and is set to be tested in combination trials with ruxolitinib and steroids in 2024 through a partnership with Incyte.

The company experienced substantial financial growth, reporting a net loss attributable to common stockholders of $72.4 million for the first quarter of 2024, compared to a $41.1 million loss in the same period the previous year. This increase was driven by heightened research and development costs, notably due to clinical development, manufacturing expenses, and employee-related costs, as well as commercialization activities for revumenib and axatilimab.

Syndax's Chief Executive Officer, Michael A. Metzger, emphasized the company's readiness to transition into a commercial organization, leveraging its expert team to ensure the successful launch of its therapies. The company appointed Steven Closter as Chief Commercial Officer, who brings over 30 years of commercial experience in the biopharmaceutical industry.

As for financial guidance, Syndax projects research and development expenses between $50 to $55 million for the second quarter of 2024, with total operating expenses ranging from $80 to $85 million. For the full year, research and development expenses are expected to be between $240 to $260 million, with total operating expenses between $355 to $375 million, including non-cash stock compensation expenses estimated at $43 million. The company believes it has sufficient funds to support its operations through 2026.

In summary, Syndax Pharmaceuticals is on the cusp of significant breakthroughs with its leading candidates, revumenib and axatilimab, potentially hitting the market soon. The company remains focused on advancing its pipeline and transitioning into a commercial entity, aiming to deliver innovative cancer therapies to patients in need.