Syndax Highlights Revumenib and Niktimvo Progress in Q3 2024 Update
核心洞察
Syndax's revumenib NDA for R/R KMT2Ar acute leukemia (搜索) is under FDA review with a PDUFA date of December 26, 2024, potentially launching the drug by year-end.
Topline data from the AUGMENT-101 trial of revumenib in R/R mNPM1 AML (搜索) is expected in Q4 2024, with a possible sNDA filing in H1 2025.
Niktimvo (axatilimab-csfr) has been approved by the FDA for chronic GVHD and is expected to launch in early Q1 2025, co-commercialized with Incyte.
Syndax Pharmaceuticals reported its Q3 2024 financial results and provided a business update, highlighting significant progress with its pipeline, particularly revumenib and Niktimvo. The company is poised for potential FDA approval and U.S. launch of revumenib for relapsed or refractory (R/R) KMT2A (搜索)-rearranged (KMT2Ar) acute leukemia, while Niktimvo has already secured FDA approval for chronic graft-versus-host disease (搜索) (GVHD).
Revumenib Advances in Acute Leukemia
The New Drug Application (NDA) for revumenib, an oral menin inhibitor, is under Priority Review by the FDA for the treatment of adult and pediatric R/R KMT2Ar acute leukemia (搜索). The Prescription Drug User Fee Act (PDUFA) target action date is set for December 26, 2024. Furthermore, Syndax anticipates topline data from the AUGMENT-101 pivotal trial cohort of patients with R/R mutant nucleophosmin (mNPM1) acute myeloid leukemia (搜索) (AML) in the fourth quarter of 2024. Positive data could support a supplemental NDA (sNDA) filing for revumenib in R/R mNPM1 AML (搜索) in the first half of 2025.
Data from the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib in adult and pediatric patients with R/R KMT2Ar AML and acute lymphoid leukemia (搜索) (ALL) have been published in the Journal of Clinical Oncology. A larger data set and longer follow-up from the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib in R/R KMT2Ar acute leukemia (搜索) will be presented at the upcoming 66th American Society of Hematology (ASH) Annual Meeting. The updated analysis shows that revumenib provides durable responses and robust rates of overall response, minimal residual disease (MRD) negativity, and hematopoietic stem cell transplantation (HSTC). With seven months of additional follow-up, the median duration of CR/CRh extended to 13 months among the 13 CR/CRh responders included in the interim analysis presented at ASH 2023.
Multiple trials are underway to evaluate the potential of revumenib across the mNPM1 and KMT2Ar acute leukemia (搜索) treatment landscape, including combinations with venetoclax and azacitidine in front-line AML patients (BEAT AML trial) and with venetoclax and decitabine/cedazuridine in R/R AML or mixed phenotype acute leukemias (SAVE trial).
Niktimvo Approved for Chronic GVHD
Niktimvo (axatilimab-csfr) received FDA approval for the treatment of chronic graft-versus-host disease (搜索) (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. The company anticipates launching Niktimvo in the U.S. no later than early first quarter 2025. In the U.S., Niktimvo will be co-commercialized by Syndax and Incyte.
Niktimvo was added to the latest NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) as a category 2A recommendation for the treatment of GVHD after the failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. Results from the pivotal Phase 2 AGAVE-201 trial of Niktimvo in adult and pediatric patients with recurrent/refractory active chronic GVHD who had received at least two prior lines of systemic therapy were published in the New England Journal of Medicine.
A secondary analysis of overall and organ-specific responses from the pivotal Phase 2 AGAVE-201 trial of Niktimvo will be presented at the 66th ASH Annual Meeting, demonstrating rapid responses and symptom improvement in inflammatory and fibrotic manifestations of chronic GVHD in heavily pretreated patients.
Financial Position and Outlook
Syndax reported cash, cash equivalents, and short- and long-term investments of $399.6 million as of September 30, 2024. A $350 million royalty funding agreement with Royalty Pharma, based on U.S. net sales of Niktimvo, is expected to fund the company through profitability. For the full year 2024, Syndax expects research and development expenses to be $245 to $250 million and total operating expenses to be $365 to $370 million.