SpringWorks Therapeutics Reports Strong OGSIVEO Growth and Mirdametinib Regulatory Progress
核心洞察
SpringWorks Therapeutics reported $49.3 million in OGSIVEO® (nirogacestat) net product revenue for the third quarter of 2024, marking a 23% increase over the previous quarter.
Long-term data from the Phase 3 DeFi trial showed further tumor size reduction and symptom improvement with nirogacestat in desmoid tumors (搜索).
The FDA granted Priority Review to the NDA for mirdametinib for Neurofibromatosis Type 1-associated Plexiform Neurofibromas (搜索) (NF1-PN (搜索)), with a PDUFA date of February 28, 2025.
SpringWorks Therapeutics, Inc. (Nasdaq: SWTX) announced its third-quarter 2024 financial results, highlighting the continued growth of OGSIVEO® (nirogacestat) and regulatory advancements for mirdametinib. The company is focused on developing and commercializing therapies for severe rare diseases and cancer.
OGSIVEO Performance and Desmoid Tumor Data
OGSIVEO, approved for adult patients with progressing desmoid tumors (搜索), generated $49.3 million in net product revenue during the third quarter, a 23% increase from the second quarter. As of September, over 800 unique patients filled an OGSIVEO prescription, and approximately 420 treatment centers have ordered the drug since its approval. SpringWorks is transitioning to blister packages for OGSIVEO, expected to be completed by the end of the year.
Long-term follow-up data from the Phase 3 DeFi trial, with a data cutoff in August 2024, demonstrated that extended treatment with nirogacestat (median duration: 34 months) led to further reductions in tumor size, an increased objective response rate, sustained improvements in desmoid tumor symptoms (including pain), and a consistent safety profile compared to the initial results from April 2022.
Mirdametinib Regulatory Updates and NF1-PN
The FDA granted Priority Review to SpringWorks' New Drug Application (NDA) for mirdametinib for the treatment of children and adults with Neurofibromatosis Type 1-associated Plexiform Neurofibromas (搜索) (NF1-PN (搜索)), setting a Prescription Drug User Fee Act (PDUFA) action date of February 28, 2025. The European Medicines Agency (EMA) has also validated the Marketing Authorization Application (MAA) for mirdametinib, which is currently under review.
Data from the Phase 2b ReNeu trial, published in the Journal of Clinical Oncology, showed that tumor volume reductions were achieved regardless of age, sex, baseline tumor volume, tumor location, or progression status. Additional data presented at the 2024 Society for Neuro-Oncology (SNO) meeting indicated that patients with deep responses had a trend of longer treatment duration and clinically meaningful improvements in health-related quality of life.
Emerging Pipeline and Financial Position
SpringWorks is advancing its emerging pipeline, including brimarafenib (BGB-3245), a selective RAF (搜索) dimer inhibitor, currently in a Phase 1b trial for adult patients with RAF mutant solid tumors. Initial data from the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with recurrent ovarian granulosa cell tumors (搜索) is expected in the first half of 2025.
As of September 30, 2024, SpringWorks reported $498.1 million in cash, cash equivalents, and marketable securities. The company anticipates this will fund operations through profitability, expected in the first half of 2026.
Saqib Islam, Chief Executive Officer of SpringWorks, stated, "Our focus for the remainder of the year is to continue building on OGSIVEO’s momentum in the U.S. while working to also bring it to patients in Europe, to advance our commercial preparations for mirdametinib in anticipation of serving patients with NF1-PN (搜索) in the U.S. and Europe, and to progress our emerging portfolio."