SpringWorks Therapeutics, a biopharmaceutical company focused on developing treatments for severe rare diseases and cancer, has announced its financial results for the third quarter of 2024, highlighting strong sales of its first commercial product, OGSIVEO (nirogacestat), and advancements in its clinical pipeline.
OGSIVEO Drives Revenue Growth
OGSIVEO, approved by the FDA in November 2023 for adult patients with progressing desmoid tumors requiring systemic treatment, has significantly contributed to the company's revenue. For the three months ended September 30, 2024, total revenue reached $49.3 million, and for the nine months ended September 30, 2024, revenue totaled $130.0 million. This growth underscores the unmet need for effective therapies in this rare tumor type.
"The commercial success of OGSIVEO demonstrates its value to patients with desmoid tumors," said [Name], CEO of SpringWorks Therapeutics. "We are committed to expanding its availability globally and continuing to develop innovative therapies for underserved patient populations."
The FDA approval of OGSIVEO was based on the Phase 3 DeFi trial, which demonstrated a statistically significant improvement in progression-free survival and objective response rate compared to placebo. In April 2024, the FDA approved a supplemental New Drug Application (sNDA) for two higher dosage strengths of OGSIVEO tablets (150 mg and 100 mg) in blister packaging, enhancing patient convenience.
Expanding Global Reach of OGSIVEO
SpringWorks is actively pursuing regulatory approvals to expand OGSIVEO's availability beyond the United States. In February 2024, the European Medicines Agency (EMA) validated a Marketing Authorization Application (MAA) for OGSIVEO, with potential approval and first launches in Germany and France expected in the first half of 2025. This expansion aims to address the needs of desmoid tumor patients in Europe, where treatment options are limited.
Mirdametinib Advances Toward Potential Approval
Mirdametinib, an investigational oral MEK inhibitor, is in development for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). The FDA has granted mirdametinib Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation. In August 2024, the FDA accepted the NDA submission for mirdametinib and granted priority review with a PDUFA target action date of February 28, 2025. The EMA also validated an MAA for mirdametinib for the treatment of NF1-PN.
Pipeline Expansion and R&D Investments
SpringWorks is advancing its clinical-stage efforts in genetically defined metastatic solid tumors, focusing on the MAPK pathway. This includes evaluating mirdametinib in combination with brimarafenib and lifirafenib in various solid tumors. In the first quarter of 2024, MapKure initiated a Phase 1b combination trial of brimarafenib with panitumumab in colorectal and pancreatic cancer patients with known MAPK pathway mutations.
The company is also pursuing several discovery programs, including the TEA Domain (TEAD) inhibitor program. In June 2024, a Phase 1a trial of SW-682, a TEAD inhibitor, was initiated in Hippo mutant solid tumors. These efforts are supported by ongoing investments in R&D infrastructure to support drug discovery and translational medicine activities, aiming to develop transformative targeted medicines for oncology patients.
Financial Position
SpringWorks reported a net loss of $(53.5) million for the three months ended September 30, 2024, and $(180.8) million for the nine months ended September 30, 2024. As of September 30, 2024, SpringWorks had cash, cash equivalents, and marketable securities of $498.1 million. The company estimates that its current liquidity will enable it to meet operating expenses through at least twelve months after the date of the financial statements.
SpringWorks plans to continue its strategic initiatives to expand the reach of OGSIVEO to additional geographies and advance its pipeline of product candidates, including mirdametinib and nirogacestat. The company anticipates increased research and development expenses as it invests in activities related to developing its product candidates and preclinical programs.
