Key Highlights of SpringWorks Therapeutics - OGSIVEO (nirogacestat) is the first and only FDA-approved therapy for desmoid tumors[15] - GOMEKLI (mirdametinib) is the first and only FDA-approved therapy for both adults and children with NF1-PN[15] - SpringWorks Therapeutics expects profitability in the first half of 2026[10] - SpringWorks Therapeutics had $461.9 million in cash, cash equivalents, and marketable securities[132] OGSIVEO (Nirogacestat) for Desmoid Tumors - OGSIVEO generated $61.5 million in net revenue in Q4 2024 and $172.0 million in FY 2024[15] - Between October 2023 and October 2024, approximately 11,000 patients with desmoid tumor ICD-10 claims were identified[15] - Objective response rate (ORR) increased from 34.3% with up to 1 year of treatment to 45.7% with up to 4 years of treatment[48] GOMEKLI (Mirdametinib) for NF1-PN - GOMEKLI is approved for the treatment of adult and pediatric patients 2 years of age and older with NF1-PN[10] - The U S market includes approximately 30,000 adult and 10,000 pediatric patients with NF1-PN[15] - In clinical trials, GOMEKLI showed a confirmed overall response rate of 41% in adults (n=58) and 52% in pediatrics (n=56)[67]

Core Viewpoint - SpringWorks Therapeutics has received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use for its MEK inhibitor, mirdametinib, aimed at treating a rare genetic disorder [1][2]. Company Developments - The CHMP has recommended conditional marketing authorization for mirdametinib to treat symptomatic, inoperable plexiform neurofibromas in patients aged two years and older with neurofibromatosis type 1 [2]. - If approved, mirdametinib will be the first therapy authorized for treating NF1-PN in the European Union [3]. - The FDA approved mirdametinib under the brand name Gomekli in February 2025, generating sales of $4.9 million in the U.S. during Q1 2025 [3]. Financial Performance - Year-to-date, SpringWorks shares have increased by 28.6%, contrasting with a 5.5% decline in the industry [5]. - Merck KGaA announced plans to acquire SpringWorks for $47 per share, totaling approximately $3.9 billion, with the transaction expected to close in the second half of 2025 [6]. Product Pipeline - Ogsiveo, an oral gamma-secretase inhibitor, was approved in the U.S. for treating desmoid tumors in November 2023 and generated $172 million in sales in 2024, with $44.1 million in Q1 2025 [7][8]. - Ongoing label expansion studies for Ogsiveo are targeting various cancer indications, with the EMA's opinion on its MAA expected in Q2 2025 [8]. Market Position - The acquisition by Merck KGaA is anticipated to enhance SpringWorks' ability to reach global markets and support patients with rare tumors [9].

Company Overview - SpringWorks Therapeutics, Inc. is a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, with a commitment to improving the lives of patients [7] - The company has developed the first and only FDA-approved medicine for both adults and children with neurofibromatosis type 1 associated plexiform neurofibromas (NF1-PN) [7] Product Development - Mirdametinib, a MEK inhibitor, has received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) for conditional marketing authorization in the EU for treating symptomatic, inoperable plexiform neurofibromas in patients aged 2 years and older with NF1 [1][2] - The European Commission is expected to make a final decision regarding mirdametinib's approval in the third quarter of 2025 [1] Clinical Trial Results - The positive opinion was based on the Marketing Authorization Application (MAA) validated by the EMA in August 2024, which included results from the Phase 2b ReNeu trial involving 114 patients [2][4] - The trial demonstrated a confirmed objective response rate (ORR) of 41% in adults and 52% in children, with significant reductions in target tumor volume [2][4] - Among patients with confirmed responses, 88% of adults and 90% of children had responses lasting at least 12 months [2] Patient Demographics and Disease Impact - Neurofibromatosis type 1 (NF1) affects approximately 3 in 10,000 people in the EU, translating to an estimated 135,000 individuals [2][5] - Patients with NF1 have a 30% to 50% lifetime risk of developing plexiform neurofibromas, which can lead to severe disfigurement, pain, and functional impairment [2][5] Safety Profile - Mirdametinib has shown a manageable safety and tolerability profile, with common adverse events in adults including rash, diarrhea, and nausea, while children experienced similar side effects along with additional issues like left ventricular dysfunction [2][6]