OGSIVEO is the first and only therapy to receive marketing authorization in the EU for the treatment of desmoid tumorsSTAMFORD, Conn., Aug. 18, 2025 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany, announced today that the European Commission (EC) granted marketing authorization for OGSIVEO® (nirogacestat), an oral gamma secretase inhibitor, as monotherapy for the treatment of adults with progressing desmoid tumors who require systemic treatment. OG ...

Core Insights - The European Commission granted conditional marketing authorization for EZMEKLY (mirdametinib) for treating symptomatic, inoperable plexiform neurofibromas (PN) in patients aged 2 years and older with neurofibromatosis type 1 (NF1), making it the first therapy approved in the EU for both adults and children with NF1-PN [1][11]. Company Overview - SpringWorks Therapeutics, a healthcare company of Merck, focuses on developing treatments for rare tumors and has commercialized the first FDA-approved medicine for adults with desmoid tumors and the first approved medicine for both adults and children with NF1-PN [19]. - The company emphasizes addressing significant unmet needs and improving outcomes for patients and their families [3]. Clinical Trial Insights - The approval of EZMEKLY is based on the Phase 2b ReNeu trial, which enrolled 114 patients (58 adults and 56 pediatric patients) and demonstrated an objective response rate (ORR) of 41% in adults and 52% in children [3][6]. - The trial showed a median best percentage change in target PN volume of -41% in adults and -42% in children, with significant improvements in pain and quality of life reported [3][6]. Treatment Profile - EZMEKLY is available in 1 mg and 2 mg capsules, as well as a 1 mg dispersible tablet that dissolves easily in water, catering to patients who have difficulty swallowing pills [5]. - The treatment has a manageable safety profile, with common adverse reactions including dermatitis acneiform (83% in adults), diarrhea (55%), and nausea (55%) [4]. Disease Context - NF1 affects approximately 3 in 10,000 people in the EU, equating to an estimated 135,000 individuals, with a lifetime risk of developing plexiform neurofibromas ranging from 30% to 50% [2][7]. - Plexiform neurofibromas can lead to severe disfigurement, pain, and functional impairment, and are often not amenable to surgical resection due to their infiltrative growth pattern [8][9].

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 Insights - Nirogacestat is poised to become the first and only approved therapy in the EU for desmoid tumors if the European Commission grants marketing authorization, with a decision expected in Q3 2025 [1][2] Company Overview - SpringWorks Therapeutics, Inc. is a biopharmaceutical company focused on severe rare diseases and cancer, currently advancing nirogacestat for desmoid tumors [2][10] - The company has previously received Orphan Drug designation for nirogacestat from the European Commission [3] Clinical Trial Results - The positive opinion from the EMA's CHMP is based on the Phase 3 DeFi trial, which demonstrated a 71% lower risk of disease progression for nirogacestat compared to placebo [3][6] - The DeFi trial involved 142 patients, with nirogacestat showing significant improvements in progression-free survival, objective response rate, and patient-reported outcomes [6] Safety Profile - Nirogacestat has a manageable safety and tolerability profile, with common adverse reactions including diarrhea, nausea, and fatigue [4] Disease Context - Desmoid tumors are rare, aggressive tumors with an estimated 1,300-2,300 new cases diagnosed annually in the EU, predominantly affecting individuals aged 20 to 44 [7][8] - Despite not metastasizing, desmoid tumors have high recurrence rates, leading to a shift in treatment guidelines recommending systemic therapies as first-line interventions [8]

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]

Company Performance - SpringWorks Therapeutics reported a quarterly loss of $1.11 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.81, representing an earnings surprise of -37.04% [1] - The company posted revenues of $49.09 million for the quarter ended March 2025, missing the Zacks Consensus Estimate by 23.53%, compared to year-ago revenues of $21.01 million [2] - Over the last four quarters, SpringWorks has surpassed consensus EPS estimates two times and topped consensus revenue estimates two times [2] Stock Movement and Outlook - SpringWorks Therapeutics shares have increased by approximately 27.9% since the beginning of the year, contrasting with the S&P 500's decline of -3.7% [3] - The current consensus EPS estimate for the upcoming quarter is -$0.69 on revenues of $73.21 million, and for the current fiscal year, it is -$2.30 on revenues of $330.84 million [7] Industry Context - The Medical - Biomedical and Genetics industry, to which SpringWorks belongs, is currently in the top 32% of over 250 Zacks industries, indicating a favorable outlook [8] - Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions, suggesting that the industry outlook can significantly impact stock performance [5][8]

Revenue and Product Performance - SpringWorks Therapeutics recorded net product revenue of $44.1 million from sales of OGSIVEO in the United States for the three months ended March 31, 2025[76]. - GOMEKLI, approved on February 11, 2025, generated net product revenue of $4.9 million in the United States for the three months ended March 31, 2025[83]. - For the three months ended March 31, 2025, the company recorded net product revenue of $49.1 million, a 134% increase from $21.0 million in the same period of 2024[103]. - The FDA approved OGSIVEO in November 2023, generating $44.1 million in net product revenue for the three months ended March 31, 2025[104]. - The FDA approved GOMEKLI in February 2025, contributing $4.9 million in net product revenue for the three months ended March 31, 2025[105]. Clinical Trials and Approvals - The FDA approved OGSIVEO on November 27, 2023, based on a Phase 3 trial showing a 71% reduction in the risk of disease progression compared to placebo[72]. - The Phase 2b ReNeu trial for GOMEKLI showed a 52% objective response rate in pediatric patients and 41% in adult patients[80]. - The company is evaluating nirogacestat in combination with BCMA-directed therapies for multiple myeloma through non-exclusive clinical collaborations[87]. - SpringWorks is also advancing mirdametinib for genetically defined metastatic solid tumors targeting the MAPK pathway[88]. Financial Performance - Operating costs and expenses totaled $129.6 million for the three months ended March 31, 2025, a 13% increase from $114.9 million in the same period of 2024[103]. - Research and development expenses decreased by 8% to $49.6 million for the three months ended March 31, 2025, down from $53.6 million in the same period of 2024[109]. - Selling, general and administrative expenses increased by 27% to $76.5 million for the three months ended March 31, 2025, compared to $60.1 million in the same period of 2024[107]. - The net loss for the three months ended March 31, 2025, was $83.2 million, a slight improvement from a net loss of $87.4 million in the same period of 2024[114]. - The accumulated deficit stood at $1.2 billion as of March 31, 2025, unchanged from December 31, 2024[114]. Merger and Corporate Strategy - The company entered into a merger agreement with Merck KGaA, with each share of common stock valued at $47.00[68]. - A termination fee of $145.6 million is required if the merger agreement is terminated under specific circumstances[69]. - The company entered into a Merger Agreement on April 27, 2025, which includes various covenants and a potential termination fee of $145.6 million[113]. Cash Flow and Funding - Net cash used in operating activities was $68.8 million for the three months ended March 31, 2025, compared to $78.9 million for the same period in 2024, reflecting a decrease of 14.1%[117]. - Net cash provided by investing activities was $73.4 million for the three months ended March 31, 2025, driven by the sale and maturities of available-for-sale debt securities, compared to $103.9 million in 2024[118]. - Net cash used in financing activities was $10.6 million for the three months ended March 31, 2025, primarily due to stock repurchases of $12.9 million, partially offset by proceeds from stock option exercises of $2.3 million[119]. - Cash and cash equivalents at the end of the period were $64.4 million as of March 31, 2025, down from $118.6 million at the end of the same period in 2024, representing a decrease of 45.6%[116]. - Future funding requirements will depend on costs associated with the commercialization of OGSIVEO and GOMEKLI, as well as the progress and costs of clinical trials for product candidates[121]. - The company expects to finance operations through a combination of equity offerings, debt financings, and collaborations, which may dilute current ownership interests[123]. Market and Economic Conditions - The company does not believe inflation has had a material impact on its business or financial condition during the reported periods[124]. - The company is unable to estimate increased capital outlays and operating expenditures associated with current and anticipated clinical studies due to numerous risks and uncertainties[122]. - There were no material changes to market risks from those described in the previous Form 10-K[128]. - There were no material changes to contractual obligations during the three months ended March 31, 2025, compared to previous disclosures[125].

Group 1 - Monteverde & Associates PC is recognized as a Top 50 Firm in the 2024 ISS Securities Class Action Services Report and has recovered millions of dollars for shareholders [1] - The firm is investigating SpringWorks Therapeutics, Inc. regarding its proposed merger with Merck KGaA, where SpringWorks shareholders will receive $47.00 in cash per share [1] - The firm operates from the Empire State Building in New York City and has a successful track record in trial and appellate courts, including the U.S. Supreme Court [2][3] Group 2 - Monteverde & Associates PC emphasizes that no company, director, or officer is above the law and offers free information to shareholders with concerns [3] - The firm encourages potential clients to inquire about their litigation history and past recoveries for shareholders [4]

Core Viewpoint - Rowley Law PLLC is investigating potential securities law violations by SpringWorks Therapeutics, Inc. related to its proposed acquisition by Merck KGaA, which is valued at approximately $3.4 billion [1]. Group 1 - The proposed acquisition will provide stockholders of SpringWorks Therapeutics with $47.00 for each share they hold [1]. - The transaction is expected to close in the second half of 2025 [1].