SpringWorks Therapeutics

Pharmaceutical M&A activity is experiencing a significant rebound with major deals including Novartis's $1.7 billion acquisition of Regulus Therapeutics and Merck KGaA's $3.9 billion purchase of SpringWorks Therapeutics.

• CG Oncology's cretostimogene grenadenorepvec demonstrated a 75.5% complete response rate in high-risk BCG-unresponsive non-muscle invasive bladder cancer patients, with 34% maintaining complete response at 24 months. • The FDA has granted Fast Track and Breakthrough Therapy designations to cretostimogene, potentially expediting its regulatory pathway for a disease with limited treatment options. • The drug's 27.9-month median duration of response may provide a competitive edge over Johnson & Johnson's TAR-200, positioning CG Oncology as a significant player in the bladder cancer treatment landscape.

German pharmaceutical giant Merck KGaA has agreed to acquire Connecticut-based SpringWorks Therapeutics for $3.9 billion, paying $47 per share in cash, representing a 26% premium over SpringWorks' unaffected 20-day average price.

SpringWorks Therapeutics expects the European Medicines Agency's CHMP to issue an opinion on nirogacestat for adult desmoid tumors in Q2 2025, potentially expanding access beyond its current FDA approval.

• SpringWorks Therapeutics' drug Ogsiveo is under review by the European Medicines Agency's CHMP, with analysts anticipating a positive opinion to be announced Friday. • Evercore ISI maintains an "Outperform" rating with a $65 price target on SpringWorks, describing it as an "emerging multi-product story" with increasing strategic value. • Geographic expansion into Europe would significantly enhance SpringWorks' market position and appeal to European pharmaceutical companies, according to analyst Cory Kasimov.

Long-term treatment with nirogacestat (Ogsiveo) demonstrated improved objective response rates reaching 45.7% at 4 years compared to 34.3% at 1 year in patients with desmoid tumors, according to updated DeFi trial data.

• SpringWorks Therapeutics recently secured FDA approval for Gomekli (mirdametinib), a drug once abandoned by Pfizer, providing the first treatment option for adults with neurofibromatosis type 1. • The Children's Tumor Foundation played a crucial role in rescuing the drug from Pfizer's intellectual property vault, demonstrating how patient advocacy groups can bridge the gap between academia and industry for rare disease treatments. • Experts estimate that approximately 30 more potential treatments for neurofibromatosis alone may be sitting idle in pharmaceutical companies' portfolios, highlighting a significant opportunity to replicate this model across rare diseases.

Merck KGaA confirms advanced acquisition discussions with SpringWorks Therapeutics, though critical conditions remain unmet and no binding agreement has been reached.

• SpringWorks Therapeutics projects OGSIVEO revenue to reach $61.5 million for Q4 and aims for EU regulatory approval in mid-2025, starting with Germany. • The company anticipates FDA approval for mirdametinib in NF1-associated plexiform neurofibromas by February 28, 2025, with EU approval and launch also planned for 2025. • Initial data from a Phase 2 trial of nirogacestat (OGSIVEO) in ovarian granulosa cell tumors is expected in the first half of 2025. • SpringWorks will continue advancing its oncology pipeline, including trials for SW-682 in Hippo-mutant solid tumors and plans to file an IND for SW-3431 by the end of 2025.

• The FDA is set to decide on Biogen and Eisai's Leqembi for monthly intravenous maintenance in early Alzheimer's disease, potentially improving patient convenience. • AstraZeneca and Daiichi Sankyo await a decision on Dato-DXd for metastatic HR-positive, HER2-negative breast cancer, offering a new antibody-drug conjugate approach. • Vertex's suzetrigine, a non-opioid analgesic for moderate-to-severe acute pain, anticipates FDA verdict, representing a novel drug class for pain management. • SpringWorks' mirdametinib is under priority review for neurofibromatosis type 1-associated plexiform neurofibromas, addressing a significant unmet need.