Mirdametinib

Jonathan Faison highlights Tarsus Pharmaceuticals and SpringWorks Therapeutics as promising biotech investments. Tarsus, with its drug XDEMVY for Demodex blepharitis, shows strong launch metrics. SpringWorks, his top holding, has Ogsiveo for desmoid tumors and Mirdametinib for NF1-PN, with potential for significant market growth.

Mirdametinib, a MEK1/2 inhibitor, showed significant improvements in health-related quality of life (HRQOL) in adult and pediatric patients with NF1-PN in the ReNeu trial, with sustained improvements in physical, emotional, social, and school/work functioning. The trial met its primary endpoint of overall response rate (ORR) and demonstrated a manageable safety profile.

No specific baseline characteristics (age, sex, demographics) predicted deep response to mirdametinib in NF1-PN patients; longer treatment duration correlated with deeper responses. Mirdametinib demonstrated manageable safety, deep responses, and QOL improvements, granted FDA priority review.

46% of NF1 patients responded to mirdametinib, with 53 of 114 achieving ≥20% PN volume reduction. Responses included 24 adults and 29 children, with reductions up to 80%. Treatment was well-tolerated, with dermatitis acneiform and diarrhea as common TRAEs. FDA approval expected in early 2025.

Mirdametinib, a MEK1/2 inhibitor, showed significant tumor volume reductions, pain, and QOL improvements in NF1-PN patients in the ReNeu trial, with 41% ORR in adults and 52% in children, highlighting its potential as a new treatment option.

SpringWorks Therapeutics reports $49.3 million in OGSIVEO® (nirogacestat) net product revenue in Q3, with long-term Phase 3 DeFi trial data showing tumor size reduction and consistent safety. The FDA granted Priority Review for mirdametinib's NDA, and SpringWorks has $498 million in cash to fund operations through anticipated profitability in H1 2026.

SpringWorks Therapeutics reported $49.3M revenue for Q3 2024, driven by OGSIVEO sales, with a net loss of $53.5M. The company launched OGSIVEO in 2023, expanded dosage strengths in 2024, and seeks EMA approval for Europe in 2025. Mirdametinib for NF1-PN is under FDA priority review. SpringWorks plans to invest in R&D and expand OGSIVEO globally, despite facing regulatory, operational, and financial risks.

SpringWorks Therapeutics announced positive mirdametinib data from its ReNeu trial, showing deep and sustained tumor volume reductions in NF1-PN patients, with 62% of adults and 52% of children achieving deep response. Quality of life improvements were clinically meaningful and sustained. A Phase 1/2 study in pediatric low-grade glioma showed a 63% objective response rate. The FDA granted Priority Review for mirdametinib's NDA with a decision date of February 28, 2025.

Mirdametinib showed significant confirmed objective response rates and manageable safety profile in NF1-PN patients, achieving deep and durable tumor volume reductions and early, sustained improvements in pain and quality of life. New Drug and Marketing Authorization Applications are under review, with a U.S. PDUFA action date set for February 28, 2025.

SpringWorks Therapeutics published Phase 2b ReNeu trial results for mirdametinib in the Journal of Clinical Oncology, showing significant confirmed objective response rates of 41% in adults and 52% in children with NF1-PN, deep and durable tumor volume reductions, and manageable safety profile. FDA PDUFA action date is February 28, 2025.