SpringWorks Therapeutics

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.

SpringWorks Therapeutics to present long-term efficacy and safety data of nirogacestat in desmoid tumours at CTOS 2024, showing sustained tumor size reductions, increased response rates, and consistent safety profile.

Long-term nirogacestat treatment in desmoid tumors shows sustained tumor size reductions, increased ORR with new PRs and CRs, improved symptoms, and consistent safety profile over 3 years.

SpringWorks Therapeutics to host Q3 financial results and business updates conference call on Nov 12, 2024, at 8:30 a.m. ET. Webcast and phone participation details available. Replay on the company’s website.

Various researchers report financial relationships with pharmaceutical companies, including Deciphera, during and outside the conduct of the study.

Various drugs and their statuses are listed, including IN-001 for anaphylaxis (Fast Track), Finereonone for heart failure (Phase 3), Tirzepatide for heart failure and obesity (Phase 3), TMB-001 for congenital ichthyosis (Phase 3), Bentracimab for ticagrelor reversal (Priority Review), Fabhalta for immunoglobulin A nephropathy (accelerated approval), RLS-0071 for acute graft-vs-host disease (Fast Track and Orphan Drug), CHIKV VLP for chikungunya (Priority Review), Diazoxide choline for Prader-Willi syndrome (Priority Review), Mirdametinib for neurofibromatosis type 1 (Priority Review), Setmelanotide for genetic obesity (Priority Review), AR882 for gout (Fast Track), SPN-830 for Parkinson disease (NDA review), BGB-16673 for chronic lymphocytic leukemia (Fast Track), Cabozantinib for neuroendocrine tumors (NDA review), Linvoseltamab for multiple myeloma (Complete Response Letter), Nivolumab plus ipilimumab for hepatocellular carcinoma (BLA review), Midomafetamine for PTSD (Complete Response Letter), and Tezepelumab-ekko for COPD (Breakthrough Therapy).

In August 2024, the FDA made several decisions on new therapeutic agents, including approving Indapta Therapeutics’ g-NK cell therapy IDP-023 for progressive multiple sclerosis, Amneal Pharmaceuticals’ carbidopa/levodopa ER capsules IPX203 for Parkinson disease, and Medtronic’s deep brain stimulation technology for use during general anesthesia. The agency also granted fast track designation to Abata Therapeutics’ Treg therapy ABA-101 for progressive MS and [18F]PI-2620 tau-PET diagnostic for neurodegenerative diseases. Additionally, the FDA cleared Clearmind Biomedical’s Neuroblade System for minimally invasive neurosurgery and granted priority review to SpringWorks Therapeutics’ mirdametinib for neurofibromatosis type 1-associated plexiform neurofibromas.

Mirdametinib, under development by SpringWorks Therapeutics, targets various cancers and tumors, with expected US revenue of $450 mn by 2034. The risk-adjusted NPV (rNPV) model, accounting for R&D risks, is used for valuation. SpringWorks reported FY2023 revenues of $5.5 mn and a net loss of $325.1 mn.