Desmoid tumors are rare and often debilitating and disfiguring soft tissue tumors for which there are currently no FDA-approved therapies. Nirogacestat is in a Phase 3 clinical trial (“DeFi”) for patients with desmoid tumors and has received Orphan Drug Designation, Fast Track and Breakthrough Therapy Designations for this indication. Learn more about nirogacestat.

In collaboration with Children’s Oncology Group (COG), a Phase 2 clinical trial is evaluating nirogacestat in children and adolescents with progressive, surgically unresectable desmoid tumors.

Under a global clinical trial agreement with GlaxoSmithKline, a Phase 1b clinical trial is evaluating nirogacestat in combination with BLENREP (belantamab mafodotin-blmf), an anti-B-cell maturation antigen (BCMA) antibody-drug conjugate (ADC), in patients with relapsed or refractory multiple myeloma.

Under a clinical trial agreement with Pfizer, a Phase 1b/2 clinical trial is planned to evaluate nirogacestat in combination with PF‐06863135, an investigational BCMA CD3 bispecific antibody, in patients with relapsed or refractory multiple myeloma.

Neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) is a rare tumor of the peripheral nerve sheath that causes significant pain and disfigurement. NF1-PN most often manifests in children. Mirdametinib is in a Phase 2b trial (“ReNeu”) for children and adults with inoperable NF1-PN, and has received Orphan Drug and Fast Track Designations for this indication. Learn more about mirdametinib.

BGB-3245 is a novel oral, selective small molecule inhibitor of monomer and dimer forms of activating B-RAF mutations including V600 B-RAF mutations, non-V600 B-RAF mutations and RAF fusions. BGB-3245 is being advanced by MapKure, an entity jointly owned by SpringWorks and BeiGene. A Phase 1 clinical trial is evaluating BGB-3245 in patients with advanced or refractory solid tumors including those with B-RAF driver mutations and fusions that are likely to respond to a RAF dimer inhibitor.