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.

Under a global clinical trial agreement with GlaxoSmithKline, a Phase 1b clinical trial is planned to evaluate nirogacestat in combination with belantamab mafodotin, an anti-B-cell maturation antigen (BCMA) antibody-drug conjugate (ADC), 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, and for which there are no FDA-approved therapies. 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.