SpringWorks Therapeutics is evaluating the safety and efficacy of our investigational therapies, both alone and in combination with other therapies, for patients with rare diseases and cancer.
For more information about ongoing trials, follow the links below to the study listings on clinicaltrials.gov. Medical professionals may also email us at email@example.com for more information about study participation. Patients and families should speak to their physician to ask about whether a study is right for them.
Rare Oncology Trials
SpringWorks is conducting a Phase 3 trial for patients with desmoid tumors, which are rare and debilitating soft-tissue tumors that can cause severe pain and disfigurement.
The DeFi trial is evaluating nirogacestat, an oral, selective, small molecule gamma-secretase inhibitor as a monotherapy in adult patients with progressing desmoid tumors.
Under a collaboration with Children’s Oncology Group (COG), a Phase 2 clinical trial is evaluating nirogacestat as a monotherapy in children and adolescents with progressive, surgically unresectable desmoid tumors.
Neurofibromatosis Type 1-Associated Plexiform Neurofibromas
SpringWorks is enrolling patients in a Phase 2b trial for patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN), which are rare and devastating tumors that can cause severe pain, disfigurement and functional impairment.
The ReNeu trial is evaluating mirdametinib, an oral small molecule designed to inhibit MEK1 and MEK2, in patients two years of age and older with an inoperable NF1-PN causing significant morbidity.
Under a collaboration with the St. Jude Children’s Research Hospital, a Phase 1/2 clinical trial is evaluating mirdametinib in children, adolescents, and young adults with low-grade glioma.
B-cell Maturation Antigen (BCMA) Collaborations in Multiple Myeloma Trials
Nirogacestat + BLENREP
Under a global clinical trial agreement with GlaxoSmithKline, a Phase 1b clinical trial is evaluating the safety, tolerability and preliminary efficacy of nirogacestat in combination with BLENREP (belantamab mafodotin-blmf), an anti-BCMA antibody-drug conjugate (ADC), in patients with relapsed or refractory multiple myeloma. This combination is part of DREAMM-5, a platform trial being conducted by GlaxoSmithKline that is evaluating multiple belantamab mafodotin-containing combinations in separate sub-studies.
Nirogacestat + Teclistamab
Under a clinical collaboration and supply agreement with Janssen Biotech, Inc., a Phase 1b clinical trial is evaluating the safety, tolerability and preliminary efficacy of nirogacestat in combination with teclistamab, an investigational bispecific antibody targeting BCMA and CD3, in patients with relapsed or refractory multiple myeloma. This combination is part of a multi-arm trial being conducted by Janssen Research & Development, LLC that is evaluating teclistamab with other anticancer therapies in patients with multiple myeloma.
Nirogacestat + ALLO-715
Under a clinical trial collaboration agreement with Allogene Therapeutics, a Phase 1 clinical trial is evaluating the safety, tolerability and preliminary efficacy of nirogacestat in combination with ALLO-715, Allogene’s investigational BCMA AlloCAR TTM therapy, in patients with relapsed or refractory multiple myeloma.
Biomarker Defined Metastatic Solid Tumor Trials
Advanced or Refractory Solid Tumors that Harbor RAS Mutations, RAF Mutations or Other MAPK Pathway Aberrations
Patients with advanced or refractory solid tumors that harbor RAS mutations, RAF mutations and other MAPK pathway aberrations are being enrolled in a Phase 1b trial.
The study is evaluating the safety, tolerability and preliminary efficacy of combining BeiGene’s investigational RAF dimer inhibitor, lifirafenib (BGB-283) and SpringWorks Therapeutics’ investigational MEK inhibitor, mirdametinib, in patients with advanced solid tumors.
The trial is being administered by BeiGene under a global clinical collaboration agreement between SpringWorks Therapeutics and BeiGene.
Advanced or Refractory Solid Tumors that Harbor Specific B-RAF Driver Mutations and Fusions
Patients with advanced or refractory solid tumors harboring specific B-RAF driver mutations and fusions that are likely to respond to a RAF dimer inhibitor, as well as patients who have developed resistance to first-generation B-RAF V600 inhibitors, are being enrolled in a Phase 1 trial.
The study is evaluating the safety, pharmacokinetics and antitumor activity of BGB-3245, an investigational, selective, next-generation B-RAF inhibitor, in this patient population.
BGB-3245 is being advanced by MapKure, an entity jointly owned by SpringWorks and BeiGene.