Too many loved ones live with uncertainty.
Let’s find answers.
Rare oncology is where rare diseases and cancers meet. While some rare tumors are not malignant, their presence and progression are nonetheless devastating for patients. At SpringWorks, we are relentless in the search for answers because people with these often-overlooked conditions can’t wait.
Let’s innovate for the overlooked
Our late-stage rare oncology programs have the potential to be best-in-class treatments for patients with devastating diseases, including desmoid tumors, neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) and low-grade gliomas.
Clinical trials are underway to evaluate nirogacestat for patients with desmoid tumors and ovarian granulosa cell tumors, and to evaluate mirdametinib for patients with NF1-PN and low-grade gliomas. Our Phase 3 DeFi clinical trial for adults with progressing desmoid tumors and Phase 2b ReNeu clinical trial for children and adults with NF1-PN are fully enrolled. In September 2022, positive data from the Phase 3 DeFi trial were presented at the ESMO Congress 2022 (see the SpringWorks press release).
Our Phase 3 DeFi clinical trial is evaluating nirogacestat as a monotherapy in adults with progressing desmoid tumors. In addition, 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.
Our Phase 2b ReNeu clinical trial is evaluating mirdametinib in children (2 years or older) and in adults with an inoperable NF1-PN.
A Phase 1/2 clinical trial is evaluating mirdametinib for the treatment of children, adolescents, and young adults with low-grade glioma (LGG).