Advancing the Fight Against Rare Diseases

Drug development and commercialization of treatments for rare diseases, regardless of their severity, can be overlooked or unmet by biopharmaceutical companies. The dedicated team at SpringWorks is on a mission to change this dynamic, and provide life-changing medicines for underserved patient populations.

As an example, SpringWorks licensed a molecule from Pfizer, collaborated with academics, clinicians and patients and initiated a Phase IIb study of mirdametinib in patients with Neurofibromatosis type 1 (NF-1)-associated plexiform neurofribromas. This rare genetic condition is characterized by multiple skin alterations and tumoral growth along nerves called neurofibromas. In addition to cancer, NF-1 may lead to blindness, bone abnormalities, learning disabilities and disabling pain.

There are approximately 100,000 patients in the US with NF-1, 20-40% of whom represent the plexiform neurofibroma variant. The FDA recently granted Fast Track designation to one of SpringWorks’ molecules to treat these underserved patients, and the team is highly focused on bringing an approved therapy to market, because they know patients are counting on them.

In September 2019, the company announced a successful initial public offering. With this milestone, SpringWorks is well-capitalized to continue advancing the development of Mirdametinib along with its other clinical programs, in-licensing additional molecules with significant promise, and improving access for rare disease patients with no treatment alternatives.