Springworks sees the way to a small market niche

Nirogacestat, a gamma secretase inhibitor Pfizer discarded five years ago, today gave Springworks a pivotal clinical trial win. In the phase 3 DeFi study in desmoid tumours nirogacestat beat placebo on the primary endpoint of progression-free survival, Springworks said, as well as meeting “all key” secondaries, setting the asset up for US filing later this year. Still, desmoid tumours are rare, slow growing and benign, and while Springworks argues that they cause pain and morbidity they likely do not translate into a major market opportunity. Moreover, nirogacestat has just three years’ patent life, and at least in desmoid tumours, which have no approved therapy, doctors might choose to give patients Nexavar off label; the Bayer kidney cancer drug has shown a strong PFS benefit in a small academic-run trial in desmoid tumours. Despite nirogacestat’s big opportunity in multiple myeloma, where six different combo studies with BCMA-targeting agents are under way, investors remained wary of the significant risks here, and sent Springworks down 7% this morning. And Ayala Pharmaceuticals, a micro cap biotech that boasts two gamma secretase inhibitors but lacks cash, slumped 20%.

After this story was published Springworks pointed out that it is clinically studying nirogacestat's polymorphc form, which has composition patent coverage until 2039.