SpringWorks gets its foot in the door with niche approval

SpringWorks got its first approval yesterday, with nirogacestat receiving the nod in adults with desmoid tumours following a three-month delay earlier this year. But this will not be the end goal for the company: desmoid tumours are rare, slow growing and benign, and do not translate into a major market opportunity. SpringWorks has its eye on other uses for nirogacestat, now branded Ogsiveo, with studies ongoing in ovarian granulosa cell tumours and, most interestingly, multiple myeloma, where the oral gamma secretase inhibitor is being tested alongside various BCMA-targeting agents. In its most recent corporate presentation, SpringWorks highlighted data on a nirogacestat/Blenrep combo, presented at this year’s EHA meeting, which the group claims showed comparable efficacy versus Blenrep monotherapy, but with a substantial reduction in grade 3 ocular adverse events – ocular toxicity is a well-known issue with GSK’s antibody-drug conjugate. Meanwhile, dose-limiting toxicities and adverse events were seen in a study of a combo with Johnson & Johnson’s BCMA Tecvayli – these were observed with concurrent dosing, so the focus is now on delayed administration of nirogacestat. SpringWorks' expansion plans for OgsiveoSettingTrialDetailsStatusDesmoid tumours, adultsDefiMonotherapy vs placeboApproved Nov 2023Desmoid tumours, childrenARST1921*UncontrolledCompletes Dec 2024Ovarian granulosa cell tumoursNCT05348356Monotherapy, uncontrolledData due 2024Multiple myeloma (BCMA combos)Magnetismm-4Plus Pfizer's elranatamabOngoingDreamm-5Plus GSK's BlenrepData at EHA 2023: ORR 29% with nirogacestat + Blenrep, vs 38% with Blenrep monotherapyMajestec-2Plus J&J's TecvayliData at EHA 2023: 78% ORR with teclistamab + nirogacestatNCT05137054Plus Regeneron’s linvoseltamab1st pt dosed Sep 2023NCT03582033Plus Seagen’s SEA-BCMAPlannedNCT05259839Plus AbbVie’s ABBV-383PlannedUniversalAllogene's ALLO-715Data published Nature Medicine Jan 2023NCT04171843Plus Precision's PBCAR269APBCAR269A development terminated Nov 2022*Investigator-sponsored trial. Source: OncologyPipeline.