SWX:IDIABiotechs
Could Lucerastat’s Renal Signal Reframe Idorsia (SWX:IDIA)’s Rare-Disease Strategy And Risk Profile?
Idorsia Ltd. recently reported in Nature Communications the Phase 3 MODIFY and open-label extension results for lucerastat, an oral substrate reduction therapy in adults with Fabry disease, which did not meet its primary neuropathic pain endpoint but showed sustained biomarker reductions and encouraging renal outcomes.
The data, including a slower rate of eGFR decline and stabilization of cardiac measures in patients with impaired kidney function, have guided the design of a new Phase 3...