Sanofi revealed that its investigational BTK inhibitor rilzabrutinib notched a significant win in the LUNA 3 phase 3 study, hitting the primary endpoint of durable platelet response in adults with persistent or chronic immune thrombocytopenia (ITP). The study showed a significantly higher proportion of rilzabrutinib-treated patients achieved the platelet response goal compared to placebo in…
Insilico’s AI-discovered INS018_055 graduates to phase 2
Roughly a year ago, Insilico Medicine announced that it had dosed the first patient in a phase 1 study of INS018_055, an AI-discovered, first-in-class small molecule inhibitor. Now, the company has progressed to the next stage, launching a phase 2 study for the drug candidate. Insilico, a founding member of NVIDIA Inception, developed its AI…
Amylyx Pharmaceuticals launches phase 2 trial for AMX0035 Wolfram syndrome therapy
Cambridge, Massachusetts–based Amylyx has dosed the first participant in its phase 2 HELIOS clinical trial of AMX0035 focusing on Wolfram syndrome. The condition is an ultra-rare genetic disorder involving the endocrine system. Symptoms of Wolfram syndrome can include diabetes insipidus, diabetes mellitus, optic atrophy and hearing loss. In September, Amylyx won FDA approval for AMX0035…