PTC Therapeutics, Inc. announced on June 17th that the U.S. Food and Drug Administration has authorized the initiation of a Phase 2/3 trial to investigate PTC299, a dihydroorotate dehydrogenase (DHODH) inhibitor, as a potential treatment for COVID-19. PTC299 is an oral investigational drug with a novel dual-mechanism of action that has the potential to address the two critical elements of COVID-19: 1) the high viral replication and 2) the uncontrolled inflammatory response that ensues after infection. PTC will initiate an integrated Phase 2/3 study in the U.S. in the coming days with additional sites planned globally. Enrollment for the JEWELFISH study, assessing safety and pharmacodynamics of risdiplam in previously treated patients with SMA, who are now receiving risdiplam, is complete (n=174). Among the patients who had completed 12 months of treatment with risdiplam (n=18), a rapid and sustained two-fold increase in median SMN protein levels versus baseline was observed. An early assessment of safety showed a consistent safety profile compared to treatment-naive patients. Of the 174 patients enrolled in the JEWELFISH study, 76 were previously treated with nusinersen and 14 with onasemnogene abeparvovec. The remaining patients had been treated in previous Roche SMA clinical trials.
For further information, see PTC Therapeutics (https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-2-year-data-part-1-sunfish-and-new)
