MAY 19, 2020. PTC announced delays in its gene therapy program for Friedreich’s ataxia and in the opening of a manufacturing facility in New Jersey, which is now expected to begin producing clinical material early next year.
Thursday, May 21, 2020
Minoryx Therapeutics doses first patient with leriglitazone in registration-enabling cALD NEXUS trial
20-May-2020. PharmiWeb.com. Mataró, Barcelona, Spain and Charleroi, Belgium, May 12, 2020 – Minoryx Therapeutics, a company that specializes in the development of innovative treatments for orphan Central Nervous System (CNS) diseases, today announces the initiation of the registration-enabling Phase 2 NEXUS trial to evaluate the safety and efficacy of leriglitazone in pediatric patients with early-stage cerebral ALD (cALD), the acute form of X-linked adrenoleukodystrophy (X-ALD).
Leriglitazone has also completed enrolment in FRAMES, a one year double-blind, placebo-controlled Phase 2 study in patients with Friedreich’s Ataxia. Results from ADVANCE and FRAMES are expected by the end of 2020 and results from NEXUS are expected in 2021. Leriglitazone has obtained Orphan Drug Designation from the European Commission and the FDA in X-ALD and Friedreich’s Ataxia.
Leriglitazone has also completed enrolment in FRAMES, a one year double-blind, placebo-controlled Phase 2 study in patients with Friedreich’s Ataxia. Results from ADVANCE and FRAMES are expected by the end of 2020 and results from NEXUS are expected in 2021. Leriglitazone has obtained Orphan Drug Designation from the European Commission and the FDA in X-ALD and Friedreich’s Ataxia.
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