Shots:
- OLE study assessed repinatrabit (JNT-517) in adolescents with PKU. Early time-point data remain embargoed to preserve parent-study blinding, with additional randomized-period results to be presented later
- First cohort receiving repinatrabit (75mg, PO, BID) showed a 67% mean reduction in Phe from baseline (Day 56/OLE Month 1), with responses seen in all pts, incl. prior Sapropterin responders, non-responders, & one naïve patient. The study will evaluate 150mg dose in a 2nd cohort
- Repinatrabit (75 or 150mg) is being evaluated in the P-III (PheORD) trial, where 120 adults will be randomized to repinatrabit or PBO for 6wks., followed by PBO crossover to 150mg; 1EP completion is expected in late 2026, with full study completion in 2028. Data from both OLE & P-III were presented at ACMG’26
Ref: Businesswire | Image: Otsuka | Press Release
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