Otsuka Pharmaceutical Co., Ltd. (Otsuka) and Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC) announced the launch of a global Phase 3 clinical trial for repinatrabit (JNT-517), an investigational oral small-molecule therapy for phenylketonuria (PKU).
The PheORD Phase 3 study is a randomized trial designed to evaluate the efficacy, safety, and tolerability of repinatrabit, administered twice daily, in participants living with PKU.
Repinatrabit received orphan drug designation and rare pediatric disease designation for the treatment of PKU from the U.S. Food and Drug Administration.
According to the company, interim analysis data from the Phase 1b/2 study demonstrated a robust reduction in blood Phe levels within one week of dosing, regardless of disease severity or non-responsiveness to existing treatments. The 150mg BID dose of repinatrabit led to a statistically significant mean blood Phe reduction from baseline of 60% on average across days 14, 21, and 285 and a 58% reduction from baseline to day 28.4 No serious adverse events were observed in any dosing group, no clinically significant changes in laboratory parameters, and no clinically significant changes in plasma amino acids other than Phe observed, confirming safety and tolerability.
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