BridgeBio announces proof-of-concept Phase 2b data on encaleret

BridgeBio Pharma announced that the proof-of-concept Phase 2b data evaluating the effects of orally-administered encaleret on mineral homeostasis in patients with ADH1 were published in the New England Journal of Medicine in partnership with the National Institutes of Health. As part of the Phase 2b study, participants completed one or two 5-day inpatient dose-ranging periods, followed by a 24-week outpatient period. Encaleret was administered twice daily with doses adjusted to achieve normal albumin-corrected blood calcium levels and participants stopped taking calcium and vitamin D supplements, the standard of care for ADH1. The results showed that: Encaleret corrected hypocalcemia and reduced hypercalciuria within a few days of treatment initiation with sustained effect during the 24-week outpatient period; As expected with elevated PTH levels, levels of bone turnover markers increased during the outpatient period, with levels in nine of 13 participants remaining normal. Long-term study will continue to assess skeletal effects; Kidney function and preexisting renal calcifications did not worsen and will continue to be monitored in longer-term studies. No serious adverse events were reported with encaleret. Treatment-related adverse events were limited to infrequent mild, transient, asymptomatic hypophosphatemia, or hypercalcemia that resolved either spontaneously or with dose adjustment; No treatment discontinuations or study withdrawals occurred