Efficacy and Safety of Encaleret in Autosomal Dominant Hypocalcemia Type 1

This correspondence presents findings from a phase 2b study evaluating the efficacy and safety of encaleret, an oral calcilytic, in 13 adults with autosomal dominant hypocalcemia type 1 (ADH1). ADH1, caused by gainof function variants in the calcium sensing receptor (CaSR) gene, leads to hypocalcemia, hyperphosphatemia, and hypercalciuria. Encaleret treatment normalized albumin corrected blood calcium levels, increased parathyroid hormone (PTH) and magnesium levels, and reduced urinary calcium excretion and phosphorus levels over a 24 week outpatient period. The drug was well tolerated, with no serious adverse events reported. These results suggest encaleret as a promising targeted therapy for ADH1, addressing its underlying pathophysiology without exacerbating renal complications. Further studies are ongoing to assess long term skeletal effects.

This correspondence presents findings from a phase 2b study evaluating the efficacy and safety of encaleret, an oral calcilytic, in 13 adults with autosomal dominant hypocalcemia type 1 (ADH1). ADH1, caused by gainof function variants in the calcium sensing receptor (CaSR) gene, leads to hypocalcemia, hyperphosphatemia, and hypercalciuria. Encaleret treatment normalized albumin corrected blood calcium levels, increased parathyroid hormone (PTH) and magnesium levels, and reduced urinary calcium excretion and phosphorus levels over a 24 week outpatient period. The drug was well tolerated, with no serious adverse events reported. These results suggest encaleret as a promising targeted therapy for ADH1, addressing its underlying pathophysiology without exacerbating renal complications. Further studies are ongoing to assess long term skeletal effects.