Cerecor announced the completed dosing for a Phase l study of CERC‑801 treating Phosphoglucomutase 1 (PGM1) deficiency.
Cerecor (NASDAQ:CERC) announced the completed dosing for a Phase l study of CERC‑801 treating Phosphoglucomutase 1 (PGM1) deficiency.
As quoted in the press release:
The single-center, US-based safety, tolerability and pharmacokinetic study was an open-label, randomized, single-dose, 4-way crossover study in 16 healthy adult volunteers. CERC-801 was shown to be safe and well-tolerated at the studied doses, with no serious adverse events. CERC‑801 related adverse events were mild and transient. Pharmacokinetic (PK) data is expected in the summer of 2019.
“We believe the excellent safety profile and anticipated PK data will provide a solid foundation for the ongoing development of CERC-801,” said Dr. Perry Calias, PhD, Chief Scientific Officer at Cerecor. “We have also initiated a retrospective study seeking to collect natural history, efficacy and safety data from CDG patients treated with monosaccharide substrate replacement therapy for, PGM1-CDG, MPI-CDG and Leukocyte Adhesion Deficiency Type II (LADII) also known as SLC35C1-CDG. The information gathered through this study will be instrumental in facilitating regulatory approval of all three CERC-800 programs through the 505(b)(2) pathway.”