Spruce Biosciences says CAHmelia-203 study did not meet efficacy endpoint

Spruce Biosciences announced topline results from its CAHmelia-203 study of tildacerfont in adult classic congenital adrenal hyperplasia and its CAHptain-205 study of tildacerfont in pediatric classic CAH. The clinical trial did not achieve the primary efficacy endpoint of the assessment of dose response for the change in A4 from baseline to week 12, the company said in a statement. 200mg androstenedione of tildacerfont demonstrated a placebo-adjusted reduction from baseline in A4 of -2.6% at week 12 with a non-significant p-value. Compliance with study medication and glucocorticoid was low with approximately 50% of patients reporting 80% or greater compliance, resulting in lower-than-expected tildacerfont exposure, according to Spruce. Most adverse events were reported as mild to moderate, it added. The company said, “We garnered important data from this study which will inform ongoing development of tildacerfont in adult classic CAH…Looking ahead to the third quarter of 2024, we are eager to report topline results from CAHmelia-204, which is focused on assessing GC reduction, a potentially registrational endpoint, in a different population of adult CAH patients with relatively controlled A4 levels and historically better adherence to GC therapy. Assuming positive results from CAHmelia-204 and CAHptain-205, we plan to meet with the U.S. Food and Drug Administration and comparable foreign regulatory authorities to outline the design of a registrational clinical program in adult and pediatric classic CAH.”