An Endocrine News roundup of the week’s pharmaceutical news, breakthroughs, and general information. *
Spruce Biosciences Focuses on CAH in Clinical Trial
On October 18, Spruce Biosciences, Inc., a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need, announced completion of target enrollment of 72 patients and closure of screening in the CAHmelia-203 clinical trial evaluating tildacerfont for the treatment of adult classic congenital adrenal hyperplasia (CAH) and provided updated anticipated milestones.
“Completing target enrollment in the CAHmelia-203 study is a significant milestone for our CAH program and reflects the continued strong enthusiasm from patients and study investigators alike, evidences the large unmet medical need in the CAH community, and reinforces the momentum within our broader tildacerfont program,” said company CEO and Endocrine Society member Javier Szwarcberg, MD, MPH, in a statement. “Due to substantial patient interest in CAHmelia-203, eligible adult patients currently in screening will be allowed to enter the trial. As a result, we anticipate that final enrollment will exceed our original target of 72 patients, and project topline results from this study along with the topline results from all cohorts in our CAHptain clinical trial in pediatric classic CAH in the first quarter of 2024.”
CAHmelia-203 is a randomized, double-blind, placebo-controlled, dose ranging Phase 2b clinical trial evaluating the safety and efficacy of tildacerfont in adults with classic CAH and highly elevated levels of androstenedione (A4) at baseline while on stable glucocorticoid dosing. Tildacerfont is a once-daily, potent, and highly selective, non-steroidal, oral antagonist of the corticotropin-releasing factor (CRF) 1 receptor. The primary endpoint of the clinical trial is the percentage change in A4 from baseline to week 12 with secondary endpoints including the proportion of patients with levels of 17-hydroxyprogesterone (17-OHP) and A4 within the target and normal range, respectively, and change in lesion volume of testicular adrenal rest tumors (TARTs) in men. Additional endpoints include clinical outcomes and patient and clinician reported outcomes.
Spruce has updated its anticipated upcoming milestones as follows:
- Completion of enrollment in the CAHmelia-204 clinical trial in adult classic CAH patients on supraphysiologic doses of glucocorticoids with normal or near normal levels of A4 in early first quarter of 2024
- Topline results from all cohorts in the Phase 2 CAHptain clinical trial in pediatric classic CAH patients in the first quarter of 2024
- Topline results from the CAHmelia-203 clinical trial in adult classic CAH patients with highly elevated levels of A4 in the first quarter of 2024
- Topline results from the CAHmelia-204 clinical trial in adult classic CAH patients on supraphysiologic doses of glucocorticoids with normal or near normal levels of A4 in the third quarter of 2024
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