Spruce Biosciences, a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological and endocrine disorders with significant unmet medical need, announced topline results from its CAHmelia-204 study of tildacerfont in adult CAH and its CAHptain-205 study of tildacerfont in adult and pediatric CAH.
“We are very grateful to all the patients, families, investigators, and the entire CAH community who supported the CAHmelia-204 and CAHptain-205 clinical trials. We garnered invaluable safety and exposure response data on tildacerfont from these studies, which suggests that higher doses and more frequent dosing may be necessary for efficacy in CAH,” said Javier Szwarcberg, M.D., M.P.H., Chief Executive Officer of Spruce. “Moving forward, we plan to evaluate a full range of strategic options for Spruce in addressing diseases with serious unmet need for patients. In the interim, the CAHmelia-204 and CAHptain-205 clinical trials will be discontinued, and we will be winding down Spruce’s investment in tildacerfont for the treatment of CAH as we conserve financial resources and look to maximize shareholder value.”
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CAHmelia-204 was a Phase 2b, randomized, double-blind, placebo-controlled clinical trial that evaluated the safety and efficacy of tildacerfont in reducing supraphysiologic GC usage in 100 adults with classic CAH on a mean GC dose of 35mg/day of hydrocortisone equivalents (HCe) (19mg/m2/day) and mean androstenedione (A4) level of 214 ng/dL at baseline.
The clinical trial did not achieve the primary efficacy endpoint of the absolute change in daily GC dose from baseline at week 24. 200mg QD of tildacerfont demonstrated a placebo-adjusted reduction from baseline in daily GC dose of 0.7mg HCe (95% CI: -4.3 to 2.9, p=0.7). Approximately 98% of patients were highly compliant with study drug. Tildacerfont was generally safe and well tolerated with no serious adverse events (SAEs).
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“Although the study missed its primary endpoint, the data offers valuable insights that will shape the future of CAH management and research,” said Jung Hee Kim, M.D., M.S., Ph.D., Principal Investigator and Associate Professor of Internal Medicine, Seoul National University Hospital and College of Medicine. “I am grateful to the nearly 400 CAH patients who shared their information with us and look forward to presenting our findings at upcoming conferences in 2025.”
CAHptain-205 was a Phase 2 open-label, 4-week, sequential cohort clinical trial, that evaluated the safety, pharmacodynamics (changes in A4 levels), and pharmacokinetics of QD and BID doses of tildacerfont from 50mg QD to 400mg BID in pediatric and adult patients with CAH. A trend was observed of larger reductions from baseline in A4 levels with higher BID doses of tildacerfont. Tildacerfont was generally safe and well tolerated across all doses with no drug-related SAEs.
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“This study was well-run with excellent compliance,” said Paul Thornton, M.B.B.S., Principal Investigator and Medical Director of the Endocrine and Diabetes Program at a CAH Center of Excellence. “The data suggests that a twice-daily dosing may be more effective.”
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Source – businesswire
