Spruce Biosciences announced topline results from two clinical trials, CAHmelia-204 and CAHptain-205, evaluating tildacerfont for congenital adrenal hyperplasia (CAH). The CAHmelia-204 trial in adults did not meet its primary endpoint of glucocorticoid (GC) dose reduction, while CAHptain-205 in adults and children suggested higher and more frequent tildacerfont doses might be needed for efficacy. As a result, Spruce is discontinuing both trials and exploring strategic options while reducing investment in tildacerfont for CAH.
These results are important for the CAH community because they impact the development of a potential new treatment option. CAH is a challenging condition requiring lifelong management with high doses of GCs, which can have significant side effects. Tildacerfont offered a potential alternative approach by targeting the underlying hormonal imbalance driving the disease. The trial outcomes underscore the complexity of CAH treatment and the need for further research to identify effective therapies that minimize GC reliance and improve patient outcomes.
CAHmelia-204 showed a negligible placebo-adjusted reduction in daily GC dose with 200mg once-daily tildacerfont. CAHptain-205 indicated a potential dose-response relationship, with higher twice-daily doses of tildacerfont showing a trend towards greater reduction in androstenedione levels, a key hormone marker in CAH. While both trials confirmed the general safety and tolerability of tildacerfont, the efficacy data prompted Spruce to reassess its development strategy. The company’s decision to discontinue the trials and explore strategic alternatives reflects the challenging financial landscape for biotech companies and the need to prioritize resources.
Looking ahead, the results from these trials offer valuable data for future CAH research, particularly regarding dosing regimens and potential patient subgroups. While tildacerfont’s development for CAH is being curtailed, the data generated may inform other research efforts and contribute to a better understanding of this complex disease. This shift in strategy by Spruce allows them to explore other opportunities and potentially address other unmet medical needs, while also preserving shareholder value. The search for effective CAH treatments continues, and researchers will likely incorporate the learnings from these studies to refine future clinical trial designs and explore alternative therapeutic approaches.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
