Pipeline
Tildacerfont is a CRF1 receptor antagonist designed to potentially offer markedly improved disease control and reduced steroid burden in patients with classic congenital adrenal hyperplasia (CAH) and other diseases characterized by elevated levels of adrenocorticotropic hormone (ACTH). Tildacerfont binds to CRF1 receptors on the pituitary gland, inhibiting excessive production of ACTH, and ultimately limiting the production of adrenal androgens such as androstenedione, a precursor to testosterone.
| Product Candidate | Indication | Status | |
|---|---|---|---|
| Tildacerfont | Adult Classic Congenital Adrenal Hyperplasia | Topline results from CAHmelia-203 in the second half of 2023. Topline results from CAHmelia-204 in the second half of 2024. | CAHmelia Studies |
| Pediatric Classic Congenital Adrenal Hyperplasia | Topline data in adolescents in the second half of 2023 | ||
| Polycystic Ovary Syndrome | Topline results in the first half of 2023 | P.O.W.E.R. Study |
Tildacerfont has been evaluated in eight clinical trials to date in 235 patients, including two Phase 2 clinical trials in patients with classic CAH: a two-week proof of mechanism dose-ranging trial, and a 12-week proof of concept trial. In the 12-week trial, tildacerfont treated patients with highly elevated adrenal androgens at baseline and achieved robust reductions in disease driving hormones, with many patients in the study achieving normal levels of these hormones.
Spruce is currently enrolling patients in two double-blind, placebo-controlled late-stage clinical trials in adult patients with classic CAH.
Spruce has also initiated a Phase 2 trial in pediatric classic CAH patients.
A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia
