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-204 anticipated in the third quarter of 2024. | CAHmelia Studies |
| Pediatric Classic Congenital Adrenal Hyperplasia | Topline results from additional dose ranging in the fourth quarter of 2024. | CAHptain Studies | |
| Polycystic Ovary Syndrome | Planned presentation of final data in an upcoming medical conference. Partnering opportunities available. | P.O.W.E.R. Study |
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