Spruce Biosciences Announces Pediatric Classic CAH Program Details During Virtual R&D Day
Phase 2 Program in Pediatric Classic Congenital Adrenal Hyperplasia (CAH) on Track to Initiate in 2021 with Data Expected by 1H 2023
Phase 3 Registrational Program in Pediatric Classic CAH Expected to Initiate in 2023
R&D Day Webcast Featuring Spruce Management, KOL Panel Discussion and Q&A Begins Today at
“We are pleased to initiate our Phase 2 clinical program of tildacerfont in children with classic CAH, a rare endocrine disorder where a significant unmet medical need exists for a non-steroidal treatment approach, this year,” said
The Phase 2 open-label study will utilize a sequential 3 cohort design to evaluate the safety, pharmacokinetics, and exploratory pharmacodynamics of tildacerfont in children aged 6 to 17 with classic CAH for up to 3 weight-adjusted doses, equivalent to adult doses of 50 mg, 100 mg, or 200 mg once daily of tildacerfont, for a duration of 2 weeks. Cohort 1 will include children between 11 and 17 years of age, at a dose of 50 mg once a day. Cohort 2 will also include children between 11 and 17 years of age, at a dose of up to 200mg once daily. Cohort 3 will include children between 6 and 10 years of age, at a dose of up to 200 mg once daily. The study drug will be dosed with an evening meal and will be formulated as granules to be sprinkled over food.
The Phase 2 program in pediatric classic CAH remains on track to initiate later this year with data expected by the first half of 2023. The Phase 3 registrational program in pediatric classic CAH is expected to initiate in 2023.
Spruce Biosciences R&D Day Webcast Details: Tildacerfont for Adult & Pediatric Classic CAH
Registration and Webcast Link
Interested parties may also access the webcast from the Events section of the company’s investor relations website. An archived replay of the webcast will be available after the conclusion of the presentation.
Tildacerfont is a potent and highly selective, non-steroidal, oral antagonist of the CRF1 receptor, which is the receptor for corticotropin-releasing factor (CRF), a hormone that is secreted by the hypothalamus. The CRF1 receptor is abundantly expressed in the pituitary gland where it is the primary regulator of the HPA axis. By blocking the CRF1 receptor, tildacerfont has the potential to address the uncontrolled cortisol feedback regulatory pathway in CAH, and in turn reduce the production of ACTH in the pituitary, limiting the amount of androgen produced downstream from the adrenal gland. Tildacerfont has been evaluated in 235 patients across eight clinical trials in which it has been generally well tolerated. No drug-related serious adverse events have been reported related to tildacerfont treatment.
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the results, conduct, progress and timing of Spruce’s clinical trials. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “plans,” “will,” “believe,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Spruce’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Spruce’s business in general, the impact of the COVID-19 pandemic, and the other risks described in Spruce’s filings with the