Spruce Biosciences, a clinical-stage biotechnology company developing novel therapies for rare endocrine disorders, today announced positive results from a Phase 2a, multicenter, 12-week trial of tildacerfont, an investigational, oral, once-a-day corticotropin-releasing factor type-1 receptor antagonist, in development for the treatment of congenital adrenal hyperplasia (CAH).
| SAN FRANCISCO, Sept. 19, 2019 /PRNewswire/ -- Spruce Biosciences, a clinical-stage biotechnology company developing novel therapies for rare endocrine disorders, today announced positive results from a Phase 2a, multicenter, 12-week trial of tildacerfont, an investigational, oral, once-a-day corticotropin-releasing factor type-1 receptor antagonist, in development for the treatment of congenital adrenal hyperplasia (CAH).
The Phase 2a study enrolled patients with classic CAH accompanied by elevated androgens at baseline who were treated with tildacerfont once-daily over 12 weeks, resulting in clinically relevant reductions in key parameters. These included mean reductions from baseline of 74% for ACTH, 82% for 17-OHP and 55% for A4. ACTH is the direct target of tildacerfont, while A4 is the critical downstream biomarker used in clinical management of patients with CAH. Maximum mean reductions observed at any timepoint in the study were 84% for ACTH, 82% for 17-OHP and 79% for A4. In addition, 60% of patients with elevated, abnormal ACTH and 40% of patients with elevated A4 saw reductions to normalization by week 12. The primary endpoint of safety and tolerability was achieved, with no new safety signals observed in the study and the majority of adverse events classified as mild, not related to study drug and single event occurrences. No serious adverse events were observed. “There is a significant and urgent need for new CAH treatments as there are currently no FDA-approved therapies,” said Richard Auchus, M.D., Ph.D., an investigator in the study and professor of internal medicine and pharmacology, division of metabolism, endocrinology & diabetes at University of Michigan, Ann Arbor. “The results of this study are encouraging as they demonstrate, for the first time, that a novel non-steroidal treatment can produce clinically meaningful reductions across key biomarkers over a period of 12 weeks. These data indicate tildacerfont could be a promising new treatment option for patients with CAH.” Further, Richard King, chief executive officer at Spruce Biosciences, commented, “The magnitude of these reductions coupled with patients having been able to achieve normalization after only 12 weeks of treatment suggests that tildacerfont may provide a-first-in-class option for patients to better manage their disease by reducing their exposure to elevated androgens and, potentially, their lifelong steroid burden.” About Congenital Adrenal Hyperplasia (CAH) In CAH, the adrenal glands often produce excessive levels of male sex hormones or androgens. While both sexes need androgens for proper growth and development, an excess can cause problems that may include precocious puberty, short stature, hirsutism, increased risk of testicular adrenal rest tumors (TART) in men, and virilization and menstrual dysfunction in women. Although CAH testing is part of the newborn screening program, there are currently no FDA-approved therapies for CAH. Glucocorticoids (such as hydrocortisone, prednisone and dexamethasone) are commonly used to treat CAH but are associated with a wide-range of side effects, including weight gain, reduced bone mineral density, metabolic abnormalities and increased cardiovascular risk. Tildacerfont Phase 2a Study Design Full results of the study will be reported at an upcoming medical conference. About Tildacerfont Tildacerfont has been granted orphan drug status by both the FDA and EMA. For more information on tildacerfont, please visit www.sprucebiosciences.com. About Spruce Biosciences
SOURCE Spruce Biosciences |
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