Spruce Biosciences Presents Phase 2 Data for Tildacerfont in Adults with Congenital Adrenal Hyperplasia at 23rd European Congress of Endocrinology

Rhea-AI Summary

Spruce Biosciences, Inc. (Nasdaq: SPRB) presented promising data from its Phase 2 clinical trial of tildacerfont for treating classic congenital adrenal hyperplasia (CAH) at the 23rd European Congress of Endocrinology. The open-label study showed significant reductions in disease-driving hormones such as ACTH, 17-OHP, and A4 in patients with poor disease control, achieving up to 84% reduction in ACTH levels. Notably, 60% of patients experienced normalization of ACTH levels without increasing steroid doses. The ongoing CAHmelia program aims to further evaluate the treatment's potential to reduce glucocorticoid usage.

Positive

• 84% reduction in ACTH levels in poorly controlled patients.
• 60% of patients normalized ACTH levels over twelve weeks.
• First investigational product to normalize hormone levels in classic CAH without steroid dose increases.

Negative

• No significant changes in hormone levels for patients with good disease control.
• Trial did not evaluate reduction in glucocorticoid dosing for patients in good control.

Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need, today presented data from its Phase 2 clinical trial of tildacerfont in adults with classic congenital adrenal hyperplasia (CAH) at the European Society of Endocrinology’s 23^rd European Congress of Endocrinology (ECE 2021), taking place virtually May 22 – 26, 2021.

SPR001-202 was an open-label, 12-week Phase 2a clinical trial, which assessed the ability of a daily dose of 400mg of tildacerfont to lower disease-driving hormones such as adrenocorticotropic hormone (ACTH), 17-hydroxyprogesterone (17-OHP), and androstenedione (A4) over a 12-week dosing period. These hormones are used by physicians as a means of measuring the severity of CAH. Patients were classified into two groups based on disease control using these baseline hormone levels, defined as either poor disease control or good disease control.

Patients with poor disease control upon study entry had mean baseline levels of ACTH, 17-OHP and A4 that were significantly above the upper limit of normal. Administration of tildacerfont to these patients resulted in mean maximum reductions of 84% in ACTH, 80% in 17-OHP, and 79% in A4 compared to baseline across the study period. This enabled reduction in the levels of these key hormones to normal or near normal levels, including normalization of ACTH levels in 60% and normalization of A4 levels in 40% of poor disease control patients during month three. Normalization of these highly elevated hormones in classic CAH patients without increases to daily steroid doses has not been reported to date with any other investigational product candidate. Patients with good disease control upon study entry, achieved by supraphysiologic glucocorticoid dosing, had mean baseline levels of ACTH, 17-OHP and A4 that were below the upper limit of normal. Administration of tildacerfont to these patients did not lead to significant changes in these levels. While this study did not evaluate the ability of tildacerfont to reduce the need for high glucocorticoid dosing in patients with good disease control, this objective is an important unmet need due to the long-term and serious side effects associated with chronic use of high doses of glucocorticoids and is currently being evaluated in the CAHmelia-204 study.

“It is encouraging to see that tildacerfont, an oral, once-daily, and non-steroidal investigational therapy, produced meaningful reductions in highly elevated hormone levels for classic CAH patients, including, in some cases, normalization of these hormone levels over twelve weeks,” said Richard Auchus, MD, PhD, the study’s lead investigator and Professor of Internal Medicine and Pharmacology, University of Michigan, Ann Arbor. “The data suggest that tildacerfont has the potential to improve the adverse consequences of androgen excess and to reduce the burden of daily glucocorticoid dosing for these patients. Patients with classic CAH should benefit from new treatment options, which effectively manage their disease, yet reduce the complications derived from chronic glucocorticoid exposure as the existing standard of care.”

Spruce’s CAHmelia program in adult classic CAH patients is underway and actively enrolling in sites within the United States and Europe. CAHmelia-203 is assessing the ability of tildacerfont to reduce excessive adrenal androgens in patients with poor disease control, while CAHmelia-204 is assessing the ability of tildacerfont to reduce glucocorticoid usage in patients with good disease control while maintaining control of androgens.

The presentation is on display in ECE 2021’s virtual poster hall. Learn more about the full program and how to access the poster presentation details on

FAQ

What were the results of the Phase 2 trial for SPRB's tildacerfont?

The trial showed up to 84% reduction in ACTH and significant normalization of hormone levels in patients with poor disease control.

What is the significance of the hormone level reductions in the SPRB study?

The reductions indicate potential for tildacerfont to effectively manage classic CAH without increasing steroid doses.

What are the next steps for SPRB after the Phase 2 trial?

Spruce is continuing its CAHmelia program to further assess tildacerfont's effects on glucocorticoid usage and adrenal androgens.

When was the data from the SPRB study presented?

The data was presented at the 23rd European Congress of Endocrinology from May 22 to 26, 2021.

What is the stock symbol for Spruce Biosciences?

The stock symbol for Spruce Biosciences is SPRB.