Spruce Biosciences is focused on developing innovative therapies for endocrine and neurological disorders, particularly where there is a significant unmet medical need. Their lead product candidate, tildacerfont, is a second-generation CRF 1 receptor antagonist that is currently undergoing late-stage clinical trials for conditions such as classic congenital adrenal hyperplasia (CAH) and polycystic ovary syndrome (PCOS). Tildacerfont works by selectively binding to CRF 1 receptors, which helps to inhibit the excessive production of adrenocorticotropic hormone (ACTH) and subsequently reduces the production of adrenal androgens. This mechanism aims to improve disease control and lessen the reliance on glucocorticoid steroids in patients suffering from these disorders.

In addition to its applications in CAH and PCOS, tildacerfont is being explored as a potential treatment for major depressive disorder (MDD). By blocking the CRF 1 receptor in the brain, tildacerfont may address hyperactive corticotropin-releasing factor (CRF) neurotransmission and restore normal functioning of the hypothalamic-pituitary-adrenal (HPA) axis, which is often disrupted in patients with MDD. The drug has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for its potential use in treating classic CAH, highlighting its significance in addressing rare and challenging medical conditions.

United States

Spruce Biosciences is a clinical stage biopharmaceutical company focused on developing novel therapies for rare diseases affecting the endocrine system.


Endocrine System Diseases

Genetic Diseases

Urogenital Diseases

Spruce Biosciences

Sanfilippo Syndrome

Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT)

Polycystic ovary syndrome

<p class="font_8">Spruce’s investigational lead product candidate, tildacerfont (formerly SPR001) is a potent, highly selective, oral, small-molecule antagonist of the corticotropin-releasing factor type-1 (CRF1) receptor. Preclinical studies have shown that through targeted delivery, tildacerfont binds to CRF1&nbsp;receptors to block CRF-stimulated receptor function, thereby decreasing the production of excess androgens (androstenedione [A4]), progestins (17-hydroxyprogesterone [17-OHP]) and adrenocorticotropic hormone (ACTH), the primary driver of adrenal gland enlargement. This may allow physicians to reduce the chronic, high-dose steroids used to treat patients with congenital adrenal hyperplasia (CAH), potentially allowing physicians and patients complete control of CAH.</p>

Tildacerfont 

Major depressive disorder

Congenital adrenal hyperplasia

Liver disease

Primary biliary cholangitis

<p class="font_7">Spruce’s investigational lead product candidate, tildacerfont (formerly SPR001) is a potent, highly selective, oral, small-molecule antagonist of the corticotropin-releasing factor type-1 (CRF1) receptor. Preclinical studies have shown that through targeted delivery, tildacerfont binds to CRF1&nbsp;receptors to block CRF-stimulated receptor function, thereby decreasing the production of excess androgens (androstenedione [A4]), progestins (17-hydroxyprogesterone [17-OHP]) and adrenocorticotropic hormone (ACTH), the primary driver of adrenal gland enlargement. This may allow physicians to reduce the chronic, high-dose steroids used to treat patients with congenital adrenal hyperplasia (CAH), potentially allowing physicians and patients complete control of CAH.</p>

Spruce Biosciences

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