RiverVest managing director Niall O’Donnell’s expertise in rare diseases and experience in putting together a high-performing team has been a proven formula for success.

With portfolio company Spruce Biosciences, RiverVest is once again leveraging scientific knowledge and industry-relevant operational experience to significantly address critical unmet medical needs in rare and orphan diseases.

Congenital Adrenal Hyperplasia (CAH), a group of life-threatening endocrine disorders, is characterized by an inability to produce key hormones such as cortisol and aldosterone, and also leads to overproduction of certain adrenal androgens that play a role in male traits and reproductive activity. Current treatment options for patients are limited to high doses of steroids in the form of glucocorticoids or mineralocorticoids to both replace absent hormones and control excess androgens, with no other new novel therapies approved in approximately 50 years. Patients, often diagnosed at birth, suffer from symptoms including abnormal or early puberty often resulting in short stature, infertility, amenorrhea, and other psychological effects attributable to high androgen exposures. There are also metabolic effects such as weight gain, blood pressure elevation, insulin resistance, and bone effects such as osteoporosis due to the high exposure to steroids.

Spruce Bioscience’s lead drug candidate, Tildacerfont, has the potential to improve outcomes for patients with Congenital Adrenal Hyperplasia

For clinicians treating CAH, a difficult choice must often be made between the negative effects of consistently high adrenal androgen levels or the harmful consequences of lifelong, high-dose steroid use. Additionally, it is estimated that even with steroid treatment, 70% of adult classic CAH patients are living with a disease that is “poorly controlled.” Spruce Biosciences’ lead drug candidate, tildacerfont could provide an innovative option to lessen the disease and steroid burden for these patients and improve outcomes.

The company was established through acquisition of tildacerfont from Eli Lilly. RiverVest joined a Series A financing and played a key early role in building a management team with proven experience developing rare disease therapies. Once again, RiverVest partnered with Pappas Capital Venture Partner Mike Grey, a collaborator on several RiverVest exited and active portfolio companies including Lumena, Amplyx, Reneo, and Mirum Pharmaceuticals. RiverVest has guided the company through major milestones, including:

  • A successful CEO transition as the company prepared to conduct pivotal clinical studies.
  • Positive clinical trial results from Phase 2a studies of tildacerfont in adults with classic CAH.
  • Oversubscribed $88 Million Series B fundraising in February 2020.
  • IPO in October 2020.

At Spruce, every employee is working on finding solutions for patients with rare endocrine diseases. In the case of classic congenital adrenal hyperplasia, we are focused on developing the first new therapeutic for these patients in over 50 years, with the goal of sparing patients the difficult choice between having too much androgen or too much androgen or too much steroid as a result of the disease and current standard of care therapy.

Richard King, CEO

Currently, Spruce Biosciences is conducting late-stage clinical trials globally to evaluate the safety and efficacy of tildacerfont in adults with classic CAH. In June of 2021, the company published positive Phase 2a results in the Journal of Clinical Endocrinology and Metabolism that demonstrate tildacerfont’s potential to normalize key hormones in adults with classic CAH. The company is also expanding studies to evaluate tildacerfont for use in other indications, including pediatric classic CAH, and Polycystic Ovary Syndrome arising from excess adrenal androgens.

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