Spruce Biosciences Presented Phase 2 Tildacerfont Results for PCOS at ENDO 2024

In an effort to address the significant unmet medical needs in treating polycystic ovary syndrome (PCOS), Spruce Biosciences, Inc. has presented promising results from its Phase 2 POWER study. This study evaluates the efficacy and safety of tildacerfont, a second-generation CRF1 receptor antagonist, for PCOS treatment. The findings were shared at the 2024 Annual Meeting of the Endocrine Society (ENDO 2024).

Study Background and Need for New Treatments

PCOS is a hormonal disorder affecting roughly five million women in the United States and approximately 115 million women globally. It is characterized by elevated androgen levels, ovarian cysts, and irregular menstrual cycles. Common symptoms include hirsutism, alopecia, acne, infertility, weight gain, fatigue, depression, and mood swings. The underlying causes remain largely unknown, but factors like excess insulin secretion and low-grade inflammation have been implicated. Currently, there are no FDA-approved therapies specifically for PCOS, and existing treatments only manage symptoms rather than addressing the root causes.

Expert Opinions

Dr. Lubna Pal, a specialist from Yale University School of Medicine, emphasized the need for new PCOS treatments. She noted that the study's results, which show tildacerfont's ability to reduce dehydroepiandrosterone sulfate (DHEAS), could potentially mitigate numerous health risks associated with chronic hyperandrogenism in PCOS patients.

Key Findings from the POWER Study

The POWER study enrolled 27 women diagnosed with PCOS, aged around 28 years on average. The study aimed to evaluate the safety and efficacy of tildacerfont, dosed up to 200 mg daily, over 12 weeks. Key observations from the study included:

1. Reduction in DHEAS Levels: In women with elevated baseline DHEAS, tildacerfont significantly reduced DHEAS levels compared to placebo, showing a 12.4% decrease versus a 5.7% increase in the placebo group (p = 0.020). This finding suggests that tildacerfont may help reduce adrenal androgen levels, addressing one of the underlying issues in PCOS.

2. Increase in SHBG Levels: Tildacerfont also led to a significant increase in serum sex hormone-binding globulin (SHBG) levels, with a 32.9% rise compared to an 8.1% drop in the placebo group (p = 0.012). Higher SHBG levels could potentially lower the amount of free, bioactive sex hormones, such as testosterone, which are implicated in PCOS symptoms.

3. Safety and Tolerability: Tildacerfont was well-tolerated with no significant safety concerns. Most adverse events reported were mild to moderate, and no serious adverse reactions were observed.

Implications and Future Directions

Will Charlton, the Chief Medical Officer of Spruce Biosciences, expressed gratitude to the study participants and investigators, highlighting the importance of these findings in advancing PCOS treatment options. He emphasized the need for further studies to explore the full potential of tildacerfont and referenced ongoing efforts to find strategic collaborations for future research.

About Tildacerfont

Tildacerfont is a potent, selective, non-steroidal oral antagonist of the CRF1 receptor, which plays a key role in regulating the hypothalamic-pituitary-adrenal (HPA) axis. By blocking this receptor, tildacerfont can reduce the production of adrenocorticotropic hormone (ACTH) and subsequently lower androgen levels produced by the adrenal glands. This mechanism holds promise for treating the underlying causes of PCOS, potentially alleviating symptoms such as hair growth, acne, irregular periods, and infertility.

Conclusion

The Phase 2 POWER study's results are promising, indicating that tildacerfont may offer a novel therapeutic avenue for women suffering from PCOS. By addressing the hormonal imbalances at the root of the condition, tildacerfont could provide relief from the myriad symptoms that affect millions of women worldwide. Further research is warranted to confirm these findings and fully explore the potential benefits of this treatment.

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