Despite significant advancements in medicine, women's healthcare remains underserved, particularly in the realm of drug innovation. This talk will share the current landscape of drug approvals for women's health, highlight substantial unmet medical needs, and examine funding trends that impact innovation. Additionally, the extensive opportunities for novel drug discovery programs in this area will be emphasized.

Inavolisib, recently approved by the US FDA for endocrine resistant, PIK3CA mutated, HR-positive, HER2-negative, advanced breast cancer, in combination with palbociclib and fulvestrant, is a potent and selective inhibitor of PI3K alpha, one of the most commonly mutated oncogenes in cancer. The discovery of inavolisb, along with how the molecular properties were shaped by clinical and preclinical data from earlier molecules, will be discussed.

Since the passage of the NIH Revitalization Act in 1993, the NIH Office of Research on Women's Health (ORWH) has served as the Congressionally mandated focal point for coordinating research on the health of women within the organization. In this presentation, recent NIH efforts related advancing the health of women through research will be reviewed.

Family planning is a human right. Nevertheless, almost half of all pregnancies are unintended and half of them end in abortion. In LMIC, 97% of those are unsafe, leading to 50,000 deaths of young women. User preferences worldwide indicate a preference of innovative, non-hormonal contraceptives. Compounds addressing the sperm proteome in women appear promising based on expected safety & efficacy. Prioritization of drug targets for R&D activities will be discussed.

PCOS impacts 1 in 10 women of reproductive age, yet has no specifically approved therapeutics. In this heterogeneous endocrine disorder, androgen excess is linked to reproductive dysfunction and hirsutism. In our pursuit of novel therapies for PCOS, we have successfully applied our machine learning-driven medicinal chemistry platform to rapidly optimize a series of small molecules which affect the testosterone biosynthesis pathway, demonstrating in vivo reduction of testosterone with advanced leads.

Endometriosis and alternative sources of non-hormonal contraception are neglected and challenging issues associated with women's health. Today, I will discuss how we leverage multi-omics data and AI to identify novel targets implicated in Endometriosis and how we contribute to the challenge of designing novel non-hormonal contraceptives using AI. 

Opal Therapeutics is building a biotechnology platform dedicated to drug discovery in women’s reproductive health. By integrating patient-derived uterine organoids for chemical screening and advanced AI image analysis, we are pioneering innovative therapeutic approaches for chronic gynecological conditions, including fibroids and endometriosis. Our platform aims to uncover novel pharmaceutical solutions, addressing the urgent need for targeted interventions in women’s healthcare.