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.

The NIH Revitalization Act, passed in 1993 mandated inclusion of women into federally funded clinical research. More than three decades later, significant gaps remain in our understanding of the health of women. Opportunities to align research opportunities with the health needs of women will be discussed.

The nonprofit Sanders Tri-Institutional Therapeutics Discovery Institute (TDI) is a unique alliance created to remove the barriers that impede innovative, high risk translational drug discovery in academic settings. Without expert assistance in navigating the path of drug discovery, even the most transformational research advances often do not realize their potential to become major medical breakthroughs. TDI’s scientific contributions to the discovery, optimization, and validation of inhibitors of soluble adenylyl cyclase (sAC, ADCY10) for use as non-hormonal, on demand oral contraceptives for men (NewCo created: Sacyl Pharmaceuticals) and as topical contraceptives for women (in IND-enablement) will be presented.

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.

SeNa Therapeutics is focused on developing innovative disease-modifying Kisspeptin receptor (KISS1R)-targeted therapies for hypothalamic-pituitary-gonadal (HPG) axis disorders in women. SeNa's novel KISS1R binding peptide-derived molecules therapeutically stimulate or attenuate Kisspeptin signaling, addressing both HPG hyperactivity and diminished activity.

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 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.