Women's Health: Drug Discovery to Novel Therapies

Despite remarkable progress in medical science, women's healthcare continues to face critical gaps—nowhere more evident than in drug innovation. This talk will explore the current state of drug approvals for women’s health, highlight substantial unmet medical needs, and discuss how funding dynamics shape innovation. It will highlight current trends in drug discovery and map out future directions for advancing innovation in women’s health.

Unique sex-specific biological, hormonal, and genetic factors influence cancer risk, progression, and treatment response in women, yet these differences are often underexplored in research and clinical development. This session will explore cutting-edge oncology treatments tailored to women, including novel biomarkers and targeted therapies that will improve cancer outcomes.

Proliferating cell nuclear antigen (PCNA) plays an essential role in regulating DNA synthesis and repair and is indispensable to cancer cell growth and survival. It represents an attractive molecular target to develop broad-spectrum anti-cancer agents. By targeting a cancer specific domain of PCNA, we identified a novel compound, AOH1996, which selectively kills cancer cells but causes no significant toxicity to a broad range of non-malignant cells. AOH1996 is in clinical trials. Our research suggests AOH1996 may significantly improve women’s cancer therapeutic approaches.

Breast cancer affects one in eight women during their lifetime, making it the most frequently occurring cancer among women. Globally, the World Health Organization estimated that breast cancer caused approximately 670,000 deaths worldwide in 2022. About 70% of all breast cancers express estrogen receptor alpha (ERa), making it a key target for breast cancer treatment. Giredestrant, a full ERa antagonist and degrader, has undergone multiple Phase III clinical trials for patients at various stages of breast cancer. The discovery and development of giredestrant will be shared to inspire further groundbreaking research aimed at helping patients in need.

Life-threatening pregnancy disorders such as preeclampsia remain understudied, limiting therapeutic options for mothers and infants. This talk introduces a chemoproteomic drug de-orphanization framework using activity-based protein profiling to identify actionable targets of approved drugs. Importantly, disease-relevant stress pathways defined in (pre)eclampsia converge with vulnerabilities in glioblastoma, enabling rational discovery of lead molecules and therapeutic strategies for otherwise intractable brain cancers with immediate translational relevance to oncology, neurology, and maternal medicine.

EDS is characterized by marked changes to the urogenital region and all epithelial surfaces. Within 5 years post-menopause, the vagina and other epithelial surfaces lose more than 40% in thickness and elasticity, causing drying, itching, and inflammation. ORX-102 is a safe and effective alternative to currently marketed estrogens for long-term use even in women for whom estradiol and estriol are contraindicated.

Adjuva Bio is developing next-generation cell therapies to treat endometriosis by targeting the fibrotic and inflammatory microenvironment that drives the disease. Our engineered cells are designed for precision targeting and localized immunomodulation to reduce inflammation. This presentation will highlight our therapeutic strategy, early preclinical data, and the broader potential of cell-based platforms to address major unmet needs in women’s health.

This talk will focus on the use of botulinum toxin as a novel therapy for major depressive disorder (MDD). Multiple controlled clinical trials have established the efficacy of this therapy in patients with moderate to severe MDD. Discussion will include the unique value drivers of this therapy, particularly for females, as well as the proposed biological mechanisms behind its efficacy.

The FDA's Botanical Drug Guidance offers drug developers a regulatory pathway to approval for plant-based medicines. Plants are sophisticated chemical manufacturing environments and hold therapeutic promise for a multitude of conditions. Altin Biosciences is pursuing a novel botanical therapeutic for the non-hormonal treatment of uterine fibroids. Learn about the regulatory pathway for botanicals, myths, challenges and opportunities.

There is a significant gap when it comes to understanding the underlying causes affecting women's health and funding for R&D, and there are tools and data that we can leverage to help us begin to close the gap and pave a path towards addressing diseases that result in a significant societal and economic burden. I will describe the work we did in collaboration with Gynae Solutions, the Buck Institute for Research on Aging, and the Chinese University of Hong Kong to identify novel targets implicated in immune pathways, as well as a drug being repurposed to address endometriosis.

Luteinizing Hormone/ Choriogonadotropin Receptor (LHCGR) is a critical regulator of fertility in women and men. It is necessary for follicle development and normal spermatogenesis, and loss of LHCGR activity leads to infertility. The injectable LHCGR agonist, hCG, is the current standard of care for females undergoing controlled ovarian stimulation and/or males with secondary hypogonadism. Using our machine learning platform, we identified potent, drug-like small-molecule LHCGR agonists with comparable efficacy to hCG in murine in vivo models.

Women are not just small men, and their health solutions shouldn’t be scaled-down versions of male-centered models. Daré’s novel drug-delivery technologies are designed specifically for women and address the often-overlooked areas such as sexual and reproductive health that demand more targeted, thoughtful approaches to care.

Persistent disparity in female drug pharmacokinetics and dosing leads to a "one-size-fits-all" approach, often based on male-centric data. This oversight causes suboptimal efficacy or increased adverse drug reactions in women. Examining historical clinical studies and drug approvals where female-specific data were inadequately considered is crucial for understanding these failures and informing appropriate drug-development and prescribing practices.

Personalized predictive models are transforming the landscape of women’s health. By leveraging data-driven insights and AI-powered tools, researchers and clinicians can identify unique health patterns, accelerate biomedical discoveries, and tailor interventions to individual needs. We'll discuss how these models are being applied to close gaps in diagnosis and improve clinical trial representation. We'll highlight considerations, challenges, and translational opportunities that come with deploying predictive models in real-world settings.