Endometriosis: A research and development call to action

International Women’s Day is a global movement to celebrate women’s achievements in social, economic, cultural and political life. It is also a call to action for addressing persistent unmet needs and an opportunity to shine a spotlight the R&D efforts addressing often-overlooked conditions that impact women’s health.

Endometriosis affects approximately 10% of women of reproductive age worldwide, amounting to over 190 million women. It is a chronic, oestrogen-dependent inflammatory condition characterised by the presence of endometrial-like tissue outside the uterus. Despite its high prevalence, delayed diagnosis, limited treatment options, and the profound impact on quality of life remain as significant challenges. In this blog we examine the research conditions, pipeline, challenges and opportunities in developing new treatment options.

Endometriosis : A persistent unmet need

Endometriosis represents a leading societal public health unmet need which demands innovative solutions. Despite effecting an estimated 190 million women worldwide, the disease remains underdiagnosed. There is an average diagnostic delay of 7-10 years due to normalisation of menstrual pain and the lack of non-invasive diagnostic tools. Current treatment therapies focus on intermittent symptomatic relief as opposed to lasting disease modifying solutions. The impact on fertility is central also. Up to 50% of women with infertility may have underlying endometriosis.

This therapeutic gap underscores the urgent need for innovative, targeted treatment that address both the underlying pathology and long-term disease progression, creating a critical area for investment and clinical development.

The solutions gaps centres around many factors including delayed diagnosis and reliance on invasive diagnostic methods like laparoscopy. Symptom management is short-sighted and non-hormonal options are limited. Adverse effects of current treatment options present real-world problems for endometriosis patients and their physicians. Hormonal therapies are often associated with adverse effects such as bone loss, mood changes and other unwelcome outcomes.

Market potential and investment trends

Despite historical underinvestment, the women's health research sector is now poised for explosive growth. Untapped opportunities are set to transform translational and clinical research, driving substantial economic returns while addressing critical unmet needs in women's health.

The global endometriosis therapeutics market is projected to surpass $3 billion by 2030 driven by a compound annual growth rate (CAGR) of 12.5% from 2025 to 2030. Fuelling this expansion is increasing awareness, improved diagnostics and the demand for novel non-hormonal and disease modifying treatments. Recent funding trends indicate a shift, with VC firms, biotech investors and government initiatives recognising the commercial and clinical potential of this underserved space. Public and private funding bodies including the NIH, Horizon Europe and women’s health focused investment funds (ref WHAM report) are prioritising research in inflammation, fibrosis and neuromodulation as potential therapeutic targets.

As pharmaceutical companies and biotech firms expand their pipelines, CROs and clinical trial sponsors have a crucial opportunity to accelerate the development and commercialisation of breakthrough therapies. Endometriosis is a key frontier in women’s health R&D.

Biopharmaceutical companies are intensifying their efforts to develop new treatments. Abbvie received FDA approval for Orilissa in 2018 for endometriosis related pain. Pfizer and Myovant collaborated to launch Myfembree in 2022 and Gedeon Richter received EMA approval for Ryeqo in July 2021 for uterine fibroids and more recently an indication expansion to include endometriosis associated pelvic pain.

Emerging biotech firms are contributing to the innovation landscape also. Celmatix Therapeutics has initiated a novel drug program focusing on a Jun-N-terminal kinase (JNK) inhibitor aimed at addressing endometriosis-related pain and inflammation. Similarly, Gesynta Pharma is developing vipoglanstat, a non-hormonal drug candidate targeting the PGES-1 enzyme which is implicated in endometriosis pathology.

Current R&D pipeline and innovation in clinical trials

There is a surge in endometriosis research and development with over 15 key companies actively engaged in advancing more than 20 pipeline therapies. This robust pipeline encompasses a diverse array of treatment modalities including hormonal agents, non-hormonal pharmaceutical and biologic therapies all aiming to address the multifaceted nature of endometriosis.

Examples of emerging therapies include:

Linustedastat (FOR-6219): this investigational candidate functions as a 17B hydroxy-steroid dehydrogenase type 1 (17β-HSD1) inhibitor targeting the local conversion of estrone to the more potent oestradiol within endometriotic lesions. By reducing oestradiol levels at the lesion site, linustedastat aims to alleviate oestrogen driven symptoms association with endometriosis

Selective Progesterone Receptor Modulators (SPRMs): Compounds such as telapristone (CDB-4124) are under investigation for their dual agonist-antagonist effects on progesterone receptors. SPRMs have the potential to modulate endometrial tissue response, offering a novel approach to managing endometriosis related pain.

Other emerging targets include:

Immune modulation: Strategies targeting inflammatory cytokines (e.g., IL-6 inhibitors) and macrophage activity
Angiogenesis inhibitors: Blocking vascular endothelial growth factor (VEGF) pathways
Microbiome interventions: Exploring the gut-uterine axis in endometriosis
Epigenetic modulators: Compounds targeting epigenetic changes in endometriotic lesions
Anti-NGF (Tanezumab): Nerve growth factor inhibitor. Preclinical and exploratory clinical trials for endometriosis-related pain (Pfizer/Eli Lilly)

FemTech: Transforming endometriosis and women’s health R&D

Technology-driven solutions for women’s health (FemTech) is rapidly evolving to bridge clinical gaps in diagnostics, treatment and patient engagement. In endometriosis, to address lengthy diagnostic delays, FemTech innovations are revolutionising early detection and management. Tools like AI powered symptom tracking apps, non-invasive biomarker tests, and wearable pain-monitoring devices are helping to identify and track symptoms. More broadly, FemTech is reshaping women’s health R&D, accelerating progress in areas such as menstrual health, fertility, menopause, and chronic gynaecologic conditions.

Investment in FemTech has surged, with funding surpassing $2.5 billion globally, highlighting the increasing recognition of women’s health as a critical and underserved market. As regulatory agencies, biotech firms, and investors prioritise women-centred innovation, FemTech is becoming a driving force in precision medicine, digital therapeutics, and personalised care, making it indispensable in the future of women’s healthcare.

Innovations in clinical trials

Advancements in diagnostic methodologies are enhancing the precision for clinical trials.

Non-invasive diagnostic tools: the development of blood-based biomarkers such as the Menstrual Blood Test (MBT) by Pearanta, aims to facilitate earlier, less invasive diagnosis of endometriosis. This innovation not only improves patient recruitment by identifying candidates more efficiently but also enables the monitoring of disease progression and treatment efficacy without reliance on surgical interventions.

Advanced imaging techniques: Studies exploring the use of 99mTc-maraciclatide with single-photon emission computed tomography (SPET) have demonstrated potential in detecting superficial peritoneal endometriosis. Such imaging could lead to more accurate patient stratification and assessment of therapeutic outcomes in clinical settings.

These innovations collectively aim to enhance the specificity and sensitivity of endometriosis diagnosis and thereby improve patient selection and the overall efficacy of trials. As the endometriosis R&D pipeline expands, the integration of novel therapeutic approaches with cutting edge diagnostic tools holds promise for more effective management strategies.

Regulatory considerations and related R&D challenges

The regulatory environment for endometriosis therapeutics is evolving with agencies like the FDA and EMA recognising the significant unmet medical need. In the US the FDA has granted fast track designation to innovative diagnostic agents such as 99mTc-maraciclatide aiming to expedite the development of non-invasive diagnostic tools for superficial peritoneal endometriosis. Similarly, the EMA has approved treatments such as Ryeqo a combination oral therapy for managing mode to severe symptoms of uterine fibroids and endometriosis associated pain in women who have undergone prior treatment. The FDAs Women’s Health Research Roadmap was updated in September 2024. Since its inception, the FDA Office of Women’s Health (OWH) has worked to support the FDA’s public health and consumer protection mission by leading and coordinating research, policy development, and educational and communication initiatives focused on women’s health.

Some regulatory environment challenges in R& D include heterogeneity:

Endometriosis presents variably among patients, complicating clinical trial design
Biomarker development: Lack of validated, non-invasive biomarkers for early detection and monitoring
Trial recruitment: Recruiting patients for clinical trials is challenging due to the disease's underdiagnosis and stigma
Funding gaps: Despite its prevalence, endometriosis research receives less funding compared to other chronic conditions

These challenges can be overcome with new perspectives and improved resourcing of women’s health.

Health Disparities

Socioeconomic factors, race, and geographic location significantly influence access to diagnosis and care. Women in underserved areas often experience longer delays and worse outcomes. One way to address this is through establishing endometriosis centres of excellence to streamline diagnosis and treatment pathways. Some recent examples include:

The World Endometriosis Research Foundation (WERF)
Ireland's Women's Health Taskforce, which prioritises reproductive health under its national women's health policy

Conclusion

The convergence of regulatory support, innovative research and specialised clinical trial management presents a unique opportunity to transform the environment of endometriosis treatment. As awareness and investment in women’s health continues to grow, collaboration among pharmaceutical companies, CROs and regulatory bodies is essential to accelerate the development of effective therapies.

Endometriosis demands urgent attention from the global healthcare community. By leveraging emerging therapies, fostering public-private partnerships, and prioritising policy reforms, we can address this critical unmet need in women’s health. On International Women’s Day, we reaffirm our commitment to advancing research, improving care, and empowering women worldwide to lead healthier, pain-free lives.

Contact us to find out how ICON can support your women’s health clinical trials.