Is women’s health reaching an inflection point?
Many biotech investor conferences now include a panel on women’s health. But Weatherden, the clinical development consultancy, recently decided something more in-depth was needed, resulting in a half-day symposium – titled Women’s Health: From Intractable to Investable – which was held in London on 11th June.
Why has investment in the area historically been poor? What is holding back development of new therapeutics from a scientific point of view? Does it even make sense to think of ‘women’s health’ as a discrete sector at all? These were some of the themes explored during the event at St Ermin’s Hotel in St James’s.
Explaining the reasons for holding the symposium, Emma Tinsley, CEO of Weatherden, said she and fellow colleagues had asked themselves: “Every challenge in women’s health drug development has already been solved somewhere else – so, why hasn’t anyone bothered to solve it here?
“We thought: the development playbook exists, it just needs translating – and the right people in the right room is how you start.”
The purpose was to “start a conversation”, she said, with a particular focus on practical problems.
Looking ahead, she believes women’s health is at an “inflection point” – one similar to that crossed by the likes of oncology, rare diseases, and obesity in the not-too-distant past – with scientific understanding improving, development pathways maturing, and investor confidence in the area growing.
The drive for data
The event’s three panels focused on: defining the right patient; establishing credible signals, endpoints, and biomarkers; and how to move a product successfully towards commercialisation after regulatory approval.
But there was one overarching theme that cut across all discussions: a critical need for better data on women’s health conditions.
Without that, all the panellists agreed, our understanding of the fundamental biology underlying conditions such as endometriosis, polyendocrine metabolic ovarian syndrome (PMOS), and many others, would be stymied – and so too would the development of new therapeutics.
“Unless we understand the underlying disease, we will not succeed,” said Dr Carmen Walbert, chief medical officer of Theramex.
A note on defining ‘women’s health conditions’: these can be broadly split into two categories. The first is those affecting women only, due to the clearly sex-based nature of the condition, such as the two mentioned above, menopause, and certain cancers, for example. The second is a broader group of conditions that affect both sexes, but disproportionately affect women – such as lupus (and autoimmune diseases more generally), neurological disorders including Alzheimer’s and migraine, and bone conditions like osteoporosis.
Better data will enable diseases to be better understood and help define sub-categories of patients based on the underlying biology, allowing patients to be stratified accordingly and treated with targeted, smarter drugs.
Dr Harriet Holme, a clinical development director at Weatherden said the way we have expanded our knowledge of breast cancer over the last 20 to 30 years, so we now understand it to be a collection of diseases with quite distinct underlying causes – iss a model that could be applied to other women’s health conditions. “You need to understand the biology to create effective therapies.”
Panellist Colleen Acosta, CEO of Freya Biosciences, which is developing immune-mediated therapeutics for women’s health conditions including IVF-related embryo implantation failure and bacterial vaginosis, said: “Effective clinical trial design for women’s health conditions requires a deeper understanding of the underlying disease biology than the field historically had.”
“Many women’s health conditions remain poorly characterised at a mechanistic level, making it difficult to identify validated therapeutics targets, develop meaningful biomarkers, and stratify patients effectively,” she continued. “Without this foundational scientific understanding, even well-funded programmes can struggle to generate robust and reproducible evidence required by regulators.”
However, Acosta stressed this was a common issue across biotech as a whole, and not unique to women’s health, and thus a solvable problem.
OIlie Hardick, investment director at M Ventures, a venture capital firm, said he thought “better data infrastructure” in women’s health would help bridge the “data gap”, eventually making drug development in the field “more precise, evidence-driven, investable, and ultimately more widely adopted”.
Identifying valid, practical, and meaningful biomarkers
Going hand-in-hand with better understanding of fundamental biology is the need for better biomarkers to diagnose disease, track natural progression, and measure a patient’s response to therapeutics.
A diagnosis of endometriosis, for example, can only be confirmed by a laparoscopy at present, which is invasive and potentially uncomfortable. Yet, ultrasound and MRI could increasingly be used for diagnosis and to track changes, thanks to improvements interpreting scans, said Beth Fisher of Fidēs Imaging, a point reiterated by Dr Graham Dixon, CEO of Hirunda Biosciences and head of R&D women’s health at Gedeon Richter. Better access to joined-up ‘big data’ sets would improve things still further, she argued.
Dr Agnès Arbat, CEO of Oriva Therapeutics, which has an experimental non-hormonal treatment for endometriosis in clinical development, said better biomarkers were “essential” to delineate different sub-types of endometriosis and thus improve treatment for women with the condition.
She believed progress in endometriosis was “ahead of the curve” compared to other women’s health conditions, with “a deeper understanding of disease biology enabling identification of new targets, improved diagnostics, and more personalised treatments”.
Daniel Sieiro, senior associate of European venture capital firm Sofinnova Partners, said, while developing new ways of identifying and characterising endometriosis was important, there was no point “waiting for that magic biomarker”. With today’s imaging techniques and pain scores, “we can start to stratify patients”.
Another big theme was the importance of asking women what was important for them in terms of clinical outcomes.
Dr Lisa Campbell, director of regulatory strategy at Richmond Pharmacology, said this was why the non-hormonal treatment for hot flushes, fezolinetant (Veoza, made by Astellas Pharma), had been “a big success”. Women had been asked what mattered to them, and they’d said they wanted a non-hormonal treatment that reduced the frequency and intensity of hot flushes.
“It’s not rocket science,” said Dr Campbell.
Hardick noted: “Clinical trial design matters enormously in women’s health. The patient voice needs to shape endpoints.”
Jackie Mulryne, a partner at Morgan Lewis, said firms “need to start conversations early” with regulators if they want to introduce a new type of data, because the rule-setters “need to have trust” in that data. When it came to subjective data, engaging early was doubly important, she said.
Trials on trial
Women have historically been under-represented in clinical trials, despite out-numbering men. Unsurprisingly, women are much more likely to suffer an adverse drug reaction.
When it comes to pregnant women and breastfeeding women, the picture is starker still. Dr Kirsty Wydenbach, head of regulatory strategy at Weatherden, said only 1% of UK clinical trials had included pregnant women. And of some 4,000 Phase 3 trials, just 12 had included pregnant women, and only eight breastfeeding women.
As Marie Teil, global head of the Women of Childbearing Age Programme at UCB, put it, “we have almost no [clinical trial] data” involving pregnant or breastfeeding women.
The thalidomide scandal has cast a very long shadow over the very idea of taking drugs while pregnant or breastfeeding. But panellists said the truth was women do end up taking drugs to manage health conditions while expecting – only, these tend to be prescribed off-label or are old drugs where years of post-authorisation safety data exists.
Wouldn’t it be better, asked Lawrence Tallon, CEO of the Medicines and Healthcare products Regulatory Agency (MHRA), if pregnant women were treated like grown-ups, given all possible evidence of safety concerns of an experimental drug, and then invited to take part in a clinical trial?
When it came to under-representation of women in clinical trials more generally, he said “systemic bias” was to blame and challenged people to ask themselves: “At what point do you become an active agent of change?”
“Platitudes” would not cut it, he said, before controversially suggesting trials should not be funded or green-lighted unless they included “an acceptable population of women”. The same went for other under-represented populations, he added.
Investor interest – reaching that inflection point
Despite the ongoing challenges, Dr Arbat of Oriva said “a clear shift” was now underway in women’s health, with “increasing investment and advancing technologies enabling more robust, science driven development”.
Freya’s Acosta said a new generation of “targeted treatments” was starting to emerge that would “fundamentally change the standard of care for conditions that have historically been underserved..
Returning to the title of the symposium, From Intractable to Investable, entrepreneur and investor Andy Richards, CBE, of Cambridge Angels, urged participants to recognise that while investment “moves in waves or cycles”, it was often not easy to spot what “triggered” those movements.
“Obesity was uninvestable 10 years ago,” he noted. “What changed? It wasn’t all about the science. “These things can change very quickly. And understanding the triggers, and even working proactively on some of those triggers, can be useful.”
About the author
Stephen Adams has been an associate director at Optimum Strategic Communications, a specialist life science communications agency, for two years. Prior to that he was a health journalist, spending six years at The Daily Telegraph and then a decade at The Mail on Sunday.
