Cardiometabolic disease is an overlooked therapeutic space that represents a huge and growing need as the global population ages, according to panelists speaking at Biotech Showcase.
Cardiometabolic disease is an overlooked therapeutic space that represents a huge and growing need as the global population ages, according to panelists speaking at Biotech Showcase during J.P. Morgan Week.
Specifically, cardiology and endocrinology products account for less than 10% of all pharmaceutical products in clinical development, according to data from Evaluate Pharma, despite a global population whose median age is steadily increasing (from 23.6 years in 1950 to 31.0 in 2020).
The reason for this limited investment is related directly to the size, complexity and duration of cardiometabolic trials, Komathi Stem, chief operating officer at Glooko, explained. And, “There’s been a significant amount of failure that daunted investors.”
That’s changing now, she said. Mechanistic understanding of the condition has increased, the standard of care has evolved, polypharma (combination therapies) will soon become generic and medical devices – including wearables – are enabling real-time patient monitoring. Combined, Stem said all this means “Integrated solutions are increasing the probability of the technical success of a molecule,” as well as delivering dynamic responses and more complete approaches to patients.
“We’ll see a lot coming out in the next few years to address multiple questions at once,” Crystal Nyitray, CEO of Encellin, predicted. “We’re seeing a shift in how therapeutics are approached. Many are looking at small molecules. We’re focused on cell therapy and encapsulation for curative approaches.”
The key, as with any program, is delivering value for investors, Karin Conde-Knape, SVP, global drug discovery at Novo Nordisk, said. “As we approach discovery in a different way, the more we must understand the residual risks in the populations and add more support. That will enrich trials and their outcomes, and thus reduce the size of the trials. We are at the point at which there is plenty of opportunity to rethink how we develop therapies and what type of therapies we bring to patients, and thus generate more investor excitement. This is an area in which there is still value, and it’s less scary than many other therapies, such as oncology.
“The chances that a single company will have all the tools required to provide integrated health care solutions are not high,” Conde-Knape continued. Therefore, determine which partnerships you need in order to provide an integrated solution. Then, “ensure the partners will generate value for (external and collaborative) stakeholders and identify the kind of data you need to do that. This can be tricky,” she admitted, and depends on whether you’re attempting to cure the condition permanently, or only for a few years.
The anticipated duration of benefit is changing the way payers are thinking about reimbursement in the cell and gene therapy space. What’s overlooked, however, are the benefits derived from the prevention or delay of onset of secondary complications, such as retinopathy and neuropathy in diabetes. To address that, Nyitray advised learning how preventive medicine has dealt with that issue.
“Subscription-based models are interesting, and I’m encouraged by the chance to align incentives and to have the support from the (involved) agencies,” Nyitray said. Value-based pricing, in which the ecosystem is accountable for outcomes, is another promising option.
Digital medicine benefitted tremendously from the pandemic, in which the industry learned to work remotely, Stem said, “but education is still needed, along with ways to make it simpler.” This is particularly true in the area of pediatrics, which needs frequent refreshes to keep children engaged as they mature, and gerontology, which needs larger screens and keypads and straightforward application design.
The need for good user interface transcends age and infirmity, of course, and Stem urged application designers to focus on the patient and create a path that is as frictionless as possible. For example, she said, “For onboarding, reduce the number of clicks needed, and enable data to auto-populate forms. Make it as passive as possible to get the data.” For monitoring devices, like glucose monitors, this means enabling automatic data uploads that require no user interaction at all.
The second part of digital plus drug combinations is managing the behavioral component. Tools are readily available to make people more aware of their behaviors, from step counters and caloric intake apps.
“Make the tools simple,” Conde-Knape said. “For example, ‘Here are the foods you like, and here’s what to consider in your weekly menu.’ I strongly recommend that the more we support the patients, the less thinking they need to do to manage their conditions, and the better it will be for everyone.” The challenge is preventing users from becoming bored.
Engagement is particularly important for long-term monitoring applications. “Leverage the power of passive monitoring to keep patients in these safe windows,” Nyitray advised. “Having more data points helps keep patients safer longer. The challenge for patients is that you’re preventing disease, so they aren’t seeing the immediate effects,” and so may not understand the benefit.
In terms of rolling out digital and drug combinations, the panelists urged focusing first on niche populations that can realize benefits quickly. In diabetes, for example, that may be patients with or at risk for severe hyperglycemia episodes. That high-risk subpopulation can be helped now and at a reasonable cost. Gradually, similar programs can be delivered to other groups and ultimately to the more than 1.5 million diabetics in the U.S. Eventually, applications can expand globally. Progress, the panelists agreed, will be incremental.
Looking forward 5 to 10 years, Stem said she was “super excited about precision engagement. The importance of using artificial intelligence and machine learning with real world data will help us deliver a more personalized engagement tool for clinicians, so they can spend time with those at most risk.”
For Nyitray, the excitement is the opportunity to use biology more thoroughly, “going beyond mechanistic understandings to harness the power of cells as living medicines, so they become sophisticated machines to sense conditions and, in response, secrete therapeutics.”
Conde-Knape said gene editing technical advancements were most exciting to her. It is made even more so by “a better understanding of residual risk, in which editing a single gene or expression protein can disrupt the treatment paradigm.”
