Last week, the Ovation team had the privilege of attending the STAT Summit in Boston, a premier gathering of healthcare and life sciences professionals. The event brought together leaders from top pharmaceutical companies, health systems, philanthropic foundations, academic research centers, and patients who offered their insights into the current healthcare landscape. Here are our top takeaways from the talks that focused on advancements in biomarker and drug R&D.
The Power of Persistence in Biomarker Discovery and Drug Development
Biomarker discovery and drug development requires perseverance, a theme echoed by several speakers at the summit. Emma Walmsley, CEO of GSK, spoke about their commitment to antimicrobial resistance (AMR), calling it the “slow pandemic.” She highlighted GSK’s role as a leader in this effort with three assets in the pipeline that address segments where resistance has already developed. In Walmsley’s view, AMR is a reflection of GSK’s commitment to meaningful societal work while still running a profitable business. It’s an area they’re not shying away from, despite other pharma companies backing out due to lack of incentives.
Eli Lilly’s EVP, Chief Scientific and Medical Officer, and President, Daniel M. Skovronsky, MD, PhD, emphasized the benefits of big pharma’s ability to engage in prolonged research endeavors, like tackling obesity and Alzheimer’s disease: “At big companies, we have the wherewithal to ignore that noise and just follow the science and medicine.” He explained how a drug approval 10 to 20 years out is just too long for many biotech companies to get involved in, and one example of this is Lilly’s investment in chronic pain. In Skovronsky’s words, chronic pain drug development is “super unpopular, but we keep working on it.”
Another obesity powerhouse, Novo Nordisk, required perseverance in developing their blockbuster GLP-1 obesity drug, Wegovy. Lotte Bjerre Knudsen, PhD, chief scientific adviser at the Dutch pharma company, spoke of her journey with GLP-1, which she started working on in the 1990s when big pharma was only interested in small molecule treatments for diabetes. Her message for the audience was to have patience—it takes time to progress something that is incredibly important to patient health.
Boehringer Ingelheim’s SVP, Site Head Discovery Research US & Head of Global Immunology & Respiratory Diseases Research, Carine Boustany, PhD, PharmD, spoke to how Boehringer’s status as an independent company has allowed them to take a unique approach to innovation and persist through challenges. Her team has followed the science and remained steadfast through failures, all backed by Boehringer’s philosophical approach to drug development. “We don’t think in terms of financial quarters…we think in terms of generations,” said Boustany.
The most inspiring perseverance story came from Michael J. Fox who spoke at the summit virtually. Parkinson’s disease (PD) is the second-most common neurodegenerative disorder in the United States with approximately a half a million Americans diagnosed. However, some experts estimate that twice as many people have PD without being diagnosed because there is no definitive diagnostic or screening test.
Deborah Brooks, CEO and co-founder of the Michael J. Fox Foundation, who also spoke virtually at the summit, explained that identifying PD an entire decade or more before symptoms begin would be a huge opportunity to slow progression. This mission drove the foundation’s main clinical study, the Parkinson’s Progression Markers Initiative (PPMI).
Findings from the PPMI study were recently published in The Lancet Neurology, showing that α-synuclein seed amplification assays (SAAs) have the potential to differentiate people with Parkinson’s disease from healthy controls. Currently, alpha-synuclein can only be detected by taking a spinal tap, but scientists hope that it could be detected in more accessible ways such as in blood, skin biopsy, or nasal swab. This breakthrough was the result of the decade-long longitudinal PPMI study, and is evidence of what patience and perseverance can bring to a disease that appears to be undetectable.
R&D Shift from Therapeutic Area to Underlying Mechanism
Several pharma leaders mentioned a shift in how R&D thinks about areas of interest, moving from focusing on therapeutic areas to the underlying mechanisms of disease. Biogen’s CEO, Chistopher Viehbacher, discussed expanding the company’s R&D portfolio to avoid relying on a single drug, and furthermore, suggested a shift away from thinking about diseases on the basis of their clinical symptoms.
Viehbacher explained that approach is why immunology is a natural fit for Biogen. The company has been involved in multiple sclerosis for several years, which in his view, is really an autoimmune disease despite its typical categorization as an affliction of the central nervous system. He also referenced his previous experience at Sanofi, which followed the IL-15 pathway and ended up with several indications outside of therapeutic areas of interest for the company. “If you started therapeutically, you would never have developed a drug like Dupixent,” said Viehbacher.
This approach was also discussed by Boustany from Boehringer Ingelheim, who emphasized their focus on seeing patients holistically, especially in cardiometabolic and renal diseases. She told the story of Spevigo, the first FDA-approved treatment for adults with generalized pustular psoriasis (GPP). Her team first started working on the underlying mechanism of action, IL-36, in 2009 in the field of cirrhosis. Over the years, they ultimately found the strongest signal in GPP. She also mentioned the expanded indications for Boehringer’s Jardiance, which is now on the market for type 2 diabetes, heart failure, and chronic kidney disease.
Knudsen from Novo Nordisk, shared her passion for one biology working across multiple diseases, which was a key factor in the development of liraglutide, a medication for diabetes that was eventually enhanced to semaglutide (Wegovy), the weight loss drug. The expansion to tackling obesity wasn’t an easy sell at first. Management wasn’t convinced that one molecule could contain multiple biological mechanisms, even in the face of clear evidence. Now, the times have changed, and Knudsen is excited for researchers to look to semaglutide for even more treatment opportunities including chronic kidney disease and NASH.
Genomics: The Catalyst for Next-Gen Innovation
Another theme that emerged was how genomics is driving innovation. This was best exemplified by the Michael J. Fox Foundation’s PPMI study and the follow-up studies using genomic sequencing data collected from patients to understand how different genes might affect Parkinson’s progression. Brooks expressed her conviction in the power of omic data—the foundation has made specimens and whole exome, whole genome, and RNA-sequencing data from the PPMI study available to qualified researchers.
Brooks also discussed the foundation’s involvement with the Global Parkinson’s Genetics Program (GP2), further demonstrating the significance of genomics in understanding the underlying mechanisms of diseases, emphasizing the need for diverse backgrounds and underrepresented populations.
Eli Lilly’s Skovronsky underlined the pivotal role of biomarkers when questioned about addressing the high cost and onerous patient experience of trying multiple drugs before finding relief. In his view, biomarkers are the ultimate solution to drug cycling and the problem would be immensely improved if we could better understand which patients are going to respond to which class of medications. Outside of oncology, this type of precision is woefully unavailable. But Skovronsky remains optimistic, especially about obesity, where Lilly is starting to understand there are more genotypically-defined types of the disease that could respond optimally to different therapies.
The Future of Genomics Research
The STAT Summit underscored that with unwavering perseverance, a deeper understanding of underlying disease mechanisms, and the transformative potential of genomics, we can continue to expect significant innovative breakthroughs from top pharma companies in the years to come.
At Ovation, we champion the transformative power of genomic and omic data in accelerating drug development innovations. With our expansive, high-quality genomic data linked to rich, longitudinal clinical data at population scale, we are positioned to help pharma R&D teams discover and validate the next generation of biomarkers and targets.
To learn more about how Ovation is helping life sciences organizations advance precision medicine, don’t hesitate to contact our team.