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Drug Development Insights from the 36th Annual J.P. Morgan Healthcare Conference

Posted on January 19th, 2018 by in Pharma R&D

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The 2018 J.P. Morgan Healthcare Conference, held in San Francisco from January 8-11, came and went in a whirlwind. In just a few days, there were hundreds of speeches, presentations, panels and a tremendous amount of networking. The conference was devoted to the business of healthcare, with announcements of mergers making headlines and discussions on subjects like branding, managing costs, and the role of big data. There was also much that addressed concerns specific to those working in pharmaceutical R&D.

The hottest topics at the conference included the emerging areas of gene therapy, mRNA and CAR-T. Meanwhile, there was talk of immuno-oncology, one of the most exciting new fields in recent years, being viewed as over-crowded. Simon King of First Word Pharma predicted that, as a result, “significant focus will be sharpened on emerging clinical approaches in this field, which offer potential differentiation versus competitors.”

The first day of the conference featured a key note address from Bill Gates, whose charitable foundation has had a special focus on global health issues. Gates emphasized how those needs intersect with today’s research in biotech and pharma. “Many of the solutions you’re working on – harnessing the immune system to tackle cancer, unraveling the mysteries of the brain to treat Alzheimer’s, and learning how bodies absorb nutrition to address the obesity epidemic and other diseases – also have clear applications in global health,” he said.

“You may be interested in developing products for rich-world markets, but the breakthroughs happening in your labs can also save millions of lives in the world’s poorest countries,” he explained, suggesting that insights learned about obesity could eventually find application in the fight against undernutrition, or that research into treatments for neurodegenerative disorders could ultimately help with the cognitive development of impoverished children. And it’s a two-way street. “We are backing companies like CureVac and Moderna on mRNA approaches for vaccine and drug development, which have the potential to help us tackle cancer,” he noted. “This approach is also intriguing as a potential immunological intervention for HIV, malaria, flu and the Zika virus.”

Among the hundreds of presenters at the conference was Alnylam Pharmaceuticals, a 15-year old company now preparing to move to the commercial stage with the forthcoming launch of patisiran, which treats hereditary ATTR amyloidosis, a rare orphan disease. The drug is expected to get FDA approval, which will be a big win for RNA interference (RNAi) therapeutics. RNAi is “a natural process in all of our cells that manipulates the genome,” said Alnylam president Barry Greene in an interview with The Motley Fool. “Today’s drug works by binding to protein and stopping protein action. We stop the protein from being made in the first place. If you had a leaky faucet in your kitchen, today’s drug works by mopping up the floor; we shut off the spigot. And we can leverage RNA interference to create an entirely new class of innovative medicines.”

While research of the microbiome is still in early stages, it’s viewed as having a great deal of potential and generated interest at the conference. As Ned Pagliarulo and Lisa LaMotta reported in BioPharma Drive, “A session on the microbiome noted there is more to the space than fecal transplants and ‘bugs as drugs.’ In fact, it might surprise some to hear a number of therapeutic areas are now being investigated as potential spaces where the microbiome could play a role. Traditionally, microbiome research has focused on the gastrointestinal tract and infectious skin disorders, but panelists suggested oncology actually could be a hot space as well. One participant even suggested the microbiome in cancer could outgrow immuno-oncology, and is only about five years behind.”

Presenting on the third day of the conference, Stephen Dilly, CEO of Aimmune Therapeutics, talked about the critical unmet need in peanut allergies, one of the most common food allergies (and growing). Aimmune is in Phase 3 trials of their oral biologic immunotherapy, AR101, designed to protect peanut allergic patients aged 4-17 from suffering significant allergic reactions. The goal is to reach a “bite-proof level of protection,” meaning a child who is taking the drug could go as far as accidentally biting into something containing peanuts and spit it out without experiencing a significant reaction. Aside from preventing hundreds of deaths and emergency room visits per year, providing this type of protection stands to relieve the constant anxiety that comes with worrying that simple contact with a common food product could result in death.

A recurring theme at the conference was the impact of technology. In a blog post on the conference’s website, Michele Colocci and Jeff Stute, co-heads of Global Healthcare Investment Banking, highlighted artificial intelligence, 3D visualization, drones and even blockchain as innovations that have the power to transform all aspects of modern healthcare, from analyzing patient information to helping surgeons operate to securing confidential data. In his address to investors, Alex Gorsky of Johnson & Johnson articulated the point by saying, “Going forward we won’t be classifying ourselves as just a healthcare or biopharmaceutical industry, but we’ll be a healthcare biopharmaceutical technology industry.”

This is already understood in drug development, where AI and 3D visualization have demonstrated great potential for accelerating research. At next year’s J.P. Morgan Healthcare Conference, we can be certain that these technologies will have developed in untold ways, and joined by many more fascinating new advancements.


 

All opinions shared in this post are the author’s own.

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