Every January, downtown San Francisco is taken over by a flock of investors, bankers, biotech companies, and scientists attending the annual JP Morgan Healthcare Conference. This meeting looks at the healthcare advancements over the past year and predicts the disease areas and technologies that will see the most progress and success in 2016.
According to some of the experts at the event, regenerative medicine and stem cell research are experiencing impressive, accelerated advancements, which has peaked the interest of investors, biotech, and pharmaceutical companies.
Because these are such fast paced fields, the Alliance for Regenerative Medicine (ARM) hosts the Annual Regenerative Medicine and Advanced Therapies State of the Industry Briefing during JP Morgan to discuss the recent progress and outlook for the industry in the coming year.
What happened in 2015 and what’s next?
ARM’s 6th Annual Briefing was open to the public and drew over 300 people on Monday morning. The meeting opened with an industry update from Edward Lanphier, ARM Chairman and President/CEO of Sangamo BioSciences. Then two panels featuring top leaders from biotech and pharmaceutical companies discussed the 2016 clinical data forecast and the promise of regenerative medicine and advanced therapies in oncology (cancer).
With an upbeat attitude, Lanphier gave an overview of clinical development progress in 2015, with 20 approved products worldwide and over 600 clinical trials both from academia and industry. More than 40% of these ongoing clinical trials are in cancer while approximately 12% are in heart disease/injury. These trials are not limited to Phase 1 either. In 2015, there were 376 in Phase 2 (compared to 200 in 2014) and 64 in Phase 3 (compared to 39 in 2014).
Two other areas Lanphier emphasized were CAR-T and other cell-based immunotherapies and gene therapy programs for rare diseases. He ended with 2015 statistics on clinical milestones in various disease and therapy programs, key company IPOs, the financial landscape, and predictions of major anticipated data from clinical trials in 2016.
It was a lot to take in, but this was definitely a good thing and a sign that the areas of regenerative medicine and advanced therapies are thriving. If you want more details, you can check out ARM’s State of the Industry presentation.
Major Theme: Data is King
The major theme that cropped up during the industry update and panel discussions was the importance of producing meaningful clinical data to get positive outcomes in regenerative medicine.
This was succinctly put by panelist Sven Kili, head of Gene Therapy Development at GlaxoSmithKline:
“I would say “Data is King”. A great idea is fantastic, passion is wonderful, and most companies will buy into a strong management team, but that only gets you so far. After that you need to have data, and you need to have a good plan for going forward.”
Kill added that there’s the need to work with the FDA to change the regulatory process, saying the FDA is, understandably, cautious about working with therapies that can alter a person’s genome permanently. However, he said there needs to be serious discussions with the FDA about how to speed up the process, to make it easier for the most promising projects to get approval.
Edward Lanphier also talked about the industry’s new focus on clinical data and the questions that arise when trying to advance regenerative medicine research into approved treatments and cures for patients:
“How do we communicate the value of curing blindness? How do we think about pricing that? What do we think about [drug] reimbursement? For rare diseases, we aren’t trying to talk about acute treatments – we are talking about one-time, curative outcomes. And the value and benefit to patients in this is enormous. This is what we are trying to do, and on the cusp of, in terms of generating both approvable data and also the proof of concept data that then allows us to drive that next value inflection point in terms of financings.”
The Future Looks Good
After listening to the briefing, the future of regenerative medicine and advanced therapies certainly looks bright. As Jason Kolbert, head of Healthcare Research at the Maxim Group, said:
“This industry is now rapidly maturing and regenerative medicine and gene therapy have great things in store for the next decade.”
Usman Azam, Global Head of Cell and Gene Therapies at Novartis, had a similar outlook:
“We now are going from proof of concept to commercial availability of a disruptive innovation within seven years. If somebody had said that to me four years ago, I would have said, not possible. But that gives you a sense of how quickly this field is moving.”
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