BIO2025: Sanofi R&D, FDA's Martin Makary, Paying For New Therapies
By Ben Comer, Chief Editor, Life Science Leader
The annual BIO International Convention, held this year in Boston on June 16 through 19, is a choose-your-own-adventure kind of meeting. With educational programing, theme-based session tracks, one-on-one fireside chats, and opportunities to schedule meetings with hundreds of company executives (among many other things – like playing ping pong or pickle ball on the convention center “wellness” grounds outside), it’s unlikely that any two of the more than 20,000 attendees had the same BIO experience.
I’ll be sharing more from my experiences and conversations at BIO in the coming weeks, but for now, here are a few thoughts on three events that stood out this year.
Sanofi’s R&D Facility In Cambridge
R&D leaders at Sanofi invited a group of editors and reporters to visit the company’s three-year-old R&D facility in Cambridge, MA, in the morning on day two of the BIO conference. The eight-floor facility offers “end-to-end” product discovery to finished product — including CMC — for a range of modalities, explained Matt Truppo, global head of research platforms and computational R&D. From biologics and small molecules to genetic medicines, vaccines, and an emerging ‘nanobody’ platform, the facility brings in experts of many stripes, and represents “a cultural transformation as much as a technological transformation” at Sanofi, said Truppo.
Sanofi’s concept of “immunoscience” — an approach to scientific research and discovery based on the role of the immune system in how diseases work and how they can be treated, or prevented — serves as the connective tissue between other therapeutic areas, including immunology and inflammation (I and I), neurology, rare diseases, oncology, vaccines, and Type 1 diabetes. But that’s not all: Sanofi is also building therapeutic area “adjacencies” to immunoscience, such as ophthalmology, and in the future, drugs that improve longevity and health span, according to Mike Quigley, chief scientific officer and global head of research.
In the I and I space, Sanofi aims to unlock new mechanisms that can “break the efficacy ceiling” of existing products, or make them more convenient to administer for patients. Sanofi expanded its immunology pipeline during the first half of 2025 through bispecific antibody deals with Dren Bio and Earendil Labs, and the company is “all in on AI” across R&D discovery/target identification and development, said Quigley. The company’s nanobody platform, obtained through the acquisition of Ablynx in 2018, is revving up after some initial candidate failures in 2023; the platform can now generate “pente-specific antibodies,” or nano molecules with five binders, per Quigley.
At the beginning of June, Sanofi announced plans to purchase Blueprint Medicines in a deal worth up to $9.5 billion, representing another big swing in immunology, including rare immunological diseases. Sanofi’s R&D leaders also talked up the company’s “Biologics x AI Moonshot,” or BioAIM, which includes academic and industry partners — such as Stanford and MIT, Fable Bio and BioMap — which can create “protein language models built on amino acids” as the feeder language. In the small molecule arena, Sanofi full AI robotic automation will come online in 2027.
Exciting stuff, which will require an FDA that’s on the level. In response to a reporter’s question about the implications of shake ups at the NIH and Advisory Committee on Immunization Practices (ACIP), Quigley said “we’ve had no delays with the FDA, but our communications have shifted to written,” presumably in lieu of phone or in-person communications.
Fireside Chat With FDA Commissioner Martin Makary, M.D., M.P.H
BIO President and CEO (and Life Science Leader Editorial Advisory Board Member) John Crowley was assertive in his fireside chat with FDA Commissioner Martin Makary, M.D., M.P.H., asking what Makary’s first few days at the agency were like, given the DOGE activities and layoffs. Makary, who identifies as a surgeon first and foremost (make of that what you will), responded that the FDA “is on track to meet all PDUFA targets,” anecdotal evidence to the contrary (GSK’s Nucala, Stealth Biotherapeutics’ elamipretide) notwithstanding.
On firing and hiring, Makary emphasized the “tremendous redundancies” in FDA communications, legislative affairs, IT and HR departments, and his goal to get the agency down to “2019 staffing levels ... we’re at 2020 [levels] probably right now.” The FDA is however “hiring scientists,” including scientific reviewers, and “wants fresh ideas,” said Makary. “I have reinstated people” and “no cuts are being made to scientific reviewers and inspectors,” he said. “We want to create a culture of teamwork,” said Makary, compared to the FDA’s current “fiefdom culture,” where different centers in the agency don’t talk to each other.
Makary also announced his ambition to shorten FDA review times from 10 to 12 months, to one to two months, through a new priority review voucher pilot program. The program will allow sponsor companies to start the review process by submitting partial information, including CMC data, prior to final clinical data for an NDA, or BLA application, for example. Once the clinical data is available, it can be submitted as an addendum, said Makary. The program also will include an “almost real-time dialogue” with sponsor companies. “I want to see American drug developers thrive,” Makary proclaimed, which means better communication between FDA and drug developers. “We have to continue to be creative in how we review medications” at the FDA, he said.
Paying For Innovative New Therapies
Value-based contracting pioneer Michael Sherman (background on that topic here), who is now running his own consulting shop called Sherman Healthcare Solutions, surfaced on a panel titled “Reimbursement Innovation: Unlocking the Future of Advanced Therapy Access.” Joining Sherman on the panel were Kamal Menghrajani, former assistant director, office of science and technology policy at The White House, and William Shrank, MD, a venture partner and former chief medical officer at Humana, and UPMC. (Shrank revealed that he started a new company — currently “in stealth” — focused on treatment affordability.)
Moderated by George Eastwood at the Emily Whitehead Foundation, the panelists decried Independent Blue Cross’s (IBX) decision not to cover new drugs approved through the FDA’s accelerated approval pathway for the first 18 months, except for cancer drugs. Noting that drugs receive accelerated approval due in large part to urgent and unmet patient needs, the panel hoped IBX’s policy won’t “metastasize” into other insurance policy coverages. “This policy came in right after Luigi [Mangione], which really makes you wonder what they were thinking,” said Sherman.
Sherman also noted that some payers are still refusing to cover GLP-1 drugs, despite all of their apparent benefits. “That’s new to our country, these kinds of barriers to new treatments,” said Sherman.
Shrank made the observation that figuring out how to pay for innovative, effective drugs in a sustainable way is a much easier task than say, extracting, manipulating, and inserting a gene into a patient successfully. “Buy and bill is a huge risk and cash flow problem for providers … but payers can fix this,” for example by driving volume to high quality providers and paying the up-front cost for a new drug, he said. Better provider networks and better patient risk pools could help alleviate payer risks, he said.
To the question of what happens when one employer healthcare program pays a large up-front medication cost for an employee, only to lose out on the long-term cost savings when that employee lands a job somewhere else, Shrank said “there are ways to create continuity in tracking patients through a value-based contract, across jobs and across insurers.”