Along with kicking off biopharma dealmaking season, the JPMorgan Healthcare conference offers CEOs a chance to pitch their companies to a prime-time investor audience. Among the most anticipated talks of the virtual meeting’s opening day was that of Biogen CEO Michel Vounatsos. 

The launch of Biogen’s Alzheimer’s drug Aduhelm has been dismal. Even before its US approval in June, the med has been hounded by doubts from the medical community about the product’s efficacy and safety profile. But it was on the subject of the drug’s initial price – $56,000 a year – that Vounatsos made a surprising admission. 

“We were proven wrong by the reaction of the community, physicians and eventually the reaction of patients and policy leaders,” Vounatsos acknowledged, in response to a question from JPMorgan analyst Cory Kasimov about the pricing rationale. 

He then said that the company’s decision to lower the Aduhelm price by nearly 50% was “courageous,” adding, “We could have waited and dictated the need to change over time based on competitive pressure or an access decision.”

The CEO said the initial price was “evidence-based,” that teams had worked on it “for more than a year” and that “it was a price that eventually we could defend.”

Whether providers and insurers agree is another matter. On the data front, Biogen is beginning a post-marketing trial that will take four years. According to Vounatsos, it’s also working to publish Aduhelm’s pivotal clinical trial data “very soon” in a “top-tier journal.” 

The Department of Health and Human Services Secretary Xavier Becerra on Monday announced that, given Aduhelm’s 50% price cut, “there is a compelling basis to reexamine the previous recommendation.” That means Medicare may reconsider what would have been a hike in premiums, revealed last November.

Meanwhile, Aduhelm’s cloudy reimbursement picture could become clearer by this Wednesday, as Medicare is expected to release a draft coverage decision that will have major market-access ramifications. Alisha Alaimo, president of Biogen’s US operations, said, “We can’t speculate as to [the outcome] because we actually don’t know.”

Perhaps equally anticipated (by those interested in gene therapy, at least) was Sarepta CEO Doug Ingram’s update from “Part 2” of an ongoing mid-stage clinical trial of its gene therapy SRP-9001 for Duchenne muscular dystrophy. Results for “Part 1” of the study, released last January, were less than stellar.

Changes observed at one year for the 21 placebo crossover patients showed a statistically significant, roughly two-point benefit in the North Star Ambulatory Assessment (NSAA) vs. the company’s external control. 

These results, Ingram said, were`”unequivocal” that study 102 “Part 2” adds “additional weight” to evidence supporting the thesis that SRP-9001 changes the course of DMD. Moreover, based on the three studies so far conducted on SRP-9001, Ingram added, “You can only imagine the level of confidence and conviction that we have” in the upcoming Phase 3 trial, study 301, also known as EMBARK.

One analyst’s convictions were not as strong, however. 

“Very few additional details were provided [to] challenge the interpretation of these findings,” noted Raymond James analyst Danielle Brill in an investor note Monday. Brill would have liked to see data broken out by age cohorts. The two-year follow-up was only provided for four- and five-year-olds. 

“The bottom line,” Brill wrote, is that “today’s update did not provide the clarity we were hoping for, and we do not view these data as de-risking for the SRP-9001 program.”

The FDA will want the company to wait until EMBARK reads out data before submitting a filing, given the variable nature of DMD and the modest treatment effect of SRP-9001, Brill added.