In normal years, thousands of biopharma executives and investors would be cramming into the halls of the Westin St. Francis hotel in San Francisco for the annual J.P. Morgan Healthcare Conference.
As with other meetings during the pandemic, though, the Westin has been replaced with a website, conference rooms with virtual ones. Executives are presenting safely from their homes, while investors dial into virtual Q&A sessions.
Everything else, however, remains the same: four days of wall-to-wall presentations as pharma and biotech companies attempt to put their best foot forward.
Biopharma Dive will be keeping track of what’s to come with a series of short reports from the conference. The first is below.
For Biogen, a ‘transformative year’ awaits, but what kind?
Excepting coronavirus vaccine developers, there’s perhaps no company in the drug industry facing higher stakes this year than Biogen.
In 2021, regulators in the U.S., Europe and Japan will decide the fate of its experimental Alzheimer’s drug aducanumab. If approved, aducanumab would become the first marketed medicine to treat the underlying cause of the memory-robbing disease and likely a multi-billion dollar product. A rejection, meanwhile, would be devastating and intensify the scrutiny on Biogen’s pipeline and eroding business.
On Monday, Biogen CEO Michel Vountasos pointed to several milestones outside of aducanumab that could soften the blow if things don’t go well. Its biosimilars business is growing, he noted, as is its revenue base outside of the U.S. Spinraza, a treatment for spinal muscular atrophy, remains the top-selling medicine for the disease, despite emerging competition, and the company is working to bolster its position. Several Phase 3 trials could produce results this year, including three under a brain drug alliance Biogen forged with Sage Therapeutics in November.
“This is a transformative year for Biogen,” said CEO Michel Vounatsos, during the company’s virtual presentation. Increasing investments in ophthalmology, psychiatric disorders and acute neurological problems such as stroke are part of the company’s plan to diversify, he added.
Yet the immediate risk to Biogen’s future is very real, and the focus on aducanumab during Biogen’s Q&A session reflected those concerns. A U.S. rejection of aducanumab by the March 7 deadline seems more likely following the decidedly negative vote of an independent advisory panel in November. Vounatsos and chief medical officer Al Sandrock both said they were “surprised” by the decision, and expressed confidence that aducanumab still has a good chance of approval.
“The advisory committee vote is non-binding,” Sandrock said. “And there's plenty of precedents for the FDA to not go along with the vote of the committee.”
The FDA typically sides with its advisory panels, though it’s not required to do so.
Why Vertex may go shopping
Vertex has been one of the industry’s fastest-rising companies over the past several years, thanks to the steady growth of a now-dominant cystic fibrosis business. That has put the biotech, which lost money for decades, in an unfamiliar position: consistent profitability. Vertex ended the third quarter with $6.2 billion in cash on hand, and no debt, said CEO Reshma Kewalramani.
Vertex is more aware than most of how quickly fortunes can change in biotech, having seen competition wipe out its once-promising hepatitis C business nearly a decade ago. But unlike before, the company has the financial muscle to brace for competition by acquiring new assets. And Kewalramani indicated Vertex does plan to put some of its cash to use.
The biotech has become a more active dealmaker as its CF business has matured, forming alliances with, or acquiring, biotechs pursuing cutting-edge science like CRISPR gene editing, protein degradation and messenger RNA. The programs in each of those deals, however, are early-stage. Vertex is now hunting for more advanced, and likely more expensive, assets.
“Growing financial firepower,” she said, “allows us now to look at mid- and late-stage assets.”
How Sarepta’s new data hint at a gene therapy turnaround
Sarepta came into the conference reeling, having reported disappointing results last week from a key study of its closely watched gene therapy for Duchenne muscular dystrophy. Treatment didn’t improve all patients’ motor function in a Phase 2 trial, data showed, although the therapy did appear to help younger participants more. Shares plummeted 50%, erasing billions of dollars in market value overnight.
Sarepta CEO Doug Ingram described himself as “utterly gobsmacked” when he first saw the negative data, which the company blamed on an unlucky randomization of participants between treatment and placebo. But Ingram gave investors new hope Monday: Encouraging signs from the biopsies of 11 patients who first got a placebo and then received the gene therapy after 48 weeks.
The results, Ingram said, show these patients produced similar levels of microdystrophin — the protein Sarepta’s gene therapy helps produce — to what was observed in patients in an earlier trial. The numbers were also superior to what Sarepta reported from those who received the gene therapy initially.
These so-called crossover patients will be followed for 48 weeks to measure whether their motor function improves. That could set up another critical data disclosure from Sarepta, which aims to prove its gene therapy still has a future. The biopsy results “get us excited about what we could see at the readout of part two [of the trial], by the end of this year,” Ingram said.
Sarepta shares climbed 9% on the update.