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After a one-day respite from JPM tradition, the rain returned this morning, leading to some dampened spirits and at least one canny promotional effort. (We’ve still got plenty of STAT-branded umbrellas at our coworking space, if you’re in need.) Anyway, here are the highlights from Day 2.
Will Sarepta earn a participation trophy from the FDA?
Sure seems like it, based on remarks made by Peter Marks, the agency’s chief gene therapy regulator, at STAT’s event on Monday night. By trophy, we mean full approval of some kind for Elevidys, Sarepta Therapeutics’ treatment for Duchenne muscular dystrophy.
“Hypothetically, if you had a product that used an aggregate scale and all of the components of that aggregate scale looked pretty good, or nearly all of them look pretty good, but the aggregate, for various reasons, didn’t come out perfectly. We approved products based on that,” Marks told Adam Feuerstein onstage at the STAT event.
To be clear, Marks declined to comment specifically about the ongoing FDA review of Elevidys. However, he clearly suggested the failure of an Elevidys confirmatory study in October didn’t bother him all that much.
Sarepta’s shares were up about 15% midday.
Pfizer, are you listening?
The pharma giant is developing its own gene therapy for Duchenne. A Phase 3 study is underway with final results expected at some point this year. Based on Marks’ comments, Pfizer could — and likely should — submit for approval, even if the primary goal of the study isn’t met.
Novo isn’t exactly sweating GLP-1 pills
While excitement builds for the GLP-1 obesity pills Eli Lilly and other drugmakers are developing, Novo Nordisk CEO Lars Fruergaard Jørgensen has a word of warning: watch out for safety.
Novo’s injectable semaglutide (sold as Ozempic and Wegovy) is made up of peptides, which are large molecules, and has a long track record for safety. But the pills that other companies are studying are made up of small molecules, which could get into different parts of the body and also introduce new side effects. Pfizer stopped trials of one of its small-molecule candidates due to elevated liver enzymes. “There will be very low regulatory appetite for introducing oral treatment that comes with some safety issues,” Jørgensen said.
Novo does have an oral version of semaglutide, but in peptide form, not as a small molecule. This is sold to diabetes patients as Rybelsus, and Novo has also tested a much higher dose for obesity. Jørgensen said that a key hurdle with launching this type of treatment would be building up enough supply of the underlying ingredient. The oral treatment requires a large amount of the ingredient for enough to be absorbed and circulated in the body, and Novo is currently “many-fold ramping up” supply capacity, the CEO said.
A clever and depressing justification for high U.S. drug costs
Another interesting thought from Jørgensen, the Novo Nordisk CEO. He talked a lot during his presentation about the health economics of GLP-1 drugs, arguing that they return more money to the economy than they cost. And he was asked how that changes in the U.S., where they are more expensive, compared to other countries.
Jørgensen’s answer: Everything else in U.S. health care that GLP-1 drugs might prevent by reducing obesity (he didn’t say, but think hip replacements, heart bypass surgeries, and cancer treatment) is also much more expensive in the U.S., so the math still works.
This isn’t a new idea. But it will probably make you happy if you are looking to price a new GLP-1 drug in the U.S., and depressed if you’re trying to imagine a way to lower America’s health care bill.
Today on the Cytokinetics show
J.P. Morgan began with the Wall Street Journal reporting that Cytokinetics, maker of an effective treatment for a genetic heart disease, was close to selling itself to Novartis. Hours later, Reuters reported that AstraZeneca and Johnson & Johnson had also submitted bids for the company and that a deal could materialize as early as this week.
The fun part of this all happening in the middle of the industry’s largest investor conference is that the relevant CEOs are frequently in front of live microphones. Vas Narasimhan, sitting across from CNBC’s Jim Cramer, declined to comment specifically on a Cytokinetics acquisition but did issue a sort of implied denial. “I want to highlight that while we look at larger deals, our M&A strategy is bolt-on,” Narasimhan said, which means focusing on “sub-$5 billion assets.” Cytokinetics, with a market cap of $10 billion, would fall outside that definition.
This of course could be multi-dimensional chess playing out in public while the two companies negotiate in private, a feint to confuse rival bidders, or a classic case of the world reading too much into what amounts to boilerplate words from a pharmaceutical CEO. Whatever the case, Cytokinetics’ share price fell as much as 10% after Narasimhan’s interview.
Party exit lines you only hear at JPM
“Great seeing you, but I gotta go see Takeda about a molecule.”
More reads
- JPM 2024: FogPharma’s new CEO, Mathai Mammen, unveils ‘contrarian’ plan in oncology, STAT
- Doudna institute hatches plan to ‘cure hundreds of diseases’ left behind by CRISPR revolution, STAT
- GSK to acquire Aiolos Bio in $1.4 billion deal, picking up an experimental asthma drug, STAT
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