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After a one-day respite from JPM custom, the rain returned this morning, resulting in some dampened spirits and a minimum of one canny promotional effort. (We’ve nonetheless received loads of STAT-branded umbrellas at our coworking house, in case you’re in want.) Anyway, listed here are the highlights from Day 2.
Will Sarepta earn a participation trophy from the FDA?
Certain looks as if it, primarily based on remarks made by Peter Marks, the company’s chief gene remedy regulator, at STAT’s occasion on Monday night time. By trophy, we imply full approval of some sort for Elevidys, Sarepta Therapeutics’ therapy for Duchenne muscular dystrophy.
“Hypothetically, in case you had a product that used an combination scale and the entire parts of that combination scale seemed fairly good, or practically all of them look fairly good, however the combination, for varied causes, didn’t come out completely. We authorized merchandise primarily based on that,” Marks instructed Adam Feuerstein onstage on the STAT occasion.
To be clear, Marks declined to remark particularly in regards to the ongoing FDA overview of Elevidys. Nevertheless, he clearly urged the failure of an Elevidys confirmatory examine in October didn’t trouble him all that a lot.
Sarepta’s shares have been up about 15% noon.
Learn extra.
Pfizer, are you listening?
The pharma big is creating its personal gene remedy for Duchenne. A Part 3 examine is underway with closing outcomes anticipated in some unspecified time in the future this 12 months. Based mostly on Marks’ feedback, Pfizer may — and certain ought to — submit for approval, even when the first objective of the examine isn’t met.
Novo isn’t precisely sweating GLP-1 tablets
Whereas pleasure builds for the GLP-1 weight problems tablets Eli Lilly and different drugmakers are creating, Novo Nordisk CEO Lars Fruergaard Jørgensen has a phrase of warning: be careful for security.
Novo’s injectable semaglutide (offered as Ozempic and Wegovy) is made up of peptides, that are giant molecules, and has a protracted observe document for security. However the tablets that different firms are learning are made up of small molecules, which may get into completely different components of the physique and in addition introduce new uncomfortable side effects. Pfizer stopped trials of considered one of its small-molecule candidates because of elevated liver enzymes. “There shall be very low regulatory urge for food for introducing oral therapy that comes with some questions of safety,” Jørgensen mentioned.
Novo does have an oral model of semaglutide, however in peptide type, not as a small molecule. That is offered to diabetes sufferers as Rybelsus, and Novo has additionally examined a a lot larger dose for weight problems. Jørgensen mentioned {that a} key hurdle with launching the sort of therapy could be build up sufficient provide of the underlying ingredient. The oral therapy requires a considerable amount of the ingredient for sufficient to be absorbed and circulated within the physique, and Novo is at present “many-fold ramping up” provide capability, the CEO mentioned.
A intelligent and miserable justification for prime U.S. drug prices
One other attention-grabbing thought from Jørgensen, the Novo Nordisk CEO. He talked loads throughout his presentation in regards to the well being economics of GLP-1 medicine, arguing that they return more cash to the economic system than they value. And he was requested how that adjustments within the U.S., the place they’re costlier, in comparison with different nations.
Jørgensen’s reply: All the pieces else in U.S. well being care that GLP-1 medicine would possibly forestall by decreasing weight problems (he didn’t say, however assume hip replacements, coronary heart bypass surgical procedures, and most cancers therapy) can also be way more costly within the U.S., so the mathematics nonetheless works.
This isn’t a brand new thought. However it can in all probability make you content in case you are trying to value a brand new GLP-1 drug within the U.S., and depressed in case you’re making an attempt to think about a option to decrease America’s well being care invoice.
Right this moment on the Cytokinetics present
J.P. Morgan started with the Wall Avenue Journal reporting that Cytokinetics, maker of an efficient therapy for a genetic coronary heart illness, was near promoting itself to Novartis. Hours later, Reuters reported that AstraZeneca and Johnson & Johnson had additionally submitted bids for the corporate and {that a} deal may materialize as early as this week.
The enjoyable a part of this all taking place in the course of the trade’s largest investor convention is that the related CEOs are ceaselessly in entrance of reside microphones. Vas Narasimhan, sitting throughout from CNBC’s Jim Cramer, declined to remark particularly on a Cytokinetics acquisition however did difficulty a form of implied denial. “I wish to spotlight that whereas we have a look at bigger offers, our M&A method is bolt-on,” Narasimhan mentioned, which suggests specializing in “sub-$5 billion belongings.” Cytokinetics, with a market cap of $10 billion, would fall outdoors that definition.
This after all may very well be multi-dimensional chess taking part in out in public whereas the 2 firms negotiate in personal, a feint to confuse rival bidders, or a traditional case of the world studying an excessive amount of into what quantities to boilerplate phrases from a pharmaceutical CEO. Regardless of the case, Cytokinetics’ share value fell as a lot as 10% after Narasimhan’s interview.
Get together exit strains you solely hear at JPM
“Nice seeing you, however I gotta go see Takeda a few molecule.”
Extra reads
- JPM 2024: FogPharma’s new CEO, Mathai Mammen, unveils ‘contrarian’ plan in oncology, STAT
- Doudna institute hatches plan to ‘remedy a whole lot of illnesses’ left behind by CRISPR revolution, STAT
- GSK to amass Aiolos Bio in $1.4 billion deal, selecting up an experimental bronchial asthma drug, STAT
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