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Morning! Right now, we have a look at some promising, however early, gene remedy information from Lexeo Therapeutics, look at Medicare’s new worth negotiation guidelines and their influence on innovation, and extra.
Lexeo’s gene remedy for uncommon coronary heart illness seems to be promising
Lexeo Therapeutics is growing a gene remedy meant to stop lethal coronary heart problems in sufferers with Friedreich’s ataxia, a uncommon genetic dysfunction of the guts. The outcomes are early and inconclusive however promising — and Lexeo is optimistic that it may win FDA approval, after one other examine.
“In my view, it sits squarely in the kind of framework that Peter Marks has been kind of driving,” Lexeo CEO Nolan Townsend informed STAT’s Jason Mast, talking of the director of the company’s biologics division.
In an 11-patient examine, the remedy confirmed some discount in coronary heart illness markers like left ventricle dimension and troponin ranges. Though the information are encouraging, there’s some concern that the affected person pool is just too small and the corporate has but to point out purposeful enchancment in sufferers.
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Do Medicare’s new guidelines stifle drug innovation?
Medicare’s new pointers for drug worth negotiations, a part of the Inflation Discount Act, would possibly unintentionally stifle medical innovation, opines medical advisor and American Medical Affiliation delegate Peter Rheinstein. CMS is decoding the regulation in a manner that lumps all drugs with the identical energetic ingredient right into a single drug class, even when they’re accepted individually and used for various circumstances. This might discourage pharmaceutical firms from investing in analysis to search out new makes use of or enhance present medicine, he writes.
For instance, the event of a once-daily capsule model of tofacitinib for arthritis, which reinforces affected person adherence, won’t have occurred beneath these guidelines, Rheinstein argues. Whereas the objective is to decrease drug prices, CMS’s strategy may finally restrict the event of latest and doubtlessly life-saving therapies. As an alternative, Rheinstein says that CMS ought to contemplate every drug individually if it acquired FDA approval by itself — to guarantee that new medicine have the required time earlier than dealing with worth negotiations.
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What are the chances we get an inheritor to Paxlovid?
STAT’s Matthew Herper weighs in from the Twitterverse: In case your learn Derek Lowe — and are available on, you should learn the bearded chemist who has been running a blog about pharma for twenty years — you’ve already thought about ibuzatrelvir, Pfizer’s potential inheritor to Paxlovid, which might be given as a single capsule and doubtlessly with out that metallic style.
I learn this eagerly, however I had another question that I asked virologist Michael Mina when he posted Derek’s article on X. How will we check it?
Loads of folks nonetheless get Covid and die from it. However I’ve been watching medical trials of Paxlovid and different antivirals. And these medicine maintain failing in research. Even Paxlovid labored finest when it was given to high-risk individuals who haven’t been vaccinated. Shionogi’s Paxlovid follow-up recently failed in a large study.
It’s very exhausting to show an antiviral drug works, an issue that has lengthy dogged Tamiflu. The profit solely exhibits up if persons are at very excessive threat, or if the examine may be very huge. In any other case, it simply seems the drug is decreasing signs a really tiny bit.
On this, David Boulware of the College of Minnesota had one perspective: Shionogi’s drug failed as a result of regulators wished its examine to include too few high-risk patients. However my suspicion is that getting higher Covid medicine will probably be much less an issue of chemistry than of medical trials. That is a part of why we don’t have extra monoclonal antibodies in opposition to new strains. Paxlovid gross sales have are available in larger than many analysts have been anticipating to this point this yr. Perhaps that can encourage drug firms to unravel this downside.
AI examine exhibits mind variability based mostly on intercourse, gender
Intercourse and gender uniquely form mind connectivity, in response to a brand new examine utilizing AI to investigate MRI scans of hundreds of kids. Researchers discovered that intercourse, largely influenced by biology, concerned mind areas linked to imaginative and prescient and motion, whereas gender, outlined by id, had extra diffuse mind community patterns. This highlights the necessity to contemplate each intercourse and gender individually in mind analysis.
Understanding how intercourse and gender affect mind connectivity in numerous methods may finally assist enhance how mind problems are recognized and handled — permitting interventions to be tailor-made to particular person wants.
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Extra reads
- AbbVie’s Humira loses some floor in high-stakes battle in opposition to biosimilars: report, FiercePharma
- Lilly says lupus drug dropped from pipeline failed to point out efficacy, Endpoints
