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Morning! Today we look at some promising but early gene therapy data from Lexeo Therapeutics, examine Medicare’s new price negotiation rules and their impact on innovation, and more.
Lexeo’s gene therapy for rare heart diseases looks promising
Lexeo Therapeutics is developing a gene therapy intended to prevent fatal heart complications in patients with Friedreich’s ataxia, a rare genetic disorder of the heart. The results are early and inconclusive, but promising – and Lexeo is optimistic that it could win FDA approval after another study.
“In my view, it fits right into the kind of framework that Peter Marks has driven,” Lexeo CEO Nolan Townsend told STAT’s Jason Mast, speaking of the director of the agency’s biology division.
In a study of 11 patients, the therapy showed some reduction in markers of heart disease, such as left ventricular size and troponin levels. While the data is encouraging, there is some concern that the patient pool is too small and the company has not yet shown functional improvement in patients.
Read more.
Do Medicare’s new rules stifle drug innovation?
Medicare’s new guidelines for drug price negotiations, part of the Inflation Reduction Act, could inadvertently stifle medical innovation, says medical consultant and American Medical Association delegate Peter Rheinstein. CMS interprets the law in a way that puts all drugs with the same active ingredient into one drug category, even if they are approved separately and used for different conditions. This could discourage pharmaceutical companies from investing in research to find new applications or improve existing drugs, he writes.
For example, the development of a once-daily pill version of tofacitinib for arthritis, which improves patient compliance, might not have happened under these rules, Rheinstein argues. While the goal is to reduce drug costs, CMS’ approach could ultimately limit the development of new and potentially life-saving treatments. Instead, Rheinstein says CMS should consider each drug individually if it wins FDA approval on its own — to ensure new drugs are given the necessary time before facing price negotiations.
Read more.
What are the chances we’ll get a Paxlovid heir?
STAT’s Matthew Herper weighs in from the Twitterverse: If you read Derek Lowe – and come on, you should also read the bearded chemist who has been blogging about pharmaceuticals for twenty years – you have already thought about ibuzatrelvir, Pfizer’s potential heir to Paxlovid, which would be given as a single pill and possibly without that metallic taste.
I read this with interest, but I had one more question I asked virologist Michael Mina when he posted Derek’s article about How do we test it?
There are still many people getting Covid and dying from it. But I have looked at clinical trials of Paxlovid and other antivirals. And these drugs continue to fail in trials. Even Paxlovid worked best when given to high-risk people who had not been vaccinated. Shionogi’s Paxlovid sequel recently failed in a major study.
It is very difficult to prove that an antiviral drug works, a problem that has long dogged Tamiflu. The benefit only shows up if people are at very high risk, or if the study is very large. Otherwise, the drug seems to reduce symptoms very slightly.
On this, David Boulware of the University of Minnesota had one perspective: Shionogi’s drug failed because regulators wanted the study to too few at-risk patients. But my suspicion is that getting better Covid drugs won’t be a chemical problem so much as a clinical trial problem. This is part of the reason why we don’t have more monoclonal antibodies against new strains. Paxlovid sales have been higher than many analysts expected so far this year. Maybe that will push pharmaceutical companies to solve this problem.
AI research shows the variability of the brain based on sex and gender
Sex and gender uniquely determine brain connectivity, according to a new study using AI to analyze MRI scans of thousands of children. Researchers found that sex, largely influenced by biology, involved brain areas related to vision and movement, while gender, defined by identity, had more diffuse brain network patterns. This emphasizes the need to consider both sex and gender separately in brain research.
Understanding how sex and gender affect brain connectivity differently could ultimately help improve the way brain disorders are diagnosed and treated – allowing interventions to be tailored to individual needs.
Read more.
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