April 4, 2024

The need-to-know this morning

Amylyx Pharmaceuticals is removing its ALS treatment, called Relyvrio, from the U.S. and Canadian markets, where the medicine is currently approved. The company's decision comes weeks after the drug failed to show any benefits for patients in a large clinical trial.

glp-1

GLP-1 drug shows potential in Parkinson's

The potential of GLP-1 drugs stretches far past diabetes and obesity; now, a new study suggests one such drug could even be effective in treating Parkinson's disease. The motor symptoms of patients in a Phase 2 trial testing lixisenatide, an older GLP-1 drug, didn't worsen over the course of a year, in contrast to the patients on placebo, a NEJM study shows.

Although the difference in tremors and rigidity didn't differ too dramatically between the trial arms, researchers are encouraged that patients taking the drug didn't get worse. There aren't any approved drugs to halt disease progression in Parkinson's. And though the difference was subtle over the course of a year, "the question now would be what would happen if the patients would take this drug for two years or three years or five years?" a principal investigator of the study told STAT. "That is something future trials definitely need to address."

biosecurity

Why the U.S. biotech sector must stay on top

Biotech is increasingly becoming a national security concern. The best protection, opines BIO chief John F. Crowley, is to ensure that the U.S. maintains its position as the global leader in medical and biotechnology innovation.

"The country's strategic rivals are aggressively investing in biotechnology in an effort to surpass and replace U.S. preeminence," he writes. "The nation's future may well depend on how America maintains and extends its current advantage."

China is asserting itself as a leader in biotech, and the sector is "a centerpiece of its strategic plans." And if China rises to the top by 2035, as it plans, it will control how diseases are treated, and how genetic information is used.

"This supremacy would expand its global economic influence while reshaping the global order to its interests," Crowley writes.

de-extinction

How resurrecting a mammoth might impact medicine

De-extinction of mammoths and other species of yore command a great deal of public interest: Jurassic Park, but for real? But the potential runs much deeper than theoretical amusement parks, says evolutionary biologist Beth Shapiro, chief scientific officer of Colossal Biosciences. Species are disappearing at an untenable clip, and the company is harnessing genetic editing to try and reverse that trend.

And it's not just about resurrecting the thylacine or the dodo: Shapiro says the work could impact human health.

"We have hundreds of thousands of human genomes, and we still can't pinpoint with precision what gene means what phenotype," she said. Colossal is developing a library of genomes "from across the tree of life," she said, which could help scientists to better understand the link between genotype and disease.

rare disease

Moderna's mRNA treatment for rare disease

Well before its Covid-19 vaccine, Moderna's goal with mRNA was to turn it into therapies. That may finally begin coming to fruition: It just reported interim results on a treatment for propionic acidemia, a rare metabolic condition in which the body fails to produce enzymes necessary to break down fats and proteins. Most of the Phase 1/2 trial participants are children.

Some saw a reduction in life-threatening emergencies linked to the disease — and although side effects included fever and vomiting, the majority wanted to stay on the drug even after the trial ended. Moderna wants to launch a pivotal trial for the therapy, called mRNA-3927, later this year, as well as another pivotal trial with a treatment called mRNA-3705 for a similar disease called methylmalonic acidemia.

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