Alnylam's highly anticipated readout

Alnylam this morning reported highly anticipated topline results of its HELIOS-B trial testing vutrisiran in an increasingly common heart condition called ATTR-CM. Here are the findings:

Vutrisiran cut the risk of death and recurrent cardiovascular complications by 28% vs. placebo in the overall trial population, which included people who were taking the standard of care treatment (a drug from Pfizer called Vyndaqel/Vyndamax). In a subpopulation of patients who were not on that background treatment, or the monotherapy population, vutrisiran cut the risk by 33% compared with placebo.

Additionally, vutrisiran cut the risk of all-cause death, a secondary endpoint, by 36% in the overall population and 35% in the monotherapy group. This outcome included up to six months of data from an open-label extension that followed the blinded period of the trial.

These positive results likely pave the way for the approval of the treatment, but there are still outstanding questions on how much added benefit vutrisiran provides on top of Pfizer's drug and also how the efficacy compares with other rival treatments, namely one from BridgeBio.

diabetes

Vertex cell therapy cuts insulin need in type 1 diabetes

Vertex Pharmaceuticals has been testing its stem cell-derived treatment for type 1 diabetes in a Phase 1/2 study, and found that 11 of 12 patients who received it no longer needed to inject insulin over the course of three months. And after a year, three of the people had no need to inject insulin at all.

"These cells clearly work," an endocrinologist not involved with the study told STAT's Elizabeth Cooney.

Even though this treatment could ultimately offer a new and more sustainable option for type 1 diabetes, it's physically taxing for trial participants. Although patients were able to cut down or eliminate insulin injections, they had to take immune-suppressing drugs so their bodies wouldn't attack the Vertex infusion of off-the-shelf islet cells.

legislation

House panel to vote on weight loss drugs, cancer screening

A House panel may vote on a pared-down version of two major health care bills meant to expand how Medicare covers weight loss drugs and cancer screening tests, sources tell STAT's John Wilkerson and Rachel Cohrs Zhang.

Although there's bipartisan support for full coverage in these cases, the respective price tags have been a sticking point. Right now, the Medicare program is legally prohibited from covering medicines for weight loss. One of the bills in question is the Treat and Reduce Obesity Act, which would change that. The other is the Nancy Gardner Sewell Medicare Multi-Cancer Early Detection Screening Coverage Act, which would allow Medicare to cover screening healthy people for cancer with multi-cancer blood tests.

drug resistance

Using phage therapy to combat microbial resistance

Phage therapy is an underused treatment for antimicrobial resistance. Advocate Mark H. Smith, whose daughter died from a bacterial lung infection that resisted treatment, argues that bacteriophage viruses are well-poised to address this worldwide problem. These viruses have evolved naturally to attack bacteria; though they've been studied for more than 100 years, interest in research dwindled after antibiotics took off.

Smith points out the many advantages phages have over antibiotics — such as low risk of side effects, and an inability to infect human cells. And they're highly specific, so they only attack targeted bacteria.

"Phage therapy represents a powerful tool in the arsenal, one that has been neglected for far too long," he writes. "By loosening restrictions and fostering a more flexible regulatory environment, the FDA can pave the way for a new era of innovative treatments."

More around STAT