Alnylam CEO talks about advancing vutrisiran

Alnylam Pharmaceuticals is expanding the footprint of its RNA-based gene-silencing heart drug, vutrisiran, CEO Yvonne Greenstreet said yesterday during the 2024 STAT Summit in Boston. The drug's already been approved in the disease known as ATTR-polyneuropathy, but late-stage trials showed that it can dramatically reduce the risk of death and serious cardiovascular complications in patients with ATTR cardiomyopathy, or ATTR-CM — which affects about 300,000 people around the world.

BridgeBio has its own ATTR-CM drug, called acoramidis, that will likely be approved next month by the FDA.

"The job at hand here is to make sure that patients can get diagnosed early and treated early," Greenstreet told STAT's Adam Feuerstein. "Competition is a good thing because it actually brings more education to physicians."

Alnylam's stock hit an all-time high this week, and its market value now exceeds $38 billion — placing it in the ranks of the biggest biotech companies, like Vertex, Regeneron, Gilead, and Amgen.

rsv

Potential competition for RSV prophylactic Beyfortus

From STAT's Helen Branswell: New data released by Merck suggests AstraZeneca and Sanofi's Beyfortus, the monoclonal antibody injection that protects infants against RSV, may have competition, potentially as early as next fall.

In a Phase 2b/3 trial, the Merck injection was 60.4% effective at preventing RSV-related medically attended lower respiratory tract infections, 84% effective in protecting against any RSV hospitalizations, and nearly 91% effective in protecting against hospitalization for RSV-related lower respiratory tract infections in the five months after injection. A study published in March in the CDC's online journal Morbidity and Mortality Weekly Report estimated that Beyfortus was 90% effective at preventing RSV-related hospitalization.

Should clesrovimab win FDA approval, it will have an advantage over Beyfortus, which comes in two different dose sizes — one for babies under 11 pounds and a second for heavier babies. Birthing hospitals and pediatricians who administer the injection have to stock supplies of both. Clesrovimab, on the other hand, comes in a single size, regardless of the baby's birth. The data were presented at the ID Week conference in Los Angeles.

podcast

Biotech's next gold rush

Why are biotech investors so interested in autoimmune disease? Who is behind the telehealth GLP-1 boom? And what were our favorite moments from the STAT Summit this week? We talk about all that and more on this week's episode of "The Readout LOUD," STAT's biotech podcast.

We chat about investors' interest in using CAR-T to treat autoimmune conditions, the latest developments with the supply of Eli Lilly's blockbuster weight loss drug, and the invisible medical groups behind the dozens of telehealth platforms providing GLP-1 drugs.

Listen here.

opinion

Genentech wants the big picture approach to drug costs

Some of the political proposals meant to lower drug costs for patients could ultimately harm innovation, jobs, and the U.S. lead in the biopharmaceutical sector, opines Fritz Bittenbender, a top executive at Genentech. He advocates instead a longterm bipartisan approach to improve affordability — such as the 340B Drug Pricing Program, a 1992 safety-net program designed to help support vulnerable patient populations.

"Our focus at Genentech remains on pushing for broader change rather than feeding into the politcal rhetoric that only gets us further from a solution," Bittenbender writes.