November 19, 2024

The need-to-know this morning

The FDA rejected an application from Astellas seeking to change the prescribing label for its eye drug Izervay to allow less frequent injections and include data showing the effect of the drug over two years. The setback is a win for Apellis Pharmaceuticals, which sells a competing eye drug called Syfovre.
Cytokinetics and Bayer signed a partnership agreement that gives Bayer the right to develop and sell Cytokinetics' heart drug aficamten in Japan.

cardiovascular

Lipoprotein(a) studies make waves at AHA

Two drugs targeting lipoprotein(a) are showing promise in cardiovascular disease, Phase 2 data presented at the American Heart Association show. High levels of lipoprotein(a) indicate a genetic risk factor for cardiovascular disease. This affects 1 out of 5 people worldwide, STAT’s Liz Cooney writes.

An oral small molecule drug from Eli Lilly called muvalaplin and an RNA-silencing injection from Silence Therapeutics called zerlasiran each dramatically reduced levels of lipoprotein(a) in patients with high levels of the marker as well as cardiovascular disease.

“These two new reports add to the growing evidence in at least five different drug programs directed for lowering Lp(a) that the agents are potent, capable of 80% reduction or more, with durable effects over extended treatment,” said cardiologist and geneticist Eric Topol, who is director of the Scripps Research Translational Institute. He wasn’t involved in the study.

investment

Former NCI director starts biotech to launch biotechs

Ned Sharpless, former director of the National Cancer Institute, is co-founding a new company called Jupiter with $70 million in hand. Jupiter’s approach will be to launch several early stage biotechs in tandem, and either sell them or kill them, STAT’s Allison DeAngelis and Matthew Herper write. Sharpless is launching Jupiter with another Ned — serial biotech entrepreneur Edward David, who happens to go by the same nickname.

The plan is ruthless efficiency: They’ll fund 10 sub-companies, investing about $2.5 million apiece. They expect between one and three of these ideas to succeed, and then would throw another $20 million or so at the ideas that stick.

“The point of it is we get heavily operationally involved,” David said. “I mean, we don’t just invest — we, like, you know, think through the projects and figure out the science and figure out what clinical research organization is going to do this.”

Cancer

Merck proves out new version of Keytruda in study

A new formulation of the best-selling cancer drug, Keytruda, appears to deliver similar blood levels of the medicine when it is given as a shot as when it is given intravenously, Merck reported Tuesday.

For Merck, the new under-the-skin, or subcutaneous, formulation of Keytruda could represent a major way of holding on to a larger share of Keytruda’s $25 billion in annual sales than would otherwise occur when the medicine’s U.S. patent expires in 2028.

The subcutaneous injection could be given in a doctor’s office every six weeks, instead of requiring a 30-minute IV drip at the same interval. Analysts expect that Merck might be able to continue to charge a higher price for the in-office version while competition from biosimilar medicines pushes down the price for Keytruda’s existing formulation.

global health

Global reach is lacking for many pharma companies

Although some pharma companies are trying to improve drug access in low- and middle-income countries, the progress overall is pretty lackluster. The number of licensing deals, tech transfers, and clinical trials in these regions are pretty limited — and most programs aren’t particularly transparent, STAT’s Ed Silverman writes.

“Since we began measuring these companies, we expected the pace of progress would pick up, but it’s falling short — and not a little bit short, but a lot short,” said Jayasree Iyer, head of the Access to Medicines Foundation that presented these findings. “The big concern is how do we keep momentum going if, in fact, it’s dropping down?”

cardiovascular

What the cardiologists were talking about at AHA

This year’s American Heart Association conference spent a good chunk — predictably — on obesity drugs like tirzepatide and semaglutide, featuring groundbreaking trials on their impact in heart failure and cardiovascular risk reduction. Other key studies included intensive blood pressure management in type 2 diabetes, as well as novel approaches to treat transthyretin amyloidosis using CRISPR-based and RNA therapies.

Heart rhythm disorder treatments, AI-powered echocardiogram diagnostics and reconditioned pacemakers for underserved populations were also discussed during the conference. There was discussion, too, of how the overall cardiovascular disease burden will worsen from now until 2050.

“We have a problem,” former AHA president Valentin Fuster said. “Pay attention to health and prevention — that is the challenge of the next century.”

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