November 20, 2023

The need-to-know this morning:

Bayer said it prematurely stopped a Phase 3 study in stroke prevention after independent monitors concluded its experimental blood-thinning medicine, called asundexian, showed inferior efficacy compared to a standard treatment. The asundexian study setback is a significant blow to Bayer's effort to revamp its drug-development pipeline, analysts said.
Carmot Therapeutics filed paperwork Friday for an initial public offering of undisclosed size. The Berkeley, Calif.-based company is developing drugs that target GLP-1 and GIP to treat diabetes and obesity, similar to Novo Nordisk's Ozempic/Wegovy and Eli Lilly's Mounjaro/Zepbound.
Bristol Myers Squibb and 2Seventy Bio said the FDA will not complete an on-time review of its application seeking to expand the use of Abecma, their CAR-T therapy for multiple myeloma. Instead, the FDA will convene an advisory panel meeting — date to be determined — to examine survival data from an Abecma clinical trial.
The U.K. and the pharmaceutical industry have reached a deal on a five-year plan outlining how the health system pays for drugs, as the country tries to keep a lid on its medicines spending while simultaneously building up its life sciences industry. STAT's Andrew Joseph has more here.

gene editing

A post-approval chat with the CRISPR pioneers

In June 2012, Jennifer Doudna and Emmanuelle Charpentier first unveiled CRISPR-Cas9. Now, the first CRISPR-based treatment, Casgevy, has secured approval in the U.K. for sickle cell anemia and beta thalassemia — a landmark achievement that has come a lot faster than expected.

STAT's Matt Herper chatted with Doudna, along with Feng Zhang of the Broad Institute and George Church, who were also pioneers of this powerful gene editing technology.

The researchers were astounded by how fast the science has progressed: Charpentier said she was "truly amazed" by the progress, Zhang called it amazing, and Doudna called it "awe-inspiring" — but added repeatedly that "we still have a lot of work to do." George Church called himself a "proud parent."

"Even now, more than 10 years later, I still feel like I'm living in the middle of a whirlwind," Doudna said. "There's just always things happening with CRISPR. It's hard to predict what's coming down the pike with it. I think that I've sort of gotten used to that feeling."

regulatory

Merck cough drug doesn't pass panel scrutiny

An FDA advisory panel on Friday rejected a cough medicine from Merck, saying the data supporting it didn't demonstrate enough clinical benefit for patients. The committee voted 12 to 1 against gefapixant, which only showed a small reduction in chronic coughing compared to placebo. And patients receiving the treatment had side effects like a loss of taste. Late-stage data showed that 22% of patients taking a high dose of the drug discontinued it because of such adverse events.

"If they were feeling so much benefit would they have dropped out … if that's how many [patients] are dropping out in trial, I would expect to see a bigger drop out rate in the real world," one of the advisers said, according to Reuters.

regulatory

FDA needs to up the ante with cell and gene therapies

Regulators need to get up to speed when it comes to overseeing cell and gene therapy approvals, opines former U.S. Senator Richard Burr in a new First Opinion.

The FDA is too risk-averse, and culturally, it's still slow to adapt to new science, he says. There are some 2,500 cell and gene therapy investigational new drug applications on file with the FDA, and so far this year, regulators have just approved five.

There's been movement in the FDA, to be sure: CBER chief Peter Marks recently said that accelerated approvals might be the best approach for cell and gene therapies. The agency now has a new director for its Office of Therapeutic Products, and is doubling down on hiring and training. And it's announced a pilot program for rare disease gene therapies that's meant to emulate Operation Warp Speed.

"We cannot withhold or contain the transformative innovation in genetic medicine and patients cannot wait for the regulatory scheme to catch up with the science," Burr writes.

GLP-1 drugs

Will Zepbound do more than Mounjaro?

Mounjaro, Eli Lilly's blockbuster diabetes drug, has a new moniker: For weight loss, it will now also be sold as "Zepbound," a name that is a reminder of how complex the naming strategy at biopharma companies can be, STAT's Annalisa Merelli writes.

Take Prozac, for instance. It's regarded the gold standard when it comes to drug names — quick, memorable, iconic. But when the same drug was sold for premenstrual syndrome under the name Sarafem, it was discontinued. Similarly, the glaucoma drug Lumigan was renamed when sold for eyelash growth to Latisse.

This stuff can actually be quite important in terms of securing sales and even approvals, as "it definitely isn't easy to develop a drug name that meets all the federal requirements, is available as a trademark, and communicates something about the brand," one branding expert told STAT. Whether the name Zepbound will prove more appealing, commercially, than other GLP-1 treatments remains to be seen.