March 8, 2024

The need-to-know this morning:

Amylyx Pharmaceuticals said its treatment for ALS, called Relyvrio, failed to provide any benefit for patients in a large clinical trial — a stunning outcome that now has the company considering a voluntary withdrawal of the approved medicine from the market.

"This is really hard for us, and it's really hard for our team who care so much, but it's so much harder for people with ALS and their families, and we have to keep that perspective," said a somber Justin Klee, Amylyx's co-founder and co-CEO, in an interview with STAT on Thursday evening.
In a surprise move, the Food and Drug Administration has called for a meeting of outside advisers to discuss whether an Alzheimer's drug from Eli Lilly should be approved, even as many outsiders expected the medicine to receive regulatory clearance this month.

The drug, donanemab, succeeded in its Phase 3 trial, resulting in a 35% slowing of Alzheimer's disease progression versus placebo. But Lilly on Friday said that the FDA expects to call a meeting of an advisory committee to review the trial, saying that the agency had told the company "it wants to further understand topics related to evaluating the safety and efficacy of donanemab, including the safety results in donanemab-treated patients and the efficacy implications of the unique trial design" of the study.

drug pricing

Pharma can't have liked Biden's State of the Union address

In his State of the Union address, President Biden proposed expanding Medicare price negotiation — one of his most popular policies. And, as STAT's Matt Herper writes, it's clear that the pharmaceutical industry remains one of the biggest political targets in the country — even though they stepped up when Covid-19 hit, and have developed a new wave of remarkably effective weight loss drugs.

Historically, the industry found support among pro-business Republicans, or pro-science Democrats. But now, they are politically homeless. Some of Pharma's reputation is earned. But Matt writes that "increasingly people who work in the drug industry are stuck between the liberal ideology of their research hotspots in Cambridge, Mass., and San Francisco and the pro-business conservatives who would protect their intellectual property and take a stand against price controls."

podcast

Why are addiction treatments so hard to get?

Can small companies compete in obesity? And since when is "Series E" a thing in biotech? We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast.

For the entirety of the opioid overdose epidemic, the U.S. has had readily available tools that are proven to save lives. STAT's Lev Facher joins us to explain his reporting on why virtually every sector of American society is standing in the way of their use. We also discuss the latest news in the life sciences, including the haves and have-nots of venture capital, and the race to develop effective oral treatments for obesity.

Listen here.

glp-1 drugs

Eli Lilly's anti-vanity ad campaign for Oscars

Celebrities have leaned into the GLP-1 revolution — using the new wave of diabetes and obesity drugs to squeeze into sleeker silhouettes. Eli Lilly will be unveiling a new campaign during this weekend's Oscar awards event, with a clear message: These drugs aren't meant to be used for cosmetic reasons.

"Some people have been using medicine never meant for them," one ad says, according to CNN. "For smaller dress or tux, for a big night, for vanity... People whose health is affected by obesity are the reason we work on these medications. It matters who gets them."

The ad, which features a red carpet and paparazzi flashes, doesn't specifically mention Lilly's GLP-1 drugs, Zepbound and Mounjaro. But it's sending a message that Lilly's been trying to send this year — that they're meant to treat disease. The drugs have been in shortage ever since celebrities began popularizing them, leaving gaps in care among people who need such drugs more.

"We have a point of view about how these drugs are being used," CEO David Ricks told CNN. "These medicines were invented for people with a serious health condition; they were not invented just to have someone who's famous look a little bit better."

drug pricing

Federal judge unsympathetic to pharma argument

Novartis, Novo Nordisk, and other pharma giants sent their lawyers to a New Jersey courtroom on Thursday to unravel Medicare's new drug price negotiation program — just before President Biden lionized it in his State of the Union address. The federal judge wasn't particularly receptive to the drug makers' argument that Medicare drug price negotiations would harm biopharmaceutical innovation.

"A lot of people would say pharmaceutical companies could give up an arm," said Judge Zahid Quraishi, a Biden appointee. "They have a lot of appendages."

He added: "They're businesses with the goal of profit … These companies are not Mother Teresa developing drugs for free for the American public," he said.

psychiatry

Changing up psychiatric drug research

Drug development in psychiatry lags other sectors: the existing arsenal of medication is still very limited, and it can take years to find the right balance to help patients. Part of the problem, opines Alto Neuroscience CEO Amit Etkin, is that there isn't a systematized approach to learn from failures and successes in how medications affect individuals.

Perhaps most psychiatric drugs have been destined to failure because they were studied in heterogenous populations, he writes.

"Removing guesswork from psychiatry requires data-driven, patient-centric approaches, and the road to eliminating trial-and-error begins with science," he writes. "It's time for psychiatric drug developers to rethink everything to reshape the future of mental healthcare."