May 4, 2023

The need-to-know this morning

Novo Nordisk, the maker of the obesity drug Wegovy, said this morning it would temporarily limit some "starter" doses for new patients in the United States to ensure a steady supply for people already on the medication.
Valneva said it now expected to apply for regulatory approval for the Lyme disease vaccine it is developing with Pfizer in 2026, a delay of a year. The announcement came after the companies had to scrap some data because of quality issues with a third-party trial operator.
The earnings bonanza continues: Companies also reporting this morning included Moderna, Regeneron, BeiGene, and Intellia.

Research

What happens when gene therapy wears off?

Gene therapy is often described as a "one-time treatment," because, in the ideal scenario, a single dose can effectively cure a disease — and because the immune system's reaction means a second dose is unlikely to have an effect. But as the effects of some gene therapies appear to wane over time, scientists are trying to figure out how to make redosing a reality.

As STAT's Jason Mast reports, gene therapies can currently be given only once because patients' immune cells learn to recognize the viruses used to deliver them and attack if they show up again. For patients with the muscle-destroying Duchenne muscular dystrophy, the impending potential approval of a first gene therapy could be a starting pistol for the field to devise a solution before the strength of the boys who receive it begins to decline.

Scientists are already at work. Abstracts for the American Society of Cell and Gene Therapy include more than a half-dozen efforts to engineer viruses, repurpose cancer and organ transplant drugs, and devise novel enzymes and treatment regimens that might allow patients to receive a gene therapy a second time.

Read more.

Alzheimer's

Your move, Medicare

In the next few months, Eisai and Biogen's Alzheimer's disease treatment Leqembi is likely to win full FDA approval. Months after that, Eli Lilly's donanemab will probably do the same. That means Medicare, which responded to the Aduhelm debacle by restricting coverage of all amyloid-targeting treatments, is about to come under serious pressure.

As STAT's Rachel Cohrs reports, Medicare has so far held firm on its plans to require patient registries for the entire class of anti-amyloid Alzheimer's drugs, even after they are fully approved. That would loosen the restrictions currently applied to Aduhelm, but it would mean the drugs wouldn't be as widely available as other approved medicines.

According to Sean Tunis, a consultant and former CMS official who helped develop the process Medicare is using to restrict coverage, Medicare has never before made such a requirement for fully approved medicines. The policy "is in stark contrast to scientific evidence, is unprecedented, and must be reversed immediately," said Joanne Pike, president and CEO of the Alzheimer's Association.

Read more.

R&D

How Lilly's many Alzheimer's failures bred a belated success

Daniel Skovronsky's scientific career kicked off in 1998, with an academic paper on beta amyloid, the vexing molecule thought to play a role in Alzheimer's disease. Now, as head of research at Eli Lilly, Skovronsky has presided over the development of donanemab, an amyloid-targeting medicine that looks bound for FDA approval.

"I've been pursuing the same enemy for 25 years," Skovronsky said at the STAT Breakthrough Summit in San Francisco yesterday. Donanemab's clinical success, disclosed yesterday, was born from Lilly's years of failure in Alzheimer's. After the costly 2016 failure of an amyloid-directed treatment called solanezumab, Lilly embarked on "Alzheimer's 2.0," Skovronsky said, rethinking its approach to the disease and setting in motion the development of donanemab.

"All of this work," he said, "building up to this moment where we could have a Phase 3 result for a drug that has such a potent effect on removing amyloid plaques — probably any words I use to describe my emotions at seeing this data will understate it."

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Regulatory

GSK's RSV vaccine marks a medical milestone

The FDA approval of GSK's vaccine for respiratory syncytial virus, or RSV, completes an elusive scientific quest that has been decades in the making.

As STAT's Helen Branswell reports, yesterday's approval of Arexvy dates back to the 1960s, when children who received an experimental vaccine in a trial experienced more severe disease than the children in the placebo arm. Two children, both under the age of 2, died. Scientists spent years studying what went wrong, leading to groundbreaking work by researchers at NIH scientists who mapped the exact structure of key protein on the virus' surface.

GSK beat a crowded field of competitors. Pfizer is expected to win approval this month with a similar vaccine, and Moderna is in the late stages of developing one of its own. Also up for approval soon is an antibody treatment, to be marketed in the U.S. by Sanofi, that would be given as an injection after birth or near the start of RSV season to protect in the first year of life.

Read more.

Chart of the day

What's good for pharma is good for biotech

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Eli Lilly's clinical success in Alzheimer's disease sent the closely watched XBI biotech index up as much as 5% yesterday, extending the sector's recent recovery amid a spate of high-dollar deals and positive study results.

The XBI reached its highest point since February, rising more than 10% in the last month after bottoming out in March. The index has gained about 2% on the year, lagging the S&P 500's 7% growth but substantially outperforming 2022's nearly 30% decline.

Shares of Prothena, which is developing amyloid-targeting treatments for Alzheimer's, rose by about 27% after Lilly's announcement. Shares of Biogen, partnered with Eisai on the recently approved amyloid drug Leqembi, was flat.

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