It's all coming up Eisai

About two and a half years ago, Biogen spent an entire day listening to a panel of FDA advisers debate, doubt, and dismantle its work on a novel treatment for Alzheimer's disease, concluding with a stern rebuke of what would become Aduhelm and setting in motion the medicine's disastrous rollout. Eisai, Biogen's partner on a second Alzheimer's drug, will almost certainly have a different experience when it goes before the same panel Friday.

In documents posted ahead of the meeting, FDA reviewers endorsed the modest but statistically significant cognitive effects of Leqembi, an Eisai-developed successor to Aduhelm, and said the treatment's safety risks are likely outweighed by its benefits. The 35-page document didn't surface any new concerns about Eisai's treatment. To Baird analyst Brian Skorney, the FDA's favorable tone suggests "a high level of confidence" the panelists will vote in favor of Leqembi.

We'll find out Friday, when a panel of six advisers will be asked whether Eisai's Phase 3 data merit expanding Leqembi's accelerated approval into a full one. While the vote looks likely to go Eisai's way, the panelists' views on the drug's specific risks and benefits to subgroups of patients could influence the precise wording of Leqembi's eventual label.

Washington

Now begins the pricing controversy

With Eisai's Alzheimer's disease looking likely to win over the FDA, the drama shifts to Medicare, which has to date restricted access to medicines like Leqembi.

That will change if the FDA gives the drug full approval, and, as STAT's Rachel Cohrs reports, several lawmakers are ratcheting up their criticism of how the Biden administration is planning to handle the rollout.

Sen. Bernie Sanders, who leads the Senate's health committee, wrote to health secretary Xavier Becerra yesterday asking him to "use the full extent" of his authority to ensure Medicare doesn't pay the $26,500 list price for Leqembi. And two Democratic members of the House are asking Medicare to further clarify just how patients can access Leqembi if it wins full approval this summer.

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Pharma

GSK missed Covid-19, but it's hardly giving up on vaccines

GSK has been in the vaccines business since Chester A. Arthur was president, and yet when Covid-19 struck, the storied company sputtered while smaller, younger rivals made the most of a once-in-100-years opportunity.

The British drugmaker does not intend to make that a habit. As STAT's Jason Mast reports, the job of rebuilding GSK's vaccine business falls to Phil Dormitzer, a key figure in Pfizer's Covid success who jumped to GSK in late 2021. He's now global head of R&D for a division that developed more than 20 licensed vaccines in its history but that has since seemed to fall behind more agile competitors with newer technologies.

"In the biggest picture, I'd love to return GSK to the No. 1 vaccine company by sales," Dormitzer said. In the short term, the company is investing in vaccines for bacterial infections, herpes virus, and influenza, all while scaling up its technological abilities in mRNA.

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Policy

Califf: 'Prices of drugs are too high in the U.S.'

FDA Commissioner Robert Califf got a warm welcome at the BIO conference this week, but the industry's talking points on federal drug pricing legislation don't seem to be resonating.

"We don't agree," Califf said yesterday, referring to a conversation he had backstage with Ted Love, the new chair of the Biotechnology Innovation Organization. "I think the prices of drugs are too high in the U.S."

The FDA has no authority over how much medicines cost, but Califf wasn't shy about defending the Inflation Reduction Act, which allows Medicare to negotiate certain drug prices — and which Merck, in a lawsuit filed this week, described as "tantamount to extortion."

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Gene therapy

Astellas takes second stab at gene therapy for muscle disorder

Astellas Pharma will license and develop a new gene therapy for a devastating muscle disorder, after four boys died in a clinical trial testing an earlier treatment.

As STAT's Jason Mast reports, the hope is that the new therapy will allow researchers to treat the disease, known as X-linked myotubular myopathy, or XLMTM, with much lower doses of the viruses used to shuttle genes into patients' cells. In theory that should minimize the risk of severe side effects.

Astellas licensed the new gene therapy, which is not yet ready for human testing, from Kate Therapeutics, a San Diego-based startup founded.