How can you be sure a gene therapy is working?

What does $11 billion get you in biotech? And who decides which medicines get approved? We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast.

Our colleague Helen Branswell joins us to explain the latest news on Covid-19 boosters and the implications of a Marburg outbreak in Equatorial Guinea. Then we dive into the long history of a gene therapy from Sarepta Therapeutics, a polarizing medicine that promises to change the lives of patients with Duchenne muscular dystrophy. We also break down the rest of the week's biggest news in biopharma, including some billion-dollar deals and the retirement of perhaps the most storied executive in the business.

Listen here.

monoclonals

BeiGene's tislelizumab does well in stomach cancer

BeiGene's anti-cancer antibody drug, tislelizumab, improved the overall survival of patients with advanced gastric cancer — with or without PD-L1 expression in their tumors, a new study shows. Tislelizumab combined with chemotherapy increased the median overall survival to 17.2 months, FiercePharma writes, compared with 12.6 months if patients were given chemo and placebo.

The drug is already approved for 10 indications in China, and was licensed in 2021 by Novartis for the U.S., Japan, and some European markets. However, approval has been elusive: Last year, a BLA for tislelizumab as a first-line treatment with chemo for patients with some esophageal cancers was indefinitely delayed by the FDA. But U.S. regulators plan to soon inspect BeiGene's China-based manufacturing plant, Biospace writes, which perhaps will move the approval process along more quickly. That said, it faces tight competition from antibody drugs like Keytruda and Opdivo.

european pharma

Activist investor has big ideas for Bayer

A small activist investor group is pushing to split Bayer into its crop science and pharmaceutical divisions, the Financial Times writes. Bluebell Capital Partners owns an undisclosed stake in Bayer, and is pressuring the struggling company to reconfigure its supervisory board. This comes as Bayer CEO Werner Naumann transitions out of the company, and Bill Anderson, formerly of Roche, takes the helm in June.

Bluebell argues that there are "no synergies" between the company's two divisions, and that separating them could generate up to €30 billion. This could help Bayer pay off debt and reinvest in pharmaceuticals.

"If you appoint a new chief executive when the supervisory board is the same monolith, they don't want to do anything, then the CEO is not going to have an easy job," Bluebell founding partner Marco Taricco told the FT.

middlemen

Senators further target the PBM

A fresh congressional effort to reform the ever-elusive pharmaceutical benefit manager: Two senators have unveiled a bipartisan list of policies meant to tamp down on these drug middlemen. Finance Chair Ron Widen (D-Ore.) and Sen. Mike Crapo (R.-Idaho) want to separate PBM revenue from drug prices. They also want Medicare beneficiaries to benefit from the rebates and discounts that PBMs negotiate.

The measures would also increase PBM accountability and transparency to health plan clients, since it's not always clear how much money they're taking from the middle of the supply chain. Earlier this year, Congress passed a bipartisan bill to give the FTC more power to oversee PBMs.

"If you're really going to modernize PBMs, and that is what we really feel strongly about, you can't do it without looking at those particular agencies," Wyden told STAT.