September 7, 2023

The need-to-know this morning

CymaBay Therapeutics said its experimental treatment for primary biliary cholangitis, or PBC, a rare liver disease, achieved the efficacy goals of a placebo-controlled, Phase 3 clinical trial. The drug, called seladelpar, also reduced itching, a particularly bothersome symptom for people with PBC.

Pricing

What's really at stake with Medicare negotiation?

Ask any drug company: If you force them to charge less than whatever the market will bear for their new medicines, you'll set in motion a vicious cycle that will only harm patients in the end. What you don't often hear is precisely how.

STAT asked 50 of the largest companies in the industry the straightforward question of how, specifically, the dawn of Medicare pricing negotiations is changing their plans for developing new drugs. Only two of the companies offered substantive answers, while most responded with generalities, and a few ignored the question entirely.

The reticence is understandable, as STATs Ed Silverman writes. There are competitive reasons for secrecy, and getting specific risks alienating patient groups and trial investigators. But it's also a missed opportunity for an industry with a poor reputation to offer a cogent explanations to a public that is increasingly skeptical of its motivations.

Read more.

Personnel

Maybe Biogen should mix things up

For years, the knock on Biogen was that it didn't do enough deals to complement its high-risk pipeline of neuroscience treatments. The defense was pointing to the many foundering neuroscience companies in biotech and asking, OK, which of those would have been wise to purchase? Often unspoken was a third idea: Who said Biogen had to be a neuroscience company?

Yesterday, the company finally named a permanent replacement for the venerated head scientist Al Sandrock, hiring a decorated researcher whose résumé has little to do with neurology. Jane Grogan, most recently of Graphite Bio, had a long career at Genentech developing autoimmune and oncology medicines and did pioneering work on the cancer target TIGIT.

Biogen, working under a new CEO and a freshly updated board, isn't explicitly pivoting from its focus on diseases of the brain. But hiring Grogan suggests the new administration might be listening to the critics who have urged the company to diversify.

In the lab

CAR-T's second act is coming

The dawn of CAR-T cancer therapy brought dramatic results for people with desperate diagnoses. Now, as the technology enters its second decade, the field is intent on figuring out how to extend those benefits to more and more patients.

CAR-T treatments have changed the lives of some 7,000 patients, but manufacturing roadblocks have curtailed access, and scientific hurdles have limited their scope. Democratizing the therapy will require making it "more patient-friendly," David Chang, head of Allogene Therapeutics, said at the STAT 2023 Future Summit. That means improving the tolerability of current medicines and accelerating the development of off-the-shelf CAR-T therapies that can be more easily administered.

"How we scale and bring the promise of cell therapy to more patients is really, really critical," said Lynelle Hoch, senior vice president and global cell therapy franchise lead at Bristol Myers Squibb. "We do believe at BMS that off-the-shelf solutions have to be a part of that in order to reach the full potential of cell therapy and have more patients that are eligible for cell therapy to receive that. We also believe scientifically there's probably going to need to be both."

Read more.

Policy

Astellas won't have its day in court after all

Japanese drugmaker Astellas pulled its lawsuit against the U.S. government after learning that its blockbuster cancer medicine would not, in fact, face Medicare pricing negotiation.

As STAT's Rachel Cohrs reports, Astellas' pre-emptive lawsuit claimed the Inflation Reduction Act violated its constitutional rights, echoing the claims of drugmakers including Merck, Johnson & Johnson, and Bristol Myers Squibb, among others. Then, last week, Medicare published the list of 10 drugs up for negotiation, and the prostate cancer treatment Xtandi wasn't on it, meaning Astellas' inalienable rights remain intact.

You might wonder why Astellas would go through the trouble — and expense — of drafting and filing a legal challenge before it was certain any of its drugs would actually be affected by the law. It might not be a sunk cost, though; Xtandi could end up among the next round of negotiable drugs come 2027, at which point Astellas could simply dust off its lengthy legal argument.