Pfizer gene therapy for Duchenne fails in Phase 3

Pfizer's late-stage, placebo-controlled trial for gene therapy for Duchenne muscular dystrophy failed to slow the disease's progression. This is the second large gene therapy trial for Duchenne that did not meet its primary endpoint: A similar one from Sarepta also failed last year.

"We are extremely disappointed that these results did not demonstrate the relative improvement in motor function that we had hoped," Pfizer said in a statement.

It's still unclear why these trials aren't working as well in late-stage trials as they did in earlier studies. But Pfizer said it plans on sharing more detailed results from the study at upcoming medical and patient advocacy meetings in order to "help improve future clinical research" and improve the lives of the kids with this disease.

rna therapy

Avidity's antisense drug effective in FSHD

An experimental RNA therapy succeeded in silencing the gene that causes FSHD, a form of muscular dystrophy that's being pursued by several drugmakers. The Avidity Biosciences treatment, del-brex, decreased the levels of the toxic gene by 53% in an early-stage trial treating eight patients. Blood tests also confirmed improvements in muscle function, STAT's Jason Mast writes.

"This is a big deal for Avidity and the FSHD field," analysts at Cantor Fitzgerald wrote in an investor note. Avidity's stock jumped 32% following the news.

regulatory

Europe's looming regulatory policy worries gene therapy companies

Europe will soon upend the way it determines whether medicines are effective — and gene therapy makers are worried the plan could impede patient access. Beginning next year, the continent will standardize its "health technology assessment," or HTA — a review process being done in part by individual country health systems, and also in parallel by the European Medicines Agency.

Gene therapy makers argue that the new guidelines could undervalue their products — preventing them from gaining traction in Europe. Although they cost millions, gene therapies can vastly improve patient lives — but they're often approved based on single-arm trials, as opposed to randomized, controlled trials.

European authorities are holding tight to the gold standard of RCTs, and that could play into new HTA policy — threatening gene therapy companies that are often unable to conduct large, placebo-controlled trials.

adam's take

Will Geron be acquired?

Pardon the interruption. Adam Feuerstein, here with a quick look at this week's Biotech Scorecard newsletter.

The recent FDA approval of Geron's drug to treat myelodysplastic syndrome, a type of blood cancer, has triggered investor hopes of a buyout by a larger pharma company. The stock is up about 30% on speculation that Geron, one of the industry's oldest companies, won't be independent for long. You know I love a contrarian take, so I reached out to a health care investor who's short Geron. His view? Investors expecting biotech's "buyout mania" to bless Geron will be sorely disappointed.

Next, I examine Candel Therapeutics and its immunotherapy for lung cancer, mostly to illustrate why survival claims from non-randomized studies are too often red flags. This makes me sound like a biotech nerd. Guilty, as charged.

Finally — because I can't let this story go — an update on the Novartis-pelabresib saga, including a zesty theory that internal backstabbing may have led Novartis to foolishly embrace MorphoSys while walking away from a rumored buyout of Cytokinetics.

Adam's Biotech Scorecard is delivered to STAT subscribers every Thursday morning. You can sign up for your own email copy here, or read it on the web here.

drug pricing

Who created the $35 insulin cap?

There's that widely publicized $35 cap on insulin prices — but who came up with that magic number? Both Presidents Trump and Biden have claimed it was their doing, but Eli Lilly actually gets the credit. Back in 2019, it first proposed an experiment to offer $35 monthly insulin to Medicare users, CEO David Ricks told STAT's Rachel Cohrs Zhang.

It actually took bipartisan efforts to push Lilly's pilot project through to fruition: The Trump administration ran with the $35 plan and it expanded across several states. Then, the Biden administration expanded it.

"I know the two political parties don't like to give credit to anyone on the other side," Ricks said. "But the reality is, this is like a great example of how government should work."

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