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Read newsletter: Pfizer, Avidity, Geron updates




Read newsletter: Pfizer, Avidity, Geron updates

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Good morning, it’s Meghana today! Some gene therapy talk: Pfizer’s gene therapy for Duchenne muscular dystrophy failed in Phase 3, and gene therapy developers say looming regulatory changes in Europe could jeopardize such treatments. We dive into the backstory of the $35 insulin limit, and more.

Pfizer gene therapy for Duchenne fails in phase 3

Pfizer’s late-stage, placebo-controlled gene therapy trial for Duchenne muscular dystrophy failed to slow the progression of the disease. This is the second major gene therapy trial for Duchenne that failed to meet its primary endpoint: a similar trial 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 for,” Pfizer said in a statement.

It’s still unclear why these late-stage studies don’t work as well as in previous studies. But Pfizer said it plans to share more detailed results from the study at upcoming medical and patient meetings to “help improve future clinical research” and improve the lives of the children with the disease.

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Avidity’s antisense drug is effective in FSHD

An experimental RNA therapy managed to knock out the gene that causes FSHD, a form of muscular dystrophy being pursued by several drug makers. Avidity Biosciences’ treatment, del-brex, reduced levels of the toxic gene by 53% in an early-stage study that treated eight patients. Blood tests also confirmed improvements in muscle function, writes STAT’s Jason Mast.

“This is a big deal for Avidity and the FSHD field,” Cantor Fitzgerald analysts wrote in an investor note. Avidity shares rose 32% after the news.

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The looming European regulatory policy worries gene therapy companies

Europe will soon shake up the way it determines whether drugs are effective — and gene therapy makers worry the plan could hinder patient access. Early next year, the continent will standardize its ‘health technology assessment’ or HTA – an evaluation process carried out in part by individual countries’ healthcare systems, and in parallel by the European Medicines Agency.

Gene therapy makers argue the new guidelines could undervalue their products, preventing them from gaining traction in Europe. Although they cost millions, gene therapies can vastly improve patients’ lives, but they are often approved on the basis of single-arm trials, as opposed to randomized, controlled trials.

European authorities are firmly committed to the gold standard of RCTs, and that could play a role in new HTA policies – a threat to gene therapy companies that are often unable to conduct large, placebo-controlled trials.

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Who Made the $35 Insulin Cap?

There is a well-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. In 2019, it first proposed an experiment to offer $35 monthly insulin to Medicare recipients, CEO David Ricks told STAT’s Rachel Cohrs Zhang.

In fact, it took a bipartisan effort to bring Lilly’s pilot project to fruition: The Trump administration adopted 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 that this is a good example of how government should work.”

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Will Geron be acquired?

Excuse 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 form of blood cancer, has raised investor hopes of a takeover by a larger pharmaceutical 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 like a contrarian approach, so I reached out to a healthcare investor who is short-changing Geron. His vision? Investors expecting biotech buyout mania to benefit Geron will be sorely disappointed.

I then examine Candel Therapeutics and its immunotherapy for lung cancer, mainly 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 spicy theory that internal underhandedness may have led Novartis to foolishly embrace MorphoSys while walking away from a rumored acquisition 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 here on the web.

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