Readout Newsletter: Pfizer, Avidity, Geron updates
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Morning, it’s Meghana right this moment! Some gene remedy discuss: Pfizer’s gene remedy for Duchenne muscular dystrophy failed in Part 3, and gene remedy builders say Europe’s looming regulatory adjustments could endanger such remedies. We delve into the backstory of the $35 insulin cap, and extra.
Pfizer gene remedy for Duchenne fails in Part 3
Pfizer’s late-stage, placebo-controlled trial for gene remedy for Duchenne muscular dystrophy did not gradual the illness’s development. That is the second massive gene remedy trial for Duchenne that didn’t meet its main endpoint: An identical one from Sarepta additionally failed final 12 months.
“We’re extraordinarily disenchanted that these outcomes didn’t reveal the relative enchancment in motor perform that we had hoped,” Pfizer stated in a press release.
It’s nonetheless unclear why these trials aren’t working as nicely in late-stage trials as they did in earlier research. However Pfizer stated it plans on sharing extra detailed outcomes from the research at upcoming medical and affected person advocacy conferences with a purpose to “assist enhance future scientific analysis” and enhance the lives of the children with this illness.
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Avidity’s antisense drug efficient in FSHD
An experimental RNA remedy succeeded in silencing the gene that causes FSHD, a type of muscular dystrophy that’s being pursued by a number of drugmakers. The Avidity Biosciences therapy, del-brex, decreased the degrees of the poisonous gene by 53% in an early-stage trial treating eight sufferers. Blood exams additionally confirmed enhancements in muscle perform, STAT’s Jason Mast writes.
“It is a huge deal for Avidity and the FSHD discipline,” analysts at Cantor Fitzgerald wrote in an investor observe. Avidity’s inventory jumped 32% following the information.
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Europe’s looming regulatory coverage worries gene remedy corporations
Europe will quickly upend the way in which it determines whether or not medicines are efficient — and gene remedy makers are frightened the plan may impede affected person entry. Starting subsequent 12 months, the continent will standardize its “well being know-how evaluation,” or HTA — a evaluation course of being completed partly by particular person nation well being techniques, and likewise in parallel by the European Medicines Company.
Gene remedy makers argue that the brand new tips may undervalue their merchandise — stopping them from gaining traction in Europe. Though they value hundreds of thousands, gene therapies can vastly enhance affected person lives — however they’re usually permitted based mostly on single-arm trials, versus randomized, managed trials.
European authorities are holding tight to the gold customary of RCTs, and that would play into new HTA coverage — threatening gene remedy corporations which can be usually unable to conduct massive, placebo-controlled trials.
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Who created the $35 insulin cap?
There’s that broadly publicized $35 cap on insulin costs — however who got here up with that magic quantity? Each Presidents Trump and Biden have claimed it was their doing, however Eli Lilly really will get the credit score. Again in 2019, it first proposed an experiment to supply $35 month-to-month insulin to Medicare customers, CEO David Ricks informed STAT’s Rachel Cohrs Zhang.
It really took bipartisan efforts to push Lilly’s pilot undertaking via to fruition: The Trump administration ran with the $35 plan and it expanded throughout a number of states. Then, the Biden administration expanded it.
“I do know the 2 political events don’t like to offer credit score to anybody on the opposite facet,” Ricks stated. “However the actuality is, this is sort of a nice instance of how authorities ought to work.”
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Will Geron be acquired?
Pardon the interruption. Adam Feuerstein, right here with a fast take a look at this week’s Biotech Scorecard e-newsletter.
The current FDA approval of Geron’s drug to deal with myelodysplastic syndrome, a sort of blood most cancers, has triggered investor hopes of a buyout by a bigger pharma firm. The inventory is up about 30% on hypothesis that Geron, one of many trade’s oldest corporations, gained’t be impartial for lengthy. I like a contrarian take, so I reached out to a well being care investor who’s brief Geron. His view? Buyers anticipating biotech’s “buyout mania” to bless Geron can be sorely disenchanted.
Subsequent, I look at Candel Therapeutics and its immunotherapy for lung most cancers, principally as an instance why survival claims from non-randomized research are too usually pink flags. This makes me sound like a biotech nerd. Responsible, as charged.
Lastly — as a result of I can’t let this story go — an replace on the Novartis-pelabresib saga, together with a zesty concept that inside backstabbing could have led Novartis to foolishly embrace MorphoSys whereas strolling away from a rumored buyout of Cytokinetics.
Adam’s Biotech Scorecard is delivered to STAT subscribers each Thursday morning. You’ll be able to join your personal e mail copy right here, or learn it on the net right here.
Extra reads
- Michigan’s largest insurer to drop weight reduction drug protection, Bloomberg
- With placenta-on-a-chip, researchers hope to gauge how medication and toxins influence being pregnant, STAT
