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At this time, we see a gene remedy reach serving to sufferers with a uncommon liver illness preserve their blood sugar. We see an Ionis drug do higher than already-approved rivals in a uncommon swelling illness, and we see some conflict-of-interest points from medical doctors on X.
Additionally, ASCO begins right this moment! If you happen to haven’t already, join our flash ASCO publication right here. STAT’s Matt Herper, Adam Feuerstein, Angus Chen, and Elaine Chen will likely be reporting from McCormick Place in Chicago.
The necessity-to-know this morning
- Advisers to the European Medicines Company recommended the approval of a gene remedy for hemophilia B from Pfizer.
- Gilead Sciences stated its most cancers medication Trodelvy failed to improve survival in sufferers with superior metastatic bladder most cancers. The Part 3 TROPiCS-04 research was designed to substantiate Trodelvy’s profit in bladder most cancers sufferers. The drug was granted accelerated approval in 2021.
Pfizer’s Lorbrena reduces uncommon lung most cancers development by 81%
New information being offered at ASCO on the long-approved Pfizer drug Lorbrena are exceptional: The every day capsule decreased illness development in non-small cell lung most cancers by 81% over 5 years. That signifies that 60% of sufferers taking the drug have been alive and didn’t see their tumors progress in that point, in comparison with 8% who obtained Xalkori, one other Pfizer most cancers drug.
Lorbrena was accepted all the best way again in 2001 for non-small cell lung most cancers. It’s not precisely a prime vendor — it introduced in $575 million final yr, accounting for simply 1% of the drug behemoth’s annual gross sales. So it’ll be fascinating if the up to date information will transfer the needle for gross sales of both Pfizer drug.
Learn extra.
Ultragenyx gene remedy succeeds in late-stage trial
A gene remedy developed by Ultragenyx for a uncommon liver illness was profitable in a Part 3 trial — paving the best way for a possible approval. The situation, referred to as glycogen illness sort 1A, is brought on by a genetic mutation that forestalls sufferers from sustaining blood sugar ranges. Although it was as soon as deadly, it’s now managed with common doses of cornstarch — but it surely’s crucial sufferers don’t miss even a single dose.
In a research of 49 sufferers, those that obtained the Ultragenyx remedy might take 41% much less cornstarch than initially of the trial. As compared, sufferers within the placebo arm solely took 10% much less. Whereas that’s statistically important, the outcomes are much less profound than an earlier trial, which decreased sufferers’ wants by 72%. The gene remedy additionally failed a secondary endpoint.
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Ionis drug outperforms different angioedema medicine on market
Donidalorsen, an RNA-targeting therapy for hereditary angioedema made by Ionis Prescription drugs, helped enhance affected person signs in a pair of late-stage trials — even for many who are taking drugs for the illness which have already been accepted. After 25 weeks, sufferers given the drugs month-to-month had 81% fewer bouts of extreme swelling in comparison with placebo; when sufferers got the drug each two months, the charges have been nonetheless down 55%.
One other research confirmed that as time progressed, donidalorsen’s advantages grew — with swelling assaults dropping by 92% to 93%, no matter how usually sufferers have been dosed. Apparently, the sufferers who switched to donidalorsen from present therapies from rivals like Takeda and BioCryst Prescription drugs noticed their swelling assault charges drop by 62% in comparison with their earlier therapy.
Ionis will use these information to use for FDA approval by the top of the yr. Otsuka, which has partnered with Ionis for European rights to the drug, may even apply this yr for approval there.
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An ASCO preview and one other Duchenne trial failure
Which shows are price catching at ASCO this weekend? What’s exon-skipping? And the way do you pronounce bronchiectasis?
This week on “The Readout LOUD,” we preview a few of the analysis that will likely be offered on the American Society of Scientific Oncology convention in Chicago. We additionally talk about the newest information within the well being and life sciences, together with a milestone in lung illness R&D and a Duchenne muscular dystrophy confirmatory trial failure.
Hear right here.
Medical doctors on X who endorse medicine received paid by producers
Many multitudes of physicians weigh in on X — previously Twitter — and sometimes endorse the prescribed drugs they depend on most. However a brand new research reveals that it’s possible that many have been paid, in some unspecified time in the future or one other, by the makers of those very medicine.
Monitoring 28 physicians on X, the JAMA evaluation discovered that 26 obtained not less than one cost. And the typical was greater than $27,400 for issues like meals and drinks, talking, consulting, or journey. In 24 circumstances, the funds have been linked to a drug or machine the physicians actively endorsed on social media.
Apparently, two-thirds of the medical doctors who made these endorsements didn’t rating extremely on an index used to measure tutorial productiveness, STAT’s Ed Silverman writes. Almost half of the physicians didn’t disclose compensation from the producers. The research’s authors are involved that “the battle of curiosity… is probably not obvious to the overall social media viewers” as a result of monetary ties “weren’t persistently disclosed in posts.”
Learn extra.
Extra reads
- Did Sarepta Therapeutics earn $4 billion from exon-skipping medicine that don’t work? STAT
- Summit shares soar after most cancers drug tops Merck’s Keytruda, Bloomberg
- Wegovy maker Novo Nordisk sues 9 spas, clinics and pharmacies over copycat medicine, Reuters
- ADHD recreation developer Akili Interactive to be taken non-public, after $34 million sale, FierceBiotech
