Eli Lilly, Sarepta, Alnylam Pharma news
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Good morning. Right here in Chicago, temperatures are excessive and tensions are even greater as two main scorching canine eating places are reportedly beefing. Apparently PETA is concerned and taunted one of many eating places with a “pig cellular” although that restaurant doesn’t even serve pork.
In any case, right here’s the biotech information of the day.
A uncommon genetic mutation might delay early Alzheimer’s
A brand new examine bolsters proof {that a} uncommon genetic mutation in apolipoprotein E3 (APOE3) might assist delay early Alzheimer’s, supporting efforts to develop therapies mimicking the mutation.
Researchers have lengthy studied members of an prolonged household who carry a genetic variant that ensures they’ll get Alzheimer’s. Scientists beforehand discovered that one girl on this household with two copies of the APOE3 mutation developed Alzheimer’s 30 years later than anticipated. This new examine now finds that individuals with only one copy of the APOE3 mutation additionally developed the illness a number of years later than individuals with none copies of the mutation.
The researchers discovered that whereas the individuals with the APOE3 mutation had loads of amyloid, a protein that varieties plaques within the mind related to illness, these individuals had surprisingly little tau, a special protein that accumulates inside neurons and is linked to Alzheimer’s illness severity.
Learn extra from STAT’s Jonathan Wosen.
Awaiting FDA’s determination on Sarepta’s Duchenne therapy
From STAT’s Jason Mast: Someday as we speak or tomorrow, the FDA is anticipated at hand down a call on whether or not it’s going to broaden its approval of Elevidys, Sarepta’s gene remedy for Duchenne muscular dystrophy.
The therapy has already misplaced a lot of the luster it held a number of years in the past, when there was hope it’d remedy or halt the illness, earlier than it failed two consecutive randomized, placebo-controlled medical trials. Nonetheless, many docs and households see it as the most effective factor they’ve ever been capable of provide their sufferers and the most effective factor on the horizon for at the very least a pair years.
The company appears probably — primarily based partly on feedback from prime official Peter Marks — to at the very least partially increase the approval for Elevidys, which is at present licensed just for 4- and 5-year olds. But it surely’s unclear how broadly it’s going to authorize entry. Sarepta and far of the Duchenne group are hoping the company will approve the therapy for almost all sufferers with muscular dystrophy, no matter age or wheelchair standing.
But Sarepta has performed solely restricted research in older sufferers, in whom the therapy may very well be riskier and fewer useful, and hasn’t launched any knowledge. An alternative choice is an approval for under boys who can nonetheless stroll or boys aged 4 to 7, the inhabitants studied in medical trials.
Lilly takes extra actions towards unapproved variations of Mounjaro
After taking authorized actions final yr, Eli Lilly this morning filed even more lawsuits towards med spas and wellness facilities over unapproved variations of tirzepatide, the energetic ingredient in Mounjaro and Zepbound. Lilly mentioned these entities misleadingly use the model names, posing risks for affected person security.
The pharma firm additionally issued an open letter warning the general public in regards to the dangers of those merchandise.
With the immense demand for GLP-1-based medicine and recurring shortages of the branded remedies, all kinds of companies — from telehealth corporations to med spas and compounding pharmacies — have popped as much as provide their very own variations of what they declare are GLP-1 medicine.
How a lot threat do compounded merchandise truly pose? It’s a sophisticated query. Compounding pharmacies are technically allowed to make copycat variations of authorized medicines if these therapy are in scarcity. Lilly argues in its open letter, although, that compounded merchandise in the end are usually not FDA-approved and lack the identical high quality protections as FDA-approved medicine. To be taught extra about this challenge, my colleague Ed Silverman wrote a primer on this final yr.
Except for compounding pharmacies, sufferers have additionally turned to “analysis” web sites that declare to promote the underlying chemical substances in GLP-1 medicine. Sufferers have ordered from these web sites, after which combined and injected the chemical substances themselves at residence. Lilly additionally referred to as out the dangers of those web sites in its open letter.
Process pressure recommends behavioral remedy over weight problems medicine for kids
In new suggestions issued this week, the U.S. Preventive Companies Process Power favored intensive behavioral remedy over weight problems medicine like Wegovy for kids and youths with a excessive physique mass index.
The duty pressure mentioned the proof of advantages of weight problems medicines is “insufficient” as a result of small variety of research and restricted knowledge on long-term therapy hurt. This differs from suggestions from the American Academy of Pediatrics, which mentioned weight problems medicine must be thought-about for teenagers 12 and older whose weight tops progress charts.
There’s been heated debate on whether or not youngsters and youngsters must be broadly prescribed weight problems medicine. Some docs have raised issues in regards to the lack of information not solely on the bodily long-term results of the medicine, but additionally on how the remedies might have an effect on psychological well being.
Learn extra from STAT’s Liz Cooney.
An infinite stock-moving occasion for Alnylam Prescription drugs
STAT’s Adam Feuerstein jumps in with a quick have a look at his Biotech Scorecard e-newsletter this week:
This week’s Biotech Scorecard is devoted totally to a preview of Alnylam Pharma’s HELIOS-B examine. For these unfamiliar, HELIOS-B is a Section 3 cardiovascular outcomes examine designed to reply one primary query: Can Alnylam’s RNA-interference remedy referred to as vutrisiran enhance the lives of sufferers with the progressive coronary heart illness ATTR-CM?
The examine consequence — anticipated on the finish of June or early July — is a gigantic stock-moving occasion for Alnylam. Billions of {dollars} in potential income are on the road. The outcomes may also affect opponents, together with BridgeBio Pharma and Ionis Prescription drugs.
HELIOS-B is without doubt one of the greatest binary occasions in biotech this yr. The outcomes are additionally prone to be a bit sophisticated. Hopefully, this week’s e-newsletter will function your information. Inside, you’ll discover background data, a have a look at how buyers are defining success or failure, and a few feedback from Alnylam CEO Yvonne Greenstreet.
Lastly, an investor perspective from Daniel Schneeberger, founder and chief funding officer at Adar1 Capital Administration, a biotech hedge fund. If you happen to’re a Twitter/X biotech individual, you understand Dan because the broadly adopted “Sheep of Wall Street.”
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
- How a Baltimore neuroscience examine is rewriting Black America’s relationship with medical analysis, STAT
- Substance use consultants are sounding the alarm on one other dependancy: playing, STAT
- FTC backs plan to focus on extra patent settlements it sees as anticompetitive, Endpoints
