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Hey there. At the moment, we get into why Amy Abernethy is leaving Verily, and why the continuing reckoning with AI doesn’t essentially want the voice of Google to chime in. Plus, Jason Mast pops in to present an replace on Uniqure’s puzzling method to presenting trial outcomes.
The necessity-to-know this morning
- Argenx mentioned its intently watched antibody remedy didn’t outperform placebo in a Part 3 trial in an autoimmune situation that causes the pores and skin to blister — the second setback in lower than a month for the Belgian biotech and its drug.
- Bluebird Bio raised $125 million in a inventory providing priced at $1.50 per share — 50% decrease than the worth of its shares on Monday. Bluebird not too long ago received approval for a gene remedy to deal with sickle cell illness, however a dire want for money to fund the launch meant it had no leverage with buyers.
Verily CMO Amy Abernethy leaving to begin nonprofit
Amy Abernethy, Verily’s chief medical officer, can be leaving on the finish of the primary quarter to launch a startup targeted on how well being care techniques gather knowledge. It looks as if an abrupt transfer after two and a half years, however Abernathy and Verily insist the corporate’s in a superb place for her to go away: “Now’s the time for me to say ‘you’ve bought this,’ as a result of they completely do, and allow them to maintain working, whereas I am going and maintain fixing among the different issues,” she informed STAT’s Matthew Herper solely.
Abernethy, an oncologist, was second-in-command on the FDA and earlier than that labored at well being tech unicorn Flatiron Well being. She informed STAT that she discovered that progress at Alphabet would possibly sluggish as a result of well being care isn’t its major focus as a enterprise. Quickly, she mentioned, large coverage choices can be made about how AI impacts real-world proof in well being care, and that her affect would probably wane.
“You realize, Google’s stamp, or Alphabet’s stamp, on a privateness framework is probably not what the entire world needs anyway,” Abernethy mentioned.
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The complicated factor about Uniqure’s knowledge
A yr and a half in the past, Uniqure generated pleasure within the Huntington’s group and amongst buyers when it introduced an experimental gene remedy designed to remove the poisonous, neuron-killing mutant protein believed to drive the illness appeared to only that: Ranges of mutant Huntington protein, mHTT, in sufferers’ spinal fluid fell 53% and the corporate’s inventory rose 26%.
Knowledge since then have been puzzling. In June, Uniqure introduced mHTT ranges had returned to near-normal in lots of sufferers and, in some, ranges truly appeared to extend. Then, yesterday, it declined to launch a quantity in a press launch saying the info, saying solely that it believed the mHTT exams could also be unreliable. A graph in a presentation indicated mutant protein ranges have been basically flat in comparison with a management group.
There’s purpose to suppose mHTT ranges in spinal fluid is perhaps unreliable. Huntingtin protein assays might be difficult. And the gene remedy is injected deep within the striatum, the part of the mind the place Huntington’s wreaks havoc — it’s potential ranges are falling there however not in different elements of the mind or the spinal fluid, the place it may be measured. In the meantime, different measures point out the drug may very well be slowing the illness, in comparison with pure historical past. “There’s an preliminary set of knowledge that’s beginning to make me really feel assured that we’re seeing a dose-dependent impact, and that to me is proof of organic exercise,” Chief Medical Officer Walid Abi-Saab informed STAT’s Jason Mast.
The consequence, although, is that Uniqure doesn’t have a direct measure that its gene remedy is doing what it’s meant. It’s inventory, already pummeled in June, fell one other 24% yesterday.
Three issues we’ll watch in biotech subsequent yr
This coming yr, we’ve bought our eyes on three tendencies, particularly, in biotech. As STAT’s Damian Garde writes, 2024 is the yr GLP-1 medicine get actually actual: Demand has outpaced provide fairly dramatically up to now, however as that eases up we’ll see whether or not insurers will truly reimburse for them. We’ll additionally see new knowledge on how the medicine work in MASH (previously NASH), sleep apnea, and dependancy cessation, amongst others.
There may also be key readouts in CRISPR medicines and in non-opioid ache remedies. And it’s an election yr, after all — which signifies that political tongue-wagging (recall allegations of “getting away with homicide”) might influence how the general public, and the market, understand the trade.
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Inhaled vaccines might cease Covid infections, monkey research present
Mucosal vaccines, inhaled straight to the nostril and lungs, can abruptly cease Covid viruses, a trio of monkey research revealed in Nature exhibits. That is the primary actual proof that mucosal vaccines is perhaps efficient, Nature writes. The supply technique might supply ‘sterilizing’ immunity — during which an an infection is totally blocked.
“It’s not full science fiction to consider creating vaccines that may cease transmission and an infection,” one Yale immunologist informed Nature.
Though vaccines softened the pandemic, their efficiency is short-lived. That is probably as a result of intramuscular vaccines work greatest deep within the lungs, however are much less efficient within the mucosa. So there’s hope that aerosolized vaccines, particularly, would possibly cease infections increased up within the airways. Dozens of mucosal Covid vaccines are nonetheless being developed.
Extra reads
- Otsuka pays $65 million in money for European rights to Ionis’ section 3 uncommon illness medication, FierceBiotech