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Good morning! It’s an ASCO-full e-newsletter at this time (and you’ll want to peruse the Extra Reads for extra reads) however there’s a lot extra this week. We’re previewing tomorrow’s advisory committee on the carefully watched MDMA remedy from Lykos Therapeutics, and each Jonathan Wosen and I can be at BIO! Hope to see you there.
The necessity-to-know this morning
- Agios Prescription drugs mentioned its drug referred to as mitapivat decreased the necessity for blood transfusions in sufferers with a extreme type of beta-thalassemia, an inherited blood dysfunction. The outcomes achieved the first aim of a placebo-controlled Section 3 medical trial, and if the drug is ultimately cleared by regulators, might speed up gross sales.
- Construction Therapeutics reported that its oral GLP-1 drug led to weight reduction in two research, main shares to surge in pre-market buying and selling.
- Arrowhead Prescription drugs reported positive results from a Section 3 research of its RNAi-based remedy for familial chylomicronemia syndrome, an ultra-rare dysfunction that causes excessive ranges of triglycerides.
What to anticipate from the MDMA advisory committee
Later this summer season, the FDA will resolve whether or not or to not approve MDMA as a remedy for PTSD. It’s an amazing step for validating psychedelic medication as therapies, but it surely’s one being weighed with excessive warning. On Tuesday, a panel of impartial scientific advisers will evaluation information on MDMA’s security and efficacy, and share their suggestions with regulators. STAT’s Olivia Goldhill outlines the important thing points you need to look out for on this high-profile advert comm.
One challenge the panelists could flag is expectancy bias: Though the MDMA research underneath evaluation are double-blinded, it’s onerous to fake a substance is a psychedelic when it’s not — and that information doubtless impacts how a affected person responds to a remedy. There are particular issues of safety and information suppression considerations to contemplate, and looming questions round how the FDA will regulate remedy.
However there hasn’t been a brand new therapy for PTSD in additional than 20 years — and lots of are clamoring for brand spanking new, efficient interventions for this essential psychological well being challenge.
Learn extra.
Can AstraZeneca make good on its promise?
AstraZeneca has dominated the annual assembly of the American Society of Scientific Oncology. And it’s hardly the primary time.
Actually, 2024 marks the sixth 12 months in a row that the London-based drug large has had a presentation within the ASCO plenary, the convention’s largest showcase for brand spanking new information. This 12 months, the corporate has two.
However as STAT’s Matthew Herper notes, whereas scientific credibility is nice, shareholders need one thing else: return on funding. That’s what CEO Pascal Soriot not too long ago promised, when he mentioned that in a mere six years the corporate would enhance its annual gross sales to $80 billion.
Matt, reporting from the convention, takes a more in-depth look at this time at AstraZeneca and its pipeline. Learn extra.
Enhertu might change therapy requirements for some breast cancers
The blockbuster most cancers drug Enhertu delayed tumor development for greater than a 12 months in girls with a typical type of metastatic breast most cancers, new information introduced at ASCO present. It was considerably stronger than chemotherapy, prompting makers Daiichi Sankyo and AstraZeneca to say that if it’s cleared by regulators, this may turn out to be the brand new normal of look after sufferers expressing even the faintest quantity of HER2 protein, STAT’s Adam Feuerstein writes.
“It’s not an overestimation to say that trastuzumab deruxtecan is probably the most potent drug ever developed for breast most cancers,” a Dana-Farber Most cancers Institute medical oncologist instructed STAT. “It does include unwanted effects that we’re studying to handle, however with that mentioned, I’ve by no means seen information like what’s being reported right here.”
Learn extra.
Corbus ADC outperforms Pfizer’s ADC
CRB-701, an antibody-drug conjugate made by Corbus Prescription drugs, labored higher in bladder most cancers than Pacdev, an identical drug that Pfizer acquired as a part of its $43 billion Seagen deal. This underscores, maybe, why Corbus is presently the second best-performing biotech inventory this 12 months, STAT’s Adam Feuerstein writes. At Friday’s shut of $42.80, the corporate’s inventory value had risen 609% this 12 months — second solely to Elevation Oncology, which can also be growing antibody-drug conjugates.
At ASCO, Corbus confirmed that its ADC had an total response price of 28% from 25 evaluable sufferers. In a subset of 15 sufferers, the general response price was 40%.
Learn extra.
GSK blood most cancers drug Blenrep has an opportunity to reenter the market
Lower than two years in the past, GSK pulled its blood most cancers drug Blenrep from the market. However now there’s likelihood for resurrection: In a number of myeloma, Blenrep outperformed normal therapies when mixed with one other medication, Pomalyst. That is the second constructive research of late for Blenrep, which might sign a return to the FDA for a re-approval. There was a whole response price of 40% for sufferers who obtained Blenrep, versus 16% within the management group, information introduced at ASCO present.
That mentioned, physicians produce other instruments of their arsenal that could be difficult for Blenrep gross sales, STAT’s Andrew Joseph writes — outdoors of its spotty historical past of efficacy earlier than it was pulled from the market.
Learn extra.
Extra reads
- Intellia’s CRISPR-based therapy dramatically decreased sufferers’ swelling assaults in small trial, STAT
- AstraZeneca’s Tagrisso significantly slows most cancers for some individuals with stage 3 lung most cancers, STAT
- Jazz shares ‘unprecedented’ HER2 biliary tract most cancers information forward of FDA choice, Fierce Biotech
- FDA oncology head needs medical trials to vary past China alone, STAT