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Good morning. It’s the primary actual workweek of 2025 and we’re already beginning to get a pickup in information once more, so let’s get straight into it.
The necessity-to-know this morning
- Privately held Metsera reported placebo-adjusted weight lack of 6% in a cohort of 20 members handled for 12 weeks with a titrated month-to-month dose of its experimental GLP-1 injection, referred to as MET-097i.
- Privately held Orna Therapeutics and Vertex Prescribed drugs signed a analysis collaboration to make use of Orna’s non-viral supply expertise in an effort to develop an in-the-body, genetic therapy for sickle cell illness.
Google’s anti-aging moonshot disappoints
Again in 2013, Google stated it will try to grasp and finally gradual growing older via an organization referred to as Calico. The primary knowledge have lastly emerged from a drug Calico developed — it failed.
The capsule, generally known as fosigotifator, was being examined in ALS. It wasn’t designed to gradual growing older itself. The FDA doesn’t think about growing older a illness, so most so-called “longevity” corporations have began by tackling particular age-related ailments.
In a big, multi-arm trial, fosigotifator didn’t gradual sufferers’ decline greater than placebo over a 24-week interval, though Calico did be aware that sufferers who obtained an “exploratory excessive dose” appeared to keep up muscle and respiratory operate longer than placebo sufferers.
Learn extra from STAT’s Jason Mast.
FDA tries to start out regulating AI in drug growth
The FDA launched a spate of latest steerage paperwork yesterday forward of the transition to a brand new administration. Amongst them was the company’s first draft steerage on using AI in drug growth.
The draft steerage doesn’t seize how AI is being utilized in drug discovery, however as an alternative focuses on AI fashions used to supply knowledge that assist regulatory decision-making in regards to the security, effectiveness, or high quality of medication.
It suggests sponsors attain out to the FDA early about any potential makes use of of AI, and that they arrive ready with proposed plans to ascertain the credibility of a mannequin’s output — the upper the danger, the extra stringent the factors.
Learn extra from STAT’s Katie Palmer.
FDA needs confirmatory trial enrollment to start out early
The company yesterday additionally launched draft steerage on the confirmatory trials that drugmakers should conduct if their therapies obtain accelerated approval. Again in 2023, Congress had given the FDA the authority to require {that a} confirmatory trial is “underway” previous to granting accelerated approval. This new draft steerage defines how the FDA would outline “underway.”
It says that quickly after the tip of Section 2 trials, drugmakers ought to attain an settlement with the company on the confirmatory trial design. It additionally says that enrollment within the trial ought to have already begun previous to accelerated approval.
Eva Temkin, companion at legislation agency Arnold & Porter, stated that the company had already been encouraging drug corporations to start out enrollment early on a case-by-case foundation. “The actual shift that that I feel FDA is attempting to emphasise of their steerage — that there’s now going to be a presumption for all accelerated approvals, slightly than this case-by-case” foundation, she stated.
Temkin additionally stated it was noteworthy that the draft steerage discusses how making a drug out there via an accelerated approval could make it troublesome to recruit sufferers right into a confirmatory trial. In these instances, drug corporations ought to mitigate this impact, the FDA says, “by finishing all or a good portion of enrollment previous to accelerated approval.”
The draft steerage does depart some flexibility for medication in very uncommon ailments. In conditions the place there are significantly small populations with excessive unmet want, and if acceptable justification is offered, the company might not require a confirmatory trial be “underway” previous to accelerated approval, the doc says.
J&J’s lung most cancers drug prolonged survival
From my colleague Angus Chen: A yr and a half in the past, AstraZeneca confirmed Tagrisso may cut back the danger of demise by 51% for sufferers with non-small cell lung most cancers, an achievement that consultants referred to as “extraordinary.” Quickly, sufferers might get one more main advance within the therapy of EGFR-mutant non-small cell lung most cancers. The topline outcomes of the MARIPOSA trial confirmed that Johnson & Johnson’s Rybrevant mixed with Lazcluze has a major general survival benefit over Tagrisso alone, based on firm officers.
Sufferers with superior most cancers usually reside a median of three years on Tagrisso, stated Biljana Naumovic, president of U.S. oncology stable tumors at J&J. The mix of Rybrevant and Lazcluze ought to lengthen that by a minimum of one yr, she stated. “That is actually the primary time in a decade that any mixture of therapies has proven general survival profit versus normal of care.”
The median general survival has not but been met within the MARIPOSA trial, Naumovic stated.
Can an epilepsy drug do greater than cut back seizures?
From my colleague Adam Feuerstein: In a first-of-its-kind Section 3 research, Stoke Therapeutics will assess the impact of its experimental therapy on seizures, but in addition consider potential enhancements in cognition and habits of sufferers with Dravet Syndrome, a extreme type of epilepsy.
The corporate stated as we speak that it reached an settlement with the FDA, together with regulators in Europe and Japan, for it to green-light the Section 3 research of zorevunersen in the midst of the yr.
“To point out enchancment in cognition would set us other than each different drug that’s accredited for Dravet. It’s by no means been finished earlier than,” Stoke CEO Ed Kaye instructed STAT in December, after the firm offered promising new knowledge from a mid-stage research.
The Section 3 research, dubbed EMPEROR, will consider zorevunersen in opposition to a sham comparator in roughly 150 members with Dravet aged 2-18. Whereas reductions in seizure frequency — the research’s major aim — can often be measured after three to 4 months, Stoke will run the research for a full yr in order that it might absolutely assess secondary endpoints that can monitor modifications in cognition and habits.
“EMPEROR is not like any research ever performed for Dravet syndrome. It’s going to be longer and extra concerned than research of anti-seizure medicines, however the potential impacts of illness modification, as we now have been discussing, are monumental,” stated Kim Parkerson, Stoke’s head of neurology medical growth.
Eli Lilly allowed to intervene in compounding lawsuit
Yesterday, we wrote that Eli Lilly wished to intervene in a lawsuit {that a} compounding commerce group filed in opposition to the FDA, over the company’s declaration that Eli Lilly’s tirzepatide (aka Mounjaro/Zepbound) is now not in scarcity.
A fast replace on that entrance — the decide has granted Lilly’s request to hitch the lawsuit as a defendant.
The commerce group, referred to as the Outsourcing Services Affiliation, has been arguing that the scarcity isn’t really over and compounding pharmacies ought to nonetheless be capable of make copies of the branded therapies. Lilly, in the meantime, has wished to hitch the lawsuit “defend its pursuits and assist carry this swimsuit to a swift finish,” based on a authorized submitting final week.
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