April 24, 2023

The need-to-know this morning:

No deals. Sorry, merger Mondays can't happen every week.
After a brief delay, Bluebird Bio completed the submission of its gene therapy for sickle cell disease to the Food and Drug Administration.
Foghorn Therapeutics said an experimental cancer drug was placed on clinical hold by the FDA because of a serious, heart-related safety issue reported by a single patient.

Regulation

Europe's planned pharmaceutical overhaul

For two years, the European Commission has been drafting legislation meant to make the European biopharma industry more competitive, and improve access to the medicines it makes. This week, the commission will release a new set of its ambitious proposals.

The legislation will include plans to address drug shortages, rework drug development incentives, and streamline the European Medicines Agency, STAT's Ed Silverman and Andrew Joseph report. This means incentives for orphan drug development, and tinkering with how long exclusive protections last.

"We need to strike a balance between access and availability of medicines, but at the same time, make sure that we preserve innovation, because innovation serves citizens and patients, and at the same time, we need to make sure that we have a vibrant and strong pharmaceutical industry," Stella Kyriakides, the European Commissioner for Health and Food Safety, said last month.

the week ahead

Earnings, FDA decisions, and a confab

It's a big week for earnings, kicking off Tuesday morning with Biogen and Novartis, followed by GSK and Roche on Wednesday. Thursday's earnings calendar is heavy: Abbvie, Bristol Myers Squibb, Eli Lilly, Merck, AstraZeneca and Sanofi are reporting before markets open; Amgen and Gilead Sciences report after the close.

We'll have more to say on earnings when the numbers come in, but a lot of eyes will be on Lilly given the intense interest in its diabetes/obesity treatment Mounjaro. The consensus U.S. sales estimate is $346 million, according to the Factset. Investors will also be listening for any update or Lilly management comments regarding its Alzheimer's drug donanemab, given the expected readout from a Phase 3 study due before the end of June.

From the FDA, expect the announcement of approval decisions for Biogen's ALS drug tofersen on Tuesday, followed on Wednesday by a decision on a treatment for recurrent c. difficile infection from Seres Therapeutics.

Also, this week we'll have the meeting of the Association for Research in Vision and Ophthalmology, or ARVO — which will be of import for Regeneron and Novartis, among others.

weight loss wars

Wegovy and Mounjaro will go head to head

The weight loss drug face-off between Eli Lilly and Novo Nordisk is heating up: Lilly quietly registered a new Phase 3b trial called SURMOUNT-5, FiercePharma writes, pitting the company's Mounjaro against Novo's Wegovy. The study, which will enroll 700 participants across 61 sites, will launch this week and last through February 2025. It's meant for patients with obesity or who are overweight with weight-related health conditions — including sleep apnea, hypertension, or cardiovascular disease.

It's still unclear whether it's a superiority study or a measure to test noninferiority. Wegovy is a GLP-1 agonist drug, which in one trial showed weight loss of 12.4% in patients without diabetes. Mounjaro is a dual GLP-1/GIP agonist which, in non-diabetic patients, showed weight reduction up to 21%.

Alzheimer's

The fallout from an increase in Alzheimer's testing

Testing for the APOE4 gene variant is perhaps the simplest way to calculate a person's likelihood of developing Alzheimer's disease. As new treatments arise that might slow disease progression, the number of people getting tested to see if they carry the risky variant has more than doubled, Reuters writes. But there aren't many resources available to help people deal with knowing they carry two copies of APOE4 — with the Alzheimer's community facing a shortage of genetic counselors to help navigate knowledge of a predisposition to the disease.

"People describe feeling existential dread," one bioethicist told Reuters.

Leqembi, the recently approved Alzheimer's drug that now costs $26,500 a year, is not an option for these people at higher risk for the disease but yet don't show any symptoms. But in the four months before the U.S. approval of Leqembi this January, testing for APOE4 among people over 55 increased 125%, Reuters writes. The NIH believes up to 25% of people in the U.S. have one copy of APOE4, and up to 5% have two copies. But a 2018 study showed that the U.S. had just one trained genetic counselor per 82,000 people. "We needed a scalable way to warn people about the potential benefits and risks of genetic disclosure," one Alzheimer's expert says.

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