Regeneron, Eli Lilly, Pfizer updates

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Good morning. Today we’re wondering: How much will the advisory committee meeting for Lilly’s donanemab matter? Send me your thoughts and, as always, any ideas or tips: [email protected].

Young boy dies in trial against Pfizer Duchenne

A young boy died during a trial for Pfizer’s experimental gene therapy for Duchenne muscular dystrophy, the company told patient advocates yesterday.

Pfizer said the boy, who was enrolled in a trial for boys aged 2 or 3, received the therapy last year and died of cardiac arrest, but that researchers had not yet determined exactly what happened or whether the death was related with the treatment.

The company said it is pausing the transition to patients in a separate Phase 3 trial of the treatment in older boys — meaning it will stop for now giving patients who initially received a placebo in the study.

This is the second time Pfizer has announced a death in its Duchenne gene therapy trials, after a 16-year-old boy died in late 2021. No deaths have been reported for Sarepta’s gene therapy, Elevidys, although several patients were hospitalized and safety was fully assured. data is not yet available. Elevidys received accelerated approval last year and is now being reviewed for broader approval.

Read more from STAT’s Jason Mast.

Advisory panel date set for Lilly’s donanemab – how much will it matter?

The FDA has set June 10 as the date for an advisory committee meeting on Donanemab, Eli Lilly’s Alzheimer’s candidate.

In March, news that the agency will convene advisers to discuss the drug came as a surprise. Donanemab, which aims to clear plaques of amyloid protein in the brain, has succeeded in its phase 3 trial, showing a 35% slowdown in Alzheimer’s disease progression compared to placebo. Observers had expected the treatment to receive regulatory approval earlier this year.

However, there were safety concerns. In the trial, 37% of participants treated with the drug experienced side effects related to ARIA, which causes swelling and bleeding in the brain, and three patients died. The FDA also has questions about the dosing schedule used in the trial. Lilly tested a unique, “non-chronic” strategy in which donanemab was given in monthly infusions, but only until patients’ amyloid levels fell below a certain threshold.

How much will this meeting affect Lilly? In another world where Mounjaro and Zepbound didn’t exist, it would be a big deal. But investors now seem to be focusing more on obesity than Alzheimer’s disease. For perspective, Biogen and Eisai’s new Alzheimer’s drug Leqembi, which received full approval last July, brought in $19 million in sales in the first quarter, while Lilly’s Zepbound, which was approved last November and saw a shortfall, brought in $517 million. This isn’t the fairest comparison, as the introduction of Alzheimer’s drugs will be slower given the need for infusion centers, but it still points to the enormous market opportunity for anti-obesity drugs.

Pharma has more than $500 billion in deal capital

The sector has seen a fair number of mergers and acquisitions so far this year, and more deals are likely to be completed for the remainder of 2024, a team of Jefferies analysts led by Michael Yee wrote in a note yesterday.

So far this year, there have been thirteen mergers and acquisitions with an average deal size of $2.6 billion in the pharmaceutical industry. This mainly concerns ready-made deals at an early stage, the analysts said.

They estimate that companies have a total of more than $500 billion in capacity for more deals, and these will likely be in hot areas such as cardiometabolic diseases and antibody-drug conjugates. Companies like Novo Nordisk, Eli Lilly and Merck remain open to early-stage deals, they said, and there is pressure on the industry for mergers and acquisitions given the impact of Medicare’s new drug negotiation program in the Inflation Reduction Act.

Regeneron gene therapy helps two deaf children hear

An experimental gene therapy from Regeneron helped two congenitally deaf children hear, according to data from an ongoing Phase 1/2 trial presented today at the ASGCT conference.

This trial of the therapy, called DB-OTO, aims to enroll nearly 20 children who have severe hearing loss due to changes in the otoferlin gene. The new results show that one child had only mild hearing loss after 24 weeks, and the other child saw small improvements in hearing after six weeks.

Several other companies are also studying therapies in this area, including: Akouosa subsidiary of Eli Lilly, and based in China Otovia Therapeutica.

Read more from STAT’s Timmy Broderick.

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