April 17, 2023

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Biotech Correspondent

Hey there! AACR is underway, and some interesting news is emerging — particularly cancer vaccine data from Moderna and Merck. We have a special report on Sarepta's gene therapy for Duchenne muscular dystrophy, and we see Merck acquire an immunotherapy player for $10.8 billion.

gene therapy
What a gene therapy approval would mean for the Duchenne community, and Sarepta

Just days after receiving an experimental gene therapy for his Duchenne muscular dystrophy, 4-year-old Emerson could do what he never could before: climb up the stairs, placing one foot after another. He quickly learned to ride a tricycle, and was playing T-ball. Emerson's family wants every child with this debilitating genetic disease to have that experience. And soon, that could be a reality.

An FDA decision on whether Sarepta Therapeutics' experimental gene therapy for Duchenne, SRP-9001, is expected by May 29. It is the culmination of four decades of work by patient advocates, research groups, and rival companies racing on two continents to find an effective treatment for this most common of rare diseases. About 1 in 3,500 children — mostly boys — are born with it. STAT's Adam Feuerstein and Jason Mast dive deep into the story of Sarepta's gene therapy, what it means for the Duchenne community.

cancer vaccines

Validation for the cancer vaccine approach

For the first time, we have evidence that personalized cancer vaccines can offer real clinical benefit to patients. A neoantigen cancer vaccine developed by Merck and Moderna dramatically reduced patient risk of relapse when combined with Merck's blockbuster immunotherapy pembrolizumab, or Keytruda, new data presented at AACR this week show.

The study sorted 157 patients with stage 3 or 4 melanoma to receive either pembrolizumab alone, or pembrolizumab in combination with the experimental vaccine. After 18 months, 78.6% of those who got the combination treatment had no recurrence of the cancer, compared to 62.2% of patients who got the pembrolizumab alone.

The results make clear that Moderna has a viable path forward, now that demand for Covid-19 vaccines is declining. And they underscore how mRNA technology can be powerful for a variety of diseases — especially cancer.

stat reports
Understanding clinical trial delays

Delays happen all the time in clinical trials — but companies don't tend to explain in depth why. Sometimes it's just a temporary blip, explains R&D consultant Frank David, but extending trial timelines unexpectedly is nerve-wracking and often reduces the value of a program or a company. Although they tend to give competitors a leg up, delays aren't an indication that a therapy — or even a company — is going to fail.

"That said, as the cumulative delay increases, that could be a sign that a trial might flame out before completion for operational reasons, independent of whether or not the drug works," David says in a new Q&A with STAT. He's written a series of reports for STAT on how to evaluate and interpret clinical trials. The first is publishing today.

Find it here.

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