Moderna said Wednesday afternoon that its experimental vaccine for cytomegalovirus, a cause of disability in newborns, failed in a Phase 3 trial, a significant setback for a company already facing pressure from Wall Street and the federal government.

The CMV vaccine had been the company’s lead program prior to the Covid-19 pandemic. Leadership had repeatedly said it could bring in between $2 billion and $5 billion in peak annual sales. Analysts polled by Visible Alpha forecast peak sales of $1.6 billion for the product.

“It’s obviously disappointing,” said Stephen Hoge, Moderna’s president, in an interview.

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An experimental gene therapy from Sarepta Therapeutics increased levels of the gene missing in an ultra-rare form of muscular dystrophy, according to data the company presented Friday.

The company has said it plans to file for approval in the disease, known as limb-girdle muscular dystrophy (LGMD) 2E. That would make it the first approved treatment in LGMD, a broad collection of highly rare diseases that can deprive patients of the ability to walk and in some cases shorten life. But it is likely to face a significant uphill battle. 

The LGMD 2E therapy relies on the same gene-ferrying virus that Sarepta uses in its other treatments, including its approved gene therapy for Duchenne muscular dystrophy, Elevidys, and experimental gene therapies for several other LGMD subtypes. 

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