Sarepta marches forward with its potential DMD gene therapy and an expanded Catalent manufacturing deal

Sarepta spent much of last year marching forward with its candidate SRP-9001, a gene therapy treatment for the rare but fatal Duchenne muscular dystrophy, eventually filing a BLA for an accelerated approval, with a May 29 PDUFA date and a likely adcomm on the way too.

The Cambridge, MA-based biotech on Thursday signed a commercial supply agreement with the New Jersey-based CDMO Catalent to manufacture SRP-9001 and to support other gene therapy candidates in Sarepta’s pipeline for another rare, genetic disease related to muscle deterioration, known as limb-girdle muscular dystrophy (LGMD). Financial details of the deal were not disclosed and Endpoints News did not hear back from Catalent by press time.

“Our partnership with the Sarepta team spans nearly a decade across multiple programs and modalities, and we look forward to working together to manufacture these potentially life-changing and life-saving products for patients diagnosed with DMD and LGMD,” said Alessandro Maselli, Catalent’s CEO in a statement.

Catalent maintains several gene therapy manufacturing sites, including two in Maryland, outside of Baltimore, and one in Belgium.

Sarepta originally settled on a long-term manufacturing agreement with Paragon Bioservices in 2018, but a year later, Catalent acquired Paragon for $1.2 Billion.

Alessandro Maselli

In 2019, Sarepta inked a $1.15 billion deal with Roche for the rights to the therapy outside the United States, while Sarepta held on to the US rights.

In early 2021, SRP-9001 failed in an initial Phase II study, which Sarepta attributed to differences in baseline measures between the older children of the placebo and treatment groups. That fail cut the biotech’s stock in half.

However, in the second part of that Phase II study, 20 patients who had received the placebo treatment crossed over to get Sarepta’s gene therapy, the patients showed statistically significant improvements in the motor abilities test that was also used in Part 1.

manufacturing
gene therapy
biotech