
Another Duchenne clinical hold gets lifted, this time at Dyne Therapeutics
Clinical holds have hampered a host of trials testing treatments for Duchenne muscular dystrophy, but one biotech is getting the go-ahead to start its study five and a half months after the initial roadblock.
The FDA cleared Dyne Therapeutics’ first clinical trial in boys with a certain type of Duchenne muscular dystrophy, the Massachusetts biotech said Tuesday morning, sending shares $DYN up nearly 10% before the opening bell. The regulator had requested more clinical and non-clinical information back in January, but the biotech has been mum on details of what exactly the agency wanted to look at before signing off on the Phase I/II trial.
In the months since the hold, Dyne has repeatedly said it expects to start the study in mid-2022, and the company reiterated that timeline Tuesday with the hold’s lift. The second half of the year began mere days ago.
Dyne’s candidate, dubbed DYNE-251, will be tested in 30 to 50 boys aged 4 to 16 with Duchenne who have mutations amenable to exon 51 skipping therapy. Sarepta Therapeutics markets a drug, Exondys 51, for the specific exon, among other treatments available for the progressive muscle weakness disorder.
The lift marks a key moment for Dyne as it nears the clinic with DYNE-251 and myotonic dystrophy type 1 asset DYNE-101, both slated for mid-2022 trial starts. Once DYNE-251 enters human studies, Dyne hopes to progress additional work in other DMD exons, including 53, 45 and 44, as part of its broader ambitions to compete against other DMD treatment makers.
“Today marks a significant step in our journey to build a DMD franchise to serve people across the globe with Duchenne mutations amenable to exon skipping. The clearance of our first IND is an important achievement for Dyne, and we appreciate the partnership with the FDA throughout this process,” Dyne president and CEO Joshua Brumm said in a statement.
While there are a few treatments on the market for DMD from the likes of Sarepta, PTC Therapeutics and an attempt from now-defunct Marathon Pharmaceuticals at turning a cheap overseas steroid into a steep sticker price drug, the DMD community has run into many hurdles over the years in getting additional approved therapies.
Pfizer, in its attempt to catch up to Sarepta, was able to resume its Phase III gene therapy trial in late April and, at the time, was on track to submit for FDA approval in late 2023. But other biopharmas have run into holds and elongated paths to approval in the months since.
Just last week, Santhera Pharmaceuticals said its attempt at getting a green light for its DMD treatment has been pushed back four to six months. Two weeks ago, Sarepta’s next-gen Duchenne asset got hit by a clinical hold in Part B of a Phase II study. Also last month, PTC’s years-long shot at getting the FDA to approve its European-nodded DMD drug Translarna hit another setback with mixed data from a confirmatory study.
One biotech, though, has provided some positive news to the field in recent weeks, with Capricor Therapeutics saying its DMD drug notched the primary endpoint in an open-label extension to its Phase II study.