According to a recent press release from Dyne Therapeutics, the FDA has placed a clinical hold on their Investigational New Drug (IND) application for DYNE-251. Until this hold is lifted, the muscle disease company cannot begin a clinical trial for the drug.

About the Clinical Hold

The FDA put the clinical hold on an IND for DYNE-251, an investigational treatment for Duchenne muscular dystrophy (DMD). The reasoning behind this hold is that the FDA would like to see more information, both clinical and non-clinical.

In response, Dyne expects to send the necessary data by the second quarter of this year. This information is sourced from ongoing, existing clinical trials. If all goes well and the application is approved, Dyne will initiate a Phase 1/2 multiple-ascending doses (MAD) trial. Hopefully, this will happen around mid-2022. The company will work closely with the FDA to ensure that everything runs as smoothly as possible.

About DMD

Duchenne muscular dystrophy (DMD), is one of nine forms of muscular dystrophy. A mutation passed down in an X-linked recessive pattern stops patients from producing dystrophin, ultimately resulting in progressive muscle weakness and degeneration. Other symptoms include difficulty with motor skills, fatigue, learning disabilities, frequent falls, issues with moving positions, and difficulty walking. Complications may include heart disease and respiratory failure, making treatment integral. The main form of treatment is steroids, but there is ongoing research looking into new options for DMD. Other current options include amino acids, asthma treatment, physical and occupational therapy, heart medications, assisted breathing, carnitine, creatine, and other supplements.