Design Therapeutics announce positive data from Friedreich’s ataxia trial - Ataxia UK

Design Therapeutics announce positive data from Friedreich’s ataxia trial

Post Published: December 16, 2022

Design Therapeutics have announced positive data from the first part of their Phase I clinical trial of DT-216. They found that treatment with DT-216 increased frataxin mRNA. Frataxin mRNA is decreased in people with Friedreich’s ataxia (FA) and leads to the symptoms of the condition. The safety data also showed that the drug was well-tolerated. These results support the continued advancement of DT-216 in the second part of their Phase I trial, as well as their Phase II trial which is expected to start in 2023.

The first part of the trial included 39 adults with FA, who were given a single dose of either DT-216 or a placebo. Five different doses of DT-216 were tested in this trial, ranging from 25 mg to 600 mg. People who were given 100 mg of DT-216 and above had a significant increase in frataxin mRNA at 24 hours after treatment. This was tested in a type of blood cell called peripheral blood mononuclear cells (PBMCs). The highest response had over double the amount of frataxin mRNA compared to before treatment. Safety assessments were also completed for 30 days after treatment. These showed that the treatment was generally well-tolerated throughout.

The second part of the Phase I trial has now started and is ongoing. In this, DT-216 will be given in three weekly doses (rather than a single dose). Design Therapeutics expect to report data from this part of the trial in mid-2023. They also anticipate starting a Phase II trial during 2023.

DT-216 is a small-molecule therapy, called a gene targeted chimera (GeneTAC™). It is designed to target the genetic mutation found in people with FA and restore the production of frataxin mRNA. Design Therapeutics presented their pre-clinical data on DT-216 in cell and animal models at the International Congress for Ataxia Research (ICAR) 2022. You can read more about this here. Design Therapeutics previously announced that DT-216 has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA). This designation facilitates accelerated development and review of new drugs.

For further information, read the press release on the Phase I trial here.


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