Dyne Therapeutics recently shared that the FDA issued a clinical hold on their Investigational New Drug (IND) application for the Phase I/II study of their exon skipping therapy utilizing a conjugated monoclonal antibody DYNE-251.
For our community, this news may be confusing. Our experience has been that clinical holds, when they occur, are in reference to a clinical trial with active dosing of individuals with Duchenne or Becker.
DYNE-251, however, is not yet in the clinic. Dyne has been involved in discussions with the community about their next generation PMO, held focus groups and met with the community to discuss their plans around clinical trials, but has not yet provided their investigational product to any individuals.
What Does an IND Clinical Hold Mean?
FDA defines a clinical hold as an order issued by FDA to the sponsor of an IND application to delay a proposed clinical investigation or to suspend an ongoing investigation. All or some of the investigations conducted under an IND application may be placed on clinical hold.
The grounds for a clinical hold prior to a clinical trial starting cover a range of issues; the bottom line is that FDA is seeking additional information and has additional questions that need to be addressed about the program prior to allowing the trial to begin. As an example, the request may sometimes require additional animal data or toxicity data relevant to the investigational product.
What Does This Mean for Those Who Are Interested in DYNE-251?
Dyne has indicated that they are committed to resolving the clinical hold as quickly as possible, and can respond to the FDA with data they have from existing and ongoing studies in the second quarter of 2022. Dyne has also shared that they are still prepared to begin dosing individuals with Duchenne by mid-2022 if the FDA is satisfied with their response from the data being collected.