Dyne Therapeutics Inc. (NASDAQ: DYN) has announced that the US FDA has put a clinical hold on its Investigational New Drug application to commence the clinical study of DYNE-251 in Duchenne muscular dystrophy patients amenable to skipping exon 51.
Dyne received clinical hold on January 14, 2022
On Friday, January 14, 2022, Dyne got a clinical pause notice from the US FDA, seeking more clinical and non-clinical material for DYNE-251. In accordance with current guidance, Dyne expects to submit a reply to the FDA in the q2 of 2022, along with data from ongoing and existing studies, and, if adequate to the FDA, to begin dosing subjects in a Phase 1/2 multiple ascending dose (MAD) DYNE-251clicnial study by mid-2022. Dyne plans to work cooperatively with the FDA to get the clinical hold lifted as soon as feasible.
As previously stated, Dyne plans to file an investigational new drug for DYNE-101 to treat myotonic dystrophy type 1 (DM1) in the first quarter of 2022 and to begin dosing patients in a proposed MAD clinical trial by the middle of 2022.
The biotech startup employs FORCE, a technology that breaks antibodies down into oligonucleotides (short DRNA or RDNA molecules) and delivers them to muscle cells, at which oligonucleotide is predicted to connect to the cell’s RNA that promotes disease development and then decay that RNA. In addition to DYNE-251, the company is working on DYNE-101 in the treatment of myotonic dystrophy type 1, a muscular disease resulting from a genetic abnormality, and DYNE-301 in treating FSHD, which is also caused by genetic alterations in chromosomes and impairs the neck, facial, and upper-arm muscles.
DYNE-251 resulted in dystrophin synthesis and exon skipping
DYNE-251 therapy resulted in lasting exon skipping and dystrophin synthesis in skeletal muscles and heart in a DMD mouse model, according to Dyne’s website. In non-human primates, the therapy was well tolerated and exhibited substantial exon skipping, especially in the diaphragm, a dome-shaped muscle critical in breathing and the heart
Dyne also plans to develop exon-skipping therapeutics for DMD individuals with alterations in exons 53, 45, and 43.
