" class="no-js "lang="en-US"> Dystrogen Therapeutics Investigational Chimeric Cell Therapy DT-DEC01 for Duchenne Muscular Dystrophy Demonstrates Clinically Significant Functional and Biomarker Improvements - Pharmtech Focus
Friday, January 27, 2023

Dystrogen Therapeutics Investigational Chimeric Cell Therapy DT-DEC01 for Duchenne Muscular Dystrophy Demonstrates Clinically Significant Functional and Biomarker Improvements

Dystrogen Therapeutics, Corp. the leader in chimeric cell therapies, today announced positive 6-month results from an ongoing clinical study conducted in Poland under a hospital exemption protocol of DT-DEC01, the Company’s investigational engineered cell therapy for Duchenne muscular dystrophy (DMD). Results include 6-month safety, functional, and biomarker data from three clinical trial participants in the first, low-dose cohort. All patients, independent of their genetic mutation, demonstrated both clinical and biomarker improvements when compared to baseline.  During this period, no adverse events (AEs and SAEs) associated with DEC therapy were observed. DT-DEC01 is in development for the treatment of DMD, a devastating neuromuscular disease associated with a lack of dystrophin protein. DT-DEC01 is an engineered chimeric cell that engrafts in skeletal and cardiac muscle, delivering a full-length dystrophin gene and related components of a healthy muscle cell, the absence of which is closely associated with the progressive degeneration and a shortened lifespan characteristic of the disease. DT-DEC01 requires no immunosuppression and there was no evidence of an immune reaction to the therapy on blood assays at 6 months post treatment, in any of the patients to date.

“There are currently no approved treatments for boys and young men with DMD that result in a cure or significant attenuation of the disease. It is very encouraging that we continue to see consistent positive clinical and biomarker data from our investigational DT-DEC01 engineered cell therapy, even at this low dose, and that the benefit of the therapy is sustained at 6 months,” said Kris Siemionow, M.D. Ph.D., CEO Dystrogen Therapeutics. “The improvements in functional and biomarker measures at 6 months in participants from the low dose cohorts who received DT-DEC01 are distinctly different from what an age-matched, natural history group would predict with DMD. When coupled with strong and sustained dystrophin expression in preclinical studies and encouraging clinical safety profile at 6 months, today’s results increase our confidence in DT-DEC01 and provide additional supportive evidence for this approach.”

Cohort 1 (low dose) at 6 months:

Patient #1. (7-year-old ambulatory with deletion of Exon 3-12) Improved duration and amplitude of motor unit potentials (up to 108% of baseline) on EMG (biomarker) when compared to pretreatment baseline. Improved 6MWD (430m –> 469m) and NSAA (31–>34) total score; improved 10-meter walk/run time (14%) and grip strength (5kg–>6.6kg); improved PODCI (90–>95) and others. Statistically and clinically significant increase in step count via activity tracker.

Patient #2. (15-year-old non-ambulatory with deletion of Exon 48-50).  Improved duration and amplitude of motor unit potentials (up to 287% of baseline) on EMG when compared to pretreatment baseline. Improved grip strength (8kg–>9.7kg); improved PUL (20–>23). Statistically and clinically significant increase in activity via activity tracker.

Patient #3. (6-year-old ambulatory with nonsense mutation). Improved duration and amplitude of motor unit potentials (up to 124% of baseline) on EMG when compared to pretreatment baseline. Improved 6MWD (339m –> 390m) and NSAA (26–>28), improved supine to stand (5%); improved PODCI (89–>93), PUL (32–>39) and others. Statistically and clinically significant increase in step count via activity tracker.

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