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

• Results continue to support evidence for safety, tolerability, clinical efficacy, and biomarker improvements of DT-DEC01
• At 6 months treated patients achieved mean significant increase in motor unit potential (MUP) levels of up to 200% of baseline, as measured by electromyography
• Functional outcomes in first (low dose) cohort demonstrated continued improvements in PUL and PODCI scores in non-ambulatory patient and 6MWT, NSAA and PODCI scores in ambulatory patients 6 months after DT-DEC01 administration.
• Therapy demonstrated benefit in all patients independent of genetic mutation, thus making DT-DEC01 a universal therapy for all DMD patients

MIAMI and WARSAW, Poland, Sept. 26, 2022 /PRNewswire/ -- 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.

About DT-DEC01

DT-DEC01 is a chimeric cell therapy and constitutes an advanced therapeutic medicinal product (ATMP). DT-DEC01, which is made using Dystrogen's proprietary cell engineering technology, expresses favorable HLA characteristics which carry multiple immunological advantages. In preclinical studies, DT-DEC01 has been shown to express clinically significant levels of dystrophin when compared to controls. DT-DEC01 demonstrated significant functional improvement in cardiac, diaphragm, and other skeletal muscle strength and associated function in preclinical trials. Because DT-DEC01 is designed to prevent triggering an immune system response, a major advantage is that it does not require immunosuppression. The therapy is not associated with any genetic manipulation and therefore involves no risk of off target mutation, does not use viral vectors, and its use is not dependent on the genetic mutation of the DMD patient, thus making DT-DEC01 a universal therapy for all DMD patients.

About Dystrogen Therapeutics Corp

Dystrogen Therapeutics is a clinical-stage life sciences company committed to developing therapies for rare genetic diseases and disorders associated with aging. Dystrogen has developed a patented cell engineering technology platform, which has been shown to improve function in both clinical and pre-clinical trials. 

SOURCE Dystrogen Therapeutics Corporation