Yes, the mention of further testing of delivery of a CPP+PMO into a muscle cell in an animal model of DMD is interesting. It doesn’t specify which type of muscle cell is being targeted, but perhaps heart?
In their J P Morgan Presentation last month, Sarepta says that their peptide conjugated PMO (PPMO) increases cell penetration and is able to deliver PMOs to unique muscle types, such as the heart. Naked PMOs show negligible effect in cardiac muscle, apparently because they are more likely to be trapped in endosomes in cardiomyocytes than in skeletal muscle. The ability to impact heart muscle is especially relevant for patients with DMD, who suffer from cardiomyopathy.
PYC highlighted Sarepta’s preclinical results for SRP-5051 in cardiac muscle in the November 2018 AGM Presentation (relevant slide reproduced below). An 80mg/kg dose was the minimum for any measurable effect. It was stated in the AGM slide that Sarepta’s PPMO was in Phase 1 trials and multiple follow on PPMO programs were scheduled for IND in 2019.
I followed this up. It appears to me that Sarepta didn’t progress multiple follow on PPMO programs to IND last year as previously intended. For the company’s lead PPMO, SRP-5051, Sarepta commenced a 15 patient Phase 1 trial (to evaluate safety, tolerability and PK of a single dose) in early 2018, which was completed in August last year. The company says that it doesn’t expect to disclose safety and dosing insights from this trial until mid- this year.
So, by mid-year, we might know how PYC’s CPP+PMO has performed relative to SRP-5051 in a pre-clinical animal model and we might also get some insight into the dosing being used by Sarepta in its clinical trial. Meanwhile, a Phase 2 trial of SRP-5051 was already commenced in June last year and is due to complete in August next year.
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