Sarepta Therapeutics communicated on December 7, 2020 the preliminary results of an international phase II trial (not in France) (MOMENTUM trial) which evaluates SRP-5051 in Duchenne muscular dystrophy. SRP-5051 is a new generation antisense oligonucleotide combining a peptide with the antisense oligonucleotide PPMO (Peptide phosporodiamidate morpholino oligomer). He is targeting exon 51 skipping in DMD.
A lower cumulative dose for a little more efficiency
In this trial, five doses of SRP-5051 (4 mg / kg / month to 40 mg / kg / month) were evaluated in 15 patients with DMD aged 7 to 21 years, ambulatory or not. Each patient received one of the 5 doses intravenously for a minimum of 3 months. In comparison, the cumulative doses of SRP-5051 are much lower than those usually administered for older oligonucleotides (one injection per week) such as eteplirsen from the same laboratory and also targeting exon 51 skipping (Exondys 51® authorized in the USA).
Analysis of data from 4 participants treated with 20 mg / kg / month SRP-5051 for 3 months shows:
- good penetration of SRP-5051 into muscle tissue;
- increased exon skipping rate and dystrophin production in the muscles;
- no noticeable side effects.
In comparison with the results of the PROMOVI trial in which participants received 30 mg / kg / week of eteplirsen for 6 months, the first results obtained in these 4 patients of the MOMENTUM trial treated with 20 mg / kg / month of SRP-5051 for 3 months (i.e. a cumulative dose 10 times lower) show:
- 1.6 times higher exon jump rate;
- a percentage of normal dystrophin increased by 5 times in 2 of the participants.
Results to be confirmed on more patients and over the long term with this MOMENTUM trial, which is still ongoing.
Access Sarepta Therapeutic press release, December 7, 2020 « Sarepta Therapeutics Announces Positive Clinical Results from MOMENTUM, a Phase 2 Clinical Trial of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51″
MOMENTUM clinicaltrials.gov
