Sarepta Therapeutics (SRPT) has published the results of the second part of the SRP-9001-102 study, which assesses the safety, efficacy and tolerability of patients with a single dose of SRP-9001 gene therapy (delandistrogene moxeparvovec). The study involved 41 patients with Duchenne muscular dystrophy, 21 of whom received a placebo.
The drug has demonstrated statistically significant efficacy. Participants who received SRP-9001 (n=20, age 5-8 years) scored 2.0 points more according to the average North Star Outpatient Assessment 48 weeks after administration of the drug compared to the control group. Additional results will be presented at one of the upcoming medical congresses. The safety profile of SRP-9001 is also favorable: no serious adverse events associated with treatment, as well as deaths or discontinuation of treatment due to the negative effect of the drug on the patient, were observed during the trials.
Study 102 has not yet been completed, it is ongoing to obtain longer-term data on the efficacy and safety of therapy. According to management, these data increase confidence in the success of the Phase III global clinical trials of EMBARK. It is expected that the recruitment of subjects to the program will be completed in mid-2022, although last fall management predicted that this would happen at the beginning of this year. The forced postponement is the main negative signal for investors, since the path of the potential blockbuster SRP-9001 to the market has been delayed.
Sarepta management also announced other pipeline updates in 2022. In the field of LGMD (Erb muscular dystrophy), the results of SRP-9003-101 trials are expected: a three-year follow-up in a low-dose cohort and a two-year follow-up in a high-dose cohort. PoC trials of the drug SRP-6004 (Dysferlin) will also be launched soon. In addition, in the second half of 2022, it is planned to complete the recruitment of subjects in the MOMENTUM RNA study (part B) of the drug SRP-5051.
In addition, Sarepta presented preliminary quarterly results. The issuer's balance sheet at the end of the year was $2.1 billion. Grocery revenue for the fourth quarter reached $178.7 million, coinciding with market expectations. The company's annual sales amounted to $612 million, which corresponds to the earlier forecast of management at the level of $605-615 million. Apparently, the upcoming report will not surprise investors.