More Good News for LGMD Patients

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Sarepta Therapeutics Inc. (NASDAQ: SRPT) shares jumped on Friday after the firm announced an update from its Limb-girdle muscular dystrophy Type 2E (LGMD2E) trial.

Specifically, Sarepta announced the nine-month functional results from three Limb-girdle muscular dystrophy Type 2E (LGMD2E) clinical trial participants who received SRP-9003.

In cohort 1 of the study, three participants ages 4 to 13 were treated with an infusion of SRP-9003. Improvements in functional outcomes were observed at day 270 for all three participants.

At this time, mean creatine kinase was significantly reduced compared to baseline. Creatine kinase is an enzyme biomarker strongly associated with muscle damage.

All three participants showed improvements from baseline across all functional measures, including the North Star Assessment for Dysferlinopathy, time to rise, four-stair climb, 100-meter walk test and 10-meter walk test. These results are distinctly different from what an age-matched, natural history group would predict.

Doug Ingram, Sarepta’s president and CEO, commented:

We have now observed consistent functional improvements, in addition to high levels of expression of the missing protein of interest and strong results in related biomarkers, in both of our first cohorts for Duchenne muscular dystrophy (SRP-9001) and LGMD2E (SRP-9003). We intend to test one higher dose of SRP-9003 in LGMD2E participants, select our clinical dose and then advance our SRP-9003 program, along with our other five LGMD programs, as rapidly as possible. With the results of our first LGMD2E cohort, Sarepta continues to build its gene therapy engine, an enduring model created to design, develop and bring to the medical and patient community transformative therapies for those living with, and too often dying from, rare genetic disease.

Janney Capital weighed in on what this could mean for Sarepta going forward:

We see the initial LGMD2E functional data that Sarepta reported this morning as highly encouraging. In addition to videos demonstrating evident improvement in motor function in all three patients, the consistent changes from baseline across multiple functional measures, as compared to natural history data, suggest meaningful clinical benefit from gene therapy treatment. We believe an overall strong efficacy and safety profile of MYO-101 is similar to what the microdystrophin gene therapy demonstrated in the first four DMD patients. Sarepta plans to initiate patient dosing in the higher dose cohort and patient screening is already ongoing. Management expects to provide an update on the program in 1H20 and to select the dose for the registrational study based on an optimal combination of tolerability and efficacy. Although the regulatory pathway for LGMD2E will be determined upon discussion with the FDA with data from the ongoing study, management believes there could be potential to expedite the development process and we believe Sarepta should be able to leverage the clinical data as well as experience gained from the micro-dystrophin gene therapy program. We believe this morning’s LGMD2E functional data validates Sarepta’s gene therapy platform and we continue to see the gene therapy pipeline as a major value drive for SRPT shares.

Shares of Sarepta traded up about 6% to $86.41 on Friday, in a 52-week range of $72.05 to $158.80. The consensus price target is $190.76.


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