Sarepta Therapeutics Inc Stock Price, News & Analysis

Sarepta Therapeutics (NASDAQ: SRPT) announced the completion of a rolling New Drug Application (NDA) to the FDA for casimersen, aimed at treating patients with Duchenne muscular dystrophy (DMD) who have mutations amenable to skipping exon 45. Casimersen represents the third exon-skipping medicine from Sarepta's PMO RNA-based platform. The NDA submission includes data from the ESSENCE Phase 3 study demonstrating significant dystrophin production. If approved, casimersen could potentially treat about 8% of DMD patients, contributing to Sarepta's goal of addressing nearly 30% of this patient population.

Sarepta Therapeutics (NASDAQ: SRPT) has entered a global research and option agreement with Codiak BioSciences to develop engineered exosome therapeutics targeting neuromuscular diseases. The two-year agreement covers five targets, with Codiak eligible for up to $72.5 million in upfront and near-term license payments plus research funding. This collaboration aims to leverage Codiak's exosome technology for gene therapy and RNA delivery, addressing limitations of existing treatments and enhancing precision medicine strategies in this sector.

Sarepta Therapeutics (NASDAQ: SRPT) announced positive safety and tolerability data for its gene therapy SRP-9001, aimed at treating Duchenne muscular dystrophy (DMD), published in JAMA Neurology. Over one year, four participants showed no serious adverse events, with micro-dystrophin levels reaching 81.2% expression. Functional improvements were observed, as evidenced by reduced creatine kinase levels and enhanced scores on the North Star Ambulatory Assessment scale. This underscores the therapy’s potential to improve the lives of DMD patients significantly.

Sarepta Therapeutics (NASDAQ: SRPT) announced promising results from its clinical trial of SRP-9003, a gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). The high-dose cohort demonstrated a significant 72% beta-sarcoglycan positive fibers and an 89% mean reduction in serum creatine kinase, indicating robust muscle expression and reduced damage. Patients in the low-dose cohort showed continued functional improvement at one year. The therapy aims to deliver the beta-sarcoglycan gene, targeting a critical need for patients who currently have no treatment options.

Sarepta Therapeutics (NASDAQ:SRPT) announced its participation in two virtual investor conferences. The Goldman Sachs 41st Annual Global Healthcare Conference will take place on June 9, 2020, at 3:00 p.m. E.T., followed by the BofA Securities Napa Biopharma Conference on June 24, 2020, at 12:30 p.m. E.T. Live webcasts will be available on Sarepta's website and archived for 90 days. Sarepta is a leader in precision genetic medicine, focusing on treatments for rare diseases, including Duchenne muscular dystrophy, with over 40 programs in development.

Sarepta Therapeutics (NASDAQ: SRPT) announced the granting of equity awards to 8 new employees on May 29, 2020, in accordance with its 2014 Employment Commencement Incentive Plan. The awards included options to purchase 8,015 shares of common stock and 4,015 restricted stock units (RSUs). The options have an exercise price of $152.27 per share, matching the closing price on the grant date. Vesting schedules for both options and RSUs are set to complete over four years, contingent on continued employment.