Sarepta Therapeutics Inc（SRPT）资讯与事件SRPTSarepta Therapeutics Inc

Sarepta Therapeutics Presents Preclinical Data on SRP-1003

Sarepta Therapeutics presented new preclinical data at the 22nd Annual Meeting of the Oligonucleotide Therapeutics Society, OTS, demonstrating that SRP-1003, the company's investigational small interfering RNA program for the treatment of myotonic dystrophy type 1, can directly reduce pathogenic DMPK transcripts within the nucleus, where the molecular drivers of DM1 pathology originate. In the analysis, correction of muscleblind-like-dependent mis-splicing provide functional evidence of nuclear activity. Direct measurement of the nuclear and cytoplasmic transcripts pools further demonstrated the efficiency of that activity, with SRP-1003 producing comparable, dose-dependent DMPK in both compartments and achieving up to approximately 80% knockdown in each. The results were confirmed via three independent and complementary methods: RNA fluorescence in situ hybridization foci quantification, compartment-fractionated ddPCR with purity controls, and in situ RNAscope in intact cells.

Sarepta Presents ELEVIDYS Efficacy Data in Duchenne Muscular Dystrophy Patients

Sarepta Therapeutics presented new data at the 31st Annual Congress of the World Muscle Society) that provide evidence of clinically meaningful functional benefit of ELEVIDYS in older ambulatory patients with Duchenne muscular dystrophy. In ambulatory patients treated at ages 8 to 12, an age range in which functional decline becomes even more pronounced, treatment was associated with durable benefits across multiple measures over two years compared to a well-matched external control. These findings suggest that the benefits previously observed in younger ambulatory patients are also being observed in older ambulatory patients. Sarepta also presented an analysis of patients treated at ages 2 and 3 that demonstrated robust delandistrogene moxeparvovec-dystrophin expression and sarcolemmal localization 12 weeks after infusion. Together, the analyses expand the evidence base across distinct ages and stages of ambulant Duchenne patients, while continued follow-up will be important to understand longer-term outcomes. Details from the delandistrogene moxeparvovec analyses: 1.103LBP: Functional and safety outcomes in older ambulatory patients with Duchenne muscular dystrophy treated with delandistrogene moxeparvovec: This analysis used a prospectively specified approach to compare treated patients with similar patients from several well-characterized DMD natural history datasets. Patients were matched based on the study eligibility criteria, and the groups were further balanced for key factors that can influence disease progression, with the aims of creating a reliable comparison and addressing common limitations of external control analyses. Patients from the EMBARK and ENDEAVOR studies who were aged 8-12 years at time of dosing, were compared to a well-matched EC group. Treatment with delandistrogene moxeparvovec demonstrated durable and clinically meaningful functional benefits over two years compared to EC. Clinically meaningful efficacy was observed across NSAA total score, RFF velocity and 10MWR velocity at 1 and 2 years post-infusion, with nominally statistically significant differences between delandistrogene moxeparvovec-treated and EC patients at Years 1 and 2. Results show a sustained treatment effect and slowing of disease progression and support the use of delandistrogene moxeparvovec in ambulatory patients with DMD aged 8-12 years. Safety in this patient population was manageable and consistent with the known safety profile of delandistrogene moxeparvovec to date, and the most common treatment related adverse events were nausea and vomiting. 2.080P: Delandistrogene moxeparvovec micro-dystrophin expression and safety in 2-3-year-olds with Duchenne muscular dystrophy in ENDEAVOR and ENVOL studies: In this analysis, ambulatory patients with Duchenne aged greater than or equal to2 to less than3 years from the ENDEAVOR and ENVOL clinical studies, results demonstrated robust delandistrogene moxeparvovec-dystrophin expression and sarcolemmal localization 12 weeks after infusion with delandistrogene moxeparvovec. In this patient population, higher overall mean and higher minimum delandistrogene moxeparvovec micro-dystrophin expression was observed compared with previous results in older smbulatory and non-ambulatory patients. Investigators continue to follow up with patients in order to assess functional outcomes over time and, when available, those results will provide important context for interpreting the clinical benefit in younger patients. The safety of delandistrogene moxeparvovec in these patients was manageable and consistent with the safety profile to date in ambulatory patients aged 4 years and older.

Sarepta Reports Q2 Revenue of $401.3M

Reports Q2 revenue $401.3M, consensus $364.9M. "As I begin my tenure as CEO, I am excited by the strength of Sarepta's foundation, the impact our therapies are having for patients, and the significant opportunities ahead," said Michael Severino, MD, chief executive officer, Sarepta Therapeutics. "Our second quarter results, including $328.7 million in total net product revenue and both GAAP and non-GAAP operating profitability, reflect the strength and resilience of our business. With important data readouts expected in DM1 and FSHD, continued progress across our broader pipeline, and a talented team dedicated to transforming the lives of patients with rare diseases, we have significant opportunities ahead and remain committed to delivering sustainable long-term value. Our priorities are clear: execute our commercial strategy, advance our promising siRNA pipeline, and continue allocating capital with discipline. With a strong balance sheet, an innovative pipeline, and an experienced leadership team, I believe Sarepta is well positioned to deliver on its long-term potential."