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Sarepta Therapeutics, Inc. (NASDAQ: SRPT) is a pioneering biopharmaceutical company primarily engaged in the discovery and advancement of RNA-targeted therapeutics. Headquartered in Cambridge, Massachusetts, Sarepta is dedicated to developing innovative treatments for a range of rare, infectious, and other challenging diseases.
Core Focus: Sarepta’s primary focus is on Duchenne Muscular Dystrophy (DMD), a severe type of muscular dystrophy. The company's lead product candidate, eteplirsen, is designed to skip exon 51 and potentially modify the progression of DMD. Alongside eteplirsen, Sarepta is working on other DMD drug candidates that target different exons.
Innovative Technology: The company leverages proprietary RNA-targeted technology platforms to create novel pharmaceuticals. This technology is pivotal in addressing diseases with high unmet medical needs.
Therapeutic Pipeline:
- DMD Program: Focused on rapidly advancing exon-skipping drug candidates.
- Infectious Diseases: Developing treatments for drug-resistant bacteria and other rare infectious diseases.
Financial Condition and Partnerships: Sarepta collaborates with third-party contractors for the manufacturing of its product candidates. This strategic approach allows the company to focus on innovation and development while ensuring efficient production processes. Despite most of its product candidates being in early developmental stages, Sarepta continues to secure funding and partnerships to support its ambitious research and development goals.
For more information, visit their official website at www.sarepta.com.
Sarepta Therapeutics (NASDAQ:SRPT) announced positive results from the Phase 2 MOMENTUM study of its next-generation treatment, SRP-5051, for Duchenne muscular dystrophy. The 30 mg/kg dose revealed a mean exon skipping of 10.79% and dystrophin expression of 6.55% after 12 weeks, significantly outperforming the eteplirsen comparison group. The company anticipates achieving over 10% dystrophin levels with ongoing monthly dosing. Despite manageable hypomagnesemia in some patients, Sarepta is optimistic about advancing toward regulatory discussions for accelerated approval.
Sarepta Therapeutics (NASDAQ:SRPT) announced the granting of equity awards to four new employees on April 30, 2021, under its 2014 Employment Commencement Incentive Plan. The total awards included options for 8,615 shares and 3,275 restricted stock units (RSUs). The options have an exercise price of $70.84 per share, matching the closing stock price on the grant date. Vesting for options and RSUs is set over four years, contingent on continued employment.
Sarepta Therapeutics (NASDAQ:SRPT) will host a webcast and conference call on May 3, 2021, at 8:30 am ET to present results from the 30 mg/kg arm of the MOMENTUM study. This clinical trial evaluates SRP-5051, the company's investigational treatment for Duchenne muscular dystrophy (DMD). SRP-5051 utilizes Sarepta's innovative PPMO platform to enhance drug delivery to muscle tissue. The live presentation will be accessible via Sarepta's investor relations website, with archived slides available for one year.
Sarepta Therapeutics (NASDAQ:SRPT) will announce its first quarter 2021 financial results after market close on May 5, 2021. A conference call to discuss these results and provide a corporate update will follow at 4:30 p.m. E.T. Interested parties can access the call via domestic and international numbers provided in the release, with a live webcast available on Sarepta's website. Sarepta focuses on precision genetic medicine for rare diseases, notably Duchenne muscular dystrophy, and has over 40 development programs in its pipeline.
Sarepta Therapeutics (Nasdaq:SRPT) received notice of an unsolicited mini-tender offer from TRC Capital to purchase up to 2 million shares at $68.50 each, about 4.6% below the stock's closing price on April 16, 2021. Sarepta does not endorse this offer and advises shareholders against tendering their shares, citing the below-market price and lack of affiliation with TRC Capital. The offer represents 2.52% of Sarepta's outstanding shares and is set to expire on May 18, 2021. Sarepta emphasizes caution and recommends consulting with financial advisors.
Sarepta Therapeutics (NASDAQ:SRPT) has launched the Route 79, The Duchenne Scholarship Program, now accepting applications for the 2021-2022 academic year. Up to 15 scholarships, each worth $5,000, will be awarded to individuals diagnosed with Duchenne muscular dystrophy, assessed by an independent committee based on various criteria. Over 50 scholarships have been granted in previous years, showcasing the resilience and ambition of the recipients. Applications are open until May 11, 2021, with recipients notified by August.
Sarepta Therapeutics (SRPT) reported promising two-year data from its ongoing study of SRP-9003, a gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). Key findings include a mean beta-sarcoglycan protein expression of 54% at 24 months in the low-dose cohort, up from 36% at Day 60, and a sustained mean NSAD score improvement of 5.7 points from baseline. Results indicate continued safety and tolerability. Both cohorts showed significant functional improvements, suggesting SRP-9003’s potential as an effective treatment for this severe genetic disorder.
Sarepta Therapeutics (NASDAQ:SRPT) presented ten abstracts, including four podium presentations, at the MDA Annual Clinical and Scientific Conference from March 15-18, 2021. The research focuses on advancing genetic medicine for rare neuromuscular diseases like Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). Key highlights include new data from gene therapies SRP-9001 and SRP-9003, showing promising results in clinical trials. Presentations are available on-demand, emphasizing the company's commitment to improving treatment outcomes for patients with rare diseases.
Sarepta Therapeutics reported strong financial results for Q4 and full-year 2020, with net product sales reaching $122.6 million and $455.9 million, respectively. This marks a 23% increase in Q4 sales compared to 2019 and a nearly 20% increase for the year. A key highlight is the FDA approval of AMONDYS 45 for Duchenne muscular dystrophy, enhancing treatment options for 8% of patients with specific mutations. The firm is advancing its pipeline with significant developments in gene therapy and RNA platforms, aiming to address over 80% of Duchenne mutations.
Sarepta Therapeutics (NASDAQ:SRPT) announced the grant of equity awards to two new employees on February 26, 2021, as per the Compensation Committee's approval. The awards include options for 1,820 shares of common stock at an exercise price of $87.06, equal to the closing price on the grant date, and 680 restricted stock units. Vesting is set to occur over four years, contingent upon continued employment. Sarepta focuses on precision genetic medicine for rare diseases, particularly in Duchenne muscular dystrophy and gene therapies.
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