Latest news with #Sarepta
Business Insider
3 days ago
- Business
- Business Insider
Sarepta upgraded to Outperform from Sector Perform at Scotiabank
Scotiabank analyst Louise Chen upgraded Sarepta (SRPT) to Outperform from Sector Perform with an $80 price target Confident Investing Starts Here: Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>>
Business Wire
4 days ago
- Business
- Business Wire
U.S. FDA Grants Platform Technology Designation to the Viral Vector Used in SRP-9003, Sarepta's Investigational Gene Therapy for the Treatment of Limb Girdle Muscular Dystrophy Type 2E/R4
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the rAAVrh74 viral vector used in the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy type 2E/R4, has been granted platform technology designation by the U.S. Food & Drug Administration. 'This is one of the first programs to receive platform technology designation and an important recognition by FDA of the reproducibility and adaptability of this technology across multiple therapeutic programs,' said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research & development, Sarepta. 'The designation underscores and reinforces the consistency of the data we have seen with this AAVrh74 in multiple clinical programs and is yet another example of Sarepta's continued commitment to accelerating the development of potentially transformative treatments for patients with rare genetic diseases like LGMD type 2E/R4.' In keeping with FDA's pursuit of science-driven efficiency, the platform technology designation program is a critical tool that will help support rare disease therapies by streamlining drug development, manufacturing, and review processes for drug product applications by incorporating data from designated technologies. To be eligible for this designation, the technology must be a well-understood and reproducible technology – such as a nucleic acid sequence, molecular structure, mechanism of action, delivery method, vector, or a combination of any such technologies – and can support the development of multiple drugs or biologics. A platform is also eligible for consideration if it is incorporated in an approved drug and preliminary evidence demonstrates that the technology has the potential to be incorporated in or utilized by more than one drug without an adverse effect on quality, manufacturing, or safety, including using the same manufacturing process. Once granted, the designation enables sponsors to leverage prior data from the platform to support investigational new drug applications (INDs), new drug applications (NDAs) or biologic license applications (BLAs). About SRP-9003 (bidridistrogene xeboparvovec) SRP-9003 (bidridistrogene xeboparvovec) is an investigational gene therapy designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat neuromuscular diseases. SRP-9003 is intended to deliver a full-length beta-sarcoglycan transgene and uses the MHCK7 promoter, chosen for its ability to robustly express in the heart which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking Statements This statement contains 'forward-looking statements.' Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as 'believe,' 'anticipate,' 'plan,' 'expect,' 'will,' 'may,' 'intend,' 'prepare,' 'look,' 'potential,' 'possible' and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, product candidates, the potential benefits of AAVrh74 and the potential for our therapies to be transformative for patients living with rare diseases. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results; success in clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; we may not be able to comply with all FDA requests in a timely manner or at all; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; the possible impact of regulatory decisions by, and any halts imposed by, regulatory agencies on our business; and those risks identified under the heading 'Risk Factors' in our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.
Yahoo
27-05-2025
- Business
- Yahoo
Was Jim Cramer Right About Sarepta Therapeutics Inc. (SRPT)?
We recently published a list of In this article, we are going to take a look at where Sarepta Therapeutics Inc. (NASDAQ:SRPT) stands against other stocks that Jim Cramer discusses. A caller asked about Sarepta Therapeutics Inc. (NASDAQ:SRPT), a biotech firm working on RNA therapies. Cramer responded with cautious optimism: 'You know what, I have not looked at it lately — what it's doing with RNA and some really tough diseases. It has been a company that has not made money, but is about to break out and make money. I'm not going to say no to it.' Despite being cautiously positive, Cramer missed this one as Sarepta plunged 68.98%. Sarepta Therapeutics Inc. (NASDAQ: SRPT) is focused on precision genetic medicine, including RNA therapies for rare neuromuscular diseases like Duchenne muscular dystrophy. A laboratory technician in a white coat holding a microscope and examining a vial of biopharmaceuticals. As of 2025, Cramer has changed his opinion on the stock. Here's what he said on the 15th of January when asked about it: 'You're totally right. I think the RNA-based therapeutic companies are not doing well. I wonder if this isn't like a reverse halo effect because of Moderna. I didn't quite understand, I agree with you. It, it is troubling. I don't want to be there.' Overall, SRPT ranks 2nd on our list of stocks that Jim Cramer discusses. While we acknowledge the potential of SRPT as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than SRPT and that has 100x upside potential, check out our report about this cheapest AI stock. READ NEXT: 20 Best AI Stocks To Buy Now and 30 Best Stocks to Buy Now According to Billionaires. Disclosure: None. This article is originally published at Insider Monkey. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
20-05-2025
- Business
- Yahoo
SRPT Q1 Earnings Call: Sarepta Adjusts Guidance After Safety Event and Administrative Delays
Biotech company Sarepta Therapeutics (NASDAQ:SRPT) reported Q1 CY2025 results beating Wall Street's revenue expectations , with sales up 80.2% year on year to $744.9 million. Its non-GAAP loss of $3.42 per share was significantly below analysts' consensus estimates. Is now the time to buy SRPT? Find out in our full research report (it's free). Revenue: $744.9 million vs analyst estimates of $693.5 million (80.2% year-on-year growth, 7.4% beat) Adjusted EPS: -$3.42 vs analyst estimates of -$0.65 (significant miss) Adjusted EBITDA: -$239.6 million vs analyst estimates of -$371 million (-32.2% margin, 35.4% beat) Operating Margin: -40.3%, down from 8.4% in the same quarter last year Free Cash Flow was -$627.1 million compared to -$274.5 million in the same quarter last year Market Capitalization: $3.58 billion Sarepta Therapeutics' first quarter results were shaped by significant growth in its gene therapy and exon-skipping product lines, but management acknowledged that the period was marked by operational and clinical challenges. CEO Doug Ingram highlighted that while the company treated more patients with ELEVIDYS—its Duchenne muscular dystrophy gene therapy—than ever before, administrative hurdles, site imbalances, and a recent safety event led to a more cautious outlook for the rest of the year. Looking ahead, management cited three main factors behind its revised annual guidance: the need for extensive educational outreach following a patient death related to ELEVIDYS, slower administrative processing times for gene therapy infusions, and a concentration of demand at top treatment sites nearing capacity. Ingram stated, 'We are changing our net product revenue guidance…driven by three factors,' emphasizing that these are expected to impact both timing and volume of infusions in 2025. As Sarepta navigated a complex quarter, management detailed several operational and strategic developments tied to product performance and the company's evolving market approach. The quarter's results were primarily influenced by events in gene therapy delivery, site operations, and ongoing safety monitoring. ELEVIDYS Uptake and Challenges: Management reported a sharp increase in ELEVIDYS usage, but noted that a patient death from acute liver failure led some families to delay infusions while seeking more information about the therapy's safety profile. Administrative Process Delays: The company cited a longer-than-expected timeframe from patient enrollment to infusion, particularly due to complex paperwork, insurance agreements, and scheduling bottlenecks at certain centers. Specific issues, such as delays with Medi-Cal in California, have since been addressed. Site Utilization Imbalance: Roughly 60% of ELEVIDYS revenue came from a handful of leading treatment sites, many now booked up to a year in advance. Sarepta aims to engage secondary sites to better distribute patient volume and reduce wait times. Proactive Educational Outreach: Management launched comprehensive outreach efforts targeting both physicians and patients, including direct-to-consumer campaigns and digital resources, to address safety concerns and expedite access to therapy. Pipeline and Research Momentum: Beyond ELEVIDYS, Sarepta advanced its limb-girdle muscular dystrophy (LGMD) and siRNA programs, with expectations to submit a biologics license application for its EMERGENE therapy in the second half of the year and share new data from ongoing studies. Looking to the remainder of 2025, management's outlook centers on resolving operational hurdles and rebuilding momentum for ELEVIDYS, while expanding access and maintaining investment in R&D. Broader Site Engagement: Sarepta plans to intensify support for secondary infusion centers, aiming to alleviate the backlog at top sites and improve overall patient access to ELEVIDYS. Educational Initiatives: Management believes that ongoing education of both healthcare providers and patient families about the safety and efficacy data will be critical to restoring confidence and driving new treatment initiations. Pipeline Readouts and Regulatory Milestones: Sarepta anticipates key data releases and regulatory filings, particularly in its LGMD and siRNA programs, which management expects will underpin future revenue and diversify the company's therapeutic base. Tazeen Ahmad (Bank of America Securities): Asked which factor—site capacity, administrative complexity, or the safety event—most heavily influenced revised guidance. Management cited cycle times as the principal mechanical driver, with all three contributing. Ritu Baral (TD Cowen): Inquired how Sarepta plans to direct patients to less-utilized centers and whether more sites might open. CEO Doug Ingram stated the focus will be on expanding engagement and education at existing secondary sites rather than increasing the total number. Louise Chen (Scotiabank): Queried whether the guidance revision was entirely ELEVIDYS-related and about anticipated sales trends. Management confirmed the adjustment was due to ELEVIDYS and expects demand to recover in the summer and back half of the year. Andrew Tsai (Jefferies): Questioned the risk of more severe regulatory actions, such as product withdrawal, following the safety event. Management emphasized ELEVIDYS' risk-benefit profile and ongoing regulatory support. Eliana Merle (UBS): Sought clarity on potential label updates for ELEVIDYS after the patient death. Sarepta stated a supplement had been filed with the FDA, with a review expected by year-end. In upcoming quarters, the StockStory team will monitor (1) Sarepta's progress in expanding ELEVIDYS capacity at secondary sites, (2) the impact of educational initiatives on therapy adoption following recent safety concerns, and (3) the timing and outcomes of key clinical and regulatory milestones in the LGMD and siRNA pipelines. Continued resolution of administrative bottlenecks will also be closely watched as an indicator of operational improvement. Sarepta Therapeutics currently trades at a forward P/E ratio of 3.6×. In the wake of earnings, is it a buy or sell? The answer lies in our free research report. The market surged in 2024 and reached record highs after Donald Trump's presidential victory in November, but questions about new economic policies are adding much uncertainty for 2025. While the crowd speculates what might happen next, we're homing in on the companies that can succeed regardless of the political or macroeconomic environment. 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Yahoo
13-05-2025
- Business
- Yahoo
Sarepta Therapeutics Announces Approval in Japan of ELEVIDYS, a Gene Therapy to Treat Duchenne Muscular Dystrophy
Approval based on efficacy and safety results from clinical studies, including longer-term functional outcomes from EMBARK global phase 3 study Company is eligible to receive up to $103.5M in near-term regulatory and commercial milestone payments CAMBRIDGE, Mass., May 13, 2025--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the Japanese Ministry of Health, Labour, and Welfare (MHLW) has approved ELEVIDYS (delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy (DMD) under the conditional and time-limited approval pathway in Japan. ELEVIDYS is approved for individuals ages 3- to less than 8-years-old, who do not have any deletions in exon 8 and/or exon 9 in the DMD gene, and who are negative for anti-AAVrh74 antibodies. This is the first global approval to include individuals younger than 4 years of age. The approval is based on the efficacy and safety data for ELEVIDYS, which includes muscle health and longer-term functional results from the ELEVIDYS clinical programs, including the two-year data from the global Phase 3 EMBARK clinical trial (Study SRP-9001-301). These longer-term functional outcomes showed that individuals treated with ELEVIDYS had significantly better outcomes in multiple motor function measures, compared to a well-matched external control group. No new safety signals were observed in the EMBARK study over the two-year duration. These findings from Part 2 of EMBARK were presented at the 2025 Muscular Dystrophy Association meeting. In addition, one-year data from part 1 of the EMBARK study were published in Nature Medicine in October 2024 and quantitative muscle MR (magnetic resonance) outcomes from part 1 of EMBARK were published in JAMA Neurology in May 2025. "For nearly a decade, Sarepta has been a leader in advancing the treatment of Duchenne muscular dystrophy through innovative treatment options. With the approval of ELEVIDYS in Japan, children who are living with this rare, fatal disease, one marked by progressive muscle deterioration and weakness, now have an additional treatment option," said Louise Rodino-Klapac, Sarepta's head of research and development and chief scientific officer. "Sarepta is committed to supporting our partners so we can bring this treatment to additional people with Duchenne around the world who urgently need a therapy that potentially addresses the root cause of disease." The conditional and time-limited approval pathway in Japan provides for marketing authorization in Japan for up to seven years for innovative medicines to treat serious conditions. To be eligible for the pathway, certain additional criteria must also be met, including early clinical trial results that have demonstrated significant efficacy and safety. As part of a collaboration agreement signed in 2019, Sarepta is working with Roche to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world. Commercialization of ELEVIDYS in Japan is through Chugai Pharmaceuticals via its alliance with Roche. About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated in U.S. for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval in the U.S. based on expression of ELEVIDYS micro-dystrophin (noted hereafter as "micro-dystrophin") in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). U.S. IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient's clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Preexisting Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia. Report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking Statements This statement contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believe," "anticipate," "plan," "expect," "will," "may," "intend," "prepare," "look," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to receipt of financial milestones, our future operations, research and development programs, the potential timing of clinical trials and the potential benefits and risks of ELEVIDYS. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; the possible impact of regulatory decisions by, and any halts imposed by, regulatory agencies on our business; and those risks identified under the heading "Risk Factors" in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law. Source: Sarepta Therapeutics, Inc. View source version on Contacts Investor Contact: Ian Estepan617-274-4052iestepan@ Media Contacts: Tracy Sorrentino617-301-8566tsorrentino@ Kara Hoeger617-710-3898KHoeger@ Sign in to access your portfolio
