Latest news with #SRPT
Yahoo
4 days ago
- Business
- Yahoo
The FDA granted Sarepta's rAAVrh74 Viral Vector a Platform Technology Designation
The FDA has designated the viral vector rAAVrh74, which Sarepta Therapeutics, Inc. (NASDAQ:SRPT) uses in its gene therapy candidate SRP-9003 for limb-girdle muscular dystrophy type 2E/R4 (LGMD2E/R4), as a platform technology. A laboratory technician in a white coat holding a microscope and examining a vial of biopharmaceuticals. Future approvals of treatments utilizing the same delivery technology might be accelerated by this designation. Phase III EMERGENE studies for SRP-9003, sometimes referred to as bidridistrogene xeboparvovec, are anticipated to yield data in the middle of 2025, and the FDA may file a proposal later this year. Sarepta Therapeutics, Inc. (NASDAQ:SRPT) uses the viral vector rAAVrh74, which is produced from rhesus monkeys, in a number of neuromuscular gene therapy initiatives. Only 14% of DMD patients had pre-existing antibodies, which suggests that the vector has low frequencies of these antibodies, which could improve efficacy and lower safety hazards. The FDA has acknowledged the vector's constancy and adaptability, according to Sarepta Therapeutics, Inc. (NASDAQ:SRPT)'s CSO, Louise Rodino-Klapac. The firm can now expedite development throughout its gene therapy pipeline by reusing clinical data, which will cut the timeframe for future therapies of neuromuscular diseases. 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 extremely cheap AI stock that is also a major beneficiary of Trump tariffs and onshoring, see our free report on the best short-term AI stock. READ NEXT: 10 High-Growth EV Stocks to Invest In and 13 Best Car Stocks to Buy in 2025. Disclosure. None.
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. Put yourself in the driver's seat and build a durable portfolio by checking out our Top 5 Strong Momentum Stocks for this week. This is a curated list of our High Quality stocks that have generated a market-beating return of 176% over the last five years. Stocks that made our list in 2020 include now familiar names such as Nvidia (+1,545% between March 2020 and March 2025) as well as under-the-radar businesses like the once-small-cap company Comfort Systems (+782% five-year return). Find your next big winner with StockStory today.
Yahoo
09-05-2025
- Business
- Yahoo
Why Sarepta Therapeutics, Inc. (SRPT) Nosedived on Wednesday
We recently published an article titled . In this article, we are going to take a look at where Sarepta Therapeutics, Inc. (NASDAQ:SRPT) stands against the other stocks. The stock market bounced back from the previous day's losses, with all major indices finishing higher as investors cheered the central bank's decision to keep interest rates unchanged. On Wednesday afternoon, the Federal Reserve kept rates steady at a range of 4.25 percent to 4.5 percent, saying that it was not in a hurry to cut rates and could still 'wait and see' the impacts of President Donald Trump's tariff policies. The Dow Jones rallied by 0.70 percent, the S&P 500 increased by 0.43 percent, and the Nasdaq grew by 0.27 percent. Beyond the major indices, bucked a broader market optimism as investors sold off on a series of disappointing news. To come up with the list, we considered only the stocks with a $2-billion market capitalization and $5-million trading volume. A laboratory technician in a white coat holding a microscope and examining a vial of biopharmaceuticals. Sarepta Therapeutics, Inc. (NASDAQ:SRPT) nosedived by 21.45 percent on Wednesday to end at $36.72 apiece after reporting a disappointing earnings performance in the first quarter of the year. During the period, the company swung to a net loss of $447.5 million from a $36.1 million net income in the same period last year, despite revenues jumping by 80 percent to $744.9 million from $413.5 million year-on-year, with the increase primarily driven by a $241-million higher net product revenue from Elevidys—a gene therapy for Duchenne muscular dystrophy (DMD)—as a result of its expanded label approval in June 2024. Looking ahead, Sarepta Therapeutics, Inc. (NASDAQ:SRPT) said it expects lower revenues for full-year 2025, at $2.3 billion to $2.6 billion versus the $2.9 billion to $3.1 billion as expected previously. Earlier this year, Sarepta Therapeutics, Inc. (NASDAQ:SRPT) reported that a young patient succumbed to acute liver failure after receiving the Sarepta Elevidys therapy. While the risk has already been flagged on the Elevidys label, acute liver injuries leading to death have not been previously reported in clinical testing or real-world use of Elevidys. Overall SRPT ranks 2nd on our list of Wednesday's worst performers. While we acknowledge the potential of SRPT as an investment, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns and doing so within a shorter time frame. There is an AI stock that went up since the beginning of 2025, while popular AI stocks lost around 25%. If you are looking for an AI stock that is more promising than SRPT but that trades at less than 5 times its earnings, check out our report about this . READ NEXT: and . Disclosure: None. This article is originally published at . 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
01-05-2025
- Business
- Yahoo
1 of Wall Street's Favorite Stock on Our Buy List and 2 to Approach with Caution
Wall Street is overwhelmingly bullish on the stocks in this article, with price targets suggesting significant upside potential. However, it's worth remembering that analysts rarely issue sell ratings, partly because their firms often seek other business from the same companies they cover. Luckily for you, we at StockStory have no conflicts of interest - our sole job is to help you find genuinely promising companies. Keeping that in mind, here is one stock where Wall Street's positive outlook is supported by strong fundamentals and two where analysts may be overlooking some important risks. Consensus Price Target: $20.81 (17.9% implied return) Formed in 2011 with the merger of Membase and CouchOne, Couchbase (NASDAQ:BASE) is a database-as-a-service platform that allows enterprises to store large volumes of semi-structured data. Why Does BASE Worry Us? 19.2% annual revenue growth over the last three years was slower than its software peers Customer acquisition costs take a while to recoup, making it difficult to justify sales and marketing investments that could increase revenue Historical operating losses point to an inefficient cost structure Couchbase's stock price of $17.65 implies a valuation ratio of 4x forward price-to-sales. If you're considering BASE for your portfolio, see our FREE research report to learn more. Consensus Price Target: $147.33 (135% implied return) Pioneering treatments for a devastating childhood muscle-wasting disease that primarily affects boys, Sarepta Therapeutics (NASDAQ:SRPT) develops and commercializes RNA-targeted therapies and gene therapies for rare genetic disorders, primarily Duchenne muscular dystrophy. Why Does SRPT Fall Short? Smaller revenue base of $1.90 billion means it hasn't achieved the economies of scale that some industry juggernauts enjoy (but also enables it to grow faster if it executes properly) Free cash flow margin dropped by 22.7 percentage points over the last five years, implying the company increased its investment activities to fend off competitors Limited cash reserves may force the company to seek unfavorable financing terms that could dilute shareholders At $62.62 per share, Sarepta Therapeutics trades at 5.6x forward price-to-earnings. Check out our free in-depth research report to learn more about why SRPT doesn't pass our bar. Consensus Price Target: $108.14 (20.8% implied return) Formerly part of Emerson Electric, Vertiv (NYSE:VRT) manufactures and services infrastructure technology products for data centers and communication networks. Why Will VRT Beat the Market? Core business can prosper without any help from acquisitions as its organic revenue growth averaged 18.5% over the past two years Free cash flow margin increased by 6.6 percentage points over the last five years, giving the company more capital to invest or return to shareholders Improving returns on capital reflect management's ability to monetize investments Vertiv is trading at $89.52 per share, or 22.9x forward price-to-earnings. Is now a good time to buy? See for yourself in our in-depth research report, it's free. Donald Trump's victory in the 2024 U.S. Presidential Election sent major indices to all-time highs, but stocks have retraced as investors debate the health of the economy and the potential impact of tariffs. While this leaves much uncertainty around 2025, a few companies are poised for long-term gains regardless of the political or macroeconomic climate, like our Top 9 Market-Beating Stocks. This is a curated list of our High Quality stocks that have generated a market-beating return of 175% over the last five years. Stocks that made our list in 2019 include now familiar names such as Nvidia (+2,183% between December 2019 and December 2024) as well as under-the-radar businesses like Sterling Infrastructure (+1,096% five-year return). Find your next big winner with StockStory today for free.
