Latest news with #Elevidys
Yahoo
a day ago
- Business
- Yahoo
Circle initiated, Charter upgraded: Wall Street's top analyst calls
The most talked about and market moving research calls around Wall Street are now in one place. Here are today's research calls that investors need to know, as compiled by The Upgrades: Wolfe Research upgraded Charter (CHTR) to Peer Perform from Underperform without a price target. President Trump's "Big Beautiful Bill" seems likely to reinstate 100% bonus depreciation, notes the firm, which estimates the five major connectivity providers could cumulatively gain $10.5B of cash tax relief in 2025 alone. Wells Fargo upgraded Mondelez (MDLZ) to Overweight from Equal Weight with a price target of $78, up from $68. The firm sees the "intersection" of price execution, "ever-muted" inflation in 2026 and a low relative valuation creating the "optimal bull case" for Mondelez. BMO Capital upgraded CubeSmart (CUBE) to Outperform from Market Perform with a $49 price target, up from $48, and moved to the analyst's top pick in storage. The firm sees CubeSmart's more urban exposure versus peers, exemplified in leading demographics, as likely to continue to outperform, particularly in New York City. Stifel upgraded EPR Properties (EPR) to Buy from Hold with a price target of $65, up from $52, visiting the company. With improvements in the share price and cost of capital, the company "can once again return to reasonable external growth," the firm tells investors in a research note. Top Downgrades: William Blair downgraded Sarepta (SRPT) to Market Perform from Outperform without a price target. The reduction in peak revenue opportunity following second case of fatal acute liver failure due to Elevidys treatment and "growing number of uncertain variables will be a deterrent for investors," the firm tells investors in a research note. Argus downgraded Campbell's (CPB) to Hold from Buy. The firm believes that valuations on the stock are reasonable given the company's weak volume and "less than robust" outlook, though it would look to upgrade on signs of sustained volume and margin growth. Oppenheimer downgraded Johnson Controls (JCI) to Perform from Outperform on valuation. Following re-rating during the June quarter, valuation now sits at historical highs across multiple metrics, the firm notes. BMO Capital downgraded Public Storage (PSA) to Market Perform from Outperform with a $325 price target. The firm remains positive on Public Storage's long-term fundamentals but says the shares have outperformed over the past year. Stifel downgraded Jack in the Box (JACK) to Hold from Buy with a price target of $20, down from $32. The Trump administration's aggressive immigration policies are likely to create a "significant sales headwind for an unpredictable period." Top Initiations: Seaport Research initiated coverage of Circle (CRCL) with a Buy rating and $235 price target, telling investors that the firm views Circle as "a top-tier crypto 'disruptor' with a sizeable future opportunity." Janney Montgomery Scott initiated coverage of Everest Group (EG) with a Buy rating and $425 fair value estimate. Everest is a "top-tier" global property and casualty reinsurer with a growing primary insurance business with "a long-term track record of strong performance," having generated a median operating return on equity of 12% and average annual growth in shareholder value of 11% over the last 25 years, the firm tells investors. Macquarie initiated coverage of Sportradar (SRAD) with an Outperform rating and $32 price target. The market is still underestimating more U.S. state legalization, the in-play betting shift, and further market penetration in international markets, contends the firm, which doesn't think Sportradar's current valuation is reflective of OSB in California and Texas nor a U.S. in-play shift above 50% this decade. Wolfe Research initiated coverage of TPG (TPG) with an Outperform rating and $60 price target, representing over 20% upside. The firm sees "several ways" for TPG to generate high-teens fee-related earnings growth through 2027. Wells Fargo initiated coverage of Metsera (MTSR) with an Overweight rating and $65 price target. Wells sees a good risk/reward at current levels, saying data over the next year will strengthen Metsera's "differentiated profile." 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
Epoch Times
4 days ago
- Health
- Epoch Times
Second Patient Dies After Gene Therapy for Duchenne Muscular Dystrophy: Company
A second patient has died after receiving the gene therapy Elevidys, which was approved by the Food and Drug Administration (FDA) over concerns of staffers, according to the company that makes it. The 15-year-old boy who died, and the 16-year-old boy whose death was
Forbes
4 days ago
- Health
- Forbes
Second Patient Death After Gene Therapy: Implications For The Future
As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics announced a second patient death following treatment with its gene therapy, Elevidys, for Duchenne muscular dystrophy. This news has sent shockwaves through the rare disease and biotech communities. It raises questions about the safety of adeno-associated virus-based gene therapies. The latest fatality, a 15-year-old, died from acute liver failure, a recognized but rare complication of adeno-associated virus-based gene transfer. This death follows a similar case reported in March. These recent tragedies have profound implications. Elevidys, Sarepta's gene therapy, was among the first to receive approval for the treatment of Duchenne muscular dystrophy. Yet, the deaths of two patients treated with Elevidys now force us to confront a difficult question. Do the potential benefits of this therapy truly outweigh the risks, particularly for those who can no longer walk and may be at higher risk of complications? Every advance in medicine brings new challenges, and gene therapy is no exception. The deaths associated with Elevidys have raised the regulatory bar for all investigational gene therapies that use adeno-associated virus vectors. This means more stringent safety monitoring, more careful patient selection, and better reporting of outcomes. This heightened scrutiny is not unwelcome, even if these measures may slow the pace of development in the short term. They are essential for ensuring safety and responsibility. Adeno-associated virus-based vectors have become the workhorse of gene therapy due to their efficiency and relatively low risk of complications. The field has long recognized that high-dose adeno-associated virus-based administration can trigger severe, sometimes fatal, immune-mediated toxicities. These primarily affect the liver. In 1999, Jesse Gelsinger died after receiving an adenoviral gene therapy. His tragic death marked a turning point in the field and led to decades of increased scrutiny and regulation. While adeno-associated viruses are generally less immunogenic than adenoviruses, high doses can activate the body's immune system. This leads to inflammation, liver injury, and, in rare cases, organ failure. Recent studies highlight that immune responses can contribute to toxicity, particularly in the liver. However, not all patients are impacted equally. Recent findings underscore that age and disease status can significantly influence a patient's response to adeno-associated virus-based gene therapy. Clinical studies reveal that individual factors, such as underlying infections, pre-existing liver disease, or genetic predispositions, can amplify the risks. For example, in the first reported Elevidys death, a concurrent infection may have played a role in the patient's rapid decline. Research from the University of Bristol indicates that in ocular gene therapy trials, older patients, particularly females, are more likely to experience severe inflammatory responses. However, in the Elevidys cases, both fatalities involved males who had lost the ability to walk due to advanced disease progression. This is a critical factor linked to heightened immune vulnerability. This underscores that risks are context-specific, depending on therapy type, delivery site, and patient health status. To address the pressing safety concerns, Sarepta is investigating several strategies. One approach under consideration is the addition of sirolimus, an immunosuppressant widely used in organ transplantation. While this aims to reduce immune-mediated liver damage, experience from liver transplant patients warns that sirolimus can nearly triple infection risk, even at low doses. Its use in gene therapy remains experimental and demands careful evaluation before it can be widely adopted. At the same time, non-viral delivery methods are emerging as a promising alternative. Lipid nanoparticles offer a way to bypass many of the immune challenges posed by viral vectors. Their ability to be tailored for precise tissue targeting and the absence of pre-existing immunity in patients make them particularly attractive for conditions like Duchenne muscular dystrophy, where repeated dosing may be necessary. Though still less efficient than viral vectors in some applications, the technology is advancing quickly. The second death following Elevidys for Duchenne muscular dystrophy is a sobering reminder of the risks inherent in adeno-associated virus-based gene transfer, particularly for older or more severely affected patients. Still, these tragedies are not new to the field. We have seen similar inflection points before. The history of gene therapy is marked by moments of triumph and tragedy. Each shapes the path forward. As we reflect on these recent events, it is clear that the field stands at another such crossroads—one that will require careful balancing of hope, caution, and scientific rigor to chart the best course for patients and the future of medicine. For a deeper exploration of these issues, including real-world patient stories and the latest scientific advances, see Destiny's Child No Longer: Rewriting Genetic Fate, which provides a detailed examination of this therapy and other gene therapies.
Business Wire
4 days ago
- Business
- Business Wire
SRPT BREAKING NEWS: Sarepta Therapeutics, Inc. is being Investigated for Securities Fraud after Second Elevidys Death; Investors with Losses are Notified to Contact BFA Law
NEW YORK--(BUSINESS WIRE)--Leading securities law firm Bleichmar Fonti & Auld LLP announces an investigation into Sarepta Therapeutics, Inc. (NASDAQ: SRPT) for potential violations of the federal securities laws. Leading securities law firm Bleichmar Fonti & Auld LLP announces an investigation into Sarepta Therapeutics, Inc. (NASDAQ: SRPT) for potential violations of the federal securities laws. Share If you invested in Sarepta, you are encouraged to obtain additional information by visiting: Why is Sarepta being Investigated? Sarepta is a biopharmaceutical company focused on developing treatments for rare diseases. Sarepta's most important product is Elevidys, a therapy for the treatment of Duchenne muscular dystrophy. During the relevant period, Sarepta repeatedly touted the safety profile of Elevidys and told investors that the benefits of the treatment outweighed its risks. In truth, Elevidys causes fatal acute liver failure in some patients. The Stock Declines as the Truth is Revealed On March 18, 2025, Sarepta announced that a patient that had been treated with Elevidys died after suffering acute liver failure. On this news, the price of Sarepta stock fell $27.81 per share, or over 27%, from $101.35 per share on March 17, 2025, to $73.54 per share on March 18, 2025. Nevertheless, on the same day, Sarepta assured investors that 'the benefit-risk of ELEVIDYS remains positive.' Next, on June 15, 2025, Sarepta announced that a second patient treated with Elevidys had died from acute liver failure. The company further announced that because of the second death, it was suspending certain shipments of Elevidys and paused dosing in an ongoing clinical trial of the treatment. On this news, the price of Sarepta stock fell $15.24 per share, or more than 42%, from $36.18 per share on June 13, 2025, to $20.94 per share on June 16, 2025. Click here for more information: . What Can You Do? If you invested in Sarepta you may have legal options and are encouraged to submit your information to the firm. All representation is on a contingency fee basis, there is no cost to you. Shareholders are not responsible for any court costs or expenses of litigation. The firm will seek court approval for any potential fees and expenses. Why Bleichmar Fonti & Auld LLP? Bleichmar Fonti & Auld LLP is a leading international law firm representing plaintiffs in securities class actions and shareholder litigation. It was named among the Top 5 plaintiff law firms by ISS SCAS in 2023 and its attorneys have been named Titans of the Plaintiffs' Bar by Law360 and SuperLawyers by Thompson Reuters. Among its recent notable successes, BFA recovered over $900 million in value from Tesla, Inc.'s Board of Directors, as well as $420 million from Teva Pharmaceutical Ind. Ltd. For more information about BFA and its attorneys, please visit Attorney advertising. Past results do not guarantee future outcomes.
Forbes
4 days ago
- Business
- Forbes
SRPT Stock: What's Happening With Sarepta Therapeutics?
CANADA - 2025/06/08: In this photo illustration, the Sarepta Therapeutics logo is seen displayed on ... More a smartphone screen. (Photo Illustration by Thomas Fuller/SOPA Images/LightRocket via Getty Images) Sarepta Therapeutics (NASDAQ: SRPT) suffered a devastating blow on Monday, June 16 when its stock plummeted 42% following news of a second patient death linked to its Duchenne muscular dystrophy gene therapy, Elevidys. The biotechnology company and its global partner Roche immediately suspended commercial and clinical use of the treatment in non-ambulatory patients pending a comprehensive safety review. The decision came after Roche conducted a safety profile reassessment that revealed concerning patterns following the two fatalities. Commercial distribution has ceased effective immediately, while enrollment in ongoing clinical trials has been paused until additional risk mitigation measures can be implemented in study protocols. Now, if you are looking for an upside with a smoother ride than an individual stock, consider the High-Quality portfolio, which has outperformed the S&P, and clocked >91% returns since inception. On a separate note, see – Should You Buy CRWV Stock After A Whopping 4x Rise? Elevidys represents a significant advancement in gene therapy for Duchenne Muscular Dystrophy, a devastating genetic disorder that progressively weakens muscles. The FDA granted accelerated approval in June 2023 for ambulatory patients aged 4-5, later expanding traditional approval to all ambulatory patients aged 4 and older. However, continued approval for non-ambulatory patients remained contingent on confirmatory clinical trial results. The financial implications for Sarepta are profound. Elevidys generated 43% of the company's total revenues last year, making it the cornerstone of Sarepta's commercial portfolio. Industry analysts had projected peak sales of over $3 billion for the therapy, expectations that now face substantial revision given the restricted patient population. Sarepta's financial profile already presented significant challenges before this setback. Despite achieving average revenue growth exceeding 40% over the past three years, the company remains unprofitable with an operating cash flow margin of negative 25% and a net income margin of negative 11%. The company carries $1.3 billion in debt against a current market capitalization of $2.06 billion, resulting in a concerning debt-to-equity ratio of 63%. The stock's trajectory illustrates the extreme volatility inherent in pharmaceutical investments. Shares have collapsed from their 52-week high of approximately $172 to current levels around $20, representing the kind of dramatic price swings that characterize companies heavily dependent on revenues from only a few drugs. While Sarepta's multi-year low valuations might appear attractive to contrarian investors, the investment proposition carries substantial risks. The combination of recent safety concerns with its flagship product, overwhelming dependence on a single revenue source, significant cash burn, and extreme stock volatility creates a highly speculative investment scenario suitable only for investors with high risk tolerance. Investors seeking exposure to innovative healthcare developments might consider diversified approaches, such as the Trefis High Quality (HQ) Portfolio, that spread risk across multiple companies and sectors, rather than concentrating capital in individual speculative positions. With a collection of 30 stocks, this portfolio has a track record of comfortably outperforming the S&P 500 over the last 4-year period. Why is that? As a group, HQ Portfolio stocks provided better returns with less risk versus the benchmark index; less of a roller-coaster ride, as evident in HQ Portfolio performance metrics.
