Latest news with #SamiCorwin
Yahoo
16-07-2025
- Business
- Yahoo
William Blair Maintains a Buy on Ultragenyx Pharmaceutical (RARE)
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the best oversold NASDAQ stocks to buy now. On July 14, William Blair analyst Sami Corwin maintained a bullish stance on Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), giving it a Buy rating without a price target. A research team in a laboratory peering through microscopes at a biologic product. The analyst gave a positive rating based on the company's potential despite recent setbacks. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) received a complete response letter from the FDA about UX111 for the treatment of Sanfilippo syndrome type A, citing concerns associated with chemistry, manufacturing, and controls (CMC). Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), however, maintains that the complications are not directly related to the quality of the product and are manageable. The analyst also reasoned that the FDA acknowledged the supportive nature of the clinical data for UX111, which includes biomarker evidence and strong neurodevelopmental outcomes. He stated that while the FDA requested updated clinical data, the existing data package does not have issues, which is a positive sign. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) plans to promptly address the concerns put forth by the FDA and is planning resubmission and potential approval in 2026. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) develops, acquires, and commercializes novel products to treat genetic diseases. Its product portfolio includes Crysvita, Mepsevii, Dojolvi, and Evkeeza. While we acknowledge the potential of RARE as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 30 Stocks That Should Double in 3 Years and 11 Hidden AI Stocks to Buy Right Now. 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
Business Insider
17-06-2025
- Business
- Business Insider
TD Cowen Reaffirms Their Buy Rating on Sarepta Therapeutics (SRPT)
TD Cowen analyst Ritu Baral maintained a Buy rating on Sarepta Therapeutics (SRPT – Research Report) today and set a price target of $62.00. The company's shares closed today at $20.94. 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 Baral covers the Healthcare sector, focusing on stocks such as Sarepta Therapeutics, Alnylam Pharma, and Neurogene. According to TipRanks, Baral has an average return of 7.3% and a 46.40% success rate on recommended stocks. In addition to TD Cowen, Sarepta Therapeutics also received a Buy from William Blair's Sami Corwin in a report issued today. However, on the same day, Wolfe Research initiated coverage with a Hold rating on Sarepta Therapeutics (NASDAQ: SRPT).
Yahoo
16-06-2025
- Business
- Yahoo
Sarepta and Roche halt DMD gene therapy's use after second death
Serepta and global partner Roche have discontinued the commercial and clinical use of Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec) in non-ambulatory patients after a safety profile re-assessment following two patient deaths. Roche said that the dosing restrictions are effective immediately, with commercial use stopped and enrolment in clinical trials paused until additional risk mitigation measures are implemented in the study protocol. In a separate announcement, Sarepta said it is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for the therapy. The biopharma added it is temporarily suspending shipments of Elevidys for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated. The re-assessment of the benefit-risk ratio for non-ambulatory patients follows a second fatal acute liver failure (ALF) case in a DMD patient administered with Elevidys. Two fatal ALF cases have occurred in the gene therapy's use to date. Shares in Swiss-listed Roche fell 1% at market open following the development, while Sarepta was down 31.34% in pre-market trading on the Nasdaq. 'While we ultimately believe the implementation of a modified immunosuppression regimen could prevent future safety events in non-ambulatory patients, we recognise that another patient death will amplify investor concerns surrounding the potential removal of Elevidys from the commercial market, which we continue to view as an unlikely scenario,' said William Blair analyst Sami Corwin in a research note. Corwin added that Elevidys's label will likely be updated to reflect the risk of ALF. Elevidys is approved in the US for the treatment of ambulatory and non-ambulatory individuals aged four and older with DMD with a confirmed mutation in the DMD gene. It is the only gene therapy approved in the country for the rare musculoskeletal disease. Elevidys works by inserting a functional copy of the DMD gene, providing the missing instructions used to make the protein dystrophin. Sarepta Therapeutics, who developed the therapy, holds US rights to the drug, while Roche gained rights to other global territories through a 2019 agreement. The first death due to ALF, announced in March 2025, prompted the suspension of multiple clinical trials evaluating the gene therapy in different patient populations with DMD. The clinical holds are still in effect, whilst dosing will be paused in trials operating outside of Europe. Roche and Sarepta jointly manage the clinical studies for Elevidys. The companies did not release further details on the second death. Sarepta stated the event has been reported to the US Food and Drug Administration (FDA) and global health authorities. While elevated liver enzymes are a known side effect of adeno-associated virus-based gene therapies, the exact mechanism behind liver toxicity remains unclear. "We are deeply saddened by the loss of these two young men and are urgently working to mitigate any risks related to the use of Elevidys,' Garraway said "Patient safety is always our highest priority. Therefore, we have recommended halting treatment with Elevidys in non-ambulatory patients with immediate effect,' he added. 'Our paramount priority is the safety and well-being of the patients we serve. We are taking immediate, decisive steps to better understand and mitigate the risk of acute liver failure, including enhancing the immunosuppressive regimen, for those with Duchenne who are non-ambulatory,' said Sarepta's chief scientific officer and head of R&D Louise Rodino-Klapac. "Sarepta and Roche halt DMD gene therapy's use after second death " was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
Yahoo
16-06-2025
- Business
- Yahoo
Sarepta and Roche halt DMD gene therapy's use after second death
Serepta and global partner Roche have discontinued the commercial and clinical use of Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec) in non-ambulatory patients after a safety profile re-assessment following two patient deaths. Roche said that the dosing restrictions are effective immediately, with commercial use stopped and enrolment in clinical trials paused until additional risk mitigation measures are implemented in the study protocol. In a separate announcement, Sarepta said it is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for the therapy. The biopharma added it is temporarily suspending shipments of Elevidys for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated. The re-assessment of the benefit-risk ratio for non-ambulatory patients follows a second fatal acute liver failure (ALF) case in a DMD patient administered with Elevidys. Two fatal ALF cases have occurred in the gene therapy's use to date. Shares in Swiss-listed Roche fell 1% at market open following the development, while Sarepta was down 31.34% in pre-market trading on the Nasdaq. 'While we ultimately believe the implementation of a modified immunosuppression regimen could prevent future safety events in non-ambulatory patients, we recognise that another patient death will amplify investor concerns surrounding the potential removal of Elevidys from the commercial market, which we continue to view as an unlikely scenario,' said William Blair analyst Sami Corwin in a research note. Corwin added that Elevidys's label will likely be updated to reflect the risk of ALF. Elevidys is approved in the US for the treatment of ambulatory and non-ambulatory individuals aged four and older with DMD with a confirmed mutation in the DMD gene. It is the only gene therapy approved in the country for the rare musculoskeletal disease. Elevidys works by inserting a functional copy of the DMD gene, providing the missing instructions used to make the protein dystrophin. Sarepta Therapeutics, who developed the therapy, holds US rights to the drug, while Roche gained rights to other global territories through a 2019 agreement. The first death due to ALF, announced in March 2025, prompted the suspension of multiple clinical trials evaluating the gene therapy in different patient populations with DMD. The clinical holds are still in effect, whilst dosing will be paused in trials operating outside of Europe. Roche and Sarepta jointly manage the clinical studies for Elevidys. The companies did not release further details on the second death. Sarepta stated the event has been reported to the US Food and Drug Administration (FDA) and global health authorities. While elevated liver enzymes are a known side effect of adeno-associated virus-based gene therapies, the exact mechanism behind liver toxicity remains unclear. "We are deeply saddened by the loss of these two young men and are urgently working to mitigate any risks related to the use of Elevidys,' Garraway said "Patient safety is always our highest priority. Therefore, we have recommended halting treatment with Elevidys in non-ambulatory patients with immediate effect,' he added. 'Our paramount priority is the safety and well-being of the patients we serve. We are taking immediate, decisive steps to better understand and mitigate the risk of acute liver failure, including enhancing the immunosuppressive regimen, for those with Duchenne who are non-ambulatory,' said Sarepta's chief scientific officer and head of R&D Louise Rodino-Klapac. "Sarepta and Roche halt DMD gene therapy's use after second death " was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Yahoo
28-05-2025
- Business
- Yahoo
William Blair Reaffirms Outperform Rating on Rocket Pharma Despite Trial Setback
Sami Corwin, an analyst at William Blair, maintained his Outperform rating on Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) on May 27 with no price target, despite the company's recent difficulties. The biotechnology company's trial for the gene therapy medicine RP-A501 was clinically halted and a patient tragically passed away. Corwin stated that the delay was required due to the capillary leak syndrome (CLS) safety event, which had never been observed with RP-A501 or comparable treatments before. Rather than the AAV9 vector utilized in the treatment, Corwin expressed hope that the serious adverse event could be related to the recent addition of a C3 inhibitor to the immunosuppressive protocol. Only individuals who received the C3 inhibitor had CLS cases documented, the analyst said. Although the occurrences of thrombotic microangiopathy (TMA) are alarming, the analyst also pointed out that the afflicted patients are making a full recovery. Despite the RCKT stock's price tanking by around 80% year-to-date, Corwin thinks that investors are primarily concerned about the safety of the program and when the clinical hold would be lifted. He went on to say that Rocket's other clinical cardiovascular program, RP-A601, which employs a different AAV vector and does not include the C3 inhibitor, remains unaffected, implying that the recent occurrences shouldn't impact how this distinct program is viewed. While we acknowledge the potential of RCKT to grow, 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 RCKT and that has 100x upside potential, check out our report about the cheapest AI stock. Read More: and . Disclosure: None. 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
