Latest news with #DanonDisease
Yahoo
30-05-2025
- Business
- Yahoo
Leerink Partners Downgrades Rocket Pharmaceuticals (RCKT) to Market Perform on Danon Disease Blow
Rocket Pharmaceuticals, Inc.'s (NASDAQ:RCKT) prospects have taken a significant hit on the company being forced to halt research on its experimental gene therapy for Danon Disease. The decision comes on the experimental drug RP-A501, resulting in a serious adverse event that led to a patient's demise. Likewise, Leerink Partners downgraded the stock to 'Market Perform' from an 'Outperform' and cut the price target to $8 from $37 on May 28. A successful investor with a portfolio including Finance Receivables, biotechnology, and medical device investments. The downgrade comes on Rocket Pharmaceutical recording its biggest slump in the market. The stock lost more than 50% in market value after the US Food and Drug Administration placed a hold on phase 2 trials for the candidate drug. Rocket Pharmaceuticals' webcast disclosed a serious adverse event (SAE), where a patient succumbed to acute systemic infection, leading to a clinical hold on the Phase 2 Danon disease program. It is a rare hereditary condition that results in gradual muscular weakening and damage to the heart muscle. Leerink Partners insists the company needs to address safety uncertainties and resolve clinical hold for a favorable rating. In addition, Leerink Partners insists that Rocket Pharmaceuticals faces an uphill task of re-establishing investor confidence. That's partly because the company needs to meet revised timelines for clinical programs, including LAD-I, Danon, and FA. Following the significant blow on the Danon Disease treatment, Rocket Pharmaceuticals has imploded to all-time lows. The stock is down by about 80% year to date. Rocket Pharmaceuticals, Inc. is a late-stage biotechnology company dedicated to developing gene therapies for rare and life-threatening diseases in the U.S., advancing adeno-associated viral (AAV) and lentiviral (LV) programs to target cardiac and immune disorders. Its AAV pipeline includes treatments for Danon disease (RP-A501, Phase 2), Plakophilin-2 Arrhythmogenic Cardiomyopathy (RP-A601, Phase 1), and BAG3 Dilated Cardiomyopathy (preclinical). At the same time, its LV platform develops therapies for Leukocyte Adhesion Deficiency-I (RP-L201), Fanconi Anemia (RP-L102), and Pyruvate Kinase Deficiency (RP-L301), addressing critical immune and blood disorders. While we acknowledge the potential of Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) 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 RCKT and that has 100x upside potential, check out our report about the cheapest AI stock. READ NEXT: and . Disclosure: None. Sign in to access your portfolio
Yahoo
28-05-2025
- Business
- Yahoo
Leerink Partners Downgrades Rocket Pharmaceuticals (RCKT) to Market Perform on Danon Disease Blow
Rocket Pharmaceuticals, Inc.'s (NASDAQ:RCKT) prospects have taken a significant hit on the company being forced to halt research on its experimental gene therapy for Danon Disease. The decision comes on the experimental drug RP-A501, resulting in a serious adverse event that led to a patient's demise. Likewise, Leerink Partners downgraded the stock to 'Market Perform' from an 'Outperform' and cut the price target to $8 from $37 on May 28. A successful investor with a portfolio including Finance Receivables, biotechnology, and medical device investments. The downgrade comes on Rocket Pharmaceutical recording its biggest slump in the market. The stock lost more than 50% in market value after the US Food and Drug Administration placed a hold on phase 2 trials for the candidate drug. Rocket Pharmaceuticals' webcast disclosed a serious adverse event (SAE), where a patient succumbed to acute systemic infection, leading to a clinical hold on the Phase 2 Danon disease program. It is a rare hereditary condition that results in gradual muscular weakening and damage to the heart muscle. Leerink Partners insists the company needs to address safety uncertainties and resolve clinical hold for a favorable rating. In addition, Leerink Partners insists that Rocket Pharmaceuticals faces an uphill task of re-establishing investor confidence. That's partly because the company needs to meet revised timelines for clinical programs, including LAD-I, Danon, and FA. Following the significant blow on the Danon Disease treatment, Rocket Pharmaceuticals has imploded to all-time lows. The stock is down by about 80% year to date. Rocket Pharmaceuticals, Inc. is a late-stage biotechnology company dedicated to developing gene therapies for rare and life-threatening diseases in the U.S., advancing adeno-associated viral (AAV) and lentiviral (LV) programs to target cardiac and immune disorders. Its AAV pipeline includes treatments for Danon disease (RP-A501, Phase 2), Plakophilin-2 Arrhythmogenic Cardiomyopathy (RP-A601, Phase 1), and BAG3 Dilated Cardiomyopathy (preclinical). At the same time, its LV platform develops therapies for Leukocyte Adhesion Deficiency-I (RP-L201), Fanconi Anemia (RP-L102), and Pyruvate Kinase Deficiency (RP-L301), addressing critical immune and blood disorders. While we acknowledge the potential of Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) 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 RCKT and that has 100x upside potential, check out our report about the cheapest AI stock. READ NEXT: 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
Reuters
27-05-2025
- Business
- Reuters
FDA halts Rocket Pharma's gene therapy trial as complications lead to patient death
May 27 (Reuters) - The U.S. FDA has paused a mid-stage gene therapy trial by Rocket Pharmaceuticals (RCKT.O), opens new tab after a patient suffered serious complications, which ultimately led to the person's death, the drug developer said on Tuesday. Shares of the New Jersey-based company tumbled 63% to $2.30 in premarket trading. They had closed at $6.27 on Friday. The company said the FDA had placed a hold on the trial on Friday after the report of a capillary leak syndrome, a condition where fluid and proteins leak out of tiny blood vessels into surrounding tissues, causing swelling and low blood pressure. The patient had subsequently died, it said. Rocket was testing its experimental gene therapy, RP-A501, for the treatment of Danon disease, a rare genetic disorder that causes heart muscle damage and progressive muscle weakness. The company said it had voluntarily paused further dosing in the study. It is also working with the U.S. Food and Drug Administration and experts to resolve the trial halt and resume the study quickly, but cannot provide a timeline for completion until the clinical hold is lifted. The FDA did not immediately respond to a Reuters request for comment.
Yahoo
03-04-2025
- Business
- Yahoo
Rocket Pharmaceuticals to Participate in the 24th Annual Needham Virtual Healthcare Conference
CRANBURY, N.J., April 03, 2025--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that management will participate in the 24th Annual Needham Virtual Healthcare Conference taking place on April 8, 2025. Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat at 8:45 a.m. ET on Tuesday, April 8. A webcast of the fireside chat will be available here and on the Investors section of the Company's website. An archived replay of the webcast will be available for approximately 30 days following the event. About Rocket Pharmaceuticals, Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket's innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives. Rocket's adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket's lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. For more information about Rocket, please visit and follow us on LinkedIn, YouTube, and X. Rocket Cautionary Statement Regarding Forward-Looking StatementsThis press release contains forward-looking statements concerning Rocket's future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as "could," "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket's expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket's ongoing and planned clinical trials, the expected timing and outcome of Rocket's regulatory interactions and planned submissions, including the timing and outcome of the FDA's review of the additional CMC information that Rocket will provide in response to the FDA's request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket's ability to establish key collaborations and vendor relationships for its product candidates, Rocket's ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket's ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket's ability to transition to a commercial stage pharmaceutical company, and Rocket's expectation that its cash, cash equivalents and investments will be sufficient to funds its operations into the third quarter of 2026. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket's competitors' activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket's ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket's ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket's ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2024, filed February 27, 2025 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise. View source version on Contacts Media & Investors Meg Dodgemdodge@ Media Kevin Giordanomedia@ Sign in to access your portfolio
