Latest news with #RocketPharmaceuticalsInc
Yahoo
28-05-2025
- Business
- Yahoo
Rocket Pharma's Gene Therapy Future Clouded By Regulatory Uncertainty
Rocket Pharmaceuticals Inc (NASDAQ:RCKT) on Tuesday announced an update related to RP-A501, its investigational gene therapy for Danon disease, a rare X-linked dominant genetic disorder that manifests with the clinical triad of cardiomyopathy (stiff heart muscles), skeletal myopathy (weakness) and intellectual disability. A patient participating in the Phase 2 pivotal trial of RP-A501 experienced an unexpected Serious Adverse Event (SAE). The patient passed away after an acute systemic infection. Needham analyst Gil Blum downgraded Rocket Pharmaceuticals from Buy to Hold, with a Nil price forecast from $42 earlier. The analyst cited increased uncertainty around the company's main value driver as the key reason for the downgrade.'We continue to have a favorable view of RP-A501's efficacy; however, we see increased uncertainty for the product given safety concerns,' Blum said in an analyst note. Blum anticipates limited readthrough to Rocket's other AAV programs, which utilize simpler immunosuppression regimens. Chardan Research analyst Geulah Livshits also lowered its price target from $46 to $17 while maintaining the Buy rating. While the disclosure does not negate the benefit reported for the program, the patient death and commentary regarding instances of TMA impact the benefit/risk profile of RP-A501, Livshits said in an analyst note. Livshits reduced the probability of success from 70% to 40%, given CBER leadership's focus on benefit/risk, pending more details regarding agency feedback and next steps. The analyst projects a slower uptake among the Danon population (3.6% by 2030E), assuming greater caution among prescribers. Chardan notes the company hasn't revealed how many patients have been treated in the trial so far but said the study is currently over-enrolled, with more patients ready to begin treatment. Management mentioned several possible next steps, such as switching to a simpler immunomodulatory treatment, using existing complement inhibitors known to be safe, and continuing to exclude patients who are genetically predisposed to complement activation. Chardan highlights that it's still unclear whether other immunomodulatory treatments could reduce the risk of TMA. There's no timeline for when the company might align with the FDA and restart the trial. Additional changes like enrolling more patients may be required, which could cause further delays. Given this uncertainty, the topline results are expected to come later than the previously guided mid-2026 timeframe. Other analyst reactions: Jefferies downgraded Rocket Pharmaceuticals from Buy to Hold and lowered the price target from $29 to $2.5. Leerink Partners downgraded Rocket Pharmaceuticals from Outperform to Market Perform, lowering the price target from $37 to $8. Scotiabank maintains Rocket Pharmaceuticals with a Sector Outperform, lowering the price target from $51 to $19. Price Action: RCKT stock is trading higher by 6.44% to $2.48 at last check Wednesday. Read Next:Image via Shutterstock Date Firm Action From To Feb 2022 SVB Leerink Maintains Outperform Dec 2021 Chardan Capital Maintains Buy Aug 2021 SVB Leerink Maintains Outperform View More Analyst Ratings for RCKT View the Latest Analyst Ratings Up Next: Transform your trading with Benzinga Edge's one-of-a-kind market trade ideas and tools. Click now to access unique insights that can set you ahead in today's competitive market. Get the latest stock analysis from Benzinga? This article Rocket Pharma's Gene Therapy Future Clouded By Regulatory Uncertainty originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved.
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
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
Yahoo
26-03-2025
- Business
- Yahoo
Is Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) the Best US Stock Under $10 to Buy Right Now?
We recently published a list of the . In this article, we are going to take a look at where Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) stands against other US stocks under $10 to buy right now. On March 12, Stephanie Guild of Robinhood and Marci McGregor of Bank of America Merrill Lynch shared their perspectives on the current state of the market and strategies for navigating uncertainty. Guild explained that retail investors' approach to buying the dip has shifted in line with changing market dynamics. While clients are still investing, they are focusing on specific names they favor. At the same time, she noted an increased tendency toward diversification, with investors allocating funds to index investments more than ever before. Additionally, many are taking advantage of Robinhood's 4% cash yield, which reflects a balanced approach to investing during uncertain times. McGregor emphasized the importance of focusing on long-term trends rather than reacting to short-term headlines, which often create unnecessary noise. She attributed some weak economic data from January to weather-related factors, calling it a temporary head fake. While acknowledging ongoing uncertainty around trade policy, McGregor highlighted key drivers for market recovery. She pointed out that 6 of the 11 S&P 500 sectors posted double-digit year-over-year earnings growth in Q4 and noted that market broadening is underway. Year-to-date, 63% of S&P constituents have outperformed the index compared to less than 30% over the last two years. This broadening trend signals potential strength in the market. Looking ahead, McGregor predicts a gradual easing of financial conditions later this year, potentially supported by a weaker dollar and improving corporate conditions. She suggested that if soft economic data begins to manifest more clearly, it could lead to Fed rate cuts. Factors such as deregulation, a merger cycle, and potential tax cut extensions may also contribute to recovery. Despite uncertainties, McGregor advised clients to buy on weakness and stay diversified. This includes exposure to international markets like Europe while maintaining confidence in the US as a leading market. We used the Finviz stock screener to compile a list of the top US stocks that were trading below $10 as of March 24. We then selected the 10 US stocks that were the most popular among elite hedge funds and that analysts were bullish on. The stocks are ranked in ascending order of the number of hedge funds that have stakes in them, as of Q4 2024. The hedge fund data was sourced from Insider Monkey's database which tracks the moves of over 900 elite money managers. Why are we interested in the stocks that hedge funds pile into? The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter's strategy selects 14 small-cap and large-cap stocks every quarter and has returned 373.4% since May 2014, beating its benchmark by 218 percentage points (see more details here). Scientific equipment in a lab setting, revealing the cutting edge of biotechnology research. Share Price as of March 24: $8.43 Number of Hedge Fund Holders: 45 Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) is a late-stage biotechnology company that develops gene therapies for rare and devastating diseases. It focuses on both AAV and LV programs. Its pipeline targets conditions like Danon disease, Fanconi Anemia, and Leukocyte Adhesion Deficiency-I. It aims to provide life-changing treatments. The company is invested in its cardiovascular gene therapy programs, with RP-A501 for Danon disease and RP-A601 for PKP2-ACM leading the charge. The Phase 2 pivotal study of RP-A501 is ongoing, with a program update expected in H1 2025. Data published in the New England Journal of Medicine and presented at the American Heart Association (AHA) demonstrated RP-A501's safety and meaningful efficacy. The Phase 1 study of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (ACM) has completed enrollment in the low-dose cohort, with initial data expected in H1 2025. Internal estimates indicate that PKP2-ACM affects ~50,000 people in the US and Europe. The company's strategy is to maintain resources on these AAV cardiovascular programs while managing its other pipeline assets to maximize value for patients and shareholders. Its cash runway, with $372.3 million in cash, cash equivalents, and investments, is expected to fund operations into Q3 2026. Baron Health Care Fund remains confident in the company's long-term potential due to its promising gene therapies for rare diseases and anticipated revenue generation. The fund stated the following regarding Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) in its Q2 2024 investor letter: 'Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) specializes in the development of gene therapies for rare genetic diseases outside of oncology. Currently these include Danon disease, Fanconi anemia, lysosomal acid lipase deficiency, and pyruvate kinase deficiency. The first three drug treatments are slated for commercial launch by 2025, which should generate substantial revenue. Shares detracted from performance after the FDA extended the priority review period by three months for the Kresladi gene therapy for leukocyte adhesion deficiency, potentially influenced by sluggish competitive gene therapy launches from bluebird bio in sickle cell disease and BioMarin in hemophilia B. Given the lifesaving nature of Rocket's therapies and the high unmet need for each of these life ending diseases, we retain conviction in our investment.' Overall, RCKT ranks 5th on our list of the best US stocks under $10 to buy right now. While we acknowledge the growth potential of RCKT, our conviction lies in the belief that AI stocks hold great promise for delivering high returns and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than RCKT but that trades at less than 5 times its earnings, check out our report about the 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. Sign in to access your portfolio
