Welcome to our dedicated page for Rocket Pharmaceu news (Ticker: RCKT), a resource for investors and traders seeking the latest updates and insights on Rocket Pharmaceu stock.
Rocket Pharmaceu (RCKT) is a pioneering biotechnology company developing gene therapies for rare pediatric diseases through innovative lentiviral-based approaches. This page provides investors and industry observers with a comprehensive repository of official company announcements, clinical development updates, and strategic partnership news.
Access timely updates on RCKT's research milestones, regulatory progress, and financial performance. Our curated collection includes press releases related to clinical trial outcomes, manufacturing advancements, and collaborations with leading medical institutions. Users will find verified information on therapeutic developments targeting high-need genetic disorders.
Key areas covered include novel treatment pipelines, FDA designations, intellectual property updates, and scientific presentations. Bookmark this page to stay informed about Rocket Pharmaceu's contributions to gene therapy innovation and its evolving position in the biopharmaceutical sector.
Rocket Pharmaceuticals (NASDAQ: RCKT) has completed enrollment in its global Phase 2 pivotal trial of RP-A501 for treating male patients with Danon disease. The trial enrolled 12 patients, including a two-patient safety run-in, with the remaining 10 patients enrolled across the U.S. and EU within three months. The study aims to evaluate the efficacy and safety of RP-A501, a one-time treatment designed to improve cardiac abnormalities associated with Danon disease.
The trial's co-primary endpoints are improvements in LAMP2 protein expression and reductions in left ventricular mass. Key secondary endpoints include changes in troponin, natriuretic peptides, and event-free survival to 24 months. Rocket Pharmaceuticals plans to pursue regulatory filings simultaneously in the U.S. and ex-U.S. markets, given the prevalence of Danon disease across regions.
Rocket Pharmaceuticals (NASDAQ: RCKT) has appointed Mikael Dolsten, M.D., Ph.D., as an independent director to its Board of Directors. Dr. Dolsten is currently the Chief Scientific Officer and President of Research & Development at Pfizer Inc. (NYSE: PFE), focusing on advancing gene therapies, small-molecule medicines, biotherapeutics, and vaccines. His extensive experience includes leadership roles at Wyeth, Boehringer Ingelheim, AstraZeneca, Pharmacia, and Upjohn.
Dr. Dolsten is a member of the Board of Overseers for the Scripps Research Institute and a Foreign Member of The Royal Swedish Academy of Engineering Sciences. He has also advised the Obama administration on regulatory and drug development issues and Vice President Biden's Cancer Moonshot Initiative. This appointment aims to strengthen Rocket's scientific capabilities and support its mission to develop transformative treatments for rare disorders.
Rocket Pharmaceuticals (NASDAQ: RCKT), a late-stage biotechnology company focusing on genetic therapies for rare disorders, has announced its participation in two upcoming investor conferences. The company will be present at the Morgan Stanley 22nd Annual Global Healthcare Conference on September 5, 2024, at 2:35 PM ET, and the 2024 Cantor Fitzgerald Global Healthcare Conference on September 17, 2024, at 10:55 AM ET.
CEO Gaurav Shah, M.D., will engage in fireside chats at both events, and the management team will host investor meetings. Webcasts of the presentations will be accessible on the Investors section of Rocket Pharmaceuticals' website, providing an opportunity for stakeholders to stay informed about the company's progress and strategies in developing genetic therapies for rare disorders with high unmet needs.
Rocket Pharmaceuticals (NASDAQ: RCKT) reported Q2 2024 financial results and pipeline progress. Key highlights include:
- Ongoing enrollment in pivotal Phase 2 study of RP-A501 for Danon disease and Phase 1 study of RP-A601 for PKP2 arrhythmogenic cardiomyopathy
- Presented long-term KRESLADI™ data showing 100% survival rate in LAD-I patients
- Cash position of $278.8M, expected to fund operations into 2026
- R&D expenses decreased to $91.6M from $97.8M in Q2 2023
- G&A expenses increased to $49.5M from $33.2M in Q2 2023
- Net loss of $131.7M or $1.40 per share
The company is preparing for potential FDA approval of KRESLADI for severe LAD-I and advancing its clinical pipeline in rare genetic disorders.
Rocket Pharmaceuticals (NASDAQ: RCKT) has received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application for KRESLADI™ (marnetegragene autotemcel; marne-cel), a gene therapy for severe leukocyte adhesion deficiency-I (LAD-I). The FDA has requested additional Chemistry Manufacturing and Controls (CMC) information. Rocket met with FDA leaders to expedite the approval process. The global Phase 1/2 study showed 100% overall survival at 12 months for all nine patients, with significant reductions in infections and improvements in LAD-I symptoms. KRESLADI™ was well tolerated with no serious adverse events reported.
Rocket Pharmaceuticals (NASDAQ: RCKT) has received orphan medicinal product designation from the European Commission for its gene therapy candidate, RP-A601, aimed at treating plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM). This inherited heart disease affects around 50,000 individuals in the U.S. and Europe, leading to life-threatening arrhythmias and sudden cardiac death. Currently, there are no curative treatments available. The orphan designation provides various financial and regulatory benefits, including a 10-year market exclusivity post-approval. Rocket is conducting a Phase 1 trial to evaluate the safety and preliminary efficacy of RP-A601 in adult PKP2-ACM patients.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) presented positive long-term data updates from its LV vector hematology portfolio at the ASGCT meeting. The data demonstrated sustained safety and efficacy in pivotal studies of KRESLADI™ for severe LAD-I, RP-L102 for Fanconi Anemia, and RP-L301 for PKD. KRESLADI™ showed 100% survival without HSCT, RP-L102 had genetic and phenotypic correction, and RP-L301 had clinically meaningful hemoglobin improvement. The safety profiles were well-tolerated with no drug-related serious adverse events.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) reported strong financial results for the first quarter of 2024. The company advanced its RP-L102 for Fanconi Anemia towards regulatory reviews, with an anticipated BLA submission in 2024. The EMA accepted the MAA for review. KRESLADI for severe LAD-I is on track for a PDUFA date of June 30, 2024. Aaron Ondrey was appointed as CFO. Rocket's cash, cash equivalents, and investments stood at $330.3M, providing operational runway into 2026.
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