Rocket Pharmaceu Stock Price, News & Analysis

Rocket Pharmaceuticals (NASDAQ: RCKT) announced positive clinical data from its gene therapy programs for Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD) at the 24th ASGCT Annual Meeting. The results indicate successful engraftment in FA patients, with 6 out of 9 showing clinical improvement. In the LAD-I trial, CD18 expression levels improved significantly, equating to better survival rates. Additionally, PKD patients demonstrated normalized hemoglobin levels post-treatment. The company aims to advance these therapies toward regulatory submissions.

Rocket Pharmaceuticals, Inc. (RCKT) reported financial results for Q1 2021, revealing a net loss of $40.2 million, or $0.65 per share, compared to $24.7 million, or $0.45 per share, year-over-year. The company has $466.4 million in cash, expected to fund operations into the second half of 2023. Key updates include successful Phase 1 trial results for RP-A501 in Danon Disease, and positive findings in RP-L201 for LAD-I. However, the FDA has requested a pause in patient dosing for RP-A501 to address risk mitigation, delaying enrollment by one quarter.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announces clinical data presentations at the 24th American Society of Gene and Cell Therapy Annual Meeting, May 11-14, 2021. Key presentations include interim results from a Phase 1/2 study on gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I) and updates on Pyruvate Kinase Deficiency (PKD) and Fanconi Anemia (FA). With high unmet medical needs, Rocket aims to address severe pediatric diseases with innovative gene therapies, as detailed in oral and poster sessions throughout the event.

Rocket Pharmaceuticals (NASDAQ: RCKT) has announced positive interim data from its Phase 1/2 trial of RP-L201, a gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I), a life-threatening pediatric disorder. The data shows that all treated patients exhibited substantial CD18 expression, with one patient nearing survival at one year post-treatment. Significant improvements were noted in skin lesions and infection rates. The findings point towards potential BLA/MAA filings in the US and Europe. The study involved four patients with severe LAD-I, highlighting the therapy's safety and effectiveness.

Rocket Pharmaceuticals (NASDAQ: RCKT) has announced that the European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation to its investigational gene therapy RP-L201 for treating Leukocyte Adhesion Deficiency-I (LAD-I). This designation is based on encouraging safety and efficacy data from the ongoing Phase 1/2 trial. The company aims to expedite the delivery of this potentially curative therapy for pediatric patients suffering from severe LAD-I, with initial Phase 2 trial data expected in Q2 2021.

Rocket Pharmaceuticals announces promising preliminary results from its Phase 1 trial of RP-L301 for treating Pyruvate Kinase Deficiency (PKD). The therapy demonstrated durable normalization of hemoglobin levels for up to 6 months, with patient L301-006-1001 showing an increase from ~7.4 g/dL to 13.9 g/dL and significant bilirubin improvement. RP-L301 was well tolerated, with no serious safety issues reported. The company is set to present more comprehensive data at a scientific venue later this year, affirming the therapy's potential in addressing PKD's substantial burden.

Rocket Pharmaceuticals (NASDAQ: RCKT) announced that its investigational gene therapy, RP-L201, received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. This designation, granted based on promising safety and efficacy data from an ongoing Phase 1/2 clinical trial, allows for expedited development of the therapy aimed at treating Leukocyte Adhesion Deficiency-I (LAD-I). Patient enrollment for the trial is complete, providing optimism for future FDA dialogue and potential registration. The company plans to share trial data in the second quarter of 2021.

Rocket Pharmaceuticals (NASDAQ: RCKT) reported strong operational results for 2020, boasting a cash position of $482.7 million, sufficient to fund operations into H2 2023. The company anticipates significant clinical advancements in 2021, including data from its five gene therapy programs. Positive updates were noted in trials for Danon Disease, Fanconi Anemia, and Leukocyte Adhesion Deficiency-I. Notably, Rocket strengthened its balance sheet through a $300 million equity offering and leadership expansion. The FDA granted Fast Track Designation for key programs, highlighting the potential for expedited product development.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company, is participating in two upcoming healthcare conferences. Gaurav Shah, M.D., CEO, will engage in a fireside chat at the 10th Annual SVB Leerink Global Healthcare Conference on February 25, 2021, at 2:20 p.m. ET. The company will also attend the Cowen’s 41st Annual Health Care Conference on March 2, 2021. A live audio webcast of the events will be accessible on the company’s website, with a replay available post-conference.