Business
Rocket Pharmaceuticals Reports Third Quarter 2020 Financial Results and Highlights Recent Progress
—Positive Phase 1 Study Results of RP-L201 for the Treatment of LAD-I Demonstrate Ongoing Efficacy, Durability and Validation of Rocket’s “Process B”
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n—Positive Phase 1 Study Results of RP-L201 for the Treatment of LAD-I Demonstrate Ongoing Efficacy, Durability and Validation of Rocket’s “Process B” Lentiviral Platform—\n\n—Data Readouts in PKD, FA “Process B” and Danon Disease on Track for December—\n\n—First Patient Treated in Higher Dose Cohort of Phase 1 Study of RP-A501 for the Treatment of Danon Disease—\n\n—Strong Balance Sheet with $229 Million in Cash; Capitalized into 2Q 2022—\n\n NEW YORK--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial results for the quarter that ended September 30, 2020, along with an update on the Company's key pipeline developments, business operations and upcoming milestones.\n\n“We are incredibly pleased with the steady progress we made across all five of our clinical programs this quarter. The data from our RP-L201 trial for LAD-I demonstrated the potential for a very robust and attractive profile for our ‘Process B’ lentiviral gene therapy pipeline. We also treated our first higher dose patient in our Phase 1 study of RP-A501 for the treatment of Danon Disease, and presented compelling preclinical data in IMO,” said Gaurav Shah, M.D., President and Chief Executive Officer of Rocket. “In addition, I am proud that against the backdrop of a global pandemic, while there have been some delays in patient follow up and data collection, we continued to enroll and treat patients in our LVV and AAV gene therapy programs as well as expand our clinical trials internationally. Finally, we made strong progress in the build-out of Rocket’s AAV R&D and manufacturing facility, with the ability to produce GMP product anticipated in 2021.”\n\nDr. Shah continued, “We are looking forward to sharing additional updates as we enter the fourth quarter. These data announcements include an update on the Phase 1 and 2 trials for FA ‘Process B’, and for the first time, Phase 1 clinical data from our two largest indications: RP-L301 for PKD and RP-A501 for Danon Disease. We believe we are advancing closer to our goal of taking drug products from the discovery phase to BLA submission and launch so we may help improve the lives of patients facing these rare and devastating childhood diseases.”\n\n...