Business
Rocket Pharmaceuticals Reports First Quarter 2020 Financial Results and Highlights Recent Progress
—Treatment of First Cohort Complete, Clearance from FDA and IDSMC to Advance to Higher Dose in Phase 1 Trial of RP-A501 for Danon Disease — —Longer-Term FA
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n—Treatment of First Cohort Complete, Clearance from FDA and IDSMC to Advance to Higher Dose in Phase 1 Trial of RP-A501 for Danon Disease —\n\n\n—Longer-Term FA “Process A” Data and LAD-I Update to Be Presented at ASGCT—\n\n\n—Data Readout from Danon, FA “Process B”, LAD-I, and PKD Remain on Track—\n\n\n—Strong Balance Sheet with $275.9 Million in Cash, Capitalized into 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 disorders, today reports financial results for the quarter that ended March 31, 2020, along with an update on the Company's key pipeline developments, business operations and upcoming milestones.\n\n\n“During the first quarter, we continued to advance five programs across both the AAV and Lenti platforms,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “Notably this quarter, we completed patient treatment in the low dose cohort for RP-A501 in the Phase 1 Danon Disease trial with no dose-limiting toxicities, and are now pleased to report FDA and IDSMC clearance to advance to a higher dose cohort. This has been an important priority for us as Danon Disease affects so many young patients with devastating heart, muscle and CNS manifestations. We also continued to progress our clinical programs for FA, LAD-I, and PKD. Rocket is at an exciting stage of growth, with a rich pipeline of development-stage opportunities and the potential to transform the lives of patients afflicted with rare disease. In the coming months, we look forward to presenting updated FA ‘Process A’ and LAD-I ‘Process B’ data at ASGCT, treating our first patient in a higher dose cohort for RP-A501, and bringing our fifth program, IMO, to the clinic.”\n\n\nDr. Shah continued, “With regard to the COVID-19 pandemic, we are well-positioned overall to continue our progress while we monitor and adjust to this challenging global crisis. We have experienced modest COVID-19-related pauses in patient enrollment and follow-up that are being managed on a patient-by-patient basis, along with some delays in data collection. Nonetheless, we remain on track for providing updates on our lentiviral pipeline. Assuming that COVID-19 associated delays have shorter-term impact, we remain committed ...