Business
Rocket Pharmaceuticals Reports Second Quarter 2020 Financial Results and Highlights Recent Progress
—“Process B” Demonstrates Safety and Sustained Efficacy for FA (RP-L102) and LAD-I (RP-L201)— —First Patient Treated in Global Phase 1 Study of RP-L301 for
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n—“Process B” Demonstrates Safety and Sustained Efficacy for FA (RP-L102) and LAD-I (RP-L201)—\n\n\n—First Patient Treated in Global Phase 1 Study of RP-L301 for PKD—\n\n\n—FDA Clearance of IND for IMO (RP-L401)—\n\n\n—Preliminary Data Readouts in Danon and PKD and Additional “Process B” Data in FA and LAD-I on Track for Fourth Quarter—\n\n\n—Strong Balance Sheet with $250 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 June 30, 2020, along with an update on the Company's key pipeline developments, business operations and upcoming milestones.\n\n\n“During this quarter, I am pleased to announce we continued to build pipeline momentum with positive data readouts and significant clinical trial progress,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “We continued to enroll patients in our FA, LAD-I and PKD trials, and received FDA clearance of our IND for IMO. In addition, we presented positive data demonstrating sustained efficacy for both RP-L102 for FA and RP-L201 for LAD-I at ASGCT. With the continued buildout of our manufacturing facility, we anticipate GMP clinical material produced in 2021. With steady commitment and dedication, we remain focused on establishing a truly integrated and industry-leading multi-platform gene therapy approach aimed at improving and saving lives.”\n\n\nDr. Shah continued, “Our progress comes amidst the backdrop of COVID-19, which continues to affect the lives of millions. As members of the healthcare community, we are committed to continuing to operate with a sense of responsibility to our employees and patients as we advance our pipeline aimed at addressing so many devastating rare genetic disorders. We want to also applaud the relentless work of our partners, patients and healthcare communities who skillfully navigate and respond to this pandemic with passion and care.”\n\n\nKey Pipelines and Operational Updates\n\n\n\nPhase 1 clinical trial of RP-A501 for the treatment of Danon Disease continues to progress. Rocket has been cleared by the U.S. Food and Drug Administration (FDA) and the Independent Data S...