Business
Rocket Pharmaceuticals Reports Second Quarter 2021 Financial Results and Highlights Recent Progress
— Working with FDA on Changes to Clinical Trial Protocol in Danon Disease Trial; Rocket Anticipates Trial Will Resume in 3Q — — Increasing and Durable
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n— Working with FDA on Changes to Clinical Trial Protocol in Danon Disease Trial; Rocket Anticipates Trial Will Resume in 3Q —\n\n— Increasing and Durable Benefit Observed in Low Dose Cohort (6.7e13 dose) in Danon Disease; Removing Higher Dose Cohort (1.1e14 dose) From Future Dosing Plans; Full Clinical Update Expected in 4Q –\n\n— Positive Results from FA, LAD-I, PKD Trials Presented at 24th ASGCT Annual Meeting Show Preliminary Evidence of Activity and Favorable Tolerability —\n\n— Clinical Updates in FA, LAD-I, PKD and IMO Also Expected in 4Q —\n\n— Ending Balance Sheet with $426.8 Million in Cash; Cash Runway Expected into 2H'23 —\n\n— Conference Call to Be Held at 4:30 p.m. ET Today —\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial results for the quarter ending June 30, 2021 and updates on the Company's key pipeline developments, business operations, and upcoming milestones.\n\n“We are grateful to the FDA for its support and for working with us on our Danon program toward resuming our trial, which we believe will occur in the third quarter,” said Gaurav Shah, M.D., chief executive officer of Rocket Pharma. “Further, we have observed durable clinical benefit in the low dose adult cohort, which we believe is supportive of its potential as a viable Phase 2 dose. As of July 2021, we see improvement in two of three low dose patients in NYHA class. In these two patients, we also observed substantial improvement of a key marker of heart failure, BNP, which decreased from a pretreatment baseline by 75 percent in one patient and 79 percent in the other as well as improvement in cardiac output by 35 percent in one patient and 62 percent in the other as measured by invasive hemodynamics. The third patient has demonstrated stabilization of NYHA class and BNP. Given the positive benefit/risk profile in the low dose, and additionally to mitigate safety concerns observed at the higher dose, in agreement with FDA we will no longer treat Danon patients with the higher dose (1.1e14). With our full focus on the low dose, we look forward to progressing our trial on behalf of Danon patients devastated by this disease.”\n\nDr. Shah continued, “We also presented ...