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NEW YORK - Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a $308 million market cap biotechnology company, announced Thursday that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational gene therapy RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM). According to InvestingPro data, the company maintains a strong liquidity position with more cash than debt on its balance sheet.
The designation, which provides benefits including intensive FDA guidance and expedited review, was based on positive safety and efficacy data from the Phase 1 clinical trial of RP-A601, according to a company press release. With a healthy current ratio of 9.19, the company appears well-positioned to fund its clinical development programs, though InvestingPro analysis indicates it is quickly burning through cash.
PKP2-ACM is a life-threatening inherited heart disease that causes ventricular arrhythmias and sudden cardiac death, affecting approximately 50,000 adults and children in the U.S. and Europe.
Preliminary results from the ongoing Phase 1 trial presented at the 2025 American Society of Gene and Cell Therapy meeting showed that all three adult patients treated with a single dose of RP-A601 demonstrated increased PKP2 protein expression. Two patients with low baseline levels showed increases of 110% and 398%.
The trial data also indicated improvements in right ventricular function, reduced ventricular arrhythmias, and enhanced quality of life. The therapy was generally well-tolerated with no dose-limiting toxicities reported.
"This marks the fifth RMAT designation in our history and underscores our commitment to developing potentially curative gene therapies for patients with rare and inherited cardiovascular diseases," said Kinnari Patel, President and Head of R&D at Rocket Pharmaceuticals, in the statement.
RMAT designation was established under the 21st Century Cures Act to expedite development and review of promising therapeutic candidates for serious or life-threatening diseases. The designation provides benefits including early FDA interactions and potential priority review of biologics license applications.
Rocket’s RP-A601 also holds Fast Track designation in the U.S. and Orphan Drug designation in both the U.S. and Europe.
In other recent news, Rocket Pharmaceuticals announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug application for RP-A701, a gene therapy candidate targeting BAG3-associated Dilated Cardiomyopathy. This marks an important milestone as RP-A701 becomes the company’s third clinical-stage gene therapy candidate. The company is preparing to initiate a Phase 1 clinical trial to evaluate the safety and efficacy of this therapy. Additionally, Rocket Pharmaceuticals appointed Christopher Stevens as chief operating officer, transitioning Dr. Kinnari Patel to president and head of R&D. Stevens’ compensation package includes a base salary of $525,000 and equity compensation valued at approximately $2.5 million. Meanwhile, UBS lowered its price target for Rocket Pharmaceuticals to $5.00 from $12.00, maintaining a Buy rating despite a setback in the Danon disease program. Canaccord Genuity also reduced its price target to $11 from $34, citing changes in the anticipated launch timeline for RP-A501/Danon. Both UBS and Canaccord Genuity remain optimistic about Rocket Pharmaceuticals’ future prospects, particularly with the anticipated data readout for the PKP2 program in the second half of 2025.
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