Rocket Pharmaceuticals and FDA Converge on Phase 2 Gene Therapy Trial for Danon Disease, Fueling Investor Optimism

New Jersey’s Rocket Pharmaceuticals has successfully aligned with the FDA on the design of a pivotal Phase 2 trial for a rare disease, potentially hastening the approval of their gene therapy. This open-label study will enlist 12 patients for treatment with RP-A501, an AAV9 gene therapy developed to enhance heart function by introducing a gene absent in those with Danon disease. Key evaluation metrics will include LAMP2 protein expression and variations in left ventricular mass, illuminating protein concentrations and cardiac health.

Last year, during discussions with the FDA, Rocket conveyed its intent to kick-start the trial’s initial segment by mid-2023. They apprised investors of the FDA’s receptiveness towards a “biomarker-based composite endpoint” and the agency’s understanding of the hurdles of a randomized trial for Danon disease.
While syncing Rocket’s objectives with FDA expectations was more prolonged than expected, their shared vision on the trial design has fostered mutual agreement. This alignment bolstered investor confidence, propelling Rocket’s stock up by 30%, reaching $20 in Wednesday’s premarket trading.

Gaurav Shah, M.D., Rocket’s CEO, hailed this progress as a crucial move towards offering a revolutionary treatment for Danon disease patients, who, without such therapies, could confront heart transplant necessities or potential mortality.

Post one year of treatment, Rocket intends to assess these primary outcomes and continue monitoring secondary results, like event-free survival, for two years. Plans are also afoot to introduce the trial in Europe shortly, and a pediatric safety assessment with two enrollees is currently ongoing.

Capitalizing on the favorable market reaction, Rocket initiated a stock sale, surpassing its primary goal of $150 million to rake in $175 million. With a reported fund balance of $307 million in June, Rocket anticipates this will buttress their operations through to 2025.

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