Rocket Pharmaceuticals Reports Positive Results for Danon Disease Program

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) shared encouraging updates on its Danon Disease Program through a recent investor presentation. The presentation included long-term safety and efficacy outcomes from the Phase 1 RP-A501 study, demonstrating promising results for the treatment of Danon disease.

The data revealed that RP-A501 exhibited good tolerability, with all evaluated Danon disease patients showing sustained LAMP2 protein expression at 12 months (lasting up to 60 months). Additionally, there was a notable reduction in left ventricular mass index by 10% or more at 12 months (maintained up to 54 months) after treatment. These findings were presented at an American Heart Association Scientific Sessions and published in The New England Journal of Medicine, followed by a company webinar.

The Phase 1 study, conducted in male Danon disease patients, assessed the safety and preliminary efficacy of RP-A501. Patients receiving low and high doses demonstrated tolerability, with most adverse events being mild or moderate, and not attributed to RP-A501. One patient, whose heart failure worsened, underwent successful heart transplantation.

Positive long-term improvements were observed in patients, including sustained cardiac protein expression, reduced or stable left ventricular mass, preserved normal ejection fraction, and improvements in heart failure indicators and quality of life scores. Preliminary results from a specific patient highlighted substantial heart expression at the five-year mark.

In addition to the Danon Disease Program, Rocket Pharmaceuticals disclosed progress on its Pyruvate Kinase Deficiency Phase 2 RP-L301 Study, but enrollment is yet to begin. The company plans to resume patient recruitment in 2025, focusing on other active programs in the interim.

The filing emphasized that the information provided, including the investor presentation, is furnished data and not considered filed for requirements under the Exchange Act. Rocket Pharmaceuticals clearly outlined the steady advancement of its programs while maintaining regulatory compliance and strategic resource allocation for future endeavors.

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read Rocket Pharmaceuticals’s 8K filing here.

Rocket Pharmaceuticals Company Profile

Rocket Pharmaceuticals, Inc, together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia.