Rocket Pharmaceuticals (RCKT) Announces Positive Gene Expression, Clinical Biomarker and Preliminary Functional Data from Phase 1 Trial of RP-A501 for Treatment of Danon Disease

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Go back to Rocket Pharmaceuticals (RCKT) Announces Positive Gene Expression, Clinical Biomarker and Preliminary Functional Data from Phase 1 Trial of RP-A501 for Treatment of Danon Disease

Rocket Pharmaceuticals Announces Positive Gene Expression, Clinical Biomarker and Preliminary Functional Data from Phase 1 Trial of RP-A501 for the Treatment of Danon Disease

December 8, 2020 4:00 PM EST

Low Dose Showed Positive Increases in Cardiac Protein ExpressionTwo Patients With >50% by IHC, One at Month 9 and One at Month 12

Decreases in Cardiac Biomarker BNP of >50% and Stabilization of Clinical Biomarkers CK-MB and Transaminases in Two Patients

Visible Reduction of Autophagic Vacuoles, a Primary Hallmark of Danon Disease, in Heart Muscle

Individual 1.62- and 1.35-Fold Increases in Cardiac Output Observed in Two Patients at Month 12 and Month 9, Respectively

Low Dose Cohort Generally Well-Tolerated with Manageable Safety

Safety... More