Rocket Pharmaceuticals' (RCKT) Q2 update confirmed its programs are on track, with management expecting regulatory updates on RP-A501 for Danon disease and RP-A601 for PKP2-ACM in H2, Wedbush Securities said Tuesday in a report.
Danon disease is a rare inherited disorder that causes heart and skeletal muscle weakness, and PKP2-ACM is a genetic form of arrhythmogenic cardiomyopathy that can lead to dangerous heart rhythm disturbances.
Rocket plans to dose nine additional Danon patients, bringing the total number of patients on the recalibrated dose in the pivotal Phase 2 study to 12, Wedbush said.
For RP-A601, engagement with the FDA remains ongoing to finalize the design of a potential Phase 2 study, while Phase 1 remains open and actively enrolling, the report said. Rocket also expects to dose the first patient in the Phase 1 study of RP-A701 for BAG3-DCM in H2, Wedbush said.
BAG3-DCM is a genetic form of dilated cardiomyopathy that causes progressive weakening of the heart muscle.
Wedbush lowered its price target on Rocket stock to $15 from $16 and maintained its outperform rating.
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