other material
confidence high
sentiment positive
materiality 0.60
Rocket Pharma reports positive Phase 1 data for RP-A601 in PKP2-ACM
ROCKET PHARMACEUTICALS, INC.
- RP-A601 well-tolerated; no dose-limiting toxicities in 3 patients with up to 12 months follow-up.
- PKP2 protein expression increased ~110% and ~398% from baseline in 2 patients with low expression.
- Improved RV function, KCCQ-12 scores (34-41 points), and NYHA class (II to I) in patients.
- PVCs reduced 9-63%; NSVT episodes eliminated in one patient. No further dose escalation.