Rocket Pharma Surges 28.87% on FDA Trial Resumption Enters Top 500 Volume

Rocket Pharmaceuticals (RCKT) surged 28.87% on August 20, 2025, with $0.21B in trading volume. The rise followed the FDA lifting the clinical hold on its Phase 2 trial of RP-A501 for Danon disease, a rare genetic disorder causing heart failure. The agency confirmed Rocket addressed safety concerns, allowing the trial to resume with a recalibrated dose and updated protocols.

The FDA authorized the trial to proceed with a 3.8 x 10¹³ GC/kg dose of RP-A501 in three patients, spaced at least four weeks apart. This aligns with lower-dose efficacy observed in Phase 1 trials while prioritizing safety. Rocket will also adopt an immunomodulatory regimen mirroring its Phase 1 pediatric cohort, discontinuing C3 complement inhibitors but retaining sirolimus, rituximab, and steroids. The revised protocol lowers the threshold for administering a C5 inhibitor if needed.

Having treated six patients in Phase 2 so far, Rocket expects further updates after evaluating data from the next three patients. The trial’s co-primary endpoints focus on LAMP2 protein expression and cardiac mass reduction, with secondary metrics including troponin levels and long-term survival. A concurrent natural history study supports the trial’s data collection, with all participants undergoing a three-month pre-treatment observation period.

A strategy of holding top 500 volume stocks for one day from 2022 returned 31.52% over 365 days, averaging 0.98% daily. It peaked at 7.02% in June 2023 but dropped 4.20% in September 2022, showing market volatility.