Rocket Q2 2022 Earnings Report
Key Takeaways
Rocket Pharmaceuticals reported positive results across all four clinical programs and made progress toward manufacturing readiness. The company maintains a healthy operational cash runway into the first half of 2024.
Reported positive safety data from pediatric cohort of Phase 1 Danon Disease study; efficacy update on track for late Q3 2022.
Announced top-line data from pivotal Phase 2 trial for severe LAD-I with 100% overall survival at one year; regulatory filings anticipated in first half of 2023.
Primary endpoint met for Fanconi Anemia pivotal Phase 2 trial, FDA dialogue initiated for BLA planning; continued data readouts expected in Q4 2022.
Achieved cGMP readiness milestone for AAV manufacturing facility in Cranbury, N.J.
Rocket
Rocket
Forward Guidance
Rocket Pharmaceuticals expects resources will be sufficient to fund its operating expenses and capital expenditure requirements into the first half of 2024.