Rocket Pharma to launch pivotal mid-stage study of rare anemia gene therapy

Based on FDA feedback, Rocket Pharmaceuticals (RCKT -3.8%) will launch a Phase 2 clinical trial next quarter that, it says, should support a U.S. marketing application for gene therapy RP-L102 for Fanconi anemia, a rare inherited disorder characterized by bone marrow failure.

The primary endpoint will be resistance to mitomycin-C (MMC), a DNA-damaging chemo agent, in bone marrow cells at a minimum time point of one year. The company says MMC resistance may also serve as a surrogate endpoint for accelerated approval.

Another Phase 2 is currently underway in Europe with an estimated completion date of January 2023. The results will support both U.S. and European marketing applications.

https://seekingalpha.com/news/3497039-rocket-pharma-launch-pivotal-mid-stage-study-gene-therapy-rare-anemia