What's Going On With Gene Therapy-Focused Rocket Pharmaceuticals Stock Today?

On Tuesday, Rocket Pharmaceuticals Inc RCKT announced that alignment has been reached with the FDA on the global Phase 2 pivotal trial of RP-A501 for Danon Disease. 

Danon Disease is an inherited cardiomyopathy that leads to mortality in most male patients at age ~20 and females at age ~40. 

The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe.

The global, single-arm, multi-center Phase 2 pivotal trial will evaluate the efficacy and safety of RP-A501 in 12 patients with Danon Disease, including a pediatric safety run-in (n=2), with a natural history comparator and a dose level of 6.7 x 1013 GC/kg.

To support accelerated approval, the study will assess the efficacy of RP-A501 as measured by the biomarker-based co-primary endpoint consisting of improvements in LAMP2 protein expression and reductions in left ventricular mass. Key secondary endpoint is a change in troponin.

William Blair sees the update positively and believes it aligns with management’s previous guidance and our expectations.

Analysts Sami Corwin and Brooke Schuster write that the data from the Phase 1 study of RP-A501 de-risks the pivotal trial. 

Although RP-A501 will be the main stock driver in the near term, given its larger total addressable market, William Blair says the acceptance of the BLA for RP-L201 in leukocyte adhesion deficiency type I, the BLA filing for RP-L102 in Fanconi anemia, and the initiation of the pivotal Phase 2 trial of RP-L301 for pyruvate kinase deficiency could be incremental catalysts for Rocket by year-end.

Rocket Pharmaceuticals also announced an equity offering of $175 million, including 7.8 million shares at $16.00 per share and pre-funded warrants to purchase 3.1 million shares at $15.99 per pre-funded warrant.

Price Action: RCKT shares are up 39.70% at $21.36 on the last check Wednesday.