Rocket Pharmaceuticals Inc (RCKT) shares surged 32% in pre-market trading on Wednesday after the company announced it has reached an agreement with the US Food and Drug Administration (FDA) over the global Phase 2 trial of RP-A501 to address Danon disease, a fatal inherited cardiomyopathy.
Rocket said its study would evaluate the use of the investigational gene therapy RP-A501 to treat 12 patients with Danon disease.
"As a one-time potentially curative infusion, RP-A501 has the potential to restore normal cardiac function and provide a lifetime of benefit to patients with Danon Disease who have no other viable treatment options," Rocket Pharmaceuticals CEO Gaurav Shah said in a statement.
Danon disease affects an estimated 15,000 to 30,000 patients in the US and Europe, according to the company.
Stifel analysts, meanwhile, were upbeat on the finalized trial design.
"Today's press release provided much needed/awaited clarity on RCKT's next steps in DD, and to be fair, the path forward is largely in-line (or perhaps better) than what the company and key opinion leaders had previously suggested," the analysts wrote in a note to clients.
Shares of Rocket Pharmaceuticals have fallen 20% year to date as of Tuesday’s closing price.
Contact Sean at sean@proactiveinvestors.com