Rocket Pharmaceuticals (NASDAQ:RCKT) stock rose 17.7% in premarket trading on Wednesday after the gene therapy developer said that it has reached an agreement with the U.S. Food and Drug Administration (FDA) regarding the global Phase 2 trial of RP-A501.
The trial aims to address Danon disease, a fatal inherited cardiomyopathy.
“We believe this milestone sets us on the most efficient and rapid path to delivering this potentially transformative therapy to Danon Disease patients who would otherwise progress to heart transplantation or death,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.
Research firm Stifel, while surprised by the co-primary endpoints of the trial, expressed a positive stance on the finalized trial design.
“Today's PR 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 KOLs had previously suggested,” the analysts wrote in a client note.
Additionally, Rocket Pharma initiated a $150 million offering of shares and pre-funded warrants through financial institutions including JPMorgan, Morgan Stanley, Leerink Partners, TD Cowen, and LifeSci Capital.