Rocket Pharmaceuticals (NASDAQ: RCKT) shares rose 4% on Wednesday after the biotechnology company secured access to up to $150 million under a credit facility with Hercules Capital.
The facility strengthens Rocket's financial position and provides additional capital to support its pivotal Phase 2 study of RP-A501 for Danon disease and its broader cardiovascular pipeline.
Danon disease is a rare X-linked inherited, multi-organ lysosomal-associated disorder with a devastating clinical course.
The company also highlighted progress on the FDA-aligned pivotal trial during an investor webinar, including early safety observations from the first three patients treated under a modified protocol.
The three treated under the modified protocol completed the initial observation period without clinical or laboratory evidence of thrombotic microangiopathy or capillary leak syndrome, although follow-up remains ongoing and the findings are preliminary.
The pivotal Phase 2 trial will include 12 male patients receiving commercial-grade RP-A501 at a recalibrated dose, with the first three patients treated under the modified protocol included in the efficacy population.
RP-A501 is Rocket's investigational gene therapy for Danon disease and the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies.
Rocket said the trial requires at least seven patients to meet both co-primary response criteria at 12 months, including myocardial LAMP2 protein expression of at least Grade 1 and a reduction in left ventricular mass index of at least 10% from baseline.
Longer-term Phase 1 data showed sustained biological activity, with all six patients in the principal analysis demonstrating myocardial LAMP2 expression and at least a 10% reduction in left ventricular mass index at about 12 months.
At follow-up of about three to seven years, left ventricular mass index remained below baseline in five of six patients, with reductions of at least 10% in four patients.
Rocket expects to complete pivotal trial dosing by mid-2027 and report topline results in mid-2028.
