The Rocket Pharmaceuticals Inc (NASDAQ: RCKT) stock price soared 37.4% after announcing that it had reached alignment with the US Food and Drug Administration (FDA) on a pivotal Phase 2 trial design for its RP-A501 drug treatment in Danon Disease.
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The company also noted that Danon Disease is a uniformly fatal disease in both male and female patients, with male patients dying at about age 20 years and female patients dying at about 40 years. It is an inherited genetic disease that affects an estimated 15,000 to 30,000 patients in the U.S. and Europe.
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The Phase 2 Pivotal trial is a global, single-arm, multi-centre trial that 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.
Rocket Pharma also noted that it had sufficient material for the pivotal study due to the hard work of its CMC team over the past several years. The CMC team established the company’s in-house cGMP manufacturing facilities to generate enough RP-A501 for the 12-month study.
To support accelerated approval, the study will evaluate the efficacy of RP-A501 as measured by the biomarker-based co-primary endpoint consisting of improvements in LAMP2 protein expression (≥ Grade 1, as measured by immunohistochemistry) and reductions in left ventricular (LV) mass. A critical secondary endpoint is the change in troponin.
Gaurav Shah, M.D., the CEO of Rocket Pharma, said: “I am very excited to announce our alignment with the FDA on our pivotal study design for RP-A501 for Danon Disease, which reflects the highly collaborative discussions with the review team and senior management at FDA’s Center for Biologics Evaluation and Research and marks the first-ever regulatory pathway to approval for a genetic treatment for heart disease. 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.”
“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 Pharma (RCKT) stock price.
The Rocket Pharma stock price soared 37.41% to trade at $21.01, rising from Tuesday’s closing price of $15.29.
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