Rocket Pharma gets FDA green light for gene therapy trial, stock surges

Rocket Pharmaceuticals, FDA, gene therapy, Danon disease, trial design, stock price, RP-A501

Rocket Pharmaceuticals has achieved alignment with the FDA regarding the design of a pivotal phase 2 clinical trial for a rare disease, positioning the company to initiate a 12-patient study that may pave the way for accelerated approval of its gene therapy.

Based in New Jersey, Rocket Pharmaceuticals held an end-of-phase 1 meeting with the FDA in late 2022. Following the meeting, the biotech informed investors that the FDA had expressed a willingness to consider a biomarker-based composite endpoint and recognized the challenges of conducting a randomized controlled trial for Danon disease, the targeted rare disease. Rocket had initially outlined plans to initiate the initial phase of the study in the first half of 2023.

“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. I would also like to highlight the work conducted by our CMC team over the past several years to establish our in-house cGMP manufacturing capabilities, which has already provided us with sufficient material for the pivotal study and should support our eventual commercialization efforts.”

– Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma

However, throughout the year, the process of aligning with the FDA continued to progress slowly. Rocket has now successfully overcome this hurdle by devising a trial design that aligns with the FDA’s expectations. This development sent Rocket’s share price soaring by 30% to $20 in premarket trading on Wednesday.

The pivotal trial will be a single-arm, open-label study involving the enrollment of 12 patients who will receive RP-A501, an AAV9 gene therapy engineered to restore heart function by introducing a gene that is typically mutated in individuals with Danon disease. Rocket and the FDA have agreed to employ LAMP2 protein expression and left ventricular mass change from baseline as co-primary endpoints. These endpoints focus on measuring protein expression and assessing heart health.

Rocket intends to evaluate the co-primary endpoints at the 12-month mark and will continue monitoring patients using secondary endpoints such as event-free survival up to 24 months. The biotech is planning to submit an application to initiate the study in Europe during the third quarter of this year. Furthermore, enrollment is already underway for a two-patient pediatric safety run-in.

Capitalizing on the significant surge in its stock price, Rocket swiftly filed to issue new shares. Initially targeting a fundraising goal of $150 million, the biotech surpassed this mark, ultimately securing a price that will enable it to raise $175 million. Rocket concluded June with $307 million in its coffers, a substantial sum that it anticipates will fund its operations through 2025.

This significant milestone marks a promising step forward for Rocket Pharmaceuticals in its pursuit of an innovative gene therapy to address Danon disease, offering renewed hope to patients and investors alike.

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