The company is set to initiate a Phase 2 clinical trial in the U.S. after a successful Phase 1 trial in Spain.

Rocket Pharmaceuticals, Inc., a U.S.-based biotechnology company, has recently announced the U.S. Phase 2 clinical advancement trial for RP-L102, the company’s LVV (lentiviral vector) based gene therapy for the therapy of FA (Fanconi Anemia).

According to reports, Rocket plans to begin enrollment for the U.S. Phase 2 clinical trial of RP-L102 for FA in the Q4 of 2019, based on receiving a feedback from the U.S. Food and Drug Administration (FDA) from the End-of-Phase 1 meeting.

Speaking on the announcement, Gaurav Shah, M.D., Chief Executive Officer and President, Rocket, said that the company’s End-of-Phase 1 meeting with the FDA was a crucial accomplishment for advancing the progress of RP-L102 for Fanconi Anemia.

He said that the company is thankful for the Agency’s guidance and collaboration on Phase 2 clinical trial design which will ensure an effective registration path to potentially market its first-in-class gene therapy for patients suffering from FA.

Moreover, relying on the totality of existing clinical evidence at the time of filing, MMC-resistance could provide a surrogate endpoint for quicker approval. Advancements that have enabled the company to further the development of their FA program more proficiently than expected, cites Shah.

Jonathan Schwartz, M.D., Chief Medical Officer and Senior Vice President, Rocket, said that the long-term and acute side effects accompanied by allogeneic stem cell transplantation, accentuates the immediate need for extra curative hematologic strategies in this dreadful pediatric disease.

He said that in the Spanish Phase 1 trial, the drug had shown promising results, with strong engraftment in the bone marrow and peripheral blood. Moreover, their firm has observed a growth in marrow and blood profiles, similar to those in FA patients whose blood-forming stem cells have suddenly reverted back to normal phenotype.

Source Credit: https://rocketpharmaceuticals.gcs-web.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-registration-enabling-phase-2