CRANBURY, N.J.– Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces positive top-line safety and efficacy data from its Phase 2 pivotal trial for severe Leukocyte Adhesion Deficiency-I (LAD-I) at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).
“We are excited to present positive top-line safety and efficacy data from our Phase 2 pivotal trial for LAD-I today at ASGCT, representing a significant step forward in the development of RP-L201 for the treatment of LAD-I, one of the most aggressive and highly fatal immunodeficiencies ever characterized. Moreover, they point to the great possibilities lentiviral-based gene therapies can offer for patients with devastating diseases,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “At three to 24 months after RP-L201 infusion, all nine patients sustained stable CD18 expression (median: 56%) with no therapy-related serious adverse events.”
Dr. Shah continued, “We are also pleased to report 100% overall survival at 12-months post-infusion via Kaplan Meier estimate and a statistically significant reduction in all hospitalizations, infection- and inflammatory-related hospitalizations and prolonged hospitalizations for all nine LAD-I patients with three to 24 months of available follow-up. Data also shows evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities.”
“While allogeneic transplant options are available, they continue to be associated with considerable toxicity and today’s top-line safety and efficacy data point to the potential of RP-L201 to change the treatment paradigm for patients living with severe LAD-I,” said Dr. Shah. “These results are a testament to the LAD-I patient community and focus of the Rocket team on steady, reliable execution. Based on these results, we are initiating discussions with health authorities on filing plans and anticipate filings in the first half of 2023.”
Interim Results From an Ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)
The oral presentation includes efficacy and safety data (cut-off March 9, 2022) at three to 24 months of follow-up after RP-L201 infusion for all patients and overall survival data for seven patients at 12 months or longer after infusion. RP-L201 is Rocket’s ex-vivo lentiviral gene therapy candidate for severe LAD-I.
- All patients, aged five months to nine years, demonstrated sustained CD18 restoration and expression on more than 10% of neutrophils (range: 20%-87%, median: 56%).
- At one year, the overall survival without allogeneic hematopoietic stem cell transplantation across the cohort is 100% based on the Kaplan-Meier estimate.
- All patients demonstrated a statistically significant reduction in the rate of all-cause hospitalizations and severe infections, relative to pre-treatment.
- Evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities has been shown along with sustained phenotypic correction.
- The safety profile of RP-L201 has been highly favorable in all patients with no RP-L201-related serious adverse events to date.
- Adverse events related to other study procedures, including busulfan conditioning, have been previously disclosed and consistent with the safety profiles of those agents and procedures.
