Rocket Pharmaceuticals' IND for Gene Therapy for Immune Disorder Cleared by FDA

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  • Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2
  • The disease occurs in approximately 1 in 1 million births globally


Rocket Pharmaceuticals, Inc. (Nasdaq:RCKT) (“Rocket”) announced on 11/19/18 the clearance of the Company’s Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for RP-L201. RP-L201 is the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of severe Leukocyte Adhesion Deficiency-I (LAD-I) that was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT).

“This acceptance marks the second Rocket-Sponsored IND cleared for our ex-vivo lenti platform this quarter. We are excited to achieve this important milestone ahead of schedule, and look forward to working with the medical and patient communities to evaluate RP-L201 next year,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “This IND enables Rocket to initiate a Phase 1/2 clinical trial to assess the safety, tolerability, and efficacy of severe LAD-I in children. Severe LAD-I is a devastating pediatric disease and we believe that we may have an opportunity to significantly improve the lives of very young children suffering from this disease with a potentially curative therapy.”

The planned open-label, single-arm, Phase 1 portion of the clinical trial of RP-L201 is expected to enroll two severe LAD-I patients in the U.S. The Phase 2 portion of the clinical trial of RP-L201 is expected to be a registration-enabling global study with clinical sites in the U.S., U.K. and Spain.

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, life-threatening, autosomal recessive pediatric disease caused by a mutation of the ITGB2 gene that encodes for the Beta-2 Integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and enables neutrophil extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is exceedingly rare. The unmet medical need for patients with severe LAD-I is therefore significant. The disease occurs in 1 in 1 million births globally.

In addition to its LAD-I program, Rocket has five other therapies in development. The company completed its merger with Inotek Pharmaceuticals Corporation in January of 2018.