Rocket Pharmaceuticals (RCKT) investor relations material

Introduction and purpose

• FDA granted accelerated approval to KRESLADI, the first gene therapy for severe LAD-I in pediatric patients with biallelic ITGB2 mutations lacking an HLA-matched sibling donor.

• KRESLADI addresses a devastating ultra-rare pediatric disease with high early childhood mortality and high unmet medical need.

• Approval marks the first commercial product for the company and eligibility for a Rare Pediatric Disease Priority Review Voucher.

Details of approval or decision

• KRESLADI is approved for pediatric patients with severe LAD-I due to biallelic ITGB2 mutations lacking an HLA-matched sibling donor.

• Approval is under the FDA's accelerated pathway, based on biomarker restoration of neutrophil CD18 and CD11a expression; continued approval depends on confirmatory trials.

• All treated patients in the pivotal study survived without allogeneic transplant, with follow-up of 3.6–5.7 years.

• FDA granted a Rare Pediatric Disease Priority Review Voucher to incentivize further rare disease therapy development.

Impact on industry and stakeholders

• KRESLADI is the first and only gene therapy for severe LAD-I, setting a precedent for future gene therapies in rare diseases and marking a milestone for the primary immunodeficiency community.

• Families and clinicians gain a new treatment option for a disease with limited alternatives.

• The approval enables eligibility for a PRV, providing potential non-dilutive capital for pipeline advancement; the company may monetize the PRV to enhance financial flexibility.

• Commercialization will be phased, focusing on operational excellence and patient safety, with initial treatment volumes expected in the single digits annually.