Benzinga | Jan 11, 2022 07:09AM EST

Rocket Pharmaceuticals Highlights Anticipated 2022 Milestones Across Lentiviral And AAV Gene Therapy Clinical Programs

-- Top-line data from pivotal LAD-I and Fanconi Anemia trials anticipated in Q2 and Q3, respectively --

-- Danon Disease pediatric patient cohort data expected Q3 --

-- In-house AAV GMP manufacturing initiation anticipated in Q2 --

-- Danon Disease pivotal Phase 2 trial initiation planned for Q4 --

-- PKD data from Phase 1 and pivotal Phase 2 trial initiation planned for Q4 --

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today shares expected milestones for 2022, which were discussed yesterday during the Company's virtual presentation at the 40th Annual J.P. Morgan Healthcare Conference.

"The Rocket team together with our partners made significant progress across our pipeline of first-and best-in-class lentiviral and AAV gene therapies in 2021," said Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer of Rocket Pharma. "We are excited to share our expected milestones for 2022 as we progress toward potential near-term U.S. and EU regulatory filings for our Leukocyte Adhesion Deficiency-I (LAD-I) and Fanconi Anemia programs as well as continue our strong progress across our pipeline. Notably, with the most recently treated patient now engrafting with neutrophil CD18 expression of 61% at three months, all nine severe LAD-I patients who have been treated with RP-L201 in our pivotal Phase 2 trial with at least three months of follow-up have engrafted with CD18 expression ranging from 26% to 87% of normal."

Dr. Patel continued, "As our AAV-based gene therapy for Danon Disease advances in the clinic including anticipated data from our pediatric cohort in Q3, we continue to ready our in-house AAV manufacturing capabilities in our Cranbury, New Jersey facility to support commercial product. We are excited for the year ahead and look forward to continuing to leverage Rocket's strong team and resources to drive our vision of seeking and delivering gene therapy cures for patients facing such rare and devastating diseases."

Anticipated 2022 Milestones

RP-A501 for Danon Disease (AAV)

* Report data from pediatric patient cohort of Phase 1 trial -- Q3 2022

* Initiate Phase 2 pivotal study activities -- Q4 2022

RP-L102 for Fanconi Anemia (LVV)

* Report top-line data from pivotal Phase 2 trial -- Q3 2022

RP-L201 for Leukocyte Adhesion Deficiency-I (LVV)

* Report top-line data from pivotal Phase 2 trial -- Q2 2022

RP-L301 for Pyruvate Kinase Deficiency (LVV)

* Report data from Phase 1 trial -- Q4 2022

* Initiate pivotal Phase 2 trial -- Q4 2022

Manufacturing Facility in Cranbury, New Jersey

* Achieve in-house AAV Good Manufacturing Practice (GMP) manufacturing -- Q2 2022

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Company's platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket's first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit www.rocketpharma.com.