Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) has announced its Q3 2020 financial results and also provided updates on its key pipeline developments, corporate operations, and anticipated milestones.
Rocket advancing five clinical programs
Gaurav Shah, the company’s CEO, stated that the company made steady progress across its five clinical programs in Q3. Shah said that data from the RP-L201 study for LAD-I showed potential for a healthy and attractive profile for its “Process B” lentiviral gene therapy pipeline. During the quarter, the company treated its first higher dose patient in Phase 1 RP-A501 study for Danon Disease treatment with compelling preclinical data presented in IMO.
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Despite the pandemic, which resulted in delays in data collection and patient follow up, the company continued enrolling and treating patients in the AAV and LVV therapy programs and expand clinical studies globally. The CEO confirmed that the company made substantial progress in the build-out of its AAV R&D and manufacturing facility, and they anticipate the production of GMP products in 2021.
The company expects to share additional updates in Q4 2020, which include Phase 1 and Phase 2 study for FA ‘Process B’ data. The updates will also include Phase 1 clinical data from the company’s two indications, RP-A501 for Danon Disease and RP-L301 for PKD. Shah said that the company is getting closer to its goal of taking drug products to Biologics Licence Applications submission and launch to help patients with these rare and debilitating childhood diseases.
RP-A501 received Fast Track Designation
Recently Rocket presented for encouraging clinical updates for its Leucocyte Adhesion Deficiency Program (LAD-I) at the 2020 European Society for Immunodeficiencies Meeting. The company also presented preclinical data from the Infantile Malignant Osteoporosis program at the meeting. During the quarter, Rocket also treated the first patient at a higher dose in phase 1 RP-A501 dose-escalation trial in Danon disease after FDA and Independent Data Safety and Monitoring Committee clearance to advance to higher dose cohort. The company also received Fast Track Designation for RP-L401 gene therapy and Rare Paediatric Disease Designation for RP-A501.