Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) revealed in a statement that the U.S. FDA had cleared its Investigational New Drug application. Notably, the IND application aimed at acquiring official authority to begin trials on RP-A501. The drug candidate will be the first proprietary product under the adeno-associated viral vector (AAV) platform.
Phase 1 trials to involve the University of California
The AVV platform utilizes proprietary gene therapy technology that targets early developmental stage Danon disease. As per the company, the initiative is a joint project with the San Diego School of Medicine, University of California. Further, the program will conduct its Phase 1 trials at the institution. Also, Dr. Eric Adler (Director of Cardiac Transplant and Mechanical Circulatory Support at UC San Diego Health) will take the leading role in the delivering of results for Phase 1.
Notably, Phase 1 trials for RP-A501 will evaluate the preliminary efficacy as well as tolerability and safety of the product. Also, this will be the first study for the product performed in humans. This phase will, expectedly, launch in Q22019. Besides, the company will delve into the theory of Danon disease and case studies across the globe. Interestingly, the firm anticipates the move to culminate into vital literature resources to benefit researchers and practitioners.
Rocket is now part of NASDAQ Biotechnology Index
According to Gaurav Shah, M.D., CEO, and President of Rocket, Danon has devastating effects, especially to children. The disease progresses dangerously fast and often concludes in heart failure and subsequent death.
“We look forward to advancing this potentially curative treatment option for the patients and families affected by this life-threatening disease,” said Dr. Shah.
The CEO further asserted that enrolment for participants in Phase 1 trial studies would kick off any time this quarter. Interestingly, the successful commencement of the trials will expand Rocket’s pipeline to three products.
Notably, this is a great strategic move for the company regarding bringing value to stockholders. Notably, the firm joined the NASDAQ Biotechnology Index late last year to gain more market exposure. Therefore, the addition of more products to its pipeline will further raise Rocket’s stature in the industry.