Company shares preliminary results from Phase 1/2 clinical trial for patients with advanced Retinitis Pigmentosa (RP)
NEW YORK, Feb. 18, 2025 (GLOBE NEWSWIRE) -- Bionic Sight, Inc., a company developing a novel gene therapy for retinal degenerative diseases, announced today that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational product BS01 for the treatment of retinitis pigmentosa (RP) patients with advanced stage vision loss.
RMAT designation is part of the 21st Century Cures Act, which aims to accelerate the development of regenerative medicine therapies, including gene therapy, for serious medical conditions. RMAT designation includes all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with FDA, but is specified for regenerative medicine therapies. Bionic Sight is now preparing its Phase 3 program, as it advances toward marketing approval.
“Receiving RMAT designation from the FDA is a significant validation of Bionic Sight’s BS01 therapy, given the intensity of the FDA review process on both safety and efficacy data,” said Calvin Roberts, MD, President and CEO of the Lighthouse Guild, an international organization for the blind. “The company has potentially game changing results for people with RP and the potential to have similar results for other retinal degenerative diseases as well.”
The RMAT designation for BS01 was based on the preliminary data from Bionic Sight’s ongoing first-in-human Phase 1/2 clinical trial. Briefly, the participants who received the lower doses showed a modest but consistent improvement: 5 of 5 showed an increase in light sensitivity of 100-fold or more, and several also gained the ability to detect motion. The participants who received the two highest doses showed a much more marked improvement. Ten (10) of 11 showed an increase in visual acuity (Best Corrected Visual Acuity (BCVA)), with 7 patients showing an increase that was measurable on the EDTRS eye chart. The average increase for these 7 patients was nearly 5 lines on the eye chart (4.7 lines). This gain was seen across a spectrum of patients -- from those whose baseline vision was at the “count fingers” level to those whose baseline was at “tunnel vision” (less than 10 degrees of visual field, described by the patients as looking through a paper towel roll).
In addition to the increase in BCVA, the “count fingers” patients gained the ability to recognize shapes, objects, and, in some cases, colors, and the tunnel vision patients showed both an increase in visual field size (a 3-fold increase in area) and a pronounced improvement in their ability to navigate a maze (measured as a reduction in the time to complete the maze and a reduction in the number of errors). The remaining 3 high dose patients were at the “light perception” level at baseline; these participants improved to “hand motion”. With respect to safety, BS01 was well-tolerated. No product-related serious events occurred; all events were grade 1 or 2 and generally asymptomatic.
Bionic Sight’s Phase 1/2 trial (ClinicalTrials.gov identifier: NCT04278131) is an ongoing open-label, dose escalation study in patients with advanced-stage RP. The primary focus of the study is to assess the safety of BS01 through analysis of ocular or systemic treatment-related adverse events. Efficacy is measured by assessing the detection of light, BCVA, shape/object/color recognition, and maze navigation.
About the Company
Bionic Sight, Inc. is a biotech company that develops gene therapy vectors and devices to treat retinal degenerative diseases. The company was founded by Dr. Sheila Nirenberg, a professor at the Weill Medical College of Cornell University, and builds on her lab’s work on retinal and neuro-degenerative diseases and computer vision. Her work on deciphering the retina’s neural code and using it to restore visual function earned her a MacArthur “genius” award. For more information on the company, visit https://www.bionicsightllc.com.
IR and PR Contacts:
Sheila Nirenberg, PhD
Founder
Bionic Sight, Inc.
snirenberg@bionic-sight.com
Adam Carte
Business Development
Bionic Sight, Inc.
acarte@fairleadadvisors.com
