BS-01

AbbVie recently returned the rights of eye drug Ab The first four patients that were administered BS01, Bionic Sight’s investigational gene therapy for advanced retinitis pigmentosa (RP), all reported the ability to detect light and motion. Two of the patients were also able to detect direction. Today Bionic Sight, LLC announced the preliminary results of their Phase I/II clinical trial. The first four patients that were administered BS01, Bionic Sight’s investigational gene therapy for advanced retinitis pigmentosa (RP), all reported the ability to detect light and motion. Two of the patients were also able to detect direction. The four subjects were reported to have either complete or near-complete blindness when the study was first started in March 2020 . “The key issue for producing meaningful vision is to send signals to the brain that the brain can understand. When the optic nerve communicates with the brain, it does it in a code,” said Dr. Sheila Nirenberg , the founder of Bionic Sight and professor of physiology and biophysics at the Weill Medical College of Cornell University. “The code is in the form of patterns of neural pulses that tell the brain what you’re looking at – faces, objects, a clear path ahead of you. If you can deliver signals in the language the brain understands, then meaningful perceptions become possible.” The BS01 therapy delivers optogenetic proteins to the optic nerve. These proteins act as a converter and translate light into neural signals that are then sent to the brain. Unlike other gene-specific AAV therapies for RP, BS01 is combined with a device developed by Niremberg using the retina’s neural code. The device, which is worn just like a pair of glasses, generates the vision-enabling code that is sent through the ganglion cells and optic nerve to the brain. Two to three months after the injection of BS01, the patients started reporting their observations and perceptions. This was tested objectively by asking the patients to look into a device and distinguish which stimuli are present- light or no light, motion moving to the left, or motion moving to the right. These stimuli were applied randomly and the perception of the patients was recorded by the use of buttons on a console. Todd Durham, Ph.D., Vice President of Clinical and Outcomes Research at the Foundation Fighting Blindness (FFB), noted that the “early observations reported by Bionic Sight are very encouraging. Bionic Sight’s innovative approach has the potential to make a substantial difference to the lives of patients with RP and other retinal degenerative diseases.” Dr. Nirenberg admits that the sample size of their current study is still small, but emphasizes that “the consistency is very promising, with no safety concerns so far.” The next move for Bionic Sight is to continue the study using higher doses and a larger population.

Company shares preliminary results from Phase 1/2 clinical trial with patients who have complete or near-complete blindness NEW YORK, March 30, 2021 (GLOBE NEWSWIRE) -- Bionic Sight, LLC announced today that the first four patients that received BS01, an investigational gene therapy for advanced stage Retinitis Pigmentosa, are showing biological activity. The patients, all of whom have complete or near-complete blindness, can now see light and motion, and, in two cases, can detect the direction of motion. The patients began reporting observations about their perceptions 2-3 months after BS01 was injected, and then were tested objectively and quantitatively at a testing site at 3 month and 6 month time points. For the latter, the patients were asked to look into a device, similar to a virtual reality device, and distinguish among presented stimuli (e.g., light vs. no light, leftward motion vs rightward motion). The stimuli were presented automatically and randomly to prevent investigator or patient bias, and patients entered their responses by pressing buttons on a console. These results provide substantial evidence that BS01 is reaching the targeted cells and expressing the optogenetic protein at levels sufficient to produce visual responses, even at the lowest doses. The two patients that received the lowest dose showed more than a 20-fold increase in light sensitivity compared to their baseline sensitivity (pre-injection), and the two that received a dose three times higher showed more than a 100-fold increase. “While the number of patients is still small, the consistency is very promising, with no safety concerns so far,” said Dr. Sheila Nirenberg, the company’s founder and a professor at the Weill Medical College of Cornell University. “We’re looking forward to moving to the higher doses and plan to report additional results later this year.” Bionic Sight’s approach has two key strengths. It uses a sensitive optogenetic protein, and it activates the protein with a neural coding device. The device uses the retina’s neural code to convert what the patient is viewing into signals the brain can understand. This creates the potential for meaningful perceptions – not just about light and motion, but also about the environment, objects, and possibly faces, as the trial advances to higher doses. “These early observations reported by Bionic Sight are very encouraging,” said Todd Durham, PhD, Vice President of Clinical and Outcomes Research at the Foundation Fighting Blindness (FFB). “Bionic Sight’s innovative approach has the potential to make a substantial difference to the lives of patients with RP and other retinal degenerative diseases.” Bionic Sight’s Phase 1/2 trial (ClinicalTrials.gov identifier: NCT04278131) is an open-label, dose escalation study in patients with advanced-stage RP. Trial participants are being enrolled sequentially in four dose groups. The primary focus of the study is to assess the safety of BS01 through analysis of ocular or systemic treatment-emergent adverse events. Efficacy is measured by assessing the detection of light, motion, and shape/object recognition. About the Company BIONIC SIGHT is a biotech company that develops optogenetic gene therapy vectors and devices to treat retinal degenerative diseases. The company leverages the research of Dr. Sheila Nirenberg, the company’s founder and a professor at the Weill Medical College of Cornell University. Her work focuses on neuroscience and its applications to brain/machine interfaces and computer vision. Her work on deciphering the retina’s neural code has been described in TED talks, an NBC documentary, a Bloomberg documentary, the Discovery Channel, Scientific American, as well as peer-reviewed journals and patents. It also earned her a MacArthur “genius” award. For more information on the company, visit . IR and PR Contacts: Sheila Nirenberg Founder Bionic Sight, LLC snsheilan@gmail.com Adam Carte Business Development Bionic Sight, LLC acarte@fairleadadvisors.com