ANN ARBOR — The pharmaceutical startup RetroSense Therapeutics LLC Monday announced the first patient in a Phase I/IIa clinical trial of its lead drug candidate, RST-001.
The drug is designed to restore some vision to patients with retinitis pigmentosa, a genetic condition that leads to the progressive degeneration of photoreceptors in the back of the eye informally called rods and cones, resulting in severe vision loss and blindness.
“Successfully dosing our first patient with RST-001 represents a significant milestone in the development program,” said Sean Ainsworth, RetroSense CEO. “Treating the first human patients should provide key insights into the potential for optogenetics in vision restoration, and beyond. We hope to establish the power of using a gene therapy application of optogenetics to improve vision in individuals with currently incurable blindness.”
Added David G. Birch, Ph.D., chief scientist and executive officer of the Retina Foundation of the Southwest and the principal study investigator: “Patients enrolling in the trial understand that we are exploring brand new territory but are excited about the possibility of restoring some vision.”
The initiation of this clinical study results from the culmination of several years of research and collaboration with researchers at leading institutions including Zhuo-Hua Pan, Ph.D, at the Ligon Center in Wayne State University’s Kresge Eye Institute and Richard Masland, Ph.D. at Massachusetts Eye and Ear Infirmary, early pioneers in optogenetics for vision restoration.
The study is composed of two parts. An initial dose-ranging study is proposed whereby three dose levels of RST-001 will be studied in three separate groups of adult patients with advanced disease. This first part of the study is aimed at determining a single dose of the experimental agent which is safe and well tolerated, to further evaluate in a fourth group of patients. The second part of the study is aimed at obtaining additional safety data at the highest tolerated dose and providing important additional clinical data to guide the design of future efficacy studies.
In August 2015, the Company’s Investigational New Drug application for RST-001 received clearance from the United States Food and Drug Administration. The clinical trial tracker is NCT02556736. For more information on the clinical trial, visit http://www.clinicaltrials.gov.
In Phase 1 trials, researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. In Phase 2 trials, the drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety. And in Phase 3, the drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
RetroSense Therapeutics is developing RST-001 as a gene therapy application of optogenetics, a means of conferring light sensitivity to cells that were not previously, or natively, light sensitive. By applying optogenetics to retinas in which rod and cone photoreceptors have degenerated, RetroSense is working to confer new light sensitivity to the retina, with the expectation of some degree of improved or restored vision for affected patients.
In 2014, the FDA granted Orphan Drug designation for RST-001 based on its development as a treatment of RP, a rare disease that affects an estimated 100,000 people in the United States. As a designated Orphan Drug, RST-001 is eligible for various development incentives under the Orphan Drug Act, including a potential waiver from FDA’s application user fees, certain tax incentives and Orphan Drug exclusivity.
There are currently no FDA-approved drugs to improve or restore vision in patients with RP. RetroSense has worldwide exclusive rights to the intellectual property on this drug approach from both Wayne State University and Massachusetts General Hospital. RetroSense is led by a team of seasoned veterans with deep experience in taking products from the discovery stage through to the clinic. For more information visit http://www.retro-sense.com.
The trial is being conducted at the Retina Foundation of the Southwest, located in Dallas, Texas, is a nonprofit eye research institute dedicated to preventing and restoring vision loss through innovative research and treatment. The Foundation is comprised of seven laboratories and a staff of scientists who to strive to improve the quality of life for people with retina related eye diseases.