Preliminary Results from Initial Patients in the Clinical Trial Demonstrate Safe Delivery of the CPCB-RPE1 Implant and Positive Impact on Retinal Structure and Function
MENLO PARK, CA, April 4 2018. Regenerative Patch Technologies LLC, a biotechnology company focused on the development of cell-based implant technology for the treatment of retinal diseases announced the publication of preliminary results from its Phase 1/2a clinical trial in dry age-related macular degeneration. The publication entitled, “A Bioengineered Retinal Pigment Epithelial Monolayer for Advanced Dry Age Related-Macular Degeneration” was published as the cover story in Science Translational Medicine today (stm.sciencemag.org).
The newly published report describes interim results in a Phase 1/2a clinical trial of a composite implant, referred to as CPCB-RPE1, for the potential treatment of advanced dry age-related macular degeneration. The first author of the publication and lead investigator on the clinical trial is Amir H. Kashani, MD, PhD, a clinician-scientist at the USC Roski Eye Institute at Keck Medicine of USC. The advanced dry form of age-related macular degeneration, also known as geographic atrophy, is the leading cause of legal blindness in adults affecting 10-20% of people in the United States over the age of 65. Currently, there are no FDA approved therapies for the treatment of geographic atrophy.
The publication by Dr. Kashani and his coauthors describes the CPCB-RPE1 implant, experience with its surgical implantation, and the successful delivery of the implant to the diseased portion of the retina in four subjects with very advanced disease. Furthermore, the report indicates safety of the implant for at least 120 days. The effects of the CPCB-RPE1 implant on the structure and function of the retina in the treated patients are also described. In subjects receiving CPCB-RPE1, integration of the implant with the photoreceptors, cells responsible for visual function in the retina, was noted. Reappearance of a structure resembling the external limiting membrane (ELM) in the diseased retina was observed. The ELM is a structure present in the normal retina and absent in the area of disease in the patients with geographic atrophy. These structural changes suggest that CPCB-RPE1 may promote restoration of retinal architecture, a feature critical for visual function.
Several assessments of visual function were also conducted on patients in the clinical trial. After CPCB-RPE1 implantation, patients demonstrated improved ability to focus or fixate on a target using the diseased area of the retina. Furthermore, patients receiving CPCB-RPE1 showed either stable or improved visual acuity. One patient in the trial demonstrated a 17 letter improvement in visual acuity during follow-up. Normally the vast majority of patients with geographic atrophy show a progressive decline in visual acuity over time.
“This is the first human trial of this novel stem cell–based implant, which is designed to replace a single-cell layer that degenerates in patients with dry age-related macular degeneration,” says lead author and surgeon for the study Amir H. Kashani, MD, PhD. “This implant has the potential to stop the progression of the disease or even improve patients’ vision. Proving its safety in humans is the first step in accomplishing that goal.”
CPCB-RPE1 is a bioengineered implant consisting of stem cell-derived, mature, polarized retinal pigment epithelial cells in a single layer on an ultrathin synthetic parylene membrane. The implant is designed to replace the retinal pigmented epithelium (RPE) and Bruch’s membrane which are critical functional elements of the eye that degenerate in dry age-related macular degeneration. CPCB-RPE1 is delivered to the back of the eye in an area known as the subretinal space during an outpatient surgical procedure. The implant is named after the California Project to Cure Blindness, a consortium of contributors from the University of Southern California, the California Institute of Technology, the University of California at Santa Barbara and the City of Hope, that developed and manufactured the implant. The parylene membrane technology was jointly developed by the University of Southern California and California Institute of Technology, and was manufactured at CamTek LLC, Pasadena CA.
“This potential treatment blends the stem cell technology used to produce the RPE cells with micro-electro-mechanical systems (MEMS) to produce a miniaturized implant that may replace degenerated tissue critical for function of the retina,” stated Jane Lebkowski, PhD, President of R&D at Regenerative Patch Technologies.
The Phase 1/2a clinical trial continues to enroll subjects with geographic atrophy and is being conducted at six different clinical trial sites in southern California and Arizona. Further information about the clinical trial can be found at https://clinicaltrials.gov/ct2/show/NCT02590692. Subjects in the trial will be followed for 1 year to assess visual acuity, visual field, and retinal function, with additional long-term in person safety follow-up for five years. Development of the CPCB-RPE1 implant and the current clinical trial was funded by the California Institute for Regenerative Medicine (CIRM).
“Our study shows that this unique stem cell–based retinal implant thus far is well-tolerated, and preliminary results suggest it may help people with advanced dry age-related macular degeneration,” says coauthor and lead inventor of the implant Mark S. Humayun, MD, PhD, director of the USC Institute for Biomedical Therapeutics, co-director of the USC Roski Eye Institute. “We are especially thankful to CIRM for their continuous support throughout the development of the CPCB-RPE1 implant".
About Regenerative Patch Technology LLC
Regenerative Patch Technologies LLC (RPT) is a California company developing cell-based implant technology for the treatment of retinal diseases. CPCB-RPE1 is RPT’s lead product. RPT was founded by Drs. Mark Humayun and David R. Hinton from the University of Southern California and Dr. Dennis O. Clegg from UC Santa Barbara. The technology to produce the CPCB-RPE1 implant is exclusively licensed to RPT from the University of Southern California, the California Institute of Technology and UC at Santa Barbara. Further information about the company can be found at www.regenerativepatch.com
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