Osteoporosis-Pseudoglioma Syndrome

ROCHESTER, Mich.--(BUSINESS WIRE)-- Caeregen Therapeutics announced today that it has been awarded a $1.4 million Phase 2 Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH) and National Eye Institute to advance the development of CTR-107 (Noregen™), a novel regenerative therapeutic for the treatment of retinal-related vision loss due to a wide range of inherited and age-related diseases. Caeregen is initially developing CTR-107 for the treatment of a rare inherited disorder of retinal blood vessel formation, Familial Exudative Vitreoretinopathy (FEVR). “The NIH grant will support the nonclinical development program of CTR-107 and accelerate our investigational new drug (IND) submission to begin clinical trials," said Walter Capone, CEO of Caeregen Therapeutics. “This award provides further, important validation for CTR-107 and our novel approach to retinal disease treatment, the first with the potential to restore retinal function and reverse vision loss,” said Dr. Kimberly Drenser, Chief Scientific Officer of Caeregen Therapeutics. Retinal disease is the leading cause of vision loss in developed countries and is the fastest growing cause of blindness globally. Currently, it is estimated that vision-depriving retinal diseases, such as macular degeneration, diabetic retinopathy, retinal vein occlusion, and ischemia affect more than 10 million people in the United States and Europe, respectively, and more than 50 million people worldwide1,2,3. Less common, but equally devastating causes of retina-related vision loss are driven by genetic alterations or mutations that impair normal cellular formation, or retinal structure or function, including Norrie Disease, Osteoporosis Pseudoglioma Syndrome, and FEVR. Both age-related and acquired retinal disorders share an underlying deterioration of the retinal vascular bed and related neurovascular structure. Current therapies slow the progression of vision loss but do not repair, nor reverse, retinal damage that has already occurred. CTR-107 is a synthetic targeted growth factor that mimics the properties of norrin, a naturally occurring human protein that promotes the development of organized blood vessels and neurons in the human eye, ear, and central nervous system. When injected into the eye, CTR-107 may regenerate retinal blood vessels and neurons, restoring retinal function and counteracting vision loss due to FEVR and other retinal diseases. The Phase 2 SBIR grant builds on prior research, including in a Phase 1 STTR grant, that demonstrated production feasibility, safety/tolerability, modulation of target gene expression in vitro, and in vivo functional activity. During Phase 2 SBIR grant-supported research, the company will optimize dosing through in vitro and in vivo studies, further develop analytical methods for product characterization, and conduct pharmacokinetic studies to support an IND submission. CTR-107 is the first program to obtain U.S. FDA Rare Pediatric Disease (RPD) Designation for treatment of FEVR, a designation that makes Caeregen eligible to obtain a Priority Review Voucher at the time of marketing approval. The RPD designation for CTR-107 (Noregen™) follows the Orphan Drug Designations by both the FDA and European Commission European Medicines Agency (EMA). About CTR-107 (Noregen™) CTR-107 (Noregen™) is a synthetic targeted growth-factor for retina-related vision loss modeled after norrin, a naturally occurring human protein that guides retinal formation in fetal development. CTR-107 may promote development of organized blood vessels and neurons in the human eye, ear, and central nervous system for individuals with inherited or acquired retinal diseases and potential applications in other neurosensory disorders. As a first-in-class therapeutic candidate to regenerate and repair the retinal vasculature to restore and preserve vision in patients with ischemic vitreoretinopathies, CTR-107 is currently being developed for Familial Exudative Vitreoretinopathy (FEVR), a rare disease of urgent, unmet medical need. About FAMILIAL EXUDATIVE VITREORETINOPATHY (FEVR) Familial Exudative Vitreoretinopathy, or ‘FEVR’ is a rare inherited disorder of retinal angiogenesis, leading to incomplete vascularization of the peripheral retinal and poor vascular differentiation. FEVR most often presents in childhood but may progress at any age with sight-threatening manifestations4 It is characterized by abnormal retinal vascular development with progressive vitreoretinal features, including retinal capillary dropout, vessel dragging; retinal folds, vessel leakage and exudation; hemorrhage, neovascularization, vitreoretinal interface changes, and serous, tractional, or combined retinal detachment5 - all contributing to or causing visual impairment and blindness. Currently there are no approved pharmacological therapies for FEVR. About Caeregen Therapeutics Caeregen Therapeutics, LLC, based in Rochester, MI with offices in Chapel Hill, N.C., is a regenerative medicines company developing therapeutics for neurosensory diseases. By exploiting biological pathways and signaling related to cellular and organ development, Caeregen is focused on advancing targeted therapies with the ability to repair, restore and protect neurosensory tissues affected by inherited or acquired diseases. Caeregen is currently developing Noregen, a unique, novel, recombinant protein mimetic of human norrin-derived growth factor for the potential treatment of retinal-related vision loss. For more information, visit: . 1Prevalence of Age-Related Macular Degeneration in the US in 2019”, JAMA Ophthalmology, Dec. 1, 2022 2Global epidemiology of retinal vein occlusion: a systematic review and meta-analysis of prevalence, incidence and risk factors”, Global Health, June 2019 3Global Prevalence of Diabetic Retinopathy and Projection of Burden through 2045: A Systematic Review and Meta-Analysis,” Ophthalmology, November 2021 4Clinical presentation of Familial Exudative Vitreoretinopathy,” Ophthalmology, October 2011 5Symmetry of Folds in FEVR: a genotype-phenotype correlation study,” Experimental Eye Research, September 2019