Caeregen Therapeutics Receives $1.4 Million Phase 2 SBIR Grant from NIH/NEI to Advance Development of Noregen for Retinal Diseases

Caeregen Therapeutics announced 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 (NEI) to advance the development of CTR-107 (Noregen), a regenerative therapeutic for the treatment of retinal-related vision loss due to a 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.

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 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).