• Purchase of Assets and Related Global Commercialization Rights for Oral 9-cis-Retinol (Zuretinol) for Rare Forms of Childhood Blindness.
• Clinical Stage Asset Has Potential for First Approved Oral Therapy for Leber's Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP) Caused by Mutations of the RPE65 or Lecithin:Retinol Acyltransferase (LRAT) Gene.
• Program Has Received FDA Rare Pediatric Disease and Fast Track Designation and is Eligible for a Rare Pediatric Disease Priority Review Voucher.
• Future Applications of Zuretinol Include Treatment of Impaired Dark Adaptation (Night Blindness) in Adult Patients with Early Dry Age-related Macular Degeneration (AMD).

SUZHOU, China and SAN FRANCISCO, Jan. 19, 2022 /PRNewswire/ -- Eluminex Biosciences (Suzhou) Limited (Eluminex), an ophthalmology-focused biotechnology company headquartered in Suzhou, China with a US-subsidiary office in the San Francisco Bay Area, California, announced today that it has acquired certain assets and the related global development and commercialization rights for a novel oral therapy, zuretinol acetate (zuretinol), from Retinagenix Holdings, LLC (Retinagenix), a privately-held ophthalmic company based in Seattle, Washington. Zuretinol is an investigational treatment that is currently being developed to treat rare forms of childhood blindness in patients with LCA or RP caused by mutations of the RPE65 or LRAT gene.

"The addition of zuretinol into our growing retinal disease pipeline further bolsters our commitment towards the development of innovative therapies for vision-threatening diseases around the world," said Charles Semba, MD, Chief Medical Officer of Eluminex. "Currently, the only approved treatment for LCA and RP due to RPE65 and LRAT mutations is gene therapy which requires the child to undergo surgery and can treat only a small portion of the retina. Zuretinol offers hope in the ability to treat the entire retina and both eyes simultaneously either as a monotherapy treatment or adjunctive to gene therapy in restoring vision to these children and young adults."

Under terms of the agreement, Eluminex will make an upfront payment and earnout payments to Retinagenix for the purchase of the assets. The earnout payments to Retinagenix shall include (a) clinical, regulatory, and commercial milestone payments and (b) payments based upon worldwide net sales of products and the sale or use of priority review vouchers. The closing of this transaction is subject to certain customary conditions.

"Our focus at Retinagenix has been to see the zuretinol clinical program advance and for the drug to obtain regulatory approval in order to provide a safe, oral therapy for children and adults with these debilitating retinal degenerations. We believe Eluminex Biosciences is uniquely situated to complete the zuretinol development program and efficiently gain global regulatory approval for the compound," said David Saperstein, MD, Chief Medical Officer of Retinagenix.

About the Zuretinol Retinal Rare Pediatric Disease Program

The Eluminex zuretinol program (EB-109) is a clinical stage, novel, oral therapy for the treatment of a rare form of childhood blindness in patients with LCA or RP caused by mutations of the RPE65 or LRAT gene. EB-109 is regulated as a small molecule pharmaceutical product and is anticipated to enter a global Phase 2b/3 pediatric study in 2H 2022 to confirm its efficacy and safety. Orally administered zuretinol has been previously evaluated in 144 human subjects in 8 clinical studies, including healthy subjects, patients with recessive RPE65/LCA^[1],[2], autosomal dominant RPE65^[1],[2], and adults with early age-related macular degeneration (AMD) with poor night vision^[3], which has demonstrated that zuretinol appears safe and well-tolerated and can rapidly improve visual function.

The zuretinol program was granted Orphan Drug Designation by the United States Food and Drug Administration (FDA) and the European Medicine Agency (EMA). It has also received FDA Rare Pediatric Disease and Fast Track designation and is eligible for a Rare Pediatric Disease Priority Review Voucher (PRV) which is transferable and allows the holder Priority Review (6 months) instead of the standard 10 months for any future regulatory submission.