Ophthotech Expands Gene Therapy Pipeline in Orphan Retinal Diseases with Exclusive Option for Novel Product Candidates to Treat Best Vitelliform Macular Dystrophy (Best Disease)

- Ophthotech Enters into Second Series of Gene Therapy Agreements with the University of Pennsylvania and the University of Florida for Exclusive Option for Novel Product Candidates to Treat Best Disease -

NEW YORK, NY, USA I October 31, 2018 I Ophthotech Corporation (NASDAQ:OPHT) announced today that it has entered into an exclusive option agreement with the University of Pennsylvania (Penn) and the University of Florida Research Foundation (UFRF) for rights to negotiate to acquire an exclusive global license to develop and commercialize novel adeno-associated virus (AAV) gene therapy product candidates for the treatment of Best vitelliform macular dystrophy, also known as Best disease. Best disease, which generally affects individuals in both eyes, is an orphan inherited degenerative retinal disease caused by mutations in the BEST1 gene. Preclinical anatomical proof-of-concept studies conducted by Penn and the University of Florida demonstrated promising results in a canine Best disease model. In addition to the exclusive option agreement, Ophthotech will sponsor research at Penn, facilitated by the Penn Center for Innovation (PCI), and the University of Florida to conduct preclinical and natural history studies of Best disease. Ophthotech plans to commence IND-enabling activities and based on current timelines expects to submit an Investigational New Drug Application (IND) to the U.S. Food and Drug Administration (FDA) by 2021.

“We are excited to strengthen our relationship with the team of distinguished scientists at the University of Pennsylvania and the University of Florida, to expand our gene therapy pipeline by adding rights to obtain the BEST1 gene therapy product candidates and to build upon our strategy to develop novel treatment options for patients with devastating retinal diseases,” stated Kourous A. Rezaei, M.D., Chief Medical Officer of Ophthotech.

In a scientific publication in the journal Proceedings of the National Academy of Sciences of the USA (PNAS), Karina E. Guziewicz, Ph.D., Research Assistant Professor of Ophthalmology, University of Pennsylvania School of Veterinary Medicine, and colleagues presented proof-of-concept studies revealing that a BEST1 gene therapy product candidate could reverse the vitelliform lesions in the preclinical canine disease model with distinct phenotypic similarities to human Best disease. This publication is entitled: “BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure” by Karina E. Guziewicz, Artur V. Cideciyan, William A. Beltran, András M. Komáromy, Valerie L. Dufour, Malgorzata Swider, Simone Iwabe, Alexander Sumaroka, Brian T. Kendrick, Gordon Ruthel, Vince A. Chiodo, Elise Héon, William W. Hauswirth, Samuel G. Jacobson, and Gustavo D. Aguirre. PNAS February 2018.

“We have developed a preclinical canine disease model with distinct phenotypic similarities to human Best disease. Our AAV based gene therapy achieved a clear and durable reversal of the retinal pathology, including vitelliform lesions and microdetachments, in our canine model,” stated Karina E. Guziewicz, Ph.D., Research Assistant Professor of Ophthalmology, University of Pennsylvania School of Veterinary Medicine.

Ophthotech estimates that approximately 10,000 individuals in the United States and the five major European markets have Best disease. Patients with Best disease develop an egg yolk-like vitelliform lesion in their macular region, which over time leads to macular atrophy and permanent loss of central vision. There is currently no FDA or European Medicines Agency approved therapy to treat this orphan inherited retinal disease.

“Entering into our third gene therapy collaboration with highly accomplished scientific leaders in the field of gene therapy and degenerative retinal diseases reinforces Ophthotech’s commitment to build a gene therapy portfolio in retinal diseases and create value for our shareholders,” stated Glenn P. Sblendorio, Chief Executive Officer and President of Ophthotech.

In June 2018, Ophthotech announced that it had entered into an exclusive global license agreement with UFRF and Penn for rights to develop and commercialize a novel AAV gene therapy product candidate for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), an orphan monogenic disease that is characterized by progressive and severe loss of vision leading to blindness. Based on current timelines and subject to regulatory review, Ophthotech expects to initiate a RHO-adRP Phase 1/2 clinical trial in 2020.

In February 2018, Ophthotech announced that it had entered into a collaboration with the University of Massachusetts Medical School to investigate novel gene therapies for retinal diseases utilizing a “minigene” therapy approach as well as novel gene delivery methods.

About Ophthotech Corporation

Ophthotech is a science-driven biopharmaceutical company specializing in the development of novel therapies to treat ophthalmic diseases, with a focus on age-related and orphan retinal diseases. For more information, please visit www.ophthotech.com

SOURCE: Ophthotech

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