Vanda Receives FDA Approval to Proceed with VSJ-110 for Allergic Conjunctivitis

Vanda Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application to evaluate Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) activator VSJ-110 (previously known as CFTRact-K267) for the treatment of allergic conjunctivitis.

"This is an exciting milestone in the course of our collaboration with University of California, San Francisco (UCSF) and Dr. Alan Verkman towards the development of human therapeutics targeting the Cystic Fibrosis Transmembrane Conductance Regulator," said Mihael H. Polymeropoulos, M.D., Vanda's President and CEO.  "Initiation of the clinical program for VSJ-110 marks the beginning of Vanda's development of therapeutics in ophthalmology exploring the compound's novel dual anti-inflammatory and prosecretory mechanism of action."

The initial Phase II study in human volunteers will evaluate the acute anti-inflammatory effects of VSJ-110 in an ocular allergic challenge model, and will evaluate the prosecretory effects using standard tear production assessments.

The results from this Phase II study will help guide further development of VSJ-110 to treat a variety of ocular inflammatory conditions, including dry eye, which has an estimated worldwide prevalence of 5-20%, with about 16 million affected individuals in the United States. Other potential indications include chronic inflammatory eye conditions, such as atopic keratoconjunctivitis, which remain poorly addressed with current treatment options.

"Studies in experimental animal models already provide in vivo evidence for the efficacy of CFTRact-K267 (VSJ-110) in stimulating chloride secretion and reversing corneal epithelial injury in dry eye," said Alan Verkman, M.D., Ph.D., Professor of Medicine at UCSF.

Vanda plans to initiate enrollment in the Phase II study by the end of 2020 and anticipates results of this study in 2021.

VSJ-110 is a small molecule nanomolar potency CFTR activator. VSJ-110 has shown efficacy in a dry eye model3 and exhibited anti-inflammatory properties in both in vitro and in vivo assays.