Vanda gains IND approval for CFTR activator VSJ-110

Vanda Pharmaceuticals gains FDA approval on their IND for VSJ-110 to treat allergic conjunctivitis

November17th,2020
DDN Staff

WASHINGTON—News from Vanda Pharmaceuticals, Inc. notes that the U.S. Food and Drug Administration (FDA) has approved the company’s Investigational New Drug application to evaluate the cystic fibrosis transmembrane conductance regulator (CFTR) activator VSJ-110 (CFTRact-K267) for the treatment of allergic conjunctivitis.

Vanda is planning a Phase 2 study in human volunteers that will evaluate the acute anti-inflammatory effects of VSJ-110 in an ocular allergic challenge model. The study will also evaluate prosecretory effects, using standard tear production assessments.

VSJ-110 has shown efficacy in a dry eye model, as well as anti-inflammatory properties in both in vitro and in vivo assays. It’s believed that CFTR activators and inhibitors could have broad applicability in addressing a number of disorders such as chronic dry eye, constipation, polycystic kidney disease, cholestasis and secretory diarrheas.

“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., president and CEO of Vanda Pharmaceuticals. “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.”

Vanda Pharmaceuticals entered into a license agreement with UCSF in 2017, and acquired an exclusive worldwide license to develop and commercialize a portfolio of CFTR activators and inhibitors, including VSJ-110.

Vanda hopes that the results from the Phase 2 study will help to guide development of VSJ-110 and potentially expand its ability to treat a variety of ocular inflammatory conditions — including dry eye, which has an estimated worldwide prevalence of 5-20%, with around 16 million affected in the U.S. alone.

“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,” noted Alan Verkman, M.D., Ph.D., professor of Medicine at UCSF.

Other potential indications include chronic inflammatory eye conditions, like atopic keratoconjunctivitis, that remain poorly addressed with the current treatment options.

“It is exciting to see Vanda pursue a unique and novel therapeutic mechanism of action to address common inflammatory eye conditions that impact such a significant number of patients,” added Julie Schallhorn, M.D., M.S., assistant professor of Ophthalmology at UCSF. “The mechanism of action of VSJ-110, that could address both the aqueous deficiency and inflammatory components of dry eye disease, has the potential to revolutionize how we care for dry eye patients.”

Vanda plans to initiate enrollment in the Phase 2 study by the end of 2020, and anticipates results from this study in 2021.

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