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FDA approves Vanda Pharmaceuticals’ IND application for VSJ-110 for allergic conjunctivitis

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Vanda Pharmaceuticals announced that the 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,” Mihael H. Polymeropoulos, MD, Vanda’s President and CEO, said in a company news release. “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 2 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 2 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.1 Other potential indications include chronic inflammatory eye conditions, such as atopic keratoconjunctivitis, which remain poorly addressed with current treatment options.2

“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, MD, PhD, Professor of Medicine at UCSF.

“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,” said Julie Schallhorn, MD, MS, 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 of this study in 2021.

About VSJ-11

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.

Vanda entered into a license agreement with UCSF in 2017, under which Vanda acquired an exclusive worldwide license from UCSF to develop and commercialize a portfolio of CFTR activators and inhibitors, including VSJ-110. CFTR activators and inhibitors may have broad applicability in addressing a number of disorders, including chronic dry eye, constipation, polycystic kidney disease, cholestasis, and secretory diarrheas.


  1. Stapleton, F., Alves, M., Bunya, V. Y., Jalbert, I., Lekhanont, K., Malet, F., Na, K. S., Schaumberg, D., Uchino, M., Vehof, J., Viso, E., Vitale, S., & Jones, L. (2017). TFOS DEWS II Epidemiology Report. The ocular surface, 15(3), 334–365. https://doi.org/10.1016/j.jtos.2017.05.003
  2. Pedram, H., Reza, D. (2019, September). Vernal keratoconjunctivitis. UpToDate. https://www.uptodate.com/contents/vernal-keratoconjunctivitis.
  3. Chen, X., Lee, S., Zhang, T., Duan, T., Pasricha, N. D., Schallhorn, J. M., Levin, M. H., Koprivica, V., & Verkman, A. S. (2020). Nanomolar Potency Aminophenyltriazine CFTR Activator Reverses Corneal Epithelial Injury in a Mouse Model of Dry Eye. Journal of ocular pharmacology and therapeutics: the official journal of the Association for Ocular Pharmacology and Therapeutics, 36(3), 147–153. https://doi.org/10.1089/jop.2019.0087