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Vanda and UCSF Announce License Agreement for CFTR Activators and Inhibitors

WASHINGTON, March 29, 2017 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (NASDAQ: VNDA) announced today that it has entered into a license agreement with UC San Francisco (UCSF), under which Vanda will acquire an exclusive worldwide license from UCSF to develop and commercialize a portfolio of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) activators and inhibitors.

CFTR activators and inhibitors may have broad applicability in addressing a number of high unmet medical needs, including chronic dry eye, constipation, polycystic kidney disease, cholestasis and secretory diarrheas. This portfolio of CFTR activators and inhibitors was developed in the UCSF laboratory of Alan S. Verkman, M.D., Ph.D. The portfolio of compounds are at a pre-investigational new drug (IND) stage where in addition to ongoing chemistry optimization work, several lead compounds have been identified which are ready for further IND-enabling work.

"The licensing of the CFTR portfolio of activators and inhibitors is an important milestone for Vanda, as we continue to realize our vision of developing treatments to address unmet medical needs. The recently published animal model data from Dr. Verkman's team related to dry eye, constipation, polycystic kidney disease and diarrhea indications demonstrates the broad potential applicability of these compounds," said Mihael H. Polymeropoulos, M.D., President and CEO of Vanda.

"I am excited to be working with Vanda to advance the CFTR activators and inhibitors into the clinic. While CFTR modulators have been recently approved for the treatment of certain mutations in cystic fibrosis, the work we have done suggests these unique activators and inhibitors of wild-type, non-mutated CFTR could have utility in a number of prevalent conditions, including some where there is currently no available therapy," said Verkman, professor of medicine and of physiology, and director of the Cystic Fibrosis Research Development Program at UCSF.

This licensing agreement was negotiated with UCSF's Office of Technology Management (OTM). The OTM leads UCSF's licensing and business development efforts that translate cutting-edge science into therapies and products that directly benefit patients worldwide.

Under the terms of the agreement, Vanda will pay UCSF an initial license fee of $1 million and will be responsible for all development costs. UCSF is eligible to receive up to $46 million in potential development, regulatory and sales milestones as well as single-digit tiered royalties on net sales should a product be successfully commercialized.

In 2017, Vanda intends to complete the technology transfer activities from UCSF and initiate IND enabling studies for several CFTR indications.

About CFTR

The Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein functions as a channel for the movement of chloride ions in and out of cells, which is important for salt and water balance on epithelial surfaces, such as in the lungs, intestines and the surface of the eye.

The only currently FDA-approved therapies to target CFTR in humans are KALYDECO® (ivacaftor) and its combination with lumacaftor (ORKAMBI®), both made by Vertex. The CFTR potentiator therapy ivacaftor was developed to treat cystic fibrosis (CF) patients who have one of the following mutations in their CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. The combination of ivacaftor and lumacaftor was developed to treat CF patients who have two copies of the F508del mutation in their CFTR gene.

CFTR modulators that do not target specific rare mutations are expected to have utility in a variety of conditions in patients who have a disorder related to either impairment of or excessive fluid secretion across epithelial surfaces. In these disorders, CFTR activators have the ability to enhance fluid flow while CFTR inhibitors restrict fluid flow, in both cases helping restoring balance of fluids across membranes.

As CFTR activators increase water excretion into the extracellular space they may be beneficial in treating a number of conditions such as dry eye(1,2), cholestasis, constipation(3), and some lung conditions such as COPD, among others. Work in Verkman's lab demonstrated preliminary in vivo efficacy for certain compounds in both dry eye and constipation models, and work is ongoing in identifying and validating additional CFTR activator indications.

As CFTR inhibitors decrease water excretion across epithelia, such as where aberrant CFTR activation occurs, they may be useful in the treatment of cholera, traveler's diarrhea(4), polycystic kidney disease(5), and other conditions of water hyper-excretion. To date, Verkman's work has shown evidence of blocking cholera and E. coli-toxin-induced water excretion as well as activity in models of polycystic kidney disease.

About the UCSF CFTR Modulator Portfolio

The CFTR portfolio consists of patents and recently filed application families describing CFTR activators and inhibitors, some of which have proof-of-principle efficacy data in relevant in vivo disease models and which possess high affinities for CFTR.  

About Vanda:
Vanda is a global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients.
For more on Vanda Pharmaceuticals, please visit

About UCSF
UCSF is the nation's leading university exclusively focused on health. UCSF is dedicated to transforming health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care. It includes top-ranked graduate schools of dentistry, medicine, nursing and pharmacy; a graduate division with world-renowned programs in the biological sciences, a preeminent biomedical research enterprise and top-tier hospitals, UCSF Medical Center and UCSF Benioff Children's Hospitals. For more on UCSF, please visit


  1. Lee, S., P.W. Phuan, C.M. Felix, J.A. Tan, M.H. Levin and A.S. Verkman (2017). Nanomolar-potency aminophenyl-1,3,5-triazine activators of the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel for prosecretory therapy of dry eye diseases.  J. Med. Chem. 60:1210-1218.
  2. Flores, A.M., S.D. Casey, C.M. Felix, P.W. Phuan, A.S. Verkman and M.H. Levin (2016). Small-molecule CFTR activators increase tear secretion and prevent experimental dry eye disease.  Faseb J. 30:1789-1797.
  3. Cil, O., P.W. Phuan, J.H. Son, J.S. Zhu, C.K. Ku, N.A. Tabib, A.P. Teuthorn, L. Ferrera, N.C. Zachos, R. Lin, L.J. Galietta, M. Donowitz, M.J. Kurth and A.S. Verkman (2017). Phenylquinoxalone CFTR activator as potential pro-secretory therapy for constipation.  Transl. Res. 182:14-26.
  4. Cil, O., P.W. Phuan, A.M. Gillespie, S. Lee, L. Tradtrantip, J. Yin, M. Tse, N.C. Zachos, R. Yin, M. Donowitz and A.S. Verkman (2017). Benzopyrimido-pyrrolo-oxazine-dione CFTR inhibitor (R)-BPO-27 for antisecretory therapy of diarrheas caused by bacterial enterotoxins.  Faseb J. 31:743-750.
  5. Snyder, D., L. Tradtrantip, C. Yao, M.J. Kurth and A.S. Verkman (2011).  Potent, metabolically stable pyrimido-pyrrolo-quinoxolindione CFTR inhibitor for polycystic kidney disease.  J. Med. Chem. 54:5468-5477.



Jim Kelly

Executive Vice President & Chief Financial Officer

Vanda Pharmaceuticals Inc.

(202) 734-3428


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