New License for CFTR Activators and Inhibitors

Vanda Pharmaceuticals (VNDA), UCSF Enter License Agreement for CFTR Activators and Inhibitors

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.

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