Wellstat projects making an NDA submission for uridine triacetate in 2013.
BTG obtained U.S. marketing and distribution rights to Wellstat Therapeutics’ investigational drug uridine triacetate (PN401, formerly known as vistonuridine), for the treatment of accidental overexposure to the chemotherapy drug fluorouracil (5-FU) resulting from dosing errors or impaired clearance from the body.
In return for U.S. marketing rights to the drug for the 5-FU overexposure indication BTG will pay Wellstat an up-front fee, milestones on NDA acceptance and approval, and royalties on net sales in the U.S.
Wellstat retains all commercialization rights to uridine triacetate for the 5-FU overexposure indication in all non-U.S. markets, and for all other potential uses of the drug worldwide. The firm will also be responsible for completing development and regulatory filings, and for manufacturing uridine triacetate. It anticipates filing an NDA with FDA during the first half of 2013 for the 5-FU overexposure indication. In the meantime, uridine triacetate will continue to be available to patients in the U.S. under emergency-use INDs and an expanded-access protocol.
The firm is separately developing uridine triacetate for use in combination with 5-FU against a range of cancer types. The firm has completed a Phase II study in collaboration with the Southwest Oncology Group evaluating uridine triacetate combined with 5-FU in gastric cancer patients. Data from the single-arm study demonstrated improvement in the therapeutic index of 5-FU, alongside improved survival and lower toxicity. A Phase III pancreatic cancer trial evaluating uridine triacetate and high-dose 5-FU has also been completed, and additional studies are being planned.
Clinical trials are also planned to evaluate uridine triacetate in patients with mitochondrial diseases. Wellstat says the drug has already been provided to mitochondrial disease patients on a compassionate-use basis. The compound has also demonstrated activity in models of Alzheimer disease, Huntington disease, and Parkinson disease, and other neurometabolic orders that involve mitochondrial dysfunction.
Wellstat’s clinical-stage pipeline includes an insulin sensitizer, PN2034, which has completed two Phase II trials in type 2 diabetes patients, and has been exclusively licensed to sanofi-aventis for development, manufacture, and commercialization worldwide.