Chemocentryx Inc., of Mountain View, Calif., said the FDA granted orphan drug designation for CCX168, an oral inhibitor that targets the receptor for the complement protein known as C5a, for the treatment of antineutrophil cytoplasmic autoantibodies (ANCA)-associated vasculitides (granulomatosis with polyangiitis or Wegener's granulomatosis, microscopic polyangiitis and Churg-Strauss syndrome). CCX168 currently is in the third step of the phase II CLEAR trial in patients with ANCA-associated vasculitis.
Foamix Ltd., of Rehovot, Israel, inked a collaboration with the National Eye Institute (NEI) to develop treatments for geographic atrophy (GA), with the focus on developing an effective therapy for GA using Foamix's stable topical Oilgel formulation containing minocycline, an antibiotic in the tetracycline family that also has anti-inflammatory, antioxidant, anti-apoptotic and neuro-protective properties. NEI will participate with Foamix in the development of an investigational new drug application and the refinement of a protocol in support of a potential clinical trial of minocycline gel for GA.
GW Pharmaceuticals plc, of London, disclosed an agreement with New York state to initiate clinical trials using cannabidiol (CBD) to treat children diagnosed with epilepsy who suffer from seizures and other medical complications. New York will be the second state in the U.S. to initiate trials for CBD in children who have not been treated successfully by other methods.
Hemispherx Biopharma Inc., of Philadelphia, said it confirmed that its Alferon N inhibits replication of the MERS virus in vitro. Data showed that Calu-3 cells treated with Alferon N 24 hours prior to infection with MERS coronavirus demonstrated significant dose-dependent inhibitor effects, suggesting the potential of the drug as a preventive therapy. Hemispherx provided the Alferon N for the study, which was run at the University of Texas Medical Branch at Galveston.
Hyperion Therapeutics Inc., of Brisbane, Calif., said the validation period is complete for its new drug submission to Health Canada for Ravicti (glycerol phenylbutyrate) oral liquid for the treatment of urea cycle disorders (UCDs). Ravicti was granted priority review, which is expected to shorten the review time to about six months. The drug gained approval last year in the U.S. for treating UCDs. (See BioWorld Today, Feb. 4, 2013.)
Imaginab Inc., of Los Angeles, and the Regents of the University of California, Los Angeles, said they executed a technology licensing agreement relating to immune cell-targeting agents for imaging with positron emission tomography. Under the terms, Imaginab gains exclusive access to imaging agents that target specific markers of murine T cells, enabling a new understanding of response to immunotherapeutic drugs in preclinical models.
Sirona Biochem Corp., of Vancouver, British Columbia, said licensee Wanbang Biopharmaceuticals Ltd., of China, started preclinical validation for Sirona's second-generation SGLT2 inhibitor. To date, Sirona has received Wanbang's licensing fee, the Chinese government's innovation tax application has been approved and Wanbang has successfully hired an expert in pharmacology and toxicity to lead the project. Compound synthesis has begun to ensure quantities of the compound will be available for all future clinical trials. Wanbang previously secured exclusive rights to the SGLT2 inhibitor in China for $9.5 million in up-front and milestone payments, with ongoing royalty payments as a percentage of all sales.
Theravance Inc., of South San Francisco, and Theravance Biopharma Inc., also of South San Francisco, said they completed the separation of their respective businesses, with Theravance Biopharma assuming the R&D biopharmaceutical business. Theravance will operate as a royalty management company holding rights to respiratory assets partnered with London-based Glaxosmithkline plc, under the terms of the separation, disclosed last year. Shares of Theravance (NASDAQ:THRX) fell $5.55, or 19.4 percent, to close Tuesday at $23.10. Shares of Theravance Biopharma, which began trading Tuesday on Nasdaq under the ticker TBPH, ended the day at $23.51. (See BioWorld Today, April 30, 2013.)
Thrasos Therapeutics Inc., of Montreal, presented preclinical data showing that lead compound THR-184, a small peptide designed to act on the BMP/Smad pathway, can protect against loss of kidney function following acute ischemic injury in rats. Results, presented at the European Renal Association – European Dialysis and Transplant Congress in Amsterdam, showed that THR-184 significantly decreased the severity of acute kidney injury (AKI) in an animal model, with the highest level of efficacy seen when the compound was administered both prior to and following the injury. THR-184 previously demonstrated safety and tolerability in phase I testing and was granted fast track status in AKI earlier this year.
Vanda Pharmaceuticals Inc., of Washington, said its marketing authorization application for oral Hetlioz (tasimelteon) capsules was accepted for evaluation by the EMA for the treatment of non-24-hour sleep-wake disorder. Hetlioz previously was granted orphan drug status in Europe. It gained FDA approval in January. (See BioWorld Today, Feb. 3, 2014.)