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Auven and Bellus complete pivotal Phase 3 trial of Kiacta forr the treatment of orphan disease AA Amyloidosis
ST. THOMAS, U.S. Virgin Islands, LAUSANNE, Switzerland, HAMILTON, Bermuda and LAVAL, Quebec—Auven Therapeutics, a global private equity company focused on accelerated development of breakthrough therapeutic drugs, and Bellus Health Inc., a drug development company focused on rare diseases, have announced the completion of the Kiacta (eprodisate) Phase 3 confirmatory study for the treatment of AA amyloidosis. Top-line results are expected to be announced in Q2 2016 after all remaining patients have completed final study visits, all queries have been resolved based on input from study sites and the database has been locked.
AA amyloidosis is a rare disease secondary to severe chronic inflammation or infection leading to the formation and deposition of amyloid fibrils in organs, often resulting in end-stage renal disease and death. Currently there are no therapies available that target the disease directly. In prior clinical studies, Kiacta has been shown to slow the decline of renal function in AA amyloidosis patients by its ability to interfere with the formation of amyloid fibrils A and the deposition of these fibrils in tissues.
“We designed this trial to confirm the results of the prior Phase 2/3 study in which Kiacta demonstrated significant delays in AA amyloidosis disease progression, in some cases for a number of years,” said Dr. Peter B. Corr, co-founder and managing general partner of Auven. “We are very pleased to complete this step in our pivotal study and look forward to reviewing and announcing the top-line results during the second quarter of 2016.”
The five-year Phase 3 study completed enrollment in January 2015 with a total of 261 patients. In January 2016, the event-driven study met its completion target of 120 patient events linked to the deterioration of kidney function. As all remaining patients have a final study visit in the coming weeks, the final number of events could increase.
Assuming that this Phase 3 study achieves its primary endpoint, it will support global regulatory approvals for Kiacta for the treatment of AA amyloidosis. Preclinical data also suggests Kiacta has potential to treat other diseases, including sarcoidosis. In vitro study test results in sarcoidosis indicate Kiacta may reduce SAA-induced inflammatory cytokine expression and the initiation of a Phase 2b/3 study in this second orphan indication is expected in 2016.
“The Kiacta Phase 3 confirmatory study is the most comprehensive study ever conducted in AA amyloidosis patients,” said Roberto Bellini, president and CEO of BELLUS Health. “We are excited that a key portion of the study is complete and look forward to receiving the top-line data.”
“We anticipate this trial will demonstrate conclusively Kiacta’s potential to make a major impact in the lives of those who currently have no other treatment options available to them, and we look forward to commencing a formal sale process for Kiacta later this year,” said Stephen Evans-Freke, co-funder and managing general partner of Auven.
Kiacta is an oral therapy in development for the treatment of AA amyloidosis, an orphan disease for which there is no currently approved therapy available. AA amyloidosis is a progressive, severe and potentially fatal condition that affects people with chronic inflammatory diseases. Chronic inflammation and/or infection cause amyloid deposits accumulating within internal organs resulting in potential organ failure over time. Kiacta has been studied for its ability to slow the decline of renal function in AA amyloidosis patients by interfering with the formation of amyloid fibrils and the deposition of these fibrils in tissues. Kiacta has received Orphan Drug Status in the United States for AA amyloidosis and Orphan Medicinal Product designation for AA amyloidosis in Europe and Japan.
Kiacta was originally developed by BELLUS Health. Auven Therapeutics acquired worldwide rights related to Kiacta from BELLUS Health in 2010 and is responsible for conducting and financing the Kiacta development program. Auven Therapeutics and BELLUS Health expect to share overall proceeds from a Kiacta divestiture equally, assuming that total divestment transaction proceeds reach a pre-determined threshold. Proceeds will be shared between Auven Therapeutics and BELLUS Health based on a formula that provides for Auven Therapeutics to have certain preference rights on exit proceeds related to their investment costs in Kiacta.
Auven Therapeutics is a global private equity firm that acquires and pursues accelerated development of breakthrough therapeutic drugs prior to licensing them to commercial partners. Auven’s in-house team of senior pharmaceutical development executives establishes the clinical, regulatory, manufacturing and commercial strategies for all its products and oversees their execution. Auven was founded in 2007 by Stephen Evans-Freke and Dr. Peter B. Corr and maintains operations in Lausanne, London, Ft. Lauderdale, Bermuda, and the U.S. Virgin Islands.
BELLUS Health is a drug development company focused on rare diseases. It has a portfolio of rare disease projects including Kiacta in Phase 3 for AA amyloidosis, Kiacta for sarcoidosis, clinical stage Shigamab™ for STEC-related Hemolytic Uremic Syndrome (sHUS) and a research-stage project for AL amyloidosis.