LAVAL, QC, Dec. 21, 2016 /CNW/ - BELLUS Health Inc. (TSX: BLU) (BELLUS Health or the Company), a drug development company focused on rare diseases, is providing an update on the projects in its pipeline.
- Auven Therapeutics, BELLUS Health's partner who acquired the rights to KIACTA™ from the Company in 2010, has decided to terminate the KIACTA™ program for the treatment of AA amyloidosis;
- Auven Therapeutics is evaluating the conduct of a Phase 2/3 study with KIACTA™ in sarcoidosis, a rare and sometimes lethal lung disease;
- BELLUS Health has received positive regulatory feedback from the FDA in regards to the design of a Phase 2 study for Shigamab™ in STEC related Hemolytic Uremic Syndrome (sHUS);
- Update on partnered drug candidates, including AMO-01 for the treatment of Fragile X Syndrome and ALZ-801 for the treatment of Alzheimer's Disease in APOE4 Homozygous patients.
"While the termination of the KIACTA™ AA amyloidosis program is not unexpected considering the negative Phase III study results released in June, we are still disappointed for the patients suffering from AA amyloidosis and their families," said Roberto Bellini, President and Chief Executive Officer of BELLUS Health. "We are encouraged that our partner Auven Therapeutics is evaluating a clinical study in sarcoidosis and together with Shigamab™ in sHUS, there is the potential to have two clinical studies started next year."
KIACTA™ for AA Amyloidosis
Top-line results from the KIACTA™ Phase 3 study for the treatment of AA amyloidosis were announced on June 20, 2016. In the study, KIACTA™ did not meet the primary efficacy endpoint in slowing renal function decline. As a result of the negative outcome of the Phase 3 clinical trial, Auven Therapeutics has decided to terminate the KIACTA™ program for the treatment of AA amyloidosis.
KIACTA™ for Sarcoidosis
Auven Therapeutics is currently evaluating the potential use of KIACTA™ for the treatment of patients suffering from active pulmonary sarcoidosis. Auven Therapeutics has developed a clinical Phase 2/3 study protocol to evaluate the safety and efficacy of KIACTA™ in sarcoidosis and an investigational new drug application (IND) for this clinical study could be filed with the U.S. Food and Drug Administration (FDA) in the first quarter of 2017. The sarcoidosis program is currently under review by Auven Therapeutics and a decision is expected to be made in the coming months.
Sarcoidosis is a rare condition that causes patches of red and swollen tissue - called granulomas - that can develop in multiple organs in the body, but mostly in the lungs and skin. There is no cure for chronic sarcoidosis, and treatment options are limited and can have serious adverse effects.
Shigamab™ for sHUS
BELLUS Health has received positive regulatory feedback from the FDA in relation to the clinical Phase 2 study protocol for the assessment of the efficacy and safety of Shigamab™ in the treatment of children suffering from sHUS. BELLUS Health is currently planning the next steps for the initiation of this clinical Phase 2 study and is seeking to secure a strategic partner for the further development of Shigamab™.
sHUS is a rare disease which principally affects the kidneys and often leads to acute dialysis, and in certain cases chronic kidney disease and death, primarily in children.
AMO-01 for Fragile X Syndrome
AMO Pharma Limited, the Company's licensee of AMO-01 for the treatment of neurologic and psychiatric disorders, is expected to initiate a Phase 2 study on patients with Fragile X Syndrome in the first half of 2017.
Fragile X Syndrome is the most common inherited cause of autism and intellectual disabilities, affecting approximately 1 in 4,000 males and 1 in 8,000 females. Symptoms range in severity and can include intellectual disabilities, attention deficit and hyperactivity, anxiety and seizures. There are currently no approved drugs indicated for the treatment of Fragile X Syndrome.
ALZ-801 for APOE4 Homozygous Alzheimer's Disease
ALZ-801 for the treatment of Alzheimer's disease (AD), initially developed by BELLUS Health and licensed to Alzheon Inc. (Alzheon) in 2013, is a prodrug of tramiprosate, a beta-amyloid targeting small molecule shown to improve cognition and function in AD patients who are carriers of apolipoprotein E4 (APOE4) AD genotype, and to reduce soluble beta-amyloid in the cerebral spinal fluid of AD patients.
Recent third-party positive clinical results for the treatment of early AD using anti-beta-amyloid antibodies lend further support to the concept of amyloid beta clearance as a promising approach for the treatment of AD.
Alzheon has completed two bridging Phase 1b clinical studies with ALZ-801 and is currently in preparation for a pivotal Phase 2/3 program focusing on treatment of mild AD patients who are homozygous for APOE4, the most important genetic risk factor for late-onset AD.
The Company continues to explore opportunities in order to expand its pipeline, including through acquisitions and/or in-licensing.
About BELLUS Health (www.bellushealth.com)
BELLUS Health is a drug development company focused on rare diseases. Its pipeline of rare disease projects includes KIACTA™ for sarcoidosis and clinical-stage Shigamab™ for sHUS. BELLUS Health is partnered with global private equity firm Auven Therapeutics for the development of KIACTA™. BELLUS Health also has economic interests in several other partnered drug development projects.
Certain statements contained in this news release, other than statements of fact that are independently verifiable at the date hereof, may constitute "forward-looking statements" within the meaning of Canadian securities legislation and regulations. Such statements, based as they are on the current expectations of management, inherently involve numerous important risks, uncertainties and assumptions, known and unknown, many of which are beyond BELLUS Health Inc.'s control. Such risks factors include but are not limited to: the ability to obtain financing, the impact of general economic conditions, general conditions in the pharmaceutical industry, changes in the regulatory environment in the jurisdictions in which BELLUS Health Inc. does business, stock market volatility, fluctuations in costs, changes to the competitive environment due to consolidation, achievement of forecasted burn rate, potential payments/outcomes in relation to indemnity agreements and contingent value rights, achievement of forecasted pre-clinical and clinical trial milestones, dependence on Auven Therapeutics for the development of KIACTA™ and that actual results may vary once the final and quality-controlled verification of data and analyses has been completed. In addition, the length of the KIACTA™ development process and the sharing of proceeds between Auven Therapeutics and BELLUS Health Inc. from potential future revenue of KIACTA™ are dependent upon a number of factors, including the quantum of proceeds. Consequently, actual future results and events may differ materially from the anticipated results and events expressed in the forward-looking statements. The Company believes that expectations represented by forward-looking statements are reasonable, yet there can be no assurance that such expectations will prove to be correct. The reader should not place undue reliance, if any, on any forward-looking statements included in this news release. These forward-looking statements speak only as of the date made, and BELLUS Health Inc. is under no obligation and disavows any intention to update publicly or revise such statements as a result of any new information, future event, circumstances or otherwise, unless required by applicable legislation or regulation. Please see BELLUS Health Inc.'s public filings with the Canadian securities regulatory authorities, including the Annual Information Form, for further risk factors that might affect BELLUS Health Inc. and its business.
SOURCE BELLUS Health Inc.