- Demographics and baseline characteristics comparable to patient population in first Phase III study -
LAVAL, QC, May 10, 2012 /CNW/ - BELLUS Health Inc. (TSX: BLU) ("BELLUS Health" or the "Company") today announced a study update and preliminary blinded data on patient demographics and baseline characteristics for the ongoing Phase III trial to confirm the safety and efficacy of KIACTATM (eprodisate) in the treatment of AA amyloidosis. Bellus' strategic partner for KIACTA™, Celtic Therapeutics, provided the update as part of its participation in the XIII International Symposium on Amyloidosis at the University Medical Center of Groningen in The Netherlands.
"Preliminary, blinded data from our ongoing Phase III confirmatory trial demonstrates that the baseline characteristics and demographics of the 78 patients profiled to date are very similar to the first Phase III study we completed," said Dr. Denis Garceau, Global Project Leader for Celtic Therapeutics for the KIACTA™ Phase III trial, and Senior Vice President, Drug Development of Bellus Health. "These data confirm that we constructed an effective trial design based on the strong results of our first Phase III study. We have enrolled 90 patients so far and expect to complete this process by the second half of next year."
The Phase III study is an event-driven trial that is being conducted in approximately 28 countries, across 79 sites. The current trial is being run to confirm the results of the prior Phase III clinical trial, which demonstrated statistically significant primary efficacy endpoints (p value = 0.025) and a clean safety profile. The study will conclude when 120 events are attained.
Improvement in Design of Phase III Confirmatory Trial
|First Phase III Study||Phase III Confirmatory Study|
|Duration||Two years||120 events|
|Number of patients||183||230|
|Entry criteria|| Proteinuria >1g/d or CrCl <60 ml/min
CrCl >20 ml/min
| Proteinuria >1g/d
CrCl >25 ml/min
|P value||0.01 (power ~ 80%)||0.05 (power ~90%)|
| Primary Composite Endpoint
(Time to earliest event)
| 100% increase in SCr; or
50% decrease in CrCl; or
| Persistent 80% increase in SCr; or
Persistent 40% decrease in CrCl; or
The design of the confirmatory study has been improved based on the information gathered from the first KIACTA™ Phase III study. The duration of the trial has been changed from a fixed two-year duration to an event-driven trial. The data from the first study was used in calculating the number of events (120 events) required to provide an approximate 90% statistical power (versus 80% in first study), assuming a comparable effect of KIACTA™ treatment. Considering that this Phase III confirmatory study will be the second pivotal study to support a KIACTA™ NDA application, the US and European regulatory agencies agreed that the p-value threshold for a successful study could be increased to 0.05 from 0.01 in the first study. The entry criteria regarding renal function was also changed slightly to enrol patients with high proteinuria, which is the patient subgroup that benefited most from KIACTA™ in the first study. Finally, the components of the primary endpoints were changed to improve the accuracy, sensitivity and validity of the primary efficacy endpoint.
About BELLUS Health (www.bellushealth.com)
BELLUS Health is a development-focused healthcare company concentrating on products that provide innovative health solutions and address critical unmet medical needs. The Company, in collaboration with its partner, Celtic Therapeutics, is currently advancing its novel drug candidate KIACTA™, through a Phase III trial for the treatment of AA amyloidosis, an orphan indication resulting in renal dysfunction that often rapidly leads to dialysis and eventually death. AA amyloidosis affects approximately 50,000 individuals in the United States, Europe and Japan.
Forward Looking Statements
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. Such statements, based as they are on the current expectations of management, inherently involve numerous risks and uncertainties, known and unknown, many of which are beyond BELLUS Health Inc.'s control. Such risks include but are not limited to: the ability to obtain financing immediately in current markets, the impact of general economic conditions, general conditions in the pharmaceutical and/or nutraceutical industry, changes in the regulatory environment in the jurisdictions in which the BELLUS Health Inc. does business, stock market volatility, fluctuations in costs, and changes to the competitive environment due to consolidation, achievement of forecasted burn rate, achievement of forecasted clinical trial milestones, and that actual results may vary once the final and quality-controlled verification of data and analyses has been completed. Consequently, actual future results may differ materially from the anticipated results expressed in the forward-looking statements. The reader should not place undue reliance, if any, on any forward-looking statements included in this news release. These statements speak only as of the date made and BELLUS Health Inc. is under no obligation and disavows any intention to update or revise such statements as a result of any event, circumstances or otherwise, unless required by applicable legislation or regulation. Please see the Company's public fillings including the Annual Information Form of BELLUS Health Inc. for further risk factors that might affect the Company and its business.
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