We have made important progress in our business plan in 2015 that has setup the coming year to be one of the most important in our Company history.
Developing Rare Disease Drugs is a Growing Trend
Regulatory agencies, and particularly the US Food and Drug Administration (FDA), are increasingly approving innovative drugs and in particular drugs that treat rare diseases. Last year, the FDA approved the greatest number of innovative drugs in over a decade. A large proportion of these drugs, 47%, were rare disease drugs.
KIACTATM Phase 3 Data Expected This Year
Over 5 years ago, together with our partner Auven Therapeutics, we embarked on a determined plan to conduct a second Phase 3 study for KIACTATM, a drug candidate for AA amyloidosis. The study, the largest ever conducted in AA amyloidosis patients, is now completed and we expect to release the top line data in the second quarter of 2016.
Key improvements made to the KIACTATM Phase 3 AA amyloidosis study:
A positive confirmatory study would be the basis for regulatory approval of KIACTATM in the United States and Europe. This would be an important success for BELLUS Health’s shareholders and just as importantly, provide access to a much needed treatment to AA amyloidosis patients that suffer from this deadly disease.
Rare Disease Pipeline Making Progress
The other rare disease projects in our pipeline have also taken important steps forward, including achieving the following important milestones:
- Met with the FDA regarding a Phase 2/3 study for KIACTATM targeting a second indication, a rare and sometimes lethal lung condition called Sarcoidosis;
- Presented two oral presentations and one poster presentation with clinical and preclinical data supporting the development of ShigamabTM for a rare kidney disease called STEC-related Hemolytic Uremic Syndrome at the VTEC Conference in Boston; and
- Regained the rights to a research-stage program for AL amyloidosis, another rare form of amyloid disease that mostly targets the heart and kidneys.
Strong Financial Stewardship
A key component to our business model is managing our cash resources efficiently. We finished 2015 with $9.7 million in cash reserves which, together with our low operational burn rate, provides us with enough cash to take us through the KIACTATM Phase 3 Confirmatory Study data and potential exit.
Focus on Shareholder Returns
Our passion is developing drugs for rare diseases while generating attractive financial returns for our shareholders. Our management team and our employees do outstanding work every single day striving to reach these objectives. They deal with a number of complex research, development, regulatory and business issues. The manner in which the team is able to face those daily challenges while continuing to advance our pipeline and maintain strong financial stewardship fills me with pride.
We believe in both the short term upside of our immediate projects and the long-term sustainability of our business plan. We continue to scout for new projects in which we can invest and build long term value for our shareholders.
On behalf of the entire BELLUS Health team I thank you for your continued support and look forward to working for you this year and for many to come.
President and Chief Executive Officer