Results Support Streamlined Regulatory Pathway for Novel Hypertriglyceridemia Drug
LAVAL, QUÉBEC--(Marketwired - Sept. 14, 2016) - Acasti Pharma (NASDAQ:ACST)(TSX VENTURE:APO) today announced
that its bridging study for novel drug candidate CaPre® (omega-3 phospholipid) has successfully met its objectives, supporting
Acasti's strategy to pursue the U.S. Food and Drug Administration's (FDA) 505(b)(2) regulatory pathway for approval. Acasti is
developing CaPre for the treatment of patients with severe hypertriglyceridemia, a metabolic condition that contributes to
increased risk of cardiovascular disease and pancreatitis. The 505(b)(2) regulatory pathway allows Acasti to streamline the
overall development program required to support a New Drug Application (NDA) by relying on the safety data of an approved
drug.
"We are confident that the results of this study support the 505(b)(2) regulatory pathway chosen by Acasti to gain marketing
approval of CaPre," said Jan D'Alvise, president and CEO of Acasti Pharma. "With this momentum, we look forward to working with
the FDA to confirm the pathway and optimize the design of our Phase 3 program, which will seek to demonstrate the safety and
efficacy of CaPre in patients with severe hypertriglyceridemia."
Acasti's open-label, randomized, four-way, cross-over, bioavailability study compared CaPre given as a single dose of 4 grams
in fasting and fed states with the approved hypertriglyceridemia drug LOVAZA (omega-3-acid ethyl esters) in 56 healthy
volunteers. The study met its primary objective and demonstrated that the levels of omega-3 fatty acids eicosapentaenoic acid
(EPA) and docosahexaenoic acid (DHA) following administration of CaPre did not exceed the levels following administration of
LOVAZA in subjects who were fed a high-fat meal. These results support the basis for claiming a comparable safety profile of the
two products.
Furthermore, among subjects in the fasting state, CaPre demonstrated better bioavailability than LOVAZA, as measured by blood
levels of EPA and DHA. As previously reported, the bioavailability of CaPre is not significantly reduced when taken with a
low-fat meal versus a high-fat meal. This could represent a significant clinical advantage for CaPre since the administration
with a low-fat meal represents a more attractive regimen for patients with hypertriglyceridemia who follow a restricted diet.
CaPre is a novel composition of omega-3 phospholipids sourced from krill. Its omega-3s, principally EPA and DHA, are naturally
either "free" or bound to phospholipids that help them to be better absorbed into the body. This allows for enhanced
bioavailability and EPA and DHA blood levels compared to the "esterified" fish-oil omega-3 options such as LOVAZA. CaPre is
designed to modulate the major lipids associated with cardio-metabolic disease: in two previously reported Phase 2 clinical
trials, CaPre reduced triglyceride levels, lowered non-high density lipoprotein (non-HDL-C, a useful marker of cardiovascular
disease), and increased levels of high density lipoprotein (HDL-C, or "good cholesterol") while having a neutral to positive
effect on lowering low density lipoprotein (LDL-C, or "bad cholesterol").
"The CaPre bioavailability study has reinforced the compound's unique attributes in comparison with the leading pharmaceutical
agent for hypertriglyceridemia," said Roderick Carter, M.D., chairman of Acasti Pharma. "CaPre demonstrates clinically meaningful
effects on many key markers of cardio-metabolic health. With high rates of obesity and diabetes fueling the number of patients
with elevated triglycerides and cholesterols, CaPre could fill the need for a best-in-class omega-3 medication that addresses the
full lipid profiles of these patients."
About CaPre
Sourced from krill oil, CaPre seeks to provide a full scope of health benefits to patients with hypertriglyceridemia, filling
a medical need that no other omega-3 treatment option has been able to address. CaPre successfully completed Phase 2 clinical
trials for the treatment of hypertriglyceridemia, a very common metabolic condition in which blood levels of triglycerides, a
type of lipid, are elevated, posing a risk to cardiovascular health. Severe hypertriglyceridemia, affecting more than 4 million
adults in the U.S. is associated with an increased risk of coronary artery disease and pancreatitis and is often caused or
exacerbated by uncontrolled diabetes mellitus, obesity and sedentary habits. CaPre is intended to be taken orally once per day in
capsule form.
About Acasti Pharma
Acasti Pharma is a biopharmaceutical innovator advancing a potentially best-in-class cardiovascular drug, CaPre, for the
treatment of hypertriglyceridemia, a chronic condition affecting an estimated one-third of the U.S. population. Since its
founding in 2008, Acasti Pharma has focused on addressing a critical market need for an effective, safe and well-absorbing
omega-3 therapeutic that can address patients' complete lipid profile, making a positive impact on all major lipids associated
with cardiovascular disease risk. For more information, visit www.acastipharma.com.
Forward Looking Statements
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Form, which also forms part of Acasti's latest annual report on Form 20-F, and which is available on SEDAR at www.sedar.com, on EDGAR at www.sec.gov/edgar.shtml and on the investor section of Acasti's website at acastipharma.com
(the "AIF"). All forward-looking statements in this press release are made as of the date of this press release. Acasti does not
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uncertainties that are described from time to time in Acasti's public securities filings with the Securities and Exchange
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