SHARES IN Irish-American biotech company Amarin jumped more than 60 per cent yesterday as it produced trial data that suggested it could have a blockbuster cardiovascular drug on its hands.
Amarin published top-line results from the first of two phase III trials into the efficacy of the drug, AMR101, as a treatment for patients with very high triglycerides.
Triglycerides are a type of fat stored from food intake and released as energy. At normal levels, they are not a source of concern. However, at elevated levels, particularly above 500 milligrams per decilitre (mg/dl), they are considered to increase the risk of heart disease.
The trial showed that AMR101 was successful in significantly reducing triglyceride levels – particularly in patients with triglyceride readings above 750 mg/dl, where a 4gm dose led to a 45 per cent reduction compared to a placebo.
More significantly, the trial indicated that AMR101 did not increase another risk factor for heart disease – low-density lipoprotein (LDL-C), more commonly known as “bad” cholesterol. The competitor in the market for triglyceride therapy – GlaxosmithKline’s Lovaza – reported a 45 per cent increase in LDL-C in patients in its trials.
As a result of the data, Amarin chairman and chief executive Joseph Zakrzewski said the company would bring forward to next year a new drug application to market AMR101 in the United States. It had previously not expected to be in a position to do so until 2012.
“We believe that these results and the overall profile of AMR101 position the drug candidate to be best in class in this market,” Mr Zakrzewski added.
The Nasdaq-listed company also said it believed the results of the “marine” trial “may support additional patentable claims that could further protect the company’s rights to this product through 2030”.
In safety terms, the trials suggest no deviation from the placebo.
Rival Lovaza, which generates annual sales of close to $1 billion, loses patent protection at the end of 2013.
One of the additional findings was that patients already being treated with statins – drugs used to lower cholesterol – produced even better results. The world’s biggest-selling drug, Lipitor, is a statin, which sources close to the company pointed to as an indicator of potential.
The result of the trial was warmly welcomed by Fountain Healthcare, the Irish life sciences venture capital fund which last year organised a $70 million fundraising for the company.
Dr Manus Rogan, founder and managing partner of Fountain Healthcare, described the outcome as “an amazing success”.
“This is potentially a multibillion-dollar blockbuster,” he said, noting that in a sector where many key drugs, including Lipitor, were coming off patent shortly, “there will be a lot of interest” in AMR101’s data.
He refused to be drawn on whether the company and its investors were likely to seek a partner to develop and scale up the drug.
The result marks a significant turnaround for a company that almost foundered when a previous trial of the same drug as a therapy for Huntington’s disease failed after initial high expectations.
The company retrenched and redirected its focus. Dr Rogan said the result was an indication of what could happen when the best people got involved and put together top-tier internationally focused life science venture capital teams.“You try to remove as much of the risk as you can [in trials],” he said. “Sometimes, you just get lucky.”
The company will produce full data on the trial and an associated Anchor trial later.
