Here at Stinson Brand Innovation, we're always on the look-out for novel ideas that accelerate the development of new medical treatments. So we are intrigued by what Swedish biotech company, Karo Bio, is doing with its potential cholesterol game-changer. Their problem: raising $500 million for testing to gain FDA approval.
Their solution: Karo has developed is enlisting the help of noted cardiologist and FDA advisor, Steven Nissen. With Nissen's help, Karo is working to recruit a big drugmaker to finance clinical trials, and to convince the FDA of a clinical trial that could cut the usual approval time in half.
This new drug, eprotirome, works in the liver to clear cholesterol from the body, in contrast to Lipitor, the largest selling cholesterol drug, which blocks the liver enzyme needed to produce cholesterol. Upon approval, eprotirome could reach sales over $1.3 billion a year.
The proposed expedited approval plan includes testing on more patients for less time and money. The goal is to push through the approval process in less than 3 years, saving nearly $200 million. With assets of only $35 million, however, Karo still needs a big pharma partnership. The biggest obstacles to achieving this goal come in the form of Lipitor's looming 2011 patent expiration, and the doubts many healthcare professionals have about fast-track drug approvals. The real task is determining if the drug's benefits of outweigh the risks of a fast-tracked approval.
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In addition, KARO BIO issued a release that it has chosen a niche indication with large unmet need for Phase III clinical development of Eprotirome.
Here are some excerpts:
Swedish biotech company Karo Bio AB (Reuters: KARO.ST) today announced the decision to initially develop its leading lipid-lowering candidate drug eprotirome for the treatment of high-risk patients with heterozygous familial hypercholesterolemia (HeFH).
“There is today a large unmet medical need. HeFH patients are insufficiently treated and there is currently no treatment on the market with these patients as the primary target group. We have received a very positive response for the choice of this indication from key opinion leaders, regulatory authorities in Europe, and potential industrial partners. The path we have now chosen aims to bring eprotirome to the market with reasonable development costs and a limited risk”, commented Fredrik Lindgren, President and CEO.
HeFH is a hereditary condition where patients suffer from high blood lipid levels already in early years. The prevalence is estimated to between 1 of 350 to 1 of 500 people globally. In total, it is Karo Bio’s estimation that there are about 3 million patients in mature markets and growth markets, with 1 million patients in the EU alone. Today, the degree of diagnosis (mainly through clinical diagnosis) of HeFH is about 15%, which is expected to grow substantially when existing gene tests and DNA mapping are becoming more recurrent. Like other dyslipidemia patients, HeFH patients are mainly treated with statins. Only about 20% of the patients reach their treatment goals.
Eprotirome (formerly KB2115) has in several clinical phase II studies shown to have a very compelling effect profile for the lowering of blood lipid levels. The compound has the potential to provide for the market’s need for new, innovative pharmaceuticals to be given as add-ons to statins and other existing blood lipid lowerers. Utmost, eprotirome is addressing a market of tens of millions of patients, for whom statins and other blood lipid lowerers are not sufficient to reach to the treatment goals set up by the physicians.
In financial terms, the prioritization of HeFH means that the clinical phase III program will be an investment of tens of million USD all the way up to an application of a registration of the drug, instead of the hundreds of million USD that would be needed for a primary care indication. The size of Karo Bio’s investment is dependent on the degree of involvement from potential industrial partners. Regarding the potential revenues from the drug when it has reached the market, the company estimates that eprotirome can reach annual revenues of up to a billion USD per annum solely for the HeFH indication. Karo Bio estimates that it will suffice with a limited market organization in order to offer eprotirome to the specialist market that treats HeFH patients.
”At least on some geographical markets, it is realistic to believe that Karo Bio alone can market the product. This does not however prevent us from taking new contacts with potential collaboration partners for the HeFH indication. Primarily, we look for partners with a strong interest for the European market. We are also turning to partners active on other geographical markets, e g India. The purpose is then to find a partner that has the capabilities to carry out a part of the phase III program on its own market – thereby reducing our investment – and thereafter market the product”, said Fredrik Lindgren.
The fact that Karo Bio has decided to concentrate the upcoming clinical phase III studies on a so called niche indication does not in any way mean that eprotirome’s path towards a broad indication would be closed.
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