KARO BIO CHOOSES A NICHE INDICATION WITH LARGE UNMET MEDICAL NEEDS FOR CLINICAL PHASE III DEVELOPMENT OF EPROTIROME

STOCKHOLM, July 7, 2010. The Swedish biotech company Karo Bio AB (publ) (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 of Karo Bio.

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.

”Since the HeFH patients constitute a smaller patient group, with a defined and unsufficiently treated medical need, we assess to perform a limited clinical phase III program. We are initially focusing on the EU, and have initiated a process with several European medical products agencies. Based on these contacts, together with the international recommendations in this area, we estimate a phase III program that includes some hundred patients during 12-18 months – compared to thousands of patients during several years for a broad primary care indication”, said Fredrik Lindgren.

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. It is a pharmaceutical market with sales of several billion USD per annum.

In reference to the medical risk potential, this however means that also eprotirome has to undergo extensive clinical phase III studies documenting its safety before it can get an approval for such broad use from the authorities, notably the American Food and Drug Administration (FDA). For a broad primary care indication, a clinical program including several thousands of patients for some years is probably needed, meaning a considerable investment of hundreds of million USD.

As a consequence, Karo Bio has updated its overall strategy in order to more clearly emphasize the possibilities to develop the pharmaceutical compounds in the company’s project portfolio firstly through niche indications, and thereafter, when the drug has established itself on the market, and further safety data have been generated, submit an application to use the drug for broader patient groups.

It remains for Karo Bio to anchor the study plans for the HeFH indication with the European authorities EMA, which is expected to be finalized during the second half of 2010, but in parallel, the company is already working with detail plans and preparatory activities in order to commence the clinical phase III program in 2011.
 
”Among other things, we are currently staffing the project, and have made some important recruitments for our clinical organization during the second quarter of 2010. We have also engaged Prof. Steven E. Nissen as the chairman of the steering committee of the clinical program, and Prof. John J.P. Kastelein as principal investigator. If everything goes according to plan, we should be able to submit the documentation for the product approval by the end of 2013 or early 2014”, commented Fredrik Lindgren.

Steven E. Nissen is Professor of Medicine and Chairman of the Department of Cardiovascular Medicine at the Cleveland Clinic in Ohio, US. John J.P. Kastelein is Professor of Medicine and Chairman of the Department of Vascular Medicine at the Academic Medical Center (AMC) in Amsterdam, Netherlands.

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.
 
”We will continue our discussions with the FDA in parallel with working out a concrete plan for the EU and potentially some other territories. Nothing has changed our view that eprotirome in a longer perspective will be a good and appreciated additional treatment of polygenic dyslipidemia. We estimate that the patent situation will mean that eprotirome has a good defense until at least 2026, which means that we have still have a fair amount of time ahead of us”, said Fredrik Lindgren.
For more information please contact:

Fredrik Lindgren, CEO
Tel: +46 705 61 61 77

Erika Söderberg Johnson, CFO
Tel: +46 707 20 48 20 

Karo Bio publishes this information in accordance with the Swedish Securities Markets Act and/or the Swedish Financial Instruments Trading Act. The information was published on July 7, 2010 at 08:30am CET. 

About Karo Bio
Karo Bio is a drug discovery and development company specializing in endocrinology and targeting nuclear receptors as target proteins for the development of novel pharmaceuticals. The company has a project portfolio with innovative molecules that primarily target dyslipidemia, CNS-disorders, inflammation, and women’s health. In these areas, there are significant market opportunities and a clear need for pharmaceuticals with new mechanisms of action. Karo Bio develops compounds aimed at treating broad patient populations up to clinical proof of concept before out-licensing. In therapeutic niche areas, Karo Bio has the capacity to bring selected compounds into late stage clinical development and, potentially, to the market. In addition to the proprietary projects, Karo Bio has three strategic collaborations with international pharmaceutical companies for development of innovative therapies for the treatment of common diseases. Karo Bio is listed on NASDAQ OMX Stockholm since 1998 (Reuters: KARO.ST).
This press release is also available online at: www.karobio.com (https://www.karobio.com/) and www.newsroom.cision.com (https://www.newsroom.cision.com)