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Biocrea to Develop a Human Neuron Screening Platform for the Discovery of Novel Treatments for Neurodegenerative Diseases With Neuroscientists From the Dresden University of Technology

4/22/2013 9:52:50 AM

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Radebeul, Germany, April 22, 2013 – BioCrea GmbH, the first-in-class CNS drug discovery specialists validated through pharma partnerships, today announced the signing of a new collaboration with the Department of Neurology at Dresden University of Technology (TU Dresden) to discover new treatments for neurodegeneative diseases such as Amyotrophic Lateral Sclerosis (ALS; also called Lou Gehrig’s disease). Under the agreement BioCrea will work on multiple induced pluripotent stem cell (iPSC) lines derived from patients with genetic mutations, which have recently shown links to the familial form of ALS. These IPSCs will be jointly developed with the TU Dresden. This collaboration is a gateway for new phenotypic drug discovery approaches that can target the pathological mechanisms underpinning this devastating disease.

Despite many decades of research, target-based drug discovery approaches have failed to provide any new treatment options for ALS patients. Many reasons have been offered for this dilemma, the major reason is most likely the lack of a human model of the disease. Through this collaboration BioCrea will implement a novel approach to drug discovery in ALS based on the novel human cell model of the disease using the iPSC approach (See John B. Gurdon and Shinya Yamanaka, Nobel Prize, 2012). BioCrea’s ‘patient-to-screen’ approach will generate neurons from these iPSCs for use in phenotypic screening methods. In this way, the Company will identify novel compounds that can be developed into new drugs for the treatment of ALS, a severely disabling and progressive disease that often causes death within two years of diagnosis.

Tom Kronbach, CEO of BioCrea, commented: “ALS will be our initial focus; this is a stepping stone to other neurodegenerative diseases like Frontotemporal dementia and Parkinson’s disease.” He also noted that: “We have a long track record in the use of in vivo phenotypic screening methods to discover new medicines and two of these molecules have reached market authorization. Over the past 10 years, these techniques have proven to be most successful in the discovery of ‘first-in-class’ drugs (doi:10.1038/nrd3480). It is our strategy to use stem cells as patient-derived reagents in order to take this approach even further. Through the use of these new technologies at an in vitro lab scale we will be able to focus on human disease mechanisms from the outset of our drug discovery efforts. This, combined with our use of the latest medicinal -chemistry technologies, will allow us to improve the overall timelines for the delivery of more effective and innovative medicines.”

About Amyotrophic Lateral Sclerosis (ALS)

ALS is a motor neuron disease characterized by marked and progressive degeneration of the upper and lower motor neurons that typically leads to mortality within 1-5 years after first symptoms appear. It is characterized by progressive weakness, muscle atrophy and fasciculation, spasticity, dysarthria and dysphagia and at later stages occurring respiration failure or pneumonia. Whereas the cause is not known in the majority of patients diagnosed with this disease (sporadic form of ALS) up to 10% have a known genetic background (familial form of ALS). Encouragingly, the recent genes identified that are linked to ALS are beginning to point towards the existence of conserved cellular mechanisms that may underpin the familial and sporadic forms of the disease. Since these disease mechanisms are so far poorly understood, phenotypic drug discovery approaches are considered the best approach to yield new therapies that provide benefit for a wide range of ALS patients.

About BioCrea

BioCrea is the Radebeul-based first-in-class drug discovery specialist validated through pharma partnerships. The company boasts a provable track record in effective drug discovery driven by continuing discovery and optimization of selective ion channel and enzyme modulators – agents that offer significant potential for maximizing the efficacy and tolerability of new therapeutics. BioCrea was awarded Top Innovator status of the “Top 100” Most Innovative Companies in Germany, 2012 by compamedia GmbH.

About the Department of Neurology, Technische Universität Dresden (TU Dresden)

The current collaboration is with Prof. Alexander Storch (Deputy Director, Medical Faculty, Department of Neurology) and Dr. Dr. Andreas Hermann of the TU Dresden who have established track records in the fields of neurodegeneration, stem cells and ALS.

The TU Dresden is one of eleven German universities that were identified as an “excellence university” and is, today, the largest university in the state of Saxony. The excellence of the research at TU Dresden is also underlined by the positively evaluated projects of the "State Excellence Initiative", "The Leading-Edge Cluster Competition" and "The Centres for Innovation Competence". In addition, TU Dresden continuously proves its research competence in numerous other outside-funded research projects initiated by the public and the private sector.

Contact Dr. Tom Kronbach CEO, BioCrea GmbH Tel.: +49 351 / 4043 3332 Fax: +49 351 / 4043 3216

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