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  • PIPELINE
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PIPELINE

Diversified pharmaceutical company with divisions
for novel medicines and complex generics

New Drug

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New Drug Pipeline Strategy

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Wilson’s Disease

Copper absorption, distribution and excretion in Normal (A)andWilson’s disease patient (B)(from MSD Manual)
Wilson’s Disease

Autosomal recessive genetic disorder: mutation of atp7bgene on Chromosome 13 fails to transport excess copper into bile. Excess copper builds up in the body, especially in the liver and the brain leading to liver cirrhosis, and neuropsychiatric symptoms.

Orphan disease prevalence is 1 in 30,000, Korea: 1,400 patients; Japan:3,500 patients; USA: 8,000 patients; EU5: 4,000 patients, China: 30,000 patients

Medical Unmet Needs

~50% patients: chronic liver cirrhosis (Beinhardtet al., Clin. Gastroenterol. Hepatol. 2014) ~13% patients: acute severe liver cirrhosis (the only option is livertransplantation) and major cause of death in 20-year observation (Beinhardtet al., Clin. Gastroenterol. Hepatol. 2014) No available drugs facilitating copper excretion into bile Currently marketed chelating drugs encounter frequent compliance issues with daily use of extremely high dose