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Aegerion Pharmaceuticals, Inc. (AEGR) Message Board

  • hankmanoo hankmanoo Oct 11, 2013 9:35 AM Flag

    Hybridan on PLI regarding orphan drugs. Part III

    3.1 ProMetic Life Sciences Inc.
    ProMetic Life Sciences (PLI) is tackling the affordability and efficiency theme through its two distinct businesses – Protein Technology and Small Molecule Therapeutics. PLI’s Plasma Protein Purification System (PPPS TM ) is creating the next generation of plasma drugs. In value terms, PPPS TM ’s superiority across all types of plasma proteins represents at least a seven fold improvement over the industry average of $360/litre. This is game changer for the plasma drugs industry which, in many respects, is still a niche market focused on a narrow range of products/diseases and limited to the developed world. ProMetic’s proprietary process provides for superior extraction and recovery capabilities enabling the industry to address rare diseases and unmet medical needs. Due to the nature of the process, some of the rarest and most valuable proteins can be accessed at unprecedented levels. The recovery of such proteins allows for the generation of orphan drug opportunities targeting rare diseases with greater efficiency. PPPS TM has the ability to make plasma proteins a mainstream industry by making it more accessible to emerging markets as well as meeting the demand of increasingly value conscious healthcare buyers in the developed economies.
    The Protein Technology business has formed various collaborations primarily with Asian pharmaceutical companies, and development efforts so far have generated two orphan drugs - plasminogen drug for the treatment of hypoplasminogenemia, or type I plasminogen deficiency and Alpha1-antitrypsin (AAT) for AAT deficiency. These drugs are expected to be manufactured and commercialised at PLI’s own PPPS TM facility in Laval and Hematech’s future PPPS TM facility in Taipei. AAT has been approved by the FDA for various orphan indications; however the market has grown by a CAGR of just 16% since 2000 to reach $750m in 2012. At a price point of €70,000 in the EU and $120,000 in the US, only a minority of sufferers are being treated. With a much improved recovery yield, PLI can create a much larger market for AAT not just in the US/EU, but worldwide.
    The lead programme in the Small Therapeutics business is PBI-4050, which has shown anti-fibriotic activity in several indications including Chronic Kidney Diseases (CKD) and Idiopathic Pulmonary Fibrosis (IPF). Addressable markets are large, twenty six million patients in the U.S. alone are diagnosed with CKD. IPF is an orphan disease with potential sales value of $6bn in the US and Europe. New pre- clinical data presented at the 2013 annual European Respiratory Society congress in Barcelona shows that PBI-4050 performed favourably compared with Pirfenidone, the only commercially approved drug for IPF.

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