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Aegerion Pharmaceuticals, Inc. (AEGR) Message Board

  • hankmanoo hankmanoo Aug 14, 2013 10:04 AM Flag

    PLI announces another orphan drug

    From Beacon
    • ProMetic announced this morning that it plans to develop and commercialize Alpha 1-Antitrypsin (AAT) as its second plasma derived orphan drug product. This follows on the heels of plasminogen.
    • Using its proprietary PPPS technology (ie. its resin/filtration), ProMetic has the capability to capture AAT in much higher yields (+220%) than existing techniques
    • Typically, there are 1.3 grams of AAT per litre of plasma. Existing technology’s capture rate is 0.2 grams (15% capture rate). PLI has exhibited the capability to capture 0.7 grams (54% capture rate)
    • The net result is that PLI needs significantly less plasma to generate the same amount of AAT. This will make ProMetic the lowest cost producer of AAT by a wide margin.
    • AAT is used to treat AAT deficiency (Type 1). Symptoms could include shortness of breath similar to asthma.
    • There are an estimated 100,000 people affected by Type 1 AAT deficiency in the US alone.
    • Through the orphan drug designation, there is a clear pathway for re-imbursement, which stands at ~$120,000/year/patient. Given the estimated market of ~$750 million, that would imply that only ~7,000 of those 100,000 patients seek treatment.
    • Therefore, the company believes the actual market potential is much larger than $750 million as more patients seeks care.
    • Given its anticipated low-cost position, we believe revenue potential will only be capped by capacity. Through its Laval plant, and through Hematech’s Taiwan plant, management believes it will have capacity to generate revenue of $150+ million.
    • The timeline to production is that PLI anticipates filing the IND in 2014 with market entry in 2016/2017.. The FDA calls for a cross-over pharmacokinetic bioequivalence study (PPPS ATT versus a commercial brand).
    • This is the second announced orphan drug in ProMetic’s pipeline, after plasminogen.
    • As we have discussed, plasminogen (Type 1 deficiency) is a potential $60+ million market opportunity with an

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