PLI’s strategy has attracted considerable investment from
various biopharmaceutical companies, particularly those based
in Asia, where the high cost of legacy technology has limited
demand. As the management of PLI pointed out at yesterday’s
Q2 results presentation, Asia Pacific uses less than 15% of the
IVIG supply today despite representing 60% of the world’s
Potential value of AAT sales
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.
1. What is AAT?
Alpha-1 antitrypsin is a protein made by cells in the liver. It passes out from the
liver into the bloodstream and can travel to the lungs. Its main function is to
protect the lungs from damage caused by other types of proteins called enzymes.
Enzymes are essential for the normal working and development of the body. In the
lungs, certain enzymes called proteases help to fight infection, by removing
bacteria and may also be released to try to protect the lungs from tobacco smoke.
However, the activity of these protease enzymes needs to be balanced. If the
balance tips and there is too much activity, then the tissue of the lungs can start to
become damaged by the enzymes. AAT helps to balance the protease enzymes in
the lungs and stop lung damage.
AAT deficiency is an inherited genetic condition. It can lead to lung and, in some
people, liver damage at any age. According to PLI, there is an estimated 100,000
people affected by AAT deficiency in the USA alone with less than 10% treated.
According to the Alpha-1 Foundation, there may be as many as 3% of the 20m
patients suffering from Chronic Obstructive Pulmonary Disease (COPD) that may
also have an undetect