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The Lancet Respiratory Medicine Publishes RAPID Extension Study Data Confirming Disease-modifying Effect of Respreeza® [Alpha1-Proteinase Inhibitor (Human)] in Patients with Alpha-1 Antitrypsin Deficiency (AATD)

Results show significantly reduced rate of lung density decline after switch from placebo to Respreeza® and highlight the importance of early intervention

MARBURG, Germany — 02 December 2016

Global biotherapeutics leader CSL Behring announced today that data from the RAPID Open Label Extension study, conducted in patients with alpha-1 antitrypsin deficiency (AATD), demonstrates that the use of Alpha1-Proteinase Inhibitor (A1-PI) therapy slows the progressive and irreversible loss of lung tissue, confirming that early intervention is beneficial.

"RAPID is a landmark study that provides evidence for the disease-modifying effect of A1-PI therapy on the progression of emphysema in patients with severe AATD," said Professor N. Gerard McElvaney, Head of the Department of Medicine Respiratory Research Division, at Beaumont Hospital, Dublin, Royal College of Surgeons in Ireland (RCSI) and lead author of the publication.

"With the publication of RAPID, we quantified years of life lost without A1-PI therapy to preserve lung structure. Now, with the publication of the RAPID extension study, we have found that the late introduction of A1-PI therapy is still disease modifying - but the lung structure lost by the late introduction is never recovered. RAPID's message is to intervene. The RAPID extension message is to intervene early," said Kenneth R. Chapman, MD, Director of the Asthma & Airway Centre at the University Health Network in Toronto, Canada.

The Lancet Respiratory Medicine, a specialty journal, published findings of the RAPID OLE (Randomized, Placebo-controlled Trial of Augmentation Therapy in Alpha-1 Proteinase Inhibitor Deficiency Open Label Extension) study, where eligible patients continued for another two years from the original two-year RAPID trial, the largest and longest placebo-controlled AATD trial to ever have been conducted globally. The trial set out to measure the progression of emphysema, assessed by volume-adjusted lung density (measured by CT.)

The RAPID OLE consisted of two groups of patients. The “Early-Start” group received Respreeza® A1-PI therapy during both trials, providing up to four years of continuous treatment, while the “Delayed-Start” group received placebo during the first two years and then switched to Respreeza® in the extension trial, providing up to two years of active treatment.

While a similar rate of decline was observed in both groups between months 24 and 48, an advantage was sustained over the four-year period for the "Early-Start" group, which experienced a lower overall rate of lung density decline. During the extension trial, the "Delayed-Start" group failed to catch-up to their "Early-Start" counterparts, indicating that the lung tissue lost during placebo treatment could not be regained.

The RAPID and the RAPID extension trials, investigating the efficacy of Respreeza®, provide an advancement in understanding the impact of A1-PI therapy. Available A1-PI therapies can differ in terms of product features, which should be considered when choosing the appropriate therapy for patients.

About Alpha-1 Antitrypsin Deficiency (AATD)
AATD is a hereditary condition that can severely affect a patient’s lung function. The condition is marked by a low level or absence of alpha-1-proteinase inhibitor (A1-PI), a natural protein that inhibits neutrophil elastase, thereby preventing destruction of lung tissue. Severe deficiency of A1-PI is associated with a strong tendency for the development of emphysema, a form of chronic obstructive pulmonary disease (COPD), and can significantly impact everyday life and life expectancy.

About Respreeza®
In the EU, Respreeza® is indicated for maintenance treatment, and to slow the progression of emphysema in adults with documented severe A1-PI deficiency (e.g. genotypes PiZZ, PiZ(null), Pi(null,null), PiSZ). Patients are to be under optimal pharmacologic and non-pharmacologic treatment and show evidence of progressive lung disease (e.g. lower forced expiratory volume per second (FEV1) predicted, impaired walking capacity or increased number of exacerbations) as evaluated by a healthcare professional experienced in the treatment of A1-PI deficiency. Respreeza® is contraindicated in patients with hypersensitivity to the active substance or to any of the excipients and IgA deficient patients with known antibodies against IgA, due to the risk of severe hypersensitivity and anaphylactic reactions.

CSL Behring has marketed Respreeza® as Zemaira® in the Unites States since 2003.

About CSL Behring
CSL Behring is a global biotherapeutics leader which is driven by its promise to serve patients’ needs by using the latest technologies. We develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, inherited respiratory disease, and neurological disorders. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn.

CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company CSL Limited (ASX:CSL), headquartered in Melbourne, Australia, employs more than 17,000 people with operations in more than 30 countries. For more information visit www.cslbehring.com and follow us on www.Twitter.com/CSLBehring.

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Contact:
Jennifer Purdue
CSL Behring
Office: +1 610 878 4802
Mobile: +1 610 306 9355
Email: jennifer.purdue@cslbehring.com

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