New Clinical Data on Treatment to Slow the Progression of Emphysema in Patients with Alpha-1 Antitrypsin Deficiency Presented at European Respiratory Society International Congress in Amsterdam

MARBURG, Germany — 28 September 2015

CSL Behring today hosted a symposium highlighting an option to slow the progression of emphysema in adults with documented severe alpha-1 antitrypsin deficiency (AATD), during the European Respiratory Society (ERS) International Congress, which is being held from 26-30 September in Amsterdam, Netherlands. AATD is a hereditary condition marked by a lack of the alpha-1 antitrypsin protein, whose main function is to protect the lungs from inflammation.

The symposium was chaired by Professor Gerry McElvaney, MD, Royal College of Surgeons in Ireland, and included presentations by Professor Kenneth R. Chapman, MD, (Canada), Dr. David Parr, MD (United Kingdom), Dr. Emer Reeves, MD (Ireland) and Dr. Niels Seersholm, MD (Denmark).

During the symposium, Dr. Chapman highlighted evidence from the recently published RAPID trial of alpha-1 proteinase inhibitor (A1-PI) therapy. Chapman notes, “RAPID is regarded as a landmark study validating almost two decades of focus on the lung-density endpoint as the most sensitive way to track lung tissue decline in this potentially debilitating disease. Our findings, for the first time in a single randomized-controlled trial, provided additional evidence that treatment with an alpha-1 proteinase inhibitor provides a clinical benefit by slowing the accelerated loss of lung tissue.”

Professor McElvaney also presented data on the RAPID Extension trial, where eligible subjects received A1-PI for another 2 years, in an oral presentation in a separate late-breaker session on 27 September. “The extended trial showed that, over 48 months, A1-PI reduced the rate of lung density decline. The treatment effect was not only maintained by patients treated for all 4 years, but also showed significant reduction in terms of absolute lung density loss in patients that were on placebo for the first 2 years and then switched to active therapy in the last 2 years.” said, Professor McElvaney. “These results further confirm the findings of the RAPID trial and support the use of augmentation therapy in the treatment of severe AATD.”

Data from the RAPID and RAPID Extension trials were the basis for the marketing application in the EU for Respreeza®, a highly purified alpha-1 protein derived from human plasma, in the treatment of severe AATD. The European Commission granted approval of Respreeza® on 20 August, 2015. It is now marketed by CSL Behring in Europe and the first national launches of the product are imminent. Respreeza® is the only alpha-1 proteinase inhibitor that has been proven in a prospective double blind, placebo controlled trial (the RAPID study) to significantly reduce the loss of lung tissue, slowing the progression of emphysema due to AATD.

About the RAPID Program

RAPID Trial - The RAPID trial was a multicenter, double-blind, randomized, parallel group, placebo-controlled study comparing the efficacy and safety of Respreeza®, CSL Behring’s A1-PI therapy, with placebo in patients with emphysema due to AATD. The participants, 180 non-smokers aged 18-65, were randomly assigned to receive A1-PI intravenously 60 mg/kg weekly or placebo.

RAPID Trial Study Design and Findings - According to study protocol, the effect of Respreeza® on the progression of emphysema, the primary endpoint of the study, was measured by computed tomography scan over 24 months and was assessed by the annual rate of lung density loss at total lung capacity (TLC) and functional residual capacity (FRC). The annual rate of lung density loss at TLC (i.e, full aspiration) showed a statistically significant 34 percent reduction in the annual rate lung density decline compared with placebo. There was 24 percent reduction when measured at FRC (i.e., resting exhalation), which was not significant. Serious adverse events were not statistically different between groups with one death in the A1-PI group and three deaths in the placebo group.

RAPID Trial Extension - Eligible subjects completing the 2-year RAPID trial were enrolled into a 2-year, open label RAPID Extension trial where they either continued to receive weekly infusions of Respreeza® for another 2 years (Early Start group) or were switched from placebo to receive 2 years of Respreeza® therapy (Delayed Start group).

The Early Start group maintained an advantage over placebo across the 48 month period. The Delayed Start group showed a statistically significant reduction in the absolute loss of lung density in response to the administration of Respreeza® in the last 2 years compared to the loss in the first 2 years while exposed to placebo. The number and proportion of fast decliners (annual rate of lung density loss > 2g/L/y) was reduced in association with treatment with Respreeza® across both studies. The 4-year data also demonstrated moderate, statistically significant correlations between changes in lung density decline and changes in FEV1, FEV1% predicted and FVC.

These effects across both studies demonstrate a consistent disease modifying effect of A1-PI therapy, and suggest that early treatment may be more appropriate to effectively slow the progression of emphysema.

About Respreeza®

In 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.

About CSL Behring

The people and science of CSL Behring save lives around the world. We develop and deliver innovative specialty biotherapies, driven by our 100-year promise to help people with life-threatening conditions live full lives. With 14,000 employees and operations in 30 countries, CSL applies world-class R&D, high-quality manufacturing and patient-centered management.

CSL Behring therapies are used around the world to treat coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease, and neurological disorders in certain markets. 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. CSL Behring is a global biopharmaceutical company and a member of the CSL Group of companies. The parent company, CSL Limited (ASX:CSL), is headquartered in Melbourne, Australia. For more information, visit



Jennifer Purdue
CSL Behring
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