New England Journal of Medicine Publishes Pivotal Phase III Data for CSL Behring’s Subcutaneous C1-Esterase Inhibitor in HAE Patients

• Median reduction in hereditary angioedema (HAE) attack rate relative to placebo was 95% at the highest dose studied
• Data show up to 40% of HAE patients were free from attacks throughout the study
• In the clinical study, CSL830 met the primary endpoint for preventing HAE attacks

KING OF PRUSSIA, Pa.– March 22, 2017 – Global biotherapeutics leader CSL Behring today announced that results were published in the New England Journal of Medicine (NEJM) from the COMPACT study, a pivotal Phase III study evaluating the safety and efficacy of CSL830 (a novel, investigational, self-administered, subcutaneous C1-Esterase Inhibitor [C1-INH] Human replacement therapy) for the prevention of HAE attacks. The study met its primary efficacy endpoint, significantly reducing the time-normalized number of HAE attacks. In addition, the study met its secondary endpoints, including the responder rate (patients who had at least a 50% reduction in their attack rate) and the number of rescue medication uses. If approved by the FDA, CSL830 would be the first and only subcutaneous preventative therapy for HAE.

“Subcutaneous C1-INH, as demonstrated in the COMPACT study, addresses an unmet need for patients and has the potential to change the treatment paradigm by adding a new option to prevent HAE attacks with a subcutaneous therapy,” said Bruce Zuraw, M.D., of the University of California San Diego School of Medicine, Director of the US HAEA Angioedema Center at UC San Diego, and chairman of the steering committee of the study.

The NEJM paper provides detailed data on the Phase III randomized efficacy study (NCT01912456) which evaluated type I & type II HAE patients during two 16-week treatment periods. The study looked at the number of attacks experienced by a patient given CSL830 prophylactically and the number of times HAE rescue medication was needed; both were reduced. Depending on the administered dose, HAE attack rates were reduced by a median of 89% and 95% (for the 40 IU/kg and 60 IU/kg dose, respectively). Additionally, 40% of patients on the higher dose were completely free of attacks, and patients, in general, experienced fewer and milder HAE symptoms overall. No patients on 60 IU/kg experienced a laryngeal attack within the study period.

“The study not only demonstrates very robust and dose-dependent efficacy, but also that the subcutaneous self-injection could be managed by all patients and was well tolerated,” said Hilary Longhurst, M.D., of Barts Health NHS Trust, London, United Kingdom, first author of the manuscript and member of the steering committee.

“Despite the strides that have been taken to provide current treatment options, there still remains a need for new therapies that prevent symptoms of this debilitating and potentially life-threatening disease,” added Andrew Cuthbertson, M.D., Chief Scientific Officer and R&D Director, CSL Limited. “These study results demonstrate the promise of CSL830 and its potential to significantly change the lives of people living with HAE by reliably preventing attacks with a self-administered subcutaneous therapy. CSL Behring thanks all of the patients who participated in this study for their courage and collaboration. Patient and investigating physician partnerships make clinical programs like COMPACT possible and progress could not be made without them.”

Phase III data in #HAE from @CSLBehring published in NEJM. Read the news here: Tweet

On August 30, 2016, CSL Behring announced that the US FDA accepted its Biologics License Application for CSL830.

About Hereditary Angioedema
HAE is a rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 to 1 in 50,000 people. HAE is caused by lack of or malfunctioning C1-INH. As a result, there are inadequate amounts of properly functioning C1-INH, leading fluid to build up in body tissues, causing considerable swelling episodes referred to as angioedema. HAE attacks can affect many parts of the body and can spread to multiple sites, including the face, abdomen, larynx and extremities. Patients who have abdominal attacks of HAE can experience episodes of extreme pain, diarrhea, nausea and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face or throat can result in airway closure, asphyxiation and, if untreated, death.

About CSL830 - Subcutaneous C1 Esterase Inhibitor (C1-INH)
CSL830 C1-Esterase Inhibitor (Human) is a novel, investigational, subcutaneous therapy. C1-INH replaces the missing or malfunctioning C1-INH protein in patients with a C1-INH deficiency.

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, providing its life-saving, life-changing therapies to people in more than 60 countries. For more information visit www.cslbehring.com and follow us on www.Twitter.com/CSLBehring.

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