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CSL Behring to Present New Data for IDELVION® and AFSTYLA® at the World Federation of Hemophilia 2016 World Congress

KING OF PRUSSIA, Pa. — 19 July 2016

Abstracts include pivotal efficacy and safety data for IDELVION for hemophilia B and AFSTYLA for hemophilia A; AFSTYLA and IDELVION demonstrate CSL Behring’s promise to develop and deliver innovative treatments that help patients with serious medical conditions live full lives

CSL Behring announced today that it will present new data from its recombinant coagulation factor development programs at the XXXII International Congress of the World Federation of Hemophilia (WFH) in Orlando, Fla., July 24 – 28, 2016.

Six posters will highlight new IDELVION® [Coagulation Factor IX (Recombinant), Albumin Fusion Protein] data, including interim results on CSL’s Phase IIIb safety and efficacy study. IDELVION (also known as rIX-FP) is CSL Behring’s novel, long-acting recombinant albumin fusion protein for hemophilia B. One oral and six poster presentations will showcase AFSTYLA® [Antihemophilic Factor (Recombinant), Single Chain] data, including pediatric results highlighting a Phase III pharmacokinetic, efficacy and safety study. AFSTYLA (also known as rVIII-SingleChain) is CSL Behring’s long-acting, novel recombinant factor VIII single-chain therapy for hemophilia A.

“We are excited to share new clinical findings for IDELVION and AFSTYLA during WFH,” said Dr. Andrew Cuthbertson, Chief Scientific Officer and Director of R&D, CSL Limited. “These novel recombinant therapies and new data reinforce our promise to develop and deliver innovative specialty biotherapies that have the potential to improve patients’ lives. We look forward to bringing IDELVION and AFSTYLA to patients in countries where each treatment is now available and to receiving regulatory approvals in additional markets throughout the world.”

CSL Behring representatives will be available at booth number 20 with the company’s medical affairs representatives at booth number 45 in the Orange County Convention Center’s West Concourse throughout the congress. Key sessions, including two satellite symposiums, from CSL Behring at WFH include:

IDELVION

Satellite Symposium

Sunday, July 24, 3:30 p.m. - 5:00 p.m. EDT

  • IDELVION: Revolutionizing the Treatment of Hemophilia B. Room #311

Poster Presentations

Monday, July 25, 10:00 a.m. - 6:00 p.m. EDT

  • Half-life extended recombinant fusion protein linking factor IX with albumin is recycled via the intracellular FcRn-mediated pathway. Poster #2
  • Performance of a recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in the one-stage assay. Poster #74.

Tuesday, July 26, 10:00 a.m. - 6:00 p.m. EDT

  • Efficacy and safety of long-acting recombinant fusion protein linking factor IX with albumin (rIX-FP) in hemophilia B patients undergoing surgery. Poster #84.
  • Interim results of a Phase IIIb safety and efficacy extension study of a recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in patients with hemophilia B. Poster #113.
  • Long-term safety and efficacy of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in previously treated patients with hemophilia B. Poster #122.

Wednesday, July 27, 10:00 a.m. - 6:00 p.m. EDT

  • Effect of once-weekly prophylaxis treatment with a recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) on target joints in patients with hemophilia B during the PROLONG-9FP clinical trial program. Poster #138.

AFSTYLA

Satellite Symposium

Tuesday, July 26, 2016, 12:30 p.m. – 2:00 p.m. EDT

  • rVIII-SingleChain: Novel Technology in the Treatment of Hemophilia A. Room #308

Oral Presentation

Wednesday, July 27, 2:15 p.m. - 3:45 p.m. EDT, Room #307

  • The effect of non-neutralizing anti-drug antibodies on PK and bleeding rates in children less than 12 years of age with severe hemophilia A treated with rVIII-SingleChain. Topic #3.

Poster Presentations

Monday, July 25, 10:00 a.m. - 6:00 p.m. EDT

  • Potency determination of single-chain rFVIII concentrate. Poster #10.
  • Characteristics of rVIII-SingleChain in the one-stage and the chromogenic substrate assay: results of an international field study. Poster #70.

Tuesday, July 26, 10:00 a.m. - 6:00 p.m. EDT

  • rVIII-SingleChain, results of the pivotal efficacy data from a phase III PK, efficacy and safety clinical study in children less than 12 years of age with severe hemophilia A. Poster #87.
  • rVIII-SingleChain in surgical prophylaxis: efficacy and safety in 21 major surgeries. Poster #108.

Wednesday, July 27 from 10:00 a.m. - 6:00 p.m. EDT

  • Tolerance induction in a pediatric patient with severe hemophilia A and a low titer inhibitor using an intensified prophylaxis regimen with rVIII-SingleChain. Poster #89.
  • Population pharmacokinetic model of recombinant single-chain factor VIII (rVIII-SingleChain) in patients with hemophilia A. Poster #142.

rVIIa-FP (CSL Behring’s long-acting fusion protein linking recombinant coagulation factor VIIa with recombinant albumin)

Monday, July 25, 10:00 a.m. - 6:00 p.m. EDT

  • Intracellular trafficking and FcRn-dependent recycling of recombinant factor VIIa-albumin fusion protein (rVIIa-FP) provides a mechanism for half-life extension in vivo. Poster #88.

Plasma-derived VWF/FVIII concentrate

Tuesday, July 26, 10:00 a.m. - 6:00 p.m. EDT

  • Comparison of the pharmacokinetic parameters of a plasma-derived VWF/FVIII concentrate (Voncento®) in adult/adolescent and pediatric subjects with von Willebrand disease (SWIFT-VWD and SWIFTLY-VWD study). Poster #185.

Wednesday, July 27, 10:00 a.m. - 6:00 p.m. EDT

  • Pharmacokinetics of a plasma-derived VWF/FVIII concentrate (Voncento®) in adult/adolescent and pediatric subjects with severe hemophilia A (SWIFT-HA and SWIFTLY-HA studies). Poster #141.

About Hemophilia

Hemophilia is an inherited bleeding disorder caused by deficient or defective proteins that prevent the blood from clotting properly. According to the National Hemophilia Foundation, the condition affects approximately 400,000 people worldwide, the majority of whom have hemophilia A.

About IDELVION®

IDELVION is approved in the United States, European Union and Canada. Regulatory agencies in Australia, Switzerland and Japan are also currently reviewing CSL Behring’s license applications for IDELVION. Regulatory approvals were based on data from the PROLONG-9FP clinical development program showing that IDELVION delivers high-level protection with up to 14-day dosing in appropriate patients, with a median annualized spontaneous bleeding rate (AsBR) of 0.00. This dosing interval has been achieved while maintaining high levels of factor activity, above 5 percent over 14 days. These data were published in the American Society of Hematology’s publication Blood.

For more information about IDELVION, including full prescribing information, please visit http://labeling.cslbehring.com/PI/US/IDELVION/EN/IDELVION-Prescribing-Information.pdf.

About AFSTYLA®

AFSTYLA, specifically designed for long-lasting protection from bleeds with two to three times weekly dosing available, is approved in the U.S. Regulatory agencies in Europe, Switzerland and Australia are currently reviewing CSL Behring’s marketing applications for AFSTYLA.

FDA approval of AFSTYLA was based on results from the AFFINITY clinical development program. In clinical trials, patients undergoing prophylaxis with AFSTYLA experienced a median annualized spontaneous bleeding rate (AsBR) of 0.00. Additionally, AFSTYLA demonstrated a strong safety profile with no inhibitors observed. These data were recently published in the American Society of Hematology’s publication Blood.

For more information about AFSTYLA, including full prescribing information, please visit http://labeling.cslbehring.com/PI/US/Afstyla/EN/Afstyla-Prescribing-Information.pdf.

U.S. Only: IDELVION Important Safety Information

IDELVION®, Coagulation Factor IX (Recombinant), Albumin Fusion Protein (rFIX-FP), is indicated in children and adults with hemophilia B (congenital factor IX deficiency) for:

  • On-demand control and prevention of bleeding episodes
  • Perioperative management of bleeding
  • Routine prophylaxis to prevent or reduce the frequency of bleeding episodes

IDELVION is not indicated for induction of immune tolerance in patients with hemophilia B.

IDELVION is contraindicated in patients who have had life-threatening hypersensitivity to the product or its components, including hamster proteins.

IDELVION is for intravenous use only. IDELVION can be self-administered or administered by a caregiver with training and approval from a healthcare provider or hemophilia treatment center. Higher dose per kilogram body weight or more frequent dosing may be needed for pediatric patients.

Hypersensitivity reactions, including anaphylaxis, are possible. Advise patients who self-administer to immediately report symptoms of hypersensitivity, including angioedema, chest tightness, hypotension, generalized urticaria, wheezing, and dyspnea. If symptoms occur, discontinue IDELVION and administer appropriate treatment.

Development of neutralizing antibodies (inhibitors) to IDELVION may occur. If expected factor IX activity plasma levels are not attained or bleeding is not controlled with appropriate dose, perform an assay to measure factor IX inhibitor concentration. Factor IX activity assay results may vary with the type of activated partial thromboplastin time reagent used.

Thromboembolism (e.g., pulmonary embolism, venous thrombosis, and arterial thrombosis) can occur when using factor IX-containing products. In addition, nephrotic syndrome has been reported following immune tolerance induction in hemophilia B patients with factor IX inhibitors and allergic reactions to factor IX.

The most common adverse reaction (incidence ≥1%) reported in clinical trials was headache.

U.S. Only: AFSTYLA Important Safety Information

AFSTYLA®, Antihemophilic Factor (Recombinant), Single Chain, is indicated in adults and children with hemophilia A (congenital factor VIII deficiency) for:

  • On-demand treatment and control of bleeding episodes
  • Routine prophylaxis to reduce the frequency of bleeding episodes
  • Perioperative management of bleeding

AFSTYLA is not indicated for the treatment of von Willebrand disease.

AFSTYLA is contraindicated in patients who have had life-threatening hypersensitivity reactions to AFSTYLA or its excipients, or to hamster proteins.

AFSTYLA is for intravenous use only. AFSTYLA can be self-administered or administered by a caregiver with training and approval from a healthcare provider or hemophilia treatment center.

Higher and/or more frequent dosing may be needed for patients under 12 years of age.

Hypersensitivity reactions, including anaphylaxis, are possible. Advise patients to immediately report symptoms of a hypersensitivity reaction. If symptoms occur, discontinue AFSTYLA and administer appropriate treatment.

Development of factor VIII (FVIII) neutralizing antibodies (inhibitors) can occur. If expected factor VIII activity levels are not attained or bleeding is not controlled with appropriate dose, perform an assay to measure factor VIII inhibitor concentration.

Monitor plasma factor VIII activity using a chromogenic assay or one-stage clotting assay. In the U.S., if one-stage clotting assay is used, multiply result by a conversion factor of 2 to determine factor VIII activity level.

The most common adverse reactions (>0.5%) reported in clinical trials were dizziness and hypersensitivity.

About CSL Behring

CSL Behring is a global biotherapeutics leader which is driven by its promise to save lives. Focused on serving 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 16,000 people with operations in more than 30 countries. For more information about CSL Behring visit www.CSLBehring.com or follow us at www.Twitter.com/CSLBehring.

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Contact:

Greg Healy
CSL Behring
Office: 610-878-4841
Mobile: 610-906-4564
Email: Greg.Healy@CSLBehring.com

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