CSL R&D supports and enhances CSL’s portfolio of intravenous and subcutaneous immunoglobulin therapies by developing new therapy options with greater convenience for patients and healthcare practitioners.

CSL continues to enhance its immunoglobulin portfolio by expanding global registration of Privigen^®, its state-of-the-art chromatographic intravenous immunoglobulin (IVIg) in the US and Europe. Privigen^® is a high-yield, proline-stabilised IVIg that is ready for immediate use, requiring no refrigeration or reconstitution. The product is intended for patients diagnosed with primary immunodeficiency (PI) and chronic immune thrombocytopenic purpura (ITP).

In April 2010 the US Food and Drug Administration (FDA) approved a supplemental Biologics License Application to extend the shelf life for Privigen^®, Immune Globulin Intravenous (Human), 10% Liquid, from 24 to 36 months. The approval makes Privigen^® the first liquid intravenous immunoglobulin in the US that can be stored at room temperature throughout its entire 36-month shelf life. CSL is also strengthening its presence in the neurology market and have now conducted a Phase III study in CIDP (Chronic Inflammatory Demyelinating Polyneuropathy) in Europe. The recruitment was completed in November 2011.

During 2009/10 the clinical development of CSL's 20% subcutaneous immunoglobulin product was completed. On 4 March 2010, the US FDA granted marketing approval for Hizentra™, Immune Globulin Subcutaneous (Human), 20% Liquid, for treating patients diagnosed with primary immunodeficiency (PI). Hizentra™ is the first 20% subcutaneous immunoglobulin (SCIg) approved in the US by the FDA. During 2010/11, Hizentra^® was approved by the European Commission for treating patients in Europe diagnosed with Primary Immune Deficiency or Secondary Immune Deficiency. As the first SCIg treatment with a 20% concentration of immunoglobulin, Hizentra™ represents an effective, convenient choice of at-home Ig therapy that will allow people with PI to schedule treatment around their busy lives instead of scheduling their lives around treatment. Hizentra™ is an important addition to the rapidly growing CSL Behring product portfolio, and further demonstrates our long-standing commitment to the PI and rare disease communities.

Specialty Products

CSL's focus has been on expanding the use of specialty products through new geographic markets, medical indications and/or alternate modes of administration.

Riastap^™(human fibrinogen) was approved by the FDA in January 2009 for the treatment of patients with a congenital deficiency of this coagulation protein. It is the first and only treatment for acute bleeding episodes in patients with congenital fibrinogen deficiency, an extremely rare and potentially life-threatening bleeding disorder. Following approval in Germany in late 2009, Riastap^™ also gained approval more broadly across Europe via completion of the mutual recognition process in July 2010.

In February 2011 the US FDA granted marketing approval for Corifact™, Factor XIII Concentrate (Human), for the routine prophylactic treatment of congenital factor XIII (FXIII) deficiency. Corifact™, already available for use in 12 countries throughout the world under the trade name Fibrogammin^®- P, is the first and only FXIII concentrate approved in the US.

Congenital FXIII deficiency, also known as fibrin-stabilising factor deficiency, is a rare and potentially life-threatening bleeding disorder in which blood clots normally, but the clots formed are unstable, leading to recurrent bleeding. It is estimated that the condition affects one person in two million, with an incidence in the US of approximately 150 people.

On 12 October 2009, the US FDA granted marketing approval for Berinert^® C1-Esterase Inhibitor for the treatment of acute abdominal or facial attacks associated with hereditary angioderma (HAE) a rare and serious genetic disorder. Berinert^®, the first and only therapy approved for this indication in the US, has also received approval from the Australian TGA and Health Canada. It is now licensed in 31 countries worldwide.

Haemophilia Products

CSL scientists are committed to expanding our coagulation therapies portfolio through development of recombinant coagulation factor medicines to treat haemophilia and other coagulation disorders. Recombinant forms of factors IX and Vlla are in development utilising albumin fusion technology which extends the half life of the circulating molecules. If successful, this should result in a marked reduction in the frequency of administration and would significantly increase convenience for patients. Proof of principle data in animals has been obtained showing extended half life for the recombinant FVlla and FIX albumin fusion proteins. CSL is also developing a unique single chain recombinant factor VIII (rVIII-SingleChain) for treatment of haemophilia A.

Important milestones in the past year include completion of a Phase I clinical trial of rIX-FP and completion of preclinical studies to support the development of rVIIa-FP and rFVIII products. CSL has now enrolled its first patient in a rVIII-SingleChain Phase I study. A research program has also been initiated for long acting rvWF-FP.

Breakthrough Medicines

In the Breakthrough Medicines program we aim to develop new protein based therapies to address significant unmet medical needs.

Significant progress has been made in our earlier stage recombinant monoclonal antibody (mAb) projects including the Anti-G-CSFR mAb (CSL324) for treating inflammatory conditions caused by an over activity of white blood cells called neutrophils, and also the Anti-IL-3R mAb (CSL362) for treating acute myeloid leukaemia (AML). We now have data supporting an AML minimal residual disease (MRD) indication and non-human primate and other studies also support the potentially valuable additional use of CSL362 in treating a serious autoimmune disease.

The development of reconstituted High Density Lipoprotein (rHDL), a potential additional product from human plasma, is a priority for CSL's R&D program. We have recently completed a Phase I safety study supporting possible use in Acute Coronary Syndrome. CSL plans to commence Phase II studies in early 2012.

In July 2010, CSL announced its decision to build a large scale biotechnology facility for the manufacture of therapies based on recombinant DNA technology. The new facility will enable CSL to develop innovative products from our R&D pipeline for late-stage clinical trials and ultimately patient use, such as our recombinant coagulation factor products or our recombinant monoclonal antibodies.

The project, supported by both the Federal and Victorian State Governments, will generate significant employment opportunities and further develop Australia's biotechnology sector and medical research community. Design and construction of the new facility commenced during 2010/11.

Vaccines & Licensing

Influenza Vaccines

Because of the complexity of CSL’s biological products we regularly undertake research activities as part of our commitment as a supplier of safe and efficacious medicines. One example which arose in the past year was the development and rapid implementation of a high quality research program to identify the cause of reported febrile events associated with the influenza vaccine in paediatric patients. The preliminary results of this research are being communicated to public health officials and regulatory agencies.

The advent of the H1N1 pandemic influenza threat required an immediate response from CSL scientists to develop a vaccine against the novel virus. CSL conducted several clinical trials to establish an optimum vaccine dose for protection against this new strain of influenza. The data from this trial, published in the New England Journal of Medicine, showed that over 95% of participants receiving a standard single dose of vaccine achieved antibody levels that correlate with the prevention of influenza infection. Data from this and additional clinical trials assisted governments and regulatory authorities to determine how to best deploy the vaccine. CSL's Panvax^® H1N1 vaccine is now registered in Australia, the US, New Zealand, Singapore, Germany and by the World Health Organisation.

GARDASIL^®

In 2006 Merck and Co. registered its human papillomavirus vaccine, GARDASIL^®, in the US, Europe and Australia. This vaccine provides protection against infection by Human Papillomavirus (HPV) Types 6, 11, 16, and 18. The vaccine is based on technology licensed by CSL to Merck & Co. in 1995 which was developed as a result of collaboration in the early 1990s between CSL and Professor Ian Frazer of the University of Queensland. CSL has exclusive marketing rights for GARDASIL^® in Australia and New Zealand.

In October 2009 the US FDA approved the use of GARDASIL^® in males aged 9 through 26 years of age for the prevention of genital warts (condylomata acuminata) caused by HPV types 6 and 11. Merck and Co. also submitted data to the US FDA and European Regulatory Authorities for expanded use in females aged 27-45 and its use for the prevention of anal cancer and its precurser AIN (anal intraepithelial neoplasia).

Merck have also completed a phase III trial on 9-valent HPV vaccine (ie. a vaccine that provides protection against nine different HPV types). A pivotal efficacy study in 14,000 subjects has been completed with anticipated global regulatory findings during 2012.

Partnered Projects

Our partner AstraZeneca recently successfully completed a phase IIa study of a monoclonal antibody targeting the GMCSF Receptor for the potential treatment of Rheumatoid Arthritis. Mavrilimumab showed a rapid and significant clinical effect compared to placebo with a safety profile supporting further clinical development.

In December 2009, CSL signed an agreement with Sanofi Pasteur, the world’s leading manufacturer of vaccines, to develop a vaccine to prevent and treat periodontitis, a severe gum disease affecting approximately 30% of adults. Candidate vaccine antigens against the bacterium Porphyromonas gingivalis, which causes periodontitis, are currently being trialed in mouse models of disease. This program is being conducted in collaboration with the Cooperative Research Centre for Oral Health Science.

ISCOMATRIX^® Adjuvant

ISCOMATRIX^® adjuvant activities continue with our partners, progressing from pre-clinical to clinical development with several product candidates in the past year.

Our partners also continue to show confidence in CSL's ISCOMATRIX^® adjuvant as a technology platform that could be used to enable the next generation of prophylatic and therapeutic vaccines. Additional licenses have been obtained from our major partners with more than 40 fields of interst for research and product development.

The transfer of the ISCOMATRIX^® adjuvant manufacturing process from Parkville to CSL's Kankakee, Illinois site is complete. Kankakee is now performing routine GMP manufacture of ISCOMATRIX^® adjuvant at a commercial scale.

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