Recent Highlights

Replacement Therapies
CSL's replacement therapies focus has been on expanding the geographical registrations and medical indications for existing plasma products and progressing the recombinant coagulation projects in preclinical development.

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.

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.

On October 12 2009, the USA 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^® is the first and only therapy approved for this indication in the US. Berinert^® has also received approval from the Australian TGA and Health Canada and is now licensed in 28 countries worldwide.

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. 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 new addition to the rapidly growing CSL Behring product portfolio, and further demonstrates our long-standing committment to the PI and rare disease communities.

CSL scientists are at the early stages of developing a family of recombinant coagulation factor medicines to treat haemophilia and other coagulation defects. Recombinant forms of factors IX and VIIa are in development utilizing albumin fusion technology which extends the half life of circulating molecules. If successful, this should result in a marked reduction in the frequency of administration and significantly increases convenience for patients. Proof of principle data in animals has been obtained showing extended half life for recombinant FVIIa and FIX albumin fusion proteins.

Influenza Vaccines: CSL received approval from the FDA to market Afluria^®, an influenza vaccine, in the United States in September 2007. The immediate post-marketing clinical program has now been completed with four trials in adults, the elderly and the very young involving approximately 20,000 subjects. On 17 November 2009, the FDA granted approval to include active immunization against influenza for individuals 6 months through 17 years of age.

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 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% participants receiving a standard 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 Organization.

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 the disease. This program is being conducted in collaboration with the Cooperative Research Centre for Oral Health Science.

ISCOMATRIX^® Adjuvant
The ISCOMATRIX^® adjuvant activities continue with our partners progressing from pre-clinical to clinical development with several product candidates. Recently, we also entered into a partnership with Solvay Biologicals, which has licensed CSL's ISCOMATRIX^® adjuvant.

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.

Therapeutic Proteins
In the Therapeutic Proteins program we have progressed development of antibodies which target the IL-3 growth factor receptor for treatment of acute myeloid leukemia and the CAM3001 GM-CSFR product (partnered with AstraZeneca/MedImmune) entered into Phase II clinical trials in February 2010. 

The development of reconstituted High Density Lipoprotein (HDL), a potential additional product from human plasma, is a priority for CSL's R&D program. We have recently reformulated a prototype product, completed pre-clinical testing and commenced a phase I safety study for possible use in Acute Coronary Syndrome.

We also have a portfolio of earlier stage projects including a project to develop therapeutic antibodies that inhibit the activity of the cytokine granulocyte colony stimulating factor (G-CSF) for the potential development of a new class of drugs that target arthritis and other inflammatory diseases.