Research & Development

CSL’s global R&D activities focus on the development of innovative new and improved products and manufacturing processes thereby ensuring our continued growth. Our R&D portfolio is divided into four strategic areas – speciality products, haemophilia, breakthrough medicines and immunoglobulins. Over the past year, we have achieved successes in all four strategic areas with new registrations, positive results in some of the largest clinical trials ever conducted in rare diseases and an exciting new collaboration.

Specialty Products

Strong progress has been made in our specialty products portfolio over the past year. Results of a landmark study published in December 2016 confirmed the diseasemodifying effect of RESPREEZA, a highly purified alpha-1 therapy for maintenance treatment to slow the progression of hereditary emphysema in patients with Alpha-1 Antitrypsin Deficiency (AATD). The use of RESPREEZA, in the largest and longest placebo-controlled AATD trial to ever have been conducted, slowed the progressive loss of lung tissue, which once lost is never recovered. The greatest benefit was observed in the ‘early-start’ patient group, demonstrating that early intervention with RESPREEZA is key to preventing the irreversible loss of lung tissue associated with AATD.

In June 2017, the FDA approved our Biologics Licence Application (BLA) for a low-volume subcutaneous C1-Exterase Inhibitor (C1-INH) replacement therapy to prevent Hereditary Angioedema (HAE) attacks. HAE is a rare and potentially life-threatening genetic condition caused by a lack of or malfunctioning C1-INH protein and can lead to the build-up of fluid in multiple parts of the body. If untreated, HAE attacks involving the face or throat can result in airway closure, asphyxiation and potentially death. HAEGARDA, C-1 Esterase Inhibitor Subcutaneous (Human), is the first and only self-administered subcutaneous prophylactic therapy to prevent HAE attacks and will provide a new standard of care for HAE. In addition to licence approval, the FDA granted CSL Behring seven years orphan-drug designation, enabling marketing exclusivity through to 22 June 2024.

CSL Behring’s HAEGARDA, for the prevention of Hereditary Angioedema attacks was approved by the US Food and Drug Administration in June 2017.

CSL Behring’s HAEGARDA, for the prevention of Hereditary Angioedema attacks, was approved by the US Food and Drug Administration in June 2017.


Over the past year we successfully achieved new regulatory approvals in major jurisdictions for our recombinant coagulation factor products. In January 2017, we received regulatory approval in Europe for AFSTYLA, the only recombinant factor VIII single chain indicated for the treatment of haemophilia A. AFSTYLA was also granted approval in Canada in December 2016 and approval is expected in Japan later this year.

In September 2016, we received regulatory approval in Japan for IDELVION, our long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin for the treatment of haemophilia B. The efficacy of our recombinant coagulation factors was highlighted during the presentation of data from our pivotal Phase III studies at the World Federation of Hemophilia World Congress in July 2016. Preliminary results using IDELVION in the PROLONG-9FP extension trial suggest that extended treatment intervals of up to 21 days may be possible for adults. This extended regime would provide a significantly reduced burden of treatment associated with frequent prophylactic dosing and a positive impact for patients. IDELVION is currently licensed for treatment intervals of up to 14 days.

Breakthrough Medicines

Signifi cant progress has been made in the development of new breakthrough medicines over the past year with the initiation of human trials to investigate new monoclonal antibodies with novel mechanisms of action.

CSL324 neutralises G-CSF activity and may provide a new treatment for rare inflammatory diseases associated with overactive neutrophils (white blood cells).

CSL312 is an anti-factor XIIa monoclonal antibody that is being studied for use in multiple indications including as a subcutaneous therapy for HAE with the potential for administration once every two to three weeks. Another potential indication under investigation for CSL312 is the prevention of thrombosis.

CSL346 targets VEGF-B and could potentially be used to control glucose absorption in Type 2 diabetics by targeting fatty acid metabolism. CSL346 may also be beneficial in the treatment of diabetic nephropathy, one of the most common kidney complications associated with Type 2 diabetes, where VEGF-B levels have been shown to be elevated in patients.

Phase 1 clinical trials with CSL324 and CSL312 started in July and October 2016 respectively and CSL346 is due to enter the clinic in late 2017.

Positive results from our Phase 2b clinical trial designed to evaluate the safety and proof of mechanism of CSL112, a novel apolipoprotein A-I infusion therapy, were presented in November 2016. CSL112 is being developed to reduce the high incidence of early recurrent cardiovascular events that occur in the weeks to months following a heart attack by rapidly stabilising additional atherosclerotic plaques at risk of rupture. Data from the trial demonstrated that CSL112 does not cause significant changes in liver or kidney function and demonstrated that it is well-tolerated on administration in the acute myocardial infarction (MI) setting, thereby meeting the primary safety endpoints. CSL112’s unique mechanism of action, the removal of cholesterol from atherosclerotic plaque in the arteries, was also confirmed by the study. Planning for a possible Phase III trial is now underway while we complete additional Phase II studies.


CSL’s Ig portfolio continues to grow with further expansion into neurology. In February 2017, the FDA accepted for review our BLA supplement to obtain approval for a new indication for PRIVIGEN, Immune Globulin Intravenous, 10% liquid formulation. Approval is being sought for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP), a rare and progressing disease that may cause permanent nerve damage. PRIVIGEN has already been approved to treat CIDP in Europe. Data to support the submission came from CSL’s PRIMA and PATH studies, both focused on the treatment of CIDP with our Ig therapy. Results from the PRIMA study suggest that PRIVIGEN may help decrease weakness and loss of motor function in patients with CIDP. Data from the PATH study, the largest ever clinical study to investigate the treatment of CIDP, supports the efficacy, safety and tolerability of PRIVIGEN in the treatment of CIDP. In addition, the US FDA also accepted in July our application to add CIDP to the HIZENTRA® label.

Collaboration with external partners continues to provide CSL with important new opportunities to develop novel therapies for patients. In January 2017 we announced an exclusive research collaboration and worldwide licence agreement with Momenta Pharmaceuticals, Inc. to develop and commercialise their recombinant Fc multimer proteins for use in controlling inflammation. The agreement includes Momenta’s M230 which has been shown to match the potency and efficacy of intravenous immunoglobulin at significantly lower doses in animal models of autoimmune disease. Clinical trials using M230 are expected to start in the next year.

Investment in R&D remains a key driver for CSL’s future growth. We have a high quality and potentially valuable portfolio of projects in various stages of development. We continue to make a balanced investment in the life cycle management and market development of existing products that bring short to mid-term commercial benefi ts, and we make strategic investments in longer term, higher risk and high opportunity new product development activities. In 2016/17, CSL invested US$645 million on R&D and was supported by an R&D workforce of approximately 1,400 scientists worldwide.


CSL Research and Development Investment (US$ millions)

R and D investment