CSL Continues to Advance R&D Capabilities

Unique portfolio mix of plasma, cell and gene therapy, recombinant proteins and antibody assets highlighted at Research & Development Briefing.

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SYDNEY, Australia - CSL Limited (ASX:CSL; USOTC:CSLLY) is steadily advancing its Research & Development (R&D) pipeline and capabilities to  deliver a highly differentiated product portfolio mix, addressing a broader range of patients’ unmet needs,” said Head of Research & Development Dr. William Mezzanotte today.

At CSL’s annual R&D briefing to investors, Dr. Mezzanotte noted the company is building on its leadership in plasma therapies through the identification of emerging new medicines from both within its existing portfolio of plasma-derived products, and through newer platforms such as gene and cell therapies and recombinant proteins.

To support this approach, CSL has forged targeted innovation partnerships in close proximity to its R&D locations, including at the Bio21 Institute in Melbourne, Australia, the Swiss Center for Translational Medicine in Bern, Switzerland and the University Science Center in Philadelphia, US.

“Our Phase 3 clinical program targeting the reduction of early recurrent cardiovascular events in heart attack survivors, CSL112, continues to track well.

“We continue our focus on developing new medical indications for immunoglobulins while improving manufacturing efficiencies across our plasma product portfolio,” Dr Mezzanotte said.

In FY19, CSL invested US$832 million into its R&D portfolio, representing 9.7% of total revenues.

R&D Pipeline Highlights

A novel treatment for asthma which has this month advanced to Phase 1, first-inhuman trials for patients with mild to moderate asthma. Asthma is a common chronic respiratory disease that is estimated to affect as many as 235 million1 people worldwide and is the most common chronic disease among children. Despite advances in the treatment of asthma, it is estimated that every year, more than 1,0002 people around the world die each day from this disease.

CSL’s trial will test for the safety of a therapy delivered by subcutaneous injection that asthma sufferers could self-administer at home once every two to four weeks, acting prophylactically to prevent asthma attacks.

The potential therapy, which currently holds the working title “CSL311,” is a monoclonal antibody that targets multiple inflammatory agents involved in various diseases.

CSL311 is the first monoclonal antibody to simultaneously target three cell-signaling cytokines, or molecules, that are responsible for the immune response that causes asthma and in doing so, suppresses inflammation of airways.

Commenting on the potential of the research, University of Melbourne Professor Jo Douglass, Head of the Immunology and Allergy Department at the Royal Melbourne Hospital and a research collaborator on the project, said, “Asthma is a serious disease that in extreme cases can be fatal. Currently, our treatment options for severe asthma are limited. We are excited by the potential of CSL311 to address a problem that affects the lives of so many.”

Addressing Severe Muscle Disease

Another pipeline project featured today is a Phase 3 clinical trial for novel use of CSL Behring‘s existing subcutaneous Immunoglobulin (Ig) product in patients with a severe condition called Dermatomyositis. The Ig product is currently indicated for use in a rare neurological disorder, Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) as well as primary and secondary immunodeficiencies.

Dermatomyositis is one of a group of acquired muscle diseases called inflammatory myopathies which are characterized by chronic muscle inflammation accompanied by muscle weakness. If the disease goes untreated it can lead to difficulty in walking or the need for a wheelchair or even becoming bedridden.

“Our pipeline is as robust and promising as ever,” Dr. Mezzanotte said. “Our R&D portfolio holds the potential to unlock a broad range of new therapies for people with challenging medical conditions. That promise is what drives our 1,700-plus scientists to work every day as if someone’s life depends on it – because it really does.”

1 World Health Organisation: https://who.int/respiratory/asthma/en/
2 The Global Asthma Report 2018: http://globalasthmareport.org/burden/mortality.php

About CSL

CSL (ASX:CSL) is a leading global biotechnology company with a dynamic portfolio of life-saving medicines, including those that treat haemophilia and immune deficiencies, as well as vaccines to prevent influenza. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL — including our two businesses, CSL Behring and Seqirus - provides life-saving products to more than 60 countries and employs 25,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest.

For more information about CSL Limited, visit www.csl.com


CSL Media Contact
Christina Hickie
Senior Manager Communications
CSL Limited
Phone: +61 429 609 762
Christina.Hickie@csl.com.au