CSL highlights pipeline at investor briefing

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CSL invested over A$1.2 billion in R&D in financial year 2019 as the company progressed what it described as a "highly differentiated product portfolio mix".

Speaking at its annual R&D briefing to investors, the company's head of R&D Dr William Mezzanotte highlighted its focus on new plasma therapies as well as new platforms including gene and cell therapies and recombinant proteins.

“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,” said Dr Mezzanotte, adding the company invested US$832 million (A$1.2 billion) or 9.7 per cent of total revenues into its R&D portfolio.

Dr Mezzanotte pointed to R&D highlights such as a novel treatment for asthma that recently advanced to Phase 1, first-inhuman trials for patients with mild to moderate asthma.

He said the 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, currently known as CSL311, is a monoclonal antibody that targets multiple inflammatory agents involved in various diseases.

The company said 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.

The company also highlighted a Phase 3 trial involving the 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 that are characterised 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,” said 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.”