Next-Generation mRNA, Gene Therapy, Plasma Products, Monoclonal Antibodies, and Recent Acquisitions and Collaborations Highlight CSL R&D Day 2022

Rhea-AI Summary

CSL presented advancements in its R&D pipeline during its annual investor briefing. Key highlights include the priority review acceptance of etranacogene dezaparvovec for haemophilia B, and positive Phase III results for garadacimab, a treatment for hereditary angioedema. The company is also advancing projects in nephrology and influenza vaccines, including a collaboration with Arcturus Therapeutics for mRNA capabilities. CSL invested around $1.16 billion in R&D in fiscal 2021-2022. The newly appointed Cicada Innovations will operate a biotech incubator at CSL's Melbourne headquarters, supporting innovation-driven growth.

Positive

• Etranacogene dezaparvovec accepted for priority review by the FDA, potentially the first gene therapy for haemophilia B.
• Positive Phase III results for garadacimab; plans to file for approval next year.
• Investment of $1.16 billion in R&D to enhance innovation.
• Partnership with Arcturus Therapeutics to boost mRNA development.

Negative

• None.

Pipeline advancements and investment in innovation and disruptive technologies to help fuel sustainable, profitable growth for CSL's three businesses, CSL Behring, CSL Seqirus and CSL Vifor, in the decades ahead

MELBOURNE, Australia and KING OF PRUSSIA, Pa., Nov. 2, 2022 /PRNewswire/ -- At its annual R&D investor briefing today, global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) demonstrated how its growing, innovative pipeline is well-positioned to meet the current and future needs of patients and public health. CSL revealed progress and plans in advancing assets that have the potential to disrupt current standards of care in its areas of focus (immunology, haematology, respiratory, cardiovascular and metabolic, transplant, nephrology, vaccines) using its strategic scientific platforms (plasma protein technology, recombinant technology, cell and gene therapy, and sa-mRNA, adjuvanted, cell-based and egg-based vaccines).

Among the highlights presented were the following:

Late-Stage Development Includes Disruptive Innovation and New Additions to the CSL Portfolio

• Etranacogene dezaparvovec (also known as CSL222), an investigational gene therapy for the treatment of adults with haemophilia B has been accepted for priority review by the United States (US) Food and Drug Administration (FDA) and standard review by the European Medicines Agency (EMA). If approved, etranacogene dezaparvovec would be the first-ever gene therapy treatment option for the haemophilia B community. The regulatory filings are supported by results from the pivotal HOPE-B trial, the largest gene therapy trial in haemophilia B to date. The multi-year clinical development program for etranacogene dezaparvovec was led by uniQure (Nasdaq: QURE) and sponsorship of the clinical trials has transitioned to CSL after acquiring global rights to commercialize the investigational treatment.
• Top-line Phase III results for garadacimab (CSL312, anti-FXIIa), an investigational first-in-class monoclonal antibody being developed as a long-term preventive treatment for patients with hereditary angioedema, demonstrated that the study met its primary and secondary efficacy objectives and showed favourable safety and tolerability. CSL aims to begin filing for approval with global regulatory authorities next calendar year. Garadacimab was discovered and optimised by scientists at CSL's Bio21–based Research site, with formulation and manufacturing for the clinical programs completed at the CSL Broadmeadows Biotech Manufacturing Facility.
• CSL Vifor brings a leading portfolio of therapies in nephrology, dialysis and iron deficiency. New late-stage assets to the pipeline include Sparsentan for IgA nephropathy and focal segmental glomerulosclerosis (FSGS) and SNF472 for calciphylaxis and calcific uremic arteriolopathy (CUA).

R&D Portfolio Progress Across Areas of Focus and Scientific Platforms

• Clazakizumab, an anti-IL6 monoclonal antibody, intended for the treatment of chronic active antibody-mediated rejection (AMR) in kidney transplant recipients continues to progress in the Phase III IMAGINE trial. This is CSL's leading program for clazakizumab, which will also be investigated in a Phase IIb/III study for improvement of cardiovascular (CV) outcomes in dialysis patients. CSL plans to engage Fresenius Medical Care dialysis centres in the study.
• AEGIS-II, a Phase III, multicenter, double-blind, randomised, placebo-controlled, parallel-group study to evaluate the efficacy and safety of CSL112, compared to placebo, in reducing the risk of major adverse CV events (MACE) in patients following a heart attack, will enrol the last patient by the end of 2022, with completion of the trial expected towards the end of 2023.
• CSL is advancing the Phase IIb study for its adjuvanted, cell-based high-dose quadrivalent influenza vaccine (aQIVc). This study will help CSL further build the body of clinical evidence to support the optimal formulation for when the company moves into the Phase III immunogenicity and safety study for aQIVc.
• Published results from the preclinical studies of CSL's self-amplifying messenger RNA (sa-mRNA) influenza vaccine candidates, the next generation of mRNA vaccines, indicate a potent, cross-reactive immune response against pandemic and seasonal influenza strains, A(H5N1) and A(H1N1). CSL's sa-mRNA candidate is expected to enter clinical trials in 2023.

Recently Announced

• Collaboration and license agreement with Arcturus Therapeutics aims to accelerate next-generation mRNA capabilities in influenza, pandemic preparedness and other selected respiratory viral pathogens -- including a near term COVID-19 vaccine that has recently reported interim results from a large Phase III efficacy study, meeting its primary and secondary endpoints of prevention of infection and severe disease with a favourable safety profile.
• A strategic option and license agreement with Translational Sciences to license TS23, a first-in-class anti-α2-antiplasmin monoclonal antibody. TS23 is being developed to dissolve thrombi that cause serious conditions such as pulmonary embolism (PE) and acute ischemic stroke (AIS). The treatment candidate is soon to be evaluated in the US in the NAIL-IT Phase II study, which has been designed to evaluate the safety and thrombolytic effect of ascending doses of TS23 in patients with sub-massive (intermediate risk) PE.

"CSL is on the leading-edge of innovation in areas we know well and we have strategically and methodically built a pipeline that has never been more robust with diverse sources of innovation, from in-house and external sources, that include the disruptive scientific platforms of gene therapy and sa-mRNA," said Dr. Bill Mezzanotte, Executive Vice President, Head of R&D, and Chief Medical Officer for CSL. "Our enhanced capabilities across all of our scientific platforms and therapeutic focus areas will help us in our relentless pursuit to deliver on our promise to help patients lead full lives, protect public health and sustainably grow our business in the decades ahead, while providing promising futures for our employees."     

In fiscal year 2021-2022, CSL invested approximately $1.16 billion in R&D. This includes growing CSL R&D's footprint in Melbourne, Australia; Marburg, Germany; throughout Switzerland and Waltham, Massachusetts – helping to create an integrated global organization that can conveniently collaborate with institutions everywhere -- offering scientists a wide array of opportunities for professional development and enhancing access to external innovation.

Advancing External Innovation: The Biotechnology Incubator at CSL's Global Headquarters in Melbourne, Australia Names Its Operator

CSL, WEHI and The University of Melbourne have appointed Cicada Innovations as the independent operator of the new biotech incubator which will be located in CSL's new global headquarters currently under construction in the Melbourne Biomedical Precinct. The appointment follows the project partners joining forces to create an incubator for biotech start-up companies, with the Victorian Government's landmark Breakthrough Victoria Fund providing funding to support the AU$95 million project. It will be Australia's first and only incubator that is co-located with a leading biopharmaceutical company and will have space for up to 40 start-ups.

"Incubator residents will be working in an innovation-driven environment alongside a large and focused CSL R&D team, enabling opportunities for peer-collaboration, learning and sharing of ideas," said Dr. Andrew Nash, CSL's Chief Scientific Officer and Senior Vice President, Research. "The strong collaboration between CSL, the University of Melbourne, WEHI, Breakthrough Victoria and now Cicada Innovations has been critical to bring the incubator to fruition and reflects CSL's values and desire to deliver on our promise to patients worldwide."

For more about the incubator, https://www.csl.com/news/2022/20221103-cicada-innovations-to-operate-new-biotech-incubator.

For more on CSL's R&D Investor Briefing, please visit https://www.csl.com/.

About CSL
CSL Limited (ASX: CSL; USOTC: CSLLY) is a leading global biotechnology company with a dynamic portfolio of lifesaving 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 three businesses, CSL Behring, CSL Seqirus, and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs more than 30,000 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 inspiring stories about the promise of biotechnology, visit CSLBehring.com/Vita and follow us on Twitter.com/CSL.

For more information visit  www.csl.com.

Media Contacts: Greg Healy
Email: Greg.Healy@cslbehring.com
+1 610 906 4564

In Australia: Jimmy Baker
Email: Jimmy.Baker@csl.com.au
+61 450 909 211

Kim O'Donohue
Email: Kim.ODonohue@csl.com.au
+61 449 884 603

In Europe:
Jasmin Joller
Email: jasmin.joller@cslbehring.com

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FAQ

What are the key highlights from CSL's R&D Investor Briefing on November 2, 2022?

CSL announced the priority review of etranacogene dezaparvovec for haemophilia B, positive Phase III results for garadacimab, and investment in a biotech incubator in Melbourne.

What is etranacogene dezaparvovec and why is it significant?

Etranacogene dezaparvovec is an investigational gene therapy for haemophilia B, notable for being the first potential gene therapy treatment for this condition.

How much did CSL invest in R&D for fiscal 2021-2022?

CSL invested approximately $1.16 billion in R&D during fiscal 2021-2022.

What is the goal of the partnership with Arcturus Therapeutics?

The partnership aims to accelerate next-generation mRNA capabilities, including the development of a COVID-19 vaccine.

What will the new biotech incubator in Melbourne do?

The incubator will support biotech start-ups and foster innovation by providing collaborative workspaces and resources.