Clinical Trial Milestones, Investments in aQIVc and sa-mRNA Influenza Vaccines and New Strategic Collaborations Among the Highlights from CSL R&D Day

- CSL advances novel pipeline spanning six therapeutic areas, four scientific platforms and two businesses (CSL Behring and Seqirus)

- Seqirus Awarded BARDA contract to develop aQIVc and sa-mRNA Pandemic Influenza Vaccines

- New collaboration with the Walter and Eliza Hall Institute for Medical Research in Australia to create a Centre for Biologic Therapies

News provided by

CSL Limited

Oct 18, 2021, 20:30 ET

MELBOURNE, Australia and KING OF PRUSSIA, Pa., Oct. 18, 2021 /PRNewswire/ -- During its annual R&D investment briefing earlier today, CSL Limited (ASX:CSL; USOTC:CSLLY) highlighted progress from its novel pipeline that spans six therapeutic areas (immunology, hematology, respiratory, cardiovascular and metabolic, transplant, influenza), four scientific platforms (plasma fractionation, recombinant technology, cell and gene therapy, vaccines) and two businesses (CSL Behring and Seqirus).

Amongst its highlights, the company plans to initiate a phase 3 study of 4-Factor Prothrombin Complex Concentrate to improve survival in traumatic injury and acute major bleeding, a leading cause of death. In the company's VANGUARD Phase 3 clinical trial for Garadacimab, a monoclonal antibody treatment for hereditary angioedema (HAE), the last patient was enrolled two months ahead of schedule. CSL also progressed recruitment for the AEGIS-II Phase 3 study of CSL112 (ApoA-1) for treatment of acute coronary syndrome despite COVID-19 impact on clinical trial sites and patients. To date, more than 14,000 people have been enrolled in this study.

"We continue to evolve as a leading plasma-based biotechnology company with purposeful diversity in therapeutic areas, scientific platforms and strategic alliances," said Dr. Bill Mezzanotte, Executive Vice President, Head of R&D, Chief Medical Officer for CSL. "We are continuing to invest in our core plasma business while also enhancing our other scientific platforms to better deliver on our promise to discover, develop and provide innovations that save and improve lives around the world."

The company's Seqirus business, a global leader in influenza prevention, highlighted its R&D investments in its first-of-its-kind adjuvanted, cell-based seasonal influenza vaccine (aQIVc) and next-generation self-amplifying mRNA for seasonal and pandemic influenza (sa-mRNA). Earlier this month, the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS), awarded Seqirus a multi-year contract to develop two influenza A(H2Nx) vaccine candidates for assessment in a Phase 1 clinical study with the goal of helping to safeguard communities in the event of an influenza pandemic.

Other Seqirus highlights for aQIVc – which combines two leading-edge technologies – the company's proprietary MF59^® adjuvant and cell-based vaccine manufacturing – included:

Phase 2 study is ongoing with plans to initiate the Phase 3 clinical trial of this promising vaccine candidate in the second half of 2022.
Both adjuvanted and cell-based influenza vaccines have potential benefits in seasonal influenza vaccine programs compared with more traditional manufacturing methods. Combining the technologies may elicit a greater immune response and potentially result in increased vaccine efficacy, particularly in the older adult population.

Other highlights for the development of Seqirus' next-generation sa-mRNA vaccine technology for seasonal and pandemic influenza included:

Progressing sa-mRNA-based influenza vaccine candidates, with pre-clinical results demonstrating potential.
Targeting the commencement of clinical trials for both seasonal and pandemic influenza vaccine candidates in the second half of 2022.

"We are deeply invested in evolving the vaccine landscape to create new, more effective ways of protecting our communities against both seasonal and pandemic influenza," said Dr. Russell Basser, Senior Vice President of R&D at Seqirus. "As a partner in public health, our job is to determine how best to utilize and expand our capabilities to deliver improved and inspired solutions. We are pleased to see such dynamic growth within the company, particularly in our influenza vaccine pipeline, which includes the optimization of current technologies and developing new, transformative approaches to future vaccine development."

Overall, CSL invested more than $1 billion in R&D the past fiscal year for the first time in the company's 100+ year history.

New research-industry partnership with the Walter and Eliza Hall Institute for Medical Research (WEHI) announced

CSL also announced it is collaborating with WEHI, one of the most prominent medical research and medicine development organizations in Australia, to create a Centre for Biologic Therapies.

The new Centre combines WEHI's expertise in immunology, cancer, inflammatory disorders, and infectious diseases with CSL's world-class human antibody library and experience in biologic medicine discovery and development.

The Centre will provide access to expert biologic discovery and optimization capabilities accelerating medicine development into the clinic, ultimately addressing a current gap in Australian medical research.

Biologics are made in living systems and encompass a wide range of therapies including monoclonal antibodies, vaccines, growth factors and cell therapies.

Based at WEHI, the Centre aims to generate high-quality and clinic-ready therapeutic antibodies against novel targets in human disease.

The partners will contribute equally to the funding of the Centre with a combined investment of approximately $10 million over five years.

"CSL's antibody library will be the engine room of biologics discovery at the Centre and, importantly, the knowledge transfer between the two organizations and the utilization of shared assets, resources, and facilities will be of great value," said Senior Vice President, Head of Research and Chief Scientific Officer Dr. Andrew Nash. "This expansion of our relationship with WEHI will help ensure that the long-term investment of public funds into medical research in Australia is translated into both a benefit for patients and the Australian economy."

Dr. Nash added, "The Centre is an interface of innovation between research and industry and sets the foundations for significant growth in the Australian biologics discovery and development space while offering learning and bespoke training opportunities for the next generation of Australian scientists, the future of Australia's biologics workforce."

For more information on this collaboration visit, https://www.wehi.edu.au/news/research-industry-partnership-advance-biologic-therapies.

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

About CSL
CSL (ASX:CSL) is a leading global biotechnology company with a dynamic portfolio of life-saving medicines, including those that treat hemophilia 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 100 countries and employs more than 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 visit www.csl.com.

Media Contacts:
In Australia:
Jimmy Baker
Global Finance Communications
Email: [email protected]
+61 450 909 211

Jo Cleary
Seqirus Communications
[email protected]
+61 428 816 751

Kim O'Donohue
R&D Communications
[email protected]
+61 449 884 603

Rest of World:
Greg Healy
R&D Communications
[email protected]
+1 610 906 4564

Polina Miklush
Seqirus Communications
[email protected]
+1 908 608 7170

SOURCE CSL Limited