CSL Announces Positive Top-Line Phase 3 Results for Garadacimab as Preventive Treatment in Patients with Hereditary Angioedema (HAE)

Rhea-AI Summary

CSL announced positive top-line Phase 3 results for its investigational monoclonal antibody, garadacimab (CSL312), developed as a preventive treatment for hereditary angioedema (HAE). The study successfully met primary and secondary efficacy objectives, demonstrating favorable safety and tolerability. CSL plans to file for regulatory approvals by the end of the current fiscal year and will present full results at an upcoming scientific congress. Garadacimab targets Factor XIIa to inhibit HAE attack cascades, offering a potentially transformative therapy for patients with HAE.

Positive

• Positive top-line Phase 3 results for garadacimab as a preventive HAE treatment.
• Study met primary and secondary efficacy objectives with favorable safety results.
• Plans to file for regulatory approvals by the end of the fiscal year.

Negative

• None.

CSL is on track to file for regulatory approvals at the end of the current fiscal year and will present full data set at an upcoming scientific congress

KING OF PRUSSIA, Pa., Aug. 17, 2022 /PRNewswire/ -- Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY), today announced positive top-line Phase 3 results for garadacimab (CSL312), the company's investigational first-in-class monoclonal antibody inhibiting Factor XIIa being developed as a long-term preventive treatment for patients with hereditary angioedema (HAE).  The study met its primary and secondary efficacy objectives and also demonstrated favorable safety and tolerability. CSL aims to begin filing with global health authorities at the end of the current fiscal year for full approval.

The multicenter, double-blind, randomized, placebo-controlled, parallel-arm study (also known as VANGUARD) evaluated the efficacy and safety of monthly subcutaneous garadacimab administration in the prevention of HAE attacks compared to the placebo for six months. Full results from the study will be presented at an upcoming scientific congress and published in a peer-reviewed journal.

"These results underscore our belief that garadacimab has the potential to become a transformative first-in-class therapy for people living with HAE, a patient group that CSL has been serving for many years," said Dr. Bill Mezzanotte, Executive Vice President, Head of R&D, Chief Medical Officer for CSL. "CSL's promise to patients guides us to meet their unmet need by pursuing the type of disruptive innovation we believe garadacimab represents.  We look forward to sharing the full results of our phase 3 study in the coming months."

About HAE and Garadacimab 
HAE is a rare, genetic and potentially life-threatening condition that causes painful, debilitating and unpredictable episodes of swelling of the abdomen, larynx, face and extremities, among other areas of the body. 

Garadacimab is a novel Factor XIIa-inhibitory monoclonal antibody (FXIIa mAb) currently in Phase 3 clinical development as a new type of once-monthly subcutaneous prophylactic treatment for attacks related to HAE, a form of bradykinin-mediated angioedema. Garadacimab uniquely inhibits the plasma protein, FXIIa. When FXIIa is activated, it initiates the cascade of events leading to edema formation. By targeting FXIIa, garadacimab inhibits the HAE cascade at its origin as compared with other HAE therapies that target downstream mediators.  Garadacimab was discovered and optimized 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.

Orphan-drug designation for garadacimab as an investigational therapy for hereditary angioedema has been granted by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

CSL is also investigating garadacimab for other indications, beyond HAE, where FXIIa inhibition may play an important role in improving clinical outcomes, including pulmonary fibrosis.

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 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
R&D Communications
Greg.Healy@cslbehring.com 
+1 610 906 4564

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

Kim O'Donohue
R&D Communications
Kim.ODonohue@csl.com.au 
+61 449 884 603

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SOURCE CSL

FAQ

What are the Phase 3 results for CSL's garadacimab (CSLLY)?

CSL reported positive top-line results from the Phase 3 study of garadacimab, meeting both primary and secondary efficacy objectives.

When will CSL file for regulatory approvals for garadacimab (CSLLY)?

CSL plans to file for regulatory approvals at the end of the current fiscal year.

What is the significance of garadacimab for hereditary angioedema (HAE)?

Garadacimab represents a potentially transformative therapy for patients with HAE, targeting Factor XIIa to prevent attacks.

Where will the full data for the garadacimab study be presented?

Full results from the garadacimab study will be presented at an upcoming scientific congress.

Which regulatory agencies are involved with garadacimab (CSLLY)?

Garadacimab has received orphan-drug designation from both the U.S. FDA and the European Medicines Agency.