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Asset Brief

Garadacimab

CSL·CSL Limited·Prevention of hereditary angioedema (HAE) attacks·Phase 3·NCT04739059·Primary completion: 21 Nov 2025

Plain-language Brief

The trial in plain terms

The disease: Hereditary angioedema (HAE) is a rare genetic condition caused by deficiency or dysfunction of C1 inhibitor, a protein that regulates certain inflammatory pathways. Patients experience recurrent episodes of severe swelling in the skin, gastrointestinal tract, and airway, which can be life-threatening. Attacks are unpredictable and significantly reduce quality of life. Long-term preventive (prophylactic) therapy is the standard goal for patients with frequent or severe attacks.
What the asset is trying to do: Garadacimab (also referred to as CSL312) is a monoclonal antibody — a laboratory-made protein designed to bind to and block a specific target in the body. Based on its development rationale in HAE, it is understood to target activated Factor XIIa, a protein in the contact activation pathway that contributes to the production of bradykinin, the mediator responsible for swelling in HAE. additional mechanism detail or published pharmacology citation. This mechanism differs from C1 inhibitor replacement therapies and from kallikrein inhibitors, which act further downstream in the same pathway.
What the trial is measuring, and why it matters: The completed Phase 3 long-term study (NCT04739059) enrolled up to 171 participants and assessed the safety and efficacy of garadacimab for the prophylactic (preventive) treatment of HAE attacks (per ClinicalTrials.gov NCT04739059). A separate ongoing observational study (NCT07001280) is enrolling up to 200 participants and is described as investigating effectiveness and safety in real-world use, with a primary completion date of 2028-08-31 (per ClinicalTrials.gov NCT07001280). A Phase 4 switching safety study (NCT06806657) targeting 18 participants aged 12 years and older was active and not recruiting as of available data, with a primary completion date of 2026-06-23 (per ClinicalTrials.gov NCT06806657).
Efficacy benchmarks in this setting: Approved prophylactic therapies for HAE provide a reference point for this setting. Lanadelumab (Takhzyro), a monoclonal antibody targeting plasma kallikrein, demonstrated a median reduction in HAE attack rate of approximately 87% versus placebo over 26 weeks in the HELP OLE study, with 44% of patients on the 300 mg every-two-weeks regimen achieving zero attacks during the treatment period (per Banerji et al, New England Journal of Medicine 2018, NCT02586805). Subcutaneous C1 inhibitor (Haegarda) reduced the number of HAE attacks by approximately 90% in the COMPACT trial versus placebo (per Zuraw et al, New England Journal of Medicine 2017, NCT01912456). Figures from early or single-arm studies of garadacimab are not statistically comparable to these randomised controlled trial results.

Sources for this brief (7)

Garadacimab study: Long-term Safety and Efficacy of CSL312 in the Prophylactic Treatment of Hereditary Angioedema Attacks · ClinicalTrials.gov · accessed 15 June 2026
Garadacimab observational effectiveness and safety study · ClinicalTrials.gov · accessed 15 June 2026
Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab · ClinicalTrials.gov · accessed 15 June 2026
Post Study Access of CSL312 (Garadacimab) for Pediatric Participants With HAE · ClinicalTrials.gov · accessed 15 June 2026
Lanadelumab for Hereditary Angioedema Prophylaxis (HELP OLE) · New England Journal of Medicine / ClinicalTrials.gov NCT02586805 · accessed 15 June 2026
Subcutaneous C1-Inhibitor for Hereditary Angioedema Prophylaxis (COMPACT trial) · New England Journal of Medicine / ClinicalTrials.gov NCT01912456 · accessed 15 June 2026
ALHEMO (garadacimab) FDA approval information

Trial Design

Design summary

Phase: Phase 3
Design type: The lead Phase 3 trial (NCT04739059) was an interventional study assessing long-term safety and efficacy. [ TODO: fill in ] open-label or blinded design details from the full trial registry record.
Primary endpoint: [ TODO: fill in ] — primary endpoint not specified in the available trial registry summary for NCT04739059.
Key secondary endpoints: [ TODO: fill in ]
Enrolment target: 171 patients (per ClinicalTrials.gov NCT04739059)
Current enrolment status: Completed (per ClinicalTrials.gov NCT04739059); primary completion date 2025-11-21
Sites: [ TODO: fill in ]
Expected readout window: The Phase 3 long-term study (NCT04739059) had a primary completion date of 2025-11-21 (per ClinicalTrials.gov NCT04739059). Full study results publication timing is [ TODO: fill in ].

Asset Track Record

Did CSL deliver against its own guidance?

Past catalysts on this ticker, reconciled against the announcement parse pipeline. Hover any row for the parsed outcome detail.

30 June 2026data readoutCSL112 Phase 3 AEGIS-II readoutoverdue
30 June 2026commercial milestoneSeqirus flu vaccine seasonoverdue
10 May 2026otherInterim CEO 90 Day Review and Financial Updatehitsrc ASX ↗
5 May 2026financingUpdate - Notification of buy-back - CSLhitsrc ASX ↗
3 May 2026financingUpdate - Notification of buy-back - CSLhitsrc ASX ↗
13 Apr 2026data readoutResults posted — An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Sehitsrc
13 Apr 2026data readoutResults posted — Dose Ranging Study of BMS-945429 in Subjects With Moderate to Severe Crohn's Dishitsrc
13 Apr 2026data readoutResults posted — Extension Study of Maintenance Treatment With Subcutaneous Immunoglobulin (IgProhitsrc

Class Catalyst Calendar

What's landed, what's next for Garadacimab and peers

Garadacimab's own upcoming catalysts alongside class-defining historical readouts and upcoming primary completion dates in the same mechanism classes. Each event moves the broader thesis for Garadacimab. Showing past 6 months and next 12 months · 17 later hidden.

ConferenceAACR Annual Meeting 2026· ChicagoApr 2026
ConferenceASCO Annual Meeting 2026· ChicagoMay 2026
Today
Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

Trial design comparison

How Garadacimab reads against active peer trials

Same axes, same units. Discontinued and historical pivotal trials excluded; see Competitor Landscape below for the full set.

Trial	Sponsor	Class	Phase / Line	Design	Primary endpoint	Dosing	Key eligibility	n
Garadacimab	CSL Limited	Monoclonal antibody	Phase 3Maintenance	The lead Phase 3 trial (NCT04739059) was an interventional study assessing long-term safety and efficacy. [ TODO: fill in ] open-label or blinded design details from the full trial registry record.	[ TODO: fill in ] — primary endpoint not specified in the available trial registry summary for NCT04739059.	[ TODO: fill in ]	171 patients (per ClinicalTrials.gov NCT04739059)	171

Competitor Landscape

Competing programmes by mechanism and phase

Read-across commentary attached per trial. Phase ordering preserved within each group.

Mechanism trade-offs· 2 classes comparedexpand ›

Mechanism	Speed to patient	Manufacturing	Best approved result	Durability	Moat
Fc Fusion ProteinFc-fusion trap	Off-the-shelf	Biologic	Eylea / Vabysmo class	Dosing-interval driven	High (incumbents)
Antisense oligonucleotideASO	Off-the-shelf	Synthetic	Approved (SMA/DMD)	Chronic dosing	Moderate

Curated Commentary

Third-party coverage(none curated yet)

expand ›

External analyst notes, journalism, and industry commentary cited with attribution.

No third-party coverage curated for Garadacimab yet. Broker coverage of CSL Limited is thin; named analyst notes will appear here as they become available. For now, see the company's ASX announcements and the linked sponsor / class sources elsewhere on this page.

Asset Track Record

Did CSL deliver against its own guidance?

Past catalysts on this ticker, reconciled against the announcement parse pipeline. Hover any row for the parsed outcome detail.

30 June 2026data readoutCSL112 Phase 3 AEGIS-II readoutoverdue
30 June 2026commercial milestoneSeqirus flu vaccine seasonoverdue
10 May 2026otherInterim CEO 90 Day Review and Financial Updatehitsrc ASX ↗
5 May 2026financingUpdate - Notification of buy-back - CSLhitsrc ASX ↗
3 May 2026financingUpdate - Notification of buy-back - CSLhitsrc ASX ↗
13 Apr 2026data readoutResults posted — An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Sehitsrc
13 Apr 2026data readoutResults posted — Dose Ranging Study of BMS-945429 in Subjects With Moderate to Severe Crohn's Dishitsrc
13 Apr 2026data readoutResults posted — Extension Study of Maintenance Treatment With Subcutaneous Immunoglobulin (IgProhitsrc

Class Catalyst Calendar

What's landed, what's next for Garadacimab and peers

ConferenceAACR Annual Meeting 2026· ChicagoApr 2026
ConferenceASCO Annual Meeting 2026· ChicagoMay 2026
Today
Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

Trial design comparison

How Garadacimab reads against active peer trials

Same axes, same units. Discontinued and historical pivotal trials excluded; see Competitor Landscape below for the full set.

Trial	Sponsor	Class	Phase / Line	Design	Primary endpoint	Dosing	Key eligibility	n
Garadacimab	CSL Limited	Monoclonal antibody	Phase 3Maintenance	The lead Phase 3 trial (NCT04739059) was an interventional study assessing long-term safety and efficacy. [ TODO: fill in ] open-label or blinded design details from the full trial registry record.	[ TODO: fill in ] — primary endpoint not specified in the available trial registry summary for NCT04739059.	[ TODO: fill in ]	171 patients (per ClinicalTrials.gov NCT04739059)	171

Competitor Landscape

Competing programmes by mechanism and phase

Read-across commentary attached per trial. Phase ordering preserved within each group.

Mechanism trade-offs· 2 classes comparedexpand ›

Mechanism	Speed to patient	Manufacturing	Best approved result	Durability	Moat
Fc Fusion ProteinFc-fusion trap	Off-the-shelf	Biologic	Eylea / Vabysmo class	Dosing-interval driven	High (incumbents)
Antisense oligonucleotideASO	Off-the-shelf	Synthetic	Approved (SMA/DMD)	Chronic dosing	Moderate

Garadacimabn = 171

CSL Limited·Monoclonal antibody·Phase 3·Maintenance

Design: The lead Phase 3 trial (NCT04739059) was an interventional study assessing long-term safety and efficacy. [ TODO: fill in ] open-label or blinded design details from the full trial registry record.
Primary endpoint: [ TODO: fill in ] — primary endpoint not specified in the available trial registry summary for NCT04739059.
Dosing: [ TODO: fill in ]
Eligibility: 171 patients (per ClinicalTrials.gov NCT04739059)

Berotralstat Treatment in Children With Hereditary Angioedeman = —

BioCryst Pharmaceuticals·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAEn = —

Pharvaris Netherlands B.V.·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacksn = —

Octapharma·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)n = —

Ionis Pharmaceuticals, Inc.·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant IR Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiencyn = —

Pharvaris Netherlands B.V.·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAEn = —

Pharvaris Netherlands B.V.·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studiesn = —

BioCryst Pharmaceuticals·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —