zamubafusp alfa (AT-02) / Attralus - LARVOL DELTA

Attralus Has Been Granted U.S. FDA Fast Track Designation for Zamubafusp Alfa (AT-02) for the Treatment of AL Amyloidosis (The Manila Times) - "Zamubafusp alfa, the company’s lead pan-amyloid removal candidate, has been evaluated in a completed Phase 1 study and an ongoing Phase 2 open-label trial, both of which have enrolled patients with AL amyloidosis. The granting of Fast Track designation indicates that clinical data from the Phase 1 / 2 program demonstrate that zamubafusp alfa has the potential to address an important unmet medical need in AL amyloidosis....The Fast Track designation was granted based on the interim AL amyloidosis data from the Phase 2 AT02-003 study...."

Fast track • Amyloidosis • Hematological Disorders

The antibody-peptide fusion protein, AT-02, is an effective opsonin with pan-amyloid reactivity. (PubMed, NPJ Drug Discov) - "We demonstrate that AT-02 is an effective opsonin for amyloid by enhancing phagocytosis of amyloid extracts and inducing complement fixation. These data suggest that pan-amyloid binding reagents may provide novel treatment options for amyloid removal."

Journal • Amyloidosis

Attralus Therapeutic Zamubafusp Alfa (AT-02) Receives U.S. FDA Orphan Drug Designation for the Treatment of AL Amyloidosis (The Manila Times)

Orphan drug • Amyloidosis

A Study of AT-02 in Subjects With Systemic Amyloidosis. (clinicaltrials.gov) - P2 | N=120 | Active, not recruiting | Sponsor: Attralus, Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Feb 2026 ➔ Sep 2026 | Trial primary completion date: Feb 2026 ➔ Sep 2026

Enrollment closed • Trial completion date • Trial primary completion date • Amyloidosis

Renal responses in a phase 1/2 study of at-02, a novel pan-amyloid depleter ig fusion protein for the treatment of patients with AL amyloidosis (ASH 2025) - "Improvements in eGFR were observed in participantswith kappa or lambda iFLC, in participants with hematologic VGPR or CR and in participants withhematologic responses ranging from 1-6 years prior to initiating AT-02 treatment.One participant in the q2w dose cohort also met the proteinuria criteria with a baseline uACR 1349 mg/g.uACR decreased by 70% and 84% following 8 and 40 weeks of treatment with AT-02 2500 mg q2w,respectively. The early rapid decline in uACR was followed by a slower progressive decline over thetreatment period.No renal evaluable AL participant developed new or worsening proteinuria.ConclusionThese initial data support the potential for AT-02, a novel pan-amyloid depleting Ig fusion protein, toimprove renal function in AL patients with renal disease who have achieved hematologic response."

Clinical • P1/2 data • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Chronic Kidney Disease • Nephrology • Renal Disease

Attralus Therapeutic AT-02 Receives Orphan Drug Designation from the U.S. FDA for the Treatment of ATTR Amyloidosis (GlobeNewswire) - "Attralus, Inc...announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for AT-02 for the treatment of transthyretin-associated amyloidosis (ATTR), a rare, progressive, debilitating and often fatal condition."

Orphan drug • Amyloidosis • Metabolic Disorders

Attralus Receives European Medicines Agency Committee for Orphan Medicinal Products (COMP) Positive Opinions for AT-02 for the Treatment of Both ATTR and AL Amyloidosis (GlobeNewswire) - "Attralus, Inc...announced today that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has adopted positive opinions for orphan medicinal product designations for AT-02 for the treatment of the two most common forms of systemic amyloidosis, transthyretin-associated amyloidosis (ATTR) and immunoglobulin light-chain-associated (AL) amyloidosis."

European regulatory • Amyloidosis • Metabolic Disorders

AT02-001: Study of AT-02 in Healthy Volunteers and Subjects With Systemic Amyloidosis (clinicaltrials.gov) - P1 | N=100 | Recruiting | Sponsor: Attralus, Inc. | Trial completion date: Mar 2024 ➔ Mar 2025 | Trial primary completion date: Mar 2024 ➔ Mar 2025

Trial completion date • Trial primary completion date • Amyloidosis

A Study of AT-02 in Subjects With Systemic Amyloidosis. (clinicaltrials.gov) - P2 | N=120 | Recruiting | Sponsor: Attralus, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Amyloidosis • NPPB

The Peptide Fusion Immunoglobulin, AT-02, Exhibits Highly Potent Pan-Amyloid Reactivity And Immunomodulation (HFSA 2023) - "Clearance of cardiac amyloid is a significant clinical unmet need for patients with systemic amyloidosis. The humanized IgG1-peptide fusion, AT-02, potently binds cardiac amyloid, inhibits fibril growth and may serve as an effective opsonin to induce the macrophage-mediated phagocytosis and clearance of cardiac amyloid deposits."

Immunomodulating • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy • Immunology

A Study of AT-02 in Subjects With Systemic Amyloidosis. (clinicaltrials.gov) - P2 | N=120 | Not yet recruiting | Sponsor: Attralus, Inc. | N=80 ➔ 120

Enrollment change • Amyloidosis • NPPB

A Study of AT-02 in Subjects With Systemic Amyloidosis. (clinicaltrials.gov) - P2 | N=80 | Not yet recruiting | Sponsor: Attralus, Inc.

New P2 trial • Amyloidosis • NPPB

AT02-001: Study of AT-02 in Healthy Volunteers and Subjects With Systemic Amyloidosis (clinicaltrials.gov) - P1 | N=100 | Recruiting | Sponsor: Attralus, Inc. | N=39 ➔ 100 | Trial completion date: Sep 2023 ➔ Mar 2024 | Trial primary completion date: Sep 2023 ➔ Mar 2024

Enrollment change • Trial completion date • Trial primary completion date • Amyloidosis

AT02-001: Study of AT-02 in Healthy Volunteers and Subjects With Systemic Amyloidosis (clinicaltrials.gov) - P1 | N=39 | Recruiting | Sponsor: Attralus, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Amyloidosis

AT02-001: Study of AT-02 in Healthy Volunteers and Subjects With Systemic Amyloidosis (clinicaltrials.gov) - P1 | N=39 | Not yet recruiting | Sponsor: Attralus, Inc.

New P1 trial • Amyloidosis