ulviprubart (ABC008) / Abcuro - LARVOL DELTA

Paradigm shift in the pathomechanisms and treatment of inclusion body myositis: at the crossroads of cell-autonomous muscle degeneration and immune activation. (PubMed, Curr Opin Neurol) - "A molecular cascade from cell-autonomous TDP-43 dysfunction to cryptic epitope-driven immune activation could underlie the pathogenesis of IBM. Personalized multidisciplinary care integrating early diagnosis, targeted disease-modifying therapy, and advanced rehabilitation represents the emerging treatment paradigm."

Journal • Immunology • Inflammation • Myositis • CD8 • KLRG1 • NORAD • STING • TARDBP

Abcuro has raised $66 million in Series D financing led by current investor New Leaf Venture Partners, as its investigational first-in-class monoclonal antibody enters a new clinical study for inclusion body myositis (IBM), a progressive autoimmune disease with no approved treatment (citybiz) - "The financing gives Abcuro capital to pursue a regulatory pathway that could move its lead drug candidate closer to a biologics license application, or BLA, if the new study produces positive results....Abcuro said it has been in discussions with the U.S. Food and Drug Administration regarding the design and development path for the new study and is preparing to begin the trial later this year."

Financing • New trial • Immunology • Myositis

Phase 2/3 Study of a Monoclonal Antibody Targeting KLRG1 in Inclusion Body Myositis (ICNMD 2026) - P2/3 | "This is the largest prospective interventional study of patients with IBM with a range of clinically significant disease burden. Topline efficacy and safety results will be presented."

P2/3 data • Gastrointestinal Disorder • Immunology • Myositis • CD8 • KLRG1

Impact of Anti-Cytosolic Nucleotidase 1A Antibody Status on Disease Phenotype and Therapeutic Response to Ulviprubart in Inclusion Body Myositis (EULAR 2026) - P2/3 | "Conclusions Anti-cN1A positivity appears to be associated with a higher likelihood of swallowing difficulties in IBM. Responses to ulviprubart based on anti-cN1A status will be presented at the congress."

Gastrointestinal Disorder • Immunology • Myositis • CD8 • KLRG1

ABC008 in Subjects With T-cell Large Granular Lymphocytic Leukemia (T-LGLL) (clinicaltrials.gov) - P1/2 | N=21 | Completed | Sponsor: Abcuro, Inc. | Active, not recruiting ➔ Completed | Trial completion date: Sep 2027 ➔ Mar 2026

Trial completion • Trial completion date • Hematological Malignancies • Leukemia • Oncology • T-Cell Large Granular Lymphocyte Leukemia

Phase II/III Study of Ulviprubart in Inclusion Body Myositis: Study Design, Patient Characteristics, and Topline Results (AAN 2026) - P2/3 | "Conclusions This is the largest prospective interventional study of patients with IBM with a range of clinically significant disease burden. Topline efficacy and safety results will be presented."

Clinical • P2/3 data • Gastrointestinal Disorder • Immunology • Myositis • CD8 • KLRG1

Imaging CD8+ T Cells in Patients With Inclusion Body Myositis (IBM) Treated With Ulviprubart (AAN 2026) - P1 | "In this study, 2 doses of ulviprubart treatment reduced CD8+ T cells in muscle, and ulviprubart was safe and well tolerated. These results suggest that ulviprubart has pharmacodynamic effects in skeletal muscle."

Clinical • Gastrointestinal Disorder • Immunology • Myositis • CD8 • KLRG1

A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis (clinicaltrials.gov) - P2/3 | N=272 | Completed | Sponsor: Abcuro, Inc. | Active, not recruiting ➔ Completed

Trial completion • Immunology • Myositis

Clinical activity, tolerability, and pharmacodynamics of ulviprubart in patients with T-cell large granular lymphocytic leukemia: Interim Results of A phase 1 trial (ASH 2025) - "ConclusionIn this small study, Q4W dosing of ulviprubart (1.5 or 3 mg/kg) was active in depleting cytotoxic KLRG1+ Tcells and was well tolerated. There was a 53% response rate for patients with neutropenia and 22%response in patients with anemia with Q4 week dosing, supporting further clinical study."

Clinical • P1 data • PK/PD data • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • T-Cell Large Granular Lymphocyte Leukemia • B3GAT1 • CD8 • KLRG1

Abcuro Presents Interim Phase 1 Data Evaluating Ulviprubart in Patients with T Cell Large Granular Lymphocytic Leukemia at the 67th American Society of Hematology Annual Meeting (Businesswire) - "21 patients were enrolled and evaluable for safety. 95% (n=20) of patients had neutropenia and 57% (n=12) of patients had anemia at baseline. 62% (n=13) of patients achieved >12 weeks of Q4W dosing on ulviprubart. Among evaluable patients, seven experienced sustained depletions of >50% of CD8+ CD57+ KLRG1+ T cells at two or more consecutive visits. Three patients experienced sustained depletions of >90% of both CD8+ CD57+ KLRG1+ T cells and the CD8+ CD57+ parent population. Among evaluable patients, all had neutropenia and nine patients had anemia at baseline. With treatment, seven patients had a neutropenia response....With treatment, two patients had an anemia response..."

P1 data • Large Granular Lymphocyte Leukemia

Abcuro…announced that the Company will present interim data from the Phase 1/2 clinical trial evaluating the safety, tolerability, and hematological effect of ulviprubart in patients with T cell large granular lymphocytic leukemia (T-LGLL) who suffer from neutropenia and/or anemia (Businesswire) - "The interim data will be presented in an oral presentation at the 67th American Society of Hematology (ASH) Annual Meeting..."

P1 data • T-Cell Large Granular Lymphocyte Leukemia

Ulviprubart Pharmacokinetics, Pharmacodynamics (PK/PD), and Safety: Phase 1 Study Results in Patients With Inclusion Body Myositis (IBM) (EULAR 2025) - P1 | "In patients with IBM, ulviprubart led to sustained selective depletion of peripheral blood CD8+ KLRG1+ T cells and had a favorable safety profile."

Clinical • P1 data • PK/PD data • Immunology • Myositis • CD8 • KLRG1

Abcuro to Present Two Posters at the American Academy of Neurology 2025 Annual Meeting (Businesswire) - "Abcuro, Inc...today announced that the Company will present safety, pharmacokinetic, and pharmacodynamic data from the Phase 1 clinical trial evaluating ulviprubart (ABC008) for the treatment of inclusion body myositis (IBM) at the American Academy of Neurology (AAN) Annual Meeting, taking place April 5-9, 2025, in San Diego, California."

P1 data • Myositis

Pharmacokinetics, Pharmacodynamics (PK/PD), and Safety Profile of Ulviprubart: Results from a 48-week, Open-label, Phase 1 Study in Patients with Inclusion Body Myositis (IBM) (AAN 2025) - P1 | "Ulviprubart led to deep and selective depletion of peripheral blood KLRG1+ T cells in patients with IBM. Together with the favorable safety profile, these data support the continued development of ulviprubart."

Clinical • P1 data • PK/PD data • Gastrointestinal Disorder • Immunology • Myositis • CD4 • CD8 • KLRG1

A Phase 1 Study of ABC008 in Ascending (Single Ascending Dose/Multiple Ascending Dose) Study in Patients with (IBM) (clinicaltrials.gov) - P1 | N=19 | Completed | Sponsor: Abcuro, Inc. | Active, not recruiting ➔ Completed | N=30 ➔ 19 | Trial completion date: Aug 2025 ➔ Jan 2025

Enrollment change • Trial completion • Trial completion date • Immunology • Myositis

Abcuro Announces $200 Million Series C Financing to Advance Its First-In-Class Medicine in Development for Inclusion Body Myositis (Businesswire) - "Abcuro, Inc...today announced the closing of a $200 million Series C financing led by New Enterprise Associates (NEA) with Foresite Capital joining the round and participation of existing investors...Proceeds from the Series C financing will be used to complete the registrational Phase 2/3 MUSCLE clinical trial evaluating ulviprubart (ABC008), a first-in-class monoclonal antibody targeting killer cell lectin like receptor G1 (KLRG1), for the treatment of inclusion body myositis (IBM). Assuming positive results from the MUSCLE clinical trial, Abcuro plans to file a BLA and will use a portion of the proceeds to support commercial launch preparation...'We are in a strong position to execute on our clinical development plan, including completing our ongoing, registrational Phase 2/3 MUSCLE clinical trial of ulviprubart in IBM, and expect to report initial data in the first half of 2026. We will also look to fund the expansion of manufacturing capabilities and other..."

Financing • P2/3 data • Immunology • Myositis

Long-term Extension Study of Ulviprubart (ABC008) in Subjects with Inclusion Body Myositis (clinicaltrials.gov) - P2/3 | N=270 | Active, not recruiting | Sponsor: Abcuro, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Immunology • Myositis

ABC008 in Subjects with T-cell Large Granular Lymphocytic Leukemia (T-LGLL) (clinicaltrials.gov) - P1/2 | N=21 | Active, not recruiting | Sponsor: Abcuro, Inc. | Recruiting ➔ Active, not recruiting | N=30 ➔ 21 | Trial completion date: Dec 2025 ➔ Sep 2027 | Trial primary completion date: Dec 2024 ➔ Sep 2025

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Hematological Malignancies • Leukemia • Oncology

Long-term Extension Study of Ulviprubart (ABC008) in Subjects with Inclusion Body Myositis (clinicaltrials.gov) - P2/3 | N=270 | Recruiting | Sponsor: Abcuro, Inc. | Not yet recruiting ➔ Recruiting | Trial primary completion date: Aug 2028 ➔ Jan 2029

Enrollment open • Trial primary completion date • Immunology • Myositis

Long-term Extension Study of Ulviprubart (ABC008) in Subjects With Inclusion Body Myositis (clinicaltrials.gov) - P2/3 | N=270 | Not yet recruiting | Sponsor: Abcuro, Inc.

New P2/3 trial • Immunology • Myositis

A Phase 1 Study of ABC008 in Ascending (Single Ascending Dose/Multiple Ascending Dose) Study in Patients With (IBM) (clinicaltrials.gov) - P1 | N=30 | Active, not recruiting | Sponsor: Abcuro, Inc. | Trial completion date: May 2024 ➔ Aug 2025 | Trial primary completion date: Mar 2024 ➔ Mar 2025

Trial completion date • Trial primary completion date • Immunology • Myositis

A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis (clinicaltrials.gov) - P2/3 | N=231 | Recruiting | Sponsor: Abcuro, Inc.

Trial completion date • Trial primary completion date • Immunology • Myositis

A Phase 1 Study of ABC008 in Ascending (Single Ascending Dose/Multiple Ascending Dose) Study in Patients With (IBM) (clinicaltrials.gov) - P1 | N=30 | Active, not recruiting | Sponsor: Abcuro, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Immunology • Myositis

ABC008 in Subjects With T-cell Large Granular Lymphocytic Leukemia (T-LGLL) (clinicaltrials.gov) - P1/2 | N=30 | Recruiting | Sponsor: Abcuro, Inc. | N=15 ➔ 30 | Trial completion date: Dec 2023 ➔ Dec 2025 | Trial primary completion date: Dec 2023 ➔ Dec 2024

Enrollment change • Trial completion date • Trial primary completion date • Hematological Malignancies • Leukemia • Oncology • KLRG1

A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis (clinicaltrials.gov) - P2/3 | N=231 | Recruiting | Sponsor: Abcuro, Inc. | Active, not recruiting ➔ Recruiting

Enrollment open • Immunology • Myositis