ONC001 / OncoPrecision - LARVOL DELTA

A dose-finding population pharmacokinetic/pharmacodynamic model of ginisortamab, an anti-gremlin-1 monoclonal antibody, in patients with solid tumours. (PubMed, Br J Clin Pharmacol) - P1/2 | "This population PK/PD model adequately described ginisortamab PK and binding to serum gremlin-1 in patients with advanced solid tumours. Dose simulations informed the level of gremlin-1 suppression and supported further clinical evaluation of several dosing regimens."

Journal • PK/PD data • Oncology • Solid Tumor

Preclinical development of a first-in-class CD64-ADC to target leukemic monocytes and tumor-associated macrophages (AACR 2026) - "Importantly, we extensively investigated the safety of ONC001 by evaluating its on-target and off-target activity in multiple animal models, and observed remarkable tolerability with no signs of toxicity. Together, these preclinical findings demonstrate a favorable therapeutic index for ONC001, supporting its potential as a powerful agent for depleting pathogenic monocytes in cancer treatment."

ADC • IO biomarker • Preclinical • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • Solid Tumor • Triple Negative Breast Cancer

Phase 1 dose-optimization study evaluating the safety of ginisortamab (UCB6114), an anti-gremlin-1 monoclonal antibody (mAb) with first-in-class potential, as monotherapy in advanced solid tumors (AACR 2025) - P1/2 | "ONC001 (NCT04393298) is a first-in-human, nonrandomized, open-label, Phase 1/2 study assessing ginisortamab ± standard of care (SoC) in advanced solid tumors; results from Part A (monotherapy dose-escalation) have been reported previously.1 We present primary (safety), secondary (pharmacokinetics), and exploratory (biomarker, efficacy) endpoints from Part A1 (monotherapy dose-optimization); patients (pts) were aged ≥18 years with advanced disease resistant or refractory to SoC; ginisortamab was administered as 1 of 4 intravenous dose schedules (2000 mg Q2W 30- or 60-min infusion, 4000 mg Q4W 120-min, 28-day cycle; 3000 mg Q3W 90-min 21-day cycle). In total, 32 pts received ≥1 ginisortamab dose (n=8 for each dose schedule); median age 63 (39-82) years, 59% male, median prior anti-cancer therapies 3.5 (1-8); most common tumor type colorectal adenocarcinoma (81%). In total, 32 pts received ≥1 ginisortamab dose (n=8 for each dose schedule); median age 63 (39-82)..."

Clinical • First-in-human • Metastases • Monotherapy • P1 data • Colorectal Adenocarcinoma • Colorectal Cancer • Oncology • Pancreatic Cancer • Solid Tumor • SMAD1

ONC001: A first-in-class ADC targeting CD64 for the treatment of monocytic leukemia developed through patient-guided target and payload selection (ASH 2025) - "Notably, strong single agent ex vivo activity is observed in CMML and M4/M5 AML patients,substantially outperforming SoC drugs such as Azacitidine (Aza) and Venetoclax (Ven). By leveraging high-throughput patient-derived biology we exposed a bona fide vulnerability of malignantmonocytic cells that can be therapeutically exploited by our first-in-class ADC targeting CD64. Given theunique expression profile of CD64 across the monocytic differentiation lineage, with low/null expressionin the lymphoid compartment and other non-hematologic tissues, ONC001 emerges as a promising newtreatment with monotherapy potential in areas of high unmet need such as CMML and M4/M5 AML."

Clinical • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • CD14 • CD34

OncoPrecision Announces Oral Presentation of ONC001, a First-in-Class CD64-Targeting ADC for Monocytic Leukemia, at the 67th ASH Annual Meeting (PRNewswire) - "Preclinical data to be presented by Dr. Gastón Soria, Co-Founder and Chief Scientific Officer, will highlight: Selective engagement of patient-derived CMML and AML cells; Robust activity in samples resistant to current standard-of-care therapies; Compelling in vivo efficacy across heterotopic and orthotopic models."

Preclinical • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia

Results from the First Phase 1 Clinical Study of DR-01, a Non-Fucosylated Anti-CD94 Targeting Antibody in Patients with Relapsed/Refractory Cytotoxic Lymphomas: Dose Escalation and Optimization (ASH 2024) - P1/2 | "An encouraging response rate including 3 durable CRs points to the potential for DR-01 as a viable treatment option for CTL. Study DR-01-ONC-001 is currently enrolling an international cohort of patients with CTL into phase 2, part B2 of the trial."

Clinical • P1 data • Bone Marrow Transplantation • Constipation • Extranodal Natural Killer/T-cell Lymphoma • Fatigue • Gastroenterology • Gastrointestinal Disorder • Hematological Malignancies • Lymphoma • Oncology • CD8 • KLRD1