sefaldin (ABD-3001) / Advanced BioDesign - LARVOL DELTA

ALDH-Inhibitor (ABD-3001) Plus Azacitidine-Venetoclax Induce MRD-Negative Remission in TP53 Double-Hit Acute Undifferentiated Leukemia Refractory to Flag-IDA-VEN (SOHO 2026) - "Introduction: AUL is a rare entity associated with poor outcome, particularly in the presence of TP53 alteration. Mechanistically, resistance to venetoclax has been linked to altered apoptotic priming and metabolic adaptations, particularly in TP53-mutated disease. ALDH inhibition can enhance oxidative stress and lower the apoptotic threshold, thereby restoring sensitivity to BCL-2 inhibition. ALDH inhibition can be particularly effective following anthracycline-based chemotherapy-the standard first-line backbone in acute myeloid leukemia-supporting its use after intensive chemotherapy."

Clinical • IO biomarker • Minimal residual disease • Acute Myelogenous Leukemia • B Cell Lymphoma • Leukemia • Lymphoma • Oncology • Sarcoma • Solid Tumor • TP53

ALDH1 inhibition by DIMATE maintains immune responses and promotes immunogenic remodeling in AML. (PubMed, Front Immunol) - "In parallel, DIMATE induced pro-inflammatory and endoplasmic reticulum stress pathways in AML cells, upregulated co-stimulatory molecules, and promoted ICD-compatible features, including ecto-calreticulin exposure. These findings suggest that DIMATE may couple immune preservation with enhanced leukemic immunogenicity, supporting its potential for combination with immune-engaging therapies and strategies targeting measurable residual disease in AML, but also as a single agent capable of fostering a more immunostimulatory anti-leukemic context."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ALDH1A1 • CALR

ODYSSEY: A FIRST-IN-HUMAN STUDY OF THE ALDEHYDE DEHYDROGENASE (ALDH) INHIBITOR ABD-3001 IN PATIENTS WITH REFRACTORY/RELAPSED ACUTE MYELOID LEUKEMIA (AML) OR HIGH-RISK MYELODYSPLASTIC SYNDROME (MDS) (EHA 2026) - P1/2 | "Pts received a median of 6 prior lines (range 2–69); 62.9% were previously exposed to venetoclax + azacitidine; 12.5% were post ‑ HCT. PD data confirmed on ‑ target ALDH1 inhibition and downstream pathway activation. These findings support ongoing evaluation of ABD ‑ 3001 to determine the RP2D and guide further clinical development in R/R AML and HR ‑ MDS."

Clinical • First-in-human • P1 data • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Interstitial Lung Disease • Leukemia • Myelodysplastic Syndrome • Neutropenia • Pneumonia • Pulmonary Disease • ALDH1A1 • FLT3 • KRAS • TP53

Odyssey: A first-in-human study of the aldehyde dehydrogenase (ALDH) inhibitor abd-3001 in patients with refractory/relapsed acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) (ASH 2025) - P1/2 | "Mediannumber of prior therapies was 3 cycles (range 2–10); 81% had prior venetoclax + azacitidine exposureand 2 pts (10%) were post-HCT. PD data confirmed target engagement and biologicalactivity in peripheral blasts. These findings support continued evaluation of ABD-3001 in order to select apotentially optimal dose for further clinical development."

Clinical • First-in-human • P1 data • Acute Myelogenous Leukemia • Back Pain • Hematological Disorders • Hematological Malignancies • Interstitial Lung Disease • Leukemia • Musculoskeletal Pain • Myelodysplastic Syndrome • Neutropenia • Pneumonia • Pulmonary Disease • ALDH1A1 • FLT3 • KRAS • TP53

Odyssey: A First-in-Human Study of the Aldehyde Dehydrogenase (ALDH) Inhibitor Abd-3001 in Patients with Refractory/Relapsed Acute Myeloid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS) (ASH 2024) - P1/2 | "Although efficacy was not a predefined objective, early signs of hematologic improvements were observed with three patients benefiting from a long-term treatment effect. Ongoing Part B, evaluating up to three cycles of ABD-3001 at doses informed by Part A, will provide a larger data set to further evaluate tolerability and preliminary efficacy signals to guide future studies."

Clinical • First-in-human • P1 data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • ALDH1A1

Expression of Aldehyde Dehydrogenase 1A1 in Relapse-Associated Cells in Acute Myeloid Leukemia. (PubMed, Cells) - "The ODYSSEY clinical trial is a Phase I/II study designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ABD-3001, a novel therapeutic agent, in patients with AML who have relapsed or are refractory to standard treatments. In this context, ABD-3001 is used as an inhibitor of cytosolic ALDH1 enzymes, such as ALDH1A1 and ALDH1A2."

Journal • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ALDH1A1 • ALDH1A2

ODYSSEY: A FIRST-IN-HUMAN STUDY OF FIRST-IN-CLASS INHIBITOR OF ALDEHYDE DEHYDROGENASE (ALDH), ABD-3001, IN PATIENTS WITH REFRACTORY/RELAPSED ACUTE MYELOID LEUKEMIA (AML) OR HIGH-RISK MYELODYSPLASTIC SYNDROME (MDS) (EHA 2025) - P1/2 | "Part A demonstrated that ABD-3001 was well tolerated with single administration. Early signs of hematologic improvements were observed, leading to compassionate use. The ongoing Part B, which evaluates up to three cycles of ABD-3001 at various doses, indicates that ABD-3001 has high tolerability and shows promising antileukemic activity."

Clinical • P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • ALDH1A1

ODYSSEY: First-in-human Study Aiming to Characterize the Safety, Tolerability, Pharmacokinetic and Preliminary Signs of Activity of ABD-3001 in Refractory or Relapsed AML and High Risk MDS Adult Patients (clinicaltrials.gov) - P1/2 | N=36 | Recruiting | Sponsor: Advanced BioDesign | Phase classification: P1 ➔ P1/2

Monotherapy • Phase classification • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

ODYSSEY: First-in-human Study Aiming to Characterize the Safety, Tolerability, Pharmacokinetic and Preliminary Signs of Activity of ABD-3001 in Refractory or Relapsed AML and High Risk MDS Adult Patients (clinicaltrials.gov) - P1 | N=7 | Recruiting | Sponsor: Advanced BioDesign | Active, not recruiting ➔ Recruiting

Enrollment open • Monotherapy • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Reactive oxygen species and aldehyde dehydrogenase 1A as prognosis and theragnostic biomarker in acute myeloid leukaemia patients. (PubMed, J Cell Mol Med) - P1 | "In multivariate analysis, ROS level was the most significant (p < 0.05) and the strongest predictive factor for the sensitivity of cells to ABD-3001. The safety profile of ABD-3001 is currently being assessed through the first inhuman multicenter phase 1 clinical trial "ODYSSEY" (NCT05601726) for patients with relapsed AML."

Biomarker • Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ALDH1A1

ODYSSEY: First-in-human Study Aiming to Characterize the Safety, Tolerability, Pharmacokinetic and Preliminary Signs of Activity of ABD-3001 in Refractory or Relapsed AML and High Risk MDS Adult Patients (clinicaltrials.gov) - P1 | N=7 | Active, not recruiting | Sponsor: Advanced BioDesign | Trial completion date: Dec 2024 ➔ Dec 2026 | Trial primary completion date: Oct 2024 ➔ Jun 2026

Monotherapy • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Advanced BioDesign releases positive data from ODYSSEY AML study (Clinical Trials Arena) - "Advanced BioDesign has released the first data from its ODYSSEY study evaluating the potential of its drug candidate ABD-3001 (Sefaldin) in treating acute myeloid leukaemia (AML) in relapsed patients who have proven resistant to standard therapies....Initiated 15 months ago, the Phase I/II study has an adaptive design with an initial single ascending dose in six patient cohorts. Preliminary signs of Sefaldin’s efficacy on ALDH1 have been observed in more than 65% of patients treated. Biological activity markers – the inhibition of target enzyme ALDH1A1 activity and c-Jun N-terminal kinases (JNK) protein phosphorylation – demonstrated the action of ABD-3001 from the first dose."

P1/2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

ODYSSEY: First-in-human Study Aiming to Characterize the Safety, Tolerability, Pharmacokinetic and Preliminary Signs of Activity of ABD-3001 in Refractory or Relapsed AML and High Risk MDS Adult Patients (clinicaltrials.gov) - P1 | N=7 | Active, not recruiting | Sponsor: Advanced BioDesign

Monotherapy • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

ODYSSEY: First-in-human Study Aiming to Characterize the Safety, Tolerability, Pharmacokinetic and Preliminary Signs of Activity of ABD-3001 in Refractory or Relapsed AML and High Risk MDS Adult Patients (clinicaltrials.gov) - P1 | N=3 | Active, not recruiting | Sponsor: Advanced BioDesign | Recruiting ➔ Active, not recruiting | N=50 ➔ 3

Enrollment change • Enrollment closed • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

ODYSSEY: First-in-human Study Aiming to Characterize the Safety, Tolerability, Pharmacokinetic and Preliminary Signs of Activity of ABD-3001 in Refractory or Relapsed AML and High Risk MDS Adult Patients (clinicaltrials.gov) - P1 | N=50 | Recruiting | Sponsor: Advanced BioDesign

Monotherapy • New P1 trial • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology