JBZ-001 / Jabez Biosciences - LARVOL DELTA

Jabez Biosciences Presents First-in-Human Pharmacokinetic and Pharmacodynamic Data for JBZ-001 at AACR Annual Meeting 2026 (GlobeNewswire) - "A total of 15 patients have been enrolled across the first four dose levels - 3 patients at 5 mg and 4 patients each at 10 mg, 17.5 mg, and 25 mg - with no dose-limiting toxicities reported as of March 2026. The best response observed to date is stable disease....JBZ-001 demonstrated approximately dose-proportional increases in exposure following both single and multiple doses, with low to moderate interindividual variability across all dose levels evaluated....The trial is currently enrolling patients at Dose Level 5 (32.5 mg daily)."

Enrollment status • First-in-human • P1 data • PK/PD data • Non-Hodgkin’s Lymphoma • Solid Tumor

Dihydroorotate dehydrogenase (DHODH) inhibition as a promising therapeutic strategy with synergistic targeting of ROR1 in small cell lung cancer (AACR 2026) - "To evaluate the effect of DHODH inhibition on SCLC tumor growth in vivo, we treated xenograft models with HOSU-53 alone or in combination with etoposide plus cisplatin. Together, these data demonstrated the promising efficacy of HOSU-53 as single agent as well as in combination with KAN0441571C in treatment-resistant SCLC models. These findings support the initiation of a phase l clinical trial to evaluate the preliminary efficacy and tolerability of HOSU-53 and support the strategy of targeting DHODH and ROR1 as a potential therapeutic approach for the treatment of SCLC."

Lung Cancer • Oncology • Small Cell Lung Cancer • Solid Tumor • ROR1

Clinical pharmacokinetic and pharmacodynamic of JBZ-001 (HOSU-53): Comparison of first-in-human data with translational preclinical predictions (AACR 2026) - P1 | "Early clinical PK/PD data for JBZ-001 support the predictive performance of the MIDD framework based on preclinical studies. Concordance between predicted and observed DHO suppression provides confidence in exposure-response assumptions guiding dose selection. Integration of updated PK/PD data with model-informed simulations will continue to refine the optimal biological dose and support data-driven decision-making as the Phase 1 study progresses.Clinical Trial Registration: NCT06801002, funded by Jabez Biosciences Inc."

First-in-human • P1 data • PK/PD data • Preclinical • Hematological Malignancies • Oncology

Promising therapeutic effects of pyrimidine synthesis inhibition by a novel dihydroorotate dehydrogenase inhibitor in small cell lung cancer (AACR 2025) - "To evaluate the effect of DHODH inhibition on SCLC tumor growth in vivo, we treated xenograft mice models with HOSU-53 alone or in combination with etoposide plus cisplatin as the standard chemotherapy for SCLC. Together, these data demonstrated the promising efficacy of HOSU-53 in different SCLC subtypes and support the strategy of targeting DHODH as a potential therapy to treat SCLC. These findings support the initiation of a phase l clinical trial to evaluate the preliminary efficacy and tolerability of HOSU-53; therefore, we have initiated a first-in-human phase I trial of this agent."

First-in-human • Hematological Malignancies • Leukemia • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Small Cell Lung Cancer • Solid Tumor

An open-label phase 1 study to investigate JBZ001 in adults with advanced solid tumors and non-Hodjkin lymphoma (JBZ001, trial in progress) (AACR 2025) - "Safety and antitumor activity endpoints will be summarized using descriptive statistics. Recruitment is ongoing for Part 1 at The Ohio State University Comprehensive Cancer Center (WCG IRB 20245148)."

Clinical • Metastases • P1 data • Colorectal Cancer • Gastric Cancer • Hematological Malignancies • Lung Cancer • Lymphoma • Melanoma • Non-Hodgkin’s Lymphoma • Oncology • Small Cell Lung Cancer • Solid Tumor

The Potent Dihydroorotate Dehydrogenase Inhibitor, Hosu-53, Exhibits Compelling Monotherapy Efficacy in Multiple Myeloma and Augments CD47 Targeted Therapy (ASH 2023) - "Among the most promising MM SOC agents is monoclonal antibodies (mAb) such as the CD38 antibody daratumumab, which significantly improved the management of newly diagnosed and relapsed/refractory MM...We further verified HOSU-53 efficacy using the disseminated MM1.S luciferase CDX model and found a significant prolonged survival in the HOSU-53 cohort (median survival53-days) compared to vehicle (median survival28-days) that was further enhanced with isatuximab combination resulting in superior survival benefit (median survival 69-days)...Currently there is significant clinical interest in CD47 antibody therapy such as magrolimab for both solid tumors and hematological malignancies...In summary, we show compelling survival benefit for HOSU-53 as a monotherapy which is further enhanced when combined with anti-CD38 or anti-CD47 therapies. HOSU-53 is expected to enter phase 1 clinical trials in 2024 and our data is supportive for its expansion into MM."

Clinical • IO biomarker • Monotherapy • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology • Solid Tumor • CALR

Jabez Biosciences, Inc. Opens Second Clinical Site in Phase 1 Oncology Clinical Study of JBZ-001 (GlobeNewswire) - "The Phase 1 study is now open and enrolling patients at a second site, START Mountain Region....The phase 1 study is evaluating the safety, tolerability, and preliminary efficacy of JBZ-001 in patients with advanced solid tumors and non-Hodgkin lymphoma (NHL)."

Trial status • Non-Hodgkin’s Lymphoma • Solid Tumor

Multi-institutional team awarded NCI grant to open novel AML trial (Eurekalert) - "The University of Cincinnati Cancer Center (UC), The Ohio State University Comprehensive Cancer Center - Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC - James) and Jabez Biosciences are partnering to open a new phase 1 clinical trial studying JBZ-001, a potential new treatment for acute myeloid leukemia (AML). The trial is supported by a more than $3.4 million grant from the National Institutes of Health’s National Cancer Institute awarded to UC and OSUCCC - James....The Phase 1 clinical study in AML will open at UC and OSU in 2026. This study aims to assess the safety, tolerability and preliminary efficacy of JBZ-001 in patients with AML."

Financing • New P1 trial • Acute Myelogenous Leukemia

Jabez Biosciences, Inc. Announced Phase 1 Clinical Trial of JBZ-001 at AACR 2025 (ACCESSWIRE) - "Jabez Biosciences...proudly shared the progress of its Phase 1 clinical trial for JBZ-001 (HOSU-53), a novel dihydroorotate dehydrogenase (DHODH) inhibitor, at the American Association for Cancer Research (AACR) Annual Meeting held April 25-30, 2025, in Chicago, IL....The Phase 1 study, underway at OSUCCC - James, aims to assess the safety, tolerability, and preliminary efficacy of JBZ-001 in patients with advanced solid tumors and non-Hodgkin lymphoma. The trial's open-label design allows for real-time evaluation of the drug's effects, paving the way for future clinical development."

Trial status • Non-Hodgkin’s Lymphoma • Solid Tumor

Translational Pharmacokinetic-Pharmacodynamic Modeling of a Novel Oral Dihydroorotate Dehydrogenase (DHODH) Inhibitor, HOSU-53 (JBZ-001). (PubMed, Pharmaceutics) - "Additionally, predicted human hepatocellular HOSU-53 concentrations were obtained from a physiologically based PK model constructed in PK-Sim. A first-in-human starting dose of 5 mg once daily was established from the model approaches and will be utilized in the upcoming FIH clinical study."

Journal • PK/PD data • Oncology

AACR 2025: Colon Cancer Risk Reduction, Predicting Melanoma Spread and New Drug Therapies Among Ohio State Findings (Newswise) - "The study focuses on a new drug called JBZ-001 (HOSU-53 when in preclinical testing), which was developed and then moved to commercialization at the OSUCCC - James. The trial represents the first human testing of a best-in-class DHODH inhibitor for stopping growth of various cancers, including certain lymphomas. This clinical trial is now enrolling patients with advanced solid tumors or lymphomas that have not responded to other treatments....When tested in preclinical models, HOSU-53 helped slow the growth of cancer cells. Study findings suggest a new possible way to treat small cell lung cancer and have led to starting human trials to see if HOSU-53 works well in people and if it is safe to use."

Preclinical • Trial status • Lymphoma • Solid Tumor

Jabez Biosciences, Inc. Announces First Patient Dosed in Phase 1 Oncology Clinical Study of JBZ-001 (ACCESSWIRE) - "Jabez Biosciences...announced that the first patient has been successfully dosed in its Phase 1 clinical study of JBZ-001, a next-generation small molecule inhibitor targeting dihydroorotate dehydrogenase (DHODH). This milestone marks a significant step forward in the company's mission to develop innovative treatments that improve and extend the lives of patients battling cancer....The Phase 1 study, conducted at The Ohio State University Comprehensive Cancer Center...is evaluating the safety, tolerability, and preliminary efficacy of JBZ-001 in patients with advanced solid tumors and non-Hodgkin lymphoma (NHL)."

Trial status • Non-Hodgkin’s Lymphoma • Solid Tumor

Trial to Evaluate the Safety & Tolerability of JBZ-001 in Pts With Advanced Solid and Hematological Malignancies (clinicaltrials.gov) - P1 | N=25 | Recruiting | Sponsor: Jabez Bioscience, Inc

New P1 trial • Hematological Disorders • Hematological Malignancies • Oncology • Solid Tumor