JBZ-001 for Cancer

Overseen By Steve Kaesshaefer

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Jabez Bioscience, Inc

Must not be taking: Antiretrovirals, Corticosteroids

Disqualifiers: Cardiovascular disease, CNS metastases, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 2

25 Participants Needed

Trial Summary

What is the purpose of this trial?

This will be a phase 1, open-label, dose-escalation and expansion, FIH trial to evaluate the safety, tolerability, PK, PD, and preliminary efficacy of JBZ-001, a DHODH inhibitor, in patients with refractory solid and hematological malignancies. The study design includes two independent parts: dose escalation in solid tumors and NHL (Part 1), and up to four indication expansions in selected solid tumor types and NHL (Part 2). The dose escalation will enroll patients with solid tumors and NHL following a standard "3+3" design enrolling a minimum of 3 and up to 6 patients per dose level.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

What safety data exists for JBZ-001 or similar treatments?

The safety of treatments similar to JBZ-001, like JS001 and other PD-1 inhibitors, has been evaluated in various studies. These treatments have been tested in patients with different types of cancer, such as melanoma and lung cancer, and have shown to be generally safe, although specific side effects can vary. ^{1 2 3 4 5}

Research Team

Zuzana Jirakova, MD PhD

Principal Investigator

Jabez Biosciences, Inc

Eligibility Criteria

This trial is for patients with advanced solid tumors or blood cancers (hematological malignancies) who have not responded to standard treatments. Participants must meet certain health criteria to be eligible.

Inclusion Criteria

I can perform daily activities with minimal assistance.

For dose escalation and expansion: - Solid tumors: histologically confirmed relapsed or refractory advanced solid tumor with no standard approved treatment available, or ineligible for, or did not tolerate standard approved treatment - NHL: histologically confirmed relapsed or refractory diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL) or Mantle cell lymphoma (MCL) with no standard approved treatment available, or ineligible for, or did not tolerate standard approved treatment

My side effects from previous cancer treatments have mostly gone away.

Exclusion Criteria

Known hypersensitivity to JBZ-001 or any of its excipients

Corrected interval between Q and T wave on ECG (QTc) ≥ 470 msec using Fredericia's formula

I don't have any health issues that could affect the study or make it hard for me to follow the study plan.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Participants receive escalating doses of JBZ-001 to evaluate safety and tolerability

28 days

Weekly visits for dose escalation monitoring

Indication Expansion

Participants receive JBZ-001 in selected solid tumor types and NHL to evaluate efficacy

up to 1 year

Monthly visits for efficacy assessment

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

2 visits (in-person)

Treatment Details

Interventions

• JBZ-001

Trial Overview The trial is testing JBZ-001, a DHODH inhibitor, in two parts: first, determining safe dosage levels in patients with solid tumors and non-Hodgkin lymphoma; second, assessing its effects on specific cancer types.

Participant Groups

Group I: Experimental Cohort 5 Experimental Treatment 1 Intervention

1\~6 subjects in this cohort will receive 1 dose of 25 mg orally daily

Group II: Experimental Cohort 4 Experimental Treatment 1 Intervention

1\~6 subjects in this cohort will receive 1 dose of 17.5 mg orally daily

Group III: Experimental Cohort 3 Experimental Treatment 1 Intervention

1\~6 subjects in this cohort will receive 1 dose of 10 mg orally daily

Group IV: Experimental Cohort 2 Experimental Treatment 1 Intervention

1\~6 subjects in this cohort will receive 1 dose 5 mg orally daily

Group V: Experimental Cohort 1 Experimental Treatment 1 Intervention

1\~6 subjects in this cohort will receive 1 dose 5 mg orally.

Who Is Running the Clinical Trial?

Jabez Bioscience, Inc

Lead Sponsor

Trials

1

Recruited

30+

Findings from Research

JTX-4014, a new PD-1 targeting antibody, was well tolerated in a trial with 18 patients and did not show new safety concerns compared to existing PD-1 inhibitors, establishing a recommended dose of 500 mg every 3 weeks or 1000 mg every 6 weeks.

The treatment demonstrated clinical activity with a 16.7% overall response rate and a 44.4% disease control rate, particularly in patients with PD-L1 positive tumors, suggesting potential for further development in cancer therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase I, first-in-human trial of programmed cell death receptor-1 (PD-1) inhibitor, JTX-4014, in adult patients with advanced, refractory, solid tumors. Papadopoulos, KP., Lakhani, N., Falchook, GS., et al.[2021]

In a real-world study of 1002 patients with previously treated non-small cell lung cancer (NSCLC), atezolizumab showed an 18-month overall survival rate of 41.1%, confirming its effectiveness outside of clinical trial settings.

The safety profile was acceptable, with an overall adverse event rate of 43.9% and immune-related adverse events at 19.0%, but caution is advised for patients with poorer performance status (ECOG ≥ 2), as they had a significantly lower survival rate of 14.3%.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness and Safety of Atezolizumab Monotherapy in Previously Treated Japanese Patients With Unresectable Advanced or Recurrent NSCLC: A Multicenter, Prospective, Observational Study (J-TAIL). Miura, S., Nishio, M., Akamatsu, H., et al.[2023]

In a phase 2 trial involving 36 patients with untreated brain metastases from melanoma or non-small-cell lung cancer (NSCLC), pembrolizumab demonstrated activity, achieving a brain metastasis response in 22% of melanoma patients and 33% of NSCLC patients, with responses being durable for most.

The treatment had an acceptable safety profile, with serious adverse events occurring in a small percentage of patients, indicating that systemic immunotherapy could be a viable option for those with untreated or progressive brain metastases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pembrolizumab for patients with melanoma or non-small-cell lung cancer and untreated brain metastases: early analysis of a non-randomised, open-label, phase 2 trial. Goldberg, SB., Gettinger, SN., Mahajan, A., et al.[2022]

JS001, a monoclonal antibody targeting the PD-1 receptor, was well tolerated in a phase I study involving 36 patients with advanced melanoma and urologic cancers, with no dose-limiting toxicities observed.

The treatment showed promising anti-tumor activity, achieving a 22.2% objective response rate and a 50.0% disease control rate, particularly in patients with immune-active profiles, leading to further studies focusing on specific melanoma subtypes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and clinical activity with an anti-PD-1 antibody JS001 in advanced melanoma or urologic cancer patients. Tang, B., Yan, X., Sheng, X., et al.[2020]

The PePS2 trial demonstrated that pembrolizumab can be safely administered to patients with non-small-cell lung cancer (NSCLC) and a performance status of 2, with a toxicity rate of 28% and no severe treatment-related adverse events, indicating a manageable safety profile.

Efficacy outcomes showed that 38% of first-line and 36% of subsequent-line patients experienced durable clinical benefit, with higher response rates correlating with tumor proportion scores, suggesting that pembrolizumab is effective for this underserved patient group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pembrolizumab in patients with non-small-cell lung cancer of performance status 2 (PePS2): a single arm, phase 2 trial. Middleton, G., Brock, K., Savage, J., et al.[2020]

References

1. Germany pubmed.ncbi.nlm.nih.gov

Phase I, first-in-human trial of programmed cell death receptor-1 (PD-1) inhibitor, JTX-4014, in adult patients with advanced, refractory, solid tumors. [2021]

2. United States pubmed.ncbi.nlm.nih.gov

Effectiveness and Safety of Atezolizumab Monotherapy in Previously Treated Japanese Patients With Unresectable Advanced or Recurrent NSCLC: A Multicenter, Prospective, Observational Study (J-TAIL). [2023]

3. England pubmed.ncbi.nlm.nih.gov

Pembrolizumab for patients with melanoma or non-small-cell lung cancer and untreated brain metastases: early analysis of a non-randomised, open-label, phase 2 trial. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Safety and clinical activity with an anti-PD-1 antibody JS001 in advanced melanoma or urologic cancer patients. [2020]

5. England pubmed.ncbi.nlm.nih.gov

Pembrolizumab in patients with non-small-cell lung cancer of performance status 2 (PePS2): a single arm, phase 2 trial. [2020]