Rare Diseases

All Conditions

Camurati-Engelmann Disease

Participants will be administered with PM1021 on Part A Day 1. Participants will be administered with PM1021 and PM8001 on Part B Day 1. PM1021 and PM8001 combination therapy administrated every 3 weeks from Part C Day 1 until disease progression, intolerable toxicity, until the patient withdraws/is withdrawn, or study completion.

PM1021, PM8001

Vineet Kwatra, PhD

Vineet Kwatra

The purpose of this research is to assess the safety, tolerability and effectiveness of PM1021 Monotherapy and PM1021 in Combination with PM8001 in Patients with Advanced Solid Tumours. In this study, up to 30 patients will be enrolled in Australia only.

Advanced solid cancers are associated with poor prognosis and pose a significant challenge for treatment strategies. Effective treatments for advanced metastatic malignancies that have failed available standard of care treatment represent a major unmet medical need.

Biotheus Inc. is developing PM1021, a monoclonal anti-T cell immunoglobulin and immunoreceptor tyrosine-based inhibitory motif domains (TIGIT) antibody (IgG1) and PM8001 (a PD-L1/TGF-beta bispecific Fc fusion protein) as treatment for advanced solid tumours.

This is a single-arm, open-label, Phase I study including a first-in-human study for PM1021, and the combination therapy of PM1021 with PM8001. The study will be following the accelerated titration design and the classic 3+3 design, and dose escalation will be investigated for PM1021 monotherapy (Part A) and in combination with PM8001 (Part B), respectively. In the absence of DLTs in Parts A and B, patients will continue to receive combination therapy (Part C) until disease progression, intolerable toxicity, until the patient withdraws/is withdrawn, or study completion.

Up to 30 patients are planned to be enrolled. Four dose levels (150mg, 450 mg, 900 mg, and 1200 mg) of PM1021 with or without PM8001 (20 mg/kg) treatment will be explored in this study.

A Study of PM1021 (Anti-TIGIT) With or Without PM8001 (Anti-PD-L1/TGF-β) in Patients With Advanced Solid Tumours

A Phase I Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PM1021 (Anti-TIGIT) Monotherapy and PM1021 in Combination With PM8001 (Anti-PD-L1/TGF-β) in Patients With Advanced Solid Tumours

The data will be published or presented for publications (poster, abstract, articles or papers) or maked any presentations.

The incidence of DLTs in PM1021 monotherapy and PM1021 combination therapy with PM8001, respectively.

The incidence and severity of treatment-emergent adverse events (TEAEs) and treatment related adverse events (TRAEs) graded according to NCI-CTCAE v5.0.

To determine the recommended Phase II Dose (RP2D) of PM1021 monotherapy, and PM1021 combination therapy with PM8001, respectively.

To evaluate the Cmax of PM1021 monotherapy, and PM1021 combination therapy with PM8001, respectively

To evaluate the Tmax of PM1021 monotherapy, and PM1021 combination therapy with PM8001, respectively

To evaluate the trough concentrations of PM1021 monotherapy, and PM1021 combination therapy with PM8001, respectively

To evaluate the AUC0-t of PM1021 monotherapy, and PM1021 combination therapy with PM8001, respectively

To evaluate the t1/2 of PM1021 monotherapy, and PM1021 combination therapy with PM8001, respectively

To evaluate the Rac_Cmax of PM1021 monotherapy, and PM1021 combination therapy with PM8001, respectively

Defined as the number of patients with best overall response of confirmed CR or PR per RECIST 1.1 divided by the patients with at least one tumour imaging evaluation.

Defined as the percentage of patients who have achieved CR, PR, Non-CR/Non- PD, or SD in the study.

Defined as the time from the date of first dose of study drug to the first observation of documented disease progression per RECIST 1.1 as determined by the Investigators or death due to any cause, whichever occurs first.

Defined as the time from the date of first dose of study drug to the date of documented death due to any cause.

Defined as the duration from the date of first dose of study drug until date of first documented CR or PR.

Defined as the time from the earliest date of documented CR or PR until documented disease progression or death (by any cause, in the absence of progression) as determined by the Investigators using RECIST 1.1.

To evaluate the incidence of ADA to PM1021 and PM8001.

Spots Global Cancer Trial Database for pm1021, pm8001