A Study to Assess Safety, Tolerability and Preliminary efficacy of Bexmarilimab in Combination with Standard of Care in Patients with Hematological Malignancies (BEXMAB)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Faron Pharmaceuticals Oy

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

30 May 2022 → 3 Feb 2026

Decision date (initial)

2024-10-11

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Faron Pharmaceuticals Ltd

External identifiers

EU CT number

2024-517444-64-00

EudraCT number

2021-002104-12

ClinicalTrials.gov

NCT05428969

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Dose response, Pharmacodynamic, Efficacy, Pharmacokinetic, Safety

Phase I
1. To determine the safety and tolerability of bexmarilimab in combination with SoC treatment to identify the recommended dose for expansion (RDE).

Phase II
1. To evaluate the preliminary clinical efficacy of bexmarilimab at recommended phase 2 dose (RP2D) in combination with SoC.

Secondary objectives 7

1. Phase I - 1. To assess preliminary clinical efficacy of bexmarilimab in combination with SoC treatment.
2. Phase I - 2. To characterize the pharmacokinetic (PK) profile of bexmarilimab in combination with SoC treatment.
3. Phase I - 3. To assess the immunogenicity of bexmarilimab.
4. Phase II - 1. To further evaluate the extended safety and tolerability of bexmarilimab at RDE/one dose level below the RDE/RP2D in combination with SoC treatment.
5. Phase II - 2. To investigate additional preliminary efficacy parameters of bexmarilimab in combination with SoC.
6. Phase II - 3. To characterize the pharmacokinetic (PK) profile of bexmarilimab in combination with SoC treatment.
7. Phase II - 4. To assess the immunogenicity of bexmarilimab.

Conditions and MedDRA coding

Chronic Myelomonocytic Leukemia

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Patient ≥ 18 years of age who presents with one of the following conditions: - Morphologically confirmed diagnosis of MDS with revised International Prognostic Scoring System (rIPSS) risk categories: intermediate, high and very high. - Morphologically confirmed diagnosis of CMML-2 with indication for azacitidine treatment. - CMML and MDS patient with response failure to HMA or therapy regimen including HMA. - Morphologically confirmed diagnosis of r/r AML following at least 1 line of prior therapies with indication for azacitidine treatment. - Morphologically confirmed diagnosis of AML in patients unfit for induction therapy with indication for azacitidine-venetoclax treatment.
2. Leukocyte count < 20 x10^9/L (< 25 x10^9/L for newly diagnosed AML). Hydroxycarbamide use is permitted to meet this criterion in MDS and AML but not in CMML.
3. Adequate renal function.
4. Adequate liver function.

Exclusion criteria 9

1. Patient with acute promyelocytic leukemia (APL) or myeloproliferative CMML as defined by leukocyte count > 13 x10^9/L.
2. Eastern Cooperative Oncology Group (ECOG) performance status >2 (except newly diagnosed AML where ECOG 3 is allowed for patients < 75 years).
3. Allogeneic transplantation less than 6 months prior screening.
4. Patient with active auto-immune disorder (except type I diabetes, celiac disease, hypothyroidism requiring only hormone replacement, vitiligo, psoriasis, or alopecia).
5. The patient requires systemic corticosteroid (≥10 mg/day prednisone or equivalent) or other immunosuppressive treatment.
6. Less than 21 days since the last dose of intravenous anticancer chemotherapy or less than 14 days or five half-lives (whichever is shorter) from a small molecule targeted therapy or oral anticancer chemotherapy before the first study treatment.
7. Any immunotherapy or investigational therapy within preceding 28 days from the first study treatment.
8. Pregnant or lactating women.
9. History of chronic ulcers or clinically relevant liver disease leading to Child Pugh Score C or higher.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Phase I - 1. Reporting of incidence and frequency of dose limiting toxicities (DLTs), and frequency and severity of adverse events (AE), SAEs and laboratory abnormalities.
2. Phase II - 1. Preliminary efficacy will be investigated per indication as follows: - Complete response (CR) rate for MDS and CMML-2. - Overall response rate (ORR) for MDS and CMML failure to prior HMA. - CR for r/r AML. - CR rate for newly diagnosed AML.

Secondary endpoints 6

1. Phase I - 1. Clinical efficacy measures include disease-specific response criteria and progression and survival analyses.
2. Phase I - 2. PK samples at defined timepoints of single and repeat bexmarilimab administration and derived PK parameters.
3. Phase I - 3. Anti-bexmarilimab antibody detection.
4. Phase II - 1. Frequency and severity based on NCI-CTCAE v 5.0 grading of adverse events (AE), SAE and laboratory abnormalities.
5. Phase II - 2. Extended preliminary efficacy to include disease-specific response criteria and progression and survival analyses.
6. Phase II - 3. Anti-bexmarilimab antibody (immunogenicity) detection.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Faron Pharmaceuticals Oy

Sponsor organisation

Faron Pharmaceuticals Oy

Address

Joukahaisenkatu 6

City

Turku

Postcode

20520

Country

Finland

Scientific contact point

Organisation

Faron Pharmaceuticals Oy

Contact name

Regulatory Affairs

Public contact point

Organisation

Faron Pharmaceuticals Oy

Contact name

Regulatory Affairs

Third parties 6

Locations

1 EU/EEA country · 4 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications