HaemaLogiX Doses First Patient with KMCAR™ T-Cell Therapy in Phase 1 KOALA Trial for Multiple Myeloma
核心洞察
HaemaLogiX announced the first patient has been safely dosed with KMCAR™ T-cell therapy (搜索) in the Phase 1 KOALA trial for relapsed/refractory kappa-restricted multiple myeloma (搜索).
The treatment was well tolerated with no serious adverse events, and a second patient has been enrolled to receive a higher dose.
KMCAR™ targets the Kappa Myeloma Antigen (KMA) (搜索), a tumour-specific receptor found only on malignant plasma cells, sparing healthy immune cells unlike BCMA (搜索)-directed CAR-T therapies.
HaemaLogiX Ltd (搜索) has announced a major milestone in its Phase 1 KOALA trial, with the first patient having been safely treated with KMCAR™ T-cell therapy (搜索). Following an initial infusion, the patient demonstrated that the treatment was well tolerated, with no serious adverse events. A second patient on the trial has now been enrolled and is set to be treated at a higher dose level.
The KOALA study, conducted at the Peter MacCallum Cancer Centre (搜索), is a dose-escalation trial assessing the safety and preliminary efficacy of HaemaLogiX's proprietary KMCAR™ T-cell technology (PMCC-COE-KMA). The study is being conducted in patients with multiple myeloma (搜索) who are no longer responding to standard treatment options, and who are not eligible for other CAR-T therapies. This represents the first in-human trial for the novel KMCAR™ T-cell immunotherapy.
A Tumour-Specific Target
KMCAR™ T-cell therapy (搜索) is designed to target the Kappa Myeloma Antigen (KMA) (搜索), a tumour-specific receptor found only on kappa-type myeloma cells and absent from healthy immune cells. This unique specificity differentiates the treatment from currently available therapies for multiple myeloma (搜索).
Unlike other therapies currently available that can deplete both cancerous and healthy immune cells, KMCAR™ T-cell therapy (搜索) selectively recognises and eliminates KMA myeloma cells while preserving healthy immune cells that are essential for immune defence. KMCAR™ T-cells are the patient's own immune cells that are redirected to destroy the cancer-causing cells, while sparing normal immune function and other tissues.
Delivery of the therapy involves the harvesting and genetic modification of T cells from a patient's own blood, which are engineered to create KMCAR™ T-cells that are then infused back into the patient. These cells recognise and destroy cancerous plasma cells that express KMA, while sparing healthy immune cells.
Addressing an Unmet Need
Multiple myeloma (搜索) is the world's second-most common blood cancer, with around 188,000 new cases each year and roughly 543,000 people living with the disease. It remains incurable, and 42% of patients die within five years of diagnosis. Most effective treatments only delay the progression of the cancer, but in doing so, damage or destroy the patient's immune system.
Most currently approved BCMA (搜索)-directed CAR-T therapies target both cancerous and healthy cells, leading to weakening of the immune system and opportunistic infections. This can ultimately lead to more than half of non-progressing patients facing infection-related deaths, rather than deaths from the cancer itself. In 2022, the World Health Organisation (WHO)/Global Cancer Observatory (Globocan) estimated approximately 121,400 deaths from multiple myeloma (搜索) globally.
Clinical Significance
Professor Simon Harrison, Director of the Centre of Excellence in Cellular Immunotherapy at Peter Mac, said: "This therapy is designed to provide a more targeted approach that could reduce impacts on the patient's immune system, compared with existing CAR T-cell therapies for multiple myeloma (搜索). If these potential benefits are confirmed in clinical trials, this could represent an important advance in the treatment of multiple myeloma. We welcome the treatment of the first patient in this trial and look forward to continuing to evaluate the safety and potential of this investigational therapy."
Dr Rosanne Dunn, Chief Scientific Officer at HaemaLogiX, said: "We are delighted with the encouraging results from the first safely treated patient, and very pleased to be moving forward to dosing the second patient at a higher dose of KMCAR™ T-cell therapy (搜索). Multiple myeloma (搜索) is the world's second-most common blood cancer, and there is a significant need for therapies that do not cause immunodeficiency which may result in life threatening infections. Seeing this work translate from the laboratory into human trials with positive safety data is a very important step forward for our immunotherapy programs."
Technology Foundation and Study Background
KMCAR™ T-cell therapy (搜索) is derived from HaemaLogiX's proprietary KappaMab™ (搜索) antibody technology, which has been validated across Phase 1, 2a and 2b clinical trials. KappaMab™ has also demonstrated a favourable safety profile with no antibody related lymphopenias and durable patient responses.
The KOALA study is being conducted at Peter Mac's Centre of Excellence in Cellular Immunotherapy, with Cell Therapies Pty Ltd (搜索) manufacturing the KMCAR™ T-cells. Cell Therapies Pty Ltd, which is partnered with Peter Mac, is a TGA licensed Good Manufacturing Practice (GMP) facility. The KOALA trial is registered on ClinicalTrials.gov (Identifier: NCT07541391).
HaemaLogiX Ltd (搜索), founded in 2014, is a clinical-stage biotechnology company developing novel immunotherapies for patients with blood cancers and B-cell diseases. HaemaLogiX is the first and only company developing antibody and CAR-T therapies targeting the novel Kappa Myeloma Antigen (KMA) (搜索) and Lambda Myeloma Antigen (LMA). The company was awarded "Most Promising CAR-T Pipeline in APAC" at the Asia Pacific CGT Excellence Awards 2025.