• Clinical Trial Approval (CTA) granted by Australia’s Therapeutic Goods Administration (TGA) for KMCAR™ T-cell, a first-in-human CAR-T cell therapy for multiple myeloma, the second most common blood cancer worldwide
• KMCAR™ T-cell targets a novel antigen – called Kappa Myeloma Antigen, or KMA – which is found only on myeloma cells, enabling precision targeting and sparing normal immune cells
• KMCAR™ T-cell is derived from HaemaLogiX's proprietary KappaMab antibody technology, which has been validated through Phase 1, 2a and 2b KappaMab (monoclonal antibody) clinical trials, showing a favourable safety profile, plus deep and durable patient treatment responses 
• The Phase 1 KOALA study is the culmination of a previous preclinical research collaboration between HaemaLogiX and Peter Mac teams, which demonstrated compelling proof of concept
• Ethics approval process underway with Peter Mac - first patient expected during Q2 CY26

HaemaLogiX Ltd and Peter MacCallum Cancer Centre (Peter Mac) are delighted to announce the achievement of a landmark regulatory milestone: receipt of Clinical Trial Approval from Australia’s Therapeutic Goods Administration (TGA) for the Phase 1 clinical trial of KMCAR™ T-cell immunotherapy in multiple myeloma (KOALA study). The study will represent the first clinical evaluation of KMCAR™ T-cell therapy in humans.

Multiple myeloma is the second most common blood cancer worldwide. In 2022, the World Health Organization (WHO)/Global Cancer Observatory (GLOBOCAN) estimated that approximately 543,000 people lived with multiple myeloma globally, and approximately 188,000 new cases of multiple myeloma are diagnosed each year globally with approximately 121,400 deaths, and 42% of patients dying from the disease within five years of diagnosis.

HaemaLogiX’s KMA.CAR T-cell immunotherapy (KMCAR™) is a novel autologous CAR-T cell therapy that targets Kappa Myeloma Antigen (KMA), a receptor found only on the surface of myeloma cells and not on healthy immune cells. This tumour-specific targeting means that, unlike currently approved BCMA-directed CAR-T therapies, KMCAR™ T-cell is not expected to damage healthy immune cells – potentially offering patients an effective treatment without affecting their natural ability to ward off infection.

KMCAR™ T-cell therapy is derived from HaemaLogiX's proprietary KappaMab antibody technology. The KMA target has been validated through Phase 1, 2a and 2b KappaMab (monoclonal antibody) clinical trials, with a favourable safety profile and durable patient response outcomes.

KMCAR™ T-cell therapy harnesses the patient’s own T cells and genetically modifies them to recognise and destroy cancerous plasma cells that express KMA. Pre-clinical studies using KMCAR™ T-cell therapy in multiple myeloma animal models showed specific and durable KMA-positive tumour cell killing when compared to a negative control.

The KOALA study is a Phase 1 dose-escalation trial that will evaluate the safety and efficacy of the KMCAR™ T-cell immunotherapy in patients with relapsed or refractory kappa-restricted multiple myeloma. 

With the TGA Clinical Trial Approval stage complete, the trial is now on track to commence and initiate patient recruitment in Q2 2026. 

Professor Simon Harrison, Director of the Centre of Excellence in Cellular Immunotherapy at Peter Mac, said: 

"CAR-T cell therapy has already transformed outcomes for some blood cancers, and KMCAR™ T- cell represents an exciting opportunity to expand these benefits to myeloma patients with a potentially safer approach.

"The KMA target has been extensively validated through the KappaMab antibody clinical trials conducted to date by HaemaLogiX, Alfred Health and Peter Mac. The exceptional safety profile observed in those studies is highly reassuring as we move into this first-in-human CAR-T trial for multiple myeloma. We look forward to advancing the KOALA clinical trial in the hope of bringing this innovative therapy to myeloma patients worldwide."

Dr Rosanne Dunn, Chief Scientific Officer and Founder of HaemaLogiX, said: 

"Peter Mac's Centre of Excellence in Cellular Immunotherapy is world-class and has been instrumental in bringing CAR-T therapies from the laboratory to patients. We are incredibly fortunate to have such a partner to translate our KMA targeting technology into the CAR-T modality.

"For patients with multiple myeloma who have failed or relapsed on their current treatment, it is critical that we explore novel therapeutic targets. The KMA target is unique – it is expressed on malignant plasma cells but spares normal immune cells. Through the CAR-T approach, we have an opportunity to harness this tumour specificity to potentially deliver meaningful benefits for patients without harming their immune system."

Background

Multiple myeloma is a blood cancer arising from plasma cells in the bone marrow. Despite significant advances in treatment, most patients eventually relapse and become refractory to available therapies. CAR-T cell therapy has emerged as a transformative treatment option for blood cancers, with BCMA-directed products showing remarkable response rates in heavily pre-treated patients. However, they also have the characteristic of damaging protective immune cells, leaving patients seriously at risk of infections such as pneumonia.

KMCAR™ T-cell is derived from HaemaLogiX's proprietary KappaMab antibody technology. KappaMab has been evaluated in Phase 1, 2a, Phase 2b and now a further, ongoing
Phase 2b clinical trial, demonstrating an excellent safety profile with no serious adverse events or dose-limiting toxicities. This extensive clinical validation of the KMA target provides confidence as the technology advances as a CAR-T immunotherapy.

The KOALA study will be conducted at Peter Mac's Centre of Excellence in Cellular Immunotherapy, which is at the forefront of developing and delivering cell-based immunotherapies in Australia. Peter Mac has been involved in the development of many CAR-T therapies now approved globally. Cell Therapies Pty Ltd, the Centre of Excellence in Cellular Immunotherapy’s manufacturing partner, co-located within Peter Mac, will be manufacturing the KMCAR™ T-cells.

The KMCAR™ T-cell treatment is being developed by HaemaLogiX, in collaboration with world-leading Centre of Excellence in Cellular Immunotherapy at Peter Mac.