Kelonia's In Vivo CAR-T Therapy Shows Promising Early Results in Multiple Myeloma Patients
Key Insights
Kelonia Therapeutics (search)' KLN-1010, an investigational in vivo gene therapy, achieved minimal residual disease negativity in all three patients treated in the first-in-human Phase 1 inMMyCAR study for relapsed and refractory multiple myeloma.
The therapy generated anti-BCMA (search) CAR-T cells directly inside patients' bodies without requiring lymphodepleting chemotherapy, apheresis, or ex vivo cell manufacturing, potentially overcoming major barriers to traditional CAR-T access.
Robust CAR-T cell expansion and persistent memory CAR-T cells were observed in all patients, with MRD negativity persisting through three months in the patient with longest follow-up.
Kelonia Therapeutics (search) has announced promising early results from its first-in-human Phase 1 clinical trial of KLN-1010, an investigational in vivo gene therapy that generates anti-BCMA (search) CAR-T cells directly inside patients with relapsed and refractory multiple myeloma. The preliminary data from the inMMyCAR study will be presented as a late-breaking oral presentation at the American Society of Hematology (ASH) 2025 Annual Meeting in Orlando, Florida.
Early Clinical Success Without Traditional CAR-T Barriers
The late-breaking abstract features results from the first three patients treated with KLN-1010, all of whom achieved minimal residual disease (MRD) negativity at month 1. This response persisted through three months in the patient with the longest follow-up. Notably, CAR-T cell expansion and persistence of memory CAR-T cells occurred without the use of lymphodepleting chemotherapy, apheresis, or ex vivo cell manufacturing.
"KLN-1010 is beginning to show the extraordinary clinical outcomes that may be possible with in vivo CAR-T therapy — early, deep responses from a single infusion without the barriers that limit access to traditional CAR-T treatments," said Kevin Friedman, Ph.D., Chief Executive Officer and Founder of Kelonia.
Clinical Significance and Mechanism
Simon Harrison, MBBS, MRCP(UK), FRCPath(UK), FRACP, Ph.D., Director of the Centre of Excellence in Cellular Immunotherapy at the Peter MacCallum Cancer Centre (search) and lead author of the late-breaking abstract, emphasized the clinical importance of the findings. "In these early patients, we are seeing both rapid MRD-negative responses and persistent memory-phenotype CAR-T cells, a combination that has been strongly prognostic for durable clinical benefit with existing CAR-T approaches," Harrison said.
The therapy targets BCMA (search) (B-cell maturation antigen), a protein expressed on the surface of multiple myeloma cells. Unlike traditional CAR-T treatments, KLN-1010 is administered to patients via direct transfusion and is designed to generate durable CAR-T cells inside the body after a single dose, potentially eliminating the need for long wait times to receive treatment.
Study Design and Regulatory Status
The inMMyCAR study is a Phase 1, open-label, dose-escalation clinical trial designed to assess the safety, tolerability, pharmacology and preliminary efficacy of a single dose of KLN-1010 in up to 40 patients. The primary endpoints are incidence and severity of treatment-emergent adverse events (TEAEs), including dose limiting toxicities (DLTs), and to establish the recommended Phase 2 dose of KLN-1010.
KLN-1010 has been granted Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) clearance by the Australian Therapeutic Goods Administration (TGA). This Phase 1 clinical trial marks the first time KLN-1010 will be evaluated in humans.
Technology Platform and Future Applications
The results provide clinical validation of Kelonia's in vivo gene placement system (iGPS) platform. The company's in vivo gene delivery technology uses an advanced lentiviral vector particle harboring envelope modification to improve in vivo gene transfer efficiency and tropism molecules to facilitate tissue-specific delivery.
Friedman noted that these initial data "provide clear clinical validation of our iGPS platform; enabling a growing number of partnered programs as well as our wholly owned pipeline that includes KLN-1010."
Addressing Unmet Medical Need
Multiple myeloma is a hematologic malignancy characterized by the proliferation of plasma cells in the bone marrow, leading to bone destruction, anemia, renal dysfunction, and immunosuppression. Relapsed and refractory multiple myeloma is characterized by clonal evolution, drug resistance, and increased disease heterogeneity, heightening the need for accessible, personalized therapeutic strategies.
Harrison emphasized that "achieving these outcomes without lymphodepleting chemotherapy or CAR-T cell manufacturing underscores the potential of this in vivo approach to fundamentally expand access to CAR-T therapy for patients with relapsed and refractory multiple myeloma."
The oral presentation is scheduled for Tuesday, December 9, 2025, from 7:30 – 9:00 AM EST at West Hall D2 in the Orange County Convention Center during the Late-Breaking Abstracts Session.