Kelonia Therapeutics Doses First Patient in Groundbreaking In Vivo CAR-T Trial for Multiple Myeloma
核心洞察
Kelonia Therapeutics (搜索) has dosed the first patient in the inMMyCAR Phase 1 trial evaluating KLN-1010, the first anti-BCMA (搜索) in vivo CAR-T therapy studied in a multi-center clinical trial.
KLN-1010 represents a revolutionary approach that generates CAR-T cells directly inside the patient's body without requiring apheresis, ex vivo manufacturing, or lymphodepletion chemotherapy.
The Phase 1 dose-escalation study will enroll up to 40 patients with relapsed and refractory multiple myeloma to assess safety and preliminary efficacy of this novel gene therapy approach.
Kelonia Therapeutics (搜索) has achieved a significant milestone in cancer immunotherapy by dosing the first patient in its Phase 1 inMMyCAR study, marking the debut of KLN-1010, the first anti-BCMA (搜索) in vivo CAR-T program to be evaluated in a multi-center clinical trial. The groundbreaking therapy represents a paradigm shift in CAR-T cell treatment by generating therapeutic cells directly inside patients' bodies without the complex manufacturing processes required by traditional approaches.
The first patient was dosed at Royal Prince Alfred Hospital in Sydney, Australia, by inMMyCAR Investigator Professor Joy Ho. This milestone represents the first time KLN-1010 will be evaluated in humans, targeting patients with relapsed and refractory multiple myeloma who have limited treatment options.
Revolutionary In Vivo Approach
KLN-1010 distinguishes itself from conventional CAR-T therapies through its innovative delivery mechanism. Unlike traditional CAR-T treatments that require apheresis to harvest patient T cells, followed by weeks of ex vivo manufacturing and lymphodepleting chemotherapy, KLN-1010 is administered directly to patients via transfusion. The therapy is designed to generate durable anti-BCMA (搜索) CAR-T cells inside the body after a single dose.
"KLN-1010 offers hope to multiple myeloma patients who can't access today's CAR-T cell therapies," said Kevin Friedman, Ph.D., Chief Executive Officer and Founder of Kelonia. "This first-in-human dosing marks a major milestone toward a future where CAR-T benefits can be delivered without preparative chemotherapy or bespoke manufacturing delays, and at virtually any hospital around the world."
The therapy utilizes Kelonia's proprietary in vivo Gene Placement System (iGPS®) technology, which employs an advanced lentiviral vector particle with envelope modifications to improve in vivo gene transfer efficiency and tropism molecules for tissue-specific delivery.
Addressing Critical Unmet Need
Multiple myeloma presents significant treatment challenges, as most patients experience relapse after initial treatment and many develop resistance to available therapies. The disease is characterized by plasma cell proliferation in bone marrow, leading to bone destruction, anemia, renal dysfunction, and immunosuppression. Relapsed and refractory multiple myeloma involves clonal evolution, drug resistance, and increased disease heterogeneity.
Professor Simon Harrison, Director of the Centre of Excellence in Cellular Immunotherapy at the Peter MacCallum Cancer Centre (搜索) and inMMyCAR Lead Investigator, emphasized the transformative potential of the approach. "Multiple myeloma can be a challenging disease to treat as most patients experience relapse after initial treatment, and many become resistant to currently available therapies. In vivo CAR-T cell therapies would be transformative for these patients, providing rapid access to potentially life-saving treatments."
Clinical Trial Design and Objectives
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 focus on the incidence and severity of treatment-emergent adverse events (TEAEs), including dose limiting toxicities (DLTs), and establishing the recommended Phase 2 dose.
Professor Joy Ho, who administered the first dose, highlighted the significance of the achievement. "As an in vivo CAR-T cell therapeutic candidate, KLN-1010 has the potential to deliver the full promise of CAR-T cell therapies without the complex manufacturing requirements, lengthy production timelines and toxic lymphodepleting chemotherapy that ex vivo CAR-T cell therapies require. We believe it has the potential to revolutionize CAR-T therapy in myeloma."
Regulatory Approval and Future Implications
KLN-1010 has received Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) clearance from the Australian Therapeutic Goods Administration (TGA). The trial is registered on clinicaltrials.gov under identifier NCT07075185.
The therapy targets BCMA (搜索), a protein expressed on the surface of multiple myeloma cells, potentially eliminating the need for long wait times associated with traditional CAR-T manufacturing. This approach could overcome several limitations of current CAR-T therapies, including limited access to treatment and the requirement for preconditioning chemotherapy.
Kelonia's broader vision extends beyond multiple myeloma, with the company building a pipeline of genetic medicines for various diseases using its iGPS® platform. The company aims to make genetic medicines accessible to every patient in need, when and where they need them, representing a significant advancement in the democratization of advanced cellular therapies.