Kelonia Therapeutics, Inc., a clinical-stage biotechnology company pioneering in vivo gene delivery, today shared positive updated results from inMMyCAR, the ongoing first-in-human Phase 1 study of KLN-1010 in patients with relapsed and refractory multiple myeloma (RRMM), at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Illinois.
Fourteen additional patients have been dosed since first clinical data were reported at the American Society of Hematology (ASH) 2025 Annual Meeting, bringing the total number of patients dosed to 18. The interim data presented at ASCO demonstrate a 100% overall response rate (ORR) and minimal residual disease (MRD)-negative bone marrow at one month post treatment in all evaluable patients.
Additional highlights of the updated data presented today:
- First patient treated remains in deep, ongoing MRD-negative response beyond 10 months
- Six patients with ≥ 4 months of follow up include 4 stringent complete response (sCR) and 2 very good partial response (VGPR), all with ongoing MRD-negative bone marrows
- Robust generation and sustained persistence of CAR-T cells in the peripheral blood and bone marrow exceeding what is typically seen with ex vivo CAR-T therapies
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Continued favorable safety and tolerability profiles; safety review committee has approved outpatient infusion
- 16 of 18 patients experienced cytokine release syndrome (CRS); all were Grade 1-2
- One Grade 1 and one Grade 3 immune effector-cell associated neurotoxicity syndrome (ICANS) event; Grade 3 event was limited to 3 days
- No delayed neurotoxicity observed
- Cytopenias and Grade 3-4 infections were notably limited
- No infusion-related reactions (IRRs) following dexamethasone premedication implementation
“The clinical data presented today at ASCO strengthens our conviction that the iGPS platform has the potential to transform the treatment experience and outcomes for patients with serious hematologic malignancies,” said Kevin Friedman, Ph.D., Chief Executive Officer and Founder of Kelonia. “This expanded patient cohort continues to provide clear clinical validation of KLN-1010 and our platform. Durable and deep responses were achieved without the need for preparative chemotherapy or complex CAR-T cell manufacturing that impact patient access and treatment timelines. Combined with a safety profile consistent with traditional CAR-T therapies in multiple myeloma, we are seeing growing evidence for the potential of KLN-1010 to democratize CAR-T treatment of multiple myeloma.”
“Early data from Kelonia’s lead in vivo CAR-T program continues to demonstrate highly encouraging responses in additional patients, with the benefit of extended follow-up revealing a consistent pattern of strong clinical responses over time, and a continued favorable safety profile,” said ASCO Presenter and inMMyCAR investigator, Professor Joy Ho, MBBS, D.Phil, Royal Prince Alfred Hospital, Sydney, Australia. “The persistent generation of CAR-T cells observed across the expanded cohort reinforces the potential of KLN-1010 to make a meaningful difference for multiple myeloma patients.”
Eli Lilly and Company’s proposed acquisition of Kelonia Therapeutics announced in April 2026 is pending transaction close.
About KLN-1010
KLN-1010, which has received Fast Track designation from the U.S. Food and Drug Administration (FDA), is an investigational in vivo gene therapy that generates anti-BCMA CAR-T cells, targeting the BCMA protein expressed on the surface of multiple myeloma cells. Unlike traditional CAR-T treatments, KLN-1010 is administered to patients via direct infusion 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. This may overcome several limitations faced by current CAR-T approaches, including limited access to treatment and the requirement of preconditioning chemotherapy.
About inMMyCAR
inMMyCAR 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. Additional information and study site information may be found on clinicaltrials.gov (NCT07075185).
About Relapsed and Refractory Multiple Myeloma
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. It is driven by complex genetic and epigenetic alterations that promote malignant cell survival and resistance to apoptosis. Relapsed and refractory multiple myeloma is characterized by clonal evolution, drug resistance, and increased disease heterogeneity, heightening the need for accessible, personalized therapeutic strategies.
About Kelonia Therapeutics
Kelonia Therapeutics is a clinical-stage biotechnology company pioneering a new wave of genetic medicines using its in vivo gene placement system (iGPS). Kelonia’s elegant, cutting-edge 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. Kelonia is building a pipeline of genetic medicines across a range of diseases, with the bold goal of making CAR-T cell therapies accessible to every patient in need, when and where they need them. Kelonia’s lead candidate, KLN-1010, is an in vivo anti-BCMA CAR-T therapy for multiple myeloma being evaluated in a Phase 1 clinical trial. For more information, please visit: https://www.keloniatx.com/.
Kelonia, iGPS, and inMMyCAR are trademarks of Kelonia Therapeutics, Inc.
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