Early results from the Phase 1 inMMyCAR™ study demonstrate ongoing MRD-negative responses in all patients with a tolerable safety profile
Robust CAR-T cell expansion occurred in the absence of preparative chemotherapy
Persistent, memory CAR-T cells in all patients
BOSTON–(BUSINESS WIRE)–Kelonia Therapeutics, Inc., a clinical-stage biotechnology company pioneering in vivo gene delivery, today announced that its first results from the ongoing inMMyCAR study, a Phase 1 clinical trial evaluating KLN-1010, a novel in vivo gene therapy that generates anti-BCMA CAR-T cells in patients with relapsed and refractory multiple myeloma, will be presented in a late-breaking oral presentation at the American Society of Hematology (ASH) 2025 Annual Meeting in Orlando, Florida.
The late-breaking abstract features results from the first three patients treated with KLN-1010. All patients achieved minimal residual disease (MRD) negativity at month 1 that persisted through three months in the patient with the longest follow up. 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. “These initial data point to a potentially powerful medicine while also providing 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. We are encouraged by these first-in-human results and looking forward to sharing additional details from the study with the scientific and medical communities at the ASH Annual Meeting.”
“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,” said 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 and lead author of the late-breaking abstract. “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.”
Oral Presentation Details:
Minimal residual disease (MRD)-negative outcomes following a novel, in vivo gene therapy generating anti–B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR)-T cells in patients with relapsed and refractory multiple myeloma (RRMM): Preliminary results from inMMyCAR, the first-in-human Phase 1 study of KLN-1010
Date: Tuesday, December 9, 2025, 7:30 – 9:00 AM EST
Location: West Hall D2 (Orange County Convention Center)
Session Title: Late-Breaking Abstracts Session
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. 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. 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 KLN-1010
KLN‑1010 is an investigational in vivo gene therapy that generates anti-BCMA CAR-T cells, targeting 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. This may overcome several limitations faced by current CAR-T approaches, including limited access to treatment and preconditioning chemotherapy.
About Kelonia Therapeutics
Kelonia is a clinical-stage company pioneering a new wave of genetic medicines using its in vivo gene placement system (iGPS®). The company’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. Its lead candidate, KLN-1010, is an in vivo anti-BCMA CAR-T therapy for multiple myeloma being evaluated in a Phase 1 clinical trial. Learn more about Kelonia at https://www.keloniatx.com/ and follow us on LinkedIn and X.
Kelonia, iGPS, and inMMyCAR are trademarks of Kelonia Therapeutics, Inc.
Contacts
Katherine Smith, Inizio Evoke Comms
[email protected]
