Kelonia Therapeutics Presents First-in-Human Data From Phase 1 inMMyCAR Study of KLN-1010 in vivo BCMA CAR-T Therapy at the American Society of Hematology (ASH) 2025 Annual Meeting

100% MRD-negative response rate in four patients; all remain in response through the longest follow up of 5 months

Favorable toxicity profile with no CRS grade 3 or above and no ICANS

Potent CAR-T expansion from a single, off-the-shelf infusion without chemotherapy

BOSTON--(BUSINESS WIRE)--Kelonia Therapeutics, Inc., a clinical-stage biotechnology company pioneering in vivo gene delivery, today shared first-in-human data from the ongoing inMMyCAR^TM 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, in a late-breaking oral presentation at the American Society of Hematology (ASH) 2025 Annual Meeting in Orlando, Florida.

Highlights of the data from the first four patients presented today:

• 100 percent minimal residual disease (MRD)-negative response rate across four patients; MRD-negative responses were maintained through three months in the two patients with the longest follow up.
• Robust CAR-T cell expansion similar to ex vivo therapies despite no lymphodepleting chemotherapy; reaching up to 85% of circulating T cells.
• Persistent memory CAR-T in all patients observed through three months to date.
• Favorable toxicity profile with no cytokine release syndrome (CRS) of grade 3 or above, no Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), no delayed neurotoxicity, and markedly lower cytopenias compared to ex vivo CAR-T cell therapies.
• Three of the four patients were treated at 2x10⁷ IU/kg. Deep responses and favorable safety profile compelled exploration of the therapeutic window to further reduce the cost of goods. The fourth patient was treated at a lower dose of 6x10⁶ IU/kg and achieved an MRD-negative response at month 1.
• All four patients remain in response, per International Myeloma Working Group’s (IMWG) criteria, with the longest follow-up of five months; complete response (CR) was the best overall response.

“While still early, the remarkable responses and desirable safety profiles from our off-the-shelf in vivo CAR-T therapy that does not require preparative chemotherapy demonstrates that the democratization of CAR-T therapies may be truly within reach,” said Kevin Friedman, Ph.D., Chief Executive Officer and Founder of Kelonia. “KLN-1010 is beginning to reveal what may be possible with in vivo CAR-T therapy; deep responses, a differentiated safety profile, and the potential for broad accessibility.”

“A 100% MRD-negative response rate with ongoing response for at least five months alongside a favorable safety profile offers an exciting glimpse of the transformative outcomes possible with KLN-1010,” said ASH Presenter and inMMyCAR investigator, Professor Joy Ho, MB.BS. (Hons), D.Phil (Oxon), FRACP, FRCPA, FFSc (RCPA). “The activity observed at a lower dose level suggests a wide therapeutic window and highlights the promise of this in vivo approach.”

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 and iGPS are trademarks of Kelonia Therapeutics, Inc.

Katherine Smith, Inizio Evoke Comms
katherine.smith@inizioevoke.com