Kelonia’s In Vivo CAR-T Therapy KLN-1010 Achieves 100% MRD-Negative Responses in Early Multiple Myeloma Data

BOSTON, MA – December 9, 2025 — Kelonia Therapeutics, Inc., a clinical-stage biotechnology company focused on pioneering in vivo gene delivery, today presented first-in-human data from the Phase 1 inMMyCAR study, evaluating KLN-1010, a novel in vivo gene therapy designed to generate anti-BCMA CAR-T cells inside the body.

The late-breaking oral presentation at the American Society of Hematology (ASH) 2025 Annual Meeting in Orlando showcased early, compelling efficacy and a favorable safety profile in patients with relapsed and refractory multiple myeloma.

Key Data Highlights from Initial Cohort

The data from the first four treated patients demonstrated unprecedented early results for an in vivo CAR-T approach that does not require lymphodepleting chemotherapy:

• 100% MRD-Negative Response Rate: All four patients achieved minimal residual disease (MRD)-negative responses. These deep responses were sustained through three months in the two patients with the longest follow-up.

• Robust CAR-T Expansion: The therapy showed robust CAR-T cell expansion, similar to traditional ex vivo therapies, reaching up to 85% of circulating T cells. Persistent memory CAR-T cells were observed in all patients through three months.

• Best Response: All four patients remain in response, with the longest follow-up at five months; complete response (CR) was the best overall response observed.

“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., CEO and Founder of Kelonia.

Differentiated Safety and Accessibility

A crucial highlight of KLN-1010 is its favorable toxicity profile compared to traditional ex vivo CAR-T therapies, which typically require intensive conditioning:

• No High-Grade Toxicity: No Cytokine Release Syndrome (CRS) of grade 3 or above, no Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), and markedly lower cytopenias were observed.

• Dose Exploration: Deep responses were observed at the standard dose, prompting the exploration of a lower dose. The fourth patient, treated at a lower dose (6 x 10^6 IU/kg), achieved an MRD-negative response at month one, suggesting a wide therapeutic window and the potential to reduce manufacturing costs.

Professor Joy Ho, an investigator on the inMMyCAR study, noted: “The activity observed at a lower dose level suggests a wide therapeutic window and highlights the promise of this in vivo approach.”

KLN-1010 is an investigational in vivo gene therapy that is administered via direct transfusion and designed to generate durable anti-BCMA CAR-T cells inside the patient’s body, potentially eliminating long wait times and the need for preconditioning chemotherapy, thus increasing access to this transformative therapy.