FDA Clears IND Application for KLN-1010 in R/R Multiple Myeloma

The FDA has cleared an investigational new drug (IND) application for KLN-1010, a novel in vivo gene therapy targeting BCMA, as a treatment for patients with relapsed/refractory multiple myeloma, according to a press release from the developer, Kelonia Therapeutics, Inc.¹

Developers engineered KLN-1010 to generate CAR T cells that target BCMA expressed on the surface of multiple myeloma cells. The agent’s ability to be administered via direct infusion while generating enduring CAR T cells inside the body following a single dose may avoid longer wait times for treatment, which may differentiate it from other CAR T-cell therapies. Investigators hypothesize that the gene therapy’s design may mitigate limitations associated with current CAR T-cell strategies, such as limited access to treatment and the need for preconditioning chemotherapy.

“This IND clearance is an important milestone for KLN-1010 and for the broader field of in vivo CAR-T therapy. Last month, we presented initial safety and efficacy data from the first 4 patients treated in our phase 1 study, all of whom achieved measurable residual disease [MRD]–negative responses at 1 month, with durability extending through 3 months in the patients with the longest follow-up,” Kevin Friedman, PhD, chief executive officer and founder at Kelonia, stated in the press release.¹ “The FDA’s clearance allows us to accelerate enrollment across multiple geographies and brings us a meaningful step closer to our goal of democratizing CAR [T-cell] therapies for patients with multiple myeloma.”

The FDA’s clearance of the IND permits investigators to expand their evaluation of KLN-1010 in the phase 1 inMMyCAR™ trial (NCT07075185) to multiple sites throughout the US. Investigators presented data from the first-in-human trial at the 2025 American Society of Hematology (ASH) Annual Meeting and Exposition.²

Based on International Myeloma Working Group (IMWG) guidelines, 1 patient experienced a complete response (CR) at a MRD sensitivity level of 10^–6 at 5 months after initiating treatment with KLN-1010. A second patient experienced a partial response (PR) at a sensitivity of 10^–5 after 4 months, and a third patient achieved a PR at a sensitivity of 10^–5 in month 3. Additionally, a fourth patient experienced a PR at a sensitivity of 10^–5 near the beginning of month 2 after treatment.

Safety data revealed no immune effector cell–associated neurotoxicity syndrome (ICANS) events, and all instances of cytokine release syndrome (CRS) were low-grade events. The most common treatment-emergent adverse effects (TEAEs) included infusion-related reactions, lymphocytosis, hypomagnesemia, and hypokalemia.

“These initial MRD-negative responses and persistent CAR T-cell [expansion may be] prognostic of ongoing clinical responses. Establishing durability of response remains a priority in continued follow-up, and updated results will be presented at future meetings,” study investigator Phoebe Joy Ho, MBBS, DPhil, a professor at the University of Sydney and a senior staff specialist in hematology, the director of research, and head of the Multiple Myeloma Research Unit and Thalassemia/Haemoglobinopathy Unit at the Institute of Haematology, Royal Prince Alfred Hospital, stated in a presentation of these data.²

In the dose-escalation portion of the inMMyCAR trial, patients were assigned to receive KLN-1010 at various doses, including 6 x 10⁶ IU/kg and 2 x 10⁷ IU/kg. In the dose-expansion cohort, patients will receive the agent at the recommended phase 2 dose (RP2D).

The trial’s primary end point is the incidence and severity of TEAEs, including dose-limiting toxicities.³ Secondary end points include responses per IMWG guidelines and pharmacokinetics.

Patients 18 years and older with relapsed/refractory multiple myeloma and at least 3 prior lines of treatment including a proteasome inhibitor, an immunomodulatory drug, and a CD38-targeted monoclonal antibody are eligible for enrollment on the trial. Other eligibility criteria include having an ECOG performance status of 0 or 1 and acceptable laboratory values.

References

1. Kelonia Therapeutics announces FDA clearance of investigational new drug (IND) application for KLN-1010, an in vivo BCMA CAR-T therapy for relapsed and refractory multiple myeloma. News release. January 8, 2026. Accessed January 8, 2026. https://tinyurl.com/kwn2m9fx
2. Harrison S, Ho, PJ, Lim S-L, et al. 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: results of a phase 2 trial. Blood. 2025;146(suppl 1):LBA-1. doi:10.1182/blood-2025-LBA-1
3. A study to evaluate a novel gene therapy in patients with relapsed and refractory multiple myeloma (inMMyCAR). ClinicalTrials.gov. Updated September 2, 2025. Accessed January 8, 2026. https://tinyurl.com/4uvrwdsv