KLN-1010 / Kelonia Therap - LARVOL DELTA

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 (ASH 2025) - "Tocilizumab was administered prophylactically in the latter two patients after the first IRR was observed...No clinical sequelae of the lymphocytosis were noted; dexamethasone promptly resolved the lymphocytosis in the patient with the highest ALC... Preliminary results from the first three patients dosed in the inMMyCAR Phase 1 study of KLN-1010 demonstrate that promising clinical activity and manageable toxicities are feasible with an offthe-shelf in vivo CAR-T in MM. Lymphodepletion was not required for in vivo CAR-T cell generation and expansion in the peripheral blood. CRS was consistent with that seen with ex vivo CAR-T therapies, while cytopenias were notably limited and no treatment-emergent infections occurred."

First-in-human • Gene therapy • Late-breaking abstract • Minimal residual disease • P1 data • Preclinical • Residual disease • CNS Disorders • Dyslipidemia • Gene Therapies • Hematological Malignancies • Infectious Disease • Movement Disorders • Multiple Myeloma • Neutropenia • Parkinson's Disease • Thrombocytopenia

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 (Businesswire) - "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."

First-in-human • P1 data • Multiple Myeloma

Ahead of the American Society of Hematology (ASH) meeting next week, Kelonia Therapeutics shared preliminary results from the first three patients dosed with KLN-1010, an investigational in vivo anti-BCMA CAR-T therapy for relapsed/refractory multiple myeloma (RRMM), demonstrating early efficacy and favourable safety in the ongoing Phase I study. (Firstwordpharma Press Release) - "Results showed the three patients aged 61–72 years with high-risk cytogenetics, no prior exposure to BCMA-targeted agents and no extramedullary involvement, achieved minimal residual disease (MRD)-negative responses at month one. Additionally, all patients attained partial responses by International Myeloma Working Group criteria, which deepened over time, the best response being a very good partial response at month three. Moreover, none had experienced disease progression to date."

P1 data • Multiple Myeloma

Kelonia Therapeutics Announces Late-Breaking Oral Presentation of First-in-Human Data from in vivo BCMA CAR-T Therapy at the American Society of Hematology (ASH) 2025 Annual Meeting (Businesswire) - "Early results from the Phase 1 inMMyCAR study demonstrate ongoing MRD-negative responses in all patients with a tolerable safety profile....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."

First-in-human • Late-breaking abstract • P1 data • Multiple Myeloma

Kelonia Therapeutics Doses First Patient in Phase 1 inMMyCAR Study Evaluating in vivo CAR T-Cell Therapy for Relapsed and Refractory Multiple Myeloma (Businesswire) - "inMMyCAR is a multi-center Phase 1, open-label, dose-escalation clinical trial designed to assess the safety and preliminary efficacy of a single dose of KLN-1010. The first patient was dosed at Royal Prince Alfred Hospital, Sydney, Australia."

Trial status • Multiple Myeloma

inMMyCAR: A Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: Kelonia Therapeutics, Inc.

New P1 trial • Cardiovascular • Gene Therapies • Hematological Disorders • Hematological Malignancies • Monoclonal Gammopathy • Multiple Myeloma • Oncology

Kelonia Therapeutics to Participate in Upcoming Scientific Conferences (Businesswire) - "Kelonia Therapeutics...announced preclinical data from its lead in vivo CAR-T cell therapeutic candidate KLN-1010 in multiple myeloma that will be highlighted at two upcoming scientific conferences – the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, taking place on May 7-11, 2024, in Baltimore, Maryland, and the Protein Engineering Summit (PEGS) Boston Summit 2024, taking place on May 13-17, 2024, in Boston...With KLN-1010 poised to be the first in vivo CAR-T cell therapy for multiple myeloma, we aim to provide a potentially life-changing solution for patients that need it most."

Preclinical • Hematological Malignancies • Multiple Myeloma • Oncology

T cell-specific in vivo transduction with preclinical candidate KLN-1010 generates BCMA directed CAR T cells with potent anti-multiple myeloma activity (AACR 2024) - "These data demonstrate that KLN-1010 is safe and efficacious in preclinical models and may offer greater accessibility than other therapeutic modalities to address an unmet medical need in MM. Without requirements of ex vivo manufacturing, lymphodepletion and inpatient treatment that restrict patient access to therapy, the iGPS technology is well poised to provide significant clinical benefit for a multitude of indications as an off-the-shelf, single dose treatment."

CAR T-Cell Therapy • IO biomarker • Preclinical • Hematological Malignancies • Multiple Myeloma • Oncology • CD4 • CD8

Kelonia Therapeutics to Present Preclinical Data Highlighting Therapeutic Potential of in vivo CAR-T Cell Therapy in Multiple Myeloma (Businesswire) - "Kelonia Therapeutics...announced new preclinical data from its lead program KLN-1010, which will be presented at the American Association for Cancer Research (AACR) Annual Meeting 2024 in San Diego, California. KLN-1010, a novel, in vivo CAR-T cell therapy candidate for the treatment of multiple myeloma, which leverages the company’s in vivo Gene Placement System (iGPS) technology, demonstrated that it is safe and effective in preclinical animal models....A single intravenous injection of KLN-1010 displayed potent anti-tumor efficacy and caused complete tumor regression at multiple dose levels in several preclinical animal models. In other studies, treatment of non-human primates with an iGPS particle expressing an anti-CD20 CAR resulted in potent CAR-T cell activity lasting several months without the need for additional treatments or conditioning chemotherapy."

Preclinical • Hematological Malignancies • Multiple Myeloma • Oncology

Kelonia Therapeutics Presents Research Highlighting Potential of iGPS in vivo Gene Therapy to Bring Safe, Effective CAR T Therapies to Common Medical Practice for Treatment of All Multiple Myeloma Patients (Businesswire) - "Kelonia Therapeutics...announced the results of preclinical research demonstrating that its in vivo Gene Placement System (iGPS™) technology efficiently delivered CAR molecules specifically to T cells at therapeutic dose levels in both mice and non-human primates (NHPs). Kelonia’s iGPS platform enables CAR T cell therapy without the need for lymphodepleting chemotherapy or time-consuming ex vivo manufacturing. These data demonstrate the potential for iGPS particles to be a highly effective, safe, 'off-the-shelf' therapy for patients with multiple myeloma, which Kelonia is pursuing as its lead indication. Kelonia shared the data for the first time during an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting in Los Angeles."

Preclinical • Hematological Malignancies • Multiple Myeloma • Oncology