KLN-1010 / Kelonia Therapeutics, Eli Lilly - 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

Updated results from inMMyCAR, the ongoing first-in-human phase 1 study of KLN-1010 in patients (pts) with relapsed and refractory multiple myeloma (RRMM). (ASCO 2026) - P1 | "Cytokine release syndrome (CRS) was observed in 4 pts, with median onset on day 11 and median duration of 3.5 days; all events were Gr 2 and were managed with tocilizumab and corticosteroids. Results from inMMyCAR continue to demonstrate compelling early clinical activity. All pts achieved early MRD-negative responses and deepening of IMWG response over time. The pt with the longest follow-up remains MRD-negative with a stringent CR at M6."

Clinical • First-in-human • P1 data • Hematological Malignancies • Inflammation • Multiple Myeloma

SUCCESSFUL IN VIVO CAR-T GENERATION AND MINIMAL RESIDUAL DISEASE (MRD) CLEARANCE WITH KLN-1010 ACROSS DIVERSE BASELINE T CELL PHENOTYPES IN RELAPSED/REFRACTORY MULTIPLE MYELOMA (RRMM) (EHA 2026) - "Reused with permission. This abstract was accepted and previously presented at the 2026 ASCO Annual Meeting."

CAR T-Cell Therapy • IO biomarker • Minimal residual disease • Preclinical • Residual disease • Gene Therapies • Hematological Disorders • Hematological Malignancies • Infectious Disease • Inflammation • Multiple Myeloma • CD4 • CD8 • PD-1

In vivo CAR T-cell engineering in B-cell malignancies: A systematic review of emerging clinical and translational evidence. (ASCO 2026) - " We performed a literature search of peer-reviewed publications, conference proceedings (ASH, ASCO, EHA), and registered clinical trials (2018–2025) evaluating in vivo CAR T. Identified platforms included KLN-1010 (in vivo anti-BCMA CAR), INT2104 (CD20 CAR), Umoja CD22 CAR, Capstan CD8-targeted LNP CD19 CAR, and Orna circular RNA CD19 CAR systems... In vivo CAR T engineering has now demonstrated clinical proof-of-concept with deep molecular responses, robust in vivo CAR expansion, and manageable toxicity. Translational and preclinical CD19-, CD20-, and CD22-directed platforms show potent activity in aggressive lymphoma models, supporting direct applicability to DLBCL and HGBCL. Compared with ex vivo CAR T, in vivo systems offer elimination of leukapheresis, no manufacturing delays, outpatient feasibility, repeat dosing, and substantially reduced cost, potentially enabling earlier-line and broader access to CAR-T–level therapy."

CAR T-Cell Therapy • First-in-human • IO biomarker • Preclinical • Review • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • High-grade B-cell lymphoma • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • CD19 • CD20 • CD22 • CD8

In Vivo CAR T-Cell Therapy Spurs 100% ORR in Phase I. (PubMed, Cancer Discov) - "In the phase I inMMyCAR study of the in vivo chimeric antigen receptor (CAR) T-cell therapy KLN-1010, all 18 patients with multiple myeloma responded. None of the evaluable patients had minimal residual disease 1 month after treatment, suggesting that the therapy could prove clinically useful."

Journal • Preclinical • Hematological Malignancies • Multiple Myeloma • Oncology

Lilly to spotlight growing hematology portfolio at 2026 European Hematology Association (EHA) Annual Meeting (Eli Lilly Press Release) - "Positive results from the Phase 3 BRUIN CLL-322 study comparing time-limited pirtobrutinib plus venetoclax and rituximab versus venetoclax and rituximab in patients with relapsed or refractory CLL/SLL will be highlighted in a late-breaking oral presentation...Ajax Therapeutics will present the first clinical data from the Phase 1 AJX-101 study evaluating AJ1-11095, an investigational first-in-class type II JAK2 inhibitor, in patients with myelofibrosis who have been failed by a type I JAK2 inhibitor. Kelonia Therapeutics will present additional correlative data from the Phase 1 inMMyCAR study of an investigational anti-B-cell maturation antigen (BCMA) targeted in vivo CAR-T therapy in patients with relapsed and refractory multiple myeloma. Both proposed acquisitions by Lilly are pending transaction closes."

Late-breaking abstract • P1 data • P3 data • Chronic Lymphocytic Leukemia • Multiple Myeloma • Myelofibrosis • Small Lymphocytic Lymphoma

Kelonia Therapeutics Presents Updated First-in-Human Data from Phase 1 inMMyCAR Study of KLN-1010 in vivo BCMA CAR-T Therapy at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting (Businesswire) - "The interim data presented at ASCO demonstrate a 100% overall response rate (ORR) and minimal residual disease (MRD)-negative bone marrow at one month post treatment in all evaluable patients....First patient treated remains in deep, ongoing MRD-negative response beyond 10 months. Six patients with ≥ 4 months of follow up include 4 stringent complete response (sCR) and 2 very good partial response (VGPR), all with ongoing MRD-negative bone marrows."

First-in-human • P1 data • Multiple Myeloma

Kelonia Therapeutics Presentation: KLN-1010 (investigational in vivo CAR-T therapy) (PRNewswire) - "In a rapid oral presentation, updated results will be shared from the Phase 1 inMMyCAR study of KLN-1010 in patients with relapsed and refractory multiple myeloma."

P1 data • Multiple Myeloma

An integrated nonclinical strategy to support first-in-human evaluation of KLN-1010: efficacy and safety of lentiviral vector administration and in vivo–generated CAR-T cells (ASGCT 2026) - "Across relevant in vitro and in vivo models, KLN-1010 generated potent anti-BCMA CAR-T cells with anti- tumor activity comparable or superior to similarly designed approved ex vivo CAR-T products. Collectively, these data provided a robust and regulatory-ready nonclinical foundation supporting ongoing clinical evaluation of KLN-1010 in R/R MM."

CAR T-Cell Therapy • First-in-human • IO biomarker • P1 data • Preclinical • Viral vector • Dyslipidemia • Hematological Malignancies • Multiple Myeloma

inMMyCAR: A Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma (clinicaltrials.gov) - P1 | N=70 | Recruiting | Sponsor: Kelonia Therapeutics, Inc. | N=40 ➔ 70

Enrollment change • Cardiovascular • Gene Therapies • Hematological Disorders • Hematological Malignancies • Monoclonal Gammopathy • Multiple Myeloma • Oncology

Lilly to acquire Kelonia Therapeutics to advance in vivo CAR-T cell therapies (PRNewswire) - "Kelonia's lead program, KLN-1010...targeting the BCMA protein expressed on the surface of multiple myeloma cells....Under the terms of the agreement, Lilly will acquire Kelonia, and Kelonia shareholders will receive up to $7.00 billion in cash, inclusive of an upfront payment of $3.25 billion, and subsequent payments upon achievement of certain clinical, regulatory and commercial milestones. The transaction is subject to customary closing conditions, including customary regulatory approvals, and is expected to close in the second half of 2026."

M&A • Multiple Myeloma

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 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 (Businesswire) - "U.S. IND represents the second regulatory clearance for KLN-1010 and enables the first multi-center clinical trial in the U.S. for an anti-BCMA in vivo CAR-T program....FDA clearance of the IND enables Kelonia to expand its ongoing inMMyCAR Phase 1 clinical trial, which is currently enrolling patients in Australia, to multiple clinical sites across the United States."

IND • Trial status • Multiple Myeloma

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

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