Akari Therapeutics Reports Positive Preclinical Data for AKTX-101 Demonstrating Differentiated Cytotoxicity for First-in-Class TROP2 ADC Payload Targeting RNA Splicing
Superior potency demonstrated versus leading TROP2 ADCs across bladder, lung and breast tumor models
Novel RNA spliceosome-targeting payload PH1 shows potential to overcome Topoisomerase I inhibitor resistance
Preclinical data support advancement of AKTX-101 into Phase 1 studies in a rapidly evolving TROP2 ADC class expected to reach ~$12B by 20331
TAMPA, Fla. and LONDON, April 20, 2026 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (NASDAQ:AKTX), an oncology biotechnology company developing antibody drug conjugates (ADCs) with a novel RNA splicing modulator payload, today announced the presentation of positive preclinical data for its lead TROP2-targeting ADC, AKTX-101, at the American Association for Cancer Research (AACR) Annual Meeting 2026. Access the poster here.
Unlike current TROP2-targeting ADCs that use Topoisomerase I Inhibitor (Inh.) payloads, AKTX-101 has the potential to address resistance to Topoisomerase I Inh. ADCs and contribute to durable anti-tumor efficacy due to the payload's unique cytotoxic and immune-activating mechanisms of action.
The preclinical data compares the performance of AKTX-101 versus TROP2 ADCs with Topoisomerase I Inh. payloads in the killing of different cancer types driven by different cancer genes (oncogenes). AKTX-101's ability to kill cancer cells at lower concentrations vs. TROP2 ADCs using Topoisomerase I Inh. payloads suggests that AKTX-101 is a more potent drug.
The preclinical data was published recently as an abstract in Cancer Research, an AACR journal.
Here, AKTX-101 demonstrated greater potency and/or greater maximum cancer cell killing relative to TROP2 ADC Topoisomerase I Inh. payloads in cancers of the bladder, lung and breast. AKTX-101 demonstrated sub-nanomolar potency in all bladder cancer lines tested, a key tumor in which first-in-human clinical trials for AKTX-101 are planned.
AKTX-101 also demonstrated sub-nanomolar potency in several non-small cell lung cancer cell lines driven by EGFR, BRAF, and SMARCA4, as well as potent cell killing in HER2 breast cancer cell lines with inherent resistance to Topoisomerase I Inh. ADCs such as trastuzumab deruxtecan (ENHERTU™).
"These data represent a significant step forward for our lead program, AKTX-101, and reinforce our belief that a differentiated ADC payload with multiple mechanisms of action has the potential to meaningfully improve outcomes for patients with TROP2-expressing cancers," commented Satyajit K. Mitra, Ph.D., Head of Oncology Research and Development at Akari Therapeutics. "We are seeing preclinical superior AKTX-101 potency and activity as compared to TROP2 ADCs using Topoisomerase I inhibitor payloads in bladder, lung, and breast cancer models. Together, these findings show that AKTX-101 has strong potential for targeting a broad range of cancer tumors and sub-types with superior cytotoxicity than current TROP2 ADCs that use Topoisomerase I Inhibitor payloads."
The TROP2 ADC class continues to emerge in terms of its potential, with revenue projections expected to reach ~$12B or greater by 2033 based on current and future entrants. Akari believes that AKTX-101, with its novel RNA splicing modulator payload, can grow this class further by addressing multiple solid tumors where TROP2 is overexpressed including bladder, lung, breast, pancreatic, head and neck, and others.
Key AKTX-101 Data Presented at AACR Highlights:
- AKTX-101 demonstrated strong, single-agent anti-tumor activity across multiple models across bladder, lung, and breast cancers.
- AKTX-101 demonstrated greater potency and cell killing compared to current TROP2 ADCs, including Topoisomerase I inhibitor-resistant tumor models, as well as standard-of-care chemotherapies and targeted therapies. Combination of AKTX-101 with anti-PD-1 therapy resulted in synergistic anti-tumor efficacy and tumor regressions within in vivo models, supporting future combinations with checkpoint inhibition to maximize tumor remissions rates.
- Broad in vitro cytotoxicity was observed across a diverse panel of tumor models, including those with clinically oncogenic driver mutations including FGFR3, BRAF, EGFR, and SMARCA4.
Abizer Gaslightwala, CEO of Akari Therapeutics, added, "This data continues to add to the conviction and differentiation we have in our novel ADC payload PH1 targeting RNA splicing. We are focused on rapidly advancing AKTX-101 into the clinic, with IND-enabling studies underway and plans to submit an IND in the fourth quarter of 2026, followed by initiation of a Phase 1 study in the first quarter of 2027. Our team is executing against a clear development plan designed to efficiently translate these encouraging preclinical findings into clinical proof of concept."
These data were presented at the American Association for Cancer Research (AACR) Annual Meeting 2026. Access the poster here.
¹ DataIntelo, TROP2-Targeted Therapies Market Report, 2026
About Akari Therapeutics
Akari Therapeutics is an oncology biotechnology company developing next-generation antibody drug conjugates (ADCs) with a unique payload, PH1, which targets RNA splicing. Utilizing its innovative ADC discovery platform, the Company has the ability to generate ADC candidates and optimize them based on the desired application to any antigen target of interest. Akari's lead candidate, AKTX-101, targets the TROP2 receptor on cancer cells with a proprietary linker, enabling it to deliver its novel PH1 payload directly into the tumor with minimal off-target effects. Unlike current ADCs that use microtubule inhibitors and DNA damaging agents as their payloads, PH1 is a novel payload that is a spliceosome modulator designed to disrupt RNA splicing within cancer cells. This splicing modulation has been shown in preclinical animal models to induce cancer cell death while activating both the innate and adaptive immune systems to drive robust and durable activity. In preclinical studies, AKTX-101 has shown to have significant activity and prolonged survival relative to ADCs with traditional payloads. Additionally, AKTX-101 has the potential to be synergistic with checkpoint inhibitors and has demonstrated prolonged survival as both a single agent and in combination with checkpoint inhibitors. The PH1 payload has also been demonstrated to be very active against cancer cells with key oncogenic drivers such as KRAS, BRAF, ARV7, FGFR3 fusions, and others. The Company has initiated IND enabling studies for AKTX-101 with a goal of starting its First-In-Human trial by late 2026/early 2027. Akari is also developing AKTX-102, an ADC candidate targeting CEACAM5 (Carcinoembryonic Antigen-related Cell Adhesion Molecule-5), a well-validated tumor antigen broadly expressed across multiple solid tumors. AKTX-102 is designed to leverage Akari's proprietary PH1 spliceosome-modulating payload and a novel antibody construct to enable differentiated tumor cell killing and immune activation.
For more information about the Company, please visit www.akaritx.com and connect on X and LinkedIn.
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