Axcynsis Therapeutics Receives FDA Clearance for IND Application of AT03-65, a Differentiated CLDN6-Targeting ADC, Powered by AxcynDOT™ Technology

AT03-65 is the first program utilizing AxcynDOT™, a proprietary payload with a differentiated mechanism of action, to enter clinical development

Axcynsis Therapeutics Pte Ltd ("Axcynsis"), a privately held biopharmaceutical company specialized in delivering Antibody Drug Conjugates (ADCs) with breakthrough potential, proudly announces the clearance of its Investigational New Drug application (IND) by the United States Food and Drug Administration (FDA) of AT03-65 for the treatment of patients with CLDN-6 positive solid tumors. Axcynsis is planning to initiate a Phase 1 multicentre clinical trial in the United States in 1Q 2025.

AT03-65 is a differentiated ADC that selectively binds to CLDN6 with strong affinity. It is enabled by AxcynDOT™, a proprietary payload that incorporates a derivative of an approved oncology therapeutics with unique mechanism of action and broad anti-tumor activity, and coupled with a cleavable and hydrophilic proprietary linker. CLDN6 is overexpressed in many cancers including lung, ovarian, endometrial, uterine, testicular, and gastric cancers while exhibiting minimal expression in normal tissues.  AT03-65 is designed to deliver targeted therapy to improve patient outcomes with advanced or metastatic CLDN6-positive cancers.

"This is a transformational event for Axcynsis and a significant milestone for our proprietary ADC platform using AxcynDOT™," said Dr. Zou Bin, CEO of Axcynsis. "We are pleased that FDA has cleared AT03-65 which leverages our AxcynDOT™ technology for this first-in-human study.  We are very excited with the potential of offering a transformative therapeutic option for patients with CLDN6-positive tumors as well as advancing our pipeline with  differentiated  and effective ADCs using AxcynDOT™ to improve the lives of cancer patients worldwide".

The upcoming Phase 1 multicentre clinical trial in the United States will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of AT03-65 in patients with advanced CLDN6-positive solid tumors.

About AT03-65

AT03-65 is a recombinant anti-CLDN6 monoclonal antibody conjugated to AxcynDOT™, a proprietary payload developed by Axcynsis with a differentiated mechanism of action and broad anti-cancer activities.  AT03-65 is designed to target advanced, recurrent, or metastatic CLDN6+ solid tumors in patients who have progressed on or after standard systemic treatment or for whom no standard therapies are available.

The antibody of AT03-65 is rationally engineered for high affinity and specificity to CLDN6. Upon binding to CLDN6-expressing tumor cells, the ADC is internalized into lysosomes, where it releases its payload to inhibit tumor growth effectively. Preclinical studies demonstrate that AT03-65 not only directly kill CLDN6-positive tumor cells but also exhibits bystander killing effect, targeting neighboring CLDN6-negative tumor cells to enhance its anti-tumor efficacy. AT03-65 has demonstrated promising anti-tumor activities in multiple tumor mouse models and a favorable safety profile in non-human primates.

About AxcynDOT™

This is a proprietary payload developed by Axcynsis. It is a derivative of trabectedin, an approved chemotherapy in the United States, Europe and selected Asian countries, with a unique mechanism of action that is differentiated from other DNA alkylating payloads. AxcynDOT™ is optimized with enhanced potency and improved safety profile compared to trabectedin.    

About Axcynsis Therapeutics

Axcynsis Therapeutics is a pioneering biotechnology company headquartered in Singapore with operations in the United States. The company has developed AxcynDOT™ , a proprietary linker payload platform to advance a pipeline of differentiated ADC candidates aimed at addressing key unmet medical needs. The company is committed to advance effective and targeted oncology therapeutics with breakthrough potential to improve the lives of cancer patients worldwide.