CAMBRIDGE, UK, June 8, 2026
Amphista Therapeutics announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AMX-883, the company’s lead Targeted Glue™ degrader being developed for the treatment of acute myeloid leukaemia (AML). The FDA decision enables Amphista to advance AMX-883 into its first human clinical trial, marking a major milestone for the company as it transitions into a clinical-stage biotechnology organization focused on next-generation targeted protein degradation therapies.
First Clinical Trial Planned for H2 2026
The IND clearance allows Amphista to initiate a Phase 1 monotherapy dose-escalation and optimization study evaluating AMX-883 in patients with relapsed or refractory AML as well as individuals with high-risk myelodysplastic syndrome (MDS), a serious bone marrow disorder that can progress into acute myeloid leukaemia. The study is expected to begin during the second half of 2026 and will focus on assessing the safety, tolerability, pharmacokinetics, and preliminary clinical activity of the investigational therapy. The trial represents the first clinical evaluation of AMX-883 and an important step toward validating the company’s proprietary targeted protein degradation platform in oncology.
AMX-883 Offers a Novel BRD9 Degradation Approach
AMX-883 is an orally available, non-cereblon degrader designed to target BRD9, a protein implicated in the development and progression of certain blood cancers. According to Amphista, the investigational therapy is currently the only BRD9 degrader in clinical development. The drug utilizes a DCAF16-dependent degradation mechanism, providing a differentiated approach compared with existing targeted therapies. As a broad-acting, pro-differentiation agent, AMX-883 acts independently of karyotype status, potentially allowing it to benefit a wider population of AML patients than many currently available precision medicines that are limited to specific genetic mutations. Preclinical studies submitted to the FDA demonstrated encouraging activity, supporting further clinical development in one of the most aggressive forms of blood cancer.
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Combination Development Strategy Targets Treatment Resistance
Following evaluation of AMX-883 as a monotherapy, Amphista plans to investigate the candidate in combination with venetoclax and azacitidine, two commonly used therapies in AML treatment. Resistance to existing therapies remains a significant challenge in AML, contributing to poor long-term outcomes for many patients. Company executives believe the unique mechanism of AMX-883 may offer an opportunity to overcome resistance pathways and improve treatment responses. With AML patients continuing to face limited therapeutic options and a five-year survival rate of approximately 33%, the development of innovative therapies remains a critical priority across the hematology field.
Amphista Expands Targeted Protein Degradation Pipeline
The FDA clearance represents a significant achievement for Amphista Therapeutics and further validates its Eclipsys® platform, which is designed to develop next-generation Targeted Glue™ therapeutics. The company is focused on creating bifunctional degraders capable of selectively eliminating disease-causing proteins while offering advantages beyond traditional CRBN- and VHL-based degradation approaches. Amphista’s broader pipeline includes programs targeting cancer and neurodegenerative diseases, with the goal of delivering first- and best-in-class therapies. Supported by leading healthcare investors and strategic backers, the company is positioning itself as a key innovator in the rapidly expanding field of targeted protein degradation. The advancement of AMX-883 into clinical development marks a major step toward translating this technology into potential new treatment options for patients with difficult-to-treat cancers.
Source: Amphista Therapeutics press release
