- Amphista to initiate its first clinical trial for AMX-883, a potent, orally bioavailable DCAF16-dependent degrader of BRD9, for acute myeloid leukaemia (AML) in H2 2026
- The Phase 1 monotherapy dose-escalation and optimisation trial will enrol patients with relapsed or refractory AML and high-risk myelodysplastic syndrome
- As a broad-acting, pro-differentiation agent, AMX-883 acts independently of karyotype status and has the potential to benefit a wider population of AML patients than current targeted therapies
Cambridge, UK, 8June 2026 - Amphista Therapeutics ("the Company" or "Amphista"), a leader in the discovery and development of next generation targeted protein degradation (TPD) medicines, today announces that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for AMX-883, an orally available non-cereblon degrader of BRD9, for the treatment of acute myeloid leukaemia (AML). AMX-883 is expected to enter the clinic in H2 2026.
IND clearance allows Amphista to start its Phase 1 monotherapy dose-escalation and optimisation clinical trial of AMX-883 in patients with relapsed or refractory AML and high-risk myelodysplastic syndrome (MDS), a related bone marrow disorder that often progresses to AML. After establishing the monotherapy profile, the Company intends to explore AMX-883 in combination with venetoclax and azacitidine in early lines of therapy, where treatment resistance continues to pose a major clinical challenge.
Louise Modis, Chief Executive Officer at Amphista, said: "FDA clearance of our IND for AMX-883, our lead Targeted Glue, for acute myeloid leukaemia is a significant milestone as we transition into a clinical-stage company. AMX-883 is the only BRD9 degrader currently being developed and the compelling preclinical findings submitted to the FDA support its potential as a first-line treatment option in the earlier disease setting in one of the most aggressive blood cancers. We look forward to commencing the clinical trial in H2 2026."
Patrick Kelly, Chief Medical Officer at Amphista, added: "AML remains one of the most devastating blood cancers, with a 5-year survival rate of just 33%, and resistance to standard-of-care treatments like venetoclax a critical challenge. FDA clearance of our IND for AMX-883 supports the advancement of a differentiated therapeutic approach, with the potential to establish an important new treatment pathway for patients in urgent need of innovative therapies."
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About Amphista Therapeutics
Amphista Therapeutics is focused on transforming the lives of patients with severe diseases, including cancer and neurodegenerative disorders, through the discovery of advanced, next-generation targeted protein degradation (TPD) medicines. Using its proprietary Eclipsys platform, Amphista develops unique bifunctional Targeted Glue therapeutics with a differentiated mechanism and leading drug-like properties. Its portfolio has the potential to deliver first- and/or best-in-class therapeutics with performance characteristics beyond the limitations of CRBN- and VHL-based agents. Amphista was co-founded by Advent Life Sciences and is additionally funded by a premier group of investors including Forbion, Gilde Healthcare, Novartis Venture Fund, SV's Dementia Discovery Fund and Eli Lilly. For more information, please visit: www.amphista.com
Amphista, Eclipsys, Targeted Glue, Targeted Glues and the Amphista logo are all trademarks or registered trademarks of Amphista Therapeutics Limited.
About BRD9 and Acute Myeloid Leukaemia
Acute myeloid leukaemia (AML) is one of the most aggressive blood cancers. Despite the availability of anti-proliferative treatments, survival remains poor, with a 5-year survival rate of just 33%, and AML is estimated to cause around 130,000 deaths globally each year. The disease is characterised by a differentiation block that prevents normal myeloid cell maturation and leads to a build-up of immature cells. Therapies that remove this block and allow AML blasts to mature-including ATRA, FLT-3 inhibitors and, more recently, menin inhibitors-have shown clinical benefit in certain AML subtypes. However, there remains a pressing need for broader-acting therapies that can benefit patients regardless of genetic profile.
BRD9 is a subunit of the non-canonical BAF complex and plays an important structural and functional role in regulating chromatin structure and maintaining genomic stability in AML. Degradation of BRD9 removes the differentiation block and drives the differentiation and death of AML blasts.
About AMX-883
AMX-883 is an orally bioavailable, potent and rapid degrader of BRD9. It uses Amphista's proprietary Targeted Glue technology to drive BRD9 degradation through recruitment of DCAF16, a mechanism distinct from cereblon- or VHL-based PROTACs. AMX-883 achieves near complete degradation of BRD9 within two hours of treatment, while retaining exquisite selectivity over all other bromodomain containing proteins, and beyond, as shown by global proteomics.
Amphista's preclinical data package supports the advancement of AMX-883 into clinical development as a karyotype-independent, pro-differentiation agent, with the first trial planned for H2 2026. In patient-derived tumour models, AMX-883-induced BRD9 degradation is sufficient to inhibit tumour growth in vivo as a monotherapy, while in combination AMX-883 shows synergistic efficacy with venetoclax in vivo and prevents the emergence of venetoclax resistance in vitro, addressing a major clinical challenge in acute myeloid leukaemia.
For more information please contact:
Amphista Therapeutics
John Goodall
Email: Info@amphista.com
ICR Healthcare
Namrata Taak, Ashley Tapp, Emily Johnson
Email: Amphista@icrhealthcare.com
Tel: +44 (0)20 3709 5813
