On December 8, 2025 Amphista Therapeutics ("the Company" or "Amphista"), a leader in the discovery of next generation targeted protein degradation (TPD) medicines, reported that full details of its new preclinical data with its lead Targeted Glue AMX-883, an orally bioavailable, potent and selective degrader of BRD9 were presented on 6 December during the 67th American Society of Haematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando, Florida.

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The compelling findings support the potential of AMX-883 as a first-line treatment option in the earlier disease setting of acute myeloid leukaemia (AML), one of the most aggressive blood cancers where 5-year survival rates remain at just 33% and where resistance to standard of care treatments like venetoclax remains a major clinical challenge. Based on these data, the Company will advance AMX-883 as a karyotype-independent, pro-differentiation agent into the clinic for AML in H2 2026.

The key data presented at ASH (Free ASH Whitepaper) showed that:

AMX-883 demonstrates potent degradation as a monotherapy and synergistic benefit in combination with venetoclax

AMX-883 (30mg/kg twice daily and 100mg/kg once daily) significantly reduced cancer growth by achieving picomolar potency degradation of BRD9 across a panel of AML cell lines and significantly reduced leukemic burden in bone marrow and blood in vivo, compared to venetoclax alone.
Synergistic efficacy was observed when each dose of AMX-883 was combined with venetoclax (75mg/kg once daily), leading to significant blocking of tumour growth at therapeutically relevant concentrations.
AMX-883 prevents resistance to venetoclax when dosed in combination

The combination of AMX-883 with venetoclax in a venetoclax resistant cell line, prevented the emergence of resistance, and cells had comparable sensitivity to venetoclax from their first exposure.
AMX-883 actively degrades BRD9 in venetoclax resistant cells, prevents upregulation of key resistance markers, including MCL-1 and BCL-2, and increases levels of cell death markers.
Martin Pass, Chief Development Officer at Amphista Therapeutics said, "The important preclinical data presented for the first time show that our Targeted Glue AMX-883, a selective degrader of BRD9, extends the durability and efficacy of combination therapy with venetoclax and prevents AML cancer cells from becoming resistant to venetoclax. We are very pleased to receive positive feedback from experts in the field on this compelling profile, which gives us strong reason to believe that AMX-883 could be a viable treatment option for patients with AML. We look forward to commencing the first clinical trial for this devastating blood cancer in H2 2026."

To deliver the first clinical study of AMX-883, Amphista has built its development capabilities with three key strategic leadership appointments, including Dr. Lisa Butler, Senior Vice President of Clinical Operations who was previously Head of Study Leadership, early Oncology Clinical at AstraZeneca.

The abstract presented at ASH (Free ASH Whitepaper) is available on the congress website.

(Press release, Amphista Therapeutics, DEC 8, 2025, View Source [SID1234661249])