Amphista Therapeutics is showcasing its innovative approach to targeted protein degradation (TPD) with presentations at two major scientific conferences this month. The company will detail its novel mechanism for degrading BRD9, an emerging target in Acute Myeloid Leukemia, using its proprietary Targeted Glue® technology.
The presentations, scheduled for the 2nd SMR Molecular Glues meeting in Stevenage, UK on March 21, and the 5th Annual TPD and Induced Proximity Summit Europe in London on March 26, will highlight how Amphista's technology catalyzes degradation via DCAF16, a relatively uncharacterized E3 ligase, differentiating it from traditional cereblon- or VHL-based PROTACs.
Dr. Marta Carrara, Associate Director of Target Discovery & Validation at Amphista, will deliver the oral presentations describing the generation of selective and potent degraders of BRD9. The data demonstrates the strength of the company's proprietary chemistry and its Eclipsys™ platform in enabling the rational design of orally bioavailable protein degraders.
Novel Mechanism of Action
Amphista's approach represents a significant advancement in the TPD field. Unlike conventional degraders that rely on predefined E3 ligases like CRBN or VHL, Amphista's Targeted Glue® technology creates a novel interface on the target protein that is recognized by the most effective E3 ligase for driving its degradation.
"By allowing the target protein to choose its optimal E3 ligase for degradation, we're expanding the universe of accessible E3 ligases beyond the handful currently utilized in the industry," explained Dr. Louise Modis, Chief Scientific Officer at Amphista Therapeutics. "This translates to smaller, drug-like molecules that can potentially impact a broader range of diseases."
The company's BRD9 degraders have demonstrated rapid, deep, and persistent degradation through the recruitment of DCAF16, offering potential advantages over existing approaches.
Eclipsys® Platform Capabilities
At the recent SLAS Europe 2025 Conference in Hamburg, Germany, Amphista presented its Eclipsys® platform, which enables the systematic identification of fast, potent, and selective degraders against previously undruggable targets.
Dr. Simon Gilbert, Associate Director of Discovery Biology at Amphista, presented the company's systematic process for developing Targeted Glue™ degraders. "Amphista's unique medicinal chemistry creates a novel interface on the target protein that is recognized by the E3 ligase most effective in driving its degradation," Dr. Gilbert noted.
The platform's success was demonstrated using a Hit Identification screen for an oncology target that generated diverse, potent hits. Amphista's scientists successfully deconvoluted the degrader mechanism of action and identified promising candidates for further development through cryo-EM enabled Structure Based Drug Discovery.
Clinical Development Plans
Amphista plans to progress its BRD9 degrader for Acute Myeloid Leukemia into clinical trials in 2026. The company believes its portfolio offers the potential to deliver first- and/or best-in-class therapeutics with performance characteristics beyond the limitations of CRBN and VHL-based agents.
"Our Eclipsys® platform represents a paradigm shift in the TPD field and has delivered our pipeline of first- and/or best-in-class Targeted Glues™," said Dr. Modis. The technology is designed to create therapeutics that can potentially transform the lives of patients with severe diseases, including cancer and neurodegenerative disorders.
Company Background
Amphista Therapeutics 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.
The company is focused on transforming the lives of patients with severe diseases through the discovery of advanced, next-generation targeted protein degradation medicines. Its proprietary technology creates sequentially bifunctional Targeted Glue™ therapeutics with differentiated mechanisms and leading drug-like properties.
With its innovative approach to protein degradation, Amphista is positioning itself as a leader in the next wave of targeted protein degradation therapeutics, potentially offering new treatment options for patients with limited therapeutic alternatives.
