ETERN BioPharma
Advancing Drug Discovery to Target ‘Undruggable’ Proteins

Biomolecular condensates, microscopic droplets in cells containing vital proteins and molecules, offer the potential for groundbreaking therapeutics. Yet, their targeting is deemed ‘undruggable’ due to the lack of well-defined binding sites and their fluid, ever-changing nature within cells. This complexity challenges conventional drug development, necessitating innovative approaches to tap into the therapeutic possibilities of biomolecular condensates.

ETERN BioPharma, a biotech company, has found a novel method of targeting these ‘undruggable’ biomolecular condensates through liquid-liquid phase separation (LLPS).

“LLPS is a fundamental mechanism regulating biomolecule functions, implicated in various diseases like cancer, neurodegenerative disorders, metabolic issues, and inflammation. These diseases, which don’t have effective treatments yet stand to benefit significantly from the promising LLPS technology,” says Prof. Jidong Zhu, co-founder and CEO of ETERN BioPharma.

The company’s approach involves leveraging LLPS technology to develop small molecule drugs, particularly targeting transcriptional factors. There are about 1600 transcription factors encoded by the human genome, and about 300 of them are linked to human diseases. However, transcription factors are tough to target with conventional drugs because they work by interacting with other proteins or DNA, which are not easy to bind with small molecules.

With this key understanding, ETERN is using its LLPS technology to target transcription factors and hopes to find new ways to modulate gene expression. This innovative approach opens doors for developing transcriptional factor inhibitors, crucial for diseases like cancer where oncogenic transcription factors form biomolecular condensates.

It has a focused pipeline and has made significant progress, anticipating clinical trials in both the U.S. and China next year. Confident in its distinct approach, the company is dedicated to identifying novel targets and inhibitors to address unmet medical needs, showcasing a promising avenue for drug discovery.

A Cutting-Edge, AI-Driven Platform

ETERN’s pioneering strategy, based on LLPS technology, integrates AI-assisted phase separation target discovery, high-throughput drug screening, and optimization techniques. It harnesses big data analysis and machine learning to integrate diverse experimental data, unveiling hidden correlations and trends for deeper insights.

Automated equipment ensures efficient operations and precise data recording, greatly enhancing reproducibility. By rapidly assessing numerous targets and compounds, it swiftly identifies potential drug candidates.

The platform leverages MD simulation approach to aid in the optimization of compound structures, which is beneficial for targets like transcription factors lacking stable structures.

These capabilities help ETERN in developing therapeutic modalities for challenging targets untouched by conventional drug discovery. It can address critical questions in biology, elucidating the intricate relationship between targets and diseases.

Through comprehensive analysis and validation of protein LLPS, ETERN gains profound insights into target-disease interactions, offering novel targets and research directions for drug development and therapeutic discovery. Years of dedicated exploration and research have allowed it to conduct in-depth studies on the phase separation characteristics of undruggable targets. As a result, it has formulated a comprehensive set of processes for developing new targets and screening optimized compounds.

This data-driven approach accelerates drug discovery by providing accurate information on the composition and function of the undruggable protein condensates to activate it.

A Team Backed by LLPS Knowledge and Expertise

ETERN stands out as a science-driven entity. Its co-founders, including Zhu and Prof. Junying Yuan, hail from academic backgrounds with a focus on LLPS and cell death field research, respectively. Their pride lies in deriving each drug discovery project from their own publications, such as the AR-V7 targets originating from the recent Nature Chemical Biology publication.

The progress of this program is evident, with achievements like a compound demonstrating potent anti-tumor efficacy in mouse models.

This is what propels ETERN’s team, allowing them to be at the helm of challenging drug discovery targets. It thrives on creativity, team spirit, and energy. Comprising experienced members from multinational and various other companies, the research team is navigating the previously unexplored potential of LLPS technology.

ETERN’s research group composes of the LLPS exploratory and drug discovery teams. The organization itself functions like a phase-separated research group. The LLPS platform serves as a light phase, allowing the exploratory team to explore new tools, assays, and targets. While the challenging nature of the programs may result in higher failure rates, ETERN recognizes the value of learning from these setbacks. Creating an environment that fosters scientific curiosity and creativity is a priority.

As projects progress into the drug discovery stage, the team transitions to a more disciplined and timeline-focused approach like a dense phase. The goal is to deliver effective drugs to patients. Despite the different dynamics and roles in each phase, the teams consistently exchange ideas, recognizing the importance of creativity and discipline in their drug discovery efforts.

The ETERN team’s robust publication record reflects a strong foundational understanding of LLPS, showcasing its ability to translate research into drug discovery. All its pipelines are grounded in published papers, a groundbreaking demonstration of its novel approach to drug development.

Success is rooted in the company’s motto, ‘In science we believe, to health we devote.’