E-Therapeutics Presents New Data Supporting ETX‑312 As A Differentiated And Disease-Modifying Near-Clinic Treatment For MASH
In the leading Gubra‐Amylin NASH diet‐induced obese (GAN‐DIO) mouse model, ETX‐312 demonstrated:
- Dramatic improvements in NAFLD Activity Score (NAS) when administered as monotherapy or in combination with a GLP‐1/GIP receptor agonist or an FGF‐21 analogue. All mice receiving combination therapy achieved ≥ 2‐point reductions in NAS, with improvements of up to 5 points observed.
Slowed fibrosis progression comparable to that achieved with GLP‐1/GIP receptor agonist or FGF‐21 analogue controls, supported by statistically significant reductions in hepatic collagen staining and in circulating biomarkers TIMP‐1 and PIIINP.
“These data add to a growing body of evidence supporting ETX-312 as a differentiated, disease-modifying therapy for MASH,” said Alan Whitmore, Chief Scientific Officer.“We are excited to progress the programme towards the clinic.”
ETX‐312 is currently in IND‐enabling studies, and the Company remains on track to submit a regulatory submission by the end of 2025.
Presentation Details
Title: ETX-312, a GalOmic siRNA for the treatment of MASH, effectively improves the MASH phenotype of GAN DIO-MASH mice alone or in combination with emerging therapies
Poster Number: FRI-337
Date: 9th May 2025
About ETX-312
ETX‐312 is a GalOmic GalNAc-conjugated small-interfering RNA (GalNAc-siRNA) therapeutic candidate in development to be a safe and effective treatment for metabolic dysfunction-associated steatohepatitis (MASH) with potential for a quarterly subcutaneous dosing regimen. In preclinical studies using the Gubra-Amylin NASH diet-induced obese (GAN‐DIO) mouse model, administration of ETX-312 led to reductions in NAFLD Activity Score (NAS), decreased hepatic inflammation, and slowed fibrosis progression, both as a monotherapy and in combination with emerging therapies. ETX-312 is currently progressing through IND-enabling studies, with a regulatory submission planned by the end of 2025.
About e-therapeutics plc
e-therapeutics plc ("ETX") uniquely combines computation and RNAi to discover and develop life-transforming medicines. ETX's proprietary RNAi chemistry platform, GalOmicTM, enables generation of specific, potent, and durable siRNA therapeutics for effective silencing of novel gene targets in hepatocytes. The cutting-edge HepNetTM computational platform allows ETX to discover better medicines faster through generation of novel insights and increased automation across all stages of drug development. HepNetTM encompasses an extensive hepatocyte-specific knowledgebase and a suite of advanced AI-driven approaches which enable identification of novel gene targets, rapid target-indication assessment, and predictive in silico siRNA design. The Company has specialist expertise and a robust position in applying computation to biology. Its computational approaches have been extensively validated through generation of data from pipeline programs and successful drug discovery collaborations with biopharma companies, such as Novo Nordisk, Galapagos NV, and iTeos Therapeutics.
Leveraging the combined capabilities of HepNetTM and GalOmicTM, ETX is progressing a therapeutic pipeline of highly differentiated RNAi candidates across a variety of therapeutic areas with high unmet need. The Company has generated positive proof-of-concept data on preclinical assets in metabolic dysfunction-associated steatohepatitis (MASH), dry age-related macular degeneration (dry AMD), haemophilia, heart failure, and cardiometabolic disease, further validating its computationally enhanced approach to research and development. ETX is currently progressing its GalOmicTM therapies towards the clinic with its most developed assets, ETX-312 for MASH, ETX-148 for bleeding disorders, and ETX-407 for dry AMD, at the IND-enabling stage.
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