ETX-312 well tolerated at exaggerated dose levels in GLP-compliant toxicology studies
GMP manufacturing of clinical trial batch successfully completed
On track for CTA submission in Q4 2025
LONDON, July 10, 2025 (GLOBE NEWSWIRE) -- e-therapeutics plc, a company integrating computational power and biological data to discover life-transforming RNAi medicines, today announced significant progress on its lead candidate, ETX-312, a GalOmic siRNA therapy for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). The Company has successfully de-risked the path to clinical entry for ETX-312 and remains on track to submit a clinical trial application (CTA) in Q4 2025.
ETX-312 was repeatedly administered at doses far exceeding anticipated clinical exposure in GLP-compliant toxicology and toxicokinetic studies. The candidate was well tolerated across all dose levels, with no toxicity or adverse findings observed. These non-clinical results support a broad therapeutic window for ETX-312 and strengthen confidence in the program and the GalOmic platform as it advances toward first-in-human trials.
In parallel, e-therapeutics has successfully completed GMP manufacturing of the clinical batch of ETX-312, supporting the Company's operational readiness to progress to first-in-human dosing. Productive interactions with regulatory authorities have provided further support for our clinical strategy and plans.
"Recent results and progress on ETX-312 represent a pivotal step towards the clinical development of our first GalOmic therapeutic, designed to silence a novel target identified in-house" said Ali Mortazavi, CEO of e-therapeutics. "The tolerability profile of ETX-312 provides key de-risking for our program, supporting a Q4 2025 CTA filing. We look forward to advancing ETX-312 to first-in-human studies over the coming months and continuing to execute efficiently to offer a differentiated treatment option to MASH patients."
About ETX-312
ETX-312 is a GalOmic GalNAc-conjugated small-interfering RNA (GalNAc-siRNA) therapeutic candidate in development as 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 dramatic reductions in NAFLD Activity Score (NAS), decreased hepatic inflammation, and slowed fibrosis progression, both as monotherapy and in synergistic combination with approved and 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, GalOmic, enables generation of specific, potent, and durable siRNA therapeutics for effective silencing of novel gene targets in hepatocytes. The cutting-edge HepNet computational platform allows ETX to discover better medicines faster through generation of novel insights and increased automation across all stages of drug development. HepNet 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 HepNet and GalOmic, 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 GalOmic 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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