16 January, 2024

Business Update

Proof-of-concept datasets on four assets

Two programs to progress to IND-enabling studies

Range of specialist AI agents in development


London, UK, 16 January 2024 - e-therapeutics plc (AIM: ETX), a company integrating computational power and biological data to discover life-transforming RNAi medicines, is pleased to provide the following business update.

2023 was a year of significant execution and progress in e-therapeutics’ mission of computing the future of medicine. ETX has moved rapidly to incorporate Large Language Models (LLMs) and transformer technology into its platforms, while continuing to deploy advanced analytics and AI. The Company now has multiple, successful case studies showcasing the power of AI within its drug discovery process combined with the speed and specificity of its GalOmic RNAi drug platform.

A key part of the Company’s strategy is to build a highly differentiated therapeutic pipeline, having exclusively nominated and prosecuted novel target genes not pursued by any other RNAi player. These targets were identified in-house, leveraging HepNet’s suite of proprietary datasets and advanced analytical functionality.

All nominated targets investigated to date have yielded positive results in preclinical studies. ETX believes that this outstanding hit rate is a result of the unique combination of AI-enhanced computational target selection and drug design coupled with siRNA, a well-tolerated and highly specific modality. The Company is confident that these datasets will be of significant interest to prospective partners and is actively exploring potential partnerships. Pipeline highlights include:

  • ETX-407 for the treatment of dry age-related macular degeneration (dAMD), a leading cause of blindness worldwide, has the potential to be a transformative, patient-friendly (no intravitreal injections required) therapeutic option with human genetic validation. ETX-407 demonstrates the applicability of ETX’s hepatocyte-targeting GalOmic platform to indications affecting distal organs. In dAMD, the ability to modulate disease pathology without direct injection into the eye and with infrequent administration (at least monthly) is a material differentiator from available treatments.
    Non-human primate data readouts for this target were produced in just 9 months after target nomination, demonstrating ETX’s ability to go rapidly from “idea” to in vivo results at a pace not achievable by other drug modalities.
  • ETX-312 has shown exceptional results in an industry-leading diet-induced obesity (DIO) model of NASH/MASH1. The model, developed by Danish company Gubra, is rated by experts as the most translatable murine model of NASH/MASH. ETX-312 was assessed both as monotherapy and as a combination treatment, including with GLP-1 receptor agonists. The disease modulation seen with ETX-312’s highly novel approach is considered extremely significant in the context of the approved and emerging NASH/MASH treatment landscape.
  • IND/CTA enabling studies for both ETX-407 and ETX-312 are to commence in due course.
  • Preclinical studies for ETX-148 as a treatment for haemophilia have been successfully completed. ETX-148 demonstrated protection from joint bleeds in mouse models of haemophilia with no thrombotic risk, thereby addressing a key unmet need in haemophilia of reducing haemarthrosis (joint bleeds) with a low treatment burden.
  • Preclinical studies of ETX-291 for the treatment of cardiometabolic disease have been successfully completed. In a representative disease model, ETX-291 impacted multiple cardiometabolic disease drivers resulting in a pleiotropic benefit and highlighting its potential to treat a broad range of cardiometabolic indications.
  • Continued progress on ETX-258 in an undisclosed indication.
  • Last near-term milestone successfully achieved in collaboration with iTeos in immuno-oncology, validating the Company’s computational approach to modelling human biology and identifying novel potential targets. This successful partnership with iTeos provides additional evidence of the power of ETX's computational approaches to drug discovery. The Company is also eligible to receive further undisclosed milestones.

The Company expects to end the year with a strong balance sheet with a cash position of c. £20.1 million (31 January 2023: £31.7 million), exceeding management’s expectations. This financial stability, having achieved the above milestones under historically harsh conditions with a cash reduction of only £11.5 million during the year, underscores the Company’s resilience and adeptness in financial and operational management.

1 Nonalcoholic steatohepatitis (NASH) is now known as metabolic dysfunction-associated steatohepatitis (MASH) following a recent change in disease nomenclature.


Ali Mortazavi, Chief Executive Officer of e-therapeutics, commented: “Against the backdrop of the harshest macroeconomic and sector conditions seen in decades, 2023 was a year of remarkable progress for e-therapeutics. We now have compelling evidence demonstrating the applicability of our computational/AI systems across our business. We have reproducibly and rapidly progressed multiple programs from novel target discovery to strong in vivo proof-of-concept, with an outstanding hit rate. This has all been achieved with a rigorous eye on our costs and maintaining a healthy balance sheet.

“Both ETX-407 and ETX-312 have the potential to be highly differentiated, first-on-target medicines in diseases with high unmet need and we are excited to be progressing these assets towards the clinic. ETX is now firmly established as a leader in the emergent TechBio sector, and we look forward to 2024 with great confidence.”


The information contained within this announcement is deemed by the Company to constitute inside information as stipulated under the Market Abuse Regulations (EU) No. 596/2014 ('MAR') which has been incorporated into UK law by the European Union (Withdrawal) Act 2018. Upon the publication of this announcement via Regulatory Information Service ('RIS'), this inside information is now considered to be in the public domain.