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e-therapeutics plc (ETX) listed on AIM

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ETX’s long term mission is to reshape the conventional drug discovery model with a powerful combination of computation, artificial intelligence, and the well-established RNA interference (RNAi) therapeutic modality. The aim is to translate digital discoveries into real-world, highly specific, and effective medicines in record time. The mission is nearer to being achieved with the successful incorporation of AI and machine learning models enabling the development of proprietary large-scale statistical approaches to continue reducing compute times.

ETX leverages computation and AI to transform the discovery and development of new medicines; building complex models of biological processes to identify novel targets, using machine learning to rapidly design and predict the efficacy of therapies, and integrating large language models across the entire drug development process. This allows ETX to accelerate the pace of discovery of life-transforming RNAi medicines across a variety of areas of high unmet medical need, including non-alcoholic steatohepatitis (NASH, liver), dry age-related macular degeneration (eye disease that impacts vision), cardiometabolic disease (heart), and haemophilia (bleeding disorder).


Key Points

· The strategy is to build a highly differentiated therapeutic pipeline of RNAi therapies with novel target genes. These targets lead to disease-modifying therapies which provide opportunities for increased revenue through partnering or rapid progression of assets towards clinical trials by ETX.

· ETX have developed the world’s most comprehensive knowledge resource in (liver data) hepatocyte-centric biology, (see appendix) creating a world class reference library. This enables the company to model complex biological processes within the liver, a highly influential organ that interacts with many tissues and organs throughout the body. ETX can therefore develop therapies for a wide variety of diseases using its liver-targeting approach. For example, ETX is progressing a therapy for dry age-related macular degeneration, a highly prevalent disease of the eye which causes blurred or reduced central vision. The global dry age-related macular degeneration market was valued at $1.6 billion in 2022.

· Another key focus is the cardiometabolic space which includes heart attack, stroke, angina (chest pain), insulin resistance, non-alcoholic fatty liver disease (NAFLD), and more. The global cardiometabolic drugs market size reached US$ 50 Bn in 2021. The sales of cardiometabolic drugs are estimated to surpass a valuation of US$ 70 Bn by 2032.

· ETX’s platform enables extremely fast drug development at a fraction of the conventional drug development cost.


Business Update

The recent business update reports progress with 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.

There is an in-house pipeline of life-transforming RNAi medicines, developed using both its HepNet™ computational platform and its GalOmic™ RNAi chemistry platform. This offers end-to-end capabilities for the rapid identification of novel disease associated genes and drug candidate development of therapies. This leads to medicines with disease-modifying potential which should then be rapidly progressed to the clinic and revenue. IP is being continually created with a further eight applications made over the last 15 months.

The collaboration with Arcturis Data will provide high quality, real-world clinically rich data to e-therapeutics’ computational biology platform. The collaboration will focus on delivering novel insights to support innovative therapies (drugs) for patients with metabolic dysfunction and associated fatty liver disease, including NASH. Under the terms of the agreement, e-therapeutics will have exclusive rights to nominate novel gene targets derived from the collaboration to developed drugs on its RNAi platform.

ETX has also recently achieved its final near-term milestone associated with a strategic collaboration with iTeos, a Nasdaq listed clinical-stage biotech company that designs and develops targeted immunotherapies for people living with cancer.

The revenue of £0.15m for the Interims to July 2023, is largely irrelevant at this R&D phase and relates mainly to the recognition of upfront payments and the achievement of milestones under the immuno-oncology collaboration agreement with iTeos. The operating loss of £7m included R&D of £5.3m implying a cash burn of £1.2m per-month. The net cash was £24.8 suggest all thing being equal its now around £17.6m and sufficient for over 12 months and towards the commercialisation phase.



· Expansion of artificial intelligence (AI) approaches that learn from experimental data deployed into drug design is increasing the chances of a significant discovery.

· Increasing intellectual property (IP) value with four new applications recently made to protect innovation around novel gene targets, and associated disease relevant biology as well as proprietary siRNA stabilisation.

· Scalability, the HepNet™ platform and data resources are entirely cloud-based, so there is effectively unlimited computational power and data storage.

· Non-dilutive funding opportunities via collaborations/partnerships remains a key component of the Company’s strategy with scope for early-stage revenue generation.

· It is well positioned to benefit from the change in regulators policy to use compelling computational data to help reduce preclinical timelines and start in human trials.



Investors do not need a PhD to appreciate that ETX are developing a paradigm shift in targeted drug discovery in high value markets. Its fully funded for around 12 months of AI assisted commercial development traction which could produce a jump in value.


Data Bank

List Aim

Price 18p (17.5p-18.5p) 52 week low 8p: high 24p

Mkt Cap £105m

Share in Issue 583.9m 3 Month Average Daily Volume 220,000 shares

Options 22m at 1.1p 2.6m at 22p 21m at 3.1p 11.4m at 0.5p Mosty Directors

Net Cash (Debt) Est £17.6m £24.6m at the Interims to July

Next Results Finals Jan Rns Early May-

Last Results Interims July 2023 Revenue for the six months to July was £0.2m but is irrelevant. Operating losses including £5.3m investment in R&D increased to £7m from £4.6m, although after deducting tax credits the reported loss is £5.6m.

Last Funding Sept 2022 Raised £13.5m at 20p

Directors Fees £445,000

IPO 2007 67p raised £1.3m by Wh Ireland

Shareholders Securities not in public hands: 55.69% Richard Griffiths and controlled undertakings 170,688,981 (29.24%) M&G – 101,825,000 (17.44%) Robert Quested – 51,800,000 (8.87%) Ali Mortazavi – 50,941,666 (8.73%) Trillian Ltd – 28,479,594 (4.88%) David Richardson – 27,178,003 (4.66%)



CEO AIi Mortazavi presents AI’s game-changing impact on drug discovery. Click below

Presentations and videos | e-therapeutics plc (

RNAi is a biological process that occurs naturally within our cells to help regulate gene expression. The mechanism by which RNAi mediates its biological function is specific targeting of messenger RNA (mRNA) molecules, which carry the instructions cells need to make proteins out of the genetic manual encoded in our DNA. The hepatocytes are the main functional cells of the liver. They are large and polygonal epithelial cells that constitute roughly up to 80% of the liver mass.

Cardiovascular diseases include heart attack, stroke, angina (chest pain) and other disorders of the vascular system. Cardiometabolic diseases include these cardiovascular conditions, plus others, such as insulin resistance, diabetes and non-alcoholic fatty liver disease. Cardiometabolic diseases tend to have a few risk factors in common.

Ali Mortazavi CEO since 2020, has extensive experience in the biotechnology sector and financial markets. His most recent roles include CEO of Silence Therapeutics plc, from 2012 to 2018, as well as a founder shareholder of Evolution Group, a UK-based investment bank, from 2001 to 2008.Ali is an experienced investor in small companies and has held numerous declarable stakes in listed/private biotechnology and technology companies.

Author Jon lev 29/01/2024