REG - e-Therapeutics plc - Business Update

E-TherapeuticsAnnouncement

16/01/2024 07:00

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RNS Number : 7670Z  e-Therapeutics plc  16 January 2024

 

e-therapeutics plc

("e-therapeutics" or "ETX" or the "Company")

 

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/MASH(1). 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.

 

Enquiries

 e-therapeutics plc
 Ali Mortazavi,                                                                        Tel: +44 (0)1993 883 125
 CEO

                                                                                     www.etherapeutics.co.uk
 Timothy Bretherton, CFO

      SP Angel Corporate Finance LLP                                                   Tel: +44(0)20 3470 0470
 Nominated Adviser and Broker
 Matthew Johnson/Caroline Rowe (Corporate Finance)
 Vadim Alexandre/Rob Rees (Corporate Broking)

 

 

About e-therapeutics plc

 

e-therapeutics plc ("ETX") integrates computational power and biology
information to discover life-transforming RNAi medicines. The Company's
technology uses computation to capture and model human biology, identify novel
targets, and develop RNAi medicines against those targets that can be rapidly
progressed to the clinic.

 

ETX's proprietary HepNet™ platform enables the generation and analysis of
biological network models, providing a novel and mechanistic approach to drug
discovery. This approach explicitly considers the true complexity of biology
to make more reliable predictions from large complex data sets and ETX's
proprietary hepatocyte knowledgebase - the world's most comprehensive and
integrated hepatocyte-centric data resource.  The Company generates,
prioritises, and tests millions of hypotheses in silico to identify better
therapeutic targets with higher confidence.

 

GalOmic™, ETX's proprietary RNAi platform, enables targeted delivery to
hepatocytes in the liver and the specific silencing of novel
disease-associated genes, identified by HepNet™. The focus on hepatocytes
offers the opportunity to tackle a wide variety of diseases.  The liver is a
highly metabolically active organ which performs a key role in many biological
processes and vital functions crucial for human health. ETX's GalOmic™
constructs have demonstrated compelling in vivo performance in terms of depth
of gene silencing and duration of action.

 

The Company is progressing a pipeline of first-in-class RNAi candidates across
a variety of therapeutic areas with high unmet need, including preclinical
programs in cardiometabolic and metabolic diseases, haemophilia, and other
undisclosed indications. ETX has also partnered with biopharma companies such
as Novo Nordisk, Galapagos NV and iTeos Therapeutics using its computational
network biology approach across a diverse range of drug discovery projects.

 

The Company is based in London, UK and listed on the Alternative Investment
Market of the London Stock Exchange ("AIM"), with ticker symbol ETX.

 

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