Poolbeg Pharma Plc

Artificial intelligence programme yields multiple novel RSV drug targets

First time AI has been used to identify disease targets in RSV

8 November 2022 - Poolbeg Pharma (AIM: POLB, OTCQB: POLBF, 'Poolbeg' or the 'Company'), a clinical stage infectious disease pharmaceutical company with a unique capital light clinical model, announces that it has made a significant breakthrough in its Artificial Intelligence ('AI') Programme with partner OneThree Biotech, Inc. through the discovery of novel drug targets for the treatment of Respiratory Syncytial Virus ('RSV').

Following the completion of the build and optimisation of a tailored AI model in June 2022, the OneThree team, using its clinically validated AI platform, have identified novel drug targets using Poolbeg's unique RSV human challenge trial data. The identification of these novel drug targets now allows the final stage of the programme to commence which will involve the identification of small molecule inhibitors to effectively treat RSV infections. The team are particularly excited as these include a range of targets for which there are known drugs with existing Phase I safety and tolerability data. Prioritisation of compounds with existing Phase I data aligns with Poolbeg's capital light model as these compounds will have extensive nonclinical and clinical data allowing smooth transition to early human efficacy trials.

The final stage of the research plan will utilise OneThree's proprietary ATLANTIS AI platform to identify the drugs that are most likely to be efficacious against the identified disease targets with clean safety profiles in RSV patients. Selection of drugs is traditionally a long and extensive process, however, by using an existing AI model this process can be completed in weeks. Results of this stage are expected before year end 2022.

Traditional drug design is focused on identifying a molecule that can bind with a specific target within a cell. Poolbeg's approach, utilising OneThree Biotech's advanced AI models, allows for the complex disease signature underlying each drug target to be considered during drug selection. This allows the complex outputs of this stage, which represent novel biological insights, to be advanced to the final stage which will match drugs to prioritised targets. By working from disease signatures like this, OneThree Biotech can utilise the full strength of its AI technology to pinpoint the drug molecules that can precisely and effectively reverse disease signatures and produce meaningful clinical benefits.

Jeremy Skillington, PhD, CEO of Poolbeg Pharma, said:

"This is the first time that AI has been used to identify disease targets in RSV and I am delighted to have reached this significant milestone in the programme in such a short period of time. Having identified these novel disease targets, we can now commence the final stage of the programme to identify effective drug candidates to treat RSV. In keeping with our capital-light model, this AI enabled in silico R&D approach has significantly accelerated the discovery process by having the right partners and the right expertise to unleash the potential of our unique human challenge trial data. We greatly look forward to seeing the outputs of this programme later this year."

Neel S. Madhukar, PhD, CEO of OneThree Biotech said:

"This analysis is pioneering in RSV research, and we are pleased to have identified disease targets from which effective drug candidates can be explored. We are fortunate to have access to Poolbeg's unique human challenge trial data because it was collected in a controlled and powered quarantine which increases the overall strength and predictive power you can get from it. Additionally, thanks to the combination of Poolbeg's data and our ATLANTIS platform, we were able to reach this point in our project in only eight months - significantly faster than a traditional drug discovery project - and we're excited to make meaningful progress in the treatment of infectious diseases, such as RSV."

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Enquiries

About RSV

RSV is a contagious virus that affects people's respiratory tract with children and at-risk older adults being particularly vulnerable; in severe cases, it can cause pneumonia and other life-threatening breathing difficulties. RSV is a significant public health threat affecting an estimated 50 million people annually, leading to 4 million global hospitalisations and an estimated 100,000 deaths in children under the age of 5 years. An estimated 45% of these cases and deaths occur in new-born infants under the age of 6 months. Studies have suggested that treatment for RSV in children under five alone costs governments globally €4.8bn every year.

About Poolbeg's AI Programmes

Poolbeg Pharma has access to a unique private repository of clinical samples and associated data from human challenge trials via its relationship with Open Orphan plc, a resource which is anticipated to expand over the coming years. Human challenge trial data is unique in the depth of longitudinal virology, health, biomarker and symptom data collected during the course of disease. These datasets provide clinical insights into disease that guides Poolbeg Pharma's product acquisitions and clinical development.

The Company is currently progressing two AI Programmes, the first in RSV in partnership with OneThree Biotech, and the second in influenza in partnership with CytoReason. Poolbeg believe that this is the first time that AI analysis has been undertaken on RSV and influenza human challenge trial data and samples to identify new drug targets and treatments. The unique nature of human challenge trials to produce disease progression data with high precision is revolutionising the insights into human disease.

Poolbeg's asset POLB 001, which has commenced its LPS human challenge trial with initial trial data expected before year end, was identified using such disease progression data. However, by utilising AI, the Company aims to identify more targets quicker and more cost efficiently than previously possible without this technology.

About Poolbeg Pharma

Poolbeg Pharma is a clinical stage infectious disease pharmaceutical company, with a unique capital light clinical model which aims to develop multiple products faster and more cost effectively than the conventional biotech model. The Company, headquartered in London, is led by a team with a track record of creation and delivery of shareholder value and aspires to become a "one-stop shop" for pharma and biotech seeking mid-stage products to license or acquire.

The Company is targeting the growing infectious disease market. In the wake of the COVID-19 pandemic, infectious disease has become one of the fastest growing pharma markets and is expected to exceed $250bn by 2025.

With its initial assets from hVIVO plc (formerly named Open Orphan plc), an industry leading infectious disease and human challenge trials business, Poolbeg has access to knowledge, experience, and clinical data from over 20 years of human challenge trials. The Company is using these insights to acquire new assets as well as reposition clinical stage products, reducing spend and risk. Amongst its portfolio of exciting assets, Poolbeg has a small molecule immunomodulator for severe influenza (POLB 001) which has commenced its LPS human challenge trial with initial results expected by year end 2022; a first-in-class, intranasally administered RNA-based immunotherapy for respiratory virus infections (POLB 002); and a vaccine candidate for Melioidosis (POLB 003). The Company is also developing an oral vaccine delivery platform and is progressing two artificial intelligence (AI) programmes to accelerate the power of its human challenge model data and biobank, with results from the first programme expected by year end 2022.

For more information, please go to www.poolbegpharma.com or follow us on Twitter and LinkedIn @PoolbegPharma.

About OneThree Biotech

OneThree Biotech is a private, clinically validated, biology-driven artificial intelligence (AI) company focused on using its proprietary ATLANTIS AI platform in combination with systems biology to understand the mechanisms that drive drug-target-disease relationships. This approach has been used not only to generate a pipeline of first-in-class programs, but also in partnerships with other biopharmaceutical companies. OneThree Biotech's proven technology platform integrates chemical, biological, and clinical data with cutting-edge computational tools to answer complex questions surrounding disease biology and drug discovery. The company's AI platform has shown success in identifying new targets for the treatment of various cancers and optimizing early-stage drug discovery and development by quickly and accurately generating new, testable insights and hypotheses. The OneThree platform has been validated across early-stage drug discovery through multiple partnerships, clinical trials, and more than 20 peer-reviewed publications. OneThree Biotech is based in New York City.

Twitter: @onethreebiotech