Successful completion of toxicology study

REACH

23 December 2020

Open Orphan plc

("Open Orphan" or the "Company")

Successful completion of PrEP Biopharm Toxicology study for respiratory 'viral mimic'

Phase IIb study to validate efficacy of PrEP-001 against

circulating respiratory viruses such as COVID-19, flu and the common cold

Open Orphan plc (AIM: ORPH), a rapidly growing specialist pharmaceutical services CRO which is the world leader in the testing of vaccines and antivirals using human challenge clinical trials, announces that drug development company PrEP Biopharm Limited ("PrEP Biopharm"), which is 62.6% owned by Open Orphan, has successfully completed a 12-week toxicology study for its novel pan-viral prophylactic asset, PrEP-001.

The animal model study provides safety data needed to move PrEP-001 into longer duration dosing in clinical studies. PrEP Biopharm intends to move forward with a real-world, field trial intended to validate the efficacy of its PrEP-001 against all circulating respiratory viruses, including COVID-19, influenza and the common cold.

PrEP-001 is neither a vaccine nor an antiviral. Rather, it is a synthetic RNA 'viral mimic' that stimulates the body's pan-viral innate immune response locally in the upper respiratory tract. Administered via a once daily nasal spray it leverages the innate immune system to create an antiviral environment to stop invading respiratory viruses. As such, it is possible that PrEP-001 could be used as a stand-alone pan-viral prophylaxis or as an adjuvant for live and inactivated or peptide intranasal vaccines targeting specific respiratory viruses. Previous studies in animals have demonstrated that intranasal administration of poly IC shortly after vaccination with live attenuated, inactivated and haemagglutinin antigen preparations of influenza virus enhanced respiratory dendritic cell function, CD8 T cell formation, and production of neutralising antibodies.

When Open Orphan acquired hVIVO in January 2020, in addition to acquiring the world leader in the testing of vaccines and antivirals using human challenge studies, hVIVO also came with a number of non-core assets including a 62.6% stake in PrEP Biopharm. The previous hVIVO Board had used shareholders' funds to invest in these non-core assets. The Open Orphan Board and management team are primarily focussed upon cementing its position as a unique CRO and is committed to directing all resources to further improving the quality of earnings in both Venn Life Sciences and hVIVO and with shareholder funds only being used to support the profitable CRO services business. In recent months, Open Orphan has made it clear that it intends to monetise these non-core assets, however, as part of monetising these non-core assets, Open Orphan does not intend to invest any of the Open Orphan shareholders' funds but will continue to work with the PrEP Biopharm management team to secure other sources of funding outside of the Open Orphan group while maximising the potential of these non-core assets.

Cathal Friel, Executive Chairman of Open Orphan, said: "It is very encouraging to note PrEP Biopharm's progress towards the ultimate goal of validating the effectiveness of their one dosage nasal spray for key respiratory viruses like COVID-19, flu and the common cold. This safety data allows them to move into Phase 2b field studies and we are very excited that PrEP-001 could play a meaningful role in the current COVID-19 pandemic and future potential pandemics caused by new respiratory viruses." 

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About Open Orphan (www.openorphan.com)

Open Orphan is a rapidly growing niche CRO pharmaceutical services company which is a world leader in the testing of vaccines and antivirals through the use of human challenge clinical trials. Conducted from Europe's only 24-bedroom quarantine clinic with onsite virology providing individually isolated rooms and connected to our specialist laboratory facility. hVIVO's challenge studies require healthy volunteers to take part, volunteers are recruited through FluCamp, learn more at www.FluCamp.com. The hVIVO facility offers highly specialised virology and immunology laboratory services to support pre-clinical and clinical respiratory drug, antiviral, and vaccine discovery and development. Reliable laboratory analysis underpinned by scientific expertise is essential when processing and analysing clinical samples. Robust quality processes support our team of scientists in the delivery of submission ready data.

The Company has a leading portfolio of 8 viral challenge study models which are: 2 FLU, 2 RSV, 1 HRV, 1 Asthma, 1 cough and 1 COPD viral challenge models. As announced in early March, Open Orphan is rapidly advancing a number of Coronavirus challenge study models and expects to be helping many COVID-19 vaccine development companies to test their vaccines. No other company in the world has such a portfolio, with only two competitors globally having 1 challenge study model each. hVIVO also works with companies in the UK and Ireland to provide COVID-19 testing to staff to protect staff and customers from a workplace COVID-19 outbreak through its COVID Clear offering. 

Open Orphan comprises of two commercial specialist CRO services businesses, hVIVO  and  Venn Life Sciences and is also building out a valuable data platform business. hVIVO has built up one of the world's largest databases of infectious disease progression data and we are populating our Open Orphan Health Data platform with this historical hVIVO data. In our clinical trials going forward, we are also planning to collect data on volunteer's via wearables during clinical trials. Therefore, Open Orphan's data, which may yield valuable digital biomarkers, could be one of the more sought-after datasets by many of the large wearables /smart watch wearables providers around the world. In June 2019, Open Orphan acquired AIM-listed Venn Life Sciences Holdings plc in a reverse take-over and in January 2020 it completed the merger with hVIVO plc in January 2020. Venn is an integrated drug development consultancy firm which offers CMC (chemistry, manufacturing and controls), preclinical, Phase I & II clinical trials design and execution. The merger with hVIVO created a European full pharma services company broadening the Company's customer base and with complementary specialist CRO services, widened the range of the Company's service offerings.

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