ORPH.L Regulatory News (ORPH)

RNS Reach

15 June 2020

Open Orphan plc

("Open Orphan" or the "Company")

Positive Phase I study results published in The Lancet

Positive results from the Phase I study of the AGS-v vaccine developed by an Open Orphan joint venture, Imutex Limited, have been published in the peer reviewed journal The Lancet

AGS-v is a first-in-class mosquito saliva, standalone, vaccine candidate designed to protect against mosquito-borne diseases carried in the saliva such as Zika, Malaria, Dengue Fever and West Nile Virus

Open Orphan plc (ORPH), a rapidly growing specialist CRO pharmaceutical services company which is the world leader in the testing of vaccines and antivirals using human challenge study models is pleased to announce that the results of a randomised, placebo-controlled, double-blind, phase 1 trial of AGS-v have been published in The Lancet. AGS-v has been developed by Imutex Limited, in which Open Orphan owns a 49% stake in conjunction with the SEEK Group.

Highlights:

· The first trial of AGS-v, a first-in-class "mosquito saliva vaccine" in humans

· The trial indicated that the vaccine is safe and induces a strong immune response in healthy volunteers

· These positive findings suggest that AGS-v is now ready to advance to Phase II

Numerous studies have shown that protection from diseases carried in saliva is possible if you alter the immune response to saliva. Using natural saliva to achieve this not viable. AGS-v is designed as a transformational vaccine, the first ever mosquito synthetic saliva vaccine designed in to protect against mosquito-borne diseases carried in the saliva such as Zika, Malaria, Dengue Fever, West Nile Virus etc. AGS-v is designed to provoke an immune response against mosquito saliva, rather than any specific parasites, viruses, or bacteria the mosquito might transmit making it an important tool in the fight against mosquito-borne diseases.

In the new study published in The Lancet, NIAID scientists describe the results of the Phase 1 trial of the vaccine as "encouraging and worthy of further study".

The Trial Results

The double-blind study, which began in 2017 at the NIH Clinical Center in Bethesda, Maryland, was the first trial of this so-called "mosquito saliva vaccine" in humans.

The volunteers' blood tests showed that the vaccine in combination with the adjuvant produced a significant immune response to mosquito salivary peptides. Further, this immune response was not accompanied by a worse reaction to mosquito bites.

The study's results are promising and suggest that further research to test the vaccine's efficacy against individual pathogens, followed by larger field studies, would be worthwhile. A widely available "universal" vaccine could provide protection against emerging and re-emerging mosquito-borne diseases as they arise, allowing public health officials to quickly respond to new outbreaks and epidemics without waiting for new treatments or vaccines to be developed.

Cathal Friel, Executive Chairman of Open Orphan, said:

"We are delighted with the results of this very important and successful trial of Imutex's universal vaccine for mosquito-borne diseases. This further confirms and reinforces our belief that our 49% shareholding in Imutex has a lot of unrealised potential value and we look forward to working closely with Gregory Stoloff and his team in SEEK to see how we can commercialise and monetise the true value of Imutex over the coming months."

Gregory Stoloff, Chief Executive Office, Imutex Ltd, said:

"We all know too well today the cost of pandemics to society and mosquitoes have been causing these problems for many countries for too long. It has been known for some time that injecting saliva in a particular way from an insect provides protection from the disease carried in that saliva. Harvesting natural saliva however, was not practical. We are very excited that our synthetic made saliva produced an immune response and was safe in humans. This makes a vaccine that can protect people against so many diseases that plague the world, a step closer to reality. We are excited that we now are a step closer to making a vaccine that could deal with these issues."

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Notes to Editors - Open Orphan:

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. Which 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.  In June 2020 hVIVO COVID Clear Test was launched, the most accurate antibody test available to UK employers, helping them to get their people back to work.

Open Orphan comprises of two commercial specialist CRO services businesses, hVIVO and Venn Life Sciences, and is developing an early stage orphan drug genomics data platform business. Open Orphan comprises of two commercial specialist CRO services businesses (hVIVO and Venn) and is developing an early stage orphan drug genomics data platform business. This platform captures valuable genetic data from patient populations with specific diseases with designated orphan drug status and incorporating AI tools. 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. Venn, as an integrated drug development consultancy, 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.