ORPH.L Regulatory News (ORPH)

28 July 2020

Open Orphan plc

("Open Orphan" or the "Company")

Awarded Contract from Codagenix Inc. New York USA for COVID-19 Vaccine Trial

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 clinical trials is pleased to announce a new contract with Codagenix Inc. ("Codagenix") to conduct a first in human (Phase I) trial evaluating safety and immunogenicity of a single-dose, intranasal SARS-CoV-2 (COVID-19) vaccine candidate in 48 healthy young adult volunteers.

Highlights:

· First in human (Phase I) study of CodaVax-COVID, a codon deoptimized SARS-CoV-2 (COVID-19) single-dose, intranasal vaccine candidate

· Vaccine being trialled at hVIVO's state-of-the-art Quarantine Facility in Whitechapel in London

· Phase I study to start in early Autumn with initial data expected by the end of 2020

· Study will include second dose as surrogate viral challenge at Day 28

hVIVO, part of Open Orphan, will conduct the COVID-19 vaccine trial for Codagenix at its state-of-the-art Quarantine Facility. CodaVax-COVID is a live-attenuated vaccine that replicates very slowly, but contains all SARS-CoV-2 proteins, unlike most other vaccines that only contain the viral spike. This vaccine has the potential to induce broad antibody, cellular and mucosal immunity with a single intranasal dose. hVIVO is currently kicking off the initial steps of this exciting study and are expected to start the trial in the quarantine unit this autumn.

This contract reinforces Open Orphan's position as the world leader in the testing of vaccines and antivirals.

Cathal Friel, Executive Chairman, Open Orphan, said:

"We are proud to be conducting this Phase I study for Codagenix, a leader in their field, as it will help bring a promising vaccine candidate to the public to help combat the COVID-19 pandemic as quickly as possible.

hVIVO, with its unique quarantine facility, is one of the few organisations in the world that has now publicly stated that it is working on testing COVID-19 vaccines for efficacy using human challenge model clinical trials.

Today's announcement also shows that hVIVO's quarantine facility is uniquely suited to conducting Phase I studies for infectious disease vaccines such as this.

Codagenix's expertise in viral design technology, combined with hVIVO's Phase I trial capability, gives us confidence that this trial will be successful and a step forward for the world as it seeks to develop a COVID-19 vaccine to bring the pandemic to a close."

Sybil Tasker, MD, MPH, Chief Medical Officer of Codagenix, said:

"We are very excited to be collaborating with hVIVO for the first-in-human study of CodaVax-COVID. hVIVO is world-recognized for expertise in evaluation of live viruses in human volunteers. Based on our animal data we expect this vaccine to be effective with a single dose, but will also evaluate a repeat dose to assess potential for boosting and as a model for protection from wildtype challenge.

We plan to move quickly from this initial demonstration of safety and immunogenicity in a controlled inpatient setting to larger Phase 2/3 program in conjunction with our partners at Serum Institute of India who are currently scaling up manufacturing to support rapid acceleration of our joint development program."

For further information please contact

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 ) 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.

Notes to Editors - Codagenix Inc.

Codagenix is a clinical-stage synthetic biology company that uses software to recode the genomes of viruses, constructing live-attenuated vaccines or viruses to prevent viral infections or treat solid tumours. Codagenix's recoded vaccine candidates are a perfect antigenic match to the target virus and induce a robust immune response to all viral antigens. For cancer, our nimble platform allows us to turn a virus into a potential oncolytic immuno-oncology therapy. Both programs rely on scalable, low-cost manufacturing. Codagenix Inc. spun out of the laboratory of National Academy of Science Member Eckard Wimmer at Stony Brook University in 2012 and is located at the Broad Hollow Bioscience Research Park in Farmingdale, N.Y. Our programs are supported by Adjuvant Capital, TopSpin Partners, Euclidean Capital, and government agencies such as the USDA, NIH/NIAID and the Department of Defense.

For more information, visit www.codagenix.com.