Press Release

Open Orphan leads weighty Wellcome Trust bid to define human challenge drug standards

By Mark Sheridan

Published:

Disseminated on behalf of Open Orphan Plc

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Open Orphan (LSE:ORPH)

Open Orphan subsidiary hVIVO has been selected to lead a highly-respected group to create international standards for drugs used in human challenge trials. 

The Dublin-headquartered contract research firm will lead a consortium to generate regulatory guidelines to support the safe manufacture and storage of human challenge agents. 

The work will develop and extend existing World Health Organisation rules. 

Announcing the partnership Open Orphan executive chairman Cathal Friel said:

We are proud to have been selected to lead the consortium on this important and much-needed guideline. hVIVO is at the forefront of human challenge studies and has world-leading capabilities. This collaboration is recognition of the work we do in the scientific community.”

Wellcome Trust is an historic London research charity with a £25.9bn endowment, making it the fourth-wealthiest charitable foundation in the world. It provides grants of up to £5m to research teams developing faster vaccine trials using human challenge studies in low to middle income countries. 

Challenging vaccine expectations

By infecting healthy volunteers with an extremely low dose of a disease, human challenge studies can rapidly accelerate vaccines for use in the general population. They are also designed to compare the safety and effectiveness of multiple vaccine candidates. 

So even as the first wave of potential Covid-19 vaccines from Pfizer, Moderna, Astrazeneca/Oxford University and Johnson & Johnson come through, testing to determine how the world’s governments will access vaccines is supremely necessary. 

There are currently over 300 candidate vaccines in development to treat Covid-19. Scientists will not be able to conduct large-scale phase 3 studies for all of them. Challenge studies can prioritise the best drug candidates and prove efficacy in cases where transmission is low. 

Public support for human challenge studies is also extremely strong. In the current pandemic, over 40,000 people in the UK have signed up to participate in Covid-19 human challenge trials. 

Conventional vaccine field trials are very slow because researchers have to wait for participants to get infected in the real world before they are treated with a drug candidate.

Phase 2 to Phase 3 trials typically take years to complete, compared to human challenge studies which can provide results within months. Human challenge studies could bring a much faster end to the Covid-19 pandemic, experts argue. 

Opening up 

The first human challenge study started in the UK in the 18th century and scientists have been using the model widely since the Second World War.

Open Orphan won a £40m contract with the British government in October to develop the UK’s first human challenge study models for Covid-19. As well as using Europe’s only 24-bed quarantine unit for the trials, the UK government has donated the use of the Royal Free Hospital, which hosts the UK’s only high-level isolation unit for the treatment of infectious disease. 

The deal will see hVIVO create the challenge virus for use on patients and the first-in-human characterisation study. After hVIVO scientists select an appropriate strain and dose of the virus, vaccinated volunteers are intentionally infected, bringing forward results much faster than in conventional trials. Since the beginning of the 2020 Open Orphan’s market cap has grown from £36m to £165m.

Nobel backing

In October, 15 Nobel Prize winners were among the scientific experts who signed an open letter to the UK government backing human challenge studies.  

Writing for the British Medical Journal, experts at non-profit volunteer advocacy group 1DaySooner suggested human challenge trials “may prove essential for second and third generation vaccines since rich countries are likely to outbid poorer ones for the first batch of vaccines.”

Vaccines for 15 major pathogens have been fast-tracked in the last two decades using human challenge studies, including cholera, typhoid and Type-A influenza. Approximately 30,000 participants have taken part showing no serious adverse effects. 

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