Mobilizing Stalled Assets from Big Pharma for Neglected Patient Groups

green,

ARTICLE

Mobilizing Stalled Assets from Big Pharma for Neglected Patient Groups

Author(s)
Pete Jackson, PhD
Pete Jackson, PhD
Executive Director of AMR Centre and Senior Advisor to FasterCures

As part of the BRIDGE initiative, FasterCures seeks to highlight novel partnerships involving deprioritized drugs that can serve as models for future transactions. This article discusses an innovative case study from the UK’s AMR Centre.

Non-cystic fibrosis bronchiectasis (NCFB) is a neglected, chronic condition that results in progressive respiratory decline following earlier lung damage, for example, from tuberculosis or viruses, such as COVID-19. The disease is characterized by the irreversible and permanent dilation of the airways, a chronic cough, overproduction of sputum, recurrent respiratory infections (typically with exacerbations 8-12 times a year), and chronic inflammation resulting in progressive lung damage. 

Of most concern, around half of NCFB sufferers develop chronic Pseudomonas aeruginosa (Pa) bacterial infections, which cause increased inflammation, higher hospitalization rates, higher health-care costs, and greater morbidity and mortality. 

There are an estimated 1.5 million people living with NCFB in the major markets around the world, with countless more in low- and middle-income countries, so new treatment options are desperately needed to address these long-term recurrent infections. As of today, the standard of care for Pa-colonised NCFB patients is long-term treatment with inhaled antibiotics. This provides a deadly opportunity for the development of antimicrobial resistance (AMR), where bacteria evolve to become highly resistant to the available antibiotic treatments.

The AMR Centre (AMRC), based in North-West England, was created to fulfill a key role in the translation of new treatments for critical-priority infections caused by bacteria and viruses. As a for-profit organization with public, private, and philanthropic shareholders that works alongside regional government agencies, universities, and the UK National Health Service (NHS), AMRC partners with early-stage drug developers with under-invested assets to fast-track exciting new drug technologies from the lab to patients in clinical trials.

In March 2017, I was invited by Shionogi, a Japanese global pharmaceutical company, to speak on an expert AMR panel at the Nikkei Asian Conference on Communicable Diseases in Okinawa. As a result of our conversations, Shionogi approached AMRC in November of that year with a project that had been placed on hold: a new anti-virulence antibody treatment, COT-143, that "de-activates" Pa and allows the body’s natural immune system to fight off infections more easily.  

Despite impressive pre-clinical data, there was no clear route through clinical trials for Shionogi’s preferred indication of acute ventilator-acquired pneumonia, so Shionogi sought a partner to advance this asset in other indications.

Working with our own expert scientists and specialist respiratory clinical advisors from the NHS, AMRC uncovered the potential of the product as a treatment for Pa infections in NCFB.

In particular, the drug is predicted to circulate for a long time in the bloodstream after dosing, meaning that a once-monthly injection could be used as a preventative strategy to reduce the number and severity of exacerbations.

With a new, agreed upon clinical development plan in place, Shionogi licensed the product to AMRC to fund and execute the plan up to the completion of Phase 2 clinical trials in NCFB patients.

In addition to securing funding from its investors, AMRC recently won a non-dilutive grant from the UK government to support the manufacture of the drug product to be used in the clinical trials.

Although AMRC has the primary responsibility for the program, it continues to work closely with Shionogi’s senior scientists with quarterly review meetings to monitor the progress against key developmental milestones.

An exciting part of the collaboration is the innovative approach to commercialization: once the critical Phase 2 proof-of-concept has been achieved, Shionogi has the option to license back the program from the AMRC to take forward to commercialization globally.

If AMRC is successful, this will mean additional capital to re-invest in new programs and provide a return for its public, private, and philanthropic shareholders.

But more importantly, AMRC will have demonstrated the potential of an exciting new product to significantly improve the lives of NCFB patients suffering from long-term recurring and debilitating infections.

Partnerships such as this can serve as a model for other organizations that seek to pave a path for the continued development of deprioritized drugs. When companies make their deprioritized drugs available for study by other entities—as did Shionogi—patients benefit. Through the BRIDGE initiative, FasterCures is exploring mechanisms that could facilitate the placement of good science into good homes. The potential of all promising drugs should be investigated regardless of their commercial prospects. 

Published September 9, 2020