A novel PLpro inhibitor improves outcomes in a pre-clinical model of long COVID

S Stefanie M. Bader D Dale J. Calleja S Shane M. Devine N Nathan W. Kuchel B Bernadine G. C. Lu X Xinyu Wu R Richard W. Birkinshaw R Reet Bhandari K Katie Loi R Rohan Volpe Y Yelena Khakham A Amanda E. Au T Timothy R. Blackmore L Liana Mackiewicz M Merle Dayton J Jan Schaefer L Lena Scherer A Angus T. Stock J James P. Cooney K Kael Schoffer A Ana Maluenda E Elizabeth A. Kleeman K Kathryn C. Davidson C Cody C. Allison G Gregor Ebert G Gong Chen (State Key Laboratory and Institute of Elemento-Organic Chemistry, College of Chemistry) K Kasiram Katneni T Theresa A. Klemm U Ueli Nachbur S Smitha Rose Georgy P Peter E. Czabotar A Anthony J. Hannan T Tracy L. Putoczki M Maria Tanzer M Marc Pellegrini B Bernhard C. Lechtenberg S Susan A. Charman M Melissa J. Call J Jeffrey P. Mitchell K Kym N. Lowes G Guillaume Lessene M Marcel Doerflinger D David Komander

Abstract

Abstract The COVID-19 pandemic caused by the coronavirus SARS-CoV-2 has highlighted the vulnerability of a globally connected population to zoonotic viruses. The FDA-approved coronavirus antiviral Paxlovid targets the essential SARS-CoV-2 main protease, Mpro. Whilst effective in the acute phase of a COVID infection, Paxlovid cannot be used by all patients, can lead to viral recurrence, and does not protect against post-acute sequelae of COVID-19 (PASC), commonly known as long COVID, an emerging significant health burden that remains poorly understood and untreated. Alternative antivirals that are addressing broader patient needs are urgently required. We here report our drug discovery efforts to target PLpro, a further essential coronaviral protease, for which we report a novel chemical scaffold that targets SARS-CoV-2 PLpro with low nanomolar activity, and which exhibits activity against PLpro of other pathogenic coronaviruses. Our lead compound shows excellent in vivo efficacy in a mouse model of severe acute disease. Importantly, our mouse model recapitulates long-term pathologies matching closely those seen in PASC patients. Our lead compound offers protection against a range of PASC symptoms in this model, prevents lung pathology and reduces brain dysfunction. This provides proof-of-principle that PLpro inhibition may have clinical relevance for PASC prevention and treatment going forward.

Article Details

Volume / Issue Vol. 16, Issue 1
Published April 03, 2025
ISSN 2041-1723
Publisher Nature Portfolio

Journal Info

Nature Communications

Nature Portfolio

ISSN: 2041-1723 Open Access Life Sciences

Authors (43)

S

Stefanie M. Bader

D

Dale J. Calleja

S

Shane M. Devine

N

Nathan W. Kuchel

B

Bernadine G. C. Lu

X

Xinyu Wu

R

Richard W. Birkinshaw

R

Reet Bhandari

K

Katie Loi

R

Rohan Volpe

Y

Yelena Khakham

A

Amanda E. Au

T

Timothy R. Blackmore

L

Liana Mackiewicz

M

Merle Dayton

J

Jan Schaefer

L

Lena Scherer

A

Angus T. Stock

J

James P. Cooney

K

Kael Schoffer

A

Ana Maluenda

E

Elizabeth A. Kleeman

K

Kathryn C. Davidson

C

Cody C. Allison

G

Gregor Ebert

G

Gong Chen

State Key Laboratory and Institute of Elemento-Organic Chemistry, College of Chemistry

K

Kasiram Katneni

T

Theresa A. Klemm

U

Ueli Nachbur

S

Smitha Rose Georgy

P

Peter E. Czabotar

A

Anthony J. Hannan

T

Tracy L. Putoczki

M

Maria Tanzer

M

Marc Pellegrini

B

Bernhard C. Lechtenberg

S

Susan A. Charman

M

Melissa J. Call

J

Jeffrey P. Mitchell

K

Kym N. Lowes

G

Guillaume Lessene

M

Marcel Doerflinger

D

David Komander