Clinical safety and efficacy of novel antifungal, fosmanogepix, for the treatment of candidaemia: results from a Phase 2 trial.


Journal

The Journal of antimicrobial chemotherapy
ISSN: 1460-2091
Titre abrégé: J Antimicrob Chemother
Pays: England
ID NLM: 7513617

Informations de publication

Date de publication:
03 10 2023
Historique:
received: 06 04 2023
accepted: 28 07 2023
medline: 4 10 2023
pubmed: 19 8 2023
entrez: 19 8 2023
Statut: ppublish

Résumé

Fosmanogepix is a first-in-class antifungal targeting the fungal enzyme Gwt1, with broad-spectrum activity against yeasts and moulds, including multidrug-resistant fungi, formulated for intravenous (IV) and oral administration. This global, multicenter, non-comparative study evaluated the safety and efficacy of fosmanogepix for first-line treatment of candidaemia in non-neutropenic adults. Participants with candidaemia, defined as a positive blood culture for Candida spp. within 96 h prior to study entry, with ≤2 days of prior systemic antifungals, were eligible. Participants received fosmanogepix for 14 days: 1000 mg IV twice daily on Day 1, followed by maintenance 600 mg IV once daily, and optional switch to 700 mg orally once daily from Day 4. Eligible participants who received at least one dose of fosmanogepix and had confirmed diagnosis of candidaemia (<96 h of treatment start) composed the modified intent-to-treat (mITT) population. Primary efficacy endpoint was treatment success at the end of study treatment (EOST) as determined by the Data Review Committee. Success was defined as clearance of Candida from blood cultures with no additional antifungal treatment and survival at the EOST. Treatment success was 80% (16/20, mITT; EOST) and Day 30 survival was 85% (17/20; 3 deaths unrelated to fosmanogepix). Ten of 21 (48%) were switched to oral fosmanogepix. Fosmanogepix was well tolerated with no treatment-related serious adverse events/discontinuations. Fosmanogepix had potent in vitro activity against baseline isolates of Candida spp. (MICrange: CLSI, 0.002-0.03 mg/L). Results from this single-arm Phase 2 trial suggest that fosmanogepix may be a safe, well-tolerated, and efficacious treatment for non-neutropenic patients with candidaemia, including those with renal impairment.

Sections du résumé

BACKGROUND
Fosmanogepix is a first-in-class antifungal targeting the fungal enzyme Gwt1, with broad-spectrum activity against yeasts and moulds, including multidrug-resistant fungi, formulated for intravenous (IV) and oral administration.
METHODS
This global, multicenter, non-comparative study evaluated the safety and efficacy of fosmanogepix for first-line treatment of candidaemia in non-neutropenic adults. Participants with candidaemia, defined as a positive blood culture for Candida spp. within 96 h prior to study entry, with ≤2 days of prior systemic antifungals, were eligible. Participants received fosmanogepix for 14 days: 1000 mg IV twice daily on Day 1, followed by maintenance 600 mg IV once daily, and optional switch to 700 mg orally once daily from Day 4. Eligible participants who received at least one dose of fosmanogepix and had confirmed diagnosis of candidaemia (<96 h of treatment start) composed the modified intent-to-treat (mITT) population. Primary efficacy endpoint was treatment success at the end of study treatment (EOST) as determined by the Data Review Committee. Success was defined as clearance of Candida from blood cultures with no additional antifungal treatment and survival at the EOST.
RESULTS
Treatment success was 80% (16/20, mITT; EOST) and Day 30 survival was 85% (17/20; 3 deaths unrelated to fosmanogepix). Ten of 21 (48%) were switched to oral fosmanogepix. Fosmanogepix was well tolerated with no treatment-related serious adverse events/discontinuations. Fosmanogepix had potent in vitro activity against baseline isolates of Candida spp. (MICrange: CLSI, 0.002-0.03 mg/L).
CONCLUSIONS
Results from this single-arm Phase 2 trial suggest that fosmanogepix may be a safe, well-tolerated, and efficacious treatment for non-neutropenic patients with candidaemia, including those with renal impairment.

Identifiants

pubmed: 37596890
pii: 7246201
doi: 10.1093/jac/dkad256
pmc: PMC10545531
doi:

Substances chimiques

Antifungal Agents 0

Types de publication

Clinical Trial, Phase II Multicenter Study Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

2471-2480

Informations de copyright

© The Author(s) 2023. Published by Oxford University Press on behalf of British Society for Antimicrobial Chemotherapy.

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Auteurs

Peter G Pappas (PG)

Division of Infectious Diseases, Department of Internal Medicine, University of Alabama at Birmingham, Birmingham, AL, USA.

Jose A Vazquez (JA)

Division of Infectious Disease, Department of Medicine, Medical College of Georgia/Augusta University, Augusta, GA, USA.

Ilana Oren (I)

Infectious Disease Unit, Rambam Health Care Campus, Haifa, Israel.

Galia Rahav (G)

Sheba Medical Center, Ramat Gan, Israel.
Chaim Sheba Medical Center, Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel.

Mickael Aoun (M)

Department of Internal Medicine, Institut Jules Bordet, Brussels, Belgium.

Pierre Bulpa (P)

Intensive Care Medicine, University Hospital Mont-Godinne, CHU UCL Namur, Yvoir, Belgium.

Ronen Ben-Ami (R)

Chaim Sheba Medical Center, Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel.
Tel Aviv Sourasky Medical Center, Tel Aviv, Israel.

Ricard Ferrer (R)

Vall d'Hebron Hospital Universitari, Shock, Organ Dysfunction, and Resuscitation (SODIR) Research Group, Vall d'Hebron Institut de Recerca (VHIR), Vall d´Hebron Barcelona Hospital Campus, Passeig de la Vall d'Hebron, Barcelona, Spain.

Todd Mccarty (T)

Division of Infectious Diseases, Department of Internal Medicine, University of Alabama at Birmingham, Birmingham, AL, USA.

George R Thompson (GR)

Division of Infectious Diseases, Department of Internal Medicine, and Department of Medical Microbiology and Immunology, University of California Davis, Sacramento, CA, USA.

Haran Schlamm (H)

Amplyx Pharmaceuticals Inc., San Diego, CA, USA.

Paul A Bien (PA)

Pfizer, Inc., Groton, CT, USA.

Sara H Barbat (SH)

Amplyx Pharmaceuticals Inc., San Diego, CA, USA.

Pamela Wedel (P)

Amplyx Pharmaceuticals Inc., San Diego, CA, USA.

Iwona Oborska (I)

Amplyx Pharmaceuticals Inc., San Diego, CA, USA.

Michael R Hodges (MR)

Amplyx Pharmaceuticals Inc., San Diego, CA, USA.

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