18 and older, any sex, with Candidemia or Invasive Candidiases. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage of Participants With Treatment Success at End of Study Treatment (EOST) as Determined by Data Review Committee (DRC)Primary· EOST: any day from Day 1 up to maximum of Day 42
Treatment success was defined as meeting all of the following criteria:
1\) Two consecutive blood cultures negative for Candida species, and/or for participants with a deep-seated site of infection, at least 1 negative tissue culture or aspirate/fluid culture. For participants with a deep-seated site of infection involving visceral organs from which a tissue culture was not obtainable, resolution of the attributable clinical signs of infection recorded at Baseline, and as applicable, radiological improvement associated with the site of infection. 2) Alive at EOST and 3) No concomitant use of
Group
Value
95% CI
APX001 (Fosmanogepix)
88.9
51.8 – 99.7
Time to First Negative Blood CultureSecondary· Day 1 up to maximum of Day 42
Time to first negative blood culture was defined as the number of days from date of first dose of study drug to the date of first negative blood culture plus 1. Participants without a negative blood culture at post-baseline visits were censored at the last assessment date. Kaplan-Meier method was used for analysis.
Group
Value
95% CI
APX001 (Fosmanogepix)
6
5.0 – 15.0
Percentage of Participants With Mycological Outcomes at EOST and 2 and 4 Weeks After EOSTSecondary· EOST: any day from Day 1 up to maximum of Day 42, 2 and 4 weeks after EOST
Mycological outcomes were determined based on eradication and presumed eradication. Eradication was defined as a negative blood (and/or other infection site) culture(s) for Candida species. Presumed eradication (applicable to invasive candidiasis) was defined as clinical resolution of invasive Candida species infection where tissue samples were unavailable. These would be applicable only if there were no concomitant or additional systemic antifungal usage.
EOST
Group
Value
95% CI
APX001 (Fosmanogepix)
66.7
29.9 – 92.5
2 Weeks after EOST
Group
Value
95% CI
APX001 (Fosmanogepix)
66.7
29.9 – 92.5
4 Weeks after EOST
Group
Value
95% CI
APX001 (Fosmanogepix)
66.7
29.9 – 92.5
Percentage of Participants With Treatment Success at EOST Determined by InvestigatorSecondary· EOST: any day from Day 1 up to maximum of Day 42
Treatment success was defined as meeting all of the following criteria:
1\) Two consecutive blood cultures negative for Candida species, and/or for participants with a deep-seated site of infection, at least 1 negative tissue culture or aspirate/fluid culture. For participants with a deep-seated site of infection involving visceral organs from which a tissue culture was not obtainable, resolution of the attributable clinical signs of infection recorded at Baseline, and as applicable, radiological improvement associated with the site of infection. 2) Alive at EOST and 3) No concomitant use of
Group
Value
95% CI
APX001 (Fosmanogepix)
88.9
51.8 – 99.7
Percentage of Participants With Treatment Success at 2 and 4 Weeks After EOST Determined by Investigator and by the DRCSecondary· 2 and 4 weeks after EOST (where EOST is any day from Day 1 up to maximum of Day 42)
Treatment success was defined as meeting all of the following criteria:
1\) Two consecutive blood cultures negative for Candida species, and/or for participants with a deep-seated site of infection, at least 1 negative tissue culture or aspirate/fluid culture. For participants with a deep-seated site of infection involving visceral organs from which a tissue culture was not obtainable, resolution of the attributable clinical signs of infection recorded at Baseline, and as applicable, radiological improvement associated with the site of infection. 2) Alive at EOST and 3) No concomitant use of
2 Weeks after EOST: DRC
Group
Value
95% CI
APX001 (Fosmanogepix)
66.7
29.9 – 92.5
4 Weeks after EOST: DRC
Group
Value
95% CI
APX001 (Fosmanogepix)
66.7
29.9 – 92.5
2 Weeks after EOST: Investigator
Group
Value
95% CI
APX001 (Fosmanogepix)
77.8
40.0 – 97.2
4 Weeks after EOST: Investigator
Group
Value
95% CI
APX001 (Fosmanogepix)
77.8
40.0 – 97.2
All-Cause Mortality Through Study Day 30Secondary· Day 1 through Day 30
Percentage of participants who died through study Day 30 is reported in this outcome measure.
Group
Value
95% CI
APX001 (Fosmanogepix)
11.1
Number of Participants With Treatment Emergent Adverse Events (TEAEs)Secondary· Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days)
An adverse event was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs were events between first dose of study drug and up to Week 4 (+4 days) post last dose of study drug that were absent before treatment or that worsened relative to pretreatment state.
Group
Value
95% CI
APX001 (Fosmanogepix)
9
Adverse events — posted to ClinicalTrials.gov
Time frame: Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days).
Reporting threshold: 2%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This is a multicenter, open-label, single arm study to evaluate the efficacy and safety of APX001 for the treatment of candidemia and/or invasive candidiasis caused by C. auris in patients aged 18 years and over with limited antifungal treatment options.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT05491733 — A Bioequivalence Study of APX001 High-load and Low-load Tablets
· Phase 1
· completed
NCT04166669 — A Drug-Drug Interaction Study of CYP3A4 Inhibition and Pan-CYP Induction on APX001
· Phase 1
· completed
NCT03604705 — An Efficacy and Safety Study of APX001 in Non-Neutropenic Patients With Candidemia
· Phase 2
· completed
Other recruiting trials for Candidemia
Currently open trials in the same condition.
NCT05421858 — A Phase 3 Efficacy and Safety Study of Fosmanogepix for the Treatment of Adult Participants With Candidemia and/or Invas
· Phase 3
· recruiting
NCT04979052 — Safety and Efficacy of Interferon-Gamma 1b in Patients With Candidemia
· Phase 2
· active not recruiting
Other Basilea Pharmaceutica trials
Trials by the same sponsor.
NCT06961708 — A Study of Fosmanogepix in Healthy Adult Chinese Subjects
· Phase 1
· completed
NCT06808646 — A Study to Assess the Effect of Ceftobiprole on the PK of Pitavastatin and on Plasma Levels of Coproporphyrin
· Phase 1
· completed
NCT06733675 — Safety and PK of Ceftibuten-ledaborbactam Etzadroxil Fixed-dose Combination
· Phase 1
· recruiting
NCT05421858 — A Phase 3 Efficacy and Safety Study of Fosmanogepix for the Treatment of Adult Participants With Candidemia and/or Invas
· Phase 3
· recruiting
NCT06665555 — Plasma and Intrapulmonary Pharmacokinetics of Ceftibuten and Ledaborbactam in Healthy Male and Female Participants 18 to
· Phase 1
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Basilea Pharmaceutica
Last refreshed: 16 September 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04148287.