An Efficacy and Safety Study of APX001 in Non-Neutropenic Patients With Candidemia

• APX001 (Drug): APX001

Primary outcomes

• Treatment Success at End of Study Treatment (EOST) as Determined by the Data Review Committee (DRC) [One to forty-two days]: Treatment Success is defined as meeting all of the following criteria: Two consecutive blood cultures negative for Candida spp. Alive at EOST No concomitant use of any other systemic antifungal therapies through end of study treatment

Secondary outcomes

• Time to First Negative Blood Culture [One to forty-nine days]: Time to first negative blood culture was defined as the number of days from first dose date of study drug to the date of first post-Baseline negative blood culture + 1. Patients without a negative blood culture at post-Baseline visits were censored at the last assessment date.
• Percentage of Patients With Mycological Outcomes at End of Study Treatment (EOST), End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT) [End of study treatment (EOST), end of treatment (EOT), and 2 and 4 weeks after end of treatment (EOT)]
• Percentage of Patients With Treatment Success at End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT) [2 and 4 weeks after end of treatment (EOT)]
• Overall Survival at Study Day 30 [Day 30]
• Number of Patients With Treatment Emergent Adverse Events (TEAEs) [One to forty-nine days]

Sex: All, ages: 18 Years to any

Key Inclusion Criteria:

* Provision of written consent
* Adults ages 18 and above male or female
* New diagnosis of candidemia
* Able to have pre-existing intravascular catheters removed and replaced (as necessary)

Key Exclusion Criteria:

* neutropenia
* deep-seated Candida-related infections
* hepatosplenic candidiasis
* received more than 2 days of prior systemic antifungal treatment for current candidemia episode
* severe hepatic impairment

Participant flow

Outcome results

• Treatment Success at End of Study Treatment (EOST) as Determined by the Data Review Committee (DRC) (Primary), Count of participants, Participants: Treatment Period - MITT 16
• Time to First Negative Blood Culture (Secondary), Mean, Days: MITT Population 2.4 +/- 1.13
• Percentage of Patients With Mycological Outcomes at End of Study Treatment (EOST), End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT) (Secondary), Count of participants, Participants: EOST (End of Study Drug Treatment) 16; EOT (End of Antifungal Treatment) 15; Follow-up 2 Weeks After EOT 1; Follow-up 4 Weeks After EOT 0
• Percentage of Patients With Treatment Success at End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT) (Secondary), Number, percentage of participants: Treatment Success 2 Weeks After EOT 60.0; Treatment Success 4 Weeks After EOT 55.0
• Overall Survival at Study Day 30 (Secondary), Count of participants, Participants: MITT Population 17
• Number of Patients With Treatment Emergent Adverse Events (TEAEs) (Secondary), Count of participants, Participants: Safety Population 20

Adverse events

Time frame: One to forty-nine days

Serious adverse events by term

Other adverse events by term

• Results publication: Pappas PG, Vazquez JA, Oren I, Rahav G, Aoun M, Bulpa P, Ben-Ami R, Ferrer R, Mccarty T, Thompson GR, Schlamm H, Bien PA, Barbat SH, Wedel P, Oborska I, Tawadrous M, Hodges MR. Clinical safety and efficacy of novel antifungal, fosmanogepix, for the treatment of candidaemia: results from a Phase 2 trial. J Antimicrob Chemother. 2023 Oct 3;78(10):2471-2480. doi: 10.1093/jac/dkad256. PMID 37596890

• Study Protocol (PDF, 2019-06-03)
• Statistical Analysis Plan (PDF, 2020-04-27)

• Marc Engelhardt, Study director, Basilea Pharmaceutica International Ltd, Allschwil