Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as AN2-501971
Epetraborole · 2 trials · 2 indications
Improvement in severity of at least 50% of the symptoms present at baseline and no deterioration in severity of symptoms present at baseline
Defined as 3 consecutive negative cultures one month apart without intervening or subsequent positives
Defined as improvement in sputum colony count category as defined by Griffith et al, 2015
QOL-B Domain scores range from 0 to 100, with higher scores indicating better health-related quality of life
MACrO2 scores range from 0 to 100, with lower scores indicating better symptom-related quality of life
Defined as 2 consecutive negative cultures collected one month apart without intervening or subsequent positives
Determination of the maximum observed plasma concentration (Cmax)
Determine the area under the plasma concentration versus time curve from time 0 to the last time point evaluated (AUC0-t)
Determine the area under the plasma concentration versus time curve from time 0 to 24 hours (AUC0-24)
Determine area under the drug concentration versus time curve, from time zero to infinity (AUC0-∞)
Determine the apparent total plasma clearance of drug (CL/F)
Determination the time to maximum plasma concentration (Tmax)
Determine the AUC from 0 to the last quantifiable concentration AUClast immediately after the end of dialysis period from inflow (arterial) line
Determine the AUC from 0 to the last quantifiable concentration AUClast immediately after the end of dialysis period from inflow (venous) line
Determine the cumulative amount excreted over all time intervals (0 to T), calculated as the sum of all amounts excreted from each interval (t1-t2)
Determine the fraction of unchanged drug excreted in urine during the time interval (expressed in %, calculated) for parent drug only
Determine renal clearance (Ae(0-t)/ AUC0-T) for parent drug only
Determine amount of drug measured in the whole dialysate volume during dialysis period
Determine the Dialysis clearance over the dialysis period
Incidence, relatedness, and severity of adverse events
Incidence of changes in clinical laboratory measurements from baseline
Incidence of changes in blood pressure, pulse, respiratory rate, and temperature
Incidence of changes in 12-lead ECG parameters from baseline
Incidence of physical exam abnormalities
| Arm | Type | Description |
|---|---|---|
| High Dose Epetraborole | EXPERIMENTAL | This arm is a daily treatment regimen of a 750mg oral dose of Epetraborole. |
| Low Dose Epetraborole | EXPERIMENTAL | This arm is a daily treatment regimen of a 500mg oral dose of Epetraborole. |
| High Dose Placebo | PLACEBO_COMPARATOR | This arm is a daily treatment regimen of a placebo, matching the high dose experimental arm. |
| Low Dose Placebo | PLACEBO_COMPARATOR | This arm is a daily treatment regimen of a placebo, matching the low dose experimental arm. |
| Open Label | EXPERIMENTAL | All subjects in Cohorts 1-4 receive 500 mg epetraborole once; Subjects on Cohort 5 receive 500 mg epetraborole twice |
| Name | Type | Description |
|---|---|---|
| Epetraborole | DRUG | High-dose intervention (750mg daily) |
| Placebo | DRUG | Placebo intervention (matching the high-dose experimental intervention) |
Inclusion Criteria: 1. Male or female patients who are 18 years of age or older. 2. Willing and able to provide written informed consent. 3. Patients with MABc lung disease, meeting the following (a) Microbiological, (b) Clinical, (c) Radiographic a. Microbiological criteria: i. Documentation o...
Epetraborole is an investigational oral antibiotic being developed for Mycobacterium abscessus lung disease, a difficult-to-treat nontuberculous mycobacterial infection. It is also being studied in people with renal insufficiency to characterize how the drug behaves across different levels of kidney function. It is not approved for any indication.
Epetraborole targets leucyl-tRNA synthetase, or LeuRS, an essential bacterial enzyme involved in protein synthesis. By inhibiting LeuRS, the drug interferes with the bacterium's ability to build proteins. This mechanism is distinct from that of many older antibiotics used against mycobacterial infections.
Epetraborole is being developed by AN2 Therapeutics, Inc., a biopharmaceutical company that trades under the ticker ANTX. The company is advancing the drug as an oral small molecule for infectious disease indications, with Mycobacterium abscessus lung disease as its lead program.
Epetraborole is in Phase 2 clinical development for Mycobacterium abscessus lung disease. A separate Phase 1 study in subjects with varying degrees of renal function has been completed. The drug remains investigational and has not been approved by the FDA for any indication.
The ongoing trial is NCT07301320, a Phase 2 study of Epetraborole in patients with Mycobacterium abscessus lung disease. It is randomized, double-blind, placebo-controlled, recruiting in the United States, and plans to enroll 84 adults. A completed Phase 1 renal function study, NCT05283746, enrolled 40 participants.