Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as clemizole, clemizole hydrochloride (HCL), Clemizole hydrochloride, Clemizole HCl
EPX-100 · 4 trials · 4 indications
Percent change in CMMS-28 from the Baseline Period through the end of the DB Period
Percent change in CMS-28 from the Baseline Period through the end of the DB period.
Percent change in CMS-28 from the Baseline Period through the end of the maintenance period.
| Arm | Type | Description |
|---|---|---|
| Placebo | PLACEBO_COMPARATOR | Participants will receive their first dose of study drug following randomization. |
| Double-blind clemizole HCl | EXPERIMENTAL | Participants will receive their first dose of study drug following randomization. |
| Open-label clemizole HCl | EXPERIMENTAL | Eligible participants who complete the DB Period will have the option to continue in the OLE Period, during which they will receive clemizole HCl for up to 3 years. |
| Part 1 SAD: EPX-100 | EXPERIMENTAL | Sequential cohorts of participants will receive a single ascending dose of EPX-100. |
| Part 1 SAD: Placebo | PLACEBO_COMPARATOR | Participants will receive a single dose of EPX-100-matching placebo. |
| Part 2 MAD: Treatment Arm | EXPERIMENTAL | Participants will receive EPX-100 twice daily on Days 1 to 13, and a morning dose of EPX-100 on Day 14. Moxifloxacin-matching placebo will be administrated on Days 1 and 15. Participants will also receive a single dose of EPX-100-matching placebo on the morning of Days -1 and 15. |
| Part 2 MAD: Control Arm | ACTIVE_COMPARATOR | Participants will receive moxifloxacin or moxifloxacin-matching placebo on the morning of Day 1 then will cross over to the alternate treatment on the morning of Day 15. EPX-100-matching placebo will be administered twice daily on Days 1 to 13 with a morning dose on Day 14. Participants will also receive a single dose of EPX-100-matching placebo on the morning of Days -1 and 15. |
| Part 1 - Itraconazole | EXPERIMENTAL | Treatment A (Period 1): EPX-100 on Day 1. Treatment B (Period 2): itraconazole twice daily (BID) on Day 1; then once daily (QD) on Days 2-5, with EPX-100 administered approximately 2 hours after itraconazole dosing on Day 5. |
| Part 2 - Phenytoin | EXPERIMENTAL | Treatment C (Period 1): EPX-100 on Day 1. Treatment D (Period 2): phenytoin three times daily (TID) on Days 1 to Day 14, with EPX-100 coadministered on the morning of Day 14. |
| Part 3 - Omeprazole, Midazolam, and Bupropion | EXPERIMENTAL | Treatment E (Period 1): omeprazole on Day 1; midazolam on Day 2; bupropion on Day 3. Treatment F (Period 2): EPX-100 BID on Days 1-18 with omeprazole coadministered on the morning of Day 12; midazolam coadministered on the morning of Day 14; bupropion coadministered on the morning of Day 15. |
| Name | Type | Description |
|---|---|---|
| Clemizole HCl | DRUG | Clemizole HCl will be administered as an oral solution. |
| Placebo | DRUG | Placebo will be administered as an oral solution. |
| EPX-100 | DRUG | Oral solution |
| EPX-100-Matching Placebo | DRUG | Oral Solution |
| Moxifloxacin | DRUG | Over-encapsulated tablet |
| Moxifloxacin-Matching Placebo | DRUG | Capsule |
| Itraconazole | DRUG | Oral solution |
| Phenytoin | DRUG | Phenytoin sodium extended-release capsules |
| Omeprazole | DRUG | Delayed-release capsule |
| Midazolam | DRUG | Midazolam hydrochloride (HCl) syrup |
| Bupropion | DRUG | Bupropion HCl tablets |
Key Inclusion Criteria: 1. Males or females, ages ≥2 to ≤55 years, at the time of Screening. 2. Participant/parent/legal authorized representative (LAR) willing and able to give written informed consent/assent. 3. Diagnosis of LGS, including: * Evidence of at least one type of countable major m...
EPX-100 is an investigational small molecule developed by Harmony Biosciences Holdings, Inc. It is being studied in Phase 1 clinical trials in healthy adult participants, including a single and multiple ascending dose study and a drug-drug interaction study. Its target indications have not been disclosed.
EPX-100 is developed by Harmony Biosciences Holdings, Inc., which trades under the ticker HRMY. The company is running the Phase 1 clinical program for the asset, including studies in healthy adult volunteers in the United States.
EPX-100 is in Phase 1 clinical development. It is investigational and is being evaluated in early-stage studies in healthy adult participants, including a recruiting single and multiple ascending dose study and a completed drug-drug interaction study.
EPX-100 has two Phase 1 trials. NCT07879170 is a recruiting single and multiple ascending dose study in healthy adults with planned enrollment of 88. NCT07824193 is a completed drug-drug interaction study in healthy participants with enrollment of 54. Both are in the United States.
Yes, EPX-100 is also known as clemizole. The drug-drug interaction trial is listed under the name Clemizole (EPX-100), confirming that the two names refer to the same investigational small molecule in Harmony Biosciences' Phase 1 program.
Both EPX-100 trials are Phase 1 studies in healthy adult volunteers aged 18 and older in the United States. Together they plan to enroll 142 participants. The program includes randomized, double-blind, controlled designs, and one trial is currently active while the other is completed.