Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Clemizole HCl, EPX-100
Clemizole · 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...
Clemizole is being developed for the treatment of Dravet Syndrome and Lennox-Gastaut Syndrome, two severe forms of childhood-onset epilepsy. It is an investigational small molecule and is not yet approved for these indications. Harmony Biosciences Holdings, Inc. is evaluating it in late-stage clinical trials in patients aged 2 years and older.
Clemizole targets the 5-HT2 receptor. By acting on this serotonin receptor, the drug is designed to modulate neuronal signaling relevant to seizure activity in Dravet Syndrome and Lennox-Gastaut Syndrome. This mechanism distinguishes it from many conventional anti-seizure medications that act on sodium channels or GABA pathways.
Clemizole is being developed by Harmony Biosciences Holdings, Inc., which trades on the Nasdaq under the ticker HRMY. The company is running Phase 3 clinical programs for the drug in Dravet Syndrome and Lennox-Gastaut Syndrome, with trials currently recruiting participants in the United States and several other countries.
Clemizole is in Phase 3 clinical development. It is an investigational therapy and has not been approved by the FDA for any indication. The ongoing Phase 3 trials are randomized, double-blind, and placebo-controlled, evaluating the drug in patients with Dravet Syndrome and Lennox-Gastaut Syndrome.
Clemizole is being studied in two Phase 3 trials. NCT05066217 is a Phase 3 efficacy and safety study of Clemizole HCl in patients with Lennox-Gastaut Syndrome, enrolling 260 participants in the United States. NCT04462770 is a Phase 3 study of EPX-100 (Clemizole Hydrochloride) in participants with Dravet Syndrome, enrolling 150 participants across the United States, Canada, Georgia, Hungary, Poland, Romania, Spain, and the United Kingdom. Both trials are currently recruiting.
Yes, Clemizole HCl is another name for Clemizole. Clemizole HCl refers to the hydrochloride salt form of the drug, and it is also referred to as EPX-100 in some clinical trial documentation. All these names refer to the same investigational small molecule being developed by Harmony Biosciences for Dravet Syndrome and Lennox-Gastaut Syndrome.