Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
KL1333 · 3 trials · 6 indications
Change in t-score. Higher scores indicate greater fatigue severity.
Change in number of stands
Area under the curve AUC
| Arm | Type | Description |
|---|---|---|
| KL1333 | EXPERIMENTAL | Twice daily |
| Matching Placebo | PLACEBO_COMPARATOR | Twice daily |
| Active | EXPERIMENTAL | Daily treatment |
| Name | Type | Description |
|---|---|---|
| KL1333 | DRUG | Twice daily |
| Placebo | DRUG | Twice daily |
| Flurbiprofen | DRUG | Tablet |
| Dextromethorphan | DRUG | Syrap |
| Bupropion | DRUG | Tablet |
| Midazolam injection | DRUG | solution for injection |
| Omeprazole | DRUG | Capsule |
| Caffeine | DRUG | Tablet or capsule |
| Repaglinide | DRUG | Tablet |
| Placebo Oral Tablet | DRUG | 25 and 100 mg placebo encapsulated tablets |
Inclusion Criteria: * Age 18 years or older. * A confirmed PMD diagnosis caused by a known pathogenic gene mutation or deletion of the mitochondrial genome (category 6 of the International Classification of Inborn Metabolic Disorders \[ICIMD\])12 according to American College of Medical Genetics (A...
KL1333 is an investigational small molecule being developed for the treatment of primary mitochondrial disease, a group of disorders caused by mitochondrial dysfunction. It is also being studied in related conditions such as MELAS syndrome and mitochondrial myopathies. The drug is currently in clinical development and has not been approved by regulatory authorities.
KL1333 is being developed by Pharming Group N.V., a biopharmaceutical company listed on the stock exchange under the ticker PHAR. The company is conducting clinical trials to evaluate the safety and efficacy of KL1333 in patients with mitochondrial disease.
KL1333 is currently in Phase 2 clinical development. It has completed Phase 1 trials in healthy subjects and patients with primary mitochondrial disease, and a Phase 2 efficacy study is now recruiting participants. The drug is investigational and has not received FDA approval.
KL1333 has been studied in several clinical trials. NCT03888716 was a Phase 1 single and multiple ascending dose study in healthy subjects and patients with primary mitochondrial disease. NCT04643249 was a drug-drug interaction study in healthy subjects. NCT05650229 is an ongoing Phase 2 efficacy trial in adult patients with primary mitochondrial disease.
KL1333 is a distinct investigational small molecule developed by Pharming Group N.V. for primary mitochondrial disease. It is not known to be the same as any other approved or investigational treatment for this condition. Its mechanism of action is not publicly detailed in the available clinical trial information.