Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as KL1333
Napazimone · 4 trials · 6 indications
Number of adverse events will be monitored throughout the study for all subjects
The following parameters and body systems will be examined and any abnormalities described: height and weight; general appearance; skin; head, ears, eyes, nose, and throat; lungs; heart; lower extremity examination; abdomen; neurologic and lymph nodes. Any clinically significant changes from baseline should be recorded as AEs.
Body temperature, systolic and diastolic cuff blood pressure, pulse rate and pulse oximetry will be measured and any clinically significant changes from baseline should be recorded as AEs.
Changes from baseline of ECG parameters will be evaluated.
Monitoring of the clinically significant laboratory results for sodium, potassium, chloride, bicarbonate/carbon dioxide;, blood urea nitrogen, serum creatinine, glucose, albumin, total protein, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, total bilirubin, direct bilirubin, indirect bilirubin, calcium, gamma-glutamyl transferase, creatine kinase
Monitoring of the clinically significant laboratory results for specific gravity, pH, semi-quantitative "dipstick" evaluation of glucose, protein, bilirubin, ketones, leukocytes, blood microscopy and/or culture to be performed if clinically indicated or if urinalysis results positive.
Monitoring of the clinically significant laboratory results for Hemoglobin, hematocrit, white blood cell with differentials (monocytes, eosinophils, basophils, neutrophils, lymphocytes) as an absolute value, red blood cell count, platelet count, C-reactive protein
These events will be monitored throughout the study.
C-SSRS assesses suicidal ideation and behavior risk through a series of questions to assess for suicidal ideation and behavior, the severity and immediacy of the risk, and the level of support the subject may need. C-SSRS Severity of Ideation scores of 4 or 5 are considered SAEs.
Change in t-score. Higher scores indicate greater fatigue severity.
Change in number of stands
Area under the curve AUC
| Arm | Type | Description |
|---|---|---|
| Open label extension | EXPERIMENTAL | Subjects will receive medication twice a day for 48 weeks minimum |
| KL1333 | EXPERIMENTAL | Twice daily |
| Matching Placebo | PLACEBO_COMPARATOR | Twice daily |
| Active | EXPERIMENTAL | Daily treatment |
| Name | Type | Description |
|---|---|---|
| Napazimone | DRUG | Product: KL1333 (international nonproprietary name: napazimone) Dose: Each subject will be up-titrated to his/her maximum well tolerated dose. The starting dose will be 25 mg KL1333 twice daily (BID; total daily dose of 50 mg). If KL1333 is considered to be well tolerated after 4 weeks of treatment, the dose will be increased to 50 mg KL1333 BID (total daily dose of 100 mg). The dose may be lowered from 50 mg BID to 25 mg BID at the investigator's discretion throughout the study in case of tolerability issues. Frequency: Twice daily Route: Oral |
| 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: * Completed the FALCON study (age 18 years or older), and in the opinion of the investigator and sponsor has been compliant with the study requirements * Willingness and ability to attend study appointments within the specified time windows * Willingness and ability to complete ...
Napazimone is being developed for the treatment of mitochondrial disease, including primary mitochondrial disease. It is an investigational small molecule in Phase 2 clinical development and is not yet approved for any indication. Clinical trials are enrolling adults aged 18 years and older with primary mitochondrial disease.
Napazimone targets NQO1, an enzyme involved in cellular redox regulation. By acting on NQO1, the small molecule is designed to influence mitochondrial energy metabolism. This mechanism underlies its development as a potential therapy for primary mitochondrial disease, a rare disorder with limited treatment options.
Napazimone is being developed by Pharming Group N.V., a biopharmaceutical company listed under the ticker PHAR. The company is advancing the asset through Phase 2 clinical development for mitochondrial disease, with additional trials planned or ongoing in multiple countries across Europe, North America, and Australia.
Napazimone is in Phase 2 clinical development. It is an investigational therapy and has not been approved by the FDA or any other regulatory agency. The Phase 2 program includes two recruiting trials in adults with primary mitochondrial disease, supported by earlier completed Phase 1 studies.
Napazimone is being studied in several clinical trials. The Phase 2 trials include NCT07514338, an open-label extension assessing long-term safety and efficacy, and NCT05650229, evaluating efficacy in adults with primary mitochondrial disease. Completed Phase 1 studies include NCT04643249, a drug-drug interaction study, and NCT03888716, a single and multiple ascending dose study.
Yes, Napazimone is also known as KL1333. Both names refer to the same investigational small molecule being developed by Pharming Group N.V. for mitochondrial disease. The KL1333 designation appears in the clinical trial identifiers and study titles for the Phase 1 and Phase 2 programs.