Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as WTX101, Bis-choline tetrathiomolybdate, Tiomolibdate choline, formerly WTX101
ALXN1840 · 6 trials · 1 indication
Liver biopsy samples were taken for the assessment of liver Cu concentration. Multiple imputation was used to impute missing data at Week 48 due to any reason based on Baseline values.
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Normalized concentrations of NCC was defined as who achieving or maintaining normalized levels of NCC (0.8 to 2.3 micromole \[μmol\]l/liter \[L\]\]) adjusted for Mo plasma concentration or reaching a reduction of at least 25% in NCC corrected for Mo if above the normal reference range at the time of enrollment. NCC was calculated by subtracting the amount of copper (Cu) bound to ceruloplasmin (CP) from the total plasma Cu concentration. Post-baseline NCC values were adjusted (corrected) to account for Cu bound in tripartite complexes with ALXN1840 and albumin. Descriptive statistics are reported.
Blood samples were collected for pharmacokinetics (PK) analysis of celecoxib. Cmax is reported as nanograms (ng)/milliliter (mL).
Blood samples were collected for pharmacokinetic (PK) analysis of celecoxib. Area Under The Plasma Concentration Versus Time Curve From Time 0 To The Last Quantifiable Concentration (AUCt) is reported as hours•ng/mL (h•ng/mL).
Blood samples were collected for pharmacokinetic (PK) analysis of celecoxib. Area Under The Plasma Concentration Versus Time Curve From Time 0 To Infinity (AUCinf) is reported as h•ng/mL.
| Arm | Type | Description |
|---|---|---|
| ALXN1840 | EXPERIMENTAL | Participants will receive ALXN1840. |
| Treatment A | EXPERIMENTAL | Participants will receive bupropion. |
| Treatment B | EXPERIMENTAL | Participants will receive bupropion with ALXN1840. |
| Treatment Sequence A-B | EXPERIMENTAL | Participants received 1 treatment during each study period in the following sequence: * Treatment A: Celecoxib. * Treatment B: Celecoxib plus ALXN1840. |
| Treatment Sequence B-A | EXPERIMENTAL | Participants received 1 treatment during each study period in the following sequence: * Treatment B: Celecoxib plus ALXN1840. * Treatment A: Celecoxib. |
| Cohort 1: Japanese Participants | EXPERIMENTAL | All Japanese participants will receive a single dose of ALXN1840 15 milligrams (mg) in Dosing Period 1 and will receive a single dose of ALXN1840 60 mg in Dosing Period 2. |
| Cohort 2: Non-Japanese Participants | EXPERIMENTAL | All non-Japanese participants will receive a single dose of ALXN1840 15 mg in Dosing Period 1 and will receive a single dose of ALXN1840 60 mg in Dosing Period 2. |
| Name | Type | Description |
|---|---|---|
| Bis-Choline Tetrathiomolybdate | DRUG | Participants will be initiated at 15 milligrams once daily, then the dose will be increased to 30 milligrams once daily at Week 6. |
| ALXN1840 | DRUG | Administered orally as tablets. |
| Bupropion Hydrochloride | DRUG | Bupropion hydrochloride will be administered orally as a single dose as one 150 milligrams (mg) tablet with 240 milliliters of water (fasting). |
| Celecoxib | DRUG | Celecoxib was administered orally as a single dose as one 200-mg tablet with 240 mL of water (fasting). |
Inclusion Criteria: 1. Diagnosis of WD by Leipzig Criteria ≥ 4 or by historical test results. 2. Continuous treatment for WD with penicillamine, trientine or zinc for at least 1 year prior to screening. 3. Body mass index \< 30 kilograms/meter squared. 4. Able to cooperate with a percutaneous liver...
ALXN1840 is an investigational small molecule being developed for the treatment of Wilson Disease, a rare inherited disorder of copper metabolism. It is being studied in adult patients with Wilson Disease as well as in healthy adult volunteers in supporting clinical pharmacology studies. It has not been approved for any use.
ALXN1840 is being developed by AstraZeneca PLC, which trades on the Nasdaq under the ticker AZN. AstraZeneca is the sponsor of the clinical program evaluating the compound in Wilson Disease.
ALXN1840 is in Phase 2 clinical development for Wilson Disease. Five trials have been completed, including Phase 1 pharmacokinetic and drug interaction studies and a Phase 2 copper and molybdenum balance study. No trials are currently listed as active, and the drug remains investigational.
Completed ALXN1840 trials include NCT04573309, a Phase 2 copper and molybdenum balance study in Wilson Disease patients, plus Phase 1 studies NCT05641311 in Japanese and non-Japanese healthy participants, NCT04526210 evaluating a CYP2B6 substrate interaction, and NCT04526197 evaluating a CYP2C9 substrate interaction. Total enrollment across the program is 152 participants.
ALXN1840 is not approved; it is an investigational drug still in clinical development. Its completed studies include Phase 1 and Phase 2 trials in Wilson Disease and healthy volunteers, and the program has not advanced beyond Phase 2 based on the available development information.
ALXN1840 has been evaluated in controlled, randomized clinical trials, including a Phase 2 study of copper and molybdenum balance in adults with Wilson Disease conducted in the United States, New Zealand, and the United Kingdom. Supporting Phase 1 studies enrolled healthy adult volunteers, including Japanese and non-Japanese participants.