Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Maralixibat · 7 trials · 7 indications
ItchRO(Obs) severity score = Itch Reported Outcome Observer assessment severity score; scale between 0 (not itchy at all) and 4 (extremely itchy); the lower the score the better.
The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch)
The primary efficacy endpoint is the mean change in the average morning ItchRO(Obs) severity score between baseline and Weeks 15-26, using 4-week average morning ItchRO(Obs) severity scores (Mixed Model Repeated Measures). The baseline average morning ItchRO(Obs) severity score is defined as the 4-week average morning ItchRO(Obs) severity score prior to the first dose of the study medication. ItchRo(Obs) Severity Score = Itch Reported Outcome Observer assessment severity score; scale between 0 (not itchy at all) and 4 (extremely itchy); the lower the score the better.
TEAEs = Treatment-emergent Adverse Events
TEAE = Treatment-emergent Adverse Event; AESI = Adverse Event of Special Interest..
Fecal bile acid excretion was determined by the concentration of total bile acids in stool samples over each 24-hour collection window during the lead-in and treatment periods.
| Arm | Type | Description |
|---|---|---|
| Maralixibat | EXPERIMENTAL | Participants will receive maralixibat oral solution 300 μg/kg orally once daily for 1 week and then twice daily for 39 weeks. |
| Placebo | PLACEBO_COMPARATOR | Participants will receive placebo matched to maralixibat oral solution orally once daily for 1 week and then twice daily for 19 weeks. After 20 weeks, participants will receive maralixibat oral solution 300 μg/kg orally once daily for 1 week and then twice daily for 19 weeks. |
| Double Blind - Maralixibat | EXPERIMENTAL | The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm. |
| Double Blind - Placebo | PLACEBO_COMPARATOR | The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm. |
| Open Label - Maralixibat | EXPERIMENTAL | The Open-Label period comprised of 4-8 weeks of dose escalation followed by 70 - 74 weeks of stable dosing treatment. During the OLE, all participants, regardless of treatment assignment in the double-blind period, received maralixibat. |
| Maralixibat 10mg | EXPERIMENTAL | Participants will receive maralixibat 10 mg liquid formulation orally QD for 7 days. |
| Volixibat 10mg | EXPERIMENTAL | Participants will receive volixibat 10 mg capsule orally QD for 7 days. |
| Maralixibat 20mg | EXPERIMENTAL | Participants will receive maralixibat 20 mg liquid formulation orally QD for 7 days. |
| Volixibat 20mg | EXPERIMENTAL | Participants will receive volixibat 20 mg capsule orally QD for 7 days. |
| Maralixibat 50mg | EXPERIMENTAL | Participants will receive maralixibat 50 mg liquid formulation orally QD for 7 days. |
| Maralixibat 50mg BID | EXPERIMENTAL | Participants will receive maralixibat 50 mg liquid formulation orally BID for 7 days. |
| Maralixibat 100mg | EXPERIMENTAL | Participants will receive maralixibat 100 mg liquid formulation orally QD for 7 days. |
| Name | Type | Description |
|---|---|---|
| Maralixibat | DRUG | Maralixibat will be provided as an oral solution along with 0.5-, 1.0-, and 3.0-mL sized dosing dispensers. During the double-blind dose escalation period (4 weeks), the study drug (maralixibat) will be administered once daily for 1 week and then twice daily (BID; morning and evening). During the double-blind stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the double-blind dose-escalation period) of maralixibat. During the open-label dose escalation period (4 weeks), all participants will receive maralixibat treatment once daily for 1 week and then twice daily (BID; morning and evening). During the open-label stable dosing period (at least 16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the open-label dose-escalation period) of maralixibat. |
| Placebo | OTHER | Placebo matched to maralixibat will be provided as an oral solution along with 0.5-, 1.0-, and 3.0-mL sized dosing dispensers. During the double-blind dose escalation period (4 weeks), study drug will be administered once daily for 1 week and then twice daily (BID; morning and evening). During the double-blind stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the double-blind dose-escalation period) of study drug. During the open-label dose escalation period (4 weeks), all participants will receive maralixibat treatment once daily for 1 week and then twice daily (BID; morning and evening). During the open-label stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the open-label dose-escalation period) of maralixibat. |
| Volixibat | DRUG | Participants will receive volixibat in 10 mg and 20 mg doses. |
Inclusion Criteria: 1. Informed consent and assent (as applicable) 2. Age ≥6 months at time of baseline visit 3. Diagnosis of a rare cholestatic liver disease with cholestatic pruritus based on the following: 1. Chronic liver biochemical abnormalities (\>90 days) and/or pathological evidence of...
Maralixibat is an investigational small molecule being studied for cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC), biliary atresia, and other cholestatic conditions. It is also being evaluated in healthy volunteers. The drug is in clinical development and has not been approved by the FDA.
Maralixibat is being developed by Mirum Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker MIRM. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with cholestatic liver diseases.
Maralixibat is in Phase 2 and Phase 3 clinical trials. Most completed studies are Phase 2, while one active trial, NCT06553768, is Phase 3. The drug is investigational and not yet approved for any indication.
Maralixibat has been studied in several trials, including NCT04168385, a long-term safety study in cholestatic liver disease; NCT04524390, evaluating response post-Kasai in biliary atresia; NCT04729751, in infants with PFIC and Alagille syndrome; and NCT06553768, the EXPAND study for pruritus in general cholestatic liver disease.
Yes, Maralixibat is also known as MRX-800. One clinical trial, NCT04168385, is titled 'MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease.' This alternative name may appear in clinical trial records and publications.
Maralixibat is a small molecule that targets the apical sodium-dependent bile acid transporter (ASBT) in the ileum. By inhibiting this transporter, it reduces bile acid reabsorption and lowers systemic bile acid levels, which may alleviate pruritus and liver damage in cholestatic diseases.