Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05619783 | Extension Study Evaluating The Safety And Tolerability of AMX0035 | PHASE3 | COMPLETED | 352 | — | — | Dec 29, 2022 | Oct 30, 2024 | Jan 13, 2025 | 46 | Belgium, France +9 |
| NCT05021536 | Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment | PHASE3 | ACTIVE NOT_RECRUITING | 664 | — | — | Oct 28, 2021 | Jan 1, 2026 | Aug 14, 2024 | 69 | United States, Belgium +10 |
| NCT04516096 | A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS) | PHASE2 | COMPLETED | 28 | — | — | Nov 22, 2020 | Jan 31, 2023 | Apr 7, 2023 | 5 | United States |
| NCT03488524 | Open Label Extension Study of AMX0035 in Patients With ALS | PHASE2 | COMPLETED | 90 | — | — | Mar 29, 2018 | Mar 1, 2021 | Aug 13, 2025 | 24 | United States |
| NCT03127514 | AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) | PHASE2 | COMPLETED | 137 | — | — | Jun 22, 2017 | Nov 24, 2019 | May 16, 2024 | 25 | United States |
Incidence of all adverse events (AE)s; AEs leading to treatment discontinuation or study withdrawal, and all serious adverse events (SAE)s in participants treated with AMX0035
Change in slope of Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) over treatment duration. The ALSFRS-R consists of 12 items across 4 subdomains of function (bulbar, fine motor, gross motor, and breathing) with each item scored on a scale from 0 (total loss of function) to 4 (no loss of function). Total scores range from 0 to 48, with higher scores indicating better function.
Rate of treatment emergent adverse events during AMX0035 therapy
Number of participants with TEAEs from baseline in the OLE study through the last participant's last visit in the OLE
Comparison Between Groups of Number of Participants With Adverse Events Until Planned Completion
A comparison of the number of participants in each group able to remain on study drug until planned discontinuation between groups
| Arm | Type | Description |
|---|---|---|
| Active | EXPERIMENTAL | All participants will be treated with oral (or feeding tube) AMX0035 (a fixed-dose combination of Sodium Phenylbutyrate (PB) and taurursodiol). All participants will take 2 sachets daily (one morning dose and one evening dose) starting on Day 1, for the duration of the study (if twice a day dosing is poorly tolerated, dosing interruptions and reductions are further discussed in section 6.3) AMX0035 will be supplied by Amylyx as a carton box containing approximately 1 month supply of single use sachets. Each AMX0035 sachet contains active ingredients in a powder formulation with 3 g PB and 1 g taurursodiol. AMX0035 powder is mixed with water and taken orally (or via feeding tube). |
| Placebo | PLACEBO_COMPARATOR | Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating |
| AMX0035 | EXPERIMENTAL | Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating |
| AMX-0035 long term treatment extension | EXPERIMENTAL | AMX0035 administered twice daily p.o. |
| Name | Type | Description |
|---|---|---|
| AMX0035 | DRUG | Combination of 3 g phenylbutyrate and 1 g taurursodiol |
| Placebo | OTHER | Matching Placebo Comparator |
Inclusion Criteria: 1. Previous participation in Study A35-004 (PHOENIX), including completion of the randomized controlled phase through Week 48 (this timepoint may be upcoming at the time of screening). Participants who do not complete randomized-controlled phase through Week 48 for medical reaso...