Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
AMX0035 · 7 trials · 14 indications
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
Assess the impact of AMX0035 on disease progression as measured by the Progressive Supranuclear Palsy (PSP) Rating Scale (PSPRS); Total scores range from 0-96 with higher scores indicating more progressed disease
* C-peptide area under the curve (AUC) response at Week 24 using a 0-240 minute MMTT * Change from Baseline in area under the curve (AUC) in delta C-peptide at Week 24 using a 0-240 minute MMTT
* Incidence and severity of Adverse Events and Serious Adverse Events * Incidence of abnormalities in clinical laboratory assessments
Rate of treatment emergent adverse events during AMX0035 therapy
Comparison between the AMX0035 Group and Placebo of the number of participants with TEAEs
Change from Baseline in GST (global statistical test combining three measures relevant to disease trajectory (cognition \[MADCOMS: Mild/Moderate Alzheimer's Disease Composite Score\], function \[FAQ: Functional Activities Questionnaire\], and total hippocampal volume on magnetic resonance imaging)) for AMX0035 relative to placebo. For MADCOMS and FAQ, a higher score indicates a worse outcome. A larger hippocampal volume is better, so it was reversed before being normalized. Each of the three were normalized against respective baseline means and standard deviations. The mean of the three normalized scores is the final GST. A higher GST score indicates a worse outcome. Standard deviations above the mean are worse; standard deviations below the mean are better. The expected value of the GST at baseline is 0 because it is the mean of three z-scores whose expected values at baseline are 0. AD is multifaceted and the GST was designed to be sensitive to changes in multiple dimensions.
Number of participants with TEAEs from baseline in the OLE study through the last participant's last visit in the OLE
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.
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). |
| AMX0035 | EXPERIMENTAL | AMX0035 administered by mouth for 52 weeks: once daily for first 2 weeks and then twice daily for remainder of study For participants electing to continue into the open-label phase at Week 52; AMX0035 will be administered once daily for first 2 weeks and then twice daily for remainder of open-label phase |
| Placebo | PLACEBO_COMPARATOR | Placebo administered by mouth for 52 weeks: once daily for first 2 weeks and then twice daily for remainder of study |
| AMX-0035 long term treatment extension | EXPERIMENTAL | AMX0035 administered twice daily p.o. |
| Active (AMX0035) | ACTIVE_COMPARATOR | AMX0035 twice daily--a combination of Sodium Phenylbutyrate (3g) and Taurursodiol (1g) |
| 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...
AMX0035 is an investigational small molecule being studied for the treatment of Alzheimer Disease, Wolfram Syndrome, Progressive Supranuclear Palsy, and Amyotrophic Lateral Sclerosis (ALS). It is in Phase 2 clinical development for these neurological conditions.
AMX0035 is being developed by Amylyx Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol AMLX. The company is conducting clinical trials to evaluate the drug's safety and efficacy in several neurological disorders.
AMX0035 is currently in Phase 2 clinical development. It has completed Phase 2 trials in ALS and Alzheimer's disease, and an open-label extension study in ALS has also been completed. A Phase 3 trial in ALS has been initiated but its status is unknown.
AMX0035 has been studied in several clinical trials. NCT03127514 was a Phase 2 trial in ALS with 137 participants. NCT03488524 was an open-label extension study in ALS with 90 participants. NCT03533257 was a Phase 2 trial in Alzheimer's disease with 95 participants. NCT05021536 is a Phase 3 trial in ALS with 664 participants.
AMX0035 is not FDA approved. It is an investigational drug currently in clinical development. The drug has completed Phase 2 trials and is being evaluated in a Phase 3 trial for ALS, but it has not yet received regulatory approval for any indication.
AMX0035 is a small molecule being investigated for its effects on neurological diseases. While its specific molecular target is not publicly detailed, it is being studied for its potential to address neurodegenerative processes in conditions like ALS and Alzheimer's disease.