Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
AMX0035 · 8 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
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.
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
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. |
| 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 developed for Wolfram Syndrome, Amyotrophic Lateral Sclerosis, Progressive Supranuclear Palsy, and Alzheimer Disease. It is currently in Phase 2 clinical development and has not been approved by the FDA.
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 conditions.
AMX0035 is currently in Phase 2 clinical development. It has completed Phase 2 trials for Amyotrophic Lateral Sclerosis and Alzheimer Disease, and an active Phase 3 trial for ALS is ongoing but not yet recruiting participants.
AMX0035 has been studied in several clinical trials, including NCT03127514, a completed Phase 2 trial in ALS with 137 participants, and NCT03533257, a completed Phase 2 trial in Alzheimer's Disease with 95 participants. An active Phase 3 trial, NCT05021536, is ongoing for ALS with 664 participants.
AMX0035 is not known by any alternative names. It is a unique investigational compound developed by Amylyx Pharmaceuticals for the treatment of neurological disorders.