Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
AMX0114 · 1 trial · 1 indication
Incidence of adverse events (AEs), serious adverse events (SAEs) and dose limiting toxicities (DLTs). Incidence of abnormalities in clinical laboratory assessments, vital signs, physical and neurological examinations, and electrocardiograms (ECGs).
| Arm | Type | Description |
|---|---|---|
| Active Treatment: AMX0114 | EXPERIMENTAL | AMX0114 will be administered once every 4 weeks by intrathecal bolus injection for a total of up to 4 doses. Treatment will be administered on Day 1, followed by repeat dosing every 4 weeks at approximately Day 29, Day 57 and Day 85. |
| Placebo | PLACEBO_COMPARATOR | Placebo drug will be administered once every 4 weeks by intrathecal bolus injection for a total of up to 4 doses. Treatment will be administered on Day 1, followed by repeat dosing every 4 weeks at approximately Day 29, Day 57 and Day 85. |
| Name | Type | Description |
|---|---|---|
| AMX0114 | DRUG | Antisense oligonucleotides (ASOs) are a type of medicine that treats diseases by intercepting the mRNA messages sent within the cell, resulting in fewer specific proteins being made. AMX0114 is an ASO that targets the mRNA messenger that instructs the body to create a protein called calpain-2. Calpain-2 has been linked to the degeneration and death of neurons in many neurological diseases, including people living with sporadic ALS. AMX0114 is designed to reduce the levels of calpain-2, with the goal of slowing down the process that leads to neuron injury and death. |
| Placebo | OTHER | Placebo |
Inclusion Criteria: 1. Ability to understand the purpose and risks of this study, willingness to comply with the study and to provide informed consent in accordance with local laws and regulations. 2. Male or female, at least 18 years of age. 3. Diagnosis of clinically definite or clinically probab...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Amylyx Pharmaceuticals, Inc. | AMLX | 2 | PHASE3 | AMX0035 |
| Biogen Inc. | BIIB | 2 | PHASE3 | Tofersen |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE3 | ION363 |
| Novartis AG Sponsored ADR | NVS | 1 | PHASE2 | VHB937 |
| argenx SE Sponsored ADR | ARGX | 1 | PHASE2 | ARGX-119 |
| Corcept Therapeutics Incorporated. | CORT | 1 | PHASE2 | Dazucorilant |
| MediciNova, Inc. | MNOV | 1 | PHASE2 | MN-166 |
| Coya Therapeutics, Inc. | COYA | 1 | PHASE2 | COYA 302 |
| Clene Inc | CLNN | 1 | PHASE2 | CNMAu8 |
| Eli Lilly and Company | LLY | 2 | PHASE1 | 89Zr-DFO-AP-101 |
| Regeneron Pharmaceuticals, Inc. | REGN | 1 | PHASE1 | ALN-SOD |
| Insmed Incorporated | INSM | 1 | PHASE1 | INS1202 |
| uniQure N.V. | QURE | 1 | PHASE1 | AMT-162 |
| AC Immune SA | ACIU | 1 | EARLY_PHASE1 | Undisclosed |
| Design Therapeutics, Inc. | DSGN | 1 | - | Undisclosed |
AMX0114 is an investigational small molecule being developed for the treatment of amyotrophic lateral sclerosis (ALS). It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being studied in adult participants with ALS in a clinical trial.
AMX0114 targets calpain-2, a protein involved in cellular processes. By modulating this target, the drug aims to address pathways relevant to ALS. The specific mechanism of action is under investigation in clinical trials.
AMX0114 is being developed by Amylyx Pharmaceuticals, Inc., a biopharmaceutical company. The company is conducting clinical trials to evaluate the safety and efficacy of AMX0114 in patients with ALS.
AMX0114 is in Phase 1 clinical development. It is currently being evaluated in a randomized, double-blind, placebo-controlled trial in adult participants with ALS. The drug is investigational and has not yet received regulatory approval.
AMX0114 is being studied in a Phase 1 clinical trial with the identifier NCT06665165. This trial is recruiting participants with ALS in the United States and Canada, with an enrollment target of 48 participants. The study is randomized, double-blind, and placebo-controlled.