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...
Top 13 of 15 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Biogen Inc. | BIIB | 2 | PHASE3 | Tofersen |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE3 | ION363 |
| Corcept Therapeutics Incorporated. | CORT | 1 | PHASE2 | Dazucorilant |
| Novartis AG Sponsored ADR | NVS | 1 | PHASE2 | VHB937 |
| argenx SE Sponsored ADR | ARGX | 1 | PHASE2 | ARGX-119 |
| 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 | LY4256984 |
| Amylyx Pharmaceuticals, Inc. | AMLX | 1 | PHASE1 | AMX0114 |
| Regeneron Pharmaceuticals, Inc. | REGN | 1 | PHASE1 | ALN-SOD |
| Insmed Incorporated | INSM | 1 | PHASE1 | INS1202 |
| uniQure N.V. | QURE | 1 | PHASE1 | AMT-162 |
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 the FDA. The drug is being studied in adult participants with ALS in a randomized, double-blind, placebo-controlled trial.
AMX0114 targets calpain-2, a protein that belongs to the calpain family of proteases. By inhibiting calpain-2, the drug aims to modulate pathways involved in ALS pathology. This mechanism is being evaluated in early-stage clinical trials to assess its safety and tolerability in patients.
AMX0114 is being developed by Amylyx Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol AMLX. The company is conducting a Phase 1 clinical trial of AMX0114 in the United States and Canada for the treatment of ALS.
AMX0114 is currently in Phase 1 clinical development. It is an investigational drug and has not received FDA approval. The ongoing Phase 1 trial is recruiting participants with ALS and is designed to evaluate the drug's safety, tolerability, and pharmacokinetics in a randomized, placebo-controlled setting.
AMX0114 is being studied in a single Phase 1 clinical trial with the identifier NCT06665165, titled 'AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis.' The trial is recruiting 48 participants in the United States and Canada, and it is a randomized, double-blind, placebo-controlled study.