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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06665165 | AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis | PHASE1 | RECRUITING | 48 | — | — | Apr 7, 2025 | Oct 1, 2027 | Apr 14, 2026 | 14 | United States, Canada |
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, AMX0114 |
| 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 |
| Coya Therapeutics, Inc. | COYA | 1 | PHASE2 | COYA 302 |
| MediciNova, Inc. | MNOV | 1 | PHASE2 | MN-166 |
| Clene Inc | CLNN | 1 | PHASE2 | CNMAu8 |
| Eli Lilly and Company | LLY | 2 | PHASE1 | 89Zr-DFO-AP-101, LY4256984 |
| 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 |