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AMT-162 · 1 trial · 1 indication
Occurrence of TEAEs upon administration of ascending doses of AMT-162
| Arm | Type | Description |
|---|---|---|
| 3 single Ascending Dose Levels | EXPERIMENTAL | Experimental: 3 single Ascending Dose Levels The study will be open-label with an initial plan to explore 3 dose levels of AMT-162 in approximately 6 to 12 Participants in total. Each Participant will receive a single dose of AMT-162 delivered via an intrathecal (IT) infusion and will be followed for up to 5 years after AMT-162 administration. |
| EXPANSION COHORT | EXPERIMENTAL | Expansion cohort: To further test selected dose from the SAD part in approximately 6 to 8 participants The study will be open-label. Each Participant will receive a single dose of AMT-162 delivered via an intrathecal (IT) infusion and will be followed for up to 5 years after AMT-162 administration. |
| Name | Type | Description |
|---|---|---|
| AMT-162 | DRUG | AMT-162, the investigational product (IP), is a nonreplicating, rep/cap-deleted, self-complementary Recombinant adeno-associated virus (rAAV) vector based on adeno-associated virus (AAV) serotype rh10 and contains complementary deoxyribonucleic acid (cDNA) encoding an artificial miRNA targeting the SOD1 gene. |
Inclusion Criteria: * Confirmed clinical and genetic diagnosis of SOD1-mediated ALS (SOD1-ALS) experiencing signs and/or symptoms of lower motor neuron dysfunction (weakness, atrophy, cramps, fasciculations), with or without upper motor neuron symptoms (weakness, bring reflexes, spasticity). * ALSF...
AMT-162 is an investigational gene therapy being developed for the treatment of Amyotrophic Lateral Sclerosis (ALS), specifically in adult participants with SOD1-ALS. It is administered intrathecally. The drug is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
AMT-162 targets the SOD1 gene, which is implicated in a form of Amyotrophic Lateral Sclerosis known as SOD1-ALS. By targeting this gene, the therapy aims to address the underlying genetic cause of the disease. The drug is designed for intrathecal administration to deliver the gene therapy directly to the central nervous system.
AMT-162 is being developed by uniQure N.V., a biopharmaceutical company. The company's stock is traded under the ticker symbol QURE. uniQure is conducting clinical trials to evaluate the safety, tolerability, and exploratory efficacy of AMT-162 in patients with SOD1-ALS.
AMT-162 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The ongoing Phase 1 trial is active but not recruiting participants, and it is designed to assess the safety, tolerability, and exploratory efficacy of the therapy.
AMT-162 is being studied in a Phase 1 clinical trial with the identifier NCT06100276. This trial is titled 'Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)'. The trial is active but not recruiting, with an enrollment of 20 participants in the United States and Sweden.
AMT-162 is a gene therapy specifically designed for SOD1-ALS, a form of Amyotrophic Lateral Sclerosis caused by mutations in the SOD1 gene. While it is not known by other names, it is often referred to as an SOD1-ALS gene therapy in clinical trial descriptions. The therapy is administered intrathecally to target the central nervous system.