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zilganersen · 1 trial · 1 indication
10-Meter Walk Test (10MWT) is an assessment of gait speed.
| Arm | Type | Description |
|---|---|---|
| zilganersen | EXPERIMENTAL | Zilganersen will be administered by intrathecal bolus (ITB) injection once every 12 weeks through Week 49. The 60-week double-blind treatment period will be followed by the open-label, long-term and extended long-term extension periods, where participants will receive zilganersen by ITB injection from Week 61 to Week 469. |
| Placebo | PLACEBO_COMPARATOR | Matching placebo will be administered by ITB injection once every 12 weeks through Week 49. It will be followed by the open-label, long-term and extended long-term extension periods, where participants will receive zilganersen by ITB injection from Week 61 to Week 469. |
| Name | Type | Description |
|---|---|---|
| zilganersen | DRUG | zilganersen will be administered by ITB injection. |
| Placebo | DRUG | zilganersen-matching placebo will be administered by ITB injection. |
Key Inclusion Criteria: 1. Clinical phenotype and brain imaging consistent with a diagnosis of Alexander disease 2. Documented genetic mutation in the GFAP gene 3. Aged ≥ 2 to 65 years old at the time of informed consent 4. Able and willing to meet all study requirements, including travel to Study ...
Zilganersen is an investigational small molecule antisense oligonucleotide (ASO) being developed for the treatment of Alexander Disease, a rare neurological condition. It is currently in Phase 3 clinical development and has not been approved by the FDA.
Zilganersen is an antisense oligonucleotide (ASO), a class of small molecules that work by targeting specific genetic material to modulate protein production. Its mechanism is designed to address the underlying cause of Alexander Disease, though the specific molecular target is not disclosed.
Zilganersen is being developed by Ionis Pharmaceuticals, Inc. (NASDAQ: IONS). The company is conducting a Phase 3 clinical trial to evaluate the drug's safety and efficacy in patients with Alexander Disease.
Zilganersen is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. The drug has received Priority Review, Fast Track, and Breakthrough Therapy designations from the FDA.
Zilganersen is being studied in a Phase 3 trial (NCT04849741), titled 'A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)'. The trial is active but not recruiting, with an enrollment of 54 participants across multiple countries.
Yes, zilganersen is also known as ION373. The Phase 3 clinical trial NCT04849741 evaluates both names, referring to the drug as 'Zilganersen (ION373)' in its title.