Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
IONIS MAPTRx · 1 trial · 1 indication
An adverse event (AE) was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of medicinal (investigational) product, whether or not the AE was considered related to the medicinal (investigational) product. A TEAE was defined as any AE that starts or worsens on or after the date of first dose of study treatment. TEAEs were categorised as mild, moderate, and severe to aid in severity assessment.
An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of medicinal (investigational) product, whether or not the AE was considered related to the medicinal (investigational) product. A TEAE was defined as any AE that starts or worsens on or after the date of first dose of study treatment. TEAEs were categorised as mild, moderate, and severe to aid in severity assessment.
| Arm | Type | Description |
|---|---|---|
| Part 1: Cohort A: ISIS 814907 10 mg | EXPERIMENTAL | Participants received 10 milligrams (mg) ISIS 814907 diluted in 20 milliliters (mL) artificial cerebrospinal fluid (CSF), intrathecally, every four weeks (Q4W) on Days 1, 29, 57, and 85 in Part 1 of the study. |
| Part 1: Cohort B: ISIS 814907 30 mg | EXPERIMENTAL | Participants received 30 mg ISIS 814907 diluted in 20 mL in artificial CSF, intrathecally, Q4W on Days 1, 29, 57, and 85 in Part 1 of the study. |
| Part 1: Cohort C: ISIS 814907 60 mg | EXPERIMENTAL | Participants received 60 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, Q4W on Days 1, 29, 57, and 85 in Part 1 of the study. |
| Part 1: Cohort D: ISIS 814907 115 mg | EXPERIMENTAL | Participants received 115 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, every 12 weeks (Q12W) on Days 1 and 85 in Part 1 of the study. |
| Part 1: Pooled Placebo | PLACEBO_COMPARATOR | Participants received 20 mL artificial CSF, intrathecally, as placebo on Days 1, 29, 57, and 85 for the 4-dose regimens, or on Days 1 and 85 for the 2-dose regimens in Part 1 of the study. |
| Part 2: Late Start Cohort A + Cohort B + Cohort C + ISIS 814907 60 mg | EXPERIMENTAL | Participants from MAD Cohorts A, B, C that were placebo-treated, received 60 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, Q12W on Days 1, 85, 169, 253, and 337 in Part 2 of the study. |
| Part 2: Late Start Cohort D + ISIS 814907 115 mg | EXPERIMENTAL | Participants from MAD Cohort D that were placebo-treated, received 115 mg ISIS 814907 diluted up in 20 mL artificial CSF, intrathecally, Q12W on Days 1, 85, 169, 253, and 337 in Part 2 of the study. |
| Part 2: Early Start Cohort A + ISIS 814907 60 mg | EXPERIMENTAL | Participants from MAD Cohort A that were ISIS 814907 10 mg -treated, received 60 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, Q12W on Days 1, 85, 169, 253, and 337 in Part 2 of the study. |
| Part 2: Early Start Cohort B + ISIS 814907 60 mg | EXPERIMENTAL | Participants from MAD Cohort B that were ISIS 814907 30 mg-treated, received 60 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, Q12W on Days 1, 85, 169, 253, and 337 in Part 2 of the study. |
| Part 2: Early Start Cohort C + ISIS 814907 60 mg | EXPERIMENTAL | Participants from MAD Cohort C that were ISIS 814907 60 mg-treated, received 60 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, Q12W on Days 1, 85, 169, 253, and 337 in Part 2 of the study. |
| Part 2: Early Start Cohort D + ISIS 814907 115 mg | EXPERIMENTAL | Participants from MAD Cohort D that were ISIS 814907 115 mg-treated, received 115 mg ISIS 814907 diluted in 20 mL artificial CSF, intrathecally, Q12W on Days 1, 85, 169, 253, and 337 in Part 2 of the study. |
| Name | Type | Description |
|---|---|---|
| IONIS MAPTRx | DRUG | IONIS MAPTRx injections. |
| Placebo | OTHER | Artificial CSF injections. |
Inclusion Criteria for Part 1: * Males or females aged 50-74 years, inclusive, at the time of informed consent * Diagnosed with mild Alzheimers disease, including CSF biomarkers consistent with this diagnosis * Body Mass Index BMI ≥ 18 and ≤ 35 kg/m2 and total body weight \> 50 kg (110 lbs) * Able ...
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IONIS MAPTRx is an investigational drug being studied for the treatment of mild Alzheimer's disease. It is a small molecule developed by Ionis Pharmaceuticals, Inc. (IONS). The drug is currently in Phase 1 clinical development, meaning it is still being tested in clinical trials and is not yet approved for use.
IONIS MAPTRx is designed to target the production of tau protein, which is associated with Alzheimer's disease pathology. By reducing tau protein levels, the drug aims to address a key component of the disease process. This mechanism is being evaluated in patients with mild Alzheimer's disease.
IONIS MAPTRx is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company is conducting clinical research to evaluate the safety and efficacy of this investigational drug for mild Alzheimer's disease.
IONIS MAPTRx is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities. The Phase 1 trial has been completed, and the drug is still in the early stages of clinical testing for mild Alzheimer's disease.
IONIS MAPTRx has been studied in one completed Phase 1 clinical trial, identified as NCT03186989. This trial evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug in 46 patients with mild Alzheimer's disease. The study was randomized, double-blind, and placebo-controlled, and included participants aged 50 years and older.
Yes, IONIS MAPTRx is the same drug as IONIS-MAPTRx. The name is sometimes written with a hyphen, but both refer to the same investigational small molecule being developed by Ionis Pharmaceuticals for mild Alzheimer's disease. The clinical trial NCT03186989 uses the name IONIS-MAPTRx.