Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Eplontersen, AKCEA-TTR-LRx, IONIS-TTR-LRx
ION-682884 · 4 trials · 4 indications
An adverse event (AE) is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not the AE was considered related to the medicinal (investigational) product. An AE was to be regarded as a TEAE if it was present prior to receiving the first dose of study drug and subsequently worsened or was not present prior to receiving the first dose of study drug but subsequently appeared.
A concomitant therapy was any non-protocol-specified drug or substance (including over-the-counter \[OTC\] medications, herbal medications, and vitamin supplements) administered between signing of informed consent and the last study visit.
Laboratory parameters included measurement of blood chemistry, hematology, coagulation, complement, or urinalysis parameters for the single-dose and multiple-dose cohorts. Number of participants with clinically significant values in laboratory based on Investigator's assessment are reported. Any value outside the normal range was to be flagged for the attention of the investigator was to assess whether or not a flagged value is of clinical significance.
Physical examination included measurement of height and weight for body mass index (BMI) determination. Number of participants with clinically significant findings in physical examination based on Investigator's assessment are reported. Any value outside the normal range was to be flagged for the attention of the investigator was to assess whether or not a flagged value is of clinical significance.
ECG measurements included assessment of ventricular rate (VR), PR interval, QR interval, QT interval, QT corrected using Fridericia's formula (QTcF), and QT corrected using Bazett's formula (QTcB). Number of participants with clinically significant values in electrocardiogram based on Investigator's assessment are reported. Any value outside the normal range was to be flagged for the attention of the investigator was to assess whether or not a flagged value is of clinical significance.
| Arm | Type | Description |
|---|---|---|
| Eplontersen | EXPERIMENTAL | Eplontersen will be administered once every month by sub-cutaneous (SC) injection for up to 36 months or 6 months after eplontersen is approved and available in the site's country, whichever occurs first. |
| Placebo | PLACEBO_COMPARATOR | Eplontersen-matching placebo by subcutaneous injection once every 4 weeks |
| Multiple Dose Cohort: Placebo | PLACEBO_COMPARATOR | Participants received ION-682884 matching placebo, subcutaneously (SC) once every 4 weeks \[Q4W\] (total of 4 doses) along with daily oral supplemental doses of the recommended daily allowance (RDA) of vitamin A during the 13-week Treatment Period. |
| Multiple Dose Cohort A: ION-682884 45 mg | EXPERIMENTAL | Participants received ION-682884, 45 milligrams (mg), SC, Q4W (total of 4 doses) along with daily oral supplemental doses of the RDA of vitamin A during the 13-week Treatment Period. |
| Multiple Dose Cohort E: ION-682884 60 mg | EXPERIMENTAL | Participants received ION-682884, 60 mg, SC, Q4W (total of 4 doses) along with daily oral supplemental doses of the RDA of vitamin A during the 13-week Treatment Period. |
| Multiple Dose Cohort B: ION-682884 90 mg | EXPERIMENTAL | Participants received ION-682884, 90 mg, SC, Q4W (total of 4 doses) along with daily oral supplemental doses of the RDA of vitamin A during the 13-week Treatment Period. |
| Single Dose Cohort: Placebo | PLACEBO_COMPARATOR | Participants received single dose of ION-682884 matching placebo, SC along with daily oral supplemental doses of the RDA of vitamin A on Day 1. |
| Single Dose Cohort C: ION-682884 120 mg | EXPERIMENTAL | Participants received single dose of ION-682884, 120 mg, SC along with daily oral supplemental doses of the RDA of vitamin A on Day 1. |
| Name | Type | Description |
|---|---|---|
| Eplontersen | DRUG | Eplontersen will be administered by SC injection. |
| Placebo | DRUG | Eplontersen-matching placebo by subcutaneous injection |
| ION-682884 | DRUG | ION-682884 administered SC |
| Vitamin A | DIETARY_SUPPLEMENT | Oral supplement |
Inclusion Criteria: 1. Satisfactory completion of Treatment Period and the End of Treatment Visit of the Index Study (ION-682884-CS2) OR diagnosis of ATTR-CM and satisfactory participation on ISIS 420915- CS101 study as judged by the Investigator and Sponsor. 2. Investigator is willing to treat the...
ION-682884 is being developed for hereditary transthyretin-mediated amyloidosis, also called hATTR amyloidosis, a rare disease in which misfolded transthyretin protein deposits in tissues. It is an investigational therapy and has been studied in healthy volunteers as well as patients with hATTR amyloidosis.
ION-682884 is being developed by Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker IONS. Ionis is the sponsor of the clinical program evaluating the drug in hATTR amyloidosis and in healthy volunteers.
ION-682884 is in Phase 1 clinical development. It is an investigational drug and is not approved for any indication. One Phase 1 trial has been completed, and no active trials of ION-682884 are currently listed.
ION-682884 was evaluated in the Phase 1 trial NCT03728634, titled a study of single and multiple subcutaneous doses of eplontersen in healthy volunteers and patients with hereditary transthyretin-mediated amyloidosis. The trial was completed, enrolled 47 participants, was randomized, double-blind, and placebo-controlled, and was conducted in Canada.
Yes. ION-682884 is also known as eplontersen, the name used in its clinical trial NCT03728634. People searching for either name are looking at the same investigational compound being developed by Ionis Pharmaceuticals for hATTR amyloidosis.
The completed Phase 1 trial NCT03728634 was a randomized, double-blind, placebo-controlled study of single and multiple subcutaneous doses of ION-682884. It enrolled 47 participants, including healthy volunteers and patients with hATTR amyloidosis, at sites in Canada, and participants were 18 years or older.