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Label indication WAINUA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. WAINUA is a transthyretin-directed antisense oligonucleotide indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. ( 1 ) FDA label
Also known as ION-682884, IONIS-TTR-LRx, AKCEA-TTR-LRx
Eplontersen · 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...
Eplontersen is an investigational RNA therapy being developed for hereditary transthyretin-mediated amyloid polyneuropathy and transthyretin-mediated amyloid cardiomyopathy (ATTR CM). It is designed to reduce production of the transthyretin protein that causes amyloid deposits in nerves and the heart.
Eplontersen targets transthyretin (TTR) mRNA. It is an antisense inhibitor that binds TTR mRNA and reduces the production of TTR protein, the precursor to amyloid deposits in hereditary ATTR polyneuropathy and ATTR cardiomyopathy.
Eplontersen is being developed by Ionis Pharmaceuticals, Inc. (ticker: IONS). The company is running Phase 3 clinical trials in hereditary transthyretin-mediated amyloid polyneuropathy and transthyretin-mediated amyloid cardiomyopathy.
Eplontersen is in Phase 3 clinical development. It is investigational and has not been approved by the FDA for any indication. Two Phase 3 trials are listed, one completed and one active, with a total enrollment of 2,838 participants across the program.
Eplontersen has been studied in Phase 3 trials including NCT04136171 (CARDIO-TTRansform) in ATTR cardiomyopathy, which is completed, and NCT05071300 in hereditary ATTR polyneuropathy, which is active but not recruiting. An extension study, NCT05667493, is enrolling by invitation in ATTR cardiomyopathy.
Yes. Eplontersen was formerly known as ION-682884, IONIS-TTR-LRx, and AKCEA-TTR-LRx. These are all previous names for the same investigational antisense medicine targeting transthyretin, now developed under the name eplontersen by Ionis Pharmaceuticals.