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Nucresiran · 2 trials · 3 indications
The mNIS+7 is a composite score that measures neurologic impairment which includes the following components: physical exam of lower limbs, upper limbs and cranial nerves to assess motor strength/weakness and deep tendon reflexes, electrophysiologic measurement of large nerve fiber function, sensory testing and postural blood pressure. The mNIS+7 is scored from 0 (no impairment) to 304 points (maximum impairment). A higher score indicates a worse outcome.
All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) will be compared between treatment groups using an Andersen-Gill model.
| Arm | Type | Description |
|---|---|---|
| Nucresiran 300 mg | EXPERIMENTAL | Patients will be administered nucresiran 300 mg subcutaneously (SC) once every 6 months (q6M) during the Treatment Period and Treatment Extension Period |
| Vutrisiran 25 mg followed by Nucresiran 300 mg | ACTIVE_COMPARATOR | Patients will be administered vutrisiran 25 mg SC every 3 months (q3M) during the Treatment Period followed by nucresiran 300 mg SC q6M during the Treatment Extension Period |
| Placebo | PLACEBO_COMPARATOR | Participants will receive placebo administered subcutaneously (SC) once every 6 months (q6M) during the double-blind (DB) period, followed by nucresiran 300 mg administered SC q6M during the open-label extension (OLE) period. |
| Name | Type | Description |
|---|---|---|
| Nucresiran | DRUG | Nucresiran 300 mg administered SC q6M |
| Vutrisiran | DRUG | Vutrisiran 25 mg administered SC q3M |
| Sterile Normal Saline (0.9% NaCl) | DRUG | Sterile Normal Saline (0.9% NaCl) administered SC once q6M |
Inclusion Criteria: * Has documented diagnosis of hATTR-PN * Has a diagnosis of hATTR amyloidosis with polyneuropathy with a documented TTR gene variant * Has a neuropathy impairment score (NIS) of 5 to 130 (inclusive) * Has a Karnofsky Performance Status (KPS) of ≥60% Exclusion Criteria: * Has h...
Nucresiran is an investigational small molecule being developed for the treatment of transthyretin amyloidosis with cardiomyopathy and hereditary transthyretin-mediated amyloidosis with polyneuropathy. It is currently in Phase 3 clinical development and is not yet approved by regulatory authorities.
Nucresiran is a small interfering RNA (siRNA) therapeutic. It is designed to target and silence the transthyretin gene, reducing the production of transthyretin protein, which is the underlying cause of transthyretin amyloidosis.
Nucresiran is being developed by Alnylam Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol ALNY. Alnylam is conducting the Phase 3 clinical trials for this investigational drug.
Nucresiran is in Phase 3 clinical development. It is being evaluated in two Phase 3 trials, TRITON-CM and TRITON-PN, which are currently recruiting participants. Nucresiran is an investigational drug and has not received FDA approval.
Nucresiran is being studied in two Phase 3 trials. The TRITON-CM trial (NCT07052903) evaluates the drug in patients with transthyretin amyloidosis with cardiomyopathy, with an enrollment of 1,750 participants. The TRITON-PN trial (NCT07223203) evaluates it in hereditary transthyretin amyloidosis with polyneuropathy, with 125 participants.
Nucresiran is a distinct investigational siRNA therapeutic developed by Alnylam Pharmaceuticals. It is not the same as other approved transthyretin amyloidosis treatments, as it is a novel agent currently in Phase 3 trials and has not yet been approved for any indication.