Drugs / Nucresiran
last change Dec 2025 re-read 3 minutes ago

Nucresiran

Developed for
familial amyloid neuropathy
Investigated by
Alnylam Pharmaceuticals

Trials 2

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 3 NCT07223203 Dec 2025 → Dec 2027 expected familial amyloid neuropathy Alnylam Pharmaceuticals Recruiting No outcome recorded
Phase 3 NCT07052903 Jul 2025 → May 2030 expected — Alnylam Pharmaceuticals Recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 4

DateIssuerRelease
2025-07-31 Alnylam Pharmaceuticals Regulatory Alnylam Pharmaceuticals Reports Second Quarter 2025 Financial Results and Highlights Recent Period Progress alnylam.com ↗
Alnylam announces today that nucresiran has received Fast Track Designation from the FDA's Division of Cardiology and Nephrology for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin mediated amyloidosis in adults to reduce mortality, cardiovascular hospitalizations, and urgent heart failure visits.
2024-11-17 Alnylam Pharmaceuticals Results Alnylam Announces Interim Phase 1 Data of Nucresiran (ALN-TTRsc04) Showing Rapid Knockdown of TTR that is Sustained at Six Months Following a Single Dose alnylam.com ↗
At these doses, peak reduction of mean TTR levels of greater than 96% were achieved by Day 29.
2023-12-13 Alnylam Pharmaceuticals Results Alnylam Highlights Significant Progress with Platform Innovation and Clinical Pipeline at R&D Day alnylam.com ↗
In the Phase 1 study in healthy volunteers, single 300 mg doses of ALN-TTRsc04 achieved rapid knockdown with a mean reduction of greater than 90% at Day 15.
2021-09-29 Alnylam Pharmaceuticals Results Alnylam Continues Scientific Leadership in Advancement of RNAi Therapeutics at the 17th Annual Meeting of the Oligonucleotides Therapeutics Society alnylam.com ↗
The IKARIA-derived ALN-TTRsc04 molecule also demonstrated comparable potency and durability with an analog of vutrisiran in NHP studies.

Evidence & citations 2 cited values

Every value below carries the sentence it was read from. 2 sources stand behind the page.

FieldValueCited text
Known as Nucresiran ClinicalTrials.gov intervention name — accepted as the source's own label NCT07052903 ↗
1

NCT07223203 ↗

Route Subcutaneous “Nucresiran 300 mg administered SC q6M” NCT07052903 ↗