Vutrisiran Sodium Drug Asset Due Diligence Report 2026

This Vutrisiran Sodium Drug Asset Due Diligence Report was built with PatSnap Life Sciences MCP workflows. Drug & Asset MCP establishes identity, ownership and stage; Clinical Trials MCP checks design, endpoints and readouts; Company & Deal Intelligence MCP reconstructs transaction precedent. Explore the MCP servers used in this report.

Decision date: 15 July 2026. Currency fields are presented in US$ millions as returned by the deal dataset. This is a screening memorandum, not legal, medical, patent or investment advice.

Approved

Highest phase

Registered trials

Result records

Matched deals

Executive recommendation: GO

Decision memo

Advance diligence: development maturity and available evidence support continued investment, subject to indication-specific safety, IP, and commercial gates.

The central underwriting question is whether Vutrisiran Sodium can convert its siRNA profile and TTR biology into clinically meaningful differentiation while preserving an investable safety, IP and commercial position.

1. Asset identity and development status

Asset	Vutrisiran Sodium (query alias: vutrisiran)
Modality / target	siRNA; TTR; TTR inhibitors, RNAi
Highest global status	Approved
Originator	Alnylam Pharmaceuticals, Inc.
Active developers	Alnylam Pharmaceuticals, Inc., Alnylam Japan KK, Alnylam Netherlands BV

The MCP disease footprint includes Transthyretin Amyloid Cardiomyopathy, Amyloidosis, Hereditary, Transthyretin-Related, Transthyretin-related (ATTR) familial amyloid polyneuropathy. The highest-phase flag is a useful orientation point, but the licensing case depends on indication-level evidence and rights, not the global label alone.

2. Clinical program: design and endpoint audit

Registry	Phase	Status	Enrollment	Lead primary endpoint
NCT06679946	Phase 3	Enrolling by invitation	700	Frequency of Adverse Events (AEs)
NCT04153149	Phase 3	Active, not recruiting	655	Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population
NCT03759379	Phase 3	Completed	164	Change From Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]

The trial set should be diligenced for randomization, comparator relevance, endpoint hierarchy, analysis population, multiplicity, geographic mix and readout timing. For early-stage studies, safety and dose selection can be as decision-critical as response rate.

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3. Clinical readouts: what is known

Vutrisiran-Mediated Knockdown of Transthyretin in Patients with ATTR Amyloidosis

Phase 1/2; n=307; evaluation: Positive. Reported fields: Serum TTR knockdown(Week 3) = 64.2 % ( 61.6 - 67.8); Serum TTR knockdown(Week 3) = 69.0 % ( 66.0 - 72.0)

Vutrisiran in East Asian Patients With Transthyretin Amyloidosis With Cardiomyopathy

Phase 3; n=32; evaluation: Positive. Reported fields: Composite endpoint(all-cause mortality and recurrent cardiovascular events): HR = 0.2(95.0% CI, 0.04 - 0.93); Composite endpoint(all-cause mortality and recurrent cardiovascular events): HR = 0.2(95.0% CI, 0.04 - 0.93)

HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

Phase 3; n=655; evaluation: not stated. Reported fields: Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population = 125 Participants ; Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population = 159 Participants ; Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population: Hazard Ratio (HR) = 0.718(95% CI, 0.555 - 0.929), P-Value = 0.0118

These fields are structured evidence signals, not a substitute for statistical review. The next diligence pass should reconcile denominators, confidence intervals, follow-up, censoring, dose cohorts and treatment-emergent toxicity against the original abstract, registry and protocol.

4. Market and competitive position

Vutrisiran Sodium addresses Transthyretin Amyloid Cardiomyopathy, Amyloidosis, Hereditary, Transthyretin-Related, Transthyretin-related (ATTR) familial amyloid polyneuropathy. Commercial attractiveness rests on addressable biomarker-positive patients, treatment-line placement, duration, administration burden, pricing and displacement of entrenched standards. The modality—siRNA—must demonstrate a benefit large enough to offset class-specific safety and operational costs.

The strongest market-validation signal in this screen is partner behavior: 4 matched transaction record(s) indicate that sophisticated counterparties have assigned strategic value to the asset or its rights. That does not establish net present value; probability of success, remaining R&D spend, royalties, cost sharing and territorial scope still need modeling.

5. Transaction precedent and economics

Date	Transaction	Phase at deal	Disclosed economics
2025-11-04	Royalty Pharma Acquires Royalty Interest in Alnylam’s Amvuttra for $310 Million from Blackstone Life Sciences	Approved	US$310.0M upfront
2022-07-20	ORSINI SPECIALTY PHARMACY SELECTED AS LIMITED DISTRIBUTION PARTNER FOR AMVUTTRA™ (vutrisiran)	Approved	Financial terms not disclosed
2020-04-13	Blackstone and Alnylam Enter Into $2 Billion Strategic Financing Collaboration to Accelerate the Advancement of RNAi Therapeutics	NDA/BLA	US$2,000.0M stated total

Headline values are not directly comparable. Diligence should normalize upfront cash, equity, development and sales milestones, tiered royalties, opt-in mechanics, cost sharing, change-of-control clauses and geography.

6. IP and freedom-to-operate screen

No asset-specific patent-application milestone appeared in the returned milestone slice. That absence is not a freedom-to-operate conclusion; a dedicated family, claim, ownership, expiry, and legal-status search remains mandatory before signing.

The claim chart should separately test composition or sequence coverage, formulation and dosing, indication and biomarker claims, combinations, manufacturing know-how, prosecution history, term extensions and third-party blocking rights. Confirm that licensed patents, data and know-how track every granted territory and field.

7. Principal risks and diligence gates

Class crowding and differentiation versus other PD-(L)1/VEGF agents
Immune and anti-angiogenic safety, including bleeding and cardiovascular risk
Biomarker strategy, indication selection, and head-to-head endpoint execution

Cross-functional diligence should also test CMC comparability, supply chain, pharmacovigilance, regulatory correspondence, data integrity, investigator concentration, partner obligations and change-of-control restrictions.

8. Final go/no-go memorandum

GO