Patisiran sodium Drug Asset Due Diligence Report 2026: Clinical, IP, Market, Deals, and Go/No-Go
This Patisiran 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
12
Registered trials
27
Result records
8
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 Patisiran 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 | Patisiran sodium (query alias: patisiran) |
|---|---|
| Modality / target | siRNA; TTR; TTR inhibitors, RNAi |
| Highest global status | Approved |
| Originator | Alnylam Pharmaceuticals, Inc. |
| Active developers | Alnylam Netherlands BV, Alnylam Pharmaceuticals, Inc. |
The MCP disease footprint includes Polyneuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Transthyretin Amyloid Cardiomyopathy. 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 |
|---|---|---|---|---|
| NCT03997383 | Phase 3 | Completed | 360 | Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT) |
| NCT05023889 | Early Phase 1 | Completed | 10 | Change in Neurological Impairment Score |
| NCT04201418 | Not Applicable | Completed | 67 | Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline |
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
Patisiran, an RNAi therapeutic for hereditary transthyretin-mediated amyloidosis: Sub-analysis in Taiwanese patients from the APOLLO study
Phase 3; n=18; evaluation: Positive. Reported fields: mNIS+7 = -26.5 points ( -45.5 to -7.5)
A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients With Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Patisiran Clinical Study
Phase 3; n=211; evaluation: not stated. Reported fields: Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation = 49.0 percentage of participants ; Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation = 0.0 percentage of participants ; Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation = 16.8 percentage of participants
Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis
Phase 3; n=360; evaluation: Positive. Reported fields: 6MWD = decline meters ( higher); 6MWD = decline meters ( lower)
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
Patisiran sodium addresses Polyneuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Transthyretin Amyloid Cardiomyopathy. 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: 8 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 |
|---|---|---|---|
| 2020-07-08 | Alnylam Pharmaceuticals and Taiba Group Partner to Commercialize RNAi Therapeutics in the Gulf States | Approved | Financial terms not disclosed |
| 2020-03-25 | Alnylam Pharmaceuticals and Gen Sign Distribution Agreement in Turkey for ONPATTRO® (patisiran), the First-in-Class ‘Gene-Silencing’ RNAi Therapeutic. | Approved | Financial terms not disclosed |
| 2019-07-30 | Alnylam Pharmaceuticals and GENESIS Pharma Partner to Commercialize ONPATTRO® (patisiran) in South East Europe | Approved | Financial terms not disclosed |
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
The milestone feed surfaced a patent-application signal described as “Compositions and methods for treating transthyretin (TTR) mediated amyloidosis”. The milestone feed surfaced a patent-application signal described as “Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated diseases”. The milestone feed surfaced a patent-application signal described as “siRNA Therapy for Transthyretin (TTR) Related Ocular Amyloidosis”.
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
Advance diligence: development maturity and available evidence support continued investment, subject to indication-specific safety, IP, and commercial gates.
Required pre-signing gates: reproduce key efficacy analyses; complete an indication-specific safety review; run a full patent-family and freedom-to-operate search; model risk-adjusted economics by territory; and reconcile all rights, sublicenses and encumbrances.
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Data provenance: PatSnap Drug & Asset MCP, Clinical Trials MCP, and Company & Deal Intelligence MCP; accessed 15 July 2026. Counts and status fields may change as source records update.
