Fitusiran Drug Asset Due Diligence Report 2026

This Fitusiran 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 Fitusiran can convert its siRNA profile and AT III biology into clinically meaningful differentiation while preserving an investable safety, IP and commercial position.

1. Asset identity and development status

Asset	Fitusiran (query alias: Fitusiran)
Modality / target	siRNA; AT III; AT III inhibitors, RNAi
Highest global status	Approved
Originator	Alnylam Pharmaceuticals, Inc.
Active developers	Genzyme Corp., Sanofi, Sanofi-Aventis Recherche & Développement SA

The MCP disease footprint includes Hemophilia A, Hemophilia B, Hemophilia. 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
NCT06145373	Phase 4	Recruiting	20	Number of participants with Adverse events (AEs) during the fitusiran treatment
NCT05662319	Phase 3	Active, not recruiting	91	Annualized bleeding rate (ABR) in the fitusiran primary efficacy period
NCT07285460	Phase 3	Recruiting	85	Annualized treated bleeding rate (ABR) in the fitusiran primary efficacy period and in the SOC period

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

Experience with minor surgeries in people with hemophilia A or B with and without inhibitors receiving fitusiran

Phase 2/3; n=71; evaluation: Positive. Reported fields: ATIII concentrate = 99.0 %

FDA-Approved Drugs-QFITLIA-CLINICAL STUDIES

Phase 3; n=not disclosed; evaluation: Positive. Reported fields: ABR(All Treated Bleeds) = 31.4 Unit (95%CI, 20.5 - 48.2); ABR(All Treated Bleeds) = 19.1 Unit (95%CI, 11.8 - 31.0); ABR(All Treated Bleeds) = 5.1 Unit (95%CI, 2.8 - 9.5)

Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management in people with hemophilia A or B

Phase 2; n=34; evaluation: Positive. Reported fields: ABR = 0.70 Unit ; ABR = 0.87 Unit

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

Fitusiran addresses Hemophilia A, Hemophilia B, Hemophilia. 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: 2 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
2025-04-14	Soleo Health Selected as Exclusive In-Network Specialty Pharmacy for Qfitlia™ a Treatment Option for Adults With Hemophilia A and B With and Without Inhibitors	Approved	Financial terms not disclosed
2012-10-22	Alnylam and Genzyme Form Alliance to Develop and Commercialize RNAi Therapeutics in Asia	Not disclosed	US$22.5M upfront

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 “Treatment of factor x deficiency with fitusiran”. The milestone feed surfaced a patent-application signal described as “Treatment of hemophilia with fitusiran”. The milestone feed surfaced a patent-application signal described as “Treatment of hemophilia a with fitusiran”.

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