Idursulfase Drug Asset Due Diligence Report 2026

This Idursulfase 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 Idursulfase can convert its Enzyme profile and IDS biology into clinically meaningful differentiation while preserving an investable safety, IP and commercial position.

1. Asset identity and development status

Asset	Idursulfase (query alias: idursulfase)
Modality / target	Enzyme; IDS; IDS modulators
Highest global status	Approved
Originator	Shire Development LLC
Active developers	Sanofi-Aventis Korea Co., Ltd., Sanofi, Takeda Pharmaceuticals U.S.A., Inc.

The MCP disease footprint includes Mucopolysaccharidosis II, Cognitive Dysfunction. 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
NCT05494593	Phase 4	Withdrawn	Not disclosed	Rate of Anti-Idursulfase Antibodies Formation, Including Anti-Idursulfase Antibodies That Have Enzyme Neutralizing Activity
NCT05371613	Phase 2/3	Recruiting	63	Percent change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A)
NCT06031259	Phase 2/3	Active, not recruiting	6	Number of Participants with Adverse Events (AEs) by Type and Severity

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

An Open-Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Pediatric Patients With Hunter Syndrome and Cognitive Impairment

Phase 1/2; n=15; evaluation: not stated. Reported fields: -; Number of Participants With Treatment-emergent Adverse Events (TEAEs) = 4 Participants ; Number of Participants With Treatment-emergent Adverse Events (TEAEs) = 10 Participants

An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction With Elaprase® in Patients With Hunter Syndrome and Cognitive Impairment

Phase 2/3; n=56; evaluation: not stated. Reported fields: -; -; -

A Prospective, Multicenter, Single-arm, Open-label, Interventional Phase IV Study to Evaluate the Safety and Efficacy of Idursulfase (r-DNA Origin) (Elaprase™) in Indian Pediatric and Adult Population With Hunter Syndrome (Mucopolysaccharidosis II)

Phase 4; n=5; evaluation: not stated. Reported fields: TEAE = 5 Participants ; -; -

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

Idursulfase addresses Mucopolysaccharidosis II, Cognitive Dysfunction. Commercial attractiveness rests on addressable biomarker-positive patients, treatment-line placement, duration, administration burden, pricing and displacement of entrenched standards. The modality—Enzyme—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: 1 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
2005-04-21	Shire Pharmaceuticals Group plc To Build On Specialty Portfolio Through Acquisition Of Transkaryotic Therapies, Inc. For $1.6 Billion	Phase 1/2	US$1,600.0M 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

The milestone feed surfaced a patent-application signal described as “Hybrid system of culture and purification process for manufacturing antibody pharmaceuticals”. The milestone feed surfaced a patent-application signal described as “Nanostructure lipid system”.

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