Nedosiran Drug Asset Due Diligence Report 2026

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

1. Asset identity and development status

Asset	Nedosiran (query alias: Nedosiran)
Modality / target	siRNA; LDHA; LDHA inhibitors, RNAi
Highest global status	Approved
Originator	Novo Nordisk, Inc.
Active developers	Dicerna Pharmaceuticals, Inc., Novo Nordisk, Inc.

The MCP disease footprint includes Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary hyperoxaluria type III. 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
NCT04580420	Phase 2	Recruiting	28	Safety: Incidence of Events
NCT05001269	Phase 2	Completed	27	Percent Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups
LBCTR2021104866	Phase 2	Pending	5	Not disclosed

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

A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients From Birth to 11Years of Age With Primary Hyperoxaluria and Relatively Intact Renal Function

Phase 2; n=27; evaluation: not stated. Reported fields: PH1(Mean) = -68.34 Percent change (Standard Deviation, 8.574); PH1(Mean) = -74.06 Percent change (Standard Deviation, 13.193); -

Nedosiran in Pediatric Patients with PH1 (PHYOX8)

Phase 2; n=15; evaluation: Positive. Reported fields: 1.5 x ULN(spot Uox:cr ratios) = 11.0 Pts

PHYOX3: Long-Term Safety and Efficacy of Nedosiran in Patients with Primary Hyperoxaluria Type 1

Phase 3; n=40; evaluation: Positive. Reported fields: Annualized stone event rate = 0.2 Event/year

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

Nedosiran addresses Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary hyperoxaluria type III. 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: 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
2020-04-03	Alnylam and Dicerna Form RNAi Therapeutics Collaboration on Alpha-1 Antitrypsin Deficiency-Associated Liver Disease and Complete Cross-License Agreement for Primary Hyperoxaluria Programs	Phase 3	US$180.0M 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 “Methods and compositions for inhibiting expression of ldha”.

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