Deramiocel Drug Asset Due Diligence Report 2026

This Deramiocel 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.

NDA/BLA

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 Deramiocel can convert its Stem cell therapy profile and Not disclosed biology into clinically meaningful differentiation while preserving an investable safety, IP and commercial position.

1. Asset identity and development status

Asset	Deramiocel (query alias: Deramiocel)
Modality / target	Stem cell therapy; Not disclosed; Cell replacements
Highest global status	NDA/BLA
Originator	Capricor Therapeutics, Inc.
Active developers	Capricor Therapeutics, Inc.

The MCP disease footprint includes Muscular Dystrophy, Duchenne, Becker Muscular Dystrophy. 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
NCT05126758	Phase 3	Active, not recruiting	106	Change in the upper limb function
NCT04623671	Phase 2	Completed	55	Safety of CAP-1002: Incidence of All-Cause Mortality
NCT04428476	Phase 2	Active, not recruiting	13	Number of Participants With Treatment-Emergent Adverse Events (TEAEs) From Baseline Through Month 12

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

Capricor Therapeutics Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy

Phase 3; n=106; evaluation: Positive. Reported fields: PUL v2.0 = % Slowing of Progression (Deramiocel vs. Placebo) 54%, p=0.029 (n=105) Met; PUL v2.0 = % Slowing of Progression (Deramiocel vs. Placebo) 54%, p=0.029 (n=105) Met

Capricor Therapeutics Announces Positive Data Demonstrating Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular Dystrophy

Phase 2; n=not disclosed; evaluation: Positive. Reported fields: PUL 2.0 total score = -7.19 points ; PUL 2.0 total score = -3.46 points

Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial

Phase 2; n=13; evaluation: not stated. Reported fields: -; -; Number of Participants With Treatment-Emergent Adverse Events (TEAEs) From Baseline Through Month 12 = 11 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

Deramiocel addresses Muscular Dystrophy, Duchenne, Becker Muscular Dystrophy. Commercial attractiveness rests on addressable biomarker-positive patients, treatment-line placement, duration, administration burden, pricing and displacement of entrenched standards. The modality—Stem cell therapy—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
2022-01-24	Capricor Therapeutics and Nippon Shinyaku Enter Partnership for Exclusive Commercialization and Distribution of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy in the U.S.	Phase 2	US$30.0M upfront; US$705.0M milestones

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