Capricor Therapeutics, Inc. (CAPR): Business Model Canvas [Dec-2025 Updated]

You're digging into Capricor Therapeutics, Inc. (CAPR) right now, likely trying to map out how they turn that recent HOPE-3 data into actual revenue, especially after raising capital. Honestly, the business model is at a pivot point: they're burning cash-operating expenses hit $79.0 million through the first nine months of 2025-but they've got a $98.6 million war chest as of Q3 2025 to push the Deramiocel Biologics License Application (BLA) resubmission. The real prize is the potential $80 million milestone payment from their partner, Nippon Shinyaku, upon approval, which hinges on their ability to execute on manufacturing and navigate the FDA; see below for the full nine-block breakdown of how they plan to capture value from their first-in-class therapy for Duchenne muscular dystrophy (DMD) patients and the underlying StealthX™ exosome platform.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Key Partnerships

You're looking at the structure of Capricor Therapeutics, Inc. (CAPR) partnerships as of late 2025. These alliances are critical for getting Deramiocel to market and advancing the StealthX platform.

Nippon Shinyaku (NS Pharma) for US/Japan commercialization and distribution

Capricor Therapeutics has an Exclusive Commercialization and Distribution Agreement with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.) for Deramiocel in the United States and Japan, contingent on regulatory approval. This deal structure was designed to fund the HOPE-3 trial without diluting shareholders.

The financial terms associated with this partnership include:

• Initial upfront payment: $30 million.
• Potential additional milestone payments: up to $705 million.
• Capricor's share of product revenue: a meaningful, double-digit share.
• Projected U.S. sales share for Capricor by 2030: approximately $332 million.

Capricor Therapeutics reported that the upfront payment and the first development milestone payment from Nippon Shinyaku totaled $40.0 million, and the second development milestone payment was $10.0 million; both were fully recognized as revenue by December 31, 2024. As of December 2025, Capricor and NS Pharma are advancing launch readiness activities.

National Institute of Allergy and Infectious Diseases (NIAID) for StealthX™ Phase 1 trial

The Phase 1 clinical trial for the StealthX™ exosome-based vaccine is being conducted and funded by NIAID under the U.S. Department of Health and Human Services' Project NextGen. The Investigational New Drug (IND) application was cleared by the FDA, and dosing of the first subject began in August 2025.

The key timeline metric for this partnership is:

Milestone	Expected Timing
Initial data from StealthX Phase 1 trial	First quarter of 2026

The trial initially focuses on the SARS-CoV-2 spike protein and includes four dosing arms.

Academic institutions like Cedars-Sinai for research and clinical development

Capricor Therapeutics maintains a foundational relationship with Cedars-Sinai Medical Center (CSMC) stemming from an Exclusive License Agreement for intellectual property (IP) related to exosomes originating from cardiosphere-derived cells (CDCs).

Key historical and structural points include:

• Original License Agreement date: January 4, 2010.
• Amendments to the agreement: twice in 2013.
• The licensed technology is based on preclinical research led by Eduardo Marbán, M.D., Ph.D., who serves as Director of the Cedars-Sinai Heart Institute and Co-founder and Scientific Advisory Board Chairman of Capricor.

The company also lists Johns Hopkins University and the California Institute for Regenerative Medicine (CIRM) among its supportive collaborations.

Contract Manufacturing Organizations (CMOs) for supply chain scalability

Capricor Therapeutics is focused on establishing a manufacturing framework for its exosome platform, which is essential for supply chain scalability for future products like the StealthX vaccine.

Data presented at the AAEV 2025 meeting demonstrated a framework for loading therapeutic small interfering RNAs (siRNA) and phosphorodiamidate morpholino oligomers (PMO) into exosomes.

• Loading efficiencies from scale-up and scale-out strategies were comparable to standard small-volume electroporation.
• The integrated approach allows for manufacturing of significantly larger batches of therapeutic exosomes.

Patient advocacy groups for Duchenne muscular dystrophy (DMD) community outreach

Capricor Therapeutics actively engages with the DMD community through key advocacy groups. The HOPE-2 Open-Label Extension (OLE) study data was featured at the Parent Project Muscular Dystrophy (PPMD) 2025 Annual Conference on June 21, 2025.

The involvement is quantified by the presence of PPMD leadership, such as Founding President and CEO Pat Furlong, commenting on the clinical data.

The partnership with Nippon Shinyaku is also noted for its established infrastructure to support DMD patients and caregivers.

Finance: review Q4 2025 cash position against the projected runway into late 2026 by end of January.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Key Activities

You're looking at the core engine driving Capricor Therapeutics right now-the set of actions they absolutely must nail to move from clinical-stage to commercial-stage. It's all about executing on Deramiocel while simultaneously building out the next generation of their platform.

Executing Biologics License Application (BLA) resubmission for Deramiocel

The primary focus here is navigating the regulatory path after the July 2025 Complete Response Letter (CRL) from the FDA. Capricor Therapeutics plans to resubmit the BLA, leveraging the data from the pivotal HOPE-3 trial to address the CRL's concerns regarding the statutory requirement for substantial evidence of effectiveness. The FDA indicated that the HOPE-3 results can be submitted within the current BLA framework. The company expects this resubmission to be reviewed under a Type 2 classification, which carries an anticipated review period of up to six months. The initial BLA submission was supported by data from the HOPE-2 trial and its ongoing Open-Label Extension (OLE), which has continued for over 48 months.

Conducting pivotal Phase 3 clinical trials (HOPE-3 follow-up)

The HOPE-3 trial was the make-or-break activity for the BLA resubmission. This randomized, double-blind, placebo-controlled study evaluated Deramiocel in a cohort of 106 boys and young men with Duchenne muscular dystrophy (DMD). The trial was powered to measure both skeletal and cardiac function. The topline results, announced on December 3, 2025, were positive, showing statistically significant improvements. Here's the quick math on the key outcomes:

Endpoint Measure	Result vs. Placebo	Statistical Significance
Skeletal Muscle Progression (PUL v2.0)	54% slowing	p=0.029
Cardiac Function (LVEF)	91% slowing of decline	p=0.041

What this estimate hides is the baseline difference; the average baseline Left Ventricular Ejection Fraction (LVEF) for the overall trial population was 57%.

Manufacturing and quality control of allogeneic cell therapy

Capricor Therapeutics has been busy ensuring its production capabilities align with regulatory expectations. The San Diego Good Manufacturing Practice (GMP) facility, which produces the commercial-scale version of Deramiocel, successfully passed its pre-license inspection (PLI). Furthermore, all Chemistry, Manufacturing, and Controls (CMC) observations noted in the July 2025 CRL have been resolved and accepted by the FDA. This operational readiness is key for a cell therapy product. For context on the investment in this area, Research and Development (R&D) expenses specifically for the Duchenne muscular dystrophy program (Deramiocel) in fiscal year 2024 totaled $23,049,349, which included expanded manufacturing production efforts.

• Resolved all 483 observations from the Pre-License Inspection.
• Facility is operational and capable of supporting initial commercial launch.
• Published peer-reviewed data in October 2025 reinforcing Deramiocel's mechanism of action.
• Facility expenses for 2024 were $2,759,096.

Advancing the StealthX™ exosome platform pipeline development

While Deramiocel is the near-term priority, advancing the StealthX™ exosome platform is crucial for long-term value. The company is leveraging its proprietary technology for vaccinology and targeted delivery of various therapeutics. This platform entered the clinic in 2025.

• The FDA cleared the Investigational New Drug (IND) application for the StealthX™ exosome-based vaccine.
• The National Institute of Allergy and Infectious Diseases (NIAID) initiated a Phase 1 clinical trial in August 2025.
• Initial topline data from the NIAID-sponsored Phase 1 trial is currently expected in the first quarter of 2026.
• Presented data in November 2025 demonstrating a scalable framework for loading therapeutic oligonucleotides into exosomes.
• Exosomes platform research expenses in 2024 were $2,908,678.

Preparing for commercial launch of Deramiocel in 2026

Capricor Therapeutics is actively gearing up for a potential market introduction in 2026, contingent on regulatory success. This involves financial planning to bridge the gap until potential revenue hits. As of September 30, 2025, the company's cash balance was approximately $99 million (or $98.6 million), which management guided is expected to support planned operations into the fourth quarter of 2026. The company has an exclusive commercialization agreement in the U.S. and Japan with Nippon Shinyaku Co., Ltd. (NS Pharma, Inc.).

The financial reality of this preparation is reflected in the operating expenses. Total operating expenses for the third quarter of 2025 were approximately $26.3 million, resulting in a net loss of $24.6 million (or $0.54 per share). If approved before September 30, 2026, Capricor would qualify for a Priority Review Voucher (PRV). Also, the company remains eligible for an $80 million milestone payment from NS Pharma upon approval.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Key Resources

You're looking at the core assets Capricor Therapeutics, Inc. needs to execute its strategy right now. These aren't just ideas; they are tangible, validated, and financial components that define the company's current operational capacity.

The most immediate resource is the capital base. As of September 30, 2025, Capricor Therapeutics, Inc. reported cash, cash equivalents, and marketable securities totaling approximately $98.6 million. This balance is projected to cover anticipated expenses and capital requirements into the fourth quarter of 2026.

The intellectual property and clinical data surrounding Deramiocel (CAP-1002) form the primary value driver. Deramiocel is an allogeneic cardiosphere-derived cell (CDC) therapy designed to preserve cardiac and skeletal muscle function in Duchenne muscular dystrophy (DMD). The pivotal HOPE-3 Phase 3 clinical trial, which involved 105 participants, has completed its 12-month treatment phase, with topline results expected in the fourth quarter of 2025. The study randomized 106 participants who received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for a 12-month period, with an average participant age of approximately 15 years. Furthermore, the HOPE-3 study met both its primary endpoint, Performance of Upper Limb (PUL v2.0), achieving statistical significance at p=0.03, and the key secondary cardiac endpoint, Left Ventricular Ejection Fraction (LVEF), at p=0.04. A peer-reviewed paper detailing Deramiocel's anti-fibrotic and immunomodulatory mechanisms was published in Biomedicines in October 2025.

The proprietary StealthX™ exosome technology platform is a key platform asset, extending beyond Deramiocel. This platform is proprietary for designer protein surface expression, cargo loading, and targeted delivery, currently being explored in vaccinology and the targeted delivery of oligonucleotides, proteins, and small-molecule therapeutics. The platform is being advanced via a National Institute of Allergy and Infectious Diseases (NIAID)-sponsored Phase 1 clinical trial for an exosome-based vaccine, with initial data anticipated in the first quarter of 2026. The company presented data at AAEV 2025 demonstrating a scalable framework for loading therapeutic small interfering RNAs (siRNA) and phosphorodiamidate morpholino oligomers (PMO) into exosomes.

Commercial readiness is supported by physical infrastructure and regulatory recognition. Capricor Therapeutics, Inc.'s GMP facility in San Diego successfully completed its FDA Pre-License Inspection (PLI), with all 483 observations addressed and accepted, making the facility operational to support an initial commercial launch upon approval. The company also holds significant regulatory advantages for Deramiocel:

• Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S.
• Orphan Drug Designation from the U.S. FDA and the European Medicines Agency (EMA) for DMD treatment.
• Rare Pediatric Disease Designation from the FDA.

The commercial partnership with Nippon Shinyaku provides a potential non-dilutive capital event upon approval.

Financial/Operational Metric	Value/Status (as of late 2025)
Cash, Cash Equivalents, Marketable Securities (Q3 2025)	$98.6 million
Projected Cash Runway	Into Q4 2026
Potential Milestone Payment (NS Pharma)	$80 million upon U.S. approval
HOPE-3 Trial Participants	105 randomized participants
HOPE-3 Primary Endpoint PUL v2.0 Significance	p=0.03
HOPE-3 Secondary Endpoint LVEF Significance	p=0.04
StealthX™ Phase 1 Data Expectation	Q1 2026

Finance: review the Q4 2025 cash burn projection against the current runway by next Tuesday.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Value Propositions

You're looking at the core promises Capricor Therapeutics, Inc. is making to the market with Deramiocel, their investigational cell therapy for Duchenne Muscular Dystrophy (DMD). These aren't just abstract goals; they are backed by recent, hard data from their pivotal Phase 3 HOPE-3 study, which is key for any financial model you're building.

First-in-class therapy to specifically target DMD-associated cardiomyopathy is the central promise. Cardiomyopathy is the leading cause of mortality in DMD patients, so hitting this specific, fatal aspect of the disease is a massive value driver. Deramiocel is an allogeneic cardiac-derived cell therapy, meaning it's an off-the-shelf product, which significantly simplifies administration compared to autologous (patient-specific) treatments.

The clinical data from the HOPE-3 trial, which involved n=106 participants, provides the statistical muscle behind these claims. Here is a breakdown of the key efficacy results after the 12-month double-blind period:

Value Proposition Metric	Statistical Outcome (HOPE-3 Trial)	Statistical Significance
Slowing of Skeletal Muscle Disease Progression (Primary Endpoint: PUL v2.0)	54% slowing of decline	p=0.029
Slowing of Cardiac Function Decline (Key Secondary Endpoint: LVEF)	91% slowing of decline	p=0.041
Overall Trial Population (ITT) LVEF Endpoint	Met with p=0.04	Statistically Significant

The therapy is designed to preserve cardiac function, measured by Left Ventricular Ejection Fraction (LVEF). The trial demonstrated a 91 percent slowing of decline in LVEF, achieving statistical significance at p=0.041. This directly addresses the most critical survival factor in DMD. Furthermore, the primary endpoint, upper-limb function measured by the Performance of the Upper Limb (PUL v2.0) score, showed a 54% slowing of disease progression, with a p-value of 0.029. Honestly, achieving significance on both skeletal and cardiac endpoints in a single pivotal study is what positions this therapy so strongly.

The treatment mechanism involves allogeneic cardiosphere-derived cells (CDCs) that secrete exosomes to modulate immune responses and reduce fibrosis, supporting the potential for a disease-modifying treatment in this rare, life-limiting condition. The fact that Capricor Therapeutics, Inc. has maintained a cash position of approximately $98.6 million as of September 30, 2025, expected to last into the fourth quarter of 2026, shows they have the runway to pursue the planned Biologics License Application (BLA) resubmission leveraging these data.

The core value propositions can be summarized by the functional and logistical benefits:

• First-in-class therapy targeting Duchenne cardiomyopathy.
• 54% slowing of upper-limb function decline (PUL v2.0).
• 91% slowing of LVEF decline.
• Allogeneic cell therapy, simplifying administration.
• Potential to be a disease-modifying treatment.
• Company cash runway extends into Q4 2026.

The therapy's profile is further supported by prior data showing continued improvement in LVEF in the open-label extension of the HOPE-2 trial. The commitment to this development is reflected in the Q3 2025 operating expenses of approximately $26.3 million, though Q3 revenue was $0. You need to see these numbers as the cost of securing this unique value proposition.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Customer Relationships

You're preparing for a potential product launch in a rare disease space, so the relationship with the patient community and the medical experts who treat them is everything. For Capricor Therapeutics, Inc., this means a very focused, high-touch approach, especially given the late-stage clinical data for Deramiocel in Duchenne muscular dystrophy (DMD).

High-touch support for rare disease patients and caregivers

Capricor Therapeutics, Inc. builds relationships by showing deep respect for the commitment patients make to clinical research. The company is incredibly grateful to the patients and families who chose to participate in their research. The pivotal HOPE-3 Phase 3 clinical trial evaluated Deramiocel in $\mathbf{106}$ participants across $\mathbf{20}$ leading U.S. clinical sites.

The trial design itself reflects a commitment to this patient group, many of whom are facing severe progression. At the start of the trial, the average age of participants was approximately $\mathbf{15}$ years, and $\mathbf{90\%}$ were already on cardiac medications, with over $\mathbf{75\%}$ having clinical cardiomyopathy. Participants received $\mathbf{150}$ million cells per infusion intravenously every $\mathbf{three}$ months for a $\mathbf{12}$-month period.

The positive topline results announced on December $\mathbf{3}$, $\mathbf{2025}$, directly impact this relationship, showing a $\mathbf{54\%}$ slowing of skeletal muscle disease progression and a $\mathbf{91\%}$ slowing of decline in left ventricular ejection fraction (LVEF). Furthermore, across all programs, Capricor Therapeutics has administered more than $\mathbf{800}$ infusions to approximately $\mathbf{150}$ DMD patients with Deramiocel, demonstrating a consistent safety profile.

Direct engagement with key opinion leaders (KOLs) and specialized cardiologists

Engagement with the medical community is centered on presenting robust data from the late-stage program. The company has worked with experts across the industry to ensure clinical trial design and execution prioritize patient safety and wellbeing. The recent success of the HOPE-3 trial, which met its primary endpoint ($p=\mathbf{0.029}$) and key secondary endpoint ($p=\mathbf{0.041}$), is the primary driver for KOL interaction.

The relationship with the medical community is also supported by the fact that Capricor Therapeutics has received several key regulatory designations for Deramiocel, including Orphan Drug Designation from both the U.S. FDA and the EMA, plus Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S..

Here's a quick look at the clinical context that drives KOL discussions:

Metric	Value/Result	Endpoint Type
Skeletal Muscle Progression Slowing	54%	Primary (PUL v2.0)
Cardiac Function Decline Slowing	91%	Key Secondary (LVEF)
HOPE-3 Trial Site Count	20 U.S. Centers	Trial Scope
Total Deramiocel Infusions Administered (All Trials)	Over 800	Safety/Experience

Investor relations focused on clinical milestones and regulatory progress

Investor relations communication is tightly linked to achieving critical regulatory and clinical milestones, especially following the Complete Response Letter (CRL) received earlier in $\mathbf{2025}$. The company held a Type A meeting with the FDA in August $\mathbf{2025}$ to align on using the HOPE-3 results to address the CRL issues. The plan is to resubmit the Biologics License Application (BLA) leveraging the data, with review anticipated under a Type $\mathbf{2}$ classification.

Financial stability is a key relationship point for investors. As of the third quarter ended September $\mathbf{30}$, $\mathbf{2025}$, Capricor Therapeutics had a cash balance of approximately $\mathbf{\$99}$ million, which management expects will support planned operations into the fourth quarter of $\mathbf{2026}$. This is crucial as total operating expenses for Q3 $\mathbf{2025}$ were approximately $\mathbf{\$26.3}$ million, resulting in a net loss of $\mathbf{\$24.6}$ million for the quarter. The company reported $\mathbf{\$0}$ revenue for Q3 $\mathbf{2025}$.

Key milestones driving investor sentiment include:

• Topline results from the pivotal HOPE-3 Phase 3 study expected in Q4 $\mathbf{2025}$.
• Planned BLA resubmission to address the $\mathbf{2025}$ CRL.
• Commercial launch preparations underway for Deramiocel market introduction in $\mathbf{2026}$.
• FDA clearance of the IND for the StealthX™ exosome-based vaccine, with initial topline data expected in Q1 $\mathbf{2026}$ from the NIAID-sponsored Phase $\mathbf{1}$ trial.
• Successful resolution of all $\mathbf{483}$ observations noted in the FDA Pre-License Inspection, confirming readiness for commercial manufacturing.

Scientific publications to build credibility with the medical community

Building credibility with the medical community relies on peer-reviewed validation of the science behind Deramiocel. The company published a peer-reviewed paper in Biomedicines in October $\mathbf{2025}$ detailing Deramiocel's anti-fibrotic and immunomodulatory mechanisms through exosome release. This publication reinforces the mechanism of action that CDCs (cardiosphere-derived cells) exert through secreted exosomes.

The depth of scientific support is substantial, as CDCs have been investigated in more than $\mathbf{250}$ peer-reviewed scientific publications. The company is also leveraging its exosome technology with the proprietary StealthX™ platform in preclinical development.

The scientific data presented to the community includes:

• Mechanism of Action: Anti-fibrotic and immunomodulatory effects detailed in Biomedicines.
• Prior Data: Durable improvements shown in the HOPE-$\mathbf{2}$ open-label extension, which continued for over $\mathbf{48}$ months.
• Platform Progress: New data demonstrating a scalable framework for loading therapeutic oligonucleotides into exosomes presented at AAEV $\mathbf{2025}$.

Finance: draft $\mathbf{13}$-week cash view by Friday.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Channels

You're looking at how Capricor Therapeutics, Inc. gets its product, Deramiocel, to the patient, which is all tied up in regulatory success right now. The channels are heavily weighted toward partnership agreements for commercialization, given the rare disease focus.

Exclusive distribution network through partner Nippon Shinyaku/NS Pharma

Capricor Therapeutics, Inc. has established a clear path for commercialization through its existing agreements with Nippon Shinyaku Co., Ltd. and its U.S. subsidiary, NS Pharma, Inc. This structure dictates the distribution for the U.S. and Japan, contingent on regulatory approval for Deramiocel (CAP-1002).

Here's a breakdown of the key commercialization and financial terms associated with these distribution channels:

Territory/Agreement	Partner Entity	Role	Key Financial/Term Data
United States & Japan	Nippon Shinyaku Co., Ltd. / NS Pharma, Inc.	Exclusive Distribution/Commercialization (Post-US Approval)	Agreements entered January 2022 (US) and February 2023 (Japan)
Europe (EU, UK, etc.)	Nippon Shinyaku Co., Ltd.	Commercialization and Distribution (Subject to Definitive Agreement)	Binding Term Sheet signed; includes $20 Million Upfront Payment and up to $715 Million in Potential Milestones
Global Potential Milestones	Nippon Shinyaku	Combined Milestones	Total potential milestones from combined distribution agreements approximate $1.5 Billion
European Revenue Share	Nippon Shinyaku	Commercialization	Capricor to receive a double-digit percentage of product revenue

The company is also preparing for a potential influx of capital to support these future commercial activities, having announced a proposed public offering in December 2025, with gross proceeds expected to be $150 Million. The cash position as of Q2 2025 was $122.8 Million, which the company expected to fund operations into Q4 2026.

Specialized treatment centers and hospitals for cell therapy administration

As Deramiocel is an allogeneic cardiosphere-derived cell therapy, its administration channel requires specialized infrastructure. Post-approval, distribution will be limited to facilities capable of handling complex cell therapy logistics, likely a network of specialized centers treating Duchenne Muscular Dystrophy (DMD) cardiomyopathy patients.

• Cell therapy administration requires specialized handling protocols.
• Target patient population is rare, necessitating focused centers.
• The HOPE-3 trial enrolled 105 participants.

Direct sales force targeting rare disease specialists post-approval

While the primary distribution in the U.S. is through NS Pharma, Inc. post-approval, Capricor Therapeutics, Inc. will still need a focused internal team to support the launch and drive adoption among key opinion leaders and rare disease specialists.

• Talent retention is a focus, with stockholders approving an Equity Incentive Plan reserving 3,500,000 shares for awards in 2025.
• Projected annual revenue for 2025 was estimated at $827MM and projected non-GAAP EPS at 0.19.
• The company reported a last twelve month free cash flow outflow of about $63.7 Million as of late 2025.

Regulatory pathways (FDA Type 2 BLA resubmission) for market access

Market access is entirely dependent on the successful resubmission of the Biologics License Application (BLA) following the Complete Response Letter (CRL) received in July 2025.

• The FDA issued the CRL in July 2025, citing insufficient evidence of effectiveness.
• Capricor plans to resubmit the BLA in Q3 2025, incorporating data from the Phase 3 HOPE-3 trial.
• The original Prescription Drug User Fee Act (PDUFA) target action date was August 31, 2025.
• The FDA agreed to review HOPE-3 data within the current BLA framework.
• HOPE-3 primary endpoint: Performance of the Upper Limb version 2.0 (PUL v2.0).
• HOPE-3 key secondary endpoint: Left Ventricular Ejection Fraction (LVEF).
• HOPE-3 results showed a 54% slowing of skeletal muscle disease progression (P = .029) and a 91% slowing of cardiac function decline versus placebo.

Finance: draft cash flow projection for Q1 2026 based on December 2025 capital raise by next Tuesday.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Customer Segments

You're looking at the key groups Capricor Therapeutics, Inc. targets with its lead candidate, Deramiocel, and its exosome platform as of late 2025. Here is the hard data on those segments.

Duchenne muscular dystrophy (DMD) patients with cardiomyopathy represent the core patient population for Deramiocel. The Phase 3 HOPE-3 study evaluated 106 boys and young men with DMD, with 78% of that cohort presenting with cardiomyopathy at baseline. The clinical benefit observed included a 91% slowing of decline in left ventricular ejection fraction (LVEF). The total US DMD patient population is estimated around 15,000 individuals, with Capricor Therapeutics targeting an addressable population of 1,500-2,000 US DMD patients for Deramiocel. The overall DMD market is valued around $6.5B.

Metric	Value/Statistic	Context/Source Data
HOPE-3 Trial Enrollment (n)	106	Total participants in the pivotal Phase 3 study
Baseline Cardiomyopathy Prevalence (HOPE-3)	78%	Percentage of HOPE-3 participants with cardiomyopathy at baseline
LVEF Decline Slowing (Key Secondary Endpoint)	91%	Statistical significance achieved (p=0.041) in HOPE-3
Estimated US DMD Patient Population	Approximately 15,000	Total individuals affected in the United States
Targeted Addressable Patient Population (US)	1,500-2,000	Estimated segment for Deramiocel
DMD Market Valuation	$6.5B	Targeted market size

For specialists, the focus is on those treating the cardiac complications, where 78% of the HOPE-3 cohort had cardiomyopathy. In a natural history study of DMD patients, 69.9% in the $\ge$ 20 year age group showed LVEF $<$ 55%.

Global pharmaceutical companies seeking exosome technology licenses are a key partnership segment. Capricor Therapeutics has an agreement with Nippon Shinyaku Co., Ltd. for exclusive commercialization and distribution of Deramiocel in the United States and Japan. Under this deal, Capricor is eligible for 30-50% of U.S. revenues upon launch. The company recognized revenue from milestone payments, with the upfront and first development milestones totaling $40.0 million. The company reported $0 revenue in Q3 2025, down from $2.3 million in Q3 2024.

• Exclusive worldwide license agreement signed with Johns Hopkins University for engineered exosomes.
• The StealthX™ platform is being developed for vaccinology and targeted delivery of oligonucleotides, proteins, and small molecule therapeutics.

Government and academic research institutions (e.g., NIAID, DoD) represent a segment for platform technology validation and non-DMD pipeline development. The cash balance as of September 30, 2025, was approximately $98.6 million, intended to support operations into the fourth quarter of 2026.

• NIAID is sponsoring a Phase 1 clinical trial using the StealthX™ exosome-based vaccine.
• Initial topline data from the NIAID-sponsored trial is currently expected in the first quarter of 2026.
• The StealthX™ IND was cleared under Project NextGen, a U.S. Department of Health and Human Services initiative.

For context on the operating environment, the net loss for the third quarter of 2025 was approximately $24.6 million.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Cost Structure

The Cost Structure for Capricor Therapeutics, Inc. is heavily weighted toward the high-stakes, high-cost activities inherent in late-stage clinical development and preparing for commercial launch. You see this reflected in the significant increase in operating expenses year-over-year.

The primary cost drivers are centered on advancing Deramiocel through its pivotal Phase 3 trial and ensuring the infrastructure is ready for a potential 2026 market introduction. This is typical for a pre-revenue biotechnology firm focused on a rare disease indication.

Here's the quick math on the major expense categories for the first nine months of 2025:

Cost Component (9 Months Ended Sept 30, 2025)	Amount (Approximate)
Total Operating Expenses	$79.0 million
Research and Development (R&D) Expenses (Excluding Stock-Based Comp)	$54.4 million
General and Administrative (G&A) Expenses (Excluding Stock-Based Comp)	$11.1 million
Net Loss	$74.9 million

High Research and Development (R&D) expenses for late-stage trials represent the largest single cost bucket. This spending is almost entirely dedicated to the execution and analysis of the HOPE-3 Phase 3 clinical trial for Deramiocel in Duchenne muscular dystrophy (DMD). The HOPE-3 study involved 106 participants across 20 leading U.S. clinical sites, with patients receiving treatment over a 12-month period.

Clinical trial execution costs for HOPE-3 and other programs are the core of the R&D spend. This includes site management, patient monitoring, data collection, and statistical analysis required to meet the standards for a Biologics License Application (BLA) resubmission. The company is also funding the early-stage NIAID-sponsored Phase 1 clinical trial for its StealthX™ exosome-based vaccine platform, adding to the R&D burden.

Manufacturing scale-up and commercial readiness costs are a significant, non-clinical expense. Capricor Therapeutics has invested heavily to ensure its facility is ready, having completed a successful FDA Pre-License Inspection. These costs cover quality control, process validation, and inventory build-up ahead of a potential 2026 launch.

General and administrative (G&A) costs for regulatory compliance and corporate overhead are necessary to support the entire operation. For the first three quarters of 2025, these expenses, excluding stock-based compensation, totaled approximately $11.1 million. This covers essential functions like:

• Regulatory affairs personnel for BLA resubmission strategy
• Legal and compliance overhead
• Executive and corporate infrastructure support
• Investor relations activities

The overall financial position reflects these investments; as of September 30, 2025, Capricor Therapeutics held approximately $98.6 million in cash, cash equivalents, and marketable securities, which management guided was sufficient to cover anticipated expenses into the fourth quarter of 2026.

Finance: draft 13-week cash view by Friday.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of Capricor Therapeutics, Inc. (CAPR) as of late 2025, which is heavily weighted toward non-operational, financing, and future potential income sources right now. Honestly, the current revenue picture reflects a company deep in development mode.

Current revenue for Capricor Therapeutics, Inc. (CAPR) is reported as $0 for the third quarter of 2025, and for the first nine months of 2025, revenue was also $0. This compares to approximately $11.1 million recognized in the first nine months of 2024, which was from the prior recognition of upfront and development milestone payments from Nippon Shinyaku that were fully recognized as of December 31, 2024.

The primary expected revenue driver is future product sales of Deramiocel following regulatory approval, which the company is preparing for a commercial launch in 2026. Analysts suggest that if approved, Deramiocel peak sales could exceed $1 billion.

Here's a quick look at the key financial components that make up the current and near-term revenue potential:

Revenue/Funding Source	Amount/Status	Context
Q3 2025 Revenue	$0	Reported for the quarter ended September 30, 2025.
First Nine Months 2025 Revenue	$0	Reported revenue for the period ended September 30, 2025.
NS Pharma Approval Milestone	Up to $80 million	Anticipated payment from NS Pharma upon FDA approval of Deramiocel.
Recent Equity Financing Gross Proceeds	$150 million	Expected gross proceeds from the public offering priced on December 5, 2025.
Historical NIH Grant Funding (Total)	Over $30 million	Total awarded from government agencies to date to support cell and exosome candidates.

Potential milestone payments are critical for extending the company's runway until product sales begin. The partnership with NS Pharma for Deramiocel commercialization in the U.S. and Japan includes an anticipated $80 million payment contingent upon FDA approval. This milestone, along with the potential sale of a Priority Review Voucher (PRV) worth approximately $150 million, could materially extend the cash runway if approval occurs before September 30, 2026.

To bolster its balance sheet ahead of the anticipated launch, Capricor Therapeutics, Inc. (CAPR) recently executed a significant equity financing event. The company announced the pricing of an underwritten public offering on December 5, 2025, which is expected to generate gross proceeds of $150 million before fees, based on the sale of 6,000,000 common shares at $25.00 per share. The net proceeds are intended for continued product development, manufacturing, working capital, and general corporate purposes.

Research grants and collaboration funding provide non-dilutive capital to advance pipeline assets outside of the lead product. For instance, Capricor Therapeutics, Inc. (CAPR) has a collaboration with the National Institute of Allergy and Infectious Diseases (NIAID) where NIAID will conduct and fully fund a Phase 1 clinical trial for its StealthX™ exosome-based multivalent vaccine for SARS-CoV-2 prevention. Also, historically, the company was awarded up to $4.2 million from the National Institutes of Health (NIH) to evaluate exosomes for Hypoplastic Left Heart Syndrome (HLHS).

• Future Deramiocel sales potential estimated to exceed $1 billion in peak sales.
• Potential Priority Review Voucher (PRV) sale value estimated around $150 million.
• The recent equity offering involved 6,000,000 shares at $25.00 per share.
• Underwriters were granted an option to purchase up to an additional 900,000 shares.
• Cash and marketable securities stood at approximately $98.6 million as of September 30, 2025.

Finance: review the cash runway projection based on the $150 million gross proceeds by end of next week.