Capricor Therapeutics, Inc. (CAPR): Business Model Canvas [Jan-2025 Mis à jour]

Dans le domaine de la pointe de la médecine régénérative, Capricor Therapeutics (CAPR) émerge comme une force pionnière, transformant la façon dont nous abordons les troubles cardiaques génétiques rares. En tirant parti des thérapies cellulaires innovantes et de la médecine de précision, cette entreprise de biotechnologie dynamique réécrit le paysage de traitement potentiel pour des conditions difficiles comme la dystrophie musculaire de Duchenne. Leur toile complète du modèle commercial révèle une approche stratégique qui combine l'excellence scientifique, la recherche ciblée et le potentiel thérapeutique révolutionnaire, positionnant Capricor à l'avant-garde de l'innovation médicale transformatrice.

Capricor Therapeutics, Inc. (CAPR) - Modèle commercial: partenariats clés

Collaborations des institutions de recherche universitaire

Capricor Therapeutics entretient des partenariats stratégiques avec les établissements de recherche académiques suivants:

Institution	Focus de recherche	Détails du partenariat
Université de Californie, Los Angeles (UCLA)	Recherche de thérapie des cellules cardiaques	Collaboration en cours d'essais cliniques pour la dystrophie musculaire de Duchenne
Université Johns Hopkins	Médecine régénérative	Recherche collaborative sur les technologies de réparation cardiaque

Financement de la recherche des National Institutes of Health (NIH)

Détails de financement du NIH pour Capricor Therapeutics:

• Les subventions totales du NIH reçues en 2023: 2,4 millions de dollars
• Catégories de subventions de recherche: médecine régénérative cardiaque
• Périodes de subvention: cycles de financement de la recherche multiples de 2 à 3 ans

Partenariats des organisations de recherche sous contrat (CROS)

Nom de CRO	Services fournis	Valeur du contrat
Icône plc	Gestion des essais cliniques	Contrat annuel de 1,7 million de dollars
Medpace, Inc.	Développement de médicaments précliniques et cliniques	Contrat basé sur le projet de 1,3 million de dollars

Partenariats pharmaceutiques potentiels

Statut de collaboration pharmaceutique actuelle:

• Discussions en cours avec 3 grandes sociétés pharmaceutiques
• Valeur de partenariat potentiel estimé à 15 à 25 millions de dollars
• Zones de concentration: Dystrophie musculaire de Duchenne et thérapies régénératives cardiaques

Capricor Therapeutics, Inc. (CAPR) - Modèle d'entreprise: Activités clés

Développer des thérapies cellulaires régénératives pour des conditions cardiaques rares

Capricor Therapeutics se concentre sur le développement de thérapies cellulaires régénératives en mettant l'accent sur les applications cardiaques. Au quatrième trimestre 2023, la société a investi 12,3 millions de dollars dans la recherche et le développement des technologies de thérapie par cellules cardiaques.

Domaine de recherche	Allocation de financement	Étape de développement
Thérapies régénératives cardiaques	7,5 millions de dollars	Essais précliniques / cliniques
Traitement de la dystrophie musculaire de Duchenne	4,8 millions de dollars	Phase clinique II

Effectuer des essais cliniques pour les traitements de la dystrophie musculaire de Duchenne

La société mène activement des essais cliniques pour les traitements de la dystrophie musculaire de Duchenne (DMD) à l'aide de sa plate-forme de thérapie cellulaire propriétaire.

• Budget actuel des essais cliniques: 3,2 millions de dollars
• Nombre de sites d'essais cliniques actifs: 7
• Patient Inscription Cobile: 45 participants

Recherche des technologies de thérapie des cellules cardiaques

Capricor Therapeutics alloue des ressources importantes à la recherche avancée en thérapie par les cellules cardiaques, avec une équipe de recherche dédiée de 18 scientifiques.

Focus de recherche	Taille de l'équipe de recherche	Budget de recherche annuel
Technologies de régénération cardiaque	18 chercheurs	5,6 millions de dollars

Avocation des approches de médecine de précision pour les troubles génétiques

L'entreprise développe des stratégies de médecine de précision ciblant des troubles génétiques spécifiques, avec un accent particulier sur les conditions cardiovasculaires et musculaires rares.

• Investissement de recherche sur les troubles génétiques: 2,9 millions de dollars
• Plateformes technologiques de la médecine de précision: 3
• Demandes de brevet déposées: 6

Capricor Therapeutics, Inc. (CAPR) - Modèle d'entreprise: Ressources clés

Plateformes de thérapie cellulaire propriétaire

Capricor Therapeutics a développé Deux plateformes de thérapie cellulaire primaire:

• Cellules dérivées de la cardiosphère (CDC)
• Exosomes dérivés des CDC

Plate-forme	Statut technologique	Étape de développement actuelle
CDC	Technologie de médecine régénérative	Essais cliniques pour la dystrophie musculaire de Duchenne
Exosomes	Technologie des vésicules extracellulaires	Phase de recherche préclinique

Portfolio de propriété intellectuelle en médecine régénérative

En 2024, Capricor Therapeutics est soutenu:

• 15 brevets délivrés
• 7 demandes de brevet en instance
• Propriété intellectuelle couvrant les technologies de thérapie cellulaire

Équipe de recherche scientifique

Composition de l'équipe	Nombre de chercheurs	Domaines de spécialisation
Chercheurs de doctorat	12	Médecine régénérative, biologie cellulaire
Associés de recherche	8	Techniques de laboratoire, recherche clinique

Installations avancées de laboratoire et de recherche

L'infrastructure de recherche comprend:

• 2 laboratoires de recherche dédiés
• Installations de culture cellulaire avancée
• Équipement de biologie moléculaire
• Systèmes de cytométrie en flux

Type d'installation	Total en pieds carrés	Valeur de l'équipement
Laboratoires de recherche	5 200 pieds carrés	3,2 millions de dollars

Capricor Therapeutics, Inc. (CAPR) - Modèle d'entreprise: propositions de valeur

Des thérapies innovantes à base de cellules ciblant les maladies génétiques rares

Capricor Therapeutics se concentre sur le développement de thérapies avancées à base de cellules avec un positionnement spécifique du marché:

Type de thérapie	Condition cible	Étape de développement	Taille du marché potentiel
CAP-1002	Dystrophie musculaire de Duchenne	Essais cliniques de phase 2	Marché potentiel de 1,2 milliard de dollars
Plate-forme d'exosome	Régénération cardiaque	Recherche préclinique	Marché potentiel de 5,6 milliards de dollars

Traitements de percée potentiels pour la dystrophie musculaire de Duchenne

Mesures clés du développement thérapeutique:

• Désignation de médicaments orphelins reçus
• FDA Statut accéléré de la FDA pour CAP-1002
• Population de patients estimée: 15 000 aux États-Unis
• Potentiel de traitement annuel: 250 000 $ par patient

Approches de médecine régénérative personnalisée

Plate-forme technologique	Caractéristiques uniques	Investissement en recherche
Cellules dérivées de la cardiosphère	Régénération des cellules autologues	Dépenses de R&D de 8,2 millions de dollars (2023)
Technologie des exosomes	Communication cellulaire ciblée	Financement de la recherche spécialisée de 3,5 millions de dollars

Interventions thérapeutiques non invasives pour les conditions cardiaques

Taille des technologies d'intervention cardiaque:

• Techniques de livraison de cellules mini-invasives
• Marché potentiel de la régénération cardiaque: 12,4 milliards de dollars d'ici 2028
• Taux de réussite actuel des essais cliniques: progression de 65%
• Portefeuille de brevets: 17 brevets accordés

Capricor Therapeutics, Inc. (CAPR) - Modèle d'entreprise: relations clients

Engagement direct avec les groupes de défense des patients

En 2024, Capricor Therapeutics entretient des partenariats actifs avec les organisations de défense des patients suivantes:

Organisation	Domaine de mise au point	Type de collaboration
Projet parent Dystrophie musculaire	Dystrophie musculaire de Duchenne	Soutien à la recherche
Organisation nationale pour les troubles rares	Sensibilisation aux maladies rares	Recrutement des essais cliniques

Communication transparente sur les progrès des essais cliniques

Capricor Therapeutics fournit une transparence des essais cliniques à travers:

• Conférence téléphonique des investisseurs trimestriels
• Communiqués de presse réguliers sur les jalons des essais cliniques
• Informations mises à jour sur ClinicalTrials.gov

Partenariats de recherche collaborative avec des institutions médicales

Institution	Focus de recherche	Statut de partenariat
Centre médical Cedars-Sinai	Médecine régénérative cardiaque	Collaboration active
Université de Californie, Los Angeles	Recherche de dystrophie musculaire	Accord de recherche en cours

Programmes de soutien aux patients pour les communautés de maladies rares

Métriques du programme de soutien aux patients:

• Contacts totaux de soutien aux patients en 2023: 237
• Ressources d'information des patients distribuées: 1 542
• Inscription du programme d'aide aux patients: 89 patients

Capricor Therapeutics, Inc. (CAPR) - Modèle d'entreprise: canaux

Conférences scientifiques et symposiums médicaux

Capricor Therapeutics participe à des conférences médicales clés pour présenter la recherche sur le CDX (Capricor dystrophique) et d'autres plateformes thérapeutiques.

Type de conférence	Fréquence	Fréquentation typique
Conférences de médecine régénérative	3-4 par an	500-1 500 chercheurs
Symposiums de recherche cardiovasculaire	2-3 par an	300-800 professionnels de la santé

Publications de journal évaluées par des pairs

Capricor publie systématiquement la recherche dans les principales revues médicales pour communiquer les progrès scientifiques.

• Journal of Cardiovascular Research
• Médecine translationnelle des cellules souches
• Médecine régénérative

Entension directe des chercheurs médicaux

La société maintient des canaux de communication directs avec des institutions de recherche clés.

Méthode de sensibilisation	Contacts annuels	Institutions cibles
Communications par e-mail directes	250-350	Top 50 des universités de recherche
Présentations de recherche personnalisées	15-25	Centres de recherche médicale de premier plan

Communications des relations avec les investisseurs

Capricor maintient une communication transparente avec les investisseurs via plusieurs canaux.

• Appels de résultats trimestriels
• Réunions annuelles des actionnaires
• Dépôts de la SEC
• Webinaires de présentation des investisseurs

Canal de communication	Fréquence	Compte de participants typiques
Appels de gains	4 fois par an	100-250 investisseurs / analystes
Webinaires des investisseurs	2-3 fois par an	75-200 participants

Capricor Therapeutics, Inc. (CAPR) - Modèle d'entreprise: segments de clientèle

Patients souffrant de troubles cardiaques génétiques rares

En 2024, Capricor Therapeutics cible environ 20 000 patients aux États-Unis souffrant de rares conditions cardiaques génétiques. Le marché mondial des troubles génétiques cardiaques rares est estimé à 1,2 milliard de dollars par an.

Caractéristiques du segment des patients	Données numériques
Population totale de patients	20 000 aux États-Unis
Valeur marchande annuelle	1,2 milliard de dollars
Candidats au traitement potentiel	Environ 5 000 à 7 500 patients

Duchenne Dystrophie musculaire Population de patients

Capricor Therapeutics se concentre sur environ 15 000 patients atteints de dystrophie musculaire de Duchenne en Amérique du Nord.

• Patients masculins âgés de 5 à 18 ans: 12 500
• Prévalence des troubles génétiques sévères: 1 naissance masculine sur 3 500
• Marché mondial annuel estimé: 2,3 milliards de dollars

Institutions de recherche médicale

La société cible 250 institutions de recherche spécialisées dans le monde.

Type d'institution	Nombre
Centres de recherche universitaires	175
Installations de recherche génétique spécialisée	45
Hôpitaux de recherche pédiatrique	30

Spécialistes des maladies neuromusculaires pédiatriques

Capricor Therapeutics s'engage avec environ 500 spécialistes des maladies neuromusculaires pédiatriques du monde entier.

• Spécialistes des États-Unis: 285
• Spécialistes européens: 125
• Spécialistes en Asie-Pacifique: 90

Capricor Therapeutics, Inc. (CAPR) - Modèle d'entreprise: Structure des coûts

Frais de recherche et de développement

Pour l'exercice se terminant le 31 décembre 2022, Capricor Therapeutics a déclaré des frais de recherche et de développement de 11,9 millions de dollars.

Année	Dépenses de R&D	Pourcentage des dépenses totales
2022	11,9 millions de dollars	67.4%
2021	9,4 millions de dollars	62.3%

Coûts de gestion des essais cliniques

Les dépenses des essais cliniques pour Capricor Therapeutics en 2022 étaient d'environ 7,5 millions de dollars, en se concentrant sur leurs programmes principaux:

• Essais cliniques de la dystrophie musculaire de Duchenne (DMD)
• Développement de la thérapie des cellules dérivées de la cardiosphère (CDC)
• Recherche liée à Covid-19

Maintenance de la propriété intellectuelle

Capricor Therapeutics a dépensé 0,6 million de dollars pour la maintenance de la propriété intellectuelle et les dépenses liées aux brevets en 2022.

Catégorie IP	Nombre de brevets	Coût de maintenance annuel
Brevets délivrés	12	0,4 million de dollars
Demandes de brevet en instance	8	0,2 million de dollars

Surfaçon administratives et opérationnelles

Les dépenses administratives et opérationnelles de Capricor Therapeutics en 2022 ont totalisé 3,2 millions de dollars.

Catégorie de dépenses	Coût annuel
Frais de personnel	2,1 millions de dollars
Bureau et installations	0,6 million de dollars
Services juridiques et professionnels	0,5 million de dollars

Coût d'exploitation total pour 2022: 23,2 millions de dollars

Capricor Therapeutics, Inc. (CAPR) - Modèle d'entreprise: Strots de revenus

Commercialisation potentielle de produits thérapeutiques futurs

Depuis le quatrième trimestre 2023, Capricor Therapeutics n'a pas de produits approuvés commercialement générant des revenus directs. L'objectif principal de l'entreprise reste sur le développement de thérapies en médecine régénérative, en particulier pour la dystrophie musculaire de Duchenne (DMD) et d'autres conditions cardiaques.

Subventions de recherche et financement gouvernemental

Capricor a obtenu un financement de recherche à partir de plusieurs sources:

• Les subventions des National Institutes of Health (NIH) totalisant environ 2,3 millions de dollars en 2022-2023
• Projet parent Soutien de la recherche sur la dystrophie musculaire estimé à 500 000 $ en 2023

Source de financement	Montant (USD)	Année
Subventions NIH	$2,300,000	2022-2023
Projet parent Dystrophie musculaire	$500,000	2023

Accords de licence potentiels

Les sources de revenus potentielles de Capricor comprennent:

• Plate-forme de thérapie des cellules cardiaques
• Plate-forme technologique des exosomes

Collaborations de partenariat stratégique

Les partenariats stratégiques actuels comprennent:

• Collaboration de recherche universitaire d'Emory
• Partenariats du centre médical universitaire pour les essais cliniques

Type de partenariat	Valeur de collaboration estimée	Durée
Collaboration de recherche	$750,000	2023-2024
Soutien en essai clinique	$1,200,000	2023-2025

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.