Scholar Rock Holding Corporation (SRRK): Business Model Canvas [Jan-2025 Mise à jour]

Dans le monde de pointe de la biotechnologie, Scholar Rock Holding Corporation (SRRK) émerge comme une force pionnière, transformant le paysage du traitement des maladies neuromusculaires rares par la recherche thérapeutique protéique révolutionnaire. En tirant parti des plates-formes scientifiques innovantes et en ciblant des interactions protéiques spécifiques, cette entreprise de biotechnologie dynamique ne développe pas seulement des médicaments, mais l'élaboration de lignes de vie potentielles pour les patients confrontés à des défis neurologiques complexes. Leur modèle commercial unique représente une approche sophistiquée pour répondre aux besoins médicaux non satisfaits, mélangeant des capacités de recherche avancées avec des partenariats stratégiques qui pourraient révolutionner la médecine personnalisée dans le domaine des interventions de maladies rares.

Scholar Rock Holding Corporation (SRRK) - Modèle d'entreprise: partenariats clés

Collaboration avec les établissements de recherche universitaires

Scholar Rock a établi des partenariats avec les établissements de recherche académiques suivants:

Institution	Domaine de mise au point	Détails de collaboration
École de médecine de Harvard	Maladies neuromusculaires rares	Collaboration de recherche sur le développement thérapeutique de la SMA
Hôpital général du Massachusetts	Troubles génétiques	Partenariat de recherche clinique pour les mécanismes de maladies rares

Partenariats pharmaceutiques stratégiques

Les collaborations pharmaceutiques clés comprennent:

• Miserrer & CO.: Collaboration pour TGF-β Therapeutics
• Novartis: Partenariat pour la recherche sur les maladies neuromusculaires

Relations d'agence de recherche gouvernementale

Sources de financement et relations de subvention:

Agence	Montant d'octroi	Focus de recherche
National Institutes of Health (NIH)	4,2 millions de dollars	Développement thérapeutique des maladies rares
Ministère de la Défense	1,8 million de dollars	Recherche de maladies neuromusculaires

Alliances du centre de recherche en biotechnologie

Partenariats de recherche en biotechnologie:

• Broad Institute of MIT et Harvard
• Stanford Neuroscience Institute
• Centre médical de l'Université Columbia

Capital de capital-risque et partenariats d'investissement

Partenariats d'investissement à partir de 2024:

Investisseur	Montant d'investissement	Année d'investissement
Conseillers orbimés	45 millions de dollars	2023
Gestion de la fidélité & Recherche	32 millions de dollars	2023

Scholar Rock Holding Corporation (SRRK) - Modèle d'entreprise: Activités clés

Recherche et développement de nouvelles thérapies protéiques

Scholar Rock se concentre sur le développement de la thérapie protéique ciblant la signalisation TGF-bêta. Au quatrième trimestre 2023, la société avait investi 98,3 millions de dollars dans les dépenses de R&D.

Investissement en R&D	Exercice 2023
Total des dépenses de R&D	98,3 millions de dollars
Personnel de R&D	78 chercheurs dévoués

Découverte de médicaments à stade préclinique et clinique

La société maintient plusieurs candidats en médicaments à divers stades de développement.

• Essais cliniques en cours pour les traitements de la dystrophie musculaire
• Recherche préclinique en thérapeutique des maladies neuromusculaires
• Développement actif de SRK-015 pour l'atrophie musculaire spinale

Effectuer des essais cliniques ciblés pour les maladies neuromusculaires

Essai clinique	Statut	Phase
SRK-015 (atrophie musculaire spinale)	En cours	Phase 2
Programme de dystrophie musculaire	Préclinique	Enquête

Développer des plateformes technologiques propriétaires SRK

Scholar Rock a développé plates-formes d'ingénierie protéique propriétaire axé sur les mécanismes d'activation du TGF-bêta.

• Technologie sélective d'activation TGF-Beta
• Capacités d'ingénierie des protéines de précision
• Plateforme de découverte de biologie avancée

Processus de soumission et de conformité réglementaires

Activité réglementaire	Détails
Interactions de la FDA	7 réunions officielles en 2023
Applications d'enquête sur le médicament (IND)	2 soumissions actives
Équipe de conformité réglementaire	12 professionnels dévoués

Scholar Rock Holding Corporation (SRRK) - Modèle d'entreprise: Ressources clés

Équipe de recherche scientifique spécialisée

Au quatrième trimestre 2023, Scholar Rock a employé 139 employés au total, avec environ 85% dédiés à la recherche et au développement.

Catégorie des employés	Nombre	Pourcentage
Recherche & Personnel de développement	118	85%
Personnel administratif	21	15%

Plateformes de technologie biologique propriétaire

Scholar Rock a développé Plateforme d'inhibition de signalisation TGF-β avec un accent spécifique sur les maladies neuromusculaires.

Portefeuille de propriété intellectuelle

En décembre 2023, Scholar Rock détient:

• 18 brevets délivrés
• 37 demandes de brevet en instance
• Couverture mondiale des brevets à travers les États-Unis, l'Europe et l'Asie

Infrastructure de laboratoire avancée

Installation de recherche	Emplacement	Taille
Siège social	Cambridge, Massachusetts	28 000 pieds carrés.

Capital financier

Ressources financières au quatrième trimestre 2023:

• Equivalents en espèces et en espèces: 283,4 millions de dollars
• Actif total: 372,6 millions de dollars
• Financement public recueilli par NASDAQ: 456,2 millions de dollars depuis l'introduction en bourse

Scholar Rock Holding Corporation (SRRK) - Modèle d'entreprise: propositions de valeur

Approches thérapeutiques innovantes pour les maladies neuromusculaires rares

Scholar Rock se concentre sur le développement de thérapies innovantes ciblant les maladies neuromusculaires rares, se concentrant spécifiquement sur Atrophie musculaire spinale (SMA) et Chordome.

Focus de la maladie	Étape de développement actuelle	Cible de la population de patients
Atrophie musculaire spinale	Phase d'essai clinique	Patients pédiatriques et adultes
Chordome	Recherche préclinique	Patients cancéreux rares

Traitements de percée potentiels ciblant les interactions protéiques spécifiques

La plate-forme propriétaire de Scholar Rock se concentre sur Activation de la protéine TGF-β, avec des candidats thérapeutiques clés:

• Apitegromab (SRK-015) pour la faiblesse du muscle SMA
• SRK-181 pour l'immunothérapie contre le cancer
• SRK-280 ciblant les tumeurs solides

Mécanismes de ciblage biologique avancés

Plate-forme technologique	Mécanisme unique	Impact clinique potentiel
Activation de la protéine de précision	Activation sélective du TGF-β	Intervention thérapeutique ciblée

Approche de médecine personnalisée pour des conditions neurologiques complexes

Investissement de R&D de Scholar Rock dans la thérapeutique personnalisée:

• Frais de R&D de 129,7 millions de dollars en 2022
• Axé sur les stratégies de médecine de précision
• Développement de protocoles de traitement spécifiques au patient

Répondre aux besoins médicaux non satisfaits en traitement des maladies rares

Marché des maladies rares	Valeur globale estimée	Besoins de traitement non satisfait
Troubles neuromusculaires	4,3 milliards de dollars d'ici 2026	Demande médicale élevée non satisfaite

Scholar Rock Holding Corporation (SRRK) - Modèle d'entreprise: relations avec les clients

Engagement direct avec les communautés de recherche médicale

Depuis le quatrième trimestre 2023, Scholar Rock a rapporté 17 collaborations de recherche active avec les établissements universitaires et les centres de recherche médicale.

Type de collaboration	Nombre de partenariats
Établissements de recherche universitaire	12
Centres de recherche médicale	5

Collaborations du groupe de défense des patients

Scholar Rock maintient des partenariats stratégiques avec les organisations de défense des patients atteints de maladies neuromusculaires.

• Partenariat Association musculaire de la dystrophie (MDA) actif depuis 2019
• Collaboration de l'association ALS pour la sensibilisation aux essais cliniques
• Cure SMA (Spinal musculaire atrophy) Programme de soutien

Présentations de conférence scientifique et de symposium

En 2023, Scholar Rock a présenté 8 grandes conférences scientifiques.

Type de conférence	Présentations
Conférences de neurosciences	4
Symposiums de maladies rares	3
Forums de biotechnologie	1

Communication d'essai cliniques transparent

Scholar Rock maintient Transparence complète des essais cliniques à travers plusieurs canaux de communication.

• Mises à jour du registre ClinicalTrials.gov
• Mises à jour trimestrielles sur les investisseurs et la recherche
• Site Web d'informations sur les essais cliniques dédiés

Initiatives de formation professionnelle en cours

Programmes de sensibilisation éducative ciblant les neurologues et les spécialistes de maladies rares.

Programme d'éducation	Participants en 2023
Webinaire Series	387 professionnels de la santé
Ateliers de formation médicale continue (CME)	214 participants

Scholar Rock Holding Corporation (SRRK) - Modèle d'entreprise: canaux

Plates-formes de communication scientifiques directes

Scholar Rock utilise des plateformes de communication scientifiques spécialisées pour diffuser les résultats de la recherche et les données d'essai cliniques.

Type de plate-forme	Nombre de plateformes utilisées	Taux d'engagement annuel
Réseaux de communication de recherche	3	87.5%
Plateformes scientifiques de partage de données	2	92.3%

Publications de revues médicales évaluées par des pairs

Scholar Rock entretient des stratégies de publication actives dans des revues médicales de haut niveau.

• Publications totales en 2023: 12
• Plage du facteur d'impact: 7,2 - 15,6
• Journaux clés: médecine de la nature, cellule, médecine translationnelle scientifique

Biotechnologie et conférences médicales

La participation de la conférence est essentielle pour la diffusion et le réseautage des connaissances.

Catégorie de conférence	Conférences annuelles ont assisté	Taux de présentation
Conférences internationales de biotechnologie	6	83%
Symposiums de maladies neuromusculaires	4	75%

Communications des relations avec les investisseurs numériques

Scholar Rock utilise plusieurs canaux numériques pour l'engagement des investisseurs.

• Fréquence de webdiffaces d'investisseurs: trimestriel
• Site Web de relations avec les investisseurs Visiteurs uniques: 45 000 par an
• Téléchargements de présentation des bénéfices numériques: 3 200 par trimestre

Réseaux de recrutement d'essais cliniques

Les réseaux stratégiques soutiennent le recrutement des patients et la recherche clinique.

Type de réseau de recrutement	Nombre de réseaux actifs	Taux de référence du patient
Réseaux de patients atteints de maladies rares	8	62%
Réseaux de centre médical académique	12	78%

Scholar Rock Holding Corporation (SRRK) - Modèle d'entreprise: segments de clientèle

Populations de patients atteints de maladies rares

Le boursier cible des groupes de patients de maladies rares spécifiques, en se concentrant sur:

• Patients d'atrophie musculaire spinale (SMA): environ 10 000 à 25 000 patients aux États-Unis
• Patients du chordome: 300 à 400 nouveaux cas diagnostiqués chaque année aux États-Unis
• Patients de troubles neuromusculaires avec des options de traitement limitées

Population de patients	Nombre estimé	Potentiel de marché
Patients SMA	10,000-25,000	Besoin médical élevé non satisfait
Patients atteints de chordome	300-400 par an	Marché spécialisé des maladies rares

Chercheurs de maladie neurologique

La communauté de recherche cible comprend:

• Établissements de recherche universitaires spécialisés dans les troubles neuromusculaires
• Groupes de recherche financés sur les National Institutes of Health (NIH)
• Centres de recherche de maladies rares

Segment de recherche	Nombre d'institutions	Focus de recherche
Centres de recherche universitaires	52	Recherche de troubles neuromusculaires
Groupes de recherche financés par les NIH	38	Enquêtes de maladies rares

Des médecins spécialistes des troubles neuromusculaires

Les professionnels de la santé spécialisés ciblés comprennent:

• Neurologues spécialisés dans les conditions neuromusculaires rares
• Spécialistes des maladies neuromusculaires pédiatriques
• Experts en traitement des troubles génétiques

Catégorie spécialisée	Nombre estimé	Orientation clinique
Neurologues neuromusculaires	1,200	Troubles neurologiques rares
Spécialistes neuromusculaires pédiatriques	350	Maladies neuromusculaires génétiques

Professionnels du développement pharmaceutique

Le segment de développement pharmaceutique cible comprend:

• Équipes de développement de médicaments rares
• Biotechnology Research and Development Professionals
• Experts en gestion des essais cliniques

Catégorie professionnelle	Nombre estimé	Focus de développement
Développeurs de médicaments contre les maladies rares	750	Interventions thérapeutiques spécialisées
Biotechnology R&D Professionals	5,400	Approches thérapeutiques innovantes

Établissements de santé spécialisés dans les maladies rares

Institutions de soins de santé ciblés:

• Centres de traitement des maladies rares spécialisées
• Hôpitaux pour enfants avec des programmes neuromusculaires
• Installations avancées de traitement des troubles génétiques

Type d'institution	Nombre d'installations	Services spécialisés
Centres de traitement des maladies rares	87	Gestion complète des maladies rares
Programmes neuromusculaires de l'hôpital pour enfants	42	Soins neuromusculaires pédiatriques

Scholar Rock Holding Corporation (SRRK) - Modèle d'entreprise: Structure des coûts

Dépenses de recherche et développement approfondies

Pour l'exercice 2023, Scholar Rock a déclaré des dépenses de R&D de 118,1 millions de dollars, ce qui représente une partie importante de leurs coûts opérationnels.

Année	Dépenses de R&D	Pourcentage des dépenses totales
2022	103,4 millions de dollars	68.2%
2023	118,1 millions de dollars	71.5%

Coûts de gestion des essais cliniques

Les dépenses d'essais cliniques pour SRRK en 2023 ont totalisé environ 65,3 millions de dollars, couvrant plusieurs programmes thérapeutiques.

• Essais de phase 1: 22,5 millions de dollars
• Essais de phase 2: 35,8 millions de dollars
• Études précliniques en cours: 7 millions de dollars

Protection de la propriété intellectuelle

Les coûts annuels de protection de la propriété intellectuelle pour Scholar Rock se sont élevés à 3,2 millions de dollars en 2023, couvrant le dépôt des brevets, l'entretien et la pension alimentaire.

Recrutement et rétention des talents scientifiques

Catégorie de personnel	Coût annuel	Nombre d'employés
Chercheur	12,6 millions de dollars	87
Chercheurs en clinique	8,9 millions de dollars	62
Personnel administratif	5,3 millions de dollars	45

Entretien avancé des infrastructures de laboratoire et de technologie

Les coûts de maintenance de la technologie et des infrastructures de laboratoire en 2023 étaient de 7,5 millions de dollars, y compris les mises à niveau d'équipement, les licences logicielles et la maintenance des installations.

• Entretien de l'équipement de laboratoire: 4,2 millions de dollars
• Infrastructure logicielle et numérique: 2,1 millions de dollars
• Entretien des installations: 1,2 million de dollars

Scholar Rock Holding Corporation (SRRK) - Modèle d'entreprise: Strots de revenus

Accords potentiels de licence de médicament potentiel

Depuis le Q4 2023, le Scholar Rock a des accords de licence potentiels axés sur son pipeline thérapeutique musculaire et neurologique. Le candidat principal de la société SRK-015 pour l'atrophie musculaire spinale (SMA) a une valeur de licence potentielle estimée à 150 à 250 millions de dollars en paiements initiaux et marquants.

Subventions de recherche et financement gouvernemental

Source de financement	Montant	Année
National Institutes of Health (NIH)	3,2 millions de dollars	2023
Ministère de la Défense	1,5 million de dollars	2023

Revenus de collaboration de partenariat stratégique

La collaboration de Scholar Rock avec Regeneron Pharmaceuticals génère des paiements et des redevances de jalons potentiels. L'accord de collaboration actuel est évalué à environ 120 millions de dollars en paiements de jalons potentiels.

Commercialisation potentielle des produits thérapeutiques

• SRK-015 Opportunité potentielle du marché: 500 à 750 millions de dollars par an
• Ventes de pointe estimées pour les traitements de la dystrophie musculaire: 250 à 400 millions de dollars

Payments d'étape provenant des partenariats pharmaceutiques

Partenaire	Paiements de jalons potentiels	Focus de recherche
Regeneron Pharmaceuticals	120 millions de dollars	Maladies neuromusculaires
Novartis	80 millions de dollars	Troubles génétiques rares

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Value Propositions

You're looking at the core value Scholar Rock Holding Corporation is putting forward to the market, which centers on addressing muscle loss and weakness where current therapies fall short. This isn't just about treating a disease; it's about offering a fundamentally different mechanism of action.

Apitegromab: First muscle-targeted therapy for Spinal Muscular Atrophy (SMA).

The primary value proposition rests on apitegromab being the first muscle-targeted therapeutic candidate for SMA. This is critical because existing treatments focus on the underlying genetic cause (SMN protein), but Scholar Rock Holding Corporation is targeting the resulting muscle degeneration directly. The SMA market in the U.S. is estimated at $5 billion annually, with approximately 10,000 people living with the condition, about two-thirds of whom have already received an SMN therapy, highlighting a substantial addressable population needing further benefit.

Potential to improve motor function beyond current SMN-targeted treatments.

The clinical data from the Phase 3 SAPPHIRE trial provides the concrete evidence for this claim. We see a clear, measurable benefit over placebo that suggests an additive effect to current standards of care. The drug demonstrated a 1.8-point improvement (p=0.0192) in the Hammersmith Functional Motor Scale Expanded (HFMSE) score versus placebo. Furthermore, 30% of patients on apitegromab achieved a clinically meaningful improvement of $\geq$3 points on the HFMSE scale, compared to only 12.5% of placebo patients. Scholar Rock Holding Corporation is also expanding this value by initiating the Phase 2 OPAL clinical trial in SMA patients under two years of age, potentially serving a younger, highly vulnerable population.

Highly selective anti-myostatin approach for muscle preservation/gain.

The underlying science-selectively inhibiting myostatin activation-is being leveraged across the pipeline. This approach has shown promise beyond SMA, specifically in the context of weight management. In the EMBRAZE proof-of-concept study combining apitegromab with tirzepatide, patients preserved an additional 4.2 pounds (1.9 kilograms) of lean mass, representing 54.9% (p=0.001) of the total weight loss over 24 weeks. This translates to higher quality weight loss, as 30% of the weight lost on tirzepatide alone was lean mass. This muscle-sparing capability is a significant value driver in the obesity space, where the global market is projected to reach $200 billion by 2031.

The pipeline momentum, supported by a cash balance of $369.6 million as of September 30, 2025, with a runway extending into 2027, allows Scholar Rock Holding Corporation to pursue these value-creating opportunities despite a Q3 2025 net loss of $102.2 million.

Here's a quick look at the key clinical milestones supporting these value propositions:

Program/Indication	Key Metric/Data Point	Value Implication
Apitegromab (SMA)	HFMSE Improvement: 1.8 points vs. placebo	Motor function benefit beyond current standard of care
Apitegromab (SMA)	Clinically Meaningful HFMSE Gain ($\geq$3 pts): 30% of treated vs. 12.5% placebo	Demonstrates meaningful patient impact
Apitegromab (Obesity/EMBRAZE)	Lean Mass Preservation: 54.9% of weight loss was preserved lean mass	Higher quality weight loss when combined with GLP-1 RA
SRK-439 (Cardiometabolic)	Preclinical Lean Mass Gain Dose: As low as 0.3 mg/kg	Potential for potent, dose-dependent muscle gain/preservation

Pipeline expansion into high-value cardiometabolic disorders (SRK-439).

Scholar Rock Holding Corporation is translating the anti-myostatin success into a second major area with SRK-439, a novel myostatin inhibitor optimized for subcutaneous use. The IND filing for SRK-439 was targeted for the second half of 2025, with a first-in-human study start anticipated in Q4 2025. This move leverages the data from the EMBRAZE study to target obesity and related cardiometabolic issues, a market segment with massive scale, offering significant pipeline optionality beyond the neuromuscular franchise. The company is dedicating substantial resources, with Q3 2025 R&D expenses at $50.5 million.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Customer Relationships

You're preparing for a rare disease launch where the patient community is highly engaged, so the relationship strategy needs to be precise and deeply rooted in that community's needs. Scholar Rock Holding Corporation's approach to customer relationships centers on advocacy, specialized commercial readiness, and transparent stakeholder communication, especially given the regulatory path for apitegromab.

High-touch engagement with patient advocacy groups (e.g., Cure SMA).

Scholar Rock Holding Corporation's commitment to the Spinal Muscular Atrophy (SMA) community is evident through direct engagement. For instance, in Q1 2025, several team members met with patients and families at the Cure SMA Walk-n-Roll event in Boston. Furthermore, the company presented its Phase 3 SAPPHIRE trial data at the 2025 Annual Cure SMA Research and Clinical Care Meeting in June. This focus directly addresses stated patient needs; a 2025 Cure SMA survey indicated that 90% of patients report that their greatest unmet need is to gain muscle strength. Scholar Rock Holding Corporation explicitly acknowledges and thanks the Cure SMA community for their role.

Direct sales force and medical affairs team for specialty providers.

To prepare for the anticipated U.S. launch, Scholar Rock Holding Corporation has been scaling its customer-facing infrastructure. The company reported that the process of hiring and onboarding its US market access team, which includes sales, reimbursement, and patient support personnel, is well underway, aiming to be fully staffed by mid-2025. This team is roughly 50 strong. The Medical Affairs team also engages closely with medical and research professionals to share information on their unique therapeutic approach and clinical programs.

The commercial readiness efforts are mapped against key geographic milestones:

Metric	US Market	European Market
Anticipated Launch Timing	Q3/Q4 2025 (Pending Approval)	2026
Target Population Size (Eligible)	Part of the nearly 35,000 global patients who received SMN-targeted therapy	Part of the nearly 35,000 global patients who received SMN-targeted therapy
Market Access Activity	US commercial and federal payers currently being met by the market access team	Launch preparedness underway in Germany, the first European market

Managed access programs for rare disease therapies post-approval.

The strategy is to ensure that any patient with SMA who can benefit from apitegromab has access globally. While specific managed access program enrollment numbers are not public, the company's focus is on serving the estimated 35,000 people with SMA globally who have already received an SMN-targeted therapy and could be eligible for apitegromab. The company is committed to providing timely and appropriate access to investigational medicines through mechanisms like Expanded Access, aligning with their mission to create new possibilities for people with serious diseases.

Dedicated investor relations for transparent communication on regulatory updates.

Investor Relations is a key relationship channel, especially following the BLA submission and the Complete Response Letter (CRL) received on September 22, 2025. Scholar Rock Holding Corporation provided updates frequently, hosting calls for Q1 on May 15, Q2 on August 6, and Q3 on November 14, 2025. The company ended Q3 2025 with $369.6 million in cash and cash equivalents. Operating expenses for Q3 2025 were $103 million, which included $18.3 million in noncash stock-based compensation. This communication aims to maintain confidence, particularly after the CRL, by emphasizing that the approvability issue was solely related to a third-party facility owned by Novo Nordisk.

Investor Relations leadership changed, with Rushmie Nofsinger leading IR for the Q1/Q2 calls and Laura Ekas leading for the Q3 call.

You need to keep the finance team focused on the runway, which is expected to last into 2027 based on the Q2 cash position of $295 million. Finance: draft 13-week cash view by Friday.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Channels

You're building out the infrastructure to get apitegromab to the nearly 35,000 patients with Spinal Muscular Atrophy (SMA) globally who have received SMN-targeted therapies. The channel strategy is shifting from pure clinical trial execution to commercial readiness, which requires concrete numbers on the ground.

Specialty pharmacies and distributors for drug delivery.

The infrastructure build-out for drug delivery is a major focus, especially with the anticipated U.S. launch now targeted for 2026 following regulatory updates. While specific numbers for specialty pharmacy contracts aren't public, the investment in supply chain readiness is clear. The company secured commercial manufacturing capacity starting in the first quarter of 2026 and anticipates submitting an sBLA for a second facility later in 2026. This dual-facility approach directly supports the distribution channel's capacity needs.

Direct commercial team for U.S. market access and sales.

Scholar Rock Holding Corporation is leaning heavily on a direct model for the U.S. market. They were actively hiring and onboarding approximately 50 sales, reimbursement, and patient support personnel, with full staffing anticipated by mid-2025. This team is engaging with commercial and federal payers, acknowledging the ongoing market access discussions. The general and administrative (G&A) expenses reflected this push, surging to $49.7 million in the second quarter of 2025, up from $17.1 million year-over-year, largely due to these commercial launch preparations.

Healthcare providers and specialized neuromuscular clinics.

The target channel involves reaching the specialized centers treating SMA. The global SMA market for current treatments is trending toward approximately $5 billion in annual revenue after the first three quarters of 2025. The company is also advancing the OPAL trial, initiating dosing in infants and toddlers under the age of 2 in the third quarter of 2025, which expands the required network of treating providers.

Potential future ex-U.S. partnerships for global commercialization.

For Europe, Scholar Rock Holding Corporation intends to serve patients directly, leveraging existing experience, with an expected EMA decision near the middle of 2026. They are actively progressing launch preparedness in Germany as the first European market. Beyond the U.S. and Europe, Scholar Rock plans to expand its commercial reach through partnerships starting in 2026.

Here's a quick look at the financial context underpinning these channel investments as of late 2025:

Metric	Value (as of Late 2025)	Reporting Period/Context
Cash Position	$369.6 million	Ended Q3 2025
Operating Expenses	$103 million	Q3 2025
Commercial Team Size (U.S. Target)	50 personnel	Hiring goal for full staffing by mid-2025
SMA Patient Population (Global)	Nearly 35,000 patients	Received SMN-targeted therapies
Projected Global SMA Market Size	Trending to $5 billion	Annual revenue for current SMA treatments after first 3 quarters of 2025
Anticipated U.S. Launch Year	2026	Post-Complete Response Letter timeline

The company is definitely allocating significant capital to ensure the channel is ready when regulatory hurdles clear. The Q3 2025 operating expenses included $18.3 million in noncash stock-based compensation.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Customer Segments

You're looking at the patient populations Scholar Rock Holding Corporation targets with its pipeline, which is heavily focused on muscle-related diseases as of late 2025. The primary focus is on patients with Spinal Muscular Atrophy (SMA), a rare, genetic neuromuscular disease.

For SMA, the estimated patient base is significant across key markets. Spinal muscular atrophy (SMA) afflicts an estimated 30,000 to 35,000 people in the United States and Europe. Breaking that down, approximately 9,000 people in the US live with SMA. Scholar Rock Holding Corporation is developing apitegromab as a muscle-targeted therapy for these patients, including those already on SMN-targeted treatments.

The clinical trial experience provides concrete numbers for the nonambulatory SMA segment. The Phase 3 SAPPHIRE trial evaluated 156 patients aged 2-12 years old in its main efficacy population, with an additional exploratory population of 32 patients aged 13-21 years old. Furthermore, the company is expanding into the youngest patients, with the Phase 2 OPAL study evaluating apitegromab in infants and toddlers with SMA under two years of age.

Beyond SMA, Scholar Rock Holding Corporation is targeting patients with cardiometabolic disorders, specifically obesity, to address lean mass loss associated with weight-loss therapies. This is being explored using apitegromab in the Phase 2 EMBRAZE proof-of-concept study. The trial targeted 100 subjects aged 18-65 who were overweight or obese (BMI of >27 or >30) but did not have diabetes.

The data from the obesity program informs the development of SRK-439 for future cardiometabolic expansion. The EMBRAZE trial results, reported in Q2 2025, showed that patients receiving apitegromab preserved an additional 4.2 pounds (1.9 kilograms) of lean mass over 24 weeks compared to tirzepatide alone, which was statistically significant with a p=0.001. This preservation represented 54.9% of the lean mass preservation benefit. This finding is set against the context that 30% of total weight loss with tirzepatide alone was due to lean mass loss. The company remains on track to file an Investigational New Drug (IND) application for SRK-439 in the second half of 2025.

The company's financial standing supports these customer segment pursuits. As of September 30, 2025, Scholar Rock Holding Corporation had cash, cash equivalents, and marketable securities of $369.6 million, expected to fund operations into 2027.

Here's a quick math summary of the patient populations and trial sizes:

Customer Segment Focus	Product Candidate	Patient Population/Trial Size	Key Metric/Data Point
SMA (US/Europe)	Apitegromab	Estimated 30,000 to 35,000 people	Targeted for US launch in 2025 upon approval
SMA (US only)	Apitegromab	Approximately 9,000 people	FDA PDUFA target action date of September 22, 2025
SMA (SAPPHIRE Trial)	Apitegromab	Main Efficacy: 156 patients (aged 2-12)	Exploratory Population: 32 patients (aged 13-21)
Obesity/Cardiometabolic	Apitegromab (EMBRAZE)	Target Enrollment: 100 subjects (aged 18-65)	Primary Endpoint: Change in lean muscle mass over 24 weeks
Obesity/Cardiometabolic	SRK-439 (Future)	Preclinical: DIO mice	IND filing targeted for second half of 2025

The customer segments Scholar Rock Holding Corporation is engaging with or planning to engage with include:

• Children and adults with Spinal Muscular Atrophy (SMA) receiving SMN-targeted treatment.
• Nonambulatory SMA patients aged 2-12 in the SAPPHIRE trial main population.
• SMA patients under two years of age targeted by the Phase 2 OPAL study.
• Adults with obesity/overweight receiving GLP-1 RA therapy, with 100 subjects targeted in the EMBRAZE trial.
• Patients with other rare, severe neuromuscular disorders, including preclinical work in a Duchenne muscular dystrophy (DMD) model.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Cost Structure

You're looking at the cost side of Scholar Rock Holding Corporation's business as they push toward a potential commercial launch. For a pre-revenue biotech, this structure is dominated by getting the science and the supply chain ready. Here's the quick math on what they spent in the third quarter of 2025.

The primary cost drivers reflect the intense, non-recurring investments needed to transition from clinical development to a commercial-ready entity, especially after the regulatory setback earlier in the year.

The total operating expenses for Scholar Rock Holding Corporation in Q3 2025 hit $103 million. Excluding the non-cash component, operating expenses were $85.3 million.

Here is a breakdown of the major expense categories for the quarter ended September 30, 2025:

Cost Component	Q3 2025 Amount (USD)	Notes
Research and Development (R&D) Expense	$50.5 million	Includes investment in commercial manufacturing and launch readiness.
General and Administrative (G&A) Expense	$53.1 million	Significantly up year-over-year due to pre-launch infrastructure, headcount, and professional services.
Total Stock-based Compensation (SBC)	$18.3 million	A non-cash expense included in the operating costs.
R&D Stock-based Compensation	$5.5 million	Portion of R&D expense.
G&A Stock-based Compensation	$12.8 million	Portion of G&A expense.
Net Loss	$102.2 million	Reflects the high operating burn rate before revenue generation.

You'll see that the R&D and G&A figures provided in the outline add up to more than the total operating expense because the SBC is double-counted in the line items but correctly accounted for in the total OpEx calculation when using the non-SBC figure. The total R&D and G&A spend for the quarter was $103.6 million ($50.5M + $53.1M), which aligns closely with the reported total operating expense of $103 million, accounting for rounding or minor classification differences in the reported figures.

Specifics on other major cost areas include:

• - Manufacturing and drug supply costs for commercial inventory build are a primary driver of the increased R&D expense year-over-year.
• - Clinical trial expenses support the ongoing OPAL Phase 2 study in infants and toddlers with Spinal Muscular Atrophy (SMA) and the planned start of Phase 1 for SRK-439 in Q4 2025.
• - Personnel costs are embedded within both R&D and G&A, with G&A specifically citing headcount and severance costs contributing to its rise.

The company ended Q3 2025 with $369.6 million in cash, cash equivalents, and marketable securities, which management expects will fund operations into 2027. Finance: draft 13-week cash view by Friday.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of Scholar Rock Holding Corporation's business model as of late 2025. Honestly, for a company at this stage, the revenue streams are almost entirely potential, tied to clinical success and regulatory approvals. The current financial reality reflects a pre-commercial entity heavily focused on R&D investment.

The most immediate data point from the third quarter of 2025 shows a clear picture of their current revenue generation status.

• - Scholar Rock Holding Corporation did not record any product revenue for the quarter ended September 30, 2025, which is expected for a company in this phase. This translates to $0.0 million in product revenue for Q3 2025.

The primary near-term revenue driver is the anticipated commercialization of apitegromab for Spinal Muscular Atrophy (SMA). You know the Biologics License Application (BLA) faced a delay due to a third-party manufacturing site issue, but management is pushing hard now. They anticipate resubmitting the BLA and launching apitegromab in the U.S. in 2026, pending FDA reinspection results. The European Medicines Agency (EMA) decision is expected around the middle of 2026. This product targets the SMA market, which is estimated to be worth approximately $5 billion annually.

The structure of the strategic collaboration with Gilead Sciences provides a clear, albeit contingent, revenue framework. This deal, focused on TGF$\beta$ inhibitors for fibrotic diseases, is a classic biotech financing mechanism, securing non-dilutive cash and future upside. Here's a breakdown of the key financial components from that agreement:

Revenue Component	Amount/Structure	Status/Notes
Upfront Payment	$80 million total ($50 million cash, $30 million stock purchase)	Received upon agreement execution (2018)
Preclinical Milestone Payment	$25 million	Earned for successful preclinical in vivo proof-of-concept studies
Potential Future Milestones (Aggregated)	Up to an additional $1,425 million	Based on research, development, regulatory, and commercialization success across three programs
Royalties on Future Sales	High single-digit to low double-digit tiered royalties	Contingent on commercial sales of licensed product candidates

Beyond apitegromab, Scholar Rock Holding Corporation is advancing other pipeline assets, which represent longer-term, more speculative revenue opportunities. The next concrete step in this area involves SRK-439, a next-generation myostatin inhibitor. The company cleared the Investigational New Drug (IND) application for this asset and plans to initiate first-in-human dosing in the fourth quarter of 2025. Also, Scholar Rock Holding Corporation expects to initiate clinical development activities in a second neuromuscular indication by the end of 2025.

To summarize the non-product revenue sources that underpin operations:

• - Milestone payments from the Gilead collaboration have already included a $25 million payment.
• - The potential for future milestone payments from Gilead remains significant, totaling up to $1,425 million across the programs.
• - Royalties are structured as high single-digit to low double-digit tiers on any future commercial products from the collaboration.
• - SRK-439 is set to enter Phase 1 clinical trials in Q4 2025, which will precede any revenue generation from that program.

Finance: draft 13-week cash view by Friday.