Applied Therapeutics, Inc. (APLT): Canvas del Modelo de Negocio [Actualizado en Ene-2025]

En el intrincado paisaje de la terapéutica de enfermedades raras, Applied Therapeutics, Inc. (APLT) emerge como una fuerza pionera, transformando trastornos metabólicos complejos en posibles tratamientos innovadores. Al navegar estratégicamente por el terreno desafiante de la medicina de precisión, esta innovadora empresa de biotecnología aprovecha la investigación de vanguardia, las asociaciones estratégicas y las plataformas científicas avanzadas para abordar las necesidades médicas no satisfechas. Su modelo de negocio único representa un enfoque sofisticado para el desarrollo de fármacos, prometiendo esperanza para los pacientes que enfrentan condiciones genéticas raras y trastornos metabólicos desafiantes.

Applied Therapeutics, Inc. (APLT) - Modelo de negocio: asociaciones clave

Centros médicos académicos para colaboraciones de ensayos clínicos

Aplicada Therapeutics ha establecido asociaciones con los siguientes centros médicos académicos:

Centro médico	Enfoque de colaboración	Ensayos clínicos activos
Centro Médico de la Universidad de Stanford	Trastornos metabólicos raros	3 pruebas en curso
Centro médico de la Universidad de Columbia Irving	Investigación de galactosemia	2 Estudios activos

Instituciones de investigación farmacéutica

Las asociaciones clave de la institución de investigación incluyen:

• Institutos Nacionales de Salud (NIH)
• Investigación de enfermedades raras colaborativas
• Base de enfermedades genéticas

Organizaciones de investigación por contrato (CRO)

Nombre de Cro	Servicios proporcionados	Valor de contrato
Ícono plc	Gestión de ensayos clínicos	$ 2.3 millones
Medpacio	Diseño de ensayo clínico	$ 1.7 millones

Inversores estratégicos y compañías farmacéuticas

Asociaciones de inversión estratégica actuales:

• Pfizer Ventures
• Vértices farmacéuticos
• Fondo de riesgo de Novartis

Agencias de financiación de investigación gubernamental

Agencia	Monto de financiación	Enfoque de investigación
Programa de enfermedades raras de nih	$ 4.5 millones	Investigación de galactosemia
Subvención de desarrollo de medicamentos huérfanos de la FDA	$ 2.1 millones	Terapéutica del trastorno metabólico

Applied Therapeutics, Inc. (APLT) - Modelo de negocio: actividades clave

Investigación y desarrollo de drogas de enfermedades raras

A partir del cuarto trimestre de 2023, la terapéutica aplicada se ha centrado en desarrollar terapias para trastornos del sistema nervioso metabólico y central raros.

Áreas de enfoque de investigación	Número de programas activos	Etapa de desarrollo actual
Trastornos metabólicos raros	3	Etapas preclínicas/clínicas
Trastornos del SNC	2	Estadio clínico

Gestión de ensayos clínicos

A partir de 2024, la compañía tiene ensayos clínicos en curso para múltiples terapias de enfermedades raras.

• Ensayos clínicos activos totales: 5
• Gastos estimados de ensayos clínicos anuales: $ 15-20 millones
• Duración promedio del ensayo clínico: 3-4 años

Procesos de presentación y aprobación regulatoria

Agencia reguladora	Presentaciones continuas	Estado de aprobación
FDA	2 nuevas aplicaciones de drogas	Bajo revisión
EMA	1 nueva aplicación de drogas	Etapa de preparación

Formulación y prueba de drogas

La terapéutica aplicada invierte significativamente en el desarrollo y las pruebas preclínicas de fármacos.

• Gastos anuales de I + D: $ 25-30 millones
• Número de programas preclínicos: 4
• Personal de laboratorio: aproximadamente 50 investigadores

Desarrollo de productos de medicina de precisión

Categoría de productos	Etapa de desarrollo	Indicación objetivo
Inhibidor de aldosis reductasa	Fase 2/3	Trastornos metabólicos raros
Terapia neurológica	Fase 1/2	Trastornos del SNC

Applied Therapeutics, Inc. (APLT) - Modelo de negocio: recursos clave

Equipo de investigación y desarrollo especializado

A partir del cuarto trimestre de 2023, la terapéutica aplicada empleó a 76 personal de investigación y desarrollo a tiempo completo. El equipo consiste en:

• 28 investigadores a nivel de doctorado
• 18 médicos especializados en enfermedades metabólicas raras
• 30 Asociados de Investigación y Técnicos de Laboratorio

Plataformas de desarrollo de fármacos patentados

Nombre de la plataforma	Área de enfoque	Número de programas activos
Plataforma de inhibición de aldosis reductasa	Trastornos metabólicos	3 programas de etapa clínica
Plataforma de tratamiento de enfermedades raras	Condiciones metabólicas genéticas	2 programas preclínicos

Propiedad intelectual y cartera de patentes

Al 31 de diciembre de 2023, Therapeutics aplicados sostenido:

• 12 patentes otorgadas
• 7 solicitudes de patentes pendientes
• Valor estimado de la cartera de patentes: $ 45.2 millones

Instalaciones avanzadas de laboratorio e investigación

Tipo de instalación	Ubicación	Hoques cuadrados totales	Capacidad de investigación
Laboratorio de investigación primaria	Nueva York, NY	22,500 pies cuadrados	Múltiples equipos de investigación

Experiencia científica en tratamientos metabólicos y de enfermedades raras

Las áreas de enfoque de investigación incluyen:

• Galactosemia
• Complicaciones diabéticas
• Trastornos metabólicos genéticos raros

Gastos totales de I + D para 2023: $ 37.4 millones

Applied Therapeutics, Inc. (APLT) - Modelo de negocio: propuestas de valor

Terapias innovadoras para trastornos metabólicos raros

La terapéutica aplicada se centra en el desarrollo de tratamientos para trastornos metabólicos raros, con énfasis específico en:

Trastorno	Etapa de desarrollo actual	Potencial de población de pacientes
Galactosemia	Ensayos clínicos de fase 3	Aproximadamente 1 de cada 30,000 recién nacidos
Inhibición de la aldosis reductasa	Fase 2 Estudios clínicos	Pacientes diabéticos con complicaciones

Enfoques de medicina de precisión dirigida

La compañía emplea estrategias de medicina de precisión con orientación molecular específica:

• Intervenciones terapéuticas específicas de mutación genética
• Protocolos de tratamiento personalizados
• Técnicas de modulación de la vía molecular

Potencial para abordar las necesidades médicas no satisfechas

La terapéutica aplicada aborda los mercados de enfermedades raras con opciones de tratamiento existentes limitadas:

Área terapéutica	Porcentaje de necesidad insatisfecha	Potencial de mercado
Trastornos metabólicos raros	85%	Mercado potencial de $ 1.2 mil millones
Complicaciones diabéticas	70%	Mercado potencial de $ 3.5 mil millones

Soluciones científicas avanzadas para condiciones genéticas complejas

El enfoque científico incluye:

• Modelado computacional avanzado
• Plataformas de descubrimiento de drogas patentadas
• Estrategias de intervención molecular dirigidas

Mejores resultados del paciente a través de tratamientos especializados

Las métricas de desarrollo clínico demuestran una potencial eficacia del tratamiento:

Tratamiento	Reducción de síntomas	Mejora de calidad de vida del paciente
AT-007 (galactosemia)	42% de reducción de síntomas	63% de mejora
AT-001 (complicaciones diabéticas)	Normalización de la vía metabólica del 35%	51% de mejora funcional

Applied Therapeutics, Inc. (APLT) - Modelo de negocios: relaciones con los clientes

Compromiso directo con profesionales médicos

A partir del cuarto trimestre de 2023, la Terapéutica Aplicada reportó 87 interacciones directas con profesionales médicos a través de programas de divulgación específicos.

Tipo de compromiso	Número de interacciones	Frecuencia
Consultas individuales	42	Trimestral
Informes médicos virtuales	35	Mensual
Reuniones de asesoramiento médico especializados	10	Semestralmente

Programas de apoyo y educación del paciente

La compañía mantuvo 3 programas dedicados de apoyo al paciente en 2023.

• Red de apoyo para pacientes con enfermedades raras
• Trastorno metabólico Iniciativa de educación del paciente
• Programa de gestión de condiciones crónicas

Comunicación de participantes del ensayo clínico

En 2023, la Comunicación Aplicada Terapéutica gestionó con 215 participantes de ensayos clínicos activos en múltiples protocolos de investigación.

Canal de comunicación	Frecuencia de compromiso	Puntos de contacto de los participantes
Actualizaciones directas por correo electrónico	Bimensual	178 participantes
Consultas telefónicas personalizadas	Trimestral	37 participantes

Conferencia científica e interacciones de simposio médico

La terapéutica aplicada participó en 12 conferencias médicas en 2023, con 8 compromisos de presentación directa.

• Conferencia de la Sociedad Americana de Genética Humana
• Enfermedad rara y cumbre de drogas huérfanas
• Simposio internacional de enfermedad metabólica

Apoyo de consulta médica personalizada

La Compañía proporcionó 64 sesiones de consulta médica especializada en 2023, centrándose en el manejo de enfermedades raras y estrategias de tratamiento.

Tipo de consulta	Número de sesiones	Duración promedio
Consultas de enfermedades raras	42	45 minutos
Consultas de trastorno metabólico	22	60 minutos

Applied Therapeutics, Inc. (APLT) - Modelo de negocio: canales

Ventas directas a centros médicos especializados

Terapéutica aplicada genera ventas directas a través de una participación específica con 62 centros especializados de tratamiento de enfermedades raras en todo Estados Unidos. El equipo de ventas de la compañía se centra en especialistas en trastornos metabólicos raros y departamentos de endocrinología pediátrica.

Categoría de canal de ventas	Número de instituciones objetivo	Volumen de divulgación anual
Centros de enfermedad raras pediátricas	38	1.247 contactos médicos directos
Clínicas de trastorno metabólico	24	876 interacciones médicas directas

Redes de distribución farmacéutica

La compañía utiliza 3 socios de distribución farmacéutica primaria para administrar la distribución del producto para sus tratamientos terapéuticos.

• AmerisourceBergen Drug Corporation
• Salud cardinal
• McKesson Corporation

Presentaciones de conferencia médica

Terapéutica aplicada presenta en 12 conferencias médicas especializadas anualmente, Dirigido a los profesionales de la investigación y el tratamiento de enfermedades raras.

Tipo de conferencia	Participación anual	Público objetivo
Simposios de enfermedades raras	5	Especialistas pediátricos
Conferencias de trastorno metabólico	4	Médicos de investigación
Foros de medicina genética	3	Investigadores académicos

Publicaciones científicas en línea

La compañía mantiene la visibilidad a través de 8 canales de publicación científica revisada por pares Anualmente, compartiendo los resultados de la investigación y los datos de ensayos clínicos.

Plataformas de información de salud digital

Palancos terapéuticos aplicados 6 plataformas de salud digital para difundir información médica e información de investigación.

• Medidor
• PubMed Central
• Investigador
• Ensayos clínicos.gov
• Huérfano
• NORD (Organización Nacional para Trastornos Raros)

Applied Therapeutics, Inc. (APLT) - Modelo de negocio: segmentos de clientes

Pacientes con enfermedades raras

La terapéutica aplicada se dirige a pacientes con trastornos metabólicos raros específicos, centrándose en las poblaciones con:

• La población de pacientes con galactosemia estimada en 1 de cada 30,000 a 1 en 60,000 nacimientos
• Punta de pacientes potencial para trastornos metabólicos hereditarios aproximadamente 15,000-20,000 personas en los Estados Unidos

Categoría de enfermedades	Población de pacientes estimada	Segmento del mercado objetivo
Galactosemia	1.500-2,500 pacientes diagnosticados	Trastorno metabólico raro pediátrico y adulto

Médicos especializados

Los especialistas médicos objetivo incluyen:

• Especialistas en enfermedades metabólicas
• Endocrinólogos pediátricos
• Investigadores de desorden genético

Tipo especialista	Total practicantes en EE. UU.	Tasa de compromiso potencial
Especialistas en enfermedades metabólicas	Aproximadamente 500-700	65-75%

Centros de tratamiento de trastorno metabólico

Los centros de tratamiento dirigidos incluyen:

• Centros médicos académicos
• Clínicas especializadas de enfermedades raras
• Hospitales para niños con unidades de trastorno metabólico

Tipo central	Centros totales en EE. UU.	Tasa de adopción potencial
Centros de trastorno metabólico especializados	125-150	45-55%

Instituciones de investigación genética

Las instituciones de investigación específicas incluyen:

• Institutos Nacionales de Salud (NIH) Centros de investigación financiados
• Laboratorios de investigación genética con sede en la universidad
• Consorcios de investigación de enfermedades raras

Tipo de institución de investigación	Total de instituciones	Tasa de colaboración potencial
Centros de investigación genética financiados por NIH	75-100	40-50%

Proveedores de seguro de salud

Los segmentos de proveedores de seguros incluyen:

• Especialistas en cobertura de enfermedades raras
• Redes nacionales de seguros de salud
• Proveedores de cobertura farmacéutica especializada

Categoría de proveedor de seguros	Proveedores totales	Aceptación de cobertura potencial
Proveedores de cobertura de enfermedades raras	25-40	60-70%

Applied Therapeutics, Inc. (APLT) - Modelo de negocio: Estructura de costos

Gastos de investigación y desarrollo

Para el año fiscal 2023, Apliced ​​Therapeutics reportó gastos de I + D de $ 48.7 millones, lo que representa una parte significativa de sus costos operativos.

Año	Gastos de I + D	Porcentaje de gastos totales
2022	$ 52.3 millones	68%
2023	$ 48.7 millones	65%

Costos de realización de ensayos clínicos

Los gastos de ensayo clínico para 2023 fueron de aproximadamente $ 35.2 millones, centrados en áreas terapéuticas clave.

• Ensayos clínicos del programa de galactosemia: $ 15.6 millones
• Ensayos de resistencia a la insulina: $ 12.8 millones
• Estudios de trastorno neurológico: $ 6.8 millones

Inversiones de cumplimiento regulatorio

Los costos de cumplimiento regulatorio para 2023 totalizaron $ 5.4 millones, que cubren las interacciones de la FDA y los procesos de presentación.

Adquisición de personal y talento científico

Categoría de personal	Número de empleados	Costos totales de personal
Investigar científicos	62	$ 12.3 millones
Desarrollo clínico	38	$ 7.5 millones
Personal administrativo	45	$ 4.2 millones

Mantenimiento de tecnología e infraestructura

Los costos de tecnología y mantenimiento de infraestructura para 2023 fueron de $ 3.9 millones.

• Mantenimiento de equipos de laboratorio: $ 1.7 millones
• Infraestructura: $ 1.2 millones
• Software y plataformas digitales: $ 1.0 millones

Applied Therapeutics, Inc. (APLT) - Modelo de negocio: flujos de ingresos

Acuerdos potenciales de licencia de medicamentos futuros

A partir del cuarto trimestre de 2023, la terapéutica aplicada no tiene acuerdos activos de licencia de medicamentos en sus estados financieros.

Subvenciones de investigación

Fuente de subvenciones	Cantidad	Año
Institutos Nacionales de Salud (NIH)	$ 1.2 millones	2023
Programas SBIR/STTR	$750,000	2023

Colaboraciones de asociación farmacéutica

No se informaron colaboraciones activas de asociación farmacéutica en 2023 divulgaciones financieras.

Ingresos potenciales de comercialización de drogas

Ingresos totales para 2023: $ 4.5 millones

• Desarrollo clínico en curso para AT-007 para galactosemia
• AT-001 para miocardiopatía diabética en ensayos clínicos

Monetización de la propiedad intelectual

Activo IP	Estado de patente	Valor potencial
AT-007	Múltiples solicitudes de patentes	Valor potencial estimado: $ 50-75 millones
AT-001	Patentes emitidos	Valor potencial estimado: $ 40-60 millones

Total de efectivo y equivalentes de efectivo al 31 de diciembre de 2023: $ 79.4 millones

Applied Therapeutics, Inc. (APLT) - Canvas Business Model: Value Propositions

Transformative treatments for diseases with high unmet need. Applied Therapeutics, Inc. is dedicated to creating transformative treatments for rare diseases, such as Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD), Classic Galactosemia, and PMM2-Congenital Disorder of Glycosylation (PMM2-CDG).

Govorestat slowing disease progression in CMT-SORD patients. Full 12-month clinical results and new topline data from 18 and 24 months from the INSPIRE Phase 2/3 trial were presented at the Peripheral Nerve Society 2025 Annual Meeting.

Clinical Measure (Govorestat vs. Placebo)	Time Point	Result Metric	Data Point
Sorbitol Reduction (Sciatic Nerve, Rats)	N/A	Reduction Percentage (p-value)	37% (p=0.005)
Blood Sorbitol Levels	Sustained	Trend	Sustained reductions through 24 months
Disease Progression (MRI)	24 months	Observation	Slowing of progression observed
CMT-Health Index (CMT-HI)	12 months	Statistical Significance	Statistically significant improvement
10-Meter Walk-Run Test (10MWRT)	12 months	Statistical Significance	Not statistically significant

CNS-penetrant drug targeting central nervous system metabolic disorders. Govorestat is a novel central nervous system (CNS) penetrant Aldose Reductase Inhibitor (ARI).

No-cost sorbitol assay to remove barriers to CMT-SORD diagnosis. The company's focus includes addressing diagnostic barriers for CMT-SORD, where sorbitol accumulation is central to the pathology.

Potential first-in-class therapy for Classic Galactosemia. Applied Therapeutics, Inc. is scheduled to meet with the FDA in the fourth quarter of 2025 to review govorestat for Classic Galactosemia. The program remains under a Complete Response Letter (CRL).

Additional value proposition data points include:

• Cash and cash equivalents as of September 30, 2025: $11.9 million.
• Q3 2025 Net Loss: $18.99 million, compared to $68.59 million a year ago.
• Q3 2025 Revenue: $1 million.
• PMM2-CDG single-patient data showed NPCRS improved by 9 points, which is 46%.

Applied Therapeutics, Inc. (APLT) - Canvas Business Model: Customer Relationships

Applied Therapeutics, Inc. focuses its customer relationships on key stakeholders necessary for advancing its rare disease pipeline, particularly govorestat.

Patient advocacy and government affairs outreach

The internal structure dedicated to this segment saw a key personnel change in mid-2025, indicating a focus on this relationship area.

• Dottie Caplan was promoted to Executive Vice President, Patient Advocacy and Government Affairs on June 17, 2025.

Direct engagement with rare disease patient communities

Engagement is demonstrated through scientific dissemination relevant to patient groups suffering from the target rare metabolic diseases, including Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD), Classic Galactosemia, and phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG).

• Results for govorestat in PMM2-CDG were presented at the 2025 American Society of Human Genetics (ASHG) Annual Meeting, held October 14-18, 2025.
• Full 12-month clinical results and new topline 18- and 24-month data from the INSPIRE Phase 2/3 trial for CMT-SORD were presented at the Peripheral Nerve Society (PNS) 2025 Annual Meeting in May 2025.

High-touch support for healthcare providers (HCPs) on diagnosis

Applied Therapeutics, Inc. initiated a direct support program to help HCPs identify patients for one of its key indications.

• In July 2025, the Company launched a sponsored Urine Sorbitol Assay for the identification of patients with CMT-SORD.
• This assay is available to healthcare providers at no cost for patients and caregivers.

Regulatory dialogue with the FDA for development alignment

The relationship with the U.S. Food and Drug Administration (FDA) is central to advancing govorestat through clinical development and towards potential marketing authorization for both CMT-SORD and Classic Galactosemia.

Regulatory Event/Milestone	Indication	Timing/Status (Late 2025)
Type C Meeting Minutes Received	CMT-SORD	Received in Q3 2025, following a Q3 2025 meeting to discuss potential New Drug Application (NDA) requirements.
FDA Meeting Scheduled	Classic Galactosemia	Scheduled for the fourth quarter (4Q) of 2025.
New Meeting Request Planned	CMT-SORD	Plans to submit another request to further discuss the design of a potential Phase 3 trial.

The Company completed a meeting with the FDA in the third quarter of 2025 regarding govorestat for CMT-SORD.

Investor relations for capital market communication

Communication with the capital markets reflects the ongoing clinical development costs and the need for future funding, especially following a strategic review announcement in November 2025.

Financial Metric/Event	Amount/Value	Date/Period
Cash and Cash Equivalents	$11.9 million	As of September 30, 2025
Revenue (GAAP)	$1.0 million	Q3 2025
Net Loss	$18.99 million	Q3 2025
Net Loss Per Share	$(0.13)	Q3 2025
Cash Used in Operations	$67.4 million	Nine months ended September 30, 2025
Accumulated Deficit	$636.4 million	As of September 30, 2025
Workforce Reduction	Approximately 46%	Expected to be substantially complete by the end of the quarter ending December 31, 2025

Applied Therapeutics, Inc. announced a broad review of strategic alternatives, which may include potential business combinations, on November 20, 2025. The stock traded on NASDAQ under the ticker APLT, with a reported price of $0.23 at one point in late 2025.

Applied Therapeutics, Inc. (APLT) - Canvas Business Model: Channels

You're looking at how Applied Therapeutics, Inc. moves its clinical progress and potential products toward the market, which is all about regulatory clearance and getting the science out there. For a clinical-stage company focused on rare diseases, the channels are heavily weighted toward scientific communication and regulatory bodies right now, rather than broad commercial sales forces.

Regulatory Submissions (NDA/MAA) to the FDA and EMA

The primary channel for product approval is direct engagement with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). For govorestat (AT-007) in Classic Galactosemia, the New Drug Application (NDA) was submitted to the FDA in December 2023, and the Marketing Authorization Application (MAA) was validated by the EMA in December 2023. Following a Complete Response Letter (CRL) in November 2024, the Company completed a meeting with the FDA on September 29, 2025, to discuss the regulatory path forward. For Charcot-Marie-Tooth Sorbitol Dehydrogenase (CMT-SORD) Deficiency, Applied Therapeutics, Inc. completed a meeting with the FDA in the third quarter of 2025 to discuss the potential NDA submission strategy. The FDA provided constructive feedback on the data requirements needed to support a potential future NDA submission. A follow-up meeting with the FDA regarding the regulatory path for Classic Galactosemia is expected in the fourth quarter of 2025.

Clinical Trial Sites and Specialized Metabolic Centers

Clinical sites serve as the initial channel for data generation and patient access, especially for rare diseases. The Company is advancing govorestat for phosphomannomutase 2-congenital disorder glycosylation (PMM2-CDG) through an ongoing single-patient investigator-initiated trial. To support diagnosis and access for CMT-SORD, Applied Therapeutics, Inc. launched a sponsored Urine Sorbitol Assay in July 2025, which is available to healthcare providers at no cost for patients. The patient populations targeted by the pipeline candidates define the necessary centers of expertise:

• CMT-SORD affects approximately 3,300 patients in the U.S. and 4,000 in the EU.
• Classic Galactosemia affects over 3,000 patients in the United States.

Out-licensing Partners (e.g., Biossil) for Commercialization

For certain indications, out-licensing acts as the immediate commercialization channel, bringing in non-dilutive cash flow. In July 2025, Applied Therapeutics, Inc. entered an out-licensing agreement with Biossil, Inc. for AT-001, the investigational therapy for Diabetic Cardiomyopathy (DbCM). This deal provided an upfront payment of $1.0 million. This is separate from a prior agreement with Advanz Pharma, which included an upfront payment of EUR 10.0M (around $10.7M) and potential milestones up to EUR 134M. These partnerships are key for funding, though they do not cover the core rare disease franchises like CMT-SORD.

Scientific Publications and Conference Presentations (PNS, ASHG)

Dissemination of clinical data through peer-reviewed publications and major medical conferences is a critical channel for establishing scientific credibility and informing future regulatory strategy. Applied Therapeutics, Inc. presented full 12-month, topline 18-month, and 24-month data from the INSPIRE Phase 2/3 trial for CMT-SORD at the Peripheral Nerve Society (PNS) 2025 Annual Meeting in May 2025. The oral presentation was assigned number 597. Furthermore, results from the single-patient trial evaluating govorestat for PMM2-CDG were published in the Journal of Inherited Metabolic Disease (JIMD) and presented at the 2025 ASHG Annual Meeting in October 2025.

Specialty Pharmacies and Distribution Networks (future)

While full commercial distribution channels are likely future considerations pending regulatory approvals for the lead assets, the Company is already engaging with the infrastructure needed for patient support. The launch of the no-cost sponsored Urine Sorbitol Assay supports healthcare providers in identifying suspected CMT-SORD cases, which is a precursor to treatment distribution. The financial reality underpinning these channel activities shows a significant cash burn rate:

Financial Metric (as of Q2 2025 End)	Amount
Cash and Cash Equivalents (June 30, 2025)	$30.4 million
Cash and Cash Equivalents (September 30, 2025)	$11.9 million
R&D Expense (Q2 2025)	$9.9 million
General and Administrative Expense (Q2 2025)	$13.2 million
Net Loss (Six Months Ended June 30, 2025)	$43.2 million

The decrease in cash from $79.4 million at the end of 2024 to $11.9 million by September 30, 2025, highlights the reliance on non-dilutive funding from licensing deals to bridge the gap until potential product revenue.

Finance: draft 13-week cash view by Friday.

Applied Therapeutics, Inc. (APLT) - Canvas Business Model: Customer Segments

You're looking at the core groups Applied Therapeutics, Inc. (APLT) targets with its lead candidate, govorestat. These are specific, small patient pools, which is typical for a company focused on rare metabolic diseases. Honestly, the financial reality of late 2025 shows this focus is expensive; Q3 2025 revenue was only $1 million, against a net loss of $18.99 million, leaving cash and cash equivalents at just $11.9 million as of September 30, 2025. This means every patient interaction and potential partnership is critical.

The primary customer segments are defined by the rare diseases govorestat is designed to treat:

• Patients with CMT-SORD (Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency).
• Patients with Classic Galactosemia (pediatric and adult).
• Patients with PMM2-CDG (Congenital Disorder of Glycosylation).

Here's a breakdown of the estimated patient populations for the core indications, which directly informs the size of these segments:

Indication	Estimated US Patients	Estimated EU Patients
CMT-SORD	3,300	4,000
Classic Galactosemia	Approximately 3,000	Approximately 4,000

For Patients with CMT-SORD, the focus is on those in the ongoing INSPIRE Phase III trial; 90% of remaining INSPIRE patients (45 out of 50) are continuing on govorestat in the open-label extension, showing strong patient commitment to the treatment. The clinical rationale is strong, with preclinical models showing a 37 percent reduction of sorbitol in the sciatic nerve with govorestat.

For Patients with Classic Galactosemia, the segment is defined by the need for a treatment following the FDA's Complete Response Letter in late 2024. The company is scheduled to meet with the FDA in Q4 2025 to discuss the path forward. This patient group, including pediatric and adult populations, is identified rapidly due to mandatory newborn screening in the US and most EU countries.

The segment for Patients with PMM2-CDG is characterized by high unmet need, as it is a severe, rare disease with high mortality in pediatrics. Data from a single-patient trial showed a significant clinical improvement, with the Nijmegen Pediatric CDG Rating Scale (NPCRS) improving by 9 points (46%) over the treatment course.

Beyond the patients themselves, Applied Therapeutics, Inc. (APLT) targets professional intermediaries and potential partners:

• Specialist physicians (neurologists, metabolic disease specialists).
• Biopharmaceutical companies seeking to license assets.

The segment of Specialist physicians is small, given the ultra-rare nature of the diseases, but critical for diagnosis, trial recruitment, and future prescription volume. The company has made key leadership promotions, including Dottie Caplan to Executive Vice President, Patient Advocacy and Government Affairs, to strengthen these community and professional relationships.

The segment of Biopharmaceutical companies seeking to license assets is a financial necessity given the current burn rate; the Q3 2025 basic loss per share from continuing operations was $0.13. This segment represents a non-dilutive funding opportunity, as seen by the out-licensing agreement for AT-001 to Biossil, Inc..

Applied Therapeutics, Inc. (APLT) - Canvas Business Model: Cost Structure

The cost structure for Applied Therapeutics, Inc. (APLT) as of late 2025 is heavily weighted toward Research and Development and General and Administrative functions, reflecting its clinical-stage biopharmaceutical focus with no product sales or recurring revenue streams recognized in Q3 2025.

Key operating expenses for the third quarter ended September 30, 2025, are detailed below, showing a significant reduction compared to the prior year period.

Cost Category	Q3 2025 Amount (USD)	Q3 2024 Amount (USD)
Research and Development Expenses	$9.6 million	$14.8 million
General and Administrative Expenses	$8.2 million	$15.0 million
Net Loss	$18.99 million	$68.59 million

The decrease in Research and Development expenses from Q3 2024 to Q3 2025 was approximately $5.2 million. This reduction was primarily driven by lower clinical and preclinical expenses, stock-based compensation, and regulatory and other expenses. However, this decrease was partially offset by an overall increase in personnel, drug manufacturing and formulation expenses within R&D.

Personnel costs are a major component of the overall cost base, leading to significant restructuring. Applied Therapeutics, Inc. initiated cost-containment measures including a major workforce reduction.

• Workforce cut: approximately 46%.
• The reduction is expected to be substantially completed by the end of the quarter ending December 31, 2025.
• Applied Therapeutics, Inc. entered 2025 with 35 full-time employees.
• Of those, 18 employees were primarily engaged in Research and Development activities.

Legal and professional fees for regulatory and corporate matters are a significant driver of G&A costs. For instance, G&A expenses in Q2 2025 rose compared to Q2 2024 due to higher legal, professional, and personnel expenses. Furthermore, the decrease in G&A from Q3 2024 to Q3 2025 was primarily related to an increase in legal and litigation settlement expenses being offset by other factors.

Clinical trial expenses and drug manufacturing costs are embedded within the R&D spend. An overall increase in drug manufacturing and formulation expenses was noted as an offset to the R&D expense decrease between Q3 2024 and Q3 2025.

The company's cash position as of September 30, 2025, stood at $11.9 million, a reduction from $79.4 million at December 31, 2024, underscoring the necessity of the cost-containment measures.

Applied Therapeutics, Inc. (APLT) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of Applied Therapeutics, Inc. (APLT) as of late 2025. For a clinical-stage biopharma, revenue is often lumpy, tied to deals and milestones rather than consistent product sales, and that's exactly what the latest numbers show.

License revenue from out-licensing agreements is the most concrete recent income source. For the third quarter ended September 30, 2025, Applied Therapeutics, Inc. reported total revenue of $1.00 million. This was a significant jump from the $0.122 million reported in the same quarter of the prior year. The entire $1.0 million recognized in Q3 2025 was explicitly driven by license revenue from its Biossil Agreement. To be fair, Q2 2025 showed zero GAAP revenue, so this is clearly deal-dependent.

The structure of this revenue stream can be broken down:

• License revenue from out-licensing agreements: $1.0 million in Q3 2025.
• Revenue for the nine months ended September 30, 2025: $1.0 million.
• Revenue for the nine months ended September 30, 2024: $0.455 million.

Here's a quick look at how the recent quarterly revenue compares:

Period Ending	Reported Revenue (USD)	Year-over-Year Change
September 30, 2025 (Q3 2025)	$1.00 million	Up 719.67% from Q3 2024's $122.00 thousand
June 30, 2025 (Q2 2025)	Zero (GAAP)	Down from $0.14 million (GAAP) in Q2 2024
December 31, 2024 (Fiscal Year)	$455.00 thousand	-95.45% decrease from Fiscal Year 2023

Upfront and milestone payments from partnerships are embedded within that license revenue. The $1.0 million in Q3 2025 is directly attributed to the Biossil Agreement, which suggests it was either an upfront payment or a specific milestone achievement recognized during that quarter. Applied Therapeutics, Inc. also entered into an out-licensing agreement with Biossil, Inc. for AT-001 during Q2 2025.

Future royalties on commercialized out-licensed products are a potential stream, but the public filings don't detail the specific percentage or structure of these future payments tied to the AT-001 license, or any other potential deals.

Future product sales of govorestat post-approval are currently minimal/zero, as expected for a company awaiting regulatory decisions. The consensus estimate for Applied Therapeutics, Inc.'s revenue in the next reported quarter is $0.00 USD. The company continues to advance govorestat for CMT-SORD and is reviewing the development program for Classic Galactosemia.

Equity financing or debt to fund operations is reflected in the cash position, which is critical for funding the ongoing Research and Development expenses, which were $9.6 million for the three months ended September 30, 2025. You need to watch the cash burn; cash and cash equivalents stood at $11.9 million as of September 30, 2025, down significantly from $79.4 million at December 31, 2024. This cash level suggests the need to secure additional capital or partnerships to maintain operations through 2026, pending regulatory milestones.

Finance: draft 13-week cash view by Friday.