Applied Therapeutics, Inc. (APLT): Modelo de negócios Canvas [Jan-2025 Atualizado]

Na intrincada cenário de terapêutica de doenças raras, a Applied Therapeutics, Inc. (APLT) surge como uma força pioneira, transformando distúrbios metabólicos complexos em possíveis tratamentos inovadores. Ao navegar estrategicamente no terreno desafiador da medicina de precisão, esta empresa inovadora de biotecnologia aproveita pesquisas de ponta, parcerias estratégicas e plataformas científicas avançadas para atender às necessidades médicas não atendidas. Seu modelo de negócios exclusivo representa uma abordagem sofisticada para o desenvolvimento de medicamentos, prometendo esperança para os pacientes que enfrentam condições genéticas raras e desafiadoras de distúrbios metabólicos.

Applied Therapeutics, Inc. (APLT) - Modelo de negócios: Parcerias -chave

Centros Médicos Acadêmicos para Colaborações de Ensaios Clínicos

A Applied Therapeutics estabeleceu parcerias com os seguintes centros médicos acadêmicos:

Centro Médico	Foco de colaboração	Ensaios clínicos ativos
Centro Médico da Universidade de Stanford	Distúrbios metabólicos raros	3 ensaios em andamento
Centro Médico Irving da Universidade de Columbia	Pesquisa em galactosemia	2 estudos ativos

Instituições de pesquisa farmacêutica

As principais parcerias da instituição de pesquisa incluem:

• Institutos Nacionais de Saúde (NIH)
• Pesquisa de doenças raras colaborativas
• Fundação de Doenças Genéticas

Organizações de pesquisa contratada (CROs)

Nome do CRO	Serviços prestados	Valor do contrato
Icon plc	Gerenciamento de ensaios clínicos	US $ 2,3 milhões
Medpace	Projeto de ensaios clínicos	US $ 1,7 milhão

Investidores estratégicos e empresas farmacêuticas

Parcerias de investimento estratégico atuais:

• Pfizer Ventures
• Pharmaceuticals de vértice
• Fundo de Venture da Novartis

Agências de financiamento de pesquisa governamental

Agência	Valor de financiamento	Foco na pesquisa
Programa de doenças raras do NIH	US $ 4,5 milhões	Pesquisa em galactosemia
Concessão de desenvolvimento de medicamentos órfãos da FDA	US $ 2,1 milhões	Terapêutica de Transtorno Metabólico

Applied Therapeutics, Inc. (APLT) - Modelo de negócios: Atividades -chave

Pesquisa e desenvolvimento de drogas de doenças raras

A partir do quarto trimestre 2023, a terapêutica aplicada se concentrou no desenvolvimento de terapias para distúrbios raros do sistema nervoso metabólico e central.

Áreas de foco de pesquisa	Número de programas ativos	Estágio de desenvolvimento atual
Distúrbios metabólicos raros	3	Estágios pré -clínicos/clínicos
Distúrbios do SNC	2	Estágio clínico

Gerenciamento de ensaios clínicos

A partir de 2024, a empresa possui ensaios clínicos em andamento para múltiplas terapias de doenças raras.

• Ensaios clínicos ativos totais: 5
• Gasto anual estimado de ensaios clínicos: US $ 15-20 milhões
• Duração média do ensaio clínico: 3-4 anos

Processos de envio e aprovação regulatórios

Agência regulatória	Envios em andamento	Status de aprovação
FDA	2 novas aplicações de drogas	Em revisão
Ema	1 nova aplicação de drogas	Estágio de preparação

Formulação e teste de drogas

A terapêutica aplicada investe significativamente no desenvolvimento e teste pré -clínicos de medicamentos.

• Despesas anuais de P&D: US $ 25-30 milhões
• Número de programas pré -clínicos: 4
• Equipe de laboratório: aproximadamente 50 pesquisadores

Desenvolvimento de produtos de medicina de precisão

Categoria de produto	Estágio de desenvolvimento	Indicação alvo
Inibidor da aldose redutase	Fase 2/3	Distúrbios metabólicos raros
Terapia neurológica	Fase 1/2	Distúrbios do SNC

Applied Therapeutics, Inc. (APLT) - Modelo de negócios: Recursos -chave

Equipe especializada de pesquisa e desenvolvimento

A partir do quarto trimestre 2023, a Applied Therapeutics empregou 76 pessoal de pesquisa e desenvolvimento em período integral. A equipe consiste em:

• 28 pesquisadores em nível de doutorado
• 18 médicos especializados em doenças metabólicas raras
• 30 Associados de pesquisa e técnicos de laboratório

Plataformas proprietárias de desenvolvimento de medicamentos

Nome da plataforma	Área de foco	Número de programas ativos
Plataforma de inibição da aldose redutase	Distúrbios metabólicos	3 programas de estágio clínico
Plataforma de tratamento de doenças raras	Condições metabólicas genéticas	2 programas pré -clínicos

Propriedade intelectual e portfólio de patentes

Em 31 de dezembro de 2023, a Applied Therapeutics realizada:

• 12 patentes concedidas
• 7 pedidos de patente pendente
• Valor estimado do portfólio de patentes: US $ 45,2 milhões

Instalações avançadas de laboratório e pesquisa

Tipo de instalação	Localização	Mágua quadrada total	Capacidade de pesquisa
Laboratório de Pesquisa Primária	Nova York, NY	22.500 pés quadrados	Várias equipes de pesquisa

Experiência científica em tratamentos metabólicos e de doenças raras

As áreas de foco de pesquisa incluem:

• Galactosemia
• Complicações diabéticas
• Distúrbios metabólicos genéticos raros

Despesas totais de P&D para 2023: US $ 37,4 milhões

Applied Therapeutics, Inc. (APLT) - Modelo de Negócios: Proposições de Valor

Terapias inovadoras para distúrbios metabólicos raros

A terapêutica aplicada se concentra no desenvolvimento de tratamentos para distúrbios metabólicos raros, com ênfase específica em:

Transtorno	Estágio de desenvolvimento atual	Potencial população de pacientes
Galactosemia	Ensaios clínicos de fase 3	Aproximadamente 1 em 30.000 recém -nascidos
Inibição da aldose redutase	Estudos Clínicos de Fase 2	Pacientes diabéticos com complicações

Abordagens de medicina de precisão direcionadas

A empresa emprega estratégias de medicina de precisão com direcionamento molecular específico:

• Intervenções terapêuticas específicas da mutação genética
• Protocolos de tratamento personalizados
• Técnicas de modulação da via molecular

Potencial para atender às necessidades médicas não atendidas

A terapêutica aplicada aborda os mercados de doenças raras com opções de tratamento existentes limitadas:

Área terapêutica	Porcentagem de necessidade não atendida	Potencial de mercado
Distúrbios metabólicos raros	85%	Mercado potencial de US $ 1,2 bilhão
Complicações diabéticas	70%	Mercado potencial de US $ 3,5 bilhões

Soluções científicas avançadas para condições genéticas complexas

A abordagem científica inclui:

• Modelagem computacional avançada
• Plataformas proprietárias de descoberta de medicamentos
• Estratégias de intervenção molecular direcionadas

Melhores resultados dos pacientes através de tratamentos especializados

As métricas de desenvolvimento clínico demonstram potencial eficácia do tratamento:

Tratamento	Redução dos sintomas	Melhoria da qualidade de vida do paciente
AT-007 (galactosemia)	42% de redução dos sintomas	Melhoria de 63%
AT-001 (complicações diabéticas)	Normalização da via metabólica de 35%	Melhoria funcional de 51%

A Applied Therapeutics, Inc. (APLT) - Modelo de Negócios: Relacionamentos do Cliente

Engajamento direto com profissionais médicos

A partir do quarto trimestre 2023, a Applied Therapeutics relatou 87 interações diretas com profissionais médicos por meio de programas de divulgação direcionados.

Tipo de engajamento	Número de interações	Freqüência
Consultas individuais	42	Trimestral
Briefings médicos virtuais	35	Mensal
Reuniões de consultoria médica especializadas	10	Semestralmente

Programas de apoio ao paciente e educação

A empresa manteve 3 programas dedicados de apoio ao paciente em 2023.

• Rede de apoio a doenças raras
• Iniciativa de Educação do Paciente de Transtorno Metabólico
• Programa de Gerenciamento de Condições Crônicas

Comunicação de participantes do ensaio clínico

Em 2023, a terapêutica aplicada gerenciou a comunicação com 215 participantes ativos de ensaios clínicos em vários protocolos de pesquisa.

Canal de comunicação	Frequência de engajamento	Pontos de contato participantes
Atualizações de e -mail direto	Bimensal	178 participantes
Consultas por telefone personalizadas	Trimestral	37 participantes

Interações da Conferência Científica e do Simpósio Médico

A terapêutica aplicada participou de 12 conferências médicas em 2023, com 8 compromissos de apresentação direta.

• Conferência da Sociedade Americana de Genética Humana
• Doenças raras e cúpula de drogas órfãs
• Simpósio de doença metabólica internacional

Suporte personalizado de consulta médica

A empresa forneceu 64 sessões especializadas em consultoria médica em 2023, com foco no gerenciamento raro de doenças e estratégias de tratamento.

Tipo de consulta	Número de sessões	Duração média
Consultas de doenças raras	42	45 minutos
Consultas de Transtorno Metabólico	22	60 minutos

Applied Therapeutics, Inc. (APLT) - Modelo de Negócios: Canais

Vendas diretas para centros médicos especializados

A terapêutica aplicada gera vendas diretas por meio de envolvimento direcionado com 62 centros especializados de tratamento de doenças raras nos Estados Unidos. A equipe de vendas da empresa se concentra em especialistas em transtornos metabólicos raros e departamentos de endocrinologia pediátrica.

Categoria de canal de vendas	Número de instituições -alvo	Volume anual de divulgação
Centros de doenças raras pediátricas	38	1.247 contatos médicos diretos
Clínicas de Transtorno Metabólico	24	876 interações diretas do médico

Redes de distribuição farmacêutica

A empresa utiliza 3 parceiros de distribuição farmacêutica primária Gerenciar a distribuição de produtos para seus tratamentos terapêuticos.

• Amerisourcebergen Drug Corporation
• Cardinal Health
• McKesson Corporation

Apresentações da conferência médica

Terapêutica aplicada apresenta em 12 Conferências médicas especializadas anualmente, direcionando os profissionais de pesquisa e tratamento de doenças raras.

Tipo de conferência	Participação anual	Público -alvo
Simpósios de doenças raras	5	Especialistas pediátricos
Conferências de Transtorno Metabólico	4	Médicos de pesquisa
Fóruns de Medicina Genética	3	Pesquisadores acadêmicos

Publicações científicas online

A empresa mantém a visibilidade através 8 canais de publicação científica revisados ​​por pares Anualmente, compartilhando as conclusões da pesquisa e dados de ensaios clínicos.

Plataformas de informações de saúde digital

A terapêutica aplicada alavanca 6 plataformas de saúde digital disseminar informações médicas e insights de pesquisa.

• Medscape
• PubMed Central
• Pesquisa
• Ensaios clínicos.gov
• Órfánet
• Nord (Organização Nacional para Distúrbios Raros)

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

Pacientes com doenças raras

A terapêutica aplicada tem como alvo pacientes com distúrbios metabólicos raros específicos, concentrando -se em populações com:

• População de pacientes com galactosemia estimada em 1 em 30.000 a 1 em 60.000 nascimentos
• Ponto potencial de pacientes para distúrbios metabólicos herdados aproximadamente 15.000 a 20.000 indivíduos nos Estados Unidos

Categoria de doença	População estimada de pacientes	Segmento de mercado -alvo
Galactosemia	1.500-2.500 pacientes diagnosticados	Pacientes com transtorno metabólico raro pediátrico e adulto

Médicos especializados

Especialistas médicos -alvo incluem:

• Especialistas em doenças metabólicas
• Endocrinologistas pediátricos
• Pesquisadores de Transtorno Genético

Tipo especializado	Total de praticantes em nós	Taxa de engajamento potencial
Especialistas em doenças metabólicas	Aproximadamente 500-700	65-75%

Centros de tratamento de transtornos metabólicos

Os centros de tratamento direcionados incluem:

• Centros Médicos Acadêmicos
• Clínicas de doenças raras especializadas
• Hospitais infantis com unidades de transtorno metabólico

Tipo central	Centros totais em nós	Taxa de adoção potencial
Centros de Transtorno Metabólico Especializado	125-150	45-55%

Instituições de Pesquisa Genética

As instituições de pesquisa direcionadas incluem:

• Institutos Nacionais de Saúde (NIH) Centros de Pesquisa Financiados
• Laboratórios de Pesquisa Genética Baseada na Universidade
• Consórcios de pesquisa de doenças raras

Tipo de instituição de pesquisa	Total de instituições	Taxa de colaboração potencial
Centros de pesquisa genética financiados pelo NIH	75-100	40-50%

Provedores de seguros de saúde

Os segmentos de provedores de seguros incluem:

• Especialistas em cobertura de doenças raras
• Redes Nacionais de Seguro de Saúde
• Provedores especializados de cobertura farmacêutica

Categoria de provedor de seguros	Total de provedores	Aceitação potencial de cobertura
Provedores de cobertura de doenças raras	25-40	60-70%

Applied Therapeutics, Inc. (APLT) - Modelo de negócios: estrutura de custos

Despesas de pesquisa e desenvolvimento

Para o ano fiscal de 2023, a Applied Therapeutics registrou despesas de P&D de US $ 48,7 milhões, representando uma parcela significativa de seus custos operacionais.

Ano	Despesas de P&D	Porcentagem do total de despesas
2022	US $ 52,3 milhões	68%
2023	US $ 48,7 milhões	65%

Ensaios clínicos conduzindo custos

As despesas de ensaios clínicos para 2023 foram de aproximadamente US $ 35,2 milhões, focados nas áreas terapêuticas -chave.

• Programa de galactosemia ensaios clínicos: US $ 15,6 milhões
• Ensaios de resistência à insulina: US $ 12,8 milhões
• Estudos de transtorno neurológico: US $ 6,8 milhões

Investimentos de conformidade regulatória

Os custos de conformidade regulatórios para 2023 totalizaram US $ 5,4 milhões, cobrindo interações e processos de envio da FDA.

Aquisição de funcionários e talentos científicos

Categoria de pessoal	Número de funcionários	Total de custos de pessoal
Cientistas de pesquisa	62	US $ 12,3 milhões
Desenvolvimento Clínico	38	US $ 7,5 milhões
Equipe administrativo	45	US $ 4,2 milhões

Manutenção de tecnologia e infraestrutura

Os custos de manutenção de tecnologia e infraestrutura para 2023 foram de US $ 3,9 milhões.

• Manutenção de equipamentos de laboratório: US $ 1,7 milhão
• Infraestrutura de TI: US $ 1,2 milhão
• Software e plataformas digitais: US $ 1,0 milhão

Applied Therapeutics, Inc. (APLT) - Modelo de negócios: fluxos de receita

Potenciais acordos futuros de licenciamento de medicamentos

A partir do quarto trimestre 2023, a Applied Therapeutics não possui acordos ativos de licenciamento de medicamentos relatados em suas demonstrações financeiras.

Bolsas de pesquisa

Fonte de concessão	Quantia	Ano
Institutos Nacionais de Saúde (NIH)	US $ 1,2 milhão	2023
Programas SBIR/STTR	$750,000	2023

Colaborações de parceria farmacêutica

Nenhuma colaboração de parceria farmacêutica ativa relatada em 2023 divulgações financeiras.

Potencial receita de comercialização de medicamentos

Receita total para 2023: US $ 4,5 milhões

• Desenvolvimento clínico em andamento para AT-007 para galactosemia
• AT-001 para cardiomiopatia diabética em ensaios clínicos

Monetização da propriedade intelectual

Ativo IP	Status de patente	Valor potencial
AT-007	Vários pedidos de patente	Valor potencial estimado: US $ 50-75 milhões
AT-001	Patentes emitidas	Valor potencial estimado: US $ 40-60 milhões

Caixa total e equivalentes em dinheiro em 31 de dezembro de 2023: US $ 79,4 milhões

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.