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

A Gain Therapeutics, Inc. (GANX) surge como uma empresa inovadora de biotecnologia, revolucionando o cenário do tratamento raro de doenças genéticas por meio de sua inovadora plataforma See-TX. Ao direcionar com precisão os mecanismos de desdobramento de proteínas, essa empresa pioneira está na vanguarda do desenvolvimento de terapêuticas transformadoras para distúrbios neurológicos complexos, oferecendo esperança a pacientes e pesquisadores. Seu modelo de negócios abrangente representa uma abordagem estratégica para atender às necessidades médicas não atendidas, combinando conhecimentos científicos de ponta com recursos tecnológicos robustos que poderiam potencialmente remodelar o futuro da medicina genética personalizada.

Gain Therapeutics, Inc. (GANX) - Modelo de negócios: Parcerias -chave

Colaborações com instituições de pesquisa acadêmica

A partir de 2024, a Gain Therapeutics estabeleceu parcerias com as seguintes instituições de pesquisa acadêmica:

Instituição	Foco na pesquisa	Status de colaboração
Universidade da Califórnia, São Francisco	Pesquisa em doenças neurológicas	Parceria ativa
Universidade de Stanford	Estudos de desdobramento de proteínas	Colaboração em andamento

Parcerias de desenvolvimento farmacêutico estratégico

A Gain Therapeutics desenvolveu parcerias estratégicas de desenvolvimento farmacêutico com:

• Takeda Pharmaceutical Company Limited
• Biogen Inc.
• Novartis International AG

Detalhes da parceria -chave:

Parceiro	Valor da parceria	Área de foco
Takeda Pharmaceutical	Contrato de colaboração de US $ 12,5 milhões	Doenças genéticas raras
Biogen Inc.	Colaboração de pesquisa de US $ 8,3 milhões	Distúrbios neurodegenerativos

Acordos de licenciamento em potencial com empresas de biotecnologia

Status do contrato de licenciamento atual:

Empresa de biotecnologia	Tipo de licença	Valor potencial
Enzyme Therapeutics Inc.	Licença mundial exclusiva	US $ 15 milhões em potencial pagamento inicial
Genômica de precisão	Licença de tecnologia não exclusiva	US $ 5,2 milhões em potenciais pagamentos marcantes

Parcerias de rede de pesquisa e ensaios clínicos

A Gain Therapeutics estabeleceu parcerias com as seguintes redes de ensaios clínicos:

• Organização de pesquisa clínica do ICON PLC
• Parexel International Corporation
• Iqvia Holdings Inc.

Rede de ensaios clínicos	Contagem atual de julgamento	Duração da parceria
Icon plc	3 ensaios clínicos ativos	Contrato de 2 anos
Parexel International	2 estudos de pesquisa em andamento	Contrato de colaboração de três anos

Gain Therapeutics, Inc. (GANX) - Modelo de negócios: Atividades -chave

Desenvolvimento de pequenas moléculas terapêuticas para doenças incorretas de proteínas

A partir do quarto trimestre 2023, a Gain Therapeutics se concentra no desenvolvimento de pequenas moléculas terapêuticas direcionadas a doenças específicas de desdobramento de proteínas, com foco primário em:

• Doença de Alzheimer
• Doença de Parkinson
• Doença de Gaucher

Alvo de doença	Estágio de pesquisa atual	Custo estimado de desenvolvimento
Alzheimer	Pré -clínico	US $ 8,5 milhões
Parkinson's	Pré -clínico	US $ 7,2 milhões
Gaucher	Ensaios clínicos de fase I	US $ 12,3 milhões

Condução de pesquisa pré -clínica e clínica

Investimento de pesquisa para 2023-2024: US $ 15,6 milhões

• Estudos pré -clínicos em andamento para vários candidatos terapêuticos
• Ensaios clínicos ativos para candidatos a medicamentos principais
• Colaboração com instituições de pesquisa acadêmica

Avançar os processos de descoberta e triagem de medicamentos

Método de descoberta de medicamentos	Investimento anual	Taxa de sucesso
Plataforma See-tx	US $ 4,2 milhões	Identificação alvo de 62%
Triagem de alto rendimento	US $ 3,7 milhões	Validação composta de 45%

Buscando aprovações regulatórias da FDA

Orçamento de envio regulatório: US $ 2,9 milhões

• Preparando aplicativos de novos medicamentos para investigação (IND)
• Conduzindo estudos de segurança e eficácia exigidos pela FDA

Desenvolvimento e Gerenciamento da Propriedade Intelectual

Categoria de patentes	Número de patentes	Custo anual de gerenciamento de IP
Terapêutica de pequenas moléculas	12 patentes ativas	US $ 1,5 milhão
Plataforma de descoberta de medicamentos	5 patentes pendentes	$650,000

Gain Therapeutics, Inc. (GANX) - Modelo de negócios: Recursos -chave

Tecnologia proprietária de plataforma see-tx

Gain Therapeutics utiliza um Avaliação de eventos baseada em estrutura e terapêutica (ver-tx) Tecnologia da plataforma projetada para descobrir e desenvolver terapias de pequenas moléculas para distúrbios incorretos de proteínas.

Característica da plataforma	Detalhes específicos
Tipo de tecnologia	Plataforma de triagem computacional proprietária
Capacidade de triagem	Modelagem molecular avançada e análise computacional
Status de patente	Múltiplas patentes pendentes e concedidas

Experiência científica em distúrbios incorrentes de proteínas

A empresa mantém uma equipe científica especializada com experiência em:

• Distúrbios de desdobramento de proteínas neurológicas
• Técnicas avançadas de descoberta de medicamentos computacionais
• Biologia Molecular e Bioquímica

Instalações de pesquisa e desenvolvimento

Gain Therapeutics opera instalações de pesquisa focadas em doenças genéticas raras e distúrbios neurológicos.

Característica da instalação	Detalhes
Localização	San Diego, Califórnia
Foco na pesquisa	Distúrbios neurológicos genéticos raros
Investimento de equipamentos de laboratório	Estimado US $ 2,5 milhões em infraestrutura de pesquisa especializada

Recursos de triagem molecular especializados

A empresa aproveita tecnologias avançadas de triagem computacional para identificar possíveis compostos terapêuticos.

• Modelagem molecular de alto rendimento
• Algoritmos de triagem de inteligência artificial
• Técnicas de análise de estrutura de proteínas

Portfólio de propriedade intelectual

Categoria IP	Número de ativos	Valor estimado
Aplicações de patentes	12	Estimado US $ 5-7 milhões
Patentes concedidas	6	Estimado US $ 3-4 milhões
Tecnologias de triagem proprietárias	3 plataformas exclusivas	Estimado US $ 2-3 milhões

Gain Therapeutics, Inc. (GANX) - Modelo de negócios: proposições de valor

Soluções terapêuticas inovadoras para doenças genéticas raras

Gain Therapeutics se concentra em doenças genéticas raras com mecanismos de direcionamento específicos. A partir do quarto trimestre de 2023, a empresa possui três candidatos a medicamentos primários em desenvolvimento.

Candidato a drogas	Doença alvo	Estágio de desenvolvimento
GBA-par	Doença de Gaucher	Pré -clínico
SE-101	Doença de Parkinson	Fase 1/2
SE-103	Doença de Alzheimer	Pré -clínico

Direcionamento de precisão de mecanismos de desdobramento de proteínas

A plataforma de See-tx proprietária da empresa permite intervenção precisa de dobramento de proteínas com 93% de precisão computacional na identificação do alvo.

• Plataforma de descoberta de medicamentos computacional
• Segmentação de proteínas baseada em aprendizado de máquina
• Triagem molecular de alta precisão

Possíveis tratamentos inovadores para distúrbios neurológicos

Investimento em pesquisa em transtorno neurológico: US $ 12,4 milhões alocados em 2023 ano fiscal.

Transtorno	Investimento em pesquisa	Potencial população de pacientes
Doença de Parkinson	US $ 5,2 milhões	1,5 milhão de pacientes nos EUA
Doença de Alzheimer	US $ 4,7 milhões	6,7 milhões de pacientes nos EUA
Doença de Gaucher	US $ 2,5 milhões	20.000 pacientes nos EUA

Plataforma avançada de descoberta de medicamentos

Recursos da plataforma See-tx:

• Triagem computacional de 1,2 milhão de estruturas de proteínas
• Algoritmos de aprendizado de máquina com 87% de precisão preditiva
• Identificação rápida de possíveis alvos terapêuticos

Abordagem personalizada para abordar condições genéticas complexas

Métricas de personalização para 2023:

Métrica	Valor
Capacidade de análise de variantes genéticas	350.000 perfis genéticos únicos
Precisão de modelagem computacional	Previsão de interação molecular de 95%
Tentativas de design de medicamentos específicas para pacientes	127 intervenções moleculares direcionadas

Gain Therapeutics, Inc. (GANX) - Modelo de Negócios: Relacionamentos do Cliente

Engajamento direto com a comunidade de pesquisa médica

A partir do quarto trimestre 2023, a Gain Therapeutics relatou estratégias de engajamento direto com 47 instituições de pesquisa acadêmica e 12 centros de pesquisa farmacêutica globalmente.

Tipo de engajamento	Número de interações	Alcance geográfico
Colaborações de pesquisa	37	América do Norte, Europa
Reuniões consultivas científicas	22	Internacional

Colaboração com grupos de defesa de pacientes

Gain Therapeutics manteve parcerias ativas com 8 organizações de defesa de pacientes focadas em distúrbios neurológicos.

• Redes de pacientes com doenças raras: 5
• Grupos de doenças neurodegenerativas: 3

Conferência Científica e Participação de Eventos da Indústria

Em 2023, a Gain Therapeutics participou de 15 conferências científicas internacionais.

Tipo de conferência	Número de conferências	Formato de apresentação
Conferências de neurociência	7	Apresentações orais
Simpósios de doenças raras	5	Sessões de pôster
Fóruns de pesquisa farmacêutica	3	Painéis de discussão

Comunicação transparente do progresso da pesquisa

Gain Therapeutics publicou 12 atualizações de pesquisa e 4 publicações revisadas por pares em 2023.

• Relatórios trimestrais de pesquisa: 4
• Atualizações intermediárias de ensaios clínicos: 8

Estratégias de comunicação digital e científica

Métricas de engajamento digital para 2023 incluídas:

Plataforma digital	Seguidores/assinantes	Taxa de engajamento
LinkedIn	4,200	3.7%
Webinars científicos	1.800 participantes registrados	Taxa de participação de 62%
Newsletter de e -mail	2.500 assinantes	28% de taxa de abertura

Gain Therapeutics, Inc. (GANX) - Modelo de Negócios: Canais

Publicações científicas diretas

A partir do quarto trimestre 2023, Gain Therapeutics publicou 3 artigos científicos revisados ​​por pares em periódicos, incluindo:

• Jornal de Neuroquímica
• Neurodegeneração molecular
• Pesquisa de Alzheimer & Terapia

Biotecnologia e conferências médicas

Conferência	Data	Tipo de participação
Conferência de Saúde JP Morgan	Janeiro de 2024	Apresentador
Conferência Internacional da Associação de Alzheimer	Julho de 2023	Apresentação de pôsteres
Reunião da Associação Neurológica Americana	Setembro de 2023	Vitrine de pesquisa

Comunicações de Relações com Investidores

Métricas trimestrais de relatórios financeiros:

• 4 Apresentações de chamadas de ganhos em 2023
• 12 webinars de investidores
• Distribuição trimestral de cartas dos acionistas

Plataformas digitais e redes científicas

Plataformas de engajamento online:

• Seguidores do LinkedIn: 2.437
• Seguidores do Twitter: 1.856
• ResearchGate Publications: 7
• Site corporativo Visitantes mensais: 5.200

Parcerias da indústria farmacêutica

Parceiro	Foco de colaboração	Data de iniciação
Biogênio	Pesquisa de doenças neurodegenerativas	Março de 2023
Eli Lilly	Abordagem terapêutica incapaz de proteínas	Novembro de 2023

Gain Therapeutics, Inc. (GANX) - Modelo de negócios: segmentos de clientes

Pacientes com transtorno neurológico

A partir de 2024, o ganho de terapêutica tem como alvo pacientes com distúrbios neurológicos específicos, concentrando -se em condições genéticas raras.

Grupo de pacientes	População estimada	Transtorno alvo
Pacientes com Transtorno de Armazenamento Lisossômico	Aproximadamente 50.000 globalmente	Doença de Gaucher
Pacientes de variantes genéticas da doença de Parkinson	Aproximadamente 10-15% dos casos totais de Parkinson	Mutação do gene GBA

Comunidade de pesquisa de doenças genéticas raras

Ganhar terapêutica colabora com redes de pesquisa especializadas.

• Número de parcerias de pesquisa ativa: 7
• Instituições de pesquisa envolvidas: 12
• Orçamento anual de colaboração de pesquisa: US $ 3,2 milhões

Empresas farmacêuticas e de biotecnologia

Tipo de empresa	Valor potencial de colaboração	Parcerias atuais
Empresas farmacêuticas de doenças raras	US $ 5 a 10 milhões por colaboração	3 parcerias ativas
Empresas de pesquisa de biotecnologia	US $ 2-6 milhões por engajamento	5 colaborações em andamento

Instituições de pesquisa acadêmica

Gain Therapeutics mantém relações estratégicas de pesquisa acadêmica.

• Total de parcerias acadêmicas: 9
• Pesquisa Anual Grant Financiamento: US $ 1,7 milhão
• Principais áreas de foco de pesquisa: distúrbios neurológicos, engenharia enzimática

Provedores de saúde especializados em distúrbios genéticos

Tipo de provedor	Alcance potencial do paciente	Estratégia de engajamento
Clínicas genéticas especializadas	Aproximadamente 250 clínicas em todo o país	Desenvolvimento direto de consulta e protocolo de tratamento
Centros de tratamento de neurologia	Mais de 500 centros nos mercados -alvo	Abordagem terapêutica colaborativa

Gain Therapeutics, Inc. (GANX) - Modelo de negócios: estrutura de custos

Despesas de pesquisa e desenvolvimento

Para o ano fiscal encerrado em 31 de dezembro de 2023, a Gain Therapeutics registrou despesas de P&D de US $ 14,7 milhões.

Ano fiscal	Despesas de P&D	Mudança de ano a ano
2022	US $ 11,2 milhões	+31.3%
2023	US $ 14,7 milhões	+31.3%

Investimentos de ensaios clínicos

Os investimentos em ensaios clínicos para a terapêutica de ganho em 2023 totalizaram aproximadamente US $ 8,5 milhões.

• Ensaios de Fase 1 para GAN-0058 para a doença de Parkinson
• Estudos pré -clínicos em andamento para doenças genéticas raras
• Programas de pesquisa de transtornos neurológicos

Manutenção da propriedade intelectual

Os custos anuais de manutenção da propriedade intelectual foram de US $ 1,2 milhão em 2023.

Categoria IP	Número de patentes	Custo de manutenção anual
Plataforma de direcionamento molecular	12	$650,000
Candidatos específicos de drogas	8	$550,000

Aquisição de funcionários e talentos científicos

As despesas totais de pessoal para 2023 foram de US $ 7,3 milhões.

• Compensação média da equipe científica: US $ 185.000 por ano
• Total de funcionários: 42
• Custos de recrutamento: US $ 420.000

Desenvolvimento e manutenção da plataforma de tecnologia

Os custos da plataforma de tecnologia em 2023 atingiram US $ 3,6 milhões.

Componente de tecnologia	Custo de desenvolvimento	Custo de manutenção
Plataforma See-tx	US $ 2,1 milhões	$850,000
Infraestrutura computacional	US $ 1,2 milhão	$550,000

Gain Therapeutics, Inc. (GANX) - Modelo de negócios: fluxos de receita

Potenciais acordos futuros de licenciamento de medicamentos

A partir do quarto trimestre 2023, a Gain Therapeutics ainda não relatou nenhum acordos ativos de licenciamento de medicamentos. A receita total potencial de licenciamento permanece não especificada.

Bolsas de pesquisa e financiamento

Ano	Fonte de concessão	Quantia
2023	Institutos Nacionais de Saúde (NIH)	$487,000
2022	Pesquisa de Inovação em Pequenas Empresas (SBIR)	$256,000

Parcerias de pesquisa colaborativa

Os parceiros atuais de colaboração de pesquisa incluem:

• Universidade da Califórnia, São Francisco
• Hospital Geral de Massachusetts

Vendas potenciais de produtos terapêuticos

O pipeline atual se concentrou em doenças genéticas raras, sem vendas comerciais relatadas a partir de 2024.

Estratégias de monetização da propriedade intelectual

Categoria de patentes	Número de patentes	Valor potencial
Terapias de doenças raras	7	Não revelado
Plataformas de pequenas moléculas	4	Não revelado

Receita total para 2023: $ 743.000

Gain Therapeutics, Inc. (GANX) - Canvas Business Model: Value Propositions

You're looking at the core value Gain Therapeutics, Inc. (GANX) offers to patients and the market as of late 2025. It centers on delivering a true disease-modifying approach, not just masking symptoms for conditions like Parkinson's disease (PD).

Potential disease-modifying therapy for Parkinson's, not just symptomatic relief.

• Preclinical data in rodent models of both GBA1-PD and idiopathic PD demonstrated a disease-modifying effect, including rescuing deficits in motor function and gait.
• Early clinical findings from the Phase 1b study suggested a disease-slowing effect, shown by stabilization and improvement in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) scores appearing after approximately 30 days of administration.
• The company is planning for Phase 2 studies in the US and EU in the second half of 2025.

Orally administered, brain-penetrant small molecule (GT-02287).

The lead candidate, GT-02287, is an orally administered, brain-penetrant small molecule, which is a significant convenience factor compared to infusions or injections for chronic neurological conditions.

Targeting the underlying cause (GCase misfolding) for both GBA1 and idiopathic PD.

GT-02287 acts as an allosteric enzyme modulator designed to restore the function of the lysosomal enzyme glucocerebrosidase (GCase). This mechanism addresses the root issue of GCase misfolding, which is implicated in both genetically defined PD (GBA1-PD) and idiopathic PD. Results from the Phase 1 study in healthy volunteers showed target engagement with a >50% increase in glucocerebrosidase (GCase) activity among those receiving clinically relevant doses.

The market opportunity for this mechanism is substantial, with the US Market Potential for GBA1-Parkinson's Disease estimated at $3B and the overall US Parkinson's Disease market potential at $4B.

Accelerated discovery of novel allosteric modulators for difficult-to-drug targets.

The discovery engine is powered by the proprietary Magellan AI platform. This technology has already yielded multiple assets, with patent applications for 5 NCE families (New Chemical Entities) currently under review.

Pipeline expansion potential across multiple lysosomal storage disorders and oncology.

The core GCase modulation platform extends beyond PD. GT-02287 has further potential in Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease. Also, Gain Therapeutics has multiple undisclosed preclinical assets targeting other areas.

• Lysosomal Storage Disorders targets include enzymes like GALC and GLB1.
• Metabolic Diseases targets include AAT.
• Oncology targets include Solid Tumors with a focus on DDR2.

Here's a quick look at the clinical progress and the financial footing as of the third quarter of 2025:

Metric	Value/Status as of Late 2025
GT-02287 Phase 1b Enrollment (as of Sept 30, 2025)	21 participants (surpassed target of 15)
Phase 1b Study Extension Rate	Approximately 80% of participants elected to continue for 12 months
Key Data Readout Expected	90-day functional changes and biomarker activity analysis in Q4 2025
GT-02287 Patent Term (Composition of Matter)	Through 2038 (excluding extensions)
Cash Position (as of Sept 30, 2025)	$8.8 million
Shares Outstanding (as of Nov 2025)	36.0 million
Net Loss (3 months ended Sept 30, 2025)	$0.15 per share, basic and diluted

The company is defintely moving forward with its next steps, aiming for an IND Submission in the second half of 2025. Finance: review the burn rate against the $8.8 million cash on hand by next week.

Gain Therapeutics, Inc. (GANX) - Canvas Business Model: Customer Relationships

You're a clinical-stage company, so your most critical relationships revolve around the science and the capital required to prove it. For Gain Therapeutics, Inc., this means intense focus on the investigators running the trials and the investors funding the runway.

High-touch engagement with clinical investigators and trial participants

Engagement here is deep because the Phase 1b study for GT-02287 in Parkinson's disease is the core value driver. The company actively manages these relationships, evidenced by exceeding initial enrollment targets and securing extensions. The commitment from participants is high, which speaks to the perceived value of the trial protocol.

• Completed enrollment of 21 participants in the Phase 1b study, exceeding the target of 15.
• Received approval from Australian health authorities to extend treatment duration to a total of 12 months.
• Approximately 80% of eligible participants elected to transition into the study extension.
• Initial data showing stabilization/improvement in MDS-UPDRS scores after approximately 30 days was presented in October 2025.

The President and CEO, Gene Mack, expressed deep gratitude to the patients and investigators for their commitment to advancing the program, underscoring the collaborative nature of this relationship.

Direct communication with investors via earnings calls and virtual KOL events

Investor relations is highly event-driven, focusing on translating clinical milestones into digestible financial and scientific updates. The cadence of communication is tied directly to data readouts and financial reporting periods. The company completed an underwritten public offering in Q3 2025, netting approximately $7.1 million in proceeds, which directly impacts the capital relationship.

Key investor and Key Opinion Leader (KOL) touchpoints in late 2025 included:

• Reporting Q3 2025 financial results on November 12, 2025.
• Hosting a Virtual KOL Event on October 9, 2025, to discuss initial Phase 1b data.
• Presenting preclinical data at Neuroscience 2025 on November 20, 2025.
• Attending the 2025 Maxim Growth Summit on October 16, 2025.

The EPS for the quarter ending September 30, 2025, was reported at negative $0.15, meeting consensus estimates.

Here's a quick look at the recent engagement activity:

Metric/Event	Value/Date	Context
Q3 2025 EPS	-$0.15	Met consensus estimate
Phase 1b Enrollment	21 participants	Surpassed target of 15
Study Extension Rate	Approx. 80% participation	High commitment from subjects
Virtual KOL Event	October 9, 2025	Focus on initial GT-02287 data
Cash & Cash Equivalents (Sept 30, 2025)	$8.8 million	Capital position post-offering

Collaborative relationships with patient advocacy and disease foundations

While specific dollar amounts for foundation partnerships aren't public, the focus on Parkinson's disease necessitates engagement with the patient community to drive awareness and trial recruitment. The company's success in enrolling and retaining participants in the Phase 1b study suggests a functional, if not formally structured, relationship with advocacy networks that support patients seeking novel treatment options.

Business development focus on potential strategic partners (licensing/M&A)

The primary business development driver is the IND submission to the FDA, expected by year-end 2025, which is the gateway to Phase 2 development, including U.S. clinical sites. This milestone is crucial for attracting potential strategic partners for licensing or acquisition, as it de-risks the asset by moving it into a more advanced regulatory pathway. The company's Magellan drug discovery platform, which can screen 5 trillion compounds in less than 3 months, is a key asset for in-licensing or platform-based deals, though no specific deal values were reported as of late 2025.

The next major data readout, the Day-90 functional and biomarker analysis, is expected in Q4 2025, which will be the next key inflection point for business development discussions.

Finance: draft 13-week cash view by Friday.

Gain Therapeutics, Inc. (GANX) - Canvas Business Model: Channels

You're looking at how Gain Therapeutics, Inc. gets its science and its corporate story out to the world-from the clinic to the capital markets. For a clinical-stage company, the channels are all about execution and communication. Here's the quick math on how they are connecting with investigators, the scientific community, and investors as of late 2025.

Clinical Trial Execution and Geographic Reach

The primary channel for validating the lead candidate, GT-02287, is the ongoing Phase 1b clinical trial. This trial is currently centered in Australia, utilizing 7 clinical sites. Enrollment for this study, which evaluates safety and tolerability in Parkinson's disease patients with or without GBA1 mutations, was completed in the third quarter of 2025, reaching 21 participants, exceeding the initial target of 15. The channel for continued patient engagement is the study extension, approved by Australian health authorities, allowing participants to continue treatment for up to a total of 12 months. The planned expansion channel is the anticipated IND submission to the FDA by year end 2025, which is the gateway to initiating Phase 2 development and opening clinical sites in the United States.

The key milestones related to this clinical channel can be mapped out:

• Phase 1b enrollment completion: Q3 2025.
• Initial data presentation (MDS conference): October 2025.
• 90-day analysis expected: Q4 2025.
• FDA IND submission target: Year end 2025.

Scientific Data Dissemination

To communicate scientific progress, Gain Therapeutics, Inc. uses established industry forums. This is where the data, generated from the Magellan drug discovery platform and the GT-02287 trial, reaches peers and potential collaborators. For instance, preclinical data on GT-02287 was presented at the Society for Neuroscience annual meeting, Neuroscience 2025, held November 15th-19th in San Diego, CA. Furthermore, the company participated in the Drug Discovery Innovation Programme (DDIP) 2025 in Barcelona, Spain, on September 25th - 26th. These events serve as critical touchpoints for scientific validation.

Here is a look at the key scientific communication channels used through late 2025:

Channel/Event	Date	Location/Focus	Data Type
International Congress of Parkinson's Disease and Movement Disorders®	October 2025	Honolulu, HI	Initial Phase 1b clinical findings
Neuroscience 2025	November 15th-19th, 2025	San Diego, CA	Preclinical GT-02287 Data
Drug Discovery Innovation Programme (DDIP) 2025	September 25th - 26th, 2025	Barcelona, Spain	AI and Automation in Drug Discovery Talk

Corporate and Financial Disclosure

For the investment community, the channels are formal and regulated. The corporate website is the central hub for public disclosures, offering documents like the Q3 2025 Investor Factsheet and the December 2025 Corporate Presentation. Regulatory filings are the bedrock of financial transparency. The Q3 2025 financial results were released on November 12, 2025. As of September 30, 2025, cash and cash equivalents stood at $8.8 million. To bolster liquidity for future operations, Gain Therapeutics, Inc. filed a shelf registration statement on Form S-3 effective November 26, 2025, allowing for an 'at the market offering' program up to $35,530,980.56. To give you context on the shareholder base, there were 35,976,390 shares outstanding as of September 30, 2025. The company is definitely using these financial channels to manage its runway, stating management expressed substantial doubt about continuing as a going concern beyond the first quarter of 2026 without additional financing.

Partnering and Business Development Outreach

Direct outreach channels focus on securing strategic alliances for the Magellan platform or pipeline assets beyond GT-02287, which has potential in Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease. A key engagement channel for building scientific credibility with potential industry partners and prescribers is hosting Key Opinion Leader (KOL) events. Gain Therapeutics hosted a virtual KOL event on October 14th, 2025. The platform itself, which integrates AI, physics-based methods, and supercomputing to screen libraries with 5 trillion compounds in less than 3 months, is a core asset being channeled for potential licensing deals.

• KOL Event Date: October 14, 2025.
• Platform Capability: Discovery in under 3 months.
• Pipeline Breadth: GT-02287 plus multiple undisclosed preclinical assets.

Finance: draft 13-week cash view by Friday.

Gain Therapeutics, Inc. (GANX) - Canvas Business Model: Customer Segments

You're looking at the core groups that drive the value for Gain Therapeutics, Inc. (GANX) right now, focusing on the patients they target and the capital providers that fund the journey.

The primary patient segment is centered on neurodegenerative diseases, specifically Parkinson's disease (PD), with a strong focus on the genetic subtype.

• GT-02287 is being evaluated in a Phase 1b clinical study for PD patients with or without a GBA1 mutation.
• Enrollment for the Phase 1b study was completed in Q3 2025 with 21 participants, surpassing the initial target of 15 participants.
• As of September 30, 2025, 16 participants had completed 90 days of dosing.
• Approximately 80% of eligible participants elected to transition into the study extension.
• Preclinical models showed a disease-modifying effect in both GBA1-PD and idiopathic PD.

The second patient-facing segment involves rare genetic disorders, which are currently in earlier development stages.

• GT-02287 has potential for Gaucher's disease, which is a rare lysosomal storage disorder.
• Gain Therapeutics also has multiple undisclosed preclinical assets targeting lysosomal storage disorders.

For the business development side, large pharmaceutical and biotechnology companies represent a critical segment, as they are potential partners for late-stage development and commercialization.

The company is positioning itself for this segment by hitting key clinical milestones, such as planning to submit an Investigational New Drug (IND) application to the FDA by the end of 2025 to expand Phase 2 development into the United States.

The final segment is the financial community, comprising institutional and individual investors who provide the necessary capital.

Investor Metric	Value / Amount
Total Institutional Investors	7
Institutional Ownership Percentage	11.97%
Total Funding Raised (All Rounds)	$12.8M
Largest Single Offering (July 2025 Gross Proceeds)	Approx. $7.0 million
Cash and Cash Equivalents (as of Sept 30, 2025)	$8.8 million
Largest Single Investor Holding (Geode Capital Management LLC)	$523K
Analyst Consensus Rating	Moderate Buy
Average 1-Year Price Target	$7.50

The investor base is actively being engaged through capital raises; for instance, a July 2025 public offering yielded aggregate gross proceeds of approximately $7.0 million. Also, as of late November 2025, Gain Therapeutics filed to offer up to $35,530,980.56 in common stock via an at-the-market offering program. The cash position as of September 30, 2025, stood at $8.8 million.

Gain Therapeutics, Inc. (GANX) - Canvas Business Model: Cost Structure

You're looking at the hard numbers that drive the burn rate for Gain Therapeutics, Inc. as they push GT-02287 through the clinic. The cost structure is almost entirely driven by the science and keeping the lights on for a public company.

High research and development (R&D) expenses for clinical trials and preclinical work are the main cost driver. For the three months ended September 30, 2025, R&D expenses were reported at $2.8 million, up from $2.6 million for the same period in 2024. The increase was primarily due to costs for the ongoing Phase 1b clinical trial and unfavorable foreign exchange currency translation from the strengthening Swiss franc and Australian dollar against the U.S. dollar. This was partially offset by pipeline cost optimization and lower research and development personnel costs.

General and administrative (G&A) costs for corporate overhead and public company compliance also factor in. G&A expenses for the third quarter of 2025 were $1.9 million, an increase of $0.1 million compared to $1.8 million in the third quarter of 2024. These costs include items like legal and professional fees related to general corporate matters, which saw a decrease in the third quarter of 2025 compared to the prior year period.

The bottom line reflects this investment in development. Net loss for the three months ended September 30, 2025, was reported at -$5.3 million. This compares to a net loss of -$0.15 per share, basic and diluted, for the same quarter in 2025. For the nine months ending September 30, 2025, the net loss totaled -$15.6 million.

Personnel costs for specialized scientific and management teams are embedded within the R&D and G&A figures. For instance, the third quarter of 2025 saw lower research and development personnel costs, which helped offset some of the R&D expense increase. The company appointed Gene Mack as President and Chief Executive Officer effective January 6, 2025, with Gianluca Fuggetta assuming the role of Senior Vice President Finance and Principal Financial Officer.

Intellectual property maintenance and patent prosecution fees are a necessary, though often less detailed, component of the operating expenses. While specific line items for these fees aren't explicitly broken out for Q3 2025, they contribute to the overall G&A spend. The company's focus on its Magellan™ drug discovery platform and advancing GT-02287 necessitates ongoing IP protection costs.

Here is a quick look at the operating expense trends for the third quarter:

Metric	Q3 2025 Amount (USD)	Q3 2024 Amount (USD)
Research and Development (R&D) Expenses	$2.8 million	$2.6 million
General and Administrative (G&A) Expenses	$1.9 million	$1.8 million

The cash position also reflects this cost structure. Cash and cash equivalents stood at $8.8 million as of September 30, 2025, down from $10.4 million as of December 31, 2024. The company did see $11.9 million in cash inflows from financing activities over the nine months ending September 30, 2025, to help fund operations.

You can see how the quarterly losses stack up:

• Net Loss for the three months ended September 30, 2025: -$5.3 million.
• Net Loss per Share for Q3 2025: ($0.15).
• Net Loss for the nine months ended September 30, 2025: -$15.6 million.
• Cash and Cash Equivalents as of September 30, 2025: $8.8 million.
• Cash and Cash Equivalents as of December 31, 2024: $10.4 million.

For context on earlier 2025 spending, here are the Q1 figures:

• R&D Expenses for Q1 2025: $2.26 million.
• G&A Expenses for Q1 2025: $2.11 million.
• Net Loss for Q1 2025: -$4.53 million.

Finance: draft 13-week cash view by Friday.

Gain Therapeutics, Inc. (GANX) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of Gain Therapeutics, Inc. (GANX) as of late 2025, and honestly, it's what you'd expect for a clinical-stage biotech. The focus isn't on current sales, but on securing the capital needed to push the pipeline forward.

Minimal current revenue; consensus Q4 2025 revenue forecast is $0.000.

• As per analyst ratings, the consensus revenue forecast for 2025Q4 is $0.000.
• Analyst forecasts indicate that Gain Therapeutics' revenue in 2025 is projected to be $0.
• Trailing twelve months (ttm) revenue is listed as n/a.

Non-dilutive grant funding from foundations like The Michael J. Fox Foundation.

Gain Therapeutics, Inc. has secured non-dilutive funding, which directly offsets operating expenses like Research & Development (R&D). For instance, R&D expenses decreased in Q1 2025 partly due to research grant income.

• Funding support has been announced from The Michael J. Fox Foundation for Parkinson's Research (MJFF) and The Silverstein Foundation for Parkinson's with GBA for the GT-02287 program.
• The company was supported by Innosuisse, the Swiss Innovation Agency, with a grant of CHF 2.5 million (approximately $2.8 million) to develop GT-02287.
• This funding added to a prior CHF 1.5 million funding received from Innosuisse in 2021.
• The program also received support from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research.

Potential future milestone payments from strategic licensing agreements.

The business model anticipates revenue generation down the line from its proprietary platform and licensed assets. The terms of these arrangements typically outline specific non-cash or cash payments tied to progress.

Revenue Component Type	Typical Structure in Biotech Licensing	Specific Known Arrangement
Initiation Fee	Non-refundable fee upon contract signing.	The terms of collaboration and licensing agreements typically include a non-refundable initiation fee.
Milestone Payments	Payments upon achieving future development and regulatory goals.	Future development and regulatory milestone payments are a typical component.
Royalties	Percentage of net sales from a commercialized licensed product.	Royalties on net sales of the licensed product are a typical component.

Potential future royalties from commercialized products developed via the platform.

Should any of the pipeline programs, such as the lead candidate GT-02287, reach commercialization, royalties on net sales would become a significant, recurring revenue stream. The Magellan™ platform is used to identify targets for various therapeutic areas.

Equity financing through public offerings (ATM or secondary offerings).

This is the most concrete, near-term source of cash infusion to fund operations, given the lack of product sales. You've seen them execute on this recently to extend the cash runway.

• In November 2025, Gain Therapeutics, Inc. filed for an At-The-Market (ATM) offering authorization up to $35,530,980.56.
• As of November 2025, the company had already sold an aggregate of $14,469,019.44 under the original ATM agreement established in September 2024.
• In July 2025, the company priced an underwritten public offering expecting aggregate gross proceeds of approximately $7.0 million.
• For the second quarter of 2025, the company highlighted a public offering that raised $7.1 million.

The cash position as of September 30, 2025, was $8.8 million.