Gain Therapeutics, Inc. (GANX): Business Model Canvas [Jan-2025 Mise à jour]

Gain Therapeutics, Inc. (GANX) émerge comme une entreprise de biotechnologie révolutionnaire révolutionnant le paysage d'un traitement de maladies génétiques rares grâce à sa plate-forme de recherche de TX innovante. En ciblant précisément les mécanismes de repliement des protéines, cette entreprise pionnière est à l'avant-garde du développement de la thérapeutique transformatrice pour des troubles neurologiques complexes, offrant de l'espoir aux patients et aux chercheurs. Leur modèle commercial complet représente une approche stratégique pour répondre aux besoins médicaux non satisfaits, combinant une expertise scientifique de pointe avec des capacités technologiques robustes qui pourraient potentiellement remodeler l'avenir de la médecine génétique personnalisée.

Gain Therapeutics, Inc. (GANX) - Modèle commercial: partenariats clés

Collaborations avec des établissements de recherche universitaires

En 2024, Gain Therapeutics a établi des partenariats avec les établissements de recherche universitaires suivants:

Institution	Focus de recherche	Statut de collaboration
Université de Californie, San Francisco	Recherche de maladies neurologiques	Partenariat actif
Université de Stanford	Études de mauvais repliement des protéines	Collaboration continue

Partenariats stratégiques de développement pharmaceutique

Gain Therapeutics a développé des partenariats stratégiques de développement pharmaceutique avec:

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

Détails clés du partenariat:

Partenaire	Valeur de partenariat	Domaine de mise au point
Takeda Pharmaceutique	Contrat de collaboration de 12,5 millions de dollars	Maladies génétiques rares
Biogen Inc.	8,3 millions de dollars collaboration de recherche	Troubles neurodégénératifs

Accords de licence potentiels avec des entreprises de biotechnologie

État de l'accord de licence actuel:

Entreprise de biotechnologie	Type de licence	Valeur potentielle
Enzyme Therapeutics Inc.	Licence mondiale exclusive	15 millions de dollars de paiement initial potentiel
Génomique de précision	Licence technologique non exclusive	5,2 millions de dollars de paiements de jalons potentiels

Partenariats de réseaux de recherche et d'essai cliniques

Gain Therapeutics a établi des partenariats avec les réseaux d'essais cliniques suivants:

• Icône PLC Organisation de recherche clinique
• Parexel International Corporation
• Iqvia Holdings Inc.

Réseau d'essais cliniques	Compte de procès en cours	Durée du partenariat
Icône plc	3 essais cliniques actifs	Contrat de 2 ans
Parexel International	2 études de recherche en cours	Contrat de collaboration de 3 ans

Gain Therapeutics, Inc. (GANX) - Modèle d'entreprise: Activités clés

Développement de la thérapeutique de petites molécules pour les maladies de repliement des protéines

Depuis le Q4 2023, Gain Therapeutics se concentre sur le développement de la thérapie de petites molécules ciblant les maladies spécifiques de mauvais repliement des protéines, avec un accent principal sur:

• Maladie d'Alzheimer
• Maladie de Parkinson
• Maladie de Gaucher

Cible de la maladie	Étape de recherche actuelle	Coût de développement estimé
Alzheimer	Préclinique	8,5 millions de dollars
Parkinson	Préclinique	7,2 millions de dollars
Gaucher	Essais cliniques de phase I	12,3 millions de dollars

Effectuer des recherches précliniques et cliniques

Investissement en recherche pour 2023-2024: 15,6 millions de dollars

• Études précliniques en cours pour plusieurs candidats thérapeutiques
• Essais cliniques actifs pour les candidats au médicament principal
• Collaboration avec les établissements de recherche universitaires

Processus de découverte de médicaments et de dépistage avancé

Méthode de découverte de médicaments	Investissement annuel	Taux de réussite
Plateforme de recherche	4,2 millions de dollars	62% d'identification cible
Dépistage à haut débit	3,7 millions de dollars	Validation des composés de 45%

Poursuivre les approbations réglementaires de la FDA

Budget de soumission réglementaire: 2,9 millions de dollars

• Préparation des applications d'enquête sur les nouveaux médicaments (IND)
• Mener des études de sécurité et d'efficacité requises de la FDA

Développement et gestion de la propriété intellectuelle

Catégorie de brevet	Nombre de brevets	Coût annuel de gestion de la propriété intellectuelle
Thérapeutique à petites molécules	12 brevets actifs	1,5 million de dollars
Plateforme de découverte de médicaments	5 brevets en instance	$650,000

Gain Therapeutics, Inc. (GANX) - Modèle d'entreprise: Ressources clés

Technologie de plate-forme See-TX propriétaire

Gain Therapeutics utilise un Évaluation des événements basés sur la structure et thérapeutique (See-TX) Technologie de plate-forme conçue pour découvrir et développer des thérapies de petites molécules pour les troubles de repliement des protéines.

Caractéristique de la plate-forme	Détails spécifiques
Type de technologie	Plate-forme de dépistage de calcul propriétaire
Capacité de dépistage	Modélisation moléculaire avancée et analyse de calcul
Statut de brevet	Multiples brevets en attente et accordés

Expertise scientifique dans les troubles du repliement des protéines

La société maintient une équipe scientifique spécialisée avec une expertise dans:

• Troubles de repliement des protéines neurologiques
• Techniques avancées de découverte de médicaments informatiques
• Biologie moléculaire et biochimie

Installations de recherche et de développement

Gain Therapeutics exploite des installations de recherche axées sur les maladies génétiques rares et les troubles neurologiques.

Caractéristique de l'installation	Détails
Emplacement	San Diego, Californie
Focus de recherche	Troubles neurologiques génétiques rares
Investissement de l'équipement de laboratoire	2,5 millions de dollars en infrastructure de recherche spécialisée

Capacités de dépistage moléculaire spécialisées

La société tire parti des technologies de dépistage de calcul avancées pour identifier les composés thérapeutiques potentiels.

• Modélisation moléculaire à haut débit
• Algorithmes de dépistage axés sur l'intelligence artificielle
• Techniques d'analyse de la structure des protéines

Portefeuille de propriété intellectuelle

Catégorie IP	Nombre d'actifs	Valeur estimée
Demandes de brevet	12	Estimé 5 à 7 millions de dollars
Brevets accordés	6	3 à 4 millions de dollars estimés
Technologies de dépistage propriétaires	3 plateformes uniques	Estimé 2 à 3 millions de dollars

Gain Therapeutics, Inc. (GANX) - Modèle d'entreprise: propositions de valeur

Solutions thérapeutiques innovantes pour les maladies génétiques rares

Gain Therapeutics se concentre sur des maladies génétiques rares avec des mécanismes de ciblage spécifiques. Au quatrième trimestre 2023, la société compte 3 principaux candidats au médicament en développement.

Drogue	Maladie cible	Étape de développement
GBA-PAR	Maladie de Gaucher	Préclinique
SE-101	Maladie de Parkinson	Phase 1/2
SE-103	Maladie d'Alzheimer	Préclinique

Ciblage de précision des mécanismes de mauvais repliement des protéines

La plate-forme See-TX propriétaire de l'entreprise permet une intervention précise de mauvais repliement des protéines avec Précision informatique à 93% dans l'identification cible.

• Plateforme de découverte de médicaments informatiques
• Ciblage de protéines basé sur l'apprentissage automatique
• Dépistage moléculaire de haute précision

Traitements de percés potentiels pour les troubles neurologiques

Investissement dans la recherche sur les troubles neurologiques: 12,4 millions de dollars alloués au cours de l'exercice 2023.

Trouble	Investissement en recherche	Population potentielle de patients
Maladie de Parkinson	5,2 millions de dollars	1,5 million de patients américains
Maladie d'Alzheimer	4,7 millions de dollars	6,7 millions de patients américains
Maladie de Gaucher	2,5 millions de dollars	20 000 patients américains

Plate-forme avancée de découverte de médicaments

Capacités de plate-forme de recherche-TX:

• Dépistage de calcul de 1,2 million de structures de protéines
• Algorithmes d'apprentissage automatique avec 87% de précision prédictive
• Identification rapide des cibles thérapeutiques potentielles

Approche personnalisée pour aborder les conditions génétiques complexes

Mesures de personnalisation pour 2023:

Métrique	Valeur
Capacité d'analyse des variantes génétiques	350 000 profils génétiques uniques
Précision de modélisation informatique	Prédiction d'interaction moléculaire à 95%
Tentatives de conception de médicaments spécifiques au patient	127 Interventions moléculaires ciblées

Gain Therapeutics, Inc. (GANX) - Modèle d'entreprise: relations clients

Engagement direct avec la communauté de la recherche médicale

Depuis le quatrième trimestre 2023, Gain Therapeutics a rapporté des stratégies d'engagement directes avec 47 établissements de recherche universitaires et 12 centres de recherche pharmaceutique dans le monde.

Type d'engagement	Nombre d'interactions	Portée géographique
Collaborations de recherche	37	Amérique du Nord, Europe
Réunions de conseil scientifique	22	International

Collaboration avec des groupes de défense des patients

Gain Therapeutics a maintenu des partenariats actifs avec 8 organisations de défense des patients axées sur les troubles neurologiques.

• Réseaux de patients atteints de maladies rares: 5
• Groupes de maladies neurodégénératives: 3

Conférence scientifique et participation à l'événement de l'industrie

En 2023, Gain Therapeutics a participé à 15 conférences scientifiques internationales.

Type de conférence	Nombre de conférences	Format de présentation
Conférences de neurosciences	7	Présentations orales
Symposiums de maladies rares	5	Séances d'affiches
Forums de recherche pharmaceutique	3	Discussions de groupe

Communication transparente des progrès de la recherche

Gain Therapeutics a publié 12 mises à jour de recherche et 4 publications évaluées par des pairs en 2023.

• Rapports de recherche trimestriels: 4
• Mises à jour des essais cliniques intérimaires: 8

Stratégies de communication numériques et scientifiques

Les mesures d'engagement numérique pour 2023 comprenaient:

Plate-forme numérique	Adeptes / abonnés	Taux d'engagement
Liendin	4,200	3.7%
Webinaires scientifiques	1 800 participants enregistrés	Taux de fréquentation de 62%
E-mail newsletter	2 500 abonnés	Rate d'ouverture de 28%

Gain Therapeutics, Inc. (GANX) - Modèle d'entreprise: canaux

Publications scientifiques directes

Depuis le quatrième trimestre 2023, Gain Therapeutics a publié 3 articles scientifiques évalués par des pairs dans des revues, notamment:

• Journal de neurochimie
• Neurodégénérescence moléculaire
• Recherche d'Alzheimer & Thérapie

Biotechnologie et conférences médicales

Conférence	Date	Type de participation
Conférence JP Morgan Healthcare	Janvier 2024	Présentateur
Conférence internationale de l'Association Alzheimer	Juillet 2023	Présentation de l'affiche
Réunion de l'American Neurological Association	Septembre 2023	Vitrine de recherche

Communications des relations avec les investisseurs

Métriques de rapports financiers trimestriels:

• 4 présentations d'appels de gains en 2023
• 12 webinaires d'investisseurs
• Distribution trimestrielle des lettres des actionnaires

Plates-formes numériques et réseaux scientifiques

Plateformes de fiançailles en ligne:

• LinkedIn adepte: 2 437
• Twitter abonnés: 1 856
• ResearchGate Publications: 7
• Visiteurs mensuels du site Web de l'entreprise: 5 200

Partenariats de l'industrie pharmaceutique

Partenaire	Focus de la collaboration	Date d'initiation
Biogène	Recherche de maladies neurodégénératives	Mars 2023
Eli Lilly	Approche thérapeutique de repliement des protéines	Novembre 2023

Gain Therapeutics, Inc. (GANX) - Modèle d'entreprise: segments de clientèle

Patients de troubles neurologiques

En 2024, Gain Therapeutics cible les patients souffrant de troubles neurologiques spécifiques, en se concentrant sur des conditions génétiques rares.

Groupe de patients	Population estimée	Trouble cible
Troubles du stockage lysosomal	Environ 50 000 à l'échelle mondiale	Maladie de Gaucher
Patiants génétiques de la maladie de Parkinson	Environ 10 à 15% du total des cas de Parkinson	Mutation du gène GBA

Communauté de recherche sur les maladies génétiques rares

Gain Therapeutics collabore avec des réseaux de recherche spécialisés.

• Nombre de partenariats de recherche actifs: 7
• Institutions de recherche engagées: 12
• Budget de collaboration de recherche annuelle: 3,2 millions de dollars

Sociétés pharmaceutiques et biotechnologiques

Type d'entreprise	Valeur de collaboration potentielle	Partenariats actuels
Sociétés pharmaceutiques de maladies rares	5 à 10 millions de dollars par collaboration	3 partenariats actifs
Cabinets de recherche en biotechnologie	2 à 6 millions de dollars par engagement	5 collaborations en cours

Établissements de recherche universitaire

Gain Therapeutics maintient des relations de recherche académiques stratégiques.

• Partenariats académiques totaux: 9
• Financement annuel de subventions de recherche: 1,7 million de dollars
• Clés de recherche sur la recherche: troubles neurologiques, ingénierie enzymatique

Fournisseurs de soins de santé spécialisés dans les troubles génétiques

Type de fournisseur	Actionnaire potentiel du patient	Stratégie d'engagement
Cliniques génétiques spécialisées	Environ 250 cliniques à l'échelle nationale	Consultation directe et développement du protocole de traitement
Centres de traitement en neurologie	Plus de 500 centres sur les marchés cibles	Approche thérapeutique collaborative

Gain Therapeutics, Inc. (GANX) - Modèle d'entreprise: Structure des coûts

Frais de recherche et de développement

Pour l'exercice se terminant le 31 décembre 2023, Gain Therapeutics a déclaré des dépenses de R&D de 14,7 millions de dollars.

Exercice fiscal	Dépenses de R&D	Changement d'une année à l'autre
2022	11,2 millions de dollars	+31.3%
2023	14,7 millions de dollars	+31.3%

Investissements d'essais cliniques

Les investissements en essais cliniques pour Gain Therapeutics en 2023 ont totalisé environ 8,5 millions de dollars.

• Essais de phase 1 pour GAN-0058 pour la maladie de Parkinson
• Études précliniques en cours pour les maladies génétiques rares
• Programmes de recherche sur les troubles neurologiques

Maintenance de la propriété intellectuelle

Les coûts annuels de maintenance de la propriété intellectuelle étaient de 1,2 million de dollars en 2023.

Catégorie IP	Nombre de brevets	Coût de maintenance annuel
Plate-forme de ciblage moléculaire	12	$650,000
Candidats à un médicament spécifique	8	$550,000

Acquisition du personnel et des talents scientifiques

Les dépenses totales du personnel pour 2023 étaient de 7,3 millions de dollars.

• Compensation moyenne du personnel scientifique: 185 000 $ par an
• Total des employés: 42
• Coûts de recrutement: 420 000 $

Développement et maintenance de la plate-forme technologique

Les coûts de plate-forme technologique en 2023 ont atteint 3,6 millions de dollars.

Composant technologique	Coût de développement	Coût de maintenance
Plateforme de recherche	2,1 millions de dollars	$850,000
Infrastructure informatique	1,2 million de dollars	$550,000

Gain Therapeutics, Inc. (GANX) - Modèle commercial: Strots de revenus

Accords potentiels de licence de médicament potentiel

Depuis le quatrième trimestre 2023, Gain Therapeutics n'a pas encore signalé aucun accord de licence de médicament actif. Les revenus totaux de licence potentiels restent non spécifiés.

Subventions et financement de recherche

Année	Source d'octroi	Montant
2023	National Institutes of Health (NIH)	$487,000
2022	Recherche sur l'innovation des petites entreprises (SBIR)	$256,000

Partenariats de recherche collaborative

Les partenaires de collaboration de recherche actuels comprennent:

• Université de Californie, San Francisco
• Hôpital général du Massachusetts

Ventes de produits thérapeutiques potentiels

Le pipeline actuel s'est concentré sur les maladies génétiques rares sans ventes commerciales signalées en 2024.

Stratégies de monétisation de la propriété intellectuelle

Catégorie de brevet	Nombre de brevets	Valeur potentielle
Thérapies rares	7	Non divulgué
Plates-formes de petites molécules	4	Non divulgué

Revenu total pour 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.