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

Gain Therapeutics, Inc. (GANX) entwickelt sich zu einem bahnbrechenden Biotechnologieunternehmen, das mit seiner innovativen SEE-Tx-Plattform die Landschaft der Behandlung seltener genetischer Krankheiten revolutioniert. Durch die präzise Ausrichtung auf Proteinfehlfaltungsmechanismen steht dieses Pionierunternehmen an der Spitze der Entwicklung transformativer Therapeutika für komplexe neurologische Erkrankungen und gibt Patienten und Forschern gleichermaßen Hoffnung. Ihr umfassendes Geschäftsmodell stellt einen strategischen Ansatz zur Lösung ungedeckter medizinischer Bedürfnisse dar und kombiniert modernste wissenschaftliche Expertise mit robusten technologischen Fähigkeiten, die möglicherweise die Zukunft der personalisierten genetischen Medizin neu gestalten könnten.

Gain Therapeutics, Inc. (GANX) – Geschäftsmodell: Wichtige Partnerschaften

Kooperationen mit akademischen Forschungseinrichtungen

Seit 2024 hat Gain Therapeutics Partnerschaften mit den folgenden akademischen Forschungseinrichtungen aufgebaut:

Institution	Forschungsschwerpunkt	Kooperationsstatus
Universität von Kalifornien, San Francisco	Erforschung neurologischer Erkrankungen	Aktive Partnerschaft
Stanford-Universität	Studien zur Proteinfehlfaltung	Laufende Zusammenarbeit

Strategische pharmazeutische Entwicklungspartnerschaften

Gain Therapeutics hat strategische pharmazeutische Entwicklungspartnerschaften entwickelt mit:

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

Wichtige Partnerschaftsdetails:

Partner	Partnerschaftswert	Fokusbereich
Takeda Pharmaceutical	Kooperationsvereinbarung über 12,5 Millionen US-Dollar	Seltene genetische Erkrankungen
Biogen Inc.	Forschungskooperation im Wert von 8,3 Millionen US-Dollar	Neurodegenerative Erkrankungen

Mögliche Lizenzvereinbarungen mit Biotechnologieunternehmen

Aktueller Lizenzvertragsstatus:

Biotechnologieunternehmen	Lizenztyp	Potenzieller Wert
Enzyme Therapeutics Inc.	Exklusive weltweite Lizenz	Mögliche Vorauszahlung in Höhe von 15 Millionen US-Dollar
Präzisionsgenomik	Nicht-exklusive Technologielizenz	Mögliche Meilensteinzahlungen in Höhe von 5,2 Millionen US-Dollar

Netzwerkpartnerschaften für Forschung und klinische Studien

Gain Therapeutics hat Partnerschaften mit den folgenden Netzwerken für klinische Studien aufgebaut:

• Klinische Forschungsorganisation ICON plc
• Parexel International Corporation
• IQVIA Holdings Inc.

Netzwerk für klinische Studien	Aktuelle Testanzahl	Dauer der Partnerschaft
ICON plc	3 aktive klinische Studien	2-Jahres-Vertrag
Parexel International	2 laufende Forschungsstudien	3-jähriger Kooperationsvertrag

Gain Therapeutics, Inc. (GANX) – Geschäftsmodell: Hauptaktivitäten

Entwicklung niedermolekularer Therapeutika für Proteinfehlfaltungskrankheiten

Ab dem vierten Quartal 2023 konzentriert sich Gain Therapeutics auf die Entwicklung niedermolekularer Therapeutika, die auf spezifische Proteinfehlfaltungskrankheiten abzielen, mit dem Schwerpunkt auf:

• Alzheimer-Krankheit
• Parkinson-Krankheit
• Gaucher-Krankheit

Krankheitsziel	Aktueller Forschungsstand	Geschätzte Entwicklungskosten
Alzheimer	Präklinisch	8,5 Millionen US-Dollar
Parkinson	Präklinisch	7,2 Millionen US-Dollar
Gaucher	Klinische Studien der Phase I	12,3 Millionen US-Dollar

Durchführung präklinischer und klinischer Forschung

Forschungsinvestitionen für 2023-2024: 15,6 Millionen US-Dollar

• Laufende präklinische Studien für mehrere therapeutische Kandidaten
• Aktive klinische Studien für führende Arzneimittelkandidaten
• Zusammenarbeit mit akademischen Forschungseinrichtungen

Weiterentwicklung von Arzneimittelentdeckungs- und Screening-Prozessen

Methode zur Arzneimittelentdeckung	Jährliche Investition	Erfolgsquote
SEE-Tx-Plattform	4,2 Millionen US-Dollar	62 % Zielidentifikation
Hochdurchsatz-Screening	3,7 Millionen US-Dollar	45 % Compound-Validierung

Streben nach FDA-Zulassungen

Budget für die Einreichung von Vorschriften: 2,9 Millionen US-Dollar

• Vorbereitung von IND-Anträgen (Investigational New Drug).
• Durchführung von von der FDA geforderten Sicherheits- und Wirksamkeitsstudien

Entwicklung und Management von geistigem Eigentum

Patentkategorie	Anzahl der Patente	Jährliche Kosten für die IP-Verwaltung
Therapeutika für kleine Moleküle	12 aktive Patente	1,5 Millionen Dollar
Arzneimittelforschungsplattform	5 angemeldete Patente	$650,000

Gain Therapeutics, Inc. (GANX) – Geschäftsmodell: Schlüsselressourcen

Proprietäre SEE-Tx-Plattformtechnologie

Gain Therapeutics nutzt a Strukturbasierte Ereignisbewertung und Therapeutik (SEE-Tx) Plattformtechnologie zur Entdeckung und Entwicklung niedermolekularer Therapien für Proteinfehlfaltungsstörungen.

Plattformcharakteristik	Spezifische Details
Technologietyp	Proprietäre Computer-Screening-Plattform
Screening-Fähigkeit	Fortgeschrittene molekulare Modellierung und Computeranalyse
Patentstatus	Mehrere angemeldete und erteilte Patente

Wissenschaftliche Expertise bei Proteinfehlfaltungsstörungen

Das Unternehmen verfügt über ein spezialisiertes wissenschaftliches Team mit Fachkenntnissen in:

• Neurologische Proteinfehlfaltungsstörungen
• Fortgeschrittene rechnergestützte Techniken zur Arzneimittelentdeckung
• Molekularbiologie und Biochemie

Forschungs- und Entwicklungseinrichtungen

Gain Therapeutics betreibt Forschungseinrichtungen, die sich auf seltene genetische Krankheiten und neurologische Störungen konzentrieren.

Merkmale der Einrichtung	Details
Standort	San Diego, Kalifornien
Forschungsschwerpunkt	Seltene genetische neurologische Störungen
Investition in Laborausrüstung	Schätzungsweise 2,5 Millionen US-Dollar für spezialisierte Forschungsinfrastruktur

Spezialisierte molekulare Screening-Funktionen

Das Unternehmen nutzt fortschrittliche rechnergestützte Screening-Technologien zur Identifizierung potenzieller therapeutischer Verbindungen.

• Molekulare Modellierung mit hohem Durchsatz
• Auf künstlicher Intelligenz basierende Screening-Algorithmen
• Techniken zur Proteinstrukturanalyse

Portfolio für geistiges Eigentum

IP-Kategorie	Anzahl der Vermögenswerte	Geschätzter Wert
Patentanmeldungen	12	Geschätzte 5-7 Millionen US-Dollar
Erteilte Patente	6	Geschätzte 3-4 Millionen US-Dollar
Proprietäre Screening-Technologien	3 einzigartige Plattformen	Geschätzte 2-3 Millionen US-Dollar

Gain Therapeutics, Inc. (GANX) – Geschäftsmodell: Wertversprechen

Innovative therapeutische Lösungen für seltene genetische Erkrankungen

Gain Therapeutics konzentriert sich auf seltene genetische Erkrankungen mit spezifischen Targeting-Mechanismen. Im vierten Quartal 2023 befinden sich drei primäre Medikamentenkandidaten des Unternehmens in der Entwicklung.

Arzneimittelkandidat	Zielkrankheit	Entwicklungsphase
GBA-PAR	Gaucher-Krankheit	Präklinisch
SE-101	Parkinson-Krankheit	Phase 1/2
SE-103	Alzheimer-Krankheit	Präklinisch

Präzises Targeting von Proteinfehlfaltungsmechanismen

Die proprietäre SEE-Tx-Plattform des Unternehmens ermöglicht einen präzisen Eingriff in die Fehlfaltung von Proteinen 93 % Rechengenauigkeit bei der Zielidentifizierung.

• Computerbasierte Plattform zur Arzneimittelentdeckung
• Auf maschinellem Lernen basierendes Protein-Targeting
• Hochpräzises molekulares Screening

Mögliche bahnbrechende Behandlungen für neurologische Erkrankungen

Investition in die Erforschung neurologischer Störungen: 12,4 Millionen US-Dollar im Geschäftsjahr 2023 bereitgestellt.

Störung	Forschungsinvestitionen	Potenzielle Patientenpopulation
Parkinson-Krankheit	5,2 Millionen US-Dollar	1,5 Millionen US-Patienten
Alzheimer-Krankheit	4,7 Millionen US-Dollar	6,7 Millionen US-Patienten
Gaucher-Krankheit	2,5 Millionen Dollar	20.000 US-Patienten

Fortschrittliche Arzneimittelforschungsplattform

Funktionen der SEE-Tx-Plattform:

• Computergestütztes Screening von 1,2 Millionen Proteinstrukturen
• Algorithmen für maschinelles Lernen mit 87 % Vorhersagegenauigkeit
• Schnelle Identifizierung potenzieller therapeutischer Ziele

Personalisierter Ansatz zur Behandlung komplexer genetischer Erkrankungen

Personalisierungskennzahlen für 2023:

Metrisch	Wert
Kapazität zur Analyse genetischer Varianten	350.000 einzigartige genetische Profile
Präzision der rechnergestützten Modellierung	95 % molekulare Interaktionsvorhersage
Patientenspezifische Arzneimitteldesignversuche	127 gezielte molekulare Interventionen

Gain Therapeutics, Inc. (GANX) – Geschäftsmodell: Kundenbeziehungen

Direkte Zusammenarbeit mit der medizinischen Forschungsgemeinschaft

Im vierten Quartal 2023 berichtete Gain Therapeutics über Strategien zur direkten Zusammenarbeit mit 47 akademischen Forschungseinrichtungen und 12 pharmazeutischen Forschungszentren weltweit.

Engagement-Typ	Anzahl der Interaktionen	Geografische Reichweite
Forschungskooperationen	37	Nordamerika, Europa
Wissenschaftliche Beratungstreffen	22	International

Zusammenarbeit mit Patientenvertretungen

Gain Therapeutics unterhält aktive Partnerschaften mit acht Patientenorganisationen, die sich auf neurologische Erkrankungen konzentrieren.

• Patientennetzwerke für seltene Krankheiten: 5
• Neurodegenerative Krankheitsgruppen: 3

Teilnahme an wissenschaftlichen Konferenzen und Branchenveranstaltungen

Im Jahr 2023 nahm Gain Therapeutics an 15 internationalen wissenschaftlichen Konferenzen teil.

Konferenztyp	Anzahl der Konferenzen	Präsentationsformat
Neurowissenschaftliche Konferenzen	7	Mündliche Vorträge
Symposien zu seltenen Krankheiten	5	Postersitzungen
Pharmazeutische Forschungsforen	3	Podiumsdiskussionen

Transparente Kommunikation des Forschungsfortschritts

Gain Therapeutics veröffentlichte im Jahr 2023 12 Forschungsaktualisierungen und 4 peer-reviewte Veröffentlichungen.

• Vierteljährliche Forschungsberichte: 4
• Zwischenaktualisierungen klinischer Studien: 8

Digitale und wissenschaftliche Kommunikationsstrategien

Zu den Kennzahlen zum digitalen Engagement für 2023 gehörten:

Digitale Plattform	Follower/Abonnenten	Engagement-Rate
LinkedIn	4,200	3.7%
Wissenschaftliche Webinare	1.800 registrierte Teilnehmer	62 % Anwesenheitsquote
E-Mail-Newsletter	2.500 Abonnenten	28 % Öffnungsrate

Gain Therapeutics, Inc. (GANX) – Geschäftsmodell: Kanäle

Direkte wissenschaftliche Veröffentlichungen

Im vierten Quartal 2023 veröffentlichte Gain Therapeutics drei von Experten begutachtete wissenschaftliche Artikel in Fachzeitschriften, darunter:

• Zeitschrift für Neurochemie
• Molekulare Neurodegeneration
• Alzheimer-Forschung & Therapie

Biotechnologie- und Medizinkonferenzen

Konferenz	Datum	Teilnahmetyp
JP Morgan Healthcare-Konferenz	Januar 2024	Moderator
Internationale Konferenz der Alzheimer's Association	Juli 2023	Posterpräsentation
Treffen der American Neurological Association	September 2023	Forschungsschaufenster

Investor-Relations-Kommunikation

Kennzahlen der vierteljährlichen Finanzberichterstattung:

• 4 Earnings Call-Präsentationen im Jahr 2023
• 12 Investoren-Webinare
• Vierteljährliche Verteilung des Aktionärsbriefs

Digitale Plattformen und wissenschaftliche Netzwerke

Online-Engagement-Plattformen:

• LinkedIn-Follower: 2.437
• Twitter-Follower: 1.856
• ResearchGate-Veröffentlichungen: 7
• Monatliche Besucher der Unternehmenswebsite: 5.200

Partnerschaften in der Pharmaindustrie

Partner	Fokus auf Zusammenarbeit	Einführungsdatum
Biogen	Forschung zu neurodegenerativen Erkrankungen	März 2023
Eli Lilly	Therapeutischer Ansatz zur Proteinfehlfaltung	November 2023

Gain Therapeutics, Inc. (GANX) – Geschäftsmodell: Kundensegmente

Patienten mit neurologischen Störungen

Ab 2024 richtet sich Gain Therapeutics an Patienten mit bestimmten neurologischen Störungen und konzentriert sich dabei auf seltene genetische Erkrankungen.

Patientengruppe	Geschätzte Bevölkerung	Zielstörung
Patienten mit lysosomaler Speicherstörung	Ungefähr 50.000 weltweit	Gaucher-Krankheit
Patienten mit genetischer Variante der Parkinson-Krankheit	Etwa 10–15 % aller Parkinson-Fälle	GBA-Genmutation

Forschungsgemeinschaft für seltene genetische Krankheiten

Gain Therapeutics arbeitet mit spezialisierten Forschungsnetzwerken zusammen.

• Anzahl aktiver Forschungskooperationen: 7
• Beteiligte Forschungseinrichtungen: 12
• Jährliches Budget für Forschungszusammenarbeit: 3,2 Millionen US-Dollar

Pharma- und Biotechnologieunternehmen

Unternehmenstyp	Potenzieller Wert der Zusammenarbeit	Aktuelle Partnerschaften
Pharmaunternehmen für seltene Krankheiten	5–10 Millionen US-Dollar pro Zusammenarbeit	3 aktive Partnerschaften
Biotechnologie-Forschungsunternehmen	2–6 Millionen US-Dollar pro Engagement	5 laufende Kooperationen

Akademische Forschungseinrichtungen

Gain Therapeutics unterhält strategische akademische Forschungsbeziehungen.

• Gesamtzahl der akademischen Partnerschaften: 9
• Jährliche Forschungsstipendien: 1,7 Millionen US-Dollar
• Forschungsschwerpunkte: Neurologische Erkrankungen, Enzym-Engineering

Auf genetische Erkrankungen spezialisierte Gesundheitsdienstleister

Anbietertyp	Potenzielle Patientenreichweite	Engagement-Strategie
Spezialisierte genetische Kliniken	Rund 250 Kliniken bundesweit	Direkte Beratung und Entwicklung eines Behandlungsprotokolls
Neurologie-Behandlungszentren	Über 500 Zentren in Zielmärkten	Kollaborativer therapeutischer Ansatz

Gain Therapeutics, Inc. (GANX) – Geschäftsmodell: Kostenstruktur

Forschungs- und Entwicklungskosten

Für das am 31. Dezember 2023 endende Geschäftsjahr meldete Gain Therapeutics Forschungs- und Entwicklungskosten in Höhe von 14,7 Millionen US-Dollar.

Geschäftsjahr	F&E-Ausgaben	Veränderung im Jahresvergleich
2022	11,2 Millionen US-Dollar	+31.3%
2023	14,7 Millionen US-Dollar	+31.3%

Investitionen in klinische Studien

Die Investitionen in klinische Studien für Gain Therapeutics beliefen sich im Jahr 2023 auf insgesamt etwa 8,5 Millionen US-Dollar.

• Phase-1-Studien für GAN-0058 zur Behandlung der Parkinson-Krankheit
• Laufende präklinische Studien für seltene genetische Erkrankungen
• Forschungsprogramme für neurologische Störungen

Aufrechterhaltung des geistigen Eigentums

Die jährlichen Kosten für die Aufrechterhaltung des geistigen Eigentums beliefen sich im Jahr 2023 auf 1,2 Millionen US-Dollar.

IP-Kategorie	Anzahl der Patente	Jährliche Wartungskosten
Molekulare Targeting-Plattform	12	$650,000
Spezifische Arzneimittelkandidaten	8	$550,000

Personal- und wissenschaftliche Talentakquise

Die gesamten Personalkosten für 2023 beliefen sich auf 7,3 Millionen US-Dollar.

• Durchschnittliche Vergütung für wissenschaftliches Personal: 185.000 US-Dollar pro Jahr
• Gesamtzahl der Mitarbeiter: 42
• Rekrutierungskosten: 420.000 US-Dollar

Entwicklung und Wartung von Technologieplattformen

Die Kosten für Technologieplattformen beliefen sich im Jahr 2023 auf 3,6 Millionen US-Dollar.

Technologiekomponente	Entwicklungskosten	Wartungskosten
SEE-Tx-Plattform	2,1 Millionen US-Dollar	$850,000
Computerinfrastruktur	1,2 Millionen US-Dollar	$550,000

Gain Therapeutics, Inc. (GANX) – Geschäftsmodell: Einnahmequellen

Mögliche zukünftige Arzneimittellizenzvereinbarungen

Bis zum vierten Quartal 2023 hat Gain Therapeutics noch keine aktiven Lizenzvereinbarungen für Arzneimittel gemeldet. Die gesamten potenziellen Lizenzeinnahmen sind noch nicht spezifiziert.

Forschungsstipendien und Finanzierung

Jahr	Grant-Quelle	Betrag
2023	National Institutes of Health (NIH)	$487,000
2022	Small Business Innovation Research (SBIR)	$256,000

Verbundforschungspartnerschaften

Zu den aktuellen Forschungskooperationspartnern gehören:

• Universität von Kalifornien, San Francisco
• Massachusetts General Hospital

Potenzielle Verkäufe therapeutischer Produkte

Die aktuelle Pipeline konzentriert sich auf seltene genetische Krankheiten, für die ab 2024 keine kommerziellen Verkäufe gemeldet wurden.

Monetarisierungsstrategien für geistiges Eigentum

Patentkategorie	Anzahl der Patente	Potenzieller Wert
Therapien für seltene Krankheiten	7	Nicht bekannt gegeben
Plattformen für kleine Moleküle	4	Nicht bekannt gegeben

Gesamtumsatz für 2023: 743.000 US-Dollar

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.