Gain Therapeutics Presents Additional Preclinical Data and Design of Phase 1b Clinical Study of GT-02287 at AD/PD 2025 and Provides Enrollment Update
Gain Therapeutics (GANX) presented new preclinical data and Phase 1b study design for GT-02287 at AD/PD 2025 Conference. The oral presentation revealed evidence of disease-modifying activity in both GBA1 and idiopathic Parkinson's disease models.
The preclinical data showed GT-02287's ability to rescue motor deficits and prevent complex behavioral deficits, with effects persisting after compound withdrawal. Several biomarkers including α-synuclein, IRE-1, LAMP-1, Miro1, phospho-Tau, and Iba-1 showed statistically significant reductions and maintained lower levels post-treatment.
The ongoing Phase 1b study is evaluating safety, tolerability, and pharmacokinetics in Parkinson's disease patients. An early biomarker analysis is scheduled for Q2 2025, with complete endpoint analysis expected in Q4 2025. The company anticipates completing enrollment by Q2 2025, with Phase 2 planning set to begin in H2 2025.
Gain Therapeutics (GANX) ha presentato nuovi dati preclinici e il design dello studio di Fase 1b per GT-02287 alla Conferenza AD/PD 2025. La presentazione orale ha rivelato evidenze di attività modificante la malattia sia nei modelli di malattia di Parkinson GBA1 che idiopatica.
I dati preclinici hanno mostrato la capacità di GT-02287 di recuperare i deficit motori e prevenire deficit comportamentali complessi, con effetti che persistono anche dopo la sospensione del composto. Diversi biomarcatori, tra cui α-sinucleina, IRE-1, LAMP-1, Miro1, fosfo-Tau e Iba-1, hanno mostrato riduzioni statisticamente significative e hanno mantenuto livelli più bassi dopo il trattamento.
Lo studio di Fase 1b in corso sta valutando la sicurezza, la tollerabilità e la farmacocinetica nei pazienti affetti da malattia di Parkinson. Un'analisi precoce dei biomarcatori è programmata per il secondo trimestre del 2025, con un'analisi completa degli endpoint prevista per il quarto trimestre del 2025. L'azienda prevede di completare l'arruolamento entro il secondo trimestre del 2025, con la pianificazione della Fase 2 che dovrebbe iniziare nella seconda metà del 2025.
Gain Therapeutics (GANX) presentó nuevos datos preclínicos y el diseño del estudio de Fase 1b para GT-02287 en la Conferencia AD/PD 2025. La presentación oral reveló evidencia de actividad modificadora de la enfermedad tanto en modelos de enfermedad de Parkinson GBA1 como idiopática.
Los datos preclínicos mostraron la capacidad de GT-02287 para rescatar déficits motores y prevenir déficits conductuales complejos, con efectos que persisten incluso después de la retirada del compuesto. Varios biomarcadores, incluidos α-sinucleína, IRE-1, LAMP-1, Miro1, fosfo-Tau e Iba-1, mostraron reducciones estadísticamente significativas y mantuvieron niveles más bajos después del tratamiento.
El estudio de Fase 1b en curso está evaluando la seguridad, tolerabilidad y farmacocinética en pacientes con enfermedad de Parkinson. Se programó un análisis temprano de biomarcadores para el segundo trimestre de 2025, con un análisis completo de los puntos finales esperado para el cuarto trimestre de 2025. La empresa anticipa completar la inscripción para el segundo trimestre de 2025, con la planificación de la Fase 2 programada para comenzar en la segunda mitad de 2025.
Gain Therapeutics (GANX)는 AD/PD 2025 컨퍼런스에서 GT-02287에 대한 새로운 전임상 데이터와 1b상 연구 설계를 발표했습니다. 구두 발표에서는 GBA1 및 특발성 파킨슨병 모델 모두에서 질병 수정 활성을 나타내는 증거가 드러났습니다.
전임상 데이터는 GT-02287가 운동 결핍을 회복하고 복잡한 행동 결핍을 예방하는 능력을 보여주었으며, 화합물 중단 후에도 효과가 지속되었습니다. 여러 바이오마커(α-시누클레인, IRE-1, LAMP-1, Miro1, 인산화 타우 및 Iba-1)가 통계적으로 유의미한 감소를 보였으며, 치료 후에도 낮은 수준을 유지했습니다.
진행 중인 1b상 연구는 파킨슨병 환자에서 안전성, 내약성 및 약물 동태를 평가하고 있습니다. 초기 바이오마커 분석은 2025년 2분기로 예정되어 있으며, 최종 분석은 2025년 4분기에 완료될 것으로 예상됩니다. 회사는 2025년 2분기까지 등록을 완료할 것으로 예상하며, 2상 계획은 2025년 하반기에 시작될 예정입니다.
Gain Therapeutics (GANX) a présenté de nouvelles données précliniques et le design de l'étude de Phase 1b pour GT-02287 lors de la Conférence AD/PD 2025. La présentation orale a révélé des preuves d'une activité modifiant la maladie dans des modèles de maladie de Parkinson GBA1 et idiopathique.
Les données précliniques ont montré la capacité de GT-02287 à récupérer les déficits moteurs et à prévenir les déficits comportementaux complexes, avec des effets persistants même après le retrait du composé. Plusieurs biomarqueurs, y compris l'α-synucléine, l'IRE-1, le LAMP-1, le Miro1, le phospho-Tau et l'Iba-1, ont montré des réductions statistiquement significatives et ont maintenu des niveaux plus bas après le traitement.
L'étude de Phase 1b en cours évalue la sécurité, la tolérabilité et la pharmacocinétique chez des patients atteints de la maladie de Parkinson. Une analyse précoce des biomarqueurs est prévue pour le deuxième trimestre 2025, avec une analyse complète des points d'évaluation attendue pour le quatrième trimestre 2025. L'entreprise prévoit de terminer le recrutement d'ici le deuxième trimestre 2025, avec une planification de la Phase 2 qui devrait commencer au second semestre 2025.
Gain Therapeutics (GANX) hat auf der AD/PD 2025 Konferenz neue präklinische Daten und das Design der Phase 1b Studie für GT-02287 vorgestellt. Die mündliche Präsentation zeigte Beweise für eine krankheitsmodifizierende Aktivität sowohl in GBA1- als auch in idiopathischen Parkinson-Modellen.
Die präklinischen Daten zeigten die Fähigkeit von GT-02287, motorische Defizite zu beheben und komplexe Verhaltensdefizite zu verhindern, wobei die Effekte auch nach Absetzen des Wirkstoffs anhielten. Mehrere Biomarker, darunter α-Synuclein, IRE-1, LAMP-1, Miro1, Phospho-Tau und Iba-1, zeigten statistisch signifikante Reduktionen und hielten nach der Behandlung niedrigere Werte.
Die laufende Phase 1b Studie bewertet die Sicherheit, Verträglichkeit und Pharmakokinetik bei Parkinson-Patienten. Eine frühe Biomarker-Analyse ist für das zweite Quartal 2025 geplant, während eine vollständige Analyse der Endpunkte im vierten Quartal 2025 erwartet wird. Das Unternehmen rechnet damit, die Rekrutierung bis zum zweiten Quartal 2025 abzuschließen, mit der Planung für Phase 2, die in der zweiten Jahreshälfte 2025 beginnen soll.
- Preclinical data shows persistent disease-modifying effects even after drug withdrawal
- Multiple disease biomarkers showed statistically significant reductions
- Phase 1b study enrollment progressing well and on schedule
- Clear development timeline with multiple catalysts throughout 2025
- No efficacy data available yet from human trials
- Phase 1b results not expected until Q4 2025
Insights
Gain Therapeutics' clinical progress update contains multiple positive elements but remains in early clinical phases with results still pending. The company reports robust enrollment in their Phase 1b study for GT-02287, with enrollment expected to complete by Q2 2025 and an interim biomarker analysis planned mid-year to guide Phase 2 planning.
For context, this is significant for a small-cap biotech (
The notable aspect of GT-02287 is its potential as a disease-modifying therapy rather than merely symptomatic treatment for Parkinson's. The preclinical data showing persistent effects even after drug withdrawal strengthens this positioning. Key biomarkers including aggregated α-synuclein, ER stress markers, and neuroinflammation markers showed statistically significant reductions.
Investors should watch for the upcoming catalyst of interim biomarker data which could validate target engagement in humans. The design to include both GBA1 mutation carriers and non-carriers in the analysis represents a dual-market strategy targeting both genetic and sporadic Parkinson's populations.
The preclinical data for GT-02287 addresses several critical pathways in Parkinson's disease pathology. The compound's ability to normalize multiple biomarkers—including α-synuclein aggregation, ER stress, lysosomal integrity, mitochondrial health, and neuroinflammation—suggests a multi-modal mechanism potentially addressing the disease's complex pathophysiology.
Particularly noteworthy is the persistent effect after drug washout in preclinical models. This characteristic distinguishes GT-02287 from symptomatic treatments that require continuous administration to maintain benefit. True disease modification has been elusive in Parkinson's therapeutics, with most approved therapies only providing symptomatic relief.
The GCase-targeting approach has strong biological rationale. GBA1 mutations represent the most common genetic risk factor for Parkinson's, and even in idiopathic PD, GCase activity is often reduced. By enhancing GCase function, GT-02287 may address a fundamental driver of pathology across different PD subtypes.
The Phase 1b design appropriately focuses on safety, PK/PD relationships, and exploratory biomarkers that could demonstrate target engagement. The inclusion of cerebrospinal fluid biomarkers is crucial as they provide the most direct evidence of central nervous system activity. The planned analysis of multiple biomarkers—including GCase activity, sphingolipids, α-synuclein, and inflammatory markers—should provide a comprehensive picture of the compound's biological effects.
GT-02287 oral presentation demonstrates new evidence of disease-modifying capacity in GBA1 and idiopathic Parkinson’s disease models
Enrollment update provided for Phase 1b study
Phase 1b study ongoing to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of GT-02287 in people with Parkinson’s disease; biomarker analysis to be conducted in Q2 2025
BETHESDA, Md., April 10, 2025 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced that an oral presentation as well as a poster were presented at the AD/PD™ 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders held April 1-5, 2025, in Vienna, Austria. The oral presentation outlined new evidence further supporting the disease-modifying activity of GT-02287 in preclinical models of both GBA1 and idiopathic Parkinson’s disease (PD). Additionally, the poster presented detailed the design of the ongoing Phase 1b clinical study of GT-02287 in people with Parkinson’s disease.
An early biomarker analysis from the currently enrolled participants in the Phase 1b study will be conducted mid-year and will include samples from participants both with and without the GBA1 mutations for purposes of informing Phase 2 planning during the second half of 2025. Complete analysis of the Phase 1b endpoints, available after all patients have completed 90 days of treatment, is anticipated sometime during the fourth quarter of 2025.
“We continue to add to the robust preclinical dossier of GT-02287 and we believe the recent biomarker data, and the behavioral data we have presented previously further support the potential of GT-02287 to be a disease-modifying therapy for people with Parkinson’s disease,” commented Joanne Taylor, Ph.D., Senior Vice President of Research of Gain.
“We are encouraged by the robust enrollment and interest in our Phase 1b study in people with Parkinson’s disease. We believe the study is on track to complete enrollment by the end of the second quarter of 2025. We look forward to seeing how the effects on relevant pathway abnormalities that we have observed preclinically and in healthy volunteers translate to people with Parkinson’s disease,” said Jonas Hannestad, M.D., Ph.D., Chief Medical Officer of Gain.
Oral Presentation Information
Title: “Clinical Stage Glucocerebrosidase Modulator, GT-02287, Shows Disease Modifying Potential in Preclinical Models Of Both GBA1 and Idiopathic Parkinson’s Disease”
Summary: In animal models of both GBA1 and idiopathic Parkinson’s disease, GT-02287 is able to rescue motor deficits and prevent the development of deficits in complex behaviors such as nesting. These effects persist even following withdrawal of the compound, suggesting a disease-modifying potential of GT-02287. The new preclinical data that were presented additionally support a disease-modifying potential of GT-02287. Several biomarkers of disease progression, including aggregated α-synuclein, IRE-1 (a marker of ER stress), LAMP-1 (a marker of lysosomal integrity), Miro1 (a marker of damaged mitochondrial destined for mitophagy), phospho-Tau (a marker of neurodegeneration), and Iba-1 (a marker of neuroinflammation) were statistically reduced upon treatment with GT-02287 and remained reduced for several days following drug washout demonstrating a long-lasting effect.
Poster Presentation Information
Title: “Design of a Phase 1b Study to Evaluate the GCase-targeting Molecule GT-02287 in GBA1-PD and Sporadic PD”
Summary: The study will assess safety and tolerability of GT-02287 during three months of dosing in people with PD, using adverse events, laboratory tests, electrocardiograms, and vital signs as the key safety endpoints. Additionally, the study will characterize the single-dose and steady-state plasma pharmacokinetics (PK) profile of GT-02287. Exploratory endpoints include various biomarkers in the cerebrospinal fluid (CSF), dried blood spots, plasma, and whole blood. Biomarkers include GCase activity and protein levels, sphingolipid substrate levels, α-synuclein, lysosomal and mitochondrial markers, and inflammatory markers. Results from this Phase 1b study will guide the design of a subsequent randomized, placebo-controlled, double-blind dose-finding Phase 2 study to evaluate the efficacy of GT-02287 as measured by a clinically meaningful endpoint in PD over a longer treatment period. Phase 2 planning is expected to commence in the second half of 2025.
A PDF of the poster presented at AD/PD 2025 is available on the Science and Technology section of the Company’s website at https://gaintherapeutics.com/science-and-technology/posters.
Slides from the oral presentation at AD/PD 2025 will be available on the Company’s website at https://gaintherapeutics.com.
About the AD/PD™ Conference
The AD/PD conference explores the latest research, clinical trials and treatments for Alzheimer’s and Parkinson’s and other related neurological disorders. The conference has grown to be the leading event in the field of neurodegenerative disorders with over 4,700 participants from over 70 countries and over 2250 abstracts in 2024. For more information, visit https://adpd.kenes.com.
About GT-02287
Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease (PD) with or without a GBA1 mutation. The orally administered, brain-penetrant small molecule is an allosteric enzyme modulator that restores the function of the lysosomal enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to mutations in the GBA1 gene, the most common genetic abnormality associated with PD, or other age-related stress factors. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced ER stress, lysosomal and mitochondrial pathology, aggregated α-synuclein, neuroinflammation and neuronal death, as well as plasma neurofilament light chain (NfL) levels, a biomarker of neurodegeneration. In rodent models of both GBA1-PD and idiopathic PD, GT-02287 was shown to rescue deficits in motor function and gait and prevent the development of deficits in complex behaviors such as nesting.
Compelling preclinical data in models of both GBA1-PD and idiopathic PD, demonstrating a disease-modifying effect after administration of GT-02287, suggest that GT-02287 may have the potential to slow or stop the progression of Parkinson’s disease.
Results from a Phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety and tolerability, plasma and CNS exposures in the projected therapeutic range, and target engagement with a >
GT-02287 is currently being evaluated in a Phase 1b clinical trial for the treatment of Parkinson’s disease with or without a GBA1 mutation. The primary endpoint of the trial, which is currently enrolling participants across 7 sites in Australia, is to evaluate the safety and tolerability of GT-02287 after 3 months of dosing in people with Parkinson’s disease.
Gain’s lead program in Parkinson’s disease has been awarded funding support early in its development from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse – Swiss Innovation Agency.
About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate, GT-02287 is currently being evaluated for the treatment of Parkinson’s disease with or without a GBA1 mutation in a Phase 1b clinical trial. GT-02287 has further potential in Gaucher’s disease, dementia with Lewy bodies, and Alzheimer’s disease. Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors.
Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced Magellan™ platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders and oncology.
Forward-Looking Statements
This release contains “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are typically preceded by words such as “believes,” “expects,” “anticipates,” “intends,” “will,” “may,” “should,” or similar expressions. These forward-looking statements reflect management’s current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct or that those goals will be achieved, and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, statements regarding: the development of the Company’s current or future product candidates including GT-02287; expectations regarding the timing of results from a Phase 1b clinical study for GT-02287; expectations regarding the timing of patient enrollment for a Phase 1b clinical study for GT-02287; the timing of any submissions to the FDA or other regulatory bodies and agencies; and the potential therapeutic and clinical benefits of the Company’s product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the Company’s business in general, please refer to the Company’s Form 10-K for the year ended December 31, 2024. All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this release. We have no obligation, and expressly disclaim any obligation, to update, revise or correct any of the forward-looking statements, whether as a result of new information, future events or otherwise.
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Gain Therapeutics, Inc.
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FAQ
What are the key findings from GT-02287's preclinical trials for Parkinson's disease presented at AD/PD 2025?
When will GANX complete the Phase 1b trial enrollment for GT-02287?
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What is the timeline for GT-02287's Phase 1b results and Phase 2 planning?
