Gain Therapeutics Begins Its GT-02287 Phase 1b Extension Study in People with Parkinson’s Disease
Gain Therapeutics (NASDAQ:GANX) has initiated a Phase 1b extension study for its Parkinson's disease treatment GT-02287, allowing participants to continue dosing for an additional nine months beyond the initial 90-day schedule. The original Phase 1b study, which enrolled 21 participants, is set to complete in December 2025.
The company will present early results from the Phase 1b study at the International Congress of Parkinson's Disease and Movement Disorders on October 7, 2025. Additional data, including MDS-UPDRS functional changes and biomarker data from cerebrospinal fluid and blood, will be available in Q4 2025. Management reports encouraging early observations regarding safety, tolerability, pharmacokinetics, and functional impact.
Gain Therapeutics (NASDAQ:GANX) ha avviato uno studio di estensione di fase 1b per il trattamento della malattia di Parkinson GT-02287, permettendo ai partecipanti di continuare la somministrazione per ulteriori nove mesi oltre l'originario programma di 90 giorni. Lo studio originale di fase 1b, che ha arruolato 21 partecipanti, è previsto si chiuda a dicembre 2025.
L'azienda presenterà i primi risultati dello studio di fase 1b al International Congress of Parkinson's Disease and Movement Disorders il 7 ottobre 2025. Ulteriori dati, inclusi i cambiamenti funzionali MDS-UPDRS e dati biomarcatori provenienti dal liquido cefalorachidiano e dal sangue, saranno disponibili nel quarto trimestre del 2025. Il management riferisce osservazioni incoraggianti in merito alla sicurezza, tollerabilità, farmacocinetica e impatto funzionale.
Gain Therapeutics (NASDAQ:GANX) ha iniciado un estudio de extensión de Fase 1b para su tratamiento de la enfermedad de Parkinson GT-02287, permitiendo a los participantes continuar la dosificación por otros nueve meses más allá del programa inicial de 90 días. El estudio original de Fase 1b, que inscribió a 21 participantes, está previsto para completarse en diciembre de 2025.
La compañía presentará resultados preliminares del estudio de Fase 1b en el Congreso Internacional de Enfermedad de Parkinson y Trastornos del Movimiento el 7 de octubre de 2025. Datos adicionales, incluidos cambios funcionales de MDS-UPDRS y datos de biomarcadores de líquido cefalorraquídeo y sangre, estarán disponibles en el cuarto trimestre de 2025. La dirección informa observaciones alentadoras respecto a la seguridad, tolerabilidad, farmacocinética y impacto funcional.
Gain Therapeutics (NASDAQ:GANX)는 파킨슨병 치료제 GT-02287의 1상 1b 확장 연구를 시작하여 초기 90일 일정 외에 추가로 9개월 동안 투여를 지속할 수 있도록 했습니다. 21명의 참가자를 등록한 원래의 1상 1b 연구는 2025년 12월에 완료될 예정입니다.
회사는 2025년 10월 7일에 열리는 국제 파킨슨병 및 운동장애 학회에서 1상 1b 연구의 초기 결과를 발표할 예정입니다. 뇌척수액 및 혈액의 생물학적 표지자 데이터와 함께 MDS-UPDRS 기능 변화 등 추가 데이터는 2025년 4분기에 제공될 예정입니다. 경영진은 안전성, 내약성, 약동학 및 기능적 영향에 대해 고무적인 초기 관찰을 보고하고 있습니다.
Gain Therapeutics (NASDAQ:GANX) a lancé une extension de l’étude de phase 1b pour son traitement de la maladie de Parkinson, le GT-02287, permettant aux participants de poursuivre le dosage pendant neuf mois supplémentaires au-delà du planning initial de 90 jours. L’étude initiale de phase 1b, qui a recruté 21 participants, doit se terminer en décembre 2025.
L’entreprise présentera les premiers résultats de l’étude de phase 1b lors du Congrès international sur la maladie de Parkinson et les troubles du mouvement le 7 octobre 2025. Des données complémentaires, notamment les changements fonctionnels MDS-UPDRS et les données biomarqueurs du liquide céphalorachidien et du sang, seront disponibles au cours du quatrième trimestre 2025. La direction rapporte des observations encourageantes concernant la sécurité, la tolérance, la pharmacocinéthique et l’impact fonctionnel.
Gain Therapeutics (NASDAQ:GANX) hat eine Verlängerung der Phase-1b-Studie für die Parkinson-Krankheit-Behandlung GT-02287 eingeleitet, die es den Teilnehmern ermöglicht, die Dosierung für weitere neun Monate über den ursprünglichen 90-Tage-Zeitraum hinweg fortzusetzen. Die ursprüngliche Phase-1b-Studie, an der 21 Teilnehmer beteiligt waren, soll im Dezember 2025 abgeschlossen sein.
Das Unternehmen wird am International Congress of Parkinson's Disease and Movement Disorders am 7. Oktober 2025 erste Ergebnisse der Phase-1b-Studie vorstellen. Weitere Daten, einschließlich MDS-UPDRS-Funktionsveränderungen und Biomarker-Daten aus Liquor und Blut, werden im vierten Quartal 2025 verfügbar sein. Das Management meldet ermutigende erste Beobachtungen bezüglich Sicherheit, Verträglichkeit, Pharmakokinetik und funktionalen Auswirkungen.
Gain Therapeutics (NASDAQ:GANX) قد بدأ دراسة امتداد للمرحلة 1b لعلاج باركنسون GT-02287، مما يسمح للمشاركين بمواصلة الجرعة لمدة تسعة أشهر إضافية بخلاف الجدول الأصلي الذي يستغرق 90 يوماً. ستكتمل الدراسة الأصلية للمرحلة 1b، التي شملت 21 مشاركاً، في ديسمبر 2025.
ستقدم الشركة نتائج مبكرة من دراسة المرحلة 1b في المؤتمر الدولي لمرض باركنسون واضطرابات الحركة في 7 أكتوبر 2025. ستتوفر بيانات إضافية، بما في ذلك تغييرات MDS-UPDRS الوظيفية وبيانات من عينات السائل النخاعي والدم، في الربع الرابع من 2025. تشير الإدارة إلى ملاحظات مبكرة مشجعة تتعلق بالسلامة والتحمل والدوائية الحركية والتأثير الوظيفي.
Gain Therapeutics（纳斯达克股票代码：GANX） 已启动其帕金森病治疗药物 GT-02287 的1b期扩展研究，允许参与者在最初90天计划之外再进行九个月的用药。最初的1b期研究共招募了 21 名参与者，预计将于2025年12月完成。
公司将于2025年10月7日在<国际帕金森病与运动障碍大会上公布1b期研究的初步结果。包括MDS-UPDRS功能变化和来自脑脊液及血液的生物标志物数据在内的更多数据，将在2025年第四季度提供。管理层对安全性、耐受性、药代动力学及功能影响的初步观察表示乐观。
- More than 50% of participants agreed to continue treatment in the extension study
- Early observations show encouraging safety, tolerability, and functional impact results
- Study is progressing on schedule with data readouts planned for Q4 2025
- Complete Phase 1b results not yet available
- Limited participant size of only 21 subjects in the study
Insights
Gain Therapeutics' Phase 1b extension study for GT-02287 in Parkinson's indicates encouraging preliminary safety and efficacy signals.
Gain Therapeutics (NASDAQ: GANX) has initiated a 9-month extension to its Phase 1b study of GT-02287 in Parkinson's disease patients, with over half of the original 21 participants opting to continue treatment. This high retention rate suggests favorable patient experience with the experimental therapy.
The company will present early data from the Phase 1b study at the International Congress of Parkinson's Disease and Movement Disorders in October 2025, with more comprehensive 90-day analysis expected in Q4 2025. This analysis will include critical functional assessments using the MDS-UPDRS scale and biomarker data from cerebrospinal fluid and blood samples.
Management's statement that "early observations related to safety, tolerability, pharmacokinetics, and functional impact are encouraging" provides a positive signal, though without specific data points. The decision to extend treatment for nine additional months is particularly noteworthy for a novel allosteric small molecule therapy, as it provides opportunity to gather valuable long-term safety and efficacy data.
For a small biotech company, this extension study represents an important step in GT-02287's development pathway. The willingness of patients to continue beyond the initial study period suggests the treatment may be well-tolerated and potentially showing early signs of benefit, though concrete efficacy data remains pending. The upcoming data presentations in Q4 2025 will be critical in determining whether GT-02287 shows meaningful therapeutic potential in addressing Parkinson's disease.
Extension of the Phase 1b study will allow participants to continue dosing with GT-02287 for an additional nine months after completing the first 90-day dosing schedule
The Phase 1b study enrolled 21 subjects and the last participants will complete 90 days of dosing in December 2025
Early results from the Phase 1b study to be presented at the International Congress of Parkinson’s Disease (PD) and Movement Disorders in October
BETHESDA, Md., Sept. 18, 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 the start of the Phase 1b extension study allowing participants to continue treatment for an additional nine months. More than half of the participants have agreed to continue treatment.
The Phase 1b study enrolled 21 participants and is expected to complete in December 2025. Early data from the Phase 1b study will be presented on October 7, 2025, at the International Congress of Parkinson’s Disease and Movement Disorders® in Honolulu, HI.
Additional 90-day analysis from participants enrolled as of June 30, 2025, including functional changes scored according to the Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) as well as biomarker data from cerebrospinal fluid and blood, will be available as expected in Q4 2025.
“We are grateful to the individuals participating in the study and to the dedicated team of clinical investigators for their continued support,” said Gene Mack, President and Chief Executive Officer of Gain Therapeutics. “We look forward to presenting early data from our Phase 1b study at the annual MDS meeting in October, additional data will be forthcoming throughout the fourth quarter of 2025. Early observations related to safety, tolerability, pharmacokinetics, and functional impact are encouraging. We are also pleased by the commitment of participants to continue treatment with GT-02287, allowing us to further evaluate long-term safety and efficacy.”
Information on the poster to be presented at International Congress of Parkinson’s Disease and Movement Disorders® is below:
Poster Title: GT-02287 in Parkinson’s Disease: Interim Data from a Phase 1b study
E-Poster Number: 891
E-Poster Session: Tuesday, October 7, 12:00 – 2:30pm HST
The International Parkinson and Movement Disorder Society (MDS) gathers thousands of the field's clinicians, researchers, trainees and industry supporters with an interest in the current research and approaches for the diagnosis and treatment of movement disorders on an annual basis at its International Congress of Parkinson's Disease and Movement Disorders®.
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 enrolled 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 completion, quality and 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, including the dose extension study; 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 because of new information, future events or otherwise.
Investors:
Gain Therapeutics, Inc.
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Manager, Investor Relations and Public Relations
ajammu@gaintherapeutics.com
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FAQ
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