Gain Therapeutics Initiates Phase 1b Clinical Trial of Lead Candidate GT-02287 in People with GBA1 and Idiopathic Parkinson’s Disease
Gain Therapeutics (NASDAQ: GANX) has received approval to initiate a Phase 1b clinical trial in Australia for GT-02287, its lead candidate for treating Parkinson's disease (PD). The trial follows a successful Phase 1 study where GT-02287 demonstrated safety, tolerability, and >50% increase in GCase activity. The Phase 1b trial will assess safety and tolerability in PD patients over three months of dosing at 13.5 mg/kg/day, along with biomarker evaluation.
The company expects to complete enrollment in spring 2025, with data anticipated by mid-2025. The trial will be conducted across seven Australian sites, focusing on both GBA1 and idiopathic Parkinson's disease patients. Secondary endpoints include pharmacokinetics, GCase modulation, and substrate levels in plasma and cerebrospinal fluid.
Gain Therapeutics (NASDAQ: GANX) ha ricevuto l'approvazione per avviare un trial clinico di Fase 1b in Australia per GT-02287, il suo principale candidato per il trattamento della malattia di Parkinson (PD). Il trial segue uno studio di Fase 1 di successo in cui GT-02287 ha dimostrato sicurezza, tollerabilità e un aumento del >50% dell'attività di GCase. Il trial di Fase 1b valuterà la sicurezza e la tollerabilità nei pazienti con PD per un periodo di tre mesi con dosaggio di 13,5 mg/kg/giorno, insieme alla valutazione dei biomarcatori.
L'azienda prevede di completare l'arruolamento nella primavera del 2025, con dati attesi entro la metà del 2025. Il trial sarà condotto in sette sedi australiane, concentrandosi sia sui pazienti con malattia di Parkinson legata a GBA1 che sulla forma idiopatica della malattia. Gli endpoint secondari includono la farmacocinetica, la modulazione di GCase e i livelli di substrato nel plasma e nel liquido cerebrospinale.
Gain Therapeutics (NASDAQ: GANX) ha recibido aprobación para iniciar un ensayo clínico de Fase 1b en Australia para GT-02287, su candidato principal para el tratamiento de la enfermedad de Parkinson (PD). El ensayo sigue a un estudio de Fase 1 exitoso en el que GT-02287 demostró seguridad, tolerabilidad y un aumento del >50% en la actividad de GCase. El ensayo de Fase 1b evaluará la seguridad y la tolerabilidad en pacientes con PD durante tres meses de dosificación a 13,5 mg/kg/día, junto con la evaluación de biomarcadores.
La empresa espera completar la inscripción en la primavera de 2025, con datos anticipados para mediados de 2025. El ensayo se llevará a cabo en siete sitios australianos, centrado tanto en pacientes con enfermedad de Parkinson relacionada con GBA1 como en aquellos con la forma idiopática de la enfermedad. Los puntos finales secundarios incluyen la farmacocinética, la modulación de GCase y los niveles de sustrato en plasma y líquido cefalorraquídeo.
Gain Therapeutics (NASDAQ: GANX)는 PD(파킨슨병) 치료를 위한 주요 후보인 GT-02287의 임상 시험 1b 단계를 호주에서 시작할 수 있도록 승인을 받았습니다. 이 시험은 GT-02287이 안전성, 내약성 및 GCase 활성을 50% 이상 증가시킨 것을 입증한 성공적인 1단계 연구를 따릅니다. 1b 단계 시험은 PD 환자를 대상으로 13.5 mg/kg/일의 용량으로 3개월간 안전성과 내약성을 평가하며, 동시에 바이오마커 평가도 진행됩니다.
회사는 2025년 봄에 등록을 완료할 것으로 예상하며, 데이터는 2025년 중반에 예상됩니다. 이 시험은 호주의 7개 사이트에서 진행되며, GBA1 환자와 특발성 파킨슨병 환자 모두를 집중적으로 연구합니다. 2차 목표에는 약동학, GCase 조절 및 혈장과 뇌척수액의 기질 수준이 포함됩니다.
Gain Therapeutics (NASDAQ: GANX) a reçu l'autorisation de lancer un essai clinique de Phase 1b en Australie pour GT-02287, son principal candidat pour le traitement de la maladie de Parkinson (PD). L'essai fait suite à une étude de Phase 1 réussie où GT-02287 a démontré sa sécurité, sa tolérabilité et une augmentation de >50% de l'activité de GCase. L'essai de Phase 1b évaluera la sécurité et la tolérabilité chez les patients PD pendant trois mois avec un dosage de 13,5 mg/kg/jour, ainsi qu'une évaluation des biomarqueurs.
L'entreprise prévoit de compléter l'inscription au printemps 2025, avec des données attendues pour le milieu de 2025. L'essai sera réalisé dans sept sites australiens, se concentrant à la fois sur les patients présentant une maladie de Parkinson liée à GBA1 et sur la forme idiopathique de la maladie. Les points finaux secondaires incluent la pharmacocinétique, la modulation de GCase et les niveaux de substrat dans le plasma et le liquide céphalorachidien.
Gain Therapeutics (NASDAQ: GANX) hat die Genehmigung erhalten, eine klinische Studie der Phase 1b in Australien für GT-02287, seinen Hauptkandidaten zur Behandlung der Parkinson-Krankheit (PD), zu starten. Die Studie folgt einer erfolgreichen Phase-1-Studie, in der GT-02287 Sicherheit, Verträglichkeit und eine >50%ige Erhöhung der GCase-Aktivität demonstrierte. Die Phase-1b-Studie wird die Sicherheit und Verträglichkeit bei PD-Patienten über drei Monate bei einer Dosierung von 13,5 mg/kg/Tag sowie eine Biomarker-Bewertung beurteilen.
Das Unternehmen erwartet, die Einschreibung im Frühjahr 2025 abzuschließen, mit Daten, die bis Mitte 2025 erwartet werden. Die Studie wird an sieben australischen Standorten durchgeführt, wobei sowohl Patienten mit GBA1-als auch mit idiopathischer Parkinson-Krankheit fokussiert werden. Sekundäre Endpunkte umfassen Pharmakokinetik, GCase-Modulation und Substratlevels im Plasma und in der Rückenmarksflüssigkeit.
- Successful completion of Phase 1 study with favorable safety profile
- Significant target engagement with >50% increase in GCase activity
- Clear timeline for Phase 1b completion with data expected mid-2025
- Advancement from preclinical to clinical stage development
- Early-stage clinical development with no efficacy data yet
- Extended timeline to reach Phase 2 trials (2025+)
Insights
The initiation of a Phase 1b trial for GT-02287 marks a important milestone in Gain Therapeutics' clinical development program. The trial's design demonstrates several key strengths: multi-site implementation across Australia, three-month treatment duration and comprehensive biomarker analysis. The previous Phase 1 results showing
The focus on both GBA1 and idiopathic Parkinson's disease patients broadens the potential therapeutic application. The dosing regimen of 13.5 mg/kg/day was likely selected based on the pharmacokinetic and safety data from Phase 1, suggesting an optimized therapeutic window. The inclusion of cerebrospinal fluid biomarker analysis will provide critical data on central nervous system penetration and target engagement.
For a micro-cap biotech with a market cap of
The focus on disease-modifying potential rather than just symptomatic treatment positions GT-02287 uniquely in the PD therapeutic landscape. The biomarker-rich trial design will provide valuable data points for potential partnering discussions or investment decisions before the anticipated Phase 2 initiation. For investors, the relatively quick timeline to data readout (mid-2025) provides a clear value-inflection point within a defined timeframe.
The Company has received approval to begin enrollment of the Phase 1b clinical trial in Australia
Phase 1b clinical trial will assess safety and tolerability along with biomarkers during three months of dosing with GT-02287 in people diagnosed with Parkinson’s disease
Phase 1b clinical trial follows successful Phase 1 study in which GT-02287 was safe and well tolerated while demonstrating GCase target engagement
BETHESDA, Md., Dec. 23, 2024 (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 it has received approval in Australia to initiate a Phase 1b trial. Gain will be working with local Parkinson’s disease (PD) advocacy groups to support enrollment and expects enrollment to complete in the spring of 2025 with data from the study expected mid-2025. GT-02287 is the Company’s lead allosteric small molecule in clinical development for the treatment of PD with or without a GBA1 mutation. The primary goal of the Phase 1b trial is to assess the safety and tolerability of GT-02287 in people with PD.
The Phase 1b trial follows the successful Phase 1 study in healthy volunteers completed during Q3 2024, in which GT-02287 demonstrated a favorable safety and tolerability profile as well as plasma and CNS exposures in the projected therapeutic range. Importantly, the Phase 1 study also showed significant target engagement of GT-02287 demonstrated by a statistically significant increase in glucocerebrosidase (GCase) activity that was >
“It is a fitting end to what has been a transformative year for Gain Therapeutics with the initiation of our Phase 1b clinical trial for GT-02287 in Australia. During 2024, Gain Therapeutics made the significant transition from preclinical to clinical stage development with successful completion of the healthy volunteer studies. We believe GT-02287 has the potential to slow or stop the progression of Parkinson’s disease and look forward to further evaluating the safety and tolerability of GT-02287; while also observing its impact on key biomarkers of Parkinson’s disease after three months of administration in the Phase 1b trial. Gain Therapeutics welcomes the New Year and we are excited to take another critical step towards advancing GT-02287 into Phase 2 trial preparation in 2025 following results of the Phase 1b trial, expected mid-year 2025,” said Gene Mack, CFO and Interim CEO of Gain Therapeutics.
Jonas Hannestad, M.D., Ph.D., Chief Medical Officer of Gain, continued, “The Phase 1b clinical trial of GT-02287 will be conducted at seven sites covering the major metropolitan areas in Australia, all of which have experience with Parkinson’s disease trials. Historically, a limited number of clinical trials in Parkinson’s disease have included Australian sites, and we have heard from both investigators and potential participants that they are eager to have the opportunity to partake in this study.”
The Phase 1b open-label trial will assess the safety and tolerability of 13.5 mg/kg/day of GT-02287 for three months in patients with GBA1-PD or idiopathic Parkinson’s disease. Secondary endpoints include pharmacokinetics, GCase modulation, levels of GCase substrates, and other biomarkers in plasma and cerebrospinal fluid. Data from the Phase 1b trial is anticipated mid-2025.
For more information visit: https://clinicaltrials.gov/study/NCT06732180
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 protein modulator that restores the function of the lysosomal protein 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 aggregated α-synuclein, neuroinflammation and neuronal death, and improved motor function and cognitive performance. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker for neurodegeneration.
Compelling preclinical data in models of both GBA1-PD and idiopathic PD, demonstrating a disease-modifying effect after administration of GT-02287, suggests that GT-02287 may have the potential to slow or stop the progression of Parkinson’s disease.
Gain’s lead program in Parkinson’s disease has been awarded funding support 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. Results from a Phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety and tolerability, plasma exposure in the projected therapeutic range, CNS exposure, and target engagement and modulation of GCase enzyme.
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; 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, 2023 and Form 10-Q for the quarter ended September 30, 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.
Investor Contacts:
Apaar Jammu and Chuck Padala
ajammu@gaintherapeutics.com
chuck@lifesciadvisors.com
Media Contacts:
Russo Partners
Nic Johnson and Elio Ambrosio
nic.johnson@russopartnersllc.com
elio.ambrosio@russopartnersllc.com
(760) 846-9256
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