Gain Therapeutics Doses First Participant in Phase 1b Clinical Trial of GT-02287 in Parkinson’s Disease
Gain Therapeutics (NASDAQ: GANX) has announced the dosing of the first Parkinson's disease (PD) participant in its Phase 1b clinical trial of GT-02287, their lead allosteric small molecule therapy. The open-label, multi-center trial will evaluate safety and tolerability in up to 20 participants with GBA1-PD and idiopathic PD over three months.
The study follows a successful Phase 1 trial in healthy volunteers completed in Q3 2024, where GT-02287 showed favorable safety and tolerability, achieved desired plasma and CNS exposures, and demonstrated over 50% increase in glucocerebrosidase (GCase) activity. Secondary endpoints for the Phase 1b trial include pharmacokinetics, GCase modulation, and biomarker levels in plasma and cerebrospinal fluid.
An interim analysis from the Phase 1b trial is expected by the end of Q2 2025.
Gain Therapeutics (NASDAQ: GANX) ha annunciato la somministrazione del primo partecipante affetto da malattia di Parkinson (PD) nel suo studio clinico di Fase 1b su GT-02287, la loro principale terapia molecolare allosterica. Lo studio multicentrico e aperto valuterà la sicurezza e la tollerabilità in un massimo di 20 partecipanti con PD da mutazione GBA1 e PD idiopatica per un periodo di tre mesi.
Lo studio segue un precedente trial di Fase 1 su volontari sani completato nel terzo trimestre del 2024, dove GT-02287 ha mostrato una sicurezza e tollerabilità favorevoli, ha raggiunto le concentrazioni plasmatiche e nel sistema nervoso centrale desiderate, e ha dimostrato un aumento di oltre il 50% dell'attività della glucocerebrosidasi (GCase). Gli endpoint secondari per lo studio di Fase 1b includono farmacocinetica, modulazione della GCase e livelli di biomarcatori nel plasma e nel liquido cerebrospinale.
Un'analisi intermedia dello studio di Fase 1b è prevista entro la fine del secondo trimestre del 2025.
Gain Therapeutics (NASDAQ: GANX) ha anunciado la dosificación del primer participante con enfermedad de Parkinson (PD) en su ensayo clínico de Fase 1b de GT-02287, su principal terapia molecular alostérica. El ensayo multicéntrico y abierto evaluará la seguridad y tolerabilidad en hasta 20 participantes con PD por mutación GBA1 y PD idiopática durante tres meses.
El estudio sigue a un exitoso ensayo de Fase 1 en voluntarios sanos completado en el tercer trimestre de 2024, donde GT-02287 mostró una seguridad y tolerabilidad favorables, alcanzó las exposiciones plasmáticas y del sistema nervioso central deseadas, y demostró un aumento de más del 50% en la actividad de la glucocerebrosidasa (GCase). Los puntos finales secundarios para el ensayo de Fase 1b incluyen farmacocinética, modulación de GCase y niveles de biomarcadores en plasma y líquido cefalorraquídeo.
Se espera un análisis intermedio del ensayo de Fase 1b para finales del segundo trimestre de 2025.
Gain Therapeutics (NASDAQ: GANX)는 GT-02287, 그들의 주요 알로스테릭 소분자 치료제의 1상 1b 임상 시험에서 첫 번째 파킨슨병(PD) 참가자의 투약을 발표했습니다. 이 개방형 다기관 시험은 GBA1-PD 및 특발성 PD를 가진 최대 20명의 참가자에 대해 3개월 동안 안전성과 내약성을 평가할 것입니다.
이 연구는 2024년 3분기에 완료된 건강한 자원자를 대상으로 한 성공적인 1상 시험을 따르며, GT-02287는 안전성과 내약성이 우수하고, 원하는 혈장 및 CNS 노출을 달성했으며, 글루코세레브로시다제(GCase) 활성에서 50% 이상의 증가를 보여주었습니다. 1상 1b 시험의 2차 목표는 약물 동태학, GCase 조절 및 혈장과 뇌척수액의 바이오마커 수준을 포함합니다.
1상 1b 시험의 중간 분석은 2025년 2분기 말까지 예상됩니다.
Gain Therapeutics (NASDAQ: GANX) a annoncé le dosage du premier participant atteint de la maladie de Parkinson (PD) dans son essai clinique de Phase 1b sur GT-02287, leur principale thérapie moléculaire allostérique. L'essai ouvert et multicentrique évaluera la sécurité et la tolérabilité chez jusqu'à 20 participants atteints de PD liée à la mutation GBA1 et de PD idiopathique pendant trois mois.
L'étude fait suite à un essai de Phase 1 réussi sur des volontaires en bonne santé, achevé au troisième trimestre 2024, où GT-02287 a montré une sécurité et une tolérabilité favorables, a atteint les expositions plasmatiques et dans le SNC souhaitées, et a démontré une augmentation de plus de 50% de l'activité de la glucocérébrosidase (GCase). Les critères secondaires de l'essai de Phase 1b incluent la pharmacocinétique, la modulation de la GCase et les niveaux de biomarqueurs dans le plasma et le liquide céphalorachidien.
Une analyse intermédiaire de l'essai de Phase 1b est attendue d'ici la fin du deuxième trimestre 2025.
Gain Therapeutics (NASDAQ: GANX) hat die Dosierung des ersten Parkinson-Patienten (PD) in seiner klinischen Phase-1b-Studie zu GT-02287, ihrer führenden allosterischen kleinen Molekültherapie, bekannt gegeben. Die offene, multizentrische Studie wird die Sicherheit und Verträglichkeit bei bis zu 20 Teilnehmern mit GBA1-PD und idiopathischer PD über einen Zeitraum von drei Monaten bewerten.
Die Studie folgt auf eine erfolgreiche Phase-1-Studie mit gesunden Freiwilligen, die im dritten Quartal 2024 abgeschlossen wurde, wobei GT-02287 eine günstige Sicherheit und Verträglichkeit zeigte, die gewünschten Plasmakonzentrationen und ZNS-Expositionen erreichte und eine Steigerung der Glukozerebrosidase (GCase)-Aktivität um über 50% demonstrierte. Sekundäre Endpunkte für die Phase-1b-Studie umfassen Pharmakokinetik, GCase-Modulation und Biomarkerwerte im Plasma und im Liquor cerebrospinalis.
Eine Zwischenanalyse der Phase-1b-Studie wird bis Ende des zweiten Quartals 2025 erwartet.
- Successful completion of Phase 1 trial with favorable safety profile
- Significant target engagement with >50% increase in GCase activity
- Achievement of desired plasma and CNS exposures in Phase 1
- Advancement to Phase 1b trial with first patient dosed
- Results from Phase 1b trial not expected until Q2 2025
- participant size (20) in Phase 1b trial
Interim Analysis Expected End of 2Q 2025
BETHESDA, Md., March 14, 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 dosing of the first participant with Parkinson’s disease (PD) in its Phase 1b clinical trial of GT-02287, the Company’s lead allosteric small molecule in development for the treatment of PD with or without a GBA1 mutation.
“Initiation of dosing in our Phase 1b clinical trial represents an important step in the clinical development of GT-02287 and in Gain’s mission to deliver a disease-modifying therapy to people with Parkinson’s disease. We look forward to continued enrollment and anticipate an interim analysis from the towards the end of 2Q 2025,” said Gene Mack, President and CEO of Gain Therapeutics.
The Phase 1b open-label, multi-center trial is designed to evaluate the safety and tolerability of GT-02287 in people with GBA1-PD and idiopathic PD. Secondary endpoints include pharmacokinetics, GCase modulation, levels of GCase substrates, and other biomarkers in plasma and cerebrospinal fluid. The trial will enroll up to 20 participants who will receive GT-02287 daily for three months. Interim data from the Phase 1b trial are anticipated at the end of 2Q 2025.
The Phase 1b trial follows Gain’s 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 >
For more information on the Phase 1b clinical trial, 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 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 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 of neurodegeneration.
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.
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. 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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