Gain Therapeutics Announces Completion of Target Enrollment in Phase 1b Clinical Study Evaluating GT-02287 in People With Parkinson’s Disease With or Without a GBA1 Mutation
Gain Therapeutics (NASDAQ:GANX) has announced the successful completion of target enrollment in its Phase 1b clinical study of GT-02287 for Parkinson's Disease. The trial has enrolled 16 participants, exceeding the original goal of 15 participants ahead of schedule.
The company reports that analysis of 90-day biomarker activity from cerebrospinal fluid (CSF) is now expected in Q4 2025, earlier than the initially planned Q1 2026. The screening window for additional participants has been extended through July 31st, 2025, responding to continued interest from clinicians and patients.
Importantly, the independent data monitoring committee (DMC) has recommended continuing the study with no changes in dose level, and no serious treatment emergent adverse events (TEAEs) have been reported. Gain is planning to approach Australian health authorities to extend the dosing period beyond the current 90-day protocol, with long-term chronic toxicology studies nearing completion.
Gain Therapeutics (NASDAQ:GANX) ha annunciato il completamento con successo dell'arruolamento previsto nello studio clinico di Fase 1b di GT-02287 per il Morbo di Parkinson. Lo studio ha arruolato 16 partecipanti, superando l'obiettivo iniziale di 15 partecipanti in anticipo rispetto ai tempi previsti.
L'azienda comunica che l'analisi dell'attività del biomarcatore a 90 giorni dal liquido cerebrospinale (CSF) è ora prevista per il quarto trimestre 2025, anticipando rispetto alla pianificazione iniziale del primo trimestre 2026. La finestra di screening per ulteriori partecipanti è stata estesa fino al 31 luglio 2025, in risposta all'interesse continuo di medici e pazienti.
È importante sottolineare che il comitato indipendente di monitoraggio dei dati (DMC) ha raccomandato di proseguire lo studio senza modifiche al livello di dosaggio e non sono stati segnalati eventi avversi gravi correlati al trattamento (TEAEs). Gain prevede di rivolgersi alle autorità sanitarie australiane per estendere il periodo di somministrazione oltre il protocollo attuale di 90 giorni, con studi tossicologici cronici a lungo termine ormai quasi completati.
Gain Therapeutics (NASDAQ:GANX) ha anunciado la finalización exitosa del reclutamiento objetivo en su estudio clínico de Fase 1b de GT-02287 para la Enfermedad de Parkinson. El ensayo ha reclutado a 16 participantes, superando la meta original de 15 participantes antes de lo previsto.
La compañía informa que el análisis de la actividad del biomarcador a 90 días en el líquido cefalorraquídeo (LCR) se espera ahora para el cuarto trimestre de 2025, antes de lo planeado inicialmente para el primer trimestre de 2026. La ventana de selección para participantes adicionales se ha extendido hasta el 31 de julio de 2025, respondiendo al interés continuo de médicos y pacientes.
Es importante destacar que el comité independiente de monitoreo de datos (DMC) ha recomendado continuar el estudio sin cambios en el nivel de dosis, y no se han reportado eventos adversos graves emergentes del tratamiento (TEAEs). Gain planea solicitar a las autoridades sanitarias australianas extender el periodo de dosificación más allá del protocolo actual de 90 días, con estudios toxicológicos crónicos a largo plazo casi finalizados.
Gain Therapeutics (NASDAQ:GANX)는 파킨슨병 치료제 GT-02287의 1b상 임상시험 목표 등록을 성공적으로 완료했다고 발표했습니다. 이번 시험에는 원래 목표였던 15명을 초과한 16명의 참가자가 예정보다 일찍 등록되었습니다.
회사는 뇌척수액(CSF) 내 90일 바이오마커 활성 분석 결과가 당초 계획된 2026년 1분기보다 앞당겨져 2025년 4분기에 나올 것으로 예상한다고 밝혔습니다. 추가 참가자 모집 기간은 임상의와 환자의 지속적인 관심에 대응하여 2025년 7월 31일까지 연장되었습니다.
중요하게도 독립 데이터 모니터링 위원회(DMC)는 용량 변경 없이 연구를 계속할 것을 권고했으며, 심각한 치료 관련 이상반응(TEAEs)은 보고되지 않았습니다. Gain은 현재 90일 프로토콜을 넘어 투여 기간을 연장하기 위해 호주 보건 당국에 접근할 계획이며, 장기 만성 독성 연구도 거의 완료 단계에 있습니다.
Gain Therapeutics (NASDAQ:GANX) a annoncé la réussite de l'enrôlement cible dans son étude clinique de phase 1b sur le GT-02287 pour la maladie de Parkinson. L'essai a recruté 16 participants, dépassant l'objectif initial de 15 participants, et ce, en avance sur le calendrier prévu.
L'entreprise indique que l'analyse de l'activité du biomarqueur à 90 jours dans le liquide céphalo-rachidien (LCR) est désormais attendue au 4e trimestre 2025, plus tôt que le premier trimestre 2026 initialement prévu. La période de sélection pour des participants supplémentaires a été prolongée jusqu'au 31 juillet 2025, en réponse à l'intérêt continu des cliniciens et des patients.
Il est important de noter que le comité indépendant de surveillance des données (DMC) a recommandé de poursuivre l'étude sans modification du niveau de dose, et aucun événement indésirable grave lié au traitement (TEAEs) n'a été rapporté. Gain prévoit de solliciter les autorités sanitaires australiennes pour prolonger la période de dosage au-delà du protocole actuel de 90 jours, des études toxicologiques chroniques à long terme étant sur le point d'être achevées.
Gain Therapeutics (NASDAQ:GANX) hat den erfolgreichen Abschluss der Zielrekrutierung in seiner Phase-1b-Studie zu GT-02287 bei Parkinson bekanntgegeben. Die Studie hat 16 Teilnehmer eingeschlossen und damit das ursprüngliche Ziel von 15 Teilnehmern vorzeitig übertroffen.
Das Unternehmen berichtet, dass die Analyse der 90-Tage-Biomarker-Aktivität aus der Liquor cerebrospinalis (CSF) nun für das 4. Quartal 2025 erwartet wird, früher als ursprünglich für das 1. Quartal 2026 geplant. Das Screening-Fenster für weitere Teilnehmer wurde bis zum 31. Juli 2025 verlängert, um dem anhaltenden Interesse von Klinikern und Patienten gerecht zu werden.
Wichtig ist, dass das unabhängige Datenüberwachungskomitee (DMC) empfohlen hat, die Studie ohne Änderungen der Dosierung fortzusetzen, und es wurden keine schwerwiegenden behandlungsbedingten Nebenwirkungen (TEAEs) gemeldet. Gain plant, die australischen Gesundheitsbehörden anzusprechen, um den Dosierungszeitraum über das aktuelle 90-Tage-Protokoll hinaus zu verlängern, da langfristige chronische Toxikologiestudien kurz vor dem Abschluss stehen.
- Achieved target enrollment ahead of schedule with 16 participants
- Earlier timeline for biomarker analysis results (Q4 2025 vs Q1 2026)
- No serious treatment emergent adverse events reported
- DMC recommended continuation of study with no dose level changes
- Strong interest from clinicians and patients leading to extended screening window
- None.
Insights
Gain's Parkinson's drug GT-02287 shows encouraging progress with accelerated trial enrollment and clean safety profile, speeding up key biomarker readout.
Gain Therapeutics has reached a significant clinical milestone with their Phase 1b trial of GT-02287 for Parkinson's Disease, completing target enrollment ahead of schedule. The study has successfully enrolled 16 participants against an original target of 15, despite an initial two-month implementation delay. This accelerated enrollment shifts the timeline for critical cerebrospinal fluid (CSF) biomarker analysis forward to Q4 2025 from the initially projected Q1 2026.
The most encouraging aspect of this development is the clean safety profile observed thus far. The independent data monitoring committee has recommended continuation without dose modifications, and no serious treatment-emergent adverse events have been reported. This safety profile is particularly important for potential chronic administration in a neurodegenerative condition like Parkinson's.
The continued interest from both clinicians and patients has prompted the company to extend the screening window through July 31st, 2025, potentially increasing the participant pool. Furthermore, Gain is pursuing regulatory approval to extend the dosing period beyond the current 90-day protocol, supported by nearly complete long-term toxicology studies. This extension could provide valuable insights for the planned Phase 2 trial design.
The focus on cerebrospinal fluid biomarkers is strategically sound for a neurological drug candidate. CSF biomarkers provide more direct evidence of central nervous system target engagement and drug activity than blood biomarkers, offering stronger proof-of-concept data. For GT-02287, an allosteric small molecule therapy aimed at potentially modifying disease progression rather than just managing symptoms, demonstrating biomarker changes in CSF would significantly de-risk further development.
This development is particularly noteworthy given the substantial unmet need in Parkinson's Disease, which affects over one million Americans. Current treatments focus solely on symptomatic relief without addressing disease progression, highlighting the potential market opportunity for disease-modifying therapies like GT-02287.
As of June 30th, 16 participants have enrolled in the Phase 1b, ahead of the original goal of at least 15 total participants by 3Q 2025
Analysis of 90-day biomarker activity from cerebrospinal fluid (CSF) in all participants currently enrolled is now expected to also track earlier; available in 4Q 2025 instead of 1Q 2026
Gain will continue screening patients for enrollment through July 31st, 2025, at the request of clinical investigators and additional participants who wish to gain access to the study
The independent data monitoring committee (DMC) met recently and recommended to continue the study with no changes in dose level; no serious treatment emergent adverse events (TEAEs) have occurred
BETHESDA, Md., June 30, 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 provided an update on the progress of its ongoing Phase 1b clinical study evaluating the safety and tolerability of GT-02287 in people with Parkinson’s Disease with or without a GBA1 mutation. The study, which aimed to enroll 15-20 participants, reached 16 participants on June 30th, 2025.
"We are extremely pleased we’ve reached our target enrollment for our Phase 1b trial of GT-02287, even with an initial two-month delay in protocol implementation," said Gene Mack, President and CEO of Gain Therapeutics. “The faster-than-anticipated enrollment means all those participants will reach their 90-day visit in time to facilitate biomarker analysis of all CSF samples taken by fourth quarter of this year instead of the first quarter of 2026. Based on the greater relevance of CSF biomarkers as proof of drug activity, and considering our new timeline, Gain is expected to provide full results on 90-day biomarker evidence from all CSF and blood samples together during 4Q 2025, which is also earlier than originally planned.”
Additionally, to facilitate the continued interest on the part of clinicians and patients currently aware of the Phase 1b study in Australia, Gain will extend the screening window for participants through July 31st, 2025. Gain is also planning to approach the local health authorities in Australia to extend the dosing period of the Phase 1b beyond the 90-day period currently allowed in the protocol. Long-term chronic toxicology studies required to support this extension of dosing are near completion, and Gain is expected to provide an update on the progress of the extension before the end of 3Q25. Gain believes that the extension of the Phase 1b will also better inform Phase 2 protocol design and planning for early 2026.
Mr. Mack continued, "We are grateful to the patients and clinicians whose strong interest in novel, potentially disease modifying treatments for Parkinson’s Disease, together with previous evidence of GT-02287 target engagement from our Phase 1 healthy volunteer study, led to completing enrollment of the study before the end of the summer as originally planned. Current treatments for Parkinson’s disease are aimed at alleviating symptoms while there are no treatments available to impact the eventual progression of Parkinson’s Disease beyond transient symptom relief. There are over 1 million Americans with Parkinson’s Disease making this population of individuals among the larger populations with a significant unmet need.”
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 completion 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; 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.
Investors:
Gain Therapeutics, Inc.
Apaar Jammu
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ajammu@gaintherapeutics.com
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