Gain Therapeutics Receives Australian Approval of Phase 1b Dosing Extension and Reports Positive Independent Data Monitoring Committee Recommendation
Gain Therapeutics (Nasdaq: GANX) has received approval from Australian authorities to extend the dosing period in its Phase 1b clinical trial of GT-02287 by an additional nine months. The extension allows participants who completed the initial 90-day treatment period to continue for up to 12 months total.
Additionally, the independent Data Monitoring Committee (DMC) has recommended continuing the study without modifications after reviewing interim safety data, finding no safety concerns. The majority of participants who completed the initial 90-day period have expressed interest in joining the extension phase.
The extended study will assess long-term safety, tolerability, functional changes according to the Movement Disorder Society Unified Parkinson's Disease Rating Scale, and biomarker activity from cerebrospinal fluid, with data intended to support Phase 2 planning.
Gain Therapeutics (Nasdaq: GANX) ha ottenuto l'autorizzazione delle autorità australiane ad estendere di altri nove mesi il periodo di somministrazione nel suo trial di Fase 1b con GT-02287. L'estensione consente ai partecipanti che hanno completato il trattamento iniziale di 90 giorni di proseguire fino a un massimo di 12 mesi.
Inoltre, l'indipendente Comitato di Monitoraggio dei Dati (DMC) ha raccomandato di proseguire lo studio senza modifiche dopo aver esaminato i dati provvisori sulla sicurezza, non riscontrando problemi. La maggioranza dei partecipanti che ha concluso i 90 giorni iniziali ha manifestato interesse per la fase di estensione.
Lo studio prolungato valuterà la sicurezza a lungo termine, la tollerabilità, i cambiamenti funzionali secondo la Movement Disorder Society Unified Parkinson's Disease Rating Scale e l'attività dei biomarcatori nel liquido cerebrospinale, con dati destinati a supportare la pianificazione della Fase 2.
Gain Therapeutics (Nasdaq: GANX) ha recibido la aprobación de las autoridades australianas para ampliar en nueve meses el periodo de dosificación en su ensayo de fase 1b con GT-02287. La extensión permite que los participantes que completaron el tratamiento inicial de 90 días continúen hasta un total de 12 meses.
Además, el Comité Independiente de Seguimiento de Datos (DMC) recomendó continuar el estudio sin modificaciones tras revisar los datos provisionales de seguridad, sin identificar problemas. La mayoría de los participantes que finalizaron los 90 días iniciales han mostrado interés en la fase de extensión.
El estudio ampliado evaluará la seguridad a largo plazo, la tolerabilidad, los cambios funcionales según la Movement Disorder Society Unified Parkinson's Disease Rating Scale y la actividad de biomarcadores en el líquido cefalorraquídeo, con datos destinados a apoyar la planificación de la fase 2.
Gain Therapeutics (Nasdaq: GANX)가 GT-02287의 임상 1b상 투약 기간을 호주 규제당국으로부터 추가 9개월 연장하는 승인을 받았습니다. 이번 연장으로 초기 90일 치료를 완료한 참가자는 총 최대 12개월까지 계속 참여할 수 있습니다.
또한, 독립 데이터 모니터링 위원회(DMC)는 중간 안전성 데이터를 검토한 후 안전성 우려가 없다고 판단하여 수정 없이 연구를 계속할 것을 권고했습니다. 초기 90일을 완료한 참가자 대부분이 연장 단계 참여에 관심을 보였습니다.
연장된 연구는 뇌척수액의 바이오마커 활성과 함께 장기 안전성, 내약성, 기능적 변화를 Movement Disorder Society Unified Parkinson's Disease Rating Scale에 따라 평가하며, 이 데이터는 2상 계획 수립을 지원하는 데 사용될 예정입니다.
Gain Therapeutics (Nasdaq: GANX) a obtenu l'approbation des autorités australiennes pour prolonger de neuf mois la période d'administration dans son essai de phase 1b de GT-02287. Cette extension permet aux participants ayant achevé la période initiale de 90 jours de continuer jusqu'à 12 mois au total.
De plus, le Comité indépendant de surveillance des données (DMC) a recommandé la poursuite de l'étude sans modification après examen des données provisoires de sécurité, ne relevant aucun problème. La majorité des participants ayant terminé les 90 jours initiaux ont manifesté leur intérêt pour la phase d'extension.
L'étude prolongée évaluera la sûreté à long terme, la tolérance, les changements fonctionnels selon la Movement Disorder Society Unified Parkinson's Disease Rating Scale et l'activité des biomarqueurs dans le liquide cérébro-spinal, les données devant soutenir la planification de la phase 2.
Gain Therapeutics (Nasdaq: GANX) hat von den australischen Behörden die Genehmigung erhalten, den Dosierungszeitraum in seiner Phase-1b-Studie mit GT-02287 um weitere neun Monate zu verlängern. Die Verlängerung ermöglicht es Teilnehmern, die die anfängliche 90-tägige Behandlung abgeschlossen haben, bis zu insgesamt 12 Monate weiterzuführen.
Darüber hinaus hat das unabhängige Data Monitoring Committee (DMC) nach Prüfung der vorläufigen Sicherheitsdaten empfohlen, die Studie ohne Änderungen fortzusetzen, da keine Sicherheitsbedenken festgestellt wurden. Die Mehrheit der Teilnehmer, die die ersten 90 Tage abgeschlossen haben, hat ihr Interesse an der Anschlussphase bekundet.
Die verlängerte Studie wird Langzeitsicherheit, Verträglichkeit, funktionelle Veränderungen gemäß der Movement Disorder Society Unified Parkinson's Disease Rating Scale sowie Biomarker-Aktivität im Liquor untersuchen; die Daten sollen die Planung von Phase 2 unterstützen.
- Approval received to extend Phase 1b trial dosing from 3 to 12 months
- Positive DMC safety review with recommendation to continue without changes
- High participant interest in continuing treatment in extension phase
- Additional data collection will support Phase 2 planning
- None.
Insights
Gain Therapeutics' Phase 1b trial extension and positive safety review signal encouraging progress for their Parkinson's disease drug candidate.
The extension of Gain's Phase 1b trial for GT-02287 from 3 months to a full year represents a significant development in their clinical program. This extension wouldn't typically be granted without preliminary signals suggesting acceptable safety and potentially promising efficacy. Most telling is that the majority of participants who completed the initial 90-day period have expressed interest in continuing treatment - a positive indicator of both tolerability and perceived benefit.
The independent Data Monitoring Committee's recommendation to continue without modifications is particularly meaningful at this stage. DMCs evaluate unblinded data and are tasked with protecting patient safety. Their positive assessment suggests no concerning safety signals have emerged that would necessitate protocol adjustments or study termination.
For a potential disease-modifying Parkinson's therapy, the extended timeframe is crucial. Neurodegenerative conditions progress slowly, making longer observation periods essential for detecting meaningful changes. The additional nine months will allow for more robust assessments using the Movement Disorder Society Unified Parkinson's Disease Rating Scale and evaluation of cerebrospinal fluid biomarkers - both critical endpoints for demonstrating disease modification rather than just symptomatic improvement.
This extension effectively transforms what was initially a primarily safety-focused Phase 1b study into a longer trial that can generate preliminary efficacy signals, potentially accelerating GT-02287's development timeline by providing data that will inform and optimize the design of subsequent Phase 2 trials.
Ongoing Phase 1b trial of GT-02287 to be extended by an additional nine months for participants who enrolled in the original three-month study design
The independent data monitoring committee (DMC) met recently and recommended to continue the study with no changes to study design
BETHESDA, Md., Sept. 04, 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 Company has received approval from the Bellberry Human Research Ethics Committee and Alfred Hospital Ethics Committee to extend dosing by an additional nine months in its ongoing Phase 1b clinical study of GT-022887, beyond the 90-day period currently allowed in protocol.
The dosing extension will be offered to participants who have completed 90 days of treatment and will provide them the opportunity to continue treatment with GT-02287 for a total of 12 months. The majority of participants who have already completed the 90-day dosing period have confirmed their interest in joining the extension.
Separately, the independent data monitoring committee (DMC) has identified no safety concerns and recommended to continue the study with no changes after its review of interim safety data from the ongoing Phase 1b study of GT-02287.
“The approval by Australian authorities of the dosing extension and the DMC’s positive recommendation represent important milestones in GT-02287’s clinical development program,” said Gene Mack, President and CEO of Gain Therapeutics. “We look forward to transitioning eligible participants into the extension phase to further assess long-term safety, tolerability, functional changes scored according to the Movement Disorder Society Unified Parkinson’s Disease Rating Scale, and biomarker activity from cerebrospinal fluid. The additional data is intended to support Phase 2 planning and the decision to extend dosing reflects encouraging early feedback from patients and clinicians, while underscoring our commitment to developing a disease-modifying therapy for Parkinson’s disease,” concluded Mr. Mack.
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, 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.
Apaar Jammu
Manager, Investor Relations and Public Relations
ajammu@gaintherapeutics.com
LifeSci Advisors LLC
Chuck Padala
Managing Director
chuck@lifesciadvisors.com
Media:
Russo Partners LLC
Nic Johnson and Elio Ambrosio
nic.johnson@russopartnersllc.com
elio.ambrosio@russopartnersllc.com
(760) 846-9256
FAQ
What is the significance of GANX's Phase 1b trial extension approval?
What did the Data Monitoring Committee conclude about GANX's GT-02287 trial?
What will Gain Therapeutics evaluate during the extended trial period?
How many additional months of treatment were approved for GANX's Phase 1b trial?
What is GT-02287 being developed for by Gain Therapeutics?
