Gain Therapeutics Presents Preclinical Data at IAPRD 2025 30th World Congress on Parkinson’s Disease and Related Disorders
Gain Therapeutics (NASDAQ: GANX) presented new preclinical data for GT-02287, their clinical-stage therapy for Parkinson's disease, at the IAPRD 30th World Congress. The study demonstrated GT-02287's neuroprotective properties in cultured rat mesencephalic dopaminergic neurons treated with MPP+, a mitochondrial toxin.
The research showed that GT-02287 improved both lysosomal and mitochondrial function, while reducing α-synuclein aggregation and preventing the release of mitochondrial cytochrome C, a cell death signal. These findings suggest GT-02287's potential as a disease-modifying therapy through its allosteric modulation of GCase and broader neuroprotective effects.
Gain Therapeutics (NASDAQ: GANX) ha presentato nuovi dati preclinici su GT-02287, la loro terapia in fase clinica per il morbo di Parkinson, al 30° Congresso Mondiale IAPRD. Lo studio ha dimostrato le proprietà neuroprotettive di GT-02287 in neuroni dopaminergici mesencefalici di ratto coltivati e trattati con MPP+, una tossina mitocondriale.
La ricerca ha evidenziato che GT-02287 migliora sia la funzione lisosomiale che mitocondriale, riducendo l'aggregazione di α-sinucleina e prevenendo il rilascio del citocromo C mitocondriale, un segnale di morte cellulare. Questi risultati suggeriscono il potenziale di GT-02287 come terapia modificante la malattia, grazie alla sua modulazione allosterica della GCase e ai suoi effetti neuroprotettivi più ampi.
Gain Therapeutics (NASDAQ: GANX) presentó nuevos datos preclínicos de GT-02287, su terapia en fase clínica para la enfermedad de Parkinson, en el 30º Congreso Mundial IAPRD. El estudio demostró las propiedades neuroprotectoras de GT-02287 en neuronas dopaminérgicas mesencefálicas de rata cultivadas y tratadas con MPP+, una toxina mitocondrial.
La investigación mostró que GT-02287 mejoró tanto la función lisosomal como la mitocondrial, reduciendo la agregación de α-sinucleína y previniendo la liberación de citocromo C mitocondrial, una señal de muerte celular. Estos hallazgos sugieren el potencial de GT-02287 como una terapia modificadora de la enfermedad mediante su modulación alostérica de la GCase y sus efectos neuroprotectores más amplios.
Gain Therapeutics (NASDAQ: GANX)는 IAPRD 제30차 세계 총회에서 임상 단계 파킨슨병 치료제 GT-02287의 새로운 전임상 데이터를 발표했습니다. 연구에서는 MPP+라는 미토콘드리아 독소로 처리한 배양된 쥐 중뇌 도파민성 뉴런에서 GT-02287의 신경보호 효과를 입증했습니다.
연구 결과 GT-02287은 리소좀과 미토콘드리아 기능을 모두 개선했으며, α-시누클레인 응집을 감소시키고 세포 사멸 신호인 미토콘드리아 시토크롬 C의 방출을 방지했습니다. 이러한 발견은 GCase의 알로스테릭 조절과 광범위한 신경보호 효과를 통한 질병 수정 치료제로서의 GT-02287 가능성을 시사합니다.
Gain Therapeutics (NASDAQ : GANX) a présenté de nouvelles données précliniques sur GT-02287, leur thérapie en phase clinique pour la maladie de Parkinson, lors du 30e Congrès mondial de l'IAPRD. L'étude a démontré les propriétés neuroprotectrices de GT-02287 sur des neurones dopaminergiques mésencéphaliques de rat cultivés et traités avec le MPP+, une toxine mitochondriale.
La recherche a montré que GT-02287 améliorait à la fois la fonction lysosomale et mitochondriale, tout en réduisant l'agrégation de l'α-synucléine et en empêchant la libération du cytochrome C mitochondrial, un signal de mort cellulaire. Ces résultats suggèrent le potentiel de GT-02287 en tant que thérapie modifiant la maladie grâce à sa modulation allostérique de la GCase et ses effets neuroprotecteurs étendus.
Gain Therapeutics (NASDAQ: GANX) stellte neue präklinische Daten zu GT-02287, ihrer klinischen Therapie für die Parkinson-Krankheit, auf dem 30. Weltkongress der IAPRD vor. Die Studie zeigte die neuroprotektiven Eigenschaften von GT-02287 in kultivierten dopaminergen Neuronen des Rattenmittelhirns, die mit MPP+, einem mitochondrialen Toxin, behandelt wurden.
Die Forschung zeigte, dass GT-02287 sowohl die lysosomale als auch die mitochondriale Funktion verbesserte, die Aggregation von α-Synuclein reduzierte und die Freisetzung von mitochondrialem Cytochrom C, einem Signal für den Zelltod, verhinderte. Diese Ergebnisse deuten auf das Potenzial von GT-02287 als krankheitsmodifizierende Therapie durch allosterische Modulation von GCase und umfassendere neuroprotektive Effekte hin.
- GT-02287 demonstrated multiple beneficial effects: improved lysosomal and mitochondrial function
- The drug showed neuroprotective properties and prevented programmed cell death
- Results suggest potential as a disease-modifying therapy for Parkinson's disease
- None.
Insights
Gain Therapeutics reports promising preclinical data for GT-02287 in Parkinson's disease, demonstrating dual protection mechanisms for neurons.
Gain Therapeutics has presented new preclinical data for their lead compound GT-02287 at the IAPRD World Congress, revealing important mechanistic insights that strengthen its potential as a disease-modifying therapy for Parkinson's disease. The findings significantly expand our understanding of this molecule's neuroprotective capabilities.
The data demonstrates that GT-02287 protects dopaminergic neurons against MPP+, a potent mitochondrial toxin that models Parkinson's disease pathology by inhibiting mitochondrial complex 1. This suggests the compound has dual-action capabilities: it not only improves lysosomal GCase activity (which was previously known) but also appears to act on mitochondrial GCase.
This dual mechanism is particularly noteworthy as Parkinson's disease pathology involves both lysosomal dysfunction and mitochondrial impairment. The compound's ability to address α-synuclein aggregation (a hallmark of Parkinson's), reduce lysosomal dysfunction, mitigate mitochondrial stress, and prevent cytochrome C release (an apoptosis signal) represents a comprehensive approach to neuroprotection.
These preclinical results are scientifically robust as they utilize established models (rat mesencephalic dopaminergic neurons treated with MPP+) that effectively mimic key aspects of Parkinson's pathology. The findings suggest GT-02287 could potentially address multiple pathological mechanisms rather than just targeting a single aspect of the disease.
While these are preclinical findings and will require validation in clinical trials, they provide a stronger mechanistic foundation for GT-02287's development program. The compound's ability to protect neurons through multiple pathways differentiates it from many existing approaches focused solely on symptom management rather than disease modification.
GT-02287 protects dopaminergic neurons against mitochondrial toxin MPP+ suggesting an action on mitochondrial GCase in addition to its improvement of lysosomal GCase activity
BETHESDA, Md., May 12, 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 that a guided poster presentation was made at the International Association of Parkinsonism and Related Disorders (IAPRD) 30th World Congress on Parkinson’s Disease and Related Disorders, held May 7th-10th in New York City, NY. The poster outlined new evidence supporting GT-02287’s ability to provide a broader neuroprotective effect and potential as a disease-modifying therapy for Parkinson’s disease.
“We have continued to add to the preclinical dossier of GT-02287 and we believe these recent data further elucidate the mechanism of action of GT-02287 and the importance of glucocerebrosidase-mediated interactions in the disease pathology of Parkinson’s disease. Importantly, we continue to advance our understanding of stabilization of mitochondria associated with administration of GT-02287 and its allosteric modulation of GCase,” commented Joanne Taylor, Ph.D., Senior Vice President of Research of Gain.
Oral Poster Presentation Information
Title: GT-02287, A Clinical-stage Allosteric GCase Modulator For The Treatment Of Parkinson’s Disease, Protects Dopaminergic Neurons Against Mitochondrial Toxin MPP+
Summary: In cultured rat mesencephalic dopaminergic neurons treated with MPP+, a mitochondrial toxin that induces mitochondrial impairment by inhibiting mitochondrial complex 1, GT-02287 improved the function of both the lysosomes and mitochondria and exerted a broader neuroprotective effect. GT-02287 lowered α-synuclein aggregation, lysosomal dysfunction, and mitochondrial stress while also preventing the release of mitochondrial cytochrome C, a programmed cell death (apoptosis) signal, thereby promoting neuronal survival.
A PDF of the poster presented at IAPRD 2025 30th World Congress on Parkinson’s Disease and Related Disorders is available on the Science and Technology section of the Company’s website at https://gaintherapeutics.com/science-and-technology/posters.
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 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.
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Gain Therapeutics, Inc.
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FAQ
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