Gain Therapeutics Presents Phase 1 GT-02287 Data at International Congress of Parkinson’s Disease and Movement Disorders Demonstrating Increase in GCase Activity in Healthy Volunteers
Gain Therapeutics presented Phase 1 data for GT-02287, its lead drug candidate for Parkinson's disease, at the International Congress of Parkinson's Disease and Movement Disorders. The study demonstrated that GT-02287 was safe and well-tolerated in healthy volunteers, with no serious adverse events. Notably, the drug increased GCase activity by approximately 30% in subjects who received GT-02287, indicating target engagement. The drug also showed CNS exposure, with measurable levels in cerebrospinal fluid.
The company plans to initiate a trial in Parkinson's disease patients by the end of Q4 2024, aiming to demonstrate safety, tolerability, and obtain proof of mechanism based on relevant biomarkers. Gain Therapeutics remains optimistic about GT-02287's potential as a disease-modifying treatment for Parkinson's disease.
Gain Therapeutics ha presentato i dati della Fase 1 per GT-02287, il suo principale candidato farmaco per la malattia di Parkinson, durante il Congresso Internazionale sulla Malattia di Parkinson e sui Disturbi del Movimento. Lo studio ha dimostrato che GT-02287 era sicuro e ben tollerato in volontari sani, senza eventi avversi gravi. In particolare, il farmaco ha aumentato l'attività di GCase di circa il 30% nei soggetti che hanno ricevuto GT-02287, indicando un coinvolgimento del target. Il farmaco ha mostrato anche esposizione al SNC, con livelli misurabili nel fluido cerebrospinale.
L'azienda prevede di iniziare uno studio su pazienti affetti da Parkinson entro la fine del quarto trimestre del 2024, con l'obiettivo di dimostrare sicurezza, tollerabilità e ottenere la prova del meccanismo basata su biomarcatori rilevanti. Gain Therapeutics rimane ottimista riguardo al potenziale di GT-02287 come trattamento modificante la malattia per la malattia di Parkinson.
Gain Therapeutics presentó los datos de la Fase 1 para GT-02287, su principal candidato a fármaco para la enfermedad de Parkinson, en el Congreso Internacional de la Enfermedad de Parkinson y Trastornos del Movimiento. El estudio demostró que GT-02287 era seguro y bien tolerado en voluntarios sanos, sin eventos adversos graves. Notablemente, el fármaco aumentó la actividad de GCase en aproximadamente un 30% en los sujetos que recibieron GT-02287, lo que indica un compromiso del objetivo. El fármaco también mostró exposición al SNC, con niveles medibles en el líquido cefalorraquídeo.
La empresa planea iniciar un ensayo en pacientes con enfermedad de Parkinson para finales del cuarto trimestre de 2024, con el objetivo de demostrar seguridad, tolerabilidad y obtener pruebas del mecanismo basadas en biomarcadores relevantes. Gain Therapeutics sigue siendo optimista sobre el potencial de GT-02287 como un tratamiento modificador de la enfermedad para la enfermedad de Parkinson.
Gain Therapeutics는 파킨슨병에 대한 주요 후보 약물인 GT-02287의 1상 데이터를 파킨슨병 및 운동장애 국제학술대회에서 발표했습니다. 이 연구는 GT-02287가 건강한 자원자들에게 안전하고 잘 견딜 수 있었다는 것을 보여주었으며, 심각한 부작용이 없었다는 점이 주목할 만합니다. 특히, 이 약물은 GT-02287를 받은 피험자에서 GCase 활성이 약 30% 증가하여 목표와의 상호작용을 나타냈습니다. 이 약물은 또한 CNS 노출을 보여주었으며, 뇌척수액에서 측정 가능한 수준이 관찰되었습니다.
회사는 2024년 4분기 말까지 파킨슨병 환자에 대한 임상 시험을 시작할 계획이며, 안전성, 내약성을 입증하고 관련 바이오마커를 기반으로 메커니즘의 증거를 얻는 것을 목표로 하고 있습니다. Gain Therapeutics는 GT-02287가 파킨슨병에 대한 질병 수정 치료제로서의 잠재력에 대해 낙관적입니다.
Gain Therapeutics a présenté les données de Phase 1 pour GT-02287, son principal candidat médicament pour la maladie de Parkinson, lors du Congrès International sur la Maladie de Parkinson et les Troubles du Mouvement. L'étude a montré que GT-02287 était sûr et bien toléré chez des volontaires sains, sans événements indésirables graves. Notamment, le médicament a augmenté l'activité de GCase d'environ 30% chez les sujets ayant reçu GT-02287, ce qui indique une interaction avec la cible. Le médicament a également montré une exposition au SNC, avec des niveaux mesurables dans le liquide céphalorachidien.
L'entreprise prévoit de démarrer un essai chez des patients atteints de Parkinson d'ici la fin du T4 2024, visant à démontrer la sécurité, la tolérabilité et à obtenir une preuve du mécanisme basée sur des biomarqueurs pertinents. Gain Therapeutics reste optimiste quant au potentiel de GT-02287 en tant que traitement modifiant la maladie pour la maladie de Parkinson.
Gain Therapeutics hat die Daten der Phase 1 für GT-02287, seinen Hauptkandidaten für Morbus Parkinson, auf dem Internationalen Kongress für Parkinson-Krankheit und Bewegungsstörungen vorgestellt. Die Studie zeigte, dass GT-02287 bei gesunden Probanden sicher und gut verträglich war, ohne schwere unerwünschte Ereignisse. Bemerkenswerterweise erhöhte das Medikament GCase-Aktivität um etwa 30% bei den Probanden, die GT-02287 erhielten, was auf eine Zielinteraktion hinweist. Das Medikament zeigte auch ZNS-Exposition mit messbaren Konzentrationen in der Gehirn-Rückenmarksflüssigkeit.
Das Unternehmen plant, bis Ende Q4 2024 eine Studie an Patienten mit Parkinson-Krankheit zu starten, mit dem Ziel, Sicherheit und Verträglichkeit zu demonstrieren sowie einen Nachweis des Mechanismus basierend auf relevanten Biomarkern zu erhalten. Gain Therapeutics bleibt optimistisch bezüglich des Potenzials von GT-02287 als krankheitsmodifizierende Therapie für Morbus Parkinson.
- GT-02287 demonstrated safety and tolerability in healthy volunteers
- Increased GCase activity by approximately 30% in subjects receiving GT-02287
- Showed CNS exposure with measurable levels in cerebrospinal fluid
- Linear pharmacokinetic profile across tested dose ranges
- Plasma exposures within projected therapeutic range at daily doses of 7.7 mg/kg and above
- None.
Insights
The Phase 1 data for GT-02287 in healthy volunteers is encouraging for Gain Therapeutics' Parkinson's disease (PD) program. Key findings include:
- Safety and tolerability demonstrated across single and multiple ascending doses
- Linear pharmacokinetics with plasma exposures in projected therapeutic range at
7.7 mg/kg and above - CNS exposure confirmed via measurable levels in cerebrospinal fluid
- Approximately
30% increase in GCase activity in dried blood spots, indicating target engagement - Sustained increase in GCase activity 12 hours post-dose at 14 days
These results support GT-02287's potential as a disease-modifying treatment for PD. The GCase activity increase in healthy volunteers is particularly noteworthy, as it suggests a potentially more pronounced effect in PD patients. The planned Q4 2024 trial in PD patients will be important to demonstrate safety and obtain proof of mechanism via biomarkers. While promising, investors should note that early-stage clinical data doesn't guarantee success in later trials or regulatory approval.
This Phase 1 data release represents a significant milestone for Gain Therapeutics, potentially de-risking their lead asset GT-02287 and Parkinson's disease program. Key financial implications include:
- Increased probability of success for future clinical trials, potentially enhancing the company's valuation
- Stronger position for potential partnerships or licensing deals
- Justification for continued R&D investment in the program
- Potential catalyst for stock price movement, given positive data in a high-value indication
However, investors should consider that GANX is a clinical-stage biotech with a market cap of only
GT-02287 Increases Peripheral GCase Activity
GT-02287 Demonstrates CNS Exposure
Company to Host Webinar to Discuss Results at 8:30 AM ET Today
BETHESDA, Md., Sept. 30, 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 the presentation of a late-breaking abstract at the International Congress of Parkinson’s Disease and Movement Disorders. The poster and oral platform presentation summarize the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics of single- and multiple-ascending doses of GT-02287, the Company’s clinical stage lead drug candidate, in a Phase 1 first-in-human study in healthy volunteers. The International Congress of Parkinson’s Disease and Movement Disorders® is being held September 27 - October 1 in Philadelphia, PA.
“The fact that we saw an increase in GCase activity in healthy volunteers, who we assume have homeostatic mechanisms to counter-regulate a pharmacologically-induced increase in GCase enzymatic activity, indicates that it will have more pronounced effect on GCase in people with Parkinson’s disease (PD) and further confirms our conviction that GT-02287 may be able to slow or stop the progression of PD. We hope that one day we can deliver this drug to those that need it and help them improve their everyday quality of life,” commented Jonas Hannestad, M.D., Ph.D., Chief Medical Officer of Gain.
Gene Mack, interim Chief Executive Officer and current Chief Financial Officer of Gain, continued, “We remain enthusiastic about the promising profile of GT-02287 and look forward to initiating a trial in people with PD by the end of Q4 2024, with the goal of demonstrating safety and tolerability in patients with PD and obtaining proof of mechanism based on relevant biomarkers.”
The late-breaker, titled, “The novel glucocerebrosidase chaperone GT-02287 in development for GBA-PD is safe and well tolerated in healthy volunteers at oral doses that produce plasma exposures in the projected therapeutic range,” was presented on-site by Jonas Hannestad and included results from the Phase 1 study of GT-02287. The single and multiple dose levels tested were safe and generally well tolerated, with no serious adverse events or Grade 3 (severe) adverse events observed, and no other safety signals detected. The PK profile of GT-02287 was linear across the tested dose ranges, and plasma exposures at daily doses of 7.7 mg/kg and above were within the projected therapeutic range. GT-02287 was measurable in cerebrospinal fluid (CSF) at levels in line with rodent levels at effective doses, demonstrating CNS exposure. Notably, GCase activity in dried blood spots increased approximately
A PDF of the poster presented at the International Congress of Parkinson’s Disease and Movement Disorders is available on the Science and Technology section of the Company’s website at https://gaintherapeutics.com/science-and-technology/posters.
Webinar Details
Gain Therapeutics will host a webinar on Monday, September 30 at 8:30 AM ET to review results presented at the International Congress of Parkinson’s Disease and Movement Disorders. To register, please click here: https://lifescievents.com/event/gaintherapeutics/
About GT-02287
Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease 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 mouse models of GBA1-PD, including that presented at FENS Forum 2024 in June describing improvement in cognitive performance in addition to motor performance after administration of GT-02287, suggests that GT-02287 may have the potential to slow 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 for the treatment of Parkinson’s disease with or without a GBA1 mutation, is currently being evaluated in a Phase 1 clinical trial.
Leveraging AI-supported structural biology, proprietary algorithms, and supercomputer-powered physics-based models, the company’s Magellan™ drug discovery platform can identify novel allosteric binding sites on disease-implicated proteins, pinpointing pockets that cannot be found or drugged with current technologies. Its AI and machine-learning tools and virtual screening capabilities leverage the emerging on-demand compound libraries covering vast chemical spaces of over five trillion compounds to identify and select suitable small molecule hits for experimental validation.
Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced 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, risks associated with market conditions and the satisfaction of customary closing conditions related to the offering and uncertainties related to the offerings and the use of proceeds from the offerings. 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 prospectus supplement to be filed with the SEC, and the documents incorporated by reference therein, including the Company’s Form 10-K for the year ended December 31, 2023 and Form 10-Q for the quarter ended June 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
FAQ
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