Gain Therapeutics Announces Positive Topline Results from the Phase 1 Clinical Trial of GT-02287, a Novel GCase-Targeting Small Molecule Therapy for Parkinson’s Disease
Gain Therapeutics (Nasdaq: GANX) has announced positive topline results from its Phase 1 clinical trial of GT-02287, a novel GCase-targeting small molecule therapy for Parkinson's disease. The study, which enrolled 72 healthy volunteers, demonstrated that GT-02287 was safe and well-tolerated at all dose levels, with no discontinuations or serious adverse events reported. Importantly, the drug showed presence in the cerebrospinal fluid and achieved peripheral target engagement.
Based on these promising results, Gain plans to initiate a Phase 1b trial in Parkinson's disease patients by Q4 2024, with data expected by mid-2025. The company aims to demonstrate safety, tolerability, and proof of mechanism through relevant biomarkers in this upcoming trial.
Gain Therapeutics (Nasdaq: GANX) ha annunciato risultati positivi preliminari dal suo trial clinico di Fase 1 per GT-02287, una nuova terapia a piccole molecole mirata a GCase per il morbo di Parkinson. Lo studio, che ha coinvolto 72 volontari sani, ha dimostrato che GT-02287 era sicuro e ben tollerato a tutti i livelli di dosaggio, senza interruzioni o eventi avversi gravi riportati. È importante notare che il farmaco ha mostrato presenza nel liquido cerebrospinale e ha raggiunto un coinvolgimento del bersaglio periferico.
In base a questi risultati promettenti, Gain prevede di avviare un trial di Fase 1b in pazienti con morbo di Parkinson entro il quarto trimestre del 2024, con dati attesi entro la metà del 2025. L'azienda mira a dimostrare sicurezza, tollerabilità e prova di meccanismo attraverso biomarcatori rilevanti in questo prossimo trial.
Gain Therapeutics (Nasdaq: GANX) ha anunciado resultados preliminares positivos de su ensayo clínico de Fase 1 de GT-02287, una nueva terapia de moléculas pequeñas dirigida a GCase para la enfermedad de Parkinson. El estudio, que incluyó a 72 voluntarios sanos, demostró que GT-02287 era seguro y bien tolerado en todos los niveles de dosis, sin interrupciones ni eventos adversos graves reportados. Es importante destacar que el fármaco mostró presencia en el líquido cefalorraquídeo y logró un compromiso con el objetivo periférico.
Basado en estos resultados prometedores, Gain planea iniciar un ensayo de Fase 1b en pacientes con enfermedad de Parkinson para el cuarto trimestre de 2024, con datos esperados para mediados de 2025. La compañía tiene como objetivo demostrar seguridad, tolerancia y prueba del mecanismo a través de biomarcadores relevantes en este próximo ensayo.
게인 테라퓨틱스(Gain Therapeutics)(Nasdaq: GANX)는 파킨슨병을 위한 새로운 GCase 표적 소분자 치료제 GT-02287의 1상 임상 시험에서 긍정적인 주요 결과를 발표했습니다. 본 연구는 72명의 건강한 자원봉사자를 모집했으며, 모든 용량 수준에서 GT-02287가 안전하고 잘 견딜 수 있는 것으로 나타났습니다, 중단이나 심각한 부작용 보고가 없었습니다. 특히 이 약물은 척수액 내 존재가 확인되었으며 말초 표적 작용이 달성되었습니다.
이러한 유망한 결과를 바탕으로 게인은 2024년 4분기까지 파킨슨병 환자를 대상으로 하는 1b상 시험을 시작할 계획입니다, 데이터는 2025년 중반에 기대하고 있습니다. 회사는 이 다가오는 시험을 통해 관련 바이오마커를 통해 안전성, 내약성을 증명하고 메커니즘의 증거를 제시하는 것을 목표로 하고 있습니다.
Gain Therapeutics (Nasdaq: GANX) a annoncé des résultats préliminaires positifs de son essai clinique de Phase 1 pour le GT-02287, une nouvelle thérapie par petites molécules ciblant GCase pour la maladie de Parkinson. L'étude, qui a enrollé 72 volontaires en bonne santé, a démontré que le GT-02287 était sûr et bien toléré à tous les niveaux de dose, sans interruptions ni événements adverses graves signalés. Il est important de noter que le médicament a montré une présence dans le liquide céphalorachidien et a atteint un engagement périphérique ciblé.
Sur la base de ces résultats prometteurs, Gain prévoit de débuter un essai de phase 1b chez des patients atteints de la maladie de Parkinson d'ici le quatrième trimestre 2024, avec des données attendues d'ici mi-2025. L'entreprise vise à démontrer la sécurité, la tolérance et la preuve du mécanisme à travers des biomarqueurs pertinents dans cet essai à venir.
Gain Therapeutics (Nasdaq: GANX) hat positive Top-Line-Ergebnisse aus seiner Phase-1-Studie zu GT-02287, einer neuartigen kleinen Molekültherapie, die auf GCase für die Parkinson-Krankheit abzielt, bekannt gegeben. Die Studie, an der 72 gesunde Freiwillige teilnahmen, zeigte, dass GT-02287 bei allen Dosierungsstufen sicher und gut verträglich war, ohne Meldungen über Abbrüche oder schwerwiegende Nebenwirkungen. Wichtig ist, dass das Medikament im Liquor cerebrospinalis nachgewiesen wurde und periphere Zielbindung erreicht hat.
Aufgrund dieser vielversprechenden Ergebnisse plant Gain, bis zum vierten Quartal 2024 eine Phase-1b-Studie bei Parkinson-Patienten zu beginnen, mit Daten, die bis Mitte 2025 erwartet werden. Das Unternehmen strebt an, Sicherheit, Verträglichkeit und den Nachweis des Mechanismus durch relevante Biomarker in dieser kommenden Studie zu belegen.
- GT-02287 demonstrated safety and tolerability in Phase 1 trial
- Drug showed presence in cerebrospinal fluid and achieved peripheral target engagement
- No discontinuations or serious adverse events reported in the study
- Phase 1b trial in Parkinson's disease patients planned for Q4 2024
- Potential to be a lead treatment for Parkinson's disease with or without GBA1 mutation
- None.
Insights
The Phase 1 results for GT-02287 are highly encouraging for Gain Therapeutics' Parkinson's disease program. The drug demonstrated a favorable safety profile and was well-tolerated across all age groups, including those over 50. Crucially, GT-02287 showed cerebrospinal fluid penetration and peripheral target engagement, two critical factors for potential efficacy in neurodegenerative diseases. These results support advancing to a Phase 1b trial in Parkinson's patients, scheduled for Q4 2024. If successful, this could position GT-02287 as a novel treatment option for both GBA1-associated and idiopathic Parkinson's disease. The planned biomarker analysis in the upcoming patient trial will be pivotal in establishing proof-of-mechanism.
Gain Therapeutics' positive Phase 1 results for GT-02287 represent a significant milestone for the company. The favorable safety profile and target engagement data de-risk the program, potentially increasing its value. With plans to initiate a Phase 1b trial in Q4 2024 and data expected by mid-2025, Gain has clear near-term catalysts that could drive stock performance. However, investors should note that while promising, early-stage clinical success doesn't guarantee late-stage efficacy or regulatory approval. The company's cash runway and ability to fund further development will be important factors to monitor. Overall, this news positions Gain more favorably in the competitive landscape of Parkinson's disease therapeutics.
GT-02287's mechanism as a GCase-targeting small molecule is particularly intriguing for Parkinson's disease treatment. The observed CNS exposure is crucial, as many potential neurological treatments fail due to poor blood-brain barrier penetration. The peripheral target engagement suggests the drug is hitting its intended molecular target, which is promising for potential disease-modifying effects. The plan to include both GBA1-mutated and non-mutated Parkinson's patients in future trials is strategically sound, as it could broaden the drug's applicability. If GT-02287 can demonstrate neuroprotective properties in upcoming trials, it could represent a significant advance over current symptomatic treatments for Parkinson's disease.
Results Include Safety, Tolerability, Pharmacokinetics, and Target Engagement
GT-02287, in Development for GBA1 Parkinson’s Disease, on Track to Initiate Phase 1b Trial in Patients in Q4 2024
BETHESDA, Md., Aug. 29, 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, announces positive results from the Phase 1 study conducted to evaluate the safety, tolerability and pharmacokinetics of GT-02287. The Company previously announced that no discontinuations or serious adverse events were reported when the study concluded. The Phase 1 study enrolled 72 healthy volunteers, males and females, up to the age of 64 years. Review of unblinded data after database lock confirmed that single and multiple doses of GT-02287 were safe and generally well tolerated up to and including the highest planned dose levels across all age groups (approximately
“On the heels of this data, we expect to initiate a trial in people with Parkinson’s disease by Q4 2024 with the goal of demonstrating safety and tolerability in patients with Parkinson disease and to obtain proof of mechanism based on relevant biomarkers. We anticipate having data from Parkinson’s disease patients by mid-point 2025,” commented Jonas Hannestad, M.D., Ph.D., Chief Medical Officer of Gain.
Gene Mack, Interim CEO and current CFO of Gain, continued, “We are enthusiastic about the promising profile of GT-02287, particularly with the added observation of CNS exposure and target engagement. We’re proud to be executing on our near-term clinical milestones and look forward to presenting the full data and advancing GT-02287 further in the clinic with the goal of improving the lives of people with Parkinson’s disease.”
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, increased dopamine levels 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
(212) 845-4242
FAQ
What were the key results of Gain Therapeutics' Phase 1 trial for GT-02287 (GANX)?
When does Gain Therapeutics (GANX) plan to start the Phase 1b trial for GT-02287 in Parkinson's patients?
What is the target population for Gain Therapeutics' GT-02287 (GANX) in Parkinson's disease?
When does Gain Therapeutics (GANX) expect to have data from the Parkinson's disease patient trial for GT-02287?
