Gain Therapeutics CEO Gene Mack Issues Letter To Shareholders and Provides Operational Update
Gain Therapeutics (NASDAQ: GANX) provided a shareholder update outlining key milestones for 2025, focusing on the clinical development of GT-02287, a potential disease-modifying therapy for Parkinson's disease. The company received approval to initiate a Phase 1b clinical trial following successful completion of healthy volunteer studies that showed a 53% increase in GCase activity.
The Phase 1b trial will enroll 15-20 individuals with GBA1 or idiopathic Parkinson's disease, with enrollment updates expected by Q1 2025 and interim analysis guidance in Q2 2025. The company held a productive pre-IND meeting with FDA in December 2024, with no significant regulatory hurdles identified.
As of September 30, 2024, the company had approximately $12M in cash, expected to be sufficient through the interim analysis of the Phase 1b trial. The company is exploring potential licensing partnerships and financing options while preparing for Phase 2 studies anticipated in H2 2025.
Gain Therapeutics (NASDAQ: GANX) ha fornito un aggiornamento agli azionisti delineando i traguardi chiave per il 2025, concentrandosi sullo sviluppo clinico di GT-02287, una potenziale terapia modificante la malattia per il morbo di Parkinson. L'azienda ha ricevuto l'approvazione per avviare uno studio clinico di Fase 1b, dopo il completamento con successo di studi su volontari sani che hanno mostrato un aumento del 53% nell'attività di GCase.
Il trial di Fase 1b prevede l'arruolamento di 15-20 individui affetti da GBA1 o morbo di Parkinson idiopatico, con aggiornamenti sull'arruolamento previsti entro il primo trimestre del 2025 e indicazioni per l'analisi intermedia nel secondo trimestre del 2025. L'azienda ha tenuto un incontro pre-IND produttivo con la FDA nel dicembre 2024, senza ostacoli normativi significativi identificati.
Alla data del 30 settembre 2024, l'azienda disponeva di circa $12 milioni in contanti, che si prevede siano sufficienti fino all'analisi intermedia del trial di Fase 1b. L'azienda sta esplorando potenziali partenariati di licenza e opzioni di finanziamento mentre si prepara per gli studi di Fase 2 previsti nella seconda metà del 2025.
Gain Therapeutics (NASDAQ: GANX) proporcionó una actualización a los accionistas delineando hitos clave para 2025, centrándose en el desarrollo clínico de GT-02287, una terapia potencialmente modificadora de la enfermedad para el Parkinson. La empresa recibió aprobación para iniciar un ensayo clínico de Fase 1b tras la exitosa finalización de estudios en voluntarios sanos que mostraron un aumento del 53% en la actividad de GCase.
El ensayo de Fase 1b inscribirá a 15-20 individuos con enfermedad de Parkinson idiopática o GBA1, con actualizaciones de inscripción esperadas para el primer trimestre de 2025 y orientación sobre análisis intermedios en el segundo trimestre de 2025. La empresa celebró una reunión pre-IND productiva con la FDA en diciembre de 2024, sin identificar obstáculos regulatorios significativos.
Al 30 de septiembre de 2024, la empresa contaba con aproximadamente $12 millones en efectivo, que se espera sean suficientes hasta el análisis intermedio del ensayo de Fase 1b. La empresa está explorando posibles asociaciones de licencia y opciones de financiamiento mientras se prepara para los estudios de Fase 2 previstos para la segunda mitad de 2025.
게인 테라퓨틱스 (NASDAQ: GANX)는 2025년의 주요 이정표를 개략적으로 설명하는 주주 업데이트를 제공하며, 파킨슨병을 위한 잠재적 질병 과정 변경 치료제인 GT-02287의 임상 개발에 초점을 맞췄습니다. 회사는 GCase 활동에서 53% 증가를 보인 건강한 자원봉사자 연구의 성공적인 완료 후, 1b상 임상 시험을 시작할 승인을 받았습니다.
1b상 시험에는 GBA1 또는 특발성 파킨슨병을 앓고 있는 15-20명의 개인이 등록될 예정이며, 등록 업데이트는 2025년 1분기 내에 예상되며, 임시 분석에 대한 안내는 2025년 2분기에 제공될 예정입니다. 회사는 2024년 12월 FDA와의 생산적인 Pre-IND 회의를 개최했으며, 유의미한 규제 장벽은 식별되지 않았습니다.
2024년 9월 30일 기준으로 회사는 약 1200만 달러의 현금을 보유하고 있으며, 1b상 시험의 중간 분석까지 충분할 것으로 예상됩니다. 회사는 2025년 하반기에 예상되는 2상 연구를 준비하는 동안 잠재적인 라이선스 파트너십 및 자금 조달 옵션을 탐색하고 있습니다.
Gain Therapeutics (NASDAQ: GANX) a fourni une mise à jour aux actionnaires décrivant les étapes clés pour 2025, en se concentrant sur le développement clinique de GT-02287, une thérapie potentiellement modifiant l'évolution de la maladie de Parkinson. L'entreprise a obtenu l'approbation pour débuter un essai clinique de Phase 1b après l'achèvement réussi d'études sur des volontaires sains montrant une augmentation de 53% de l'activité de GCase.
L'essai de Phase 1b recrutera 15-20 individus souffrant de la maladie de Parkinson idiopathique ou de GBA1, avec des mises à jour sur le recrutement attendues d'ici le premier trimestre 2025 et des indications pour une analyse intermédiaire au deuxième trimestre 2025. L'entreprise a eu une réunion pré-IND productive avec la FDA en décembre 2024, sans obstacles réglementaires significatifs identifiés.
Au 30 septembre 2024, l'entreprise disposait d'environ 12 millions de dollars en liquidités, ce qui devrait être suffisant jusqu'à l'analyse intermédiaire de l'essai de Phase 1b. L'entreprise explore d'éventuels partenariats de licence et options de financement tout en se préparant aux études de Phase 2 anticipées dans la seconde moitié de 2025.
Gain Therapeutics (NASDAQ: GANX) hat ein Update für die Aktionäre bereitgestellt, in dem die wichtigsten Meilensteine für 2025 umrissen werden, wobei der Schwerpunkt auf der klinischen Entwicklung von GT-02287 liegt, einer potenziellen krankheitsmodifizierenden Therapie für die Parkinson-Krankheit. Das Unternehmen erhielt die Genehmigung zur Einleitung einer Phase-1b-Studie nach dem erfolgreichen Abschluss von Studien mit gesunden Freiwilligen, die eine 53%ige Steigerung der GCase-Aktivität zeigten.
Die Phase-1b-Studie wird 15-20 Personen mit GBA1 oder idiopathischem Parkinson rekrutieren, wobei die Einschreibungsupdates bis zum ersten Quartal 2025 erwartet werden und die Zwischenanalyse-Leitlinien im zweiten Quartal 2025 bereitgestellt werden. Das Unternehmen führte im Dezember 2024 ein produktives Pre-IND-Meeting mit der FDA durch, wobei keine wesentlichen regulatorischen Hürden festgestellt wurden.
Zum 30. September 2024 verfügte das Unternehmen über etwa 12 Millionen US-Dollar in bar, was voraussichtlich bis zur Zwischenanalyse der Phase-1b-Studie ausreicht. Das Unternehmen untersucht potenzielle Lizenzpartnerschaften und Finanzierungsoptionen, während es sich auf die für das zweite Halbjahr 2025 erwarteten Phase-2-Studien vorbereitet.
- Successful completion of healthy volunteer studies showing 53% increase in GCase activity
- FDA pre-IND meeting completed with no significant regulatory hurdles identified
- $12M cash position sufficient through Phase 1b interim analysis
- Phase 1b trial approval received and ready to begin patient dosing
- Chronic toxicology studies progressing for Phase 2 preparation
- Additional financing needed for Phase 2 studies in H2 2025
- Current cash runway only extends through Q2 2025
Insights
The operational update from Gain Therapeutics reveals several important developments that position GT-02287 as a potentially groundbreaking treatment for Parkinson's disease. The 53% increase in glucocerebrosidase (GCase) activity observed in the healthy volunteer study is particularly significant, as GCase dysfunction is a key pathological feature in both GBA1 and idiopathic Parkinson's disease.
The Phase 1b trial design is strategically sound, targeting both GBA1 and idiopathic Parkinson's patients, which broadens the potential market opportunity. The comprehensive biomarker evaluation strategy, including GCase activity, lysosomal substrate reduction and inflammatory markers, will provide important insights into GT-02287's mechanism of action and potential efficacy.
From a regulatory perspective, the successful pre-IND meeting with the FDA substantially derisks the development pathway. The absence of significant regulatory hurdles suggests a smoother transition to Phase 2 studies, potentially accelerating the development timeline.
The financial position requires careful attention. With
Critical upcoming catalysts include:
- Phase 1b enrollment update (Q1 2025)
- Interim analysis results (Q2 2025)
- Completion of 6-month and 9-month toxicology studies (Q2/Q3 2025)
- Phase 2 planning initiation (H2 2025)
The company's focus on elucidating GT-02287's broader neuroprotective role, particularly its effects on mitochondrial stabilization, could differentiate it from existing treatments and potentially position it as the first disease-modifying therapy for Parkinson's disease.
BETHESDA, Md., Feb. 06, 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 the following letter to shareholders from the Company’s President and CEO, Gene Mack:
To our valued shareholders,
With 2025 poised to be another pivotal year for Gain Therapeutics, I want to take a moment to provide an update on the key milestones we are expecting to achieve in 2025. Building on the accomplishments and dedicated work of the entire Gain team in 2024, we continue to advance the clinical development of GT-02287, a potentially disease-modifying therapy for Parkinson’s disease (PD) in people with or without a GBA1 mutation.
In December 2024, we received approval to initiate our Phase 1b clinical trial of GT-02287 in people with GBA1 or idiopathic Parkinson’s disease. This followed the completion of our study in healthy volunteers during 3Q 2024 that demonstrated the safety and tolerability of GT-02287 as well as target engagement with a
As a reminder, we are planning on enrolling 15-20 individuals with either GBA1 or idiopathic Parkinson’s disease – all of whom will receive oral GT-02287 daily for three months in the open-label Phase 1b clinical trial. We plan to evaluate different biomarkers of Parkinson’s disease from the cerebrospinal fluid and plasma samples taken from participants at baseline and completion of the study. Key biomarkers are anticipated to include GCase enzyme activity, lysosomal substrate reduction, inflammatory markers, and other disease markers believed to be impacted by GT-02287’s trafficking of GCase as it performs multiple neuroprotective functions in various cellular compartments.
We will also continue building upon the already extensive preclinical dossier for GT-02287 under the leadership of Joanne Taylor, Senior VP of Research, and Terenzio Ignoni, Senior VP of Technical Operations. Together, they will look to further characterize GT-02287’s mechanism of action and elucidate the broader neuroprotective role of GCase, suggested by the work of their respective teams, that goes beyond the lysosome to other cellular compartments including the endoplasmic reticulum and mitochondria. We have observed the stabilization of the mitochondria associated with administration of GT-02287 and its allosteric modulation of GCase. Importantly, we continue to advance our understanding of the pharmacokinetic and pharmacodynamic properties of GT-02287, helping our preparation for extending the dosing duration in patients beyond three months, which we anticipate to occur upon completion of ongoing six and nine month chronic animal tox studies - expected to be completed during 2Q 2025 and 3Q 2025, respectively, in preparation for Phase 2 planning during 2H 2025.
In 2025, we will remain focused on strengthening our collaboration with the FDA and other regulatory authorities to expand the clinical development of GT-02287 within the U.S. In early December 2024, we held a productive pre-IND meeting with the FDA and received encouraging feedback that aligned with our expectations. Notably, the FDA did not identify any significant regulatory hurdles that could delay our preparations for Phase 2. We believe this progress derisks our upcoming IND submission and offers flexibility to potentially expand our current Phase 1b trial. We look forward to ongoing engagement with the FDA as we advance these critical efforts and will leverage the experience of our newly formed Clinical Advisory Board as needed.
Our capital position remains in line with our guidance since the company’s 2Q 2024 capital raise of approximately
We believe that our Phase 1b clinical trial will illustrate that the broad neuroprotective effect demonstrated in pre-clinical models of Parkinson’s disease will translate to human participants with Parkinson’s disease. We believe this may unlock significant value for the GT-02287 development program through 2025 and beyond. We are grateful for the strong support we have received from the Parkinson’s community, including thought leaders and especially patients who are entrusting us to make meaningful progress towards addressing the underlying mechanisms causing the debilitating effects of Parkinson’s disease.
Parkinson’s disease remains the second most common neurodegenerative disease after Alzheimer’s and yet there are only symptomatic treatments available to patients. We hope 2025 is a year of immense progress – not only for Gain but the field as a whole - and look forward to GT-02287 taking one step closer to potentially being the first disease-modifying therapy for Parkinson’s disease. In addition to progressing GT-02287 through the clinic, we will look to add to its extensive preclinical data package with further presentations at scientific conferences and hope to inform the industry and academia of our successful progress.
The fight against Parkinson’s disease is one we at Gain are truly committed to. I’m inspired by the dedication of our team and grateful to share this important mission with you. I look forward to keeping you updated as we progress through the year and thank you for your support along the way.
Sincerely,
Gene Mack
President and CEO
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 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 models of both GBA1-PD and idiopathic PD, demonstrating a disease-modifying effect after administration of GT-02287, suggests that GT-02287 may have the potential to slow or stop 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 is currently being evaluated for the treatment of Parkinson’s disease with or without a GBA1 mutation. Results from a Phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety and tolerability, plasma exposure in the projected therapeutic range, CNS exposure, and target engagement and modulation of GCase enzyme.
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; expectations regarding the timing of the commencement of a Phase 2 clinical study for GT-02287; the amount of time current cash will finance operations; 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, 2023 and Form 10-Q for the quarter ended September 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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