Gain Therapeutics Reports Financial Results for Second Quarter 2025 and Provides Corporate Update
Gain Therapeutics (NASDAQ:GANX) has reported significant progress in its Phase 1b study of GT-02287 for Parkinson's Disease. The company achieved full enrollment earlier than expected, with 16 patients enrolled as of June 30, 2025, and total enrollment not expected to exceed 20 participants.
Key financial updates include a successful public offering raising $7.1 million in net proceeds and reduced operational expenses, with R&D expenses decreasing to $2.8 million from $4.4 million year-over-year. The company's net loss improved to $0.19 per share from $0.42 per share in the previous year.
Important upcoming milestones include analysis of functional changes and biomarker activity expected in Q4 2025, potential extension of the dosing duration beyond 90 days, and an FDA IND submission planned by year-end 2025.
Gain Therapeutics (NASDAQ:GANX) ha annunciato progressi significativi nello studio di Fase 1b di GT-02287 per il morbo di Parkinson. L'azienda ha raggiunto l'arruolamento completo prima del previsto, con 16 pazienti iscritti al 30 giugno 2025; il numero totale di partecipanti non dovrebbe superare i 20.
Tra gli aggiornamenti finanziari chiave figura un offerta pubblica riuscita che ha raccolto $7.1 milioni di proventi netti e la riduzione delle spese operative, con le spese per R&S scese a $2.8 milioni da $4.4 milioni su base annua. La perdita netta per azione è migliorata a $0.19 da $0.42 nell'anno precedente.
I prossimi traguardi importanti includono l'analisi dei cambiamenti funzionali e dell'attività dei biomarcatori prevista nel quarto trimestre 2025, la possibile estensione della durata della somministrazione oltre i 90 giorni e la presentazione di un IND alla FDA pianificata entro la fine del 2025.
Gain Therapeutics (NASDAQ:GANX) ha informado avances significativos en su estudio de Fase 1b de GT-02287 para la enfermedad de Parkinson. La compañía logró la inscripción completa antes de lo previsto, con 16 pacientes incluidos al 30 de junio de 2025, y no se espera que el total supere los 20 participantes.
Entre las actualizaciones financieras clave figura una oferta pública exitosa que recaudó $7.1 millones en ingresos netos y una reducción de los gastos operativos, con gastos de I+D que disminuyeron a $2.8 millones desde $4.4 millones interanuales. La pérdida neta por acción mejoró a $0.19 desde $0.42 el año anterior.
Los hitos importantes próximos incluyen el análisis de cambios funcionales y la actividad de biomarcadores previsto para el cuarto trimestre de 2025, la posible extensión de la duración de la dosificación más allá de los 90 días y la presentación de un IND ante la FDA planificada para finales de 2025.
Gain Therapeutics (NASDAQ:GANX)는 파킨슨병 치료제 GT-02287의 임상 1b상에서 중요한 진전을 보고했습니다. 회사는 예상보다 일찍 전원 등록을 완료했으며, 2025년 6월 30일 기준 16명의 환자가 등록되었고 전체 등록 인원은 20명을 넘지 않을 것으로 예상됩니다.
주요 재무 업데이트로는 순수익으로 $7.1 million을 확보한 성공적인 공모와 운영비 감소가 있으며, 연구개발비는 전년 대비 $4.4 million에서 $2.8 million으로 감소했습니다. 주당 순손실은 전년의 $0.42에서 $0.19로 개선되었습니다.
향후 주요 이정표로는 기능적 변화 및 바이오마커 활성 분석이 2025년 4분기 예정되어 있으며, 90일을 넘는 투약 기간 연장 가능성과 연말까지 FDA에 IND 제출 계획이 포함됩니다.
Gain Therapeutics (NASDAQ:GANX) a annoncé des progrès significatifs dans son essai de phase 1b de GT-02287 pour la maladie de Parkinson. La société a atteint le recrutement complet plus tôt que prévu, avec 16 patients inclus au 30 juin 2025 ; le nombre total de participants ne devrait pas dépasser 20.
Parmi les actualités financières clés figurent une offre publique réussie ayant généré $7.1 millions de produit net et une diminution des dépenses d'exploitation, les dépenses de R&D passant de $4.4 millions à $2.8 millions en glissement annuel. La perte nette par action s'est améliorée, passant de $0.42 à $0.19.
Les étapes importantes à venir incluent l'analyse des changements fonctionnels et de l'activité des biomarqueurs prévue au quatrième trimestre 2025, une éventuelle prolongation de la durée d'administration au-delà de 90 jours et le dépôt d'un IND auprès de la FDA prévu d'ici la fin 2025.
Gain Therapeutics (NASDAQ:GANX) hat bedeutende Fortschritte in der Phase-1b-Studie von GT-02287 bei Parkinson gemeldet. Das Unternehmen erreichte die vollständige Rekrutierung früher als erwartet und hatte zum 30. Juni 2025 16 eingeschriebene Patienten; die Gesamtteilnehmerzahl wird voraussichtlich 20 nicht überschreiten.
Wesentliche finanzielle Neuigkeiten umfassen ein erfolgreiches öffentliches Angebot, das $7.1 Millionen Nettoerlös einbrachte, sowie reduzierte Betriebskosten; die F&E-Ausgaben sanken von $4.4 Millionen auf $2.8 Millionen im Jahresvergleich. Der Nettoverlust je Aktie verbesserte sich von $0.42 auf $0.19.
Wichtige bevorstehende Meilensteine sind die Analyse funktioneller Veränderungen und der Biomarker-Aktivität im vierten Quartal 2025, eine mögliche Verlängerung der Behandlungsdauer über 90 Tage hinaus sowie die geplante Einreichung eines IND bei der FDA bis Ende 2025.
- Earlier than anticipated enrollment completion for Phase 1b study of GT-02287
- Successful public offering raising $7.1 million in net proceeds
- Reduced R&D expenses by $1.7 million year-over-year
- Improved net loss per share from $0.42 to $0.19
- Strong patient interest indicating significant market potential
- Cash position decreased to $6.7 million from $10.4 million in December 2024
- Additional funding may be needed for Phase 2 development
- Pending regulatory approval for dosing duration extension creates uncertainty
Insights
Gain's accelerated Phase 1b enrollment for GT-02287 signals strong clinical progress, with crucial Parkinson's data expected in Q4 2025.
Gain Therapeutics has achieved a significant clinical milestone by completing target enrollment for its Phase 1b study of GT-02287 in Parkinson's disease ahead of schedule. This acceleration reflects strong patient and clinician interest, which is particularly noteworthy for a disease with substantial unmet needs. The company has enrolled 16 patients as of June 30, with enrollment potentially extending to no more than 20 participants.
The upcoming Q4 2025 data readout will be pivotal, as it will include both functional assessments using the MDS-UPDRS scale and biomarker analysis from cerebrospinal fluid. These dual endpoints are crucial for neurodegenerative disease trials, as they can demonstrate both symptomatic benefit and potential disease-modifying activity. The company's request to extend dosing beyond 90 days suggests confidence in the drug's safety profile and potential efficacy signals.
The preclinical data presented at multiple scientific conferences (AD/PD 2025, IAPRD 2025, GBA1 Meeting 2025) highlights GT-02287's dual mechanism - improving both lysosomal and mitochondrial GCase activity. This is particularly important as it suggests broader neuroprotective effects beyond just GBA1 mutation carriers, potentially addressing multiple pathological mechanisms in Parkinson's disease.
The company's IND submission planned for year-end 2025 indicates they're preparing for global expansion of their clinical program, which would accelerate recruitment for the Phase 2 study. The early completion of Phase 1b enrollment suggests there should be minimal delays in transitioning to the next development stage, assuming positive data.
Gain's $7.1M offering and reduced expenses provide runway through Phase 1b completion, but more capital needed for Phase 2.
The financial position of Gain Therapeutics has improved following their $7.1 million public offering completed in July 2025. This capital raise, combined with their expense reduction initiatives, strategically extends their cash runway beyond the completion of their Phase 1b study - a critical inflection point that could drive valuation.
The company has demonstrated effective cost management, reducing R&D expenses by $1.7 million (to $2.8 million) and G&A expenses by $1.4 million (to $2.3 million) compared to Q2 2024. These reductions reflect a disciplined approach to capital allocation, focusing resources on their lead program while optimizing pipeline costs and personnel. The decrease in R&D expenses was partially offset by increased stock-based compensation, suggesting the company is using equity incentives to retain talent while preserving cash.
With $6.7 million in cash as of June 30, 2025 (down from $10.4 million at year-end 2024), and the subsequent $7.1 million offering, Gain appears positioned to reach their Q4 2025 data readout without immediate financing pressure. However, the runway likely extends only modestly beyond this point, meaning additional capital will be required to fund the planned Phase 2 program.
The narrowed quarterly net loss of $0.19 per share (compared to $0.42 in Q2 2024) represents improved operational efficiency. The company's utilization of research grant income and R&D tax incentives demonstrates awareness of non-dilutive funding sources, which is prudent for clinical-stage biotechs. The strategic timing of their recent financing - ahead of potentially value-creating Phase 1b data - likely resulted in less dilution than waiting until after results.
Reached full enrolment of Phase 1b study evaluating GT-02287 in Parkinson’s Disease (PD) with or without GBA1 mutations during 2Q 2025, earlier than anticipated
Analysis of functional changes and biomarker activity at 90 days will be available during 4Q 2025
In July 2025 following the close of 2Q 2025, Gain completed an underwritten public offering that resulted in approximately
BETHESDA, Md., Aug. 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 reported financial results for the quarter ended June 30, 2025, and provided a corporate update. Important highlights during the quarter included earlier-than-anticipated completion of target enrollment for the company’s Phase 1b study evaluating lead candidate GT-02287 in Parkinson’s disease with or without a GBA1 mutation. As a result, Gain expects the analysis of participants’ functional changes scored according to the Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and biomarker activity from cerebrospinal fluid at 90 days to be available during 4Q 2025.
As of June 30, 2025, a total of 16 patients were enrolled in the Phase 1b study. However, enrollment has been kept open to any participants screened prior to July 31 who meet the criteria for the study. Total enrollment of the Phase 1b is not expected to exceed 20 participants. Additionally, the company recently submitted a request to extend the dosing duration of the Phase 1b study beyond the current 90 days allowable in the original protocol; an update on the status of that request is expected in the coming weeks.
“Reaching target enrollment in the Phase 1b study of GT-02287 during 2Q 2025 was an exciting achievement for Gain and we look forward to reporting the analysis of both functional changes and biomarker activity during 4Q 2025, which will go a long way in planning the design of our Phase 2 clinical trial for GT-02287, an effort that has already begun in earnest,” said Gene Mack, President and CEO of Gain. “We are further encouraged by clinicians’ request to extend the screening window for participants by another month and their support to extend the dosing duration for participants in the study who wish to continue beyond the original 90-day dosing duration. We believe extending the dosing duration will also provide additional valuable feedback we can incorporate into our Phase 2 planning.”
Mr. Mack continued, “We are thankful not only to the clinicians but to the patients who have continued to express their desire to join this study or carry on as part of a potential dosing extension. The interest we have seen throughout the entirety of this study underscores the significant unmet need for a disease-modifying therapy for Parkinson’s. We believe we are well-positioned to continue executing on our milestones, all with the goal of potentially shifting the treatment paradigm and delivering a life-changing treatment to those that need it.”
Second Quarter 2025 and Recent Corporate and Clinical Program Highlights
Clinical Program Highlights
- Presented preclinical data supporting the disease-modifying potential of GT-02287 in GBA1 and idiopathic PD models, including rescue of motor deficits, prevention of deficits in complex behaviors and rescue of biomarker changes that persisted even following withdrawal of GT-02287, at AD/PD 2025 in April 2025 in Vienna, Austria.
- Presented a poster highlighting the design of the Company’s Phase 1b study to evaluate GT-02287 in people with or without a GBA1 mutation at AD/PD 2025 in April 2025 in Vienna, Austria.
- Presented preclinical data demonstrating GT-02287’s broader neuroprotective effect on dopaminergic neurons treated with mitochondrial toxin MPP+, importantly suggesting an action on mitochondrial GCase in addition to its improvement of lysosomal GCase activity, at IAPRD 2025 in May 2025 in New York City, NY.
- Presented update on GT-02287 clinical program at the GBA1 Meeting 2025 in June 2025 in Montreal, Canada.
Corporate Updates
- Completed target enrollment in the Phase 1b study for GT-02287 in people with Parkinson’s disease (PD) regardless of GBA1 status. 16 participants have been enrolled as of June 30, 2025, with full enrollment not expected to exceed 20 participants. Analysis of functional changes and biomarker activity from the Phase 1b study is expected to be available during 4Q 2025.
- Completed an underwritten public offering that resulted in approximately
$7.1 million of net proceeds, extending the current cash runway beyond the completion of the Phase 1b study.
Upcoming Anticipated Milestones
- Analysis of functional changes measured by the Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and biomarker activity in cerebrospinal fluid and blood samples from participants who completed 90 days of the Phase 1b study evaluating GT-02287 in people with Parkinson’s disease expected 4Q25.
- Approval from healthcare authorities in Australia to extend the dosing duration for participants in the Phase 1b beyond the 90 days specified under the current protocol.
- IND submission to FDA expected by year end 2025 facilitating expansion of Phase 2 clinical development for GT-02287 to include clinical sites in the United States.
Q2 2025 Financial Results
Research and Development (R&D) expenses decreased by
General and Administrative (G&A) expenses decreased by
Net loss for the three months ended June 30, 2025, was
Cash and cash equivalent were
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 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; 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
| GAIN THERAPEUTICS, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (UNAUDITED) | ||||||||||||||||
| Three Months Ended June 30 | Six Months Ended June 30 | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | (2,758,973 | ) | $ | (4,437,631 | ) | $ | (5,015,983 | ) | $ | (6,944,537 | ) | ||||
| General and administrative | (2,330,553 | ) | (3,745,193 | ) | (4,442,919 | ) | (5,615,987 | ) | ||||||||
| Total operating expenses | (5,089,526 | ) | (8,182,824 | ) | (9,458,902 | ) | (12,560,524 | ) | ||||||||
| Loss from operations | (5,089,526 | ) | (8,182,824 | ) | (9,458,902 | ) | (12,560,524 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Interest income, net | 42,568 | 84,531 | 82,981 | 199,834 | ||||||||||||
| Foreign exchange (loss) gain, net | (620,924 | ) | (43,576 | ) | (721,510 | ) | 224,501 | |||||||||
| Loss before income tax | (5,667,882 | ) | (8,141,869 | ) | (10,097,431 | ) | (12,136,189 | ) | ||||||||
| Income tax | (141,205 | ) | (1,209 | ) | (241,714 | ) | (21,083 | ) | ||||||||
| Net loss | $ | (5,809,087 | ) | $ | (8,143,078 | ) | $ | (10,339,145 | ) | $ | (12,157,272 | ) | ||||
| Net loss per share: | ||||||||||||||||
| Net loss per share attributable to common stockholders - basic and diluted | $ | (0.19 | ) | $ | (0.42 | ) | $ | (0.35 | ) | $ | (0.65 | ) | ||||
| Weighted average common stock - basic and diluted | 30,341,523 | 19,215,582 | 29,518,045 | 18,600,683 | ||||||||||||
| GAIN THERAPEUTICS, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (UNAUDITED) | ||||||||
| June 30, | December 31, | |||||||
| 2025 | 2024 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 6,694,136 | $ | 10,385,863 | ||||
| Tax credits | 317,844 | 271,079 | ||||||
| Prepaid expenses and other current assets | 2,294,723 | 945,536 | ||||||
| Total current assets | 9,306,703 | 11,602,478 | ||||||
| Noncurrent assets: | ||||||||
| Property and equipment, net | 96,190 | 103,619 | ||||||
| Internal-use software, net | 127,857 | 134,268 | ||||||
| Operating lease right-of-use assets | 233,200 | 219,715 | ||||||
| Restricted cash | 36,278 | 31,695 | ||||||
| Long-term deposits and other noncurrent assets | 33,752 | 32,109 | ||||||
| Total noncurrent assets | 527,277 | 521,406 | ||||||
| Total assets | $ | 9,833,980 | $ | 12,123,884 | ||||
| Liabilities and stockholders' equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 2,713,013 | $ | 946,259 | ||||
| Operating lease liability - current | 136,320 | 160,913 | ||||||
| Other current liabilities | 2,198,918 | 2,441,761 | ||||||
| Deferred grant income - current | 22,685 | 252,211 | ||||||
| Loans - current | 126,108 | 110,177 | ||||||
| Total current liabilities | 5,197,044 | 3,911,321 | ||||||
| Noncurrent liabilities: | ||||||||
| Defined benefit pension plan | 522,676 | 443,623 | ||||||
| Operating lease liability - noncurrent | 92,851 | 53,598 | ||||||
| Deferred grant income - noncurrent | — | 47,441 | ||||||
| Loans - noncurrent | 325,359 | 328,327 | ||||||
| Total noncurrent liabilities | 940,886 | 872,989 | ||||||
| Total liabilities | $ | 6,137,930 | $ | 4,784,310 | ||||
| Stockholders’ equity | ||||||||
| Preferred stock, | $ | — | $ | — | ||||
| Common stock, | 3,078 | 2,713 | ||||||
| Additional paid-in capital | 94,642,942 | 88,779,318 | ||||||
| Accumulated other comprehensive loss | 584,083 | (247,549 | ) | |||||
| Accumulated deficit | (81,194,908 | ) | (60,783,717 | ) | ||||
| Loss of the period | (10,339,145 | ) | (20,411,191 | ) | ||||
| Total stockholders’ equity | 3,696,050 | 7,339,574 | ||||||
| Total liabilities and stockholders’ equity | $ | 9,833,980 | $ | 12,123,884 | ||||
FAQ
What are the key findings from Gain Therapeutics' (GANX) Phase 1b study of GT-02287?
How much money did Gain Therapeutics (GANX) raise in their recent public offering?
What is GANX's current cash position and quarterly financial performance?
What are the next major milestones for Gain Therapeutics' GT-02287 program?
How many patients are enrolled in GANX's Phase 1b Parkinson's Disease study?
