Core Viewpoint - Gain Therapeutics, Inc. is actively participating in key healthcare conferences to showcase its advancements in allosteric small molecule therapies, particularly focusing on its lead drug candidate GT-02287 for Parkinson's disease [1][2]. Conference Participation - Gain Therapeutics will attend the 44th Annual J.P. Morgan Healthcare Conference and other related events, including the Sachs Neuroscience Innovation Forum on January 11, 2026, and the Demy-Colton/Informa Biotech Showcase from January 12-14, 2026 [1][3]. - The company will conduct presentations and one-on-one meetings with management during these events, providing opportunities for investors and stakeholders to engage directly [3][4]. Drug Development - GT-02287 is in clinical development for treating Parkinson's disease, targeting both patients with and without a GBA1 mutation [5][8]. - The drug functions as an allosteric enzyme modulator, aiming to restore the function of the glucocerebrosidase enzyme, which is often impaired due to genetic mutations or age-related stress [5]. - In preclinical models, GT-02287 has shown promising results, including restoration of enzymatic function and reduction of neurodegeneration biomarkers [5][6]. Clinical Trials - GT-02287 is currently undergoing a Phase 1b clinical trial across seven sites in Australia, focusing on safety and tolerability over a three-month period [6]. - An extension of the Phase 1b study allows participants to continue treatment for up to 12 months, indicating a commitment to long-term evaluation of the drug's effects [6]. Funding and Support - The development of GT-02287 has received funding from notable organizations, including The Michael J. Fox Foundation and the Eurostars-2 program, highlighting the drug's potential and the support it has garnered [7]. Company Overview - Gain Therapeutics is a clinical-stage biotechnology company specializing in the discovery and development of next-generation allosteric therapies, with a focus on neurodegenerative diseases and other challenging disorders [8][10]. - The company employs its proprietary Magellan™ platform to accelerate drug discovery and develop novel treatments for various conditions, including lysosomal storage disorders and solid tumors [10].

Summary of Gain Therapeutics Conference Call Company Overview - **Company**: Gain Therapeutics (NasdaqGM:GANX) - **Focus**: Development of GT02287 for Parkinson's disease treatment Key Industry Insights - **Parkinson's Disease Prevalence**: Second most prevalent neurodegenerative disease in the U.S. with approximately 1 million patients, 15% of whom carry a GBA1 mutation [8][2] - **GCase Activity**: The enzyme glucocerebrosidase (GCase) is crucial in the pathophysiology of Parkinson's, with mutations leading to reduced enzyme activity and associated disease progression [12][18] Core Findings from the Conference Call - **Phase 1b Study Results**: - Patients showed an average improvement of 2.2 points in MDS-UPDRS scores after 90 days of treatment with GT02287 [2][10] - The improvement aligns with expectations based on the drug's mechanism of action [3] - A clinically meaningful improvement is considered to be approximately six points over a year compared to placebo [3] - **Biomarker Evidence**: - GT02287 modulates GCase activity, leading to a reduction in glucosylsphingosine, a toxic substrate implicated in Parkinson's [4][5] - Stabilization of MDS-UPDRS scores observed without changes in dopamine administration suggests a potential disease-modifying effect [5][42] - **Study Design**: - The phase 1b study included a 90-day treatment followed by an optional open-label extension [8][26] - 21 patients enrolled, with 19 completing the first part of the study [26] - **Future Expectations**: - Continued analysis of phase 1b study extension and safety [42] - Final results expected in the second half of 2026, with plans to initiate a phase II study [43][44] Important Considerations - **Glucosylsphingosine as a Biomarker**: - Emerging evidence suggests glucosylsphingosine is a better biomarker for treatment response than glucosylceramide [35][36] - Measurement in cerebrospinal fluid (CSF) is critical for assessing central nervous system effects [36] - **Regulatory Pathway**: - Discussions with the FDA regarding the use of glucosylsphingosine as a surrogate biomarker are ongoing, but traditional UPDRS-based studies may still be required for approval [55][56] - **Patient Population**: - Future studies may focus on patients with specific glucosylsphingosine levels or GBA1 mutations, as they may respond better to treatment [46][49] Additional Insights - **Mechanism of Action**: - The drug aims to increase GCase activity, potentially leading to reduced glucosylsphingosine levels, which could have long-term benefits for Parkinson's patients [34][38] - **Clinical Implications**: - The reduction of glucosylsphingosine in CSF correlates with improvements in UPDRS scores, indicating a possible link between biomarker levels and clinical outcomes [41][45] This summary encapsulates the key points discussed during the conference call, highlighting the company's focus on GT02287, the significance of biomarker evidence, and the implications for future clinical development and regulatory interactions.

Core Insights - Gain Therapeutics, Inc. has provided additional biomarker and clinical data from the Phase 1b clinical study of GT-02287, indicating its potential as a disease-modifying therapy for Parkinson's disease [1] Group 1: Clinical Data and Results - In participants with elevated baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), GluSph decreased by an average of 81% after 90 days of treatment with GT-02287, suggesting increased GCase activity in the brain [2] - Out of 19 patients who completed Part 1 of the study, 15 patients showed an average improvement of 2.20 points in the sum of MDS-UPDRS Part II and Part III scores after treatment [3] - The study observed stabilization in MDS-UPDRS scores and anecdotal improvements in specific functional areas such as balance, gait, and sense of smell after 90 days of dosing with GT-02287 [4] Group 2: Drug Candidate Information - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric enzyme modulator, restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) [6] - Preclinical models have shown that GT-02287 can restore GCase enzymatic function and reduce various pathologies associated with Parkinson's disease [6][7] - The drug is currently being evaluated in a Phase 1b clinical trial, with the primary endpoint focused on safety and tolerability after three months of dosing [9] Group 3: Future Developments and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation for Parkinson's Research and other organizations to advance the development of GT-02287 [10] - The company is hosting a virtual event to discuss the results of the Phase 1b clinical study and engage with key opinion leaders in the field [5]

Core Insights - Premarket trading is showing notable activity with significant price movements indicating potential investment opportunities before the market opens [1] Premarket Gainers - WhiteFiber, Inc. (WYFI) is up 21% at $17.35 [3] - Rising Dragon Acquisition Corp. (RDAC) is up 21% at $12.63 [3] - Julong Holding Limited (JLHL) is up 13% at $3.45 [3] - Linkhome Holdings Inc. (LHAI) is up 11% at $12.50 [3] - A SPAC III Acquisition Corp. (ASPC) is up 9% at $13.09 [3] - Intuitive Machines, Inc. (LUNR) is up 7% at $11.61 [3] - Ascent Solar Technologies, Inc. (ASTI) is up 7% at $3.31 [3] - Bitmine Immersion Technologies, Inc. (BMNR) is up 6% at $30.39 [3] - Velo3D, Inc. (VELO) is up 5% at $12.10 [3] - Gain Therapeutics, Inc. (GANX) is up 4% at $2.36 [3] Premarket Losers - cbdMD, Inc. (YCBD) is down 17% at $1.60 [4] - Mega Fortune Company Limited (MGRT) is down 16% at $7.33 [4] - Cassava Sciences, Inc. (SAVA) is down 14% at $2.43 [4] - NIKE, Inc. (NKE) is down 10% at $59.01 [4] - InMed Pharmaceuticals Inc. (INM) is down 6% at $1.21 [4] - Datacentrex, Inc. (DTCX) is down 5% at $3.23 [4] - Abundia Global Impact Group Inc (AGIG) is down 5% at $1.50 [4] - PSQ Holdings, Inc. (PSQH) is down 5% at $1.00 [4] - Paranovus Entertainment Technology Ltd. (PAVS) is down 4% at $2.34 [4] - Worksport Ltd. (WKSP) is down 4% at $2.09 [4]

Core Viewpoint - Gain Therapeutics, Inc. is advancing its lead drug candidate GT-02287 for the treatment of Parkinson's disease, with a focus on its disease-modifying potential and biomarker data from a Phase 1b clinical study [1][2]. Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company specializing in the discovery and development of next-generation allosteric therapies [10]. - The company is currently evaluating GT-02287 in a Phase 1b clinical trial for Parkinson's disease, targeting patients with or without a GBA1 mutation [10]. Drug Candidate Details - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric enzyme modulator, restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) [5]. - Preclinical studies have shown that GT-02287 can restore GCase enzymatic function and reduce various pathologies associated with Parkinson's disease, including neuroinflammation and neuronal death [5][6]. - The drug has demonstrated a disease-modifying effect in preclinical models, suggesting potential to slow or stop the progression of Parkinson's disease [6]. Clinical Study Insights - The Phase 1 study of GT-02287 in healthy volunteers indicated favorable safety and tolerability, with target engagement reflected in increased GCase activity [7]. - The ongoing Phase 1b clinical trial has enrolled participants across seven sites in Australia, focusing on the safety and tolerability of GT-02287 after three months of dosing [8]. - An extension of the Phase 1b study allows participants to continue treatment for up to 12 months [8]. Funding and Support - Gain Therapeutics has received funding support for its lead program from The Michael J. Fox Foundation for Parkinson's Research and The Silverstein Foundation for Parkinson's with GBA, among others [9].

Core Insights - Gain Therapeutics, Inc. has reported a significant reduction in glucosylsphingosine (GluSph) levels in cerebrospinal fluid (CSF) of Parkinson's disease (PD) patients after treatment with their drug candidate GT-02287, indicating increased GCase activity in the brain, which may influence PD progression [1][2][3] Group 1: Study Results - The Phase 1b study of GT-02287 showed that all participants with elevated GluSph levels experienced substantial decreases towards levels seen in healthy individuals after 90 days of treatment [2] - The study enrolled 21 participants, with 19 completing the 90-day dosing period, and 15 (79%) opting to continue into a nine-month extension [4] - The data monitoring committee has recommended the continuation of the Phase 1b study with no changes, confirming the treatment's tolerability [5] Group 2: Future Plans and Events - A KOL event is scheduled for January 6, 2026, to discuss the results of the study and emerging biomarker data [6] - The company plans to present longer follow-up data at the AD/PD™ conference in March 2026, focusing on the effects of GT-02287 on MDS-UPDRS scores [3] Group 3: Drug Candidate Overview - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric enzyme modulator, aimed at restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) [7][8] - Preclinical studies have shown that GT-02287 can restore GCase function, reduce ER stress, and improve motor function in models of both GBA1-PD and idiopathic PD [9][10] Group 4: Funding and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation for Parkinson's Research, The Silverstein Foundation, and the Eurostars-2 joint program [12]

Core Insights - Artificial intelligence (AI) is identified as the greatest investment opportunity of the current era, with a strong emphasis on the urgency to invest now [1][13] - The energy demands of AI technologies are highlighted, with data centers consuming as much energy as small cities, leading to concerns about power grid strain and rising electricity prices [2][3] Investment Opportunity - A specific company is presented as a key player in the AI energy sector, owning critical energy infrastructure assets that are essential for meeting the increasing energy demands of AI [3][7] - This company is characterized as a "toll booth" operator in the AI energy boom, benefiting from the surge in demand for electricity driven by AI advancements [4][5] Market Position - The company is noted for its unique position in the market, being debt-free and holding a significant cash reserve, which is nearly one-third of its market capitalization [8] - It also has a substantial equity stake in another AI-related company, providing investors with indirect exposure to multiple growth engines in the AI sector [9][10] Future Trends - The article discusses the broader trends of onshoring and U.S. LNG exports, positioning the company as a beneficiary of these developments under the current political climate [6][14] - The influx of talent into the AI sector is expected to drive continuous innovation and advancements, reinforcing the importance of investing in AI-related companies [12] Conclusion - The narrative concludes with a strong call to action for investors to engage in the AI market, emphasizing the potential for significant returns within a 12 to 24-month timeframe [15][19]

Core Insights - Gain Therapeutics, Inc. announced promising preclinical data for its lead drug candidate GT-02287, which shows potential as a disease-modifying therapy for Parkinson's disease (PD) by enhancing mitochondrial and lysosomal health [1][2][7] Preclinical Data - GT-02287 demonstrated a reduction in mitochondrial stress and improved neuronal survival in rat dopaminergic neurons treated with MPP+, a mitochondrial toxin [3] - In a mouse PD model, GT-02287 decreased staining for MIRO1, indicating improved mitochondrial health [4] - The drug facilitated the trafficking of glucocerebrosidase (GCase) to mitochondria, enhancing mitochondrial function in patient-derived fibroblasts with GBA1 mutations [5][6] Mechanism of Action - The drug acts throughout the disease cascade resulting from dysfunctional GCase, contributing to neuroprotection by restoring GCase function and reducing neuroinflammation and neuronal death [2][6][7] - GT-02287's effects on mitochondrial health were highlighted as a significant aspect of its mechanism, addressing previously open questions in PD research [2][4] Clinical Development - GT-02287 is currently in a Phase 1b clinical trial for PD, with early observations suggesting disease-modifying effects consistent with preclinical findings [9] - The trial aims to evaluate safety and tolerability after 3 months of dosing, with an extension allowing treatment for up to 12 months [9] Funding and Support - The development of GT-02287 has received funding from notable organizations, including The Michael J. Fox Foundation and the Eurostars-2 joint program [10]

Core Insights - Gain Therapeutics, Inc. (GANX) has been upgraded to a Zacks Rank 2 (Buy) due to an upward trend in earnings estimates, which is a significant factor influencing stock prices [1][3] - The Zacks rating system is based on the Zacks Consensus Estimate, which reflects EPS estimates from sell-side analysts for the current and following years [1][2] Earnings Outlook - The recent upgrade indicates a positive outlook for Gain Therapeutics' earnings, suggesting potential buying pressure and an increase in stock price [3][5] - Rising earnings estimates correlate strongly with stock price movements, influenced by institutional investors who adjust their valuations based on these estimates [4][5] Earnings Estimate Revisions - For the fiscal year ending December 2025, Gain Therapeutics is expected to earn -$0.65 per share, unchanged from the previous year [8] - Over the past three months, the Zacks Consensus Estimate for Gain Therapeutics has increased by 1.1%, indicating a positive trend in earnings estimates [8] Zacks Rank System - The Zacks Rank system classifies stocks into five groups based on earnings estimates, with Zacks Rank 1 (Strong Buy) stocks historically generating an average annual return of +25% since 1988 [7] - Gain Therapeutics' upgrade to Zacks Rank 2 places it in the top 20% of Zacks-covered stocks, suggesting a strong potential for market-beating returns in the near term [10]

Financial Performance - Net loss for the three months ended September 30, 2025, was $5,284,373, compared to a net loss of $4,485,692 for the same period in 2024, indicating an increase in loss of approximately 17.8%[16] - For the nine months ended September 30, 2024, the net loss was $16,642,964, compared to a net loss of $15,623,518 for the same period in 2025, indicating a decrease in losses year-over-year[31] - The company incurred net losses of $5.3 million and $15.6 million for the three and nine months ended September 30, 2025, respectively, with negative cash flows from operations of $13.8 million during the nine months[186] - The company has not generated any revenue from product sales and has incurred significant operating losses and negative cash flows from operations[212] - The company has not yet received approval for or commercialized any products or technologies, and does not expect to generate revenue from sales in the near term[212] Assets and Liabilities - Total current assets decreased from $11,602,478 as of December 31, 2024, to $10,517,268 as of September 30, 2025, a decline of approximately 9.36%[13] - Total liabilities increased from $4,784,310 as of December 31, 2024, to $5,062,250 as of September 30, 2025, representing an increase of about 5.8%[13] - Total stockholders' equity decreased from $7,339,574 as of December 31, 2024, to $5,909,430 as of September 30, 2025, a decline of approximately 19.4%[13] - Cash and cash equivalents decreased from $10,385,863 as of December 31, 2024, to $8,807,353 as of September 30, 2025, a reduction of about 15.2%[13] - As of September 30, 2025, the Company has cash and cash equivalents of $8.8 million, which is insufficient to fund estimated operating and capital expenditures for at least the next 12 months[50] Research and Development - Research and development expenses for the nine months ended September 30, 2025, were $7,865,493, down from $9,566,259 for the same period in 2024, a decrease of about 17.8%[16] - Research and development expenses for the three months ended September 30, 2025, increased to $2.8 million, a rise of $0.2 million compared to the same period in 2024, primarily due to ongoing clinical trials[203] - For the nine months ended September 30, 2025, research and development expenses decreased to $7.9 million, down $1.7 million from $9.6 million in the same period in 2024, attributed to research grant income and tax credits[204] - The company’s clinical stage product candidate, GT-02287, is being developed for the treatment of Parkinson's disease, with positive early results from a Phase 1b clinical study presented in October 2025[36] - The Phase 1b clinical study of GT-02287 showed that participants experienced stabilization and trending improvements in their MDS-UPDRS scores after 90 days of administration[36] Capital and Financing - The company has primarily funded its operations through equity financings and research grants, indicating reliance on external capital for continued operations[47] - The company reported net proceeds from the issuance of shares in ATM offerings of $615,515 for the nine months ended September 30, 2024, and $4,910,240 for the same period in 2025, showing a significant increase in capital raised[31] - The Company completed a public offering in July 2025, raising gross proceeds of $7.0 million, with net proceeds of $6.0 million after offering expenses[137] - The company raised gross proceeds of $11.5 million from a public offering in June 2024, which included $1.2 million in underwriting commissions and expenses[147] - The company plans to finance its cash needs through public and private equity offerings, debt financings, and collaborations, but may face challenges in obtaining favorable terms[187] Operational Challenges - The Company has significant doubt about its ability to continue as a going concern due to current liquidity issues and lack of expected revenues[50] - The company has not generated any product revenues and does not expect to do so in the foreseeable future[190] - The company anticipates needing to raise additional capital to fund operations beyond the first quarter of 2026 due to projected cash outflows[186] - Management is reviewing the cost structure to optimize expenditures and improve the overall cash burn rate, including research and development expenses[51] Tax and Income - Income taxes increased to $549 thousand for the three months ended September 30, 2025, compared to $11 thousand for the same period in 2024, primarily due to higher corporate taxes in Australia[92] - Income taxes were $791 thousand for the nine months ended September 30, 2025, compared to $32 thousand in the same period of 2024[211] Currency and Foreign Exchange - The company experienced a foreign exchange loss of $26,170 for the three months ended September 30, 2025, compared to a gain of $229,047 for the same period in 2024[20] - Accumulated currency translation adjustments as of September 30, 2025, amounted to $835,949, compared to $41,247 as of December 31, 2024[57] - The Company has not utilized any foreign currency hedging strategies, exposing it to foreign currency risk[57] Stock and Compensation - The total stock-based compensation expense for the nine months ended September 30, 2025, was $1.4 million, compared to $2.0 million for the same period in 2024[158] - The Company issued 1,204,325 stock options during the nine months ended September 30, 2025, with a weighted average exercise price of $2.28[154] - The weighted average grant date fair value of stock options granted in the nine months ended September 30, 2025, was $1.63[156] - As of September 30, 2025, unrecognized compensation costs associated with stock options grants were $3.5 million, expected to be recognized over 3.5 years[154] Legal and Compliance - The Company recorded an accrual of $0.53 million related to litigation against the former CEO, which was dismissed on July 2, 2025[165]