Gain Therapeutics (NasdaqGM:GANX) FY Conference March 24, 2026 01:30 PM ET Company ParticipantsGene Mack - CEOConference Call ParticipantsBoobalan Pachaiyappan - Managing Director and Senior Biotech AnalystBoobalan PachaiyappanGood morning, everyone. My name is Boobalan Pachaiyappan. I'm Managing Director and Senior Biotech Analyst at ROTH Capital Partners. With me today is Gene Mack, who is the Chief Executive Officer of Gain Therapeutics. For those who are new to the story, Gain is focused on developing o ...

Core Insights - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation allosteric small molecule therapies [3] - The company will participate in the 38th Annual ROTH Conference from March 22-24, 2026, with a fireside chat scheduled for March 23, 2026, at 11:30 a.m. PT [1][2] Company Overview - Gain Therapeutics is leading the discovery and development of allosteric therapies, with its lead drug candidate GT-02287 currently in a Phase 1b clinical trial for Parkinson's disease [3] - The company is also exploring GT-02287's potential in treating Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease [3] - Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors [3] Technology and Approach - Gain's unique approach involves discovering novel allosteric small molecule modulators that can restore or disrupt protein function [4] - The company utilizes its advanced Magellan™ platform to accelerate drug discovery and develop disease-modifying treatments for challenging disorders, including neurodegenerative diseases and rare genetic disorders [4]

Core Insights - Gain Therapeutics, Inc. presented new clinical and biomarker data for GT-02287 at the AD/PD 2026 conference, supporting its potential as a disease-modifying treatment for Parkinson's disease [1][2][6] Group 1: Clinical Study Results - The Phase 1b study of GT-02287 showed a reduction in cerebrospinal fluid (CSF) glucosylsphingosine (GluSph) levels after 90 days of treatment, indicating a positive response in participants with elevated baseline GluSph [3][4] - MDS-UPDRS scores remained stable over 150 days of dosing, with participants having high baseline GluSph showing a 6.7-point improvement compared to those with low baseline levels [4][6] - The study's Data Monitoring Committee recommended continuing the trial without changes, reflecting the treatment's safety and tolerability [2] Group 2: Biomarker Analysis - Higher levels of DOPA decarboxylase (DDC) were observed at baseline, which decreased after 90 days of GT-02287 treatment, suggesting a positive impact on dopaminergic neuron function [5][6] - The correlation between decreased GluSph, reduced DDC levels, and improved MDS-UPDRS scores supports the drug's mechanism of action targeting the underlying biology of Parkinson's disease [6] Group 3: Future Development - Gain Therapeutics is advancing a novel chemical series of allosteric glucocerebrosidase (GCase) modulators, led by GT-04686, which is ready for IND-enabling studies for Parkinson's disease and other neurological disorders [1][7][8] - The company aims to shift the treatment paradigm from symptomatic relief to disease modification, with ongoing studies expected to yield further data [6][14]

Interim analysis of Phase 1b clinical study of GT-02287, including biomarker and clinical endpoints, will be presented in oral symposium Company remains on track to initiate Phase 2 clinical trial of GT-02287 in 3Q26 after submitting additional preclinical data requested by FDA in support of an IND BETHESDA, Md., March 12, 2026 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next genera ...

BETHESDA, Md., Feb. 19, 2026 (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 that the Company’s management team will participate in a fireside chat and one-on-one meetings at the Oppenheimer 36th Annual Healthcare Life Sciences Conference, being held Wednesday, February 25th through Thursday, February 26th. Fireside ...

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].