Duyuru • Jul 16

Gain Therapeutics Announces Clinical Progress and Operational Update for Rexaceract and GT-04686

Gain Therapeutics, Inc. provided an operational update regarding its lead drug candidate, rexaceract, formerly known as GT-02287, a disease modifying therapy for Parkinson’s disease. The company achieved critical inflection points related to the clinical development of rexaceract. The immediate focus remains the clinical development of rexaceract to address the unmet need in Parkinson’s disease, as the company endeavors to create a new backbone of therapy that slows or stops the progression of symptoms. The results from the Phase 1b study of rexaceract and open-label extension continue to support the hypothesis for the drug candidate and its mechanism of action, and safety and tolerability have been observed through five months of treatment. All 16 participants who entered the ongoing Phase 1b extension study remained on study at Day 150, and following the most recent review on July 6, 2026, an independent Data Monitoring Committee recommended the study continue without modification. Longer term outcomes in this dosing extension are anticipated to read out in October 2026. Data from Part 1 and Part 2 of the study includes the first 90 days administration of rexaceract, during which reduction in cerebrospinal fluid (CSF) levels of glucosylsphingosine (GluSph) in patients with elevated levels at baseline correlated with an early clinical benefit. That response is largely maintained after at least five months of administration, while the entire cohort of 16 patients can be categorized as non-progressors at both three and five months regardless of baseline characteristics. The company looks forward to the next updates at 270 days and 360 days, which will occur over the summer and at the conclusion of the study in October 2026. With Investigational New Drug (IND) authorization in hand, the clinical team is working to commence a Phase 2 study in people with Parkinson’s disease, anticipated to start in the third quarter of 2026. The FDA’s decision to authorize the IND application follows positive Phase 1 results in both healthy volunteers and people with Parkinson’s disease, in which rexaceract was well-tolerated and demonstrated both biomarker and clinical evidence of activity. Following the recent acceptance of “rexaceract” as the International Nonproprietary Name (INN) for GT-02287, all references made to GT-02287 within the program will transition to this established nonproprietary name. The planned Phase 2 study of rexaceract is expected to enroll participants with early Parkinson’s disease across sites in the United States, Australia, and Europe. The Clinical Advisory Board and experienced consultants have informed key protocol decisions, including patient selection, endpoint development, and statistical planning. After 90 days of treatment with rexaceract in participants with elevated baseline levels of GluSph in CSF at baseline, GluSph decreased by an average of 81%. Elevated GluSph, a hallmark of GCase dysfunction, has been shown to increase the aggregation of a-synuclein as well as to impair mitochondrial function and other intracellular processes in neurons. In individuals with high levels of CSF GluSph at baseline, levels of DOPA decarboxylase (DDC) decreased following 90 days of treatment with rexaceract. After 150 days of treatment, participants with elevated baseline GluSph demonstrated greater clinical benefit than those with low baseline levels, with a difference of 4.8 points in the sum of MDS-UPDRS Part II and III scores. Stabilization or improvement of MDS-UPDRS scores has been observed across the overall study population. Gain Therapeutics has also been advancing a second product candidate, GT-04686, and recently presented preclinical evidence of its activity in both in vitro and in vivo models. Results showed an increase in GCase activity and lipid substrate depletion in patient fibroblasts harboring both mutated and wildtype GBA1, as well as restoration of motor and non-motor function in an animal model of Parkinson’s disease. GT-04686 is a structurally distinct allosteric GCase chaperone and will be the next program in the pipeline advancing towards clinical development. Current activities are focused on optimizing the program and generating the data necessary to support IND-enabling studies, including refinement of the target product profile, preclinical pharmacology, manufacturing readiness, and development planning. These efforts are intended to position GT-04686 for a successful transition into formal IND-enabling development and, ultimately, clinical evaluation. Rexaceract is an orally administered, brain-penetrant small molecule that restores the function of the lysosomal enzyme GCase which becomes misfolded and impaired due to mutations in the GBA1 gene or other age-related stress factors. In preclinical models of Parkinson’s disease, rexaceract restored GCase enzymatic function, reduced endoplasmic reticulum stress, lysosomal and mitochondrial pathology, aggregated a-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, rexaceract 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 rexaceract, suggest that rexaceract may have the potential to slow or stop the progression of Parkinson’s disease. Results from a Phase 1 study of rexaceract in healthy volunteers demonstrated favorable safety and tolerability, plasma and CNS exposures in the projected therapeutic range, and target engagement with an increase in GCase activity among those receiving rexaceract at clinically relevant doses. Rexaceract 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 enrolled participants across seven sites in Australia, is to evaluate the safety and tolerability of rexaceract after three months of dosing in people with Parkinson’s disease. The Phase 1b study extension allows participants to continue to be treated with rexaceract for up to a total of 12 months. 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.

Duyuru • Mar 19

Gain Therapeutics Inc Presents Additional Clinical and Biomarker Data from Phase 1B Clinical Study of GT-02287 and Preclinical Data from Novel Chemical Series At AD/PD 2026

Gain Therapeutics, Inc. presented an oral presentation and poster at AD/PD 2026 International Conference on Alzheimer’s and Parkinson’s Disease and Related Neurological Disorders, being held March 17-21, 2026, in Copenhagen, Denmark. The oral presentation outlined new clinical and biomarker data from the Phase 1b clinical study of GT-02287 further supporting disease-modifying potential in both idiopathic and GBA1 Parkinson’s disease. The Company also presented a poster outlining preclinical data from a structurally distinct chemical series of allosteric glucocerebrosidase (GCase) modulators, which are ready for IND-enabling studies for the treatment of Parkinson’s disease (PD) and other neurological disorders. The oral presentation, entitled, “An Open-Label Phase 1b Study of GT-02287 in Parkinson’s Disease,” was delivered on-site by the Company’s Chief Medical Officer, Jonas Hannestad, M.D., Ph.D. Data on safety, tolerability, biomarkers, and clinical scores from the completed Part 1 of the study support continued development of GT-02287 for PD. Of the 19 participants who completed dosing in Part 1, 16 chose to continue in the ongoing nine-month extension (Part 2), further supporting the tolerability of GT-02287. A Data Monitoring Committee meeting on March 5, 2026, concluded that the study should continue without any changes. As previously presented in January 2026, in all participants with elevated baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), GluSph decreased substantially after 90 days of treatment with GT-02287. Elevated GluSph, a result of GCase dysfunction, has been shown to increase the aggregation of alpha synuclein and to impair mitochondrial and lysosomal function in neurons. As of March 10, 2026, of the 16 participants enrolled in the ongoing nine-month extension, 14 had completed dosing for five months (Day 150). MDS-UPDRS scores remained stable over 150 days of dosing. Preliminary data (subject to quality assurance) suggests that participants with elevated baseline levels of GluSph in CSF continued to benefit more than those with low baseline levels of GluSph in CSF, with a difference of 6.7 points in the sum of MDS-UPDRS Part II and Part III scores between the two groups at Day 150. Additionally, in individuals with high levels of CSF GluSph at baseline, levels of DOPA decarboxylase (DDC) decreased following 90 days of treatment with GT-02287. DDC is responsible for converting levodopa into dopamine in the brain and peripheral nervous system and is elevated in the CSF of people with Parkinson’s – likely due to dopaminergic neuron dysfunction. The poster, titled, “Novel Allosteric GCase Modulators, Different From the Clinical Stage GT-02287, for the Treatment of Parkinson’s Disease,” was presented on-site by the Company’s Head of Research Ana Maria Garcia-Collazo, Ph.D. The new, orally available, brain penetrant chemical series – led by GT-04686 – demonstrates potential for future clinical development, as evidenced by restoration of key biological activities that are impaired in Parkinson’s disease. These findings together support advancement of this novel chemical series towards the clinic and the potential to be developed for PD and other indications where GCase deficiency is implicated. 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 a-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 data in these models, demonstrating a disease-modifying effect of GT-02287, suggest that the drug candidate 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 an increase in GCase activity among those receiving GT-02287 at clinically relevant doses. 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 enrolled participants across seven sites in Australia, is to evaluate the safety and tolerability of GT-02287 after three months of dosing in people with Parkinson’s disease. The recently commenced Phase 1b study extension allows participants to continue to be treated with GT-02287 for up to a total of 12 months. Initial results from the Phase 1b clinical trial in people with Parkinson’s disease demonstrated central nervous system target engagement, a reduction to baseline levels in the prespecified endpoint glucosylsphingosine (GluSph), and improvement or stabilization in MDS-UPDRS scores. 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.

Biotechs Sektör Büyümesi	17.04%
Gelir büyüme oranı	-59.91%
Özkaynak getirisi	-178.06%
Net Marj	n/a
Son Kazanç Güncellemesi	30 Jun 2026

Tarih	Kazançlar	G+A Giderleri	Ar-Ge Giderleri
30 Jun 26	-20	9	0
31 Mar 26	-21	9	0
31 Dec 25	-20	9	0
30 Sep 25	-19	9	0
30 Jun 25	-19	9	0
31 Mar 25	-21	10	0
31 Dec 24	-20	10	0
30 Sep 24	-21	10	0
30 Jun 24	-22	10	-6
31 Mar 24	-21	10	0
31 Dec 23	-22	11	0
30 Sep 23	-22	11	0
30 Jun 23	-22	12	6
31 Mar 23	-19	10	0
31 Dec 22	-18	10	0
30 Sep 22	-16	9	0
30 Jun 22	-16	8	0
31 Mar 22	-15	8	0
31 Dec 21	-14	7	0
30 Sep 21	-12	6	0
30 Jun 21	-8	4	0
31 Mar 21	-5	2	0
31 Dec 20	-4	1	0
30 Sep 20	-3	1	0
31 Dec 19	-2	1	2

Veri	Son Güncelleme (UTC saati)
Şirket Analizi	2026/09/17 13:21
Gün Sonu Hisse Fiyatı	2026/09/16 00:00
Kazançlar	2026/06/30
Yıllık Kazançlar	2025/12/31

Paket	Veri	Zaman Çerçevesi	Örnek ABD Kaynağı ^*
Şirket Finansalları	10 yıl	Gelir tablosu Nakit akış tablosu Bilanço	SEC Form 10-K SEC Form 10-Q
Analist Konsensüs Tahminleri	+3 yıl	Finansal tahminler Analist fiyat hedefleri	Analist Araştırma Raporları Blue Matrix
Piyasa Fiyatları	30 yıl	Hisse senedi fiyatları Temettüler, Bölünmeler ve Eylemler	ICE Piyasa Verileri SEC Form S-1
Sahiplik	10 yıl	En büyük hissedarlar İçeriden öğrenenlerin ticareti	SEC Form 4 SEC Form 13D
Yönetim	10 yıl	Liderlik ekibi Yönetim Kurulu	SEC Form 10-K SEC Form DEF 14A
Önemli Gelişmeler	10 yıl	Şirket duyuruları	SEC Form 8-K

Analist	Kurum
Mayank Mamtani	B. Riley Securities, Inc.
Thomas Shrader	BTIG
Raghuram Selvaraju	H.C. Wainwright & Co.

Gain Therapeutics Geçmiş Kazanç Performansı

Geçmiş kriter kontrolleri 0/6

Anahtar bilgiler

Yakın geçmiş performans güncellemeleri

Second quarter 2023 earnings released: US$0.62 loss per share (vs US$0.43 loss in 2Q 2022)

Recent updates

Gain Therapeutics Announces Clinical Progress and Operational Update for Rexaceract and GT-04686

Gain Therapeutics Receives FDA Clearance of Ind to Advance GT-02287 into Phase 2 Clinical Development for Parkinson’s Disease

Gain Therapeutics Cleared to Begin Phase 2 Trial of GT-02287 for Parkinson’s Disease

Gain Therapeutics expands ‘compelling’ evidence base for Parkinson’s candidate

Gain Therapeutics Announces Interim Clinical and Biomarker Data from Phase 1B Study of GT-02287 in Parkinson's Disease

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Gain Therapeutics, Inc., Annual General Meeting, Jun 24, 2026

The Market Is Sleeping on This Parkinson's Biotech - And I Think That's a Mistake

Gain Therapeutics Inc Presents Additional Clinical and Biomarker Data from Phase 1B Clinical Study of GT-02287 and Preclinical Data from Novel Chemical Series At AD/PD 2026

Gain Therapeutics, Inc. Announces Presentations and Provides U.S. Fda Regulatory Update for Gt-02287

Gain Therapeutics Highlights Biomarker Evidence Supporting Disease-Modifying Potential of GT-02287

Gain Therapeutics, Inc. Announces Positive Results in Key Exploratory Endpoint from its Phase 1b Clinical Study of GT-02287 in People with Parkinson's Disease

Gain Therapeutics, Inc. has filed a Follow-on Equity Offering in the amount of $35.530981 million.

Gain Therapeutics Presents Preclinical Gt-02287 Data At Neuroscience 2025

Gain Therapeutics: Valuation Remains Low In Light Of Recently Reported Functional Improvement In PD Patients

Gain Therapeutics, Inc. Announces Resignation of Eric Richman from the Board of Directors, Effective October 6, 2025

Gain Therapeutics, Inc. Presents Initial Data from Phase 1b Clinical Study of GT-02287 in Parkinson's Patients At International Congress of Parkinson's Disease and Movement Disorders

Gain Therapeutics, Inc. Begins Its GT-02287 Phase 1b Extension Study in People with Parkinson's Disease

Gain Therapeutics, Inc. Receives Australian Approval of Phase 1B Dosing Extension and Reports Positive Independent Data Monitoring Committee Recommendation

Gain Therapeutics, Inc. has completed a Follow-on Equity Offering in the amount of $7.00005 million.

Gain Therapeutics, Inc. has filed a Follow-on Equity Offering.

Gain Therapeutics, Inc. Announces Completion of Target Enrollment in Phase 1B Clinical Study Evaluating GT-02287 in People with Parkinson's Disease With or Without GBA1 Mutation

Gain Therapeutics, Inc., Annual General Meeting, Jun 24, 2025

Gain Therapeutics Presents Additional Preclinical Data and Design of Phase 1b Clinical Study of GT-02287 at AD/PD 2025 and Provides Enrollment Update

Gain Therapeutics, Inc. Doses First Participant in Phase 1b Clinical Trial of GT-02287 in Parkinson's Disease

Gain Therapeutics, Inc. Provides Operational Update

Gain Therapeutics, Inc. Announces Formation of Clinical Advisory Board

Gain Therapeutics, Inc. Announces Board and Executive Changes

Gain Therapeutics: Parkinson's Drug Candidate GT-02287 Keeps Racking Up Good Results

Gain Therapeutics, Inc. Initiates Phase 1b Clinical Trial of Lead Candidate GT-02287 in People with GBA1 and Idiopathic Parkinson’s Disease

Gain Therapeutics, Inc. Announces Presentation of Preclinical Data for the Product Candidate, GT-02287

Gain Therapeutics Presents Phase 1 GT-02287 Data at International Congress of Parkinson’s Disease and Movement Disorders Demonstrating Increase in GCase Activity in Healthy Volunteers

Gain Therapeutics, Inc. Announces Positive Topline Results from the Phase 1 Clinical Trial of GT-02287

Gain Therapeutics, Inc. Receives Non-Compliance Letter from Nasdaq Regarding MVLS Deficiency

Gain Therapeutics, Inc. Announces the Completion of Dosing in the Multiple Ascending Dose (MAD) of the Phase 1 Study of GT-02287, a Novel Small Molecule Therapy for Parkinson’s Disease

Gain Therapeutics, Inc. Announces CEO Changes

Gain Therapeutics, Inc. has completed a Follow-on Equity Offering in the amount of $10.999898 million.

Gain Therapeutics: Phase 1 Readout Awaits For Parkinson's Drug With Great Potential

Consensus EPS estimates upgraded to US$0.97 loss, revenue downgraded

Gain Therapeutics, Inc., Annual General Meeting, Jun 24, 2024

Consensus revenue estimates fall by 40%

Gain Therapeutics, Inc. Announces Positive Results from the Single Ascending Dose (SAD) Part of the Phase 1 Clinical Trial of GT-02287

Gain Therapeutics, Inc. Appoints Gene Mack as Chief Financial Officer

Independent Director recently bought US$116k worth of stock

Gain Therapeutics, Inc. Appoints Jonas Hannestad, M.D., Ph.D. as Chief Medical Officer

New major risk - Financial position

Gain Therapeutics, Inc. Announces Xavi Barril Leaves as Chief Technology Officer

Gain Therapeutics: The Case For GCase In Parkinson's And Buyout Potential

Consensus revenue estimates decrease by 11%, EPS upgraded

Gain Therapeutics, Inc. Announces Preclinical Data Showing Restoration of Enzymatic Function with Novel Allosteric Regulators in GM1 Gangliosidosis Model

Gain Therapeutics, Inc. has completed a Follow-on Equity Offering in the amount of $6.65573 million.

Gain Therapeutics, Inc. announced that it expects to receive $5.000176 million in funding

Gain Therapeutics Announces Dosing of First Two Subjects in Phase 1 Clinical Trial of GT-02287, a Novel GCase-targeting Small Molecule Therapy for GBA1 Parkinson’s Disease

Gain Therapeutics Presents New Preclinical Data Demonstrating A Reduction of Plasma Neurodegeneration Biomarker NfL After Administration of Its Drug Candidate GT-02287 in GBA1 Parkinson's Disease Model

Consensus revenue estimates fall by 16%

New major risk - Revenue and earnings growth

Second quarter 2023 earnings released: US$0.62 loss per share (vs US$0.43 loss in 2Q 2022)

Consensus revenue estimates increase by 193%, EPS downgraded

Consensus revenue estimates increase by 17%

Gain Therapeutics, Inc. Announces Executive Changes

Consensus revenue estimates increase by 24%

High number of new and inexperienced directors

Gain Therapeutics, Inc. Presents New Preclinical Data Showing Promising Pharmacological Activity in Parkinson's Disease Patient-Derived Dopaminergic Neurons

Gain Therapeutics Announces Chief Executive Officer Changes

Gain Therapeutics appoints CEO

Gain Therapeutics, Inc. Presents New Preclinical Data Demonstrating Potential Disease Modifying Benefits of Its Novel Brain Penetrant Small Molecule Candidate GT-02287 in Two Preclinical Models of Parkinson's Disease

Price target decreased to US$19.25

Consensus forecasts updated

Consensus forecasts updated

Gain Therapeutics, Inc., Annual General Meeting, Jun 16, 2022

Price target decreased to US$22.00

High number of new and inexperienced directors

Price target decreased to US$22.00