Latest news with #Gain
Yahoo
29-05-2025
- Business
- Yahoo
Gain Therapeutics Announces Oral Presentation at 2nd GBA1 Meeting
BETHESDA, Md., May 29, 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 announced an oral presentation at the GBA1 Meeting 2025, being held June 5th-7th in Montreal, Canada. Details of the oral presentation are as follows: Date: Thursday, June 5, 2025 Time: 11:20 p.m. EST Presenter: Jonas Hannestad, M.D., Ph.D., Chief Medical Officer, Gain Therapeutics About Gain Therapeutics, 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 StatementsThis 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 as a result of new information, future events or otherwise. Investors:Gain Therapeutics, Inc. Apaar Jammu Manager, Investor Relations and Public Relationsajammu@ LifeSci Advisors LLCChuck PadalaManaging Directorchuck@ Media:Russo Partners LLC Nic Johnson and Elio 846-9256Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
23-05-2025
- Yahoo
A missing bench comes to symbolize missing solutions to homelessness
John Paul Shanks sits for a photo outside the Central Inn in Central City, April 27, 2025. (Kentucky Lantern photo by Austin Anthony) This is the final story in a Lantern series about homelessness in Western Kentucky. Read the earlier articles here. CENTRAL CITY — Sitting on his bed at the Central Inn on a bitterly cold January day, John Paul Shanks had already handwashed his clothes, after pre-soaking them in Gain detergent, and hung them to dry. Living outdoors in this Western Kentucky town has given him a lot of experience in making do. 'I'm probably one of the only people you'll see that can just sit there and lay on a piece of concrete with a pillow or nothing and go to bed,' Shanks said. 'That hardens you up.' Gwen Clements is why 41-year-old Shanks, his red beard long and his head shaved, had a motel room that day. Clements also knows about making do. She's a leader in a loose coalition of the compassionate, working to help her homeless neighbors in a place that offers them few formal resources. She met Shanks years before when she took a job at the Perdue Farms poultry processing plant in Ohio County around the time of the Great Recession. He was a production line leader. It's unclear to Clements what put Shanks on the path to what she describes as being 'chronically homeless.' But as she began seeing him walk the streets she started checking in with him and asking if he needed anything. On days when she wanted to find Shanks, she would make sure to get up early to drive around town and check a few of his haunts. Outside the Central Inn. Inside the local Wendy's. On a bench next to a local bank where people driving by gave him money, food and sometimes clothing. 'The only people that know him are the people who stop and talk to him, people that know him from the past,' Clements said in January. With deadly cold in the forecast that January week, Clements, through a Facebook group she started in early 2024 focused on homelessness, had urged her neighbors to send her money so that she could put people up in the motel and keep them safe overnight. Finding Shanks during severe weather and making sure he had shelter had become a priority for her. It was easy for Clements to check Shanks into a motel room for the night. Finding help for his deeper issues is not. Clements said that's true of other people she helps, some of them grappling with what seem to be untreated mental illness and addiction and living without permanent shelter. 'People like John Paul, there's no help for them. You can make all the appointments you want for him. He's not going to go,' Clements said. 'He doesn't have transportation if he did decide to go.' Shanks said he injured his back years ago when on the drive to work the vehicle he was in hit a patch of black ice that 'flipped the car.' The nerve pain was so intense, he said, it could take him 30 minutes to dress. In the motel room, he also described grappling with addiction and using prescription opioids, cocaine and methamphetamine. According to court records, Shanks has been arrested a number of times. Once he was screaming and throwing rocks from a train track. Shanks told police he hadn't realized one of the rocks had almost hit a woman. Another time he was arrested for disorderly conduct for allegedly yelling obscenities at a local IGA grocery store. In 2022, a Central City police officer and Chief Jason Lindsey found Shanks at a strip mall where Shanks had previously trespassed, according to an incident report. Shanks had allegedly told a minor 'he would take him out back and beat his brains out.' Shanks told law enforcement the minor 'had said things to him about him being homeless and getting a job.' Shanks was arrested and banned from entering the strip mall property. Tammy Piper, the director of business development for the city, told the Lantern last year the city had tried to help Shanks multiple times by putting him in a hotel room or offering work. Piper said in one instance, Chief Lindsey drove Shanks to live with family members several counties over and had secured a job for Shanks, only for Shanks to return to the Central City streets. In the fall of 2024, the city removed the bench next to a local bank where Shanks often sat, sometimes dozing or asking passing drivers for money. The move sparked debate on social media and made television news in Evansville, Indiana. Central City Mayor Tony Armour told the Evansville station the bench was removed because Shanks made people uncomfortable. The mayor also said the city has tried to offer Shanks work. Shanks, in the motel room in January, disputed that the city had offered him a job. The bench took on larger significance for some, including Clements, who saw its removal as a symbol of apathy and, at times, disregard by local officials and police for people who are unsheltered and struggling. 'That was just a small part of how our homeless are treated in this county and this city,' said Clements. Clements said Shanks and other people dealing with homelessness need more than a bench where they can spend their days or even a roof over their heads. She sees a need for mobile mental health services that can meet people where they're living outside, considering that homelessness can deteriorate mental health. 'He's suffered a lot of trauma from being unhoused. I don't think people understand that,' Clements said about Shanks. 'They just want to think that, 'He's lazy and a druggie, and he needs to get a job, get off drugs and he'll be fine.' It's much more than that. 'The warming shelters and stuff is the 'more.' John Paul needing mobile crisis mental health — that's part of the 'more.' It's just so much more than the bench being removed,' Clements said. When Shanks was asked in the motel room if he believed others in Central City cared about people experiencing homelessness, he said: 'I think they worry about others. I think there's just a lot to worry about.' Clements replied to Shanks: 'The problem is too big, and they don't know how to handle it.' Paramount among the needs is more housing and temporary shelter, according to Clements and others in the band of helpers pushing to address homelessness in Muhlenberg County. The Muhlenberg County Economic Growth Alliance, the economic development arm for county government, retained an Ohio-based housing research firm in November 2023 to better understand the local housing market. The study found a need for more than 300 additional rental units and more than 700 additional owner-occupied homes through 2029. The report noted the need for affordable rental units would continue because of persistent poverty in the county. But the path for creating more housing or even temporary shelter remains unclear. Kelsey Rolley, who has helped the loose coalition at times through her work at Pennyrile Allied Community Services, said some of the divisions among the community spring from fear of the unknown. She imagines questions from local 'higher ups,' such as who else might come into the county to seek shelter if more were available and whether it might attract more crime. When Armour, the mayor, raised concerns about a church's plans to turn the Central Inn into efficiency apartments to help homeless people transition into something more permanent, he worried his community could be 'destroyed' by an influx of people drawn by the assistance. 'It's going to take a village, and until that village can be formed, created and run properly, all of us work together — I feel like it's just going to keep us stuck,' Rolley said. The loose coalition is persisting, though. Clements and others recently visited Somerset to see how a nonprofit shelter and resource hub were started just a couple years ago, and Clements has been considering buildings to potentially start her own version of that nonprofit in Muhlenberg. The way forward to stable housing remains strewn with challenges and struggles for the people who talked about their experiences of being homeless in this series. Shanks remained on the concrete stoop of the Central Inn in May, waving at passing cars. He mentioned he needed a shower, a pair of socks and maybe another stay in a motel room. 'You gotta appreciate everything about everything,' he said. Courtney Phillips, who slept outside the Abundant Life Church for weeks, is still piecing together what she wants her life to be. The church has provided her a room to sleep in. At her nursing home job, she's working long hours and building relationships with residents who deal with mental health disorders including dementia. She wants to save money for a car — what she calls a 'baby step' toward where she wants to be. She made it to the top of a waiting list for a rapid rehousing program and hopes it will help her find an apartment soon. She's also been carrying on without her dog, Joker, who cuddled with her while she was sleeping outside. Joker died earlier this year; a wooden urn with Joker's ashes sits in her room at the church, and Joker's bed is still beside her bed. 'It's real different, but he's still with me,' Phillips said. Mallie Luken, who slept in the church parking lot before Clements helped her find housing, was anxious for weeks leading up to a hearing on her possible eviction from the apartment Clements had helped her find. After police left Luken in her wheelchair outside the Abundant Life Church on a stormy night in September, Clements came to her aid, helping her secure an apartment at the Greenville Housing Authority. But her housing situation was uncertain yet again by this month. Luken, 70, was served an eviction notice because of alleged complaints from neighbors about her behavior and inappropriate language that they said was directed at them. Clements, who admits Luken can be her own 'worst enemy,' also said the housing authority alleged Luken hadn't paid rent, something she said wasn't true. The stress of her predicament had Luken exhausted and apprehensive. 'Somehow or another I keep falling through the system,' Luken said weeks before the hearing. Earlier in May, in front of a district court judge, Luken with the help of a Kentucky Legal Aid attorney was able to come to an agreement with the housing authority: She can stay in her apartment until another apartment opens up at a housing authority in Beaver Dam, next door in Ohio County where Luken previously lived. Clements said Luken has friends near there, potentially a support system. In Muhlenberg County, Clements played a large role in Luken's support system. Their relationship has grown over the months they've been together. 'I can't imagine what she's done for other people,' Luken said in praise of Clements' generosity. Leaving Luken's apartment earlier this year, Clements told Luken she loved her. Out on the sidewalk, Clements, in a voice choked with emotion, said, 'I can't imagine my mother being in that predicament. I just can't.' Introduction Part 1: Homeless often means 'invisible,' but not to everyone in this small Kentucky town Part 2: After living outdoors for weeks, she got a place to sleep, a shower — and a job Part 3: A church called its vision for housing a 'Beacon of Hope.' The mayor had concerns.
Yahoo
23-05-2025
- Yahoo
A missing bench comes to symbolize missing solutions to homelessness
John Paul Shanks sits for a photo outside the Central Inn in Central City, April 27, 2025. (Kentucky Lantern photo by Austin Anthony) This is the final story in a Lantern series about homelessness in Western Kentucky. Read the earlier articles here. CENTRAL CITY — Sitting on his bed at the Central Inn on a bitterly cold January day, John Paul Shanks had already handwashed his clothes, after pre-soaking them in Gain detergent, and hung them to dry. Living outdoors in this Western Kentucky town has given him a lot of experience in making do. 'I'm probably one of the only people you'll see that can just sit there and lay on a piece of concrete with a pillow or nothing and go to bed,' Shanks said. 'That hardens you up.' Gwen Clements is why 41-year-old Shanks, his red beard long and his head shaved, had a motel room that day. Clements also knows about making do. She's a leader in a loose coalition of the compassionate, working to help her homeless neighbors in a place that offers them few formal resources. She met Shanks years before when she took a job at the Perdue Farms poultry processing plant in Ohio County around the time of the Great Recession. He was a production line leader. It's unclear to Clements what put Shanks on the path to what she describes as being 'chronically homeless.' But as she began seeing him walk the streets she started checking in with him and asking if he needed anything. On days when she wanted to find Shanks, she would make sure to get up early to drive around town and check a few of his haunts. Outside the Central Inn. Inside the local Wendy's. On a bench next to a local bank where people driving by gave him money, food and sometimes clothing. 'The only people that know him are the people who stop and talk to him, people that know him from the past,' Clements said in January. With deadly cold in the forecast that January week, Clements, through a Facebook group she started in early 2024 focused on homelessness, had urged her neighbors to send her money so that she could put people up in the motel and keep them safe overnight. Finding Shanks during severe weather and making sure he had shelter had become a priority for her. It was easy for Clements to check Shanks into a motel room for the night. Finding help for his deeper issues is not. Clements said that's true of other people she helps, some of them grappling with what seem to be untreated mental illness and addiction and living without permanent shelter. 'People like John Paul, there's no help for them. You can make all the appointments you want for him. He's not going to go,' Clements said. 'He doesn't have transportation if he did decide to go.' Shanks said he injured his back years ago when on the drive to work the vehicle he was in hit a patch of black ice that 'flipped the car.' The nerve pain was so intense, he said, it could take him 30 minutes to dress. In the motel room, he also described grappling with addiction and using prescription opioids, cocaine and methamphetamine. According to court records, Shanks has been arrested a number of times. Once he was screaming and throwing rocks from a train track. Shanks told police he hadn't realized one of the rocks had almost hit a woman. Another time he was arrested for disorderly conduct for allegedly yelling obscenities at a local IGA grocery store. In 2022, a Central City police officer and Chief Jason Lindsey found Shanks at a strip mall where Shanks had previously trespassed, according to an incident report. Shanks had allegedly told a minor 'he would take him out back and beat his brains out.' Shanks told law enforcement the minor 'had said things to him about him being homeless and getting a job.' Shanks was arrested and banned from entering the strip mall property. Tammy Piper, the director of business development for the city, told the Lantern last year the city had tried to help Shanks multiple times by putting him in a hotel room or offering work. Piper said in one instance, Chief Lindsey drove Shanks to live with family members several counties over and had secured a job for Shanks, only for Shanks to return to the Central City streets. In the fall of 2024, the city removed the bench next to a local bank where Shanks often sat, sometimes dozing or asking passing drivers for money. The move sparked debate on social media and made television news in Evansville, Indiana. Central City Mayor Tony Armour told the Evansville station the bench was removed because Shanks made people uncomfortable. The mayor also said the city has tried to offer Shanks work. Shanks, in the motel room in January, disputed that the city had offered him a job. The bench took on larger significance for some, including Clements, who saw its removal as a symbol of apathy and, at times, disregard by local officials and police for people who are unsheltered and struggling. 'That was just a small part of how our homeless are treated in this county and this city,' said Clements. Clements said Shanks and other people dealing with homelessness need more than a bench where they can spend their days or even a roof over their heads. She sees a need for mobile mental health services that can meet people where they're living outside, considering that homelessness can deteriorate mental health. 'He's suffered a lot of trauma from being unhoused. I don't think people understand that,' Clements said about Shanks. 'They just want to think that, 'He's lazy and a druggie, and he needs to get a job, get off drugs and he'll be fine.' It's much more than that. 'The warming shelters and stuff is the 'more.' John Paul needing mobile crisis mental health — that's part of the 'more.' It's just so much more than the bench being removed,' Clements said. When Shanks was asked in the motel room if he believed others in Central City cared about people experiencing homelessness, he said: 'I think they worry about others. I think there's just a lot to worry about.' Clements replied to Shanks: 'The problem is too big, and they don't know how to handle it.' Paramount among the needs is more housing and temporary shelter, according to Clements and others in the band of helpers pushing to address homelessness in Muhlenberg County. The Muhlenberg County Economic Growth Alliance, the economic development arm for county government, retained an Ohio-based housing research firm in November 2023 to better understand the local housing market. The study found a need for more than 300 additional rental units and more than 700 additional owner-occupied homes through 2029. The report noted the need for affordable rental units would continue because of persistent poverty in the county. But the path for creating more housing or even temporary shelter remains unclear. Kelsey Rolley, who has helped the loose coalition at times through her work at Pennyrile Allied Community Services, said some of the divisions among the community spring from fear of the unknown. She imagines questions from local 'higher ups,' such as who else might come into the county to seek shelter if more were available and whether it might attract more crime. When Armour, the mayor, raised concerns about a church's plans to turn the Central Inn into efficiency apartments to help homeless people transition into something more permanent, he worried his community could be 'destroyed' by an influx of people drawn by the assistance. 'It's going to take a village, and until that village can be formed, created and run properly, all of us work together — I feel like it's just going to keep us stuck,' Rolley said. The loose coalition is persisting, though. Clements and others recently visited Somerset to see how a nonprofit shelter and resource hub were started just a couple years ago, and Clements has been considering buildings to potentially start her own version of that nonprofit in Muhlenberg. The way forward to stable housing remains strewn with challenges and struggles for the people who talked about their experiences of being homeless in this series. Shanks remained on the concrete stoop of the Central Inn in May, waving at passing cars. He mentioned he needed a shower, a pair of socks and maybe another stay in a motel room. 'You gotta appreciate everything about everything,' he said. Courtney Phillips, who slept outside the Abundant Life Church for weeks, is still piecing together what she wants her life to be. The church has provided her a room to sleep in. At her nursing home job, she's working long hours and building relationships with residents who deal with mental health disorders including dementia. She wants to save money for a car — what she calls a 'baby step' toward where she wants to be. She made it to the top of a waiting list for a rapid rehousing program and hopes it will help her find an apartment soon. She's also been carrying on without her dog, Joker, who cuddled with her while she was sleeping outside. Joker died earlier this year; a wooden urn with Joker's ashes sits in her room at the church, and Joker's bed is still beside her bed. 'It's real different, but he's still with me,' Phillips said. Mallie Luken, who slept in the church parking lot before Clements helped her find housing, was anxious for weeks leading up to a hearing on her possible eviction from the apartment Clements had helped her find. After police left Luken in her wheelchair outside the Abundant Life Church on a stormy night in September, Clements came to her aid, helping her secure an apartment at the Greenville Housing Authority. But her housing situation was uncertain yet again by this month. Luken, 70, was served an eviction notice because of alleged complaints from neighbors about her behavior and inappropriate language that they said was directed at them. Clements, who admits Luken can be her own 'worst enemy,' also said the housing authority alleged Luken hadn't paid rent, something she said wasn't true. The stress of her predicament had Luken exhausted and apprehensive. 'Somehow or another I keep falling through the system,' Luken said weeks before the hearing. Earlier in May, in front of a district court judge, Luken with the help of a Kentucky Legal Aid attorney was able to come to an agreement with the housing authority: She can stay in her apartment until another apartment opens up at a housing authority in Beaver Dam, next door in Ohio County where Luken previously lived. Clements said Luken has friends near there, potentially a support system. In Muhlenberg County, Clements played a large role in Luken's support system. Their relationship has grown over the months they've been together. 'I can't imagine what she's done for other people,' Luken said in praise of Clements' generosity. Leaving Luken's apartment earlier this year, Clements told Luken she loved her. Out on the sidewalk, Clements, in a voice choked with emotion, said, 'I can't imagine my mother being in that predicament. I just can't.' SUPPORT: YOU MAKE OUR WORK POSSIBLE
Yahoo
12-05-2025
- Health
- Yahoo
Gain Therapeutics Presents Preclinical Data at IAPRD 2025 30th World Congress on Parkinson's Disease and Related Disorders
GT-02287 protects dopaminergic neurons against mitochondrial toxin MPP+ suggesting an action on mitochondrial GCase in addition to its improvement of lysosomal GCase activity BETHESDA, Md., May 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 announced that a guided poster presentation was made at the International Association of Parkinsonism and Related Disorders (IAPRD) 30th World Congress on Parkinson's Disease and Related Disorders, held May 7th-10th in New York City, NY. The poster outlined new evidence supporting GT-02287's ability to provide a broader neuroprotective effect and potential as a disease-modifying therapy for Parkinson's disease. 'We have continued to add to the preclinical dossier of GT-02287 and we believe these recent data further elucidate the mechanism of action of GT-02287 and the importance of glucocerebrosidase-mediated interactions in the disease pathology of Parkinson's disease. Importantly, we continue to advance our understanding of stabilization of mitochondria associated with administration of GT-02287 and its allosteric modulation of GCase,' commented Joanne Taylor, Ph.D., Senior Vice President of Research of Gain. Oral Poster Presentation Information Title: GT-02287, A Clinical-stage Allosteric GCase Modulator For The Treatment Of Parkinson's Disease, Protects Dopaminergic Neurons Against Mitochondrial Toxin MPP+ Summary: In cultured rat mesencephalic dopaminergic neurons treated with MPP+, a mitochondrial toxin that induces mitochondrial impairment by inhibiting mitochondrial complex 1, GT-02287 improved the function of both the lysosomes and mitochondria and exerted a broader neuroprotective effect. GT-02287 lowered α-synuclein aggregation, lysosomal dysfunction, and mitochondrial stress while also preventing the release of mitochondrial cytochrome C, a programmed cell death (apoptosis) signal, thereby promoting neuronal survival. A PDF of the poster presented at IAPRD 2025 30th World Congress on Parkinson's Disease and Related Disorders is available on the Science and Technology section of the Company's website at About GT-02287Gain 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 >50% increase in glucocerebrosidase (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 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, 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 StatementsThis 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; 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 as a result of new information, future events or otherwise. Investors:Gain Therapeutics, Inc. Apaar Jammu Manager, Investor Relations and Public Relationsajammu@ LifeSci Advisors LLCChuck PadalaManaging Directorchuck@ Media:Russo Partners LLC Nic Johnson and Elio 846-9256Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
12-05-2025
- Health
- Yahoo
Gain Therapeutics Presents Preclinical Data at IAPRD 2025 30th World Congress on Parkinson's Disease and Related Disorders
GT-02287 protects dopaminergic neurons against mitochondrial toxin MPP+ suggesting an action on mitochondrial GCase in addition to its improvement of lysosomal GCase activity BETHESDA, Md., May 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 announced that a guided poster presentation was made at the International Association of Parkinsonism and Related Disorders (IAPRD) 30th World Congress on Parkinson's Disease and Related Disorders, held May 7th-10th in New York City, NY. The poster outlined new evidence supporting GT-02287's ability to provide a broader neuroprotective effect and potential as a disease-modifying therapy for Parkinson's disease. 'We have continued to add to the preclinical dossier of GT-02287 and we believe these recent data further elucidate the mechanism of action of GT-02287 and the importance of glucocerebrosidase-mediated interactions in the disease pathology of Parkinson's disease. Importantly, we continue to advance our understanding of stabilization of mitochondria associated with administration of GT-02287 and its allosteric modulation of GCase,' commented Joanne Taylor, Ph.D., Senior Vice President of Research of Gain. Oral Poster Presentation Information Title: GT-02287, A Clinical-stage Allosteric GCase Modulator For The Treatment Of Parkinson's Disease, Protects Dopaminergic Neurons Against Mitochondrial Toxin MPP+ Summary: In cultured rat mesencephalic dopaminergic neurons treated with MPP+, a mitochondrial toxin that induces mitochondrial impairment by inhibiting mitochondrial complex 1, GT-02287 improved the function of both the lysosomes and mitochondria and exerted a broader neuroprotective effect. GT-02287 lowered α-synuclein aggregation, lysosomal dysfunction, and mitochondrial stress while also preventing the release of mitochondrial cytochrome C, a programmed cell death (apoptosis) signal, thereby promoting neuronal survival. A PDF of the poster presented at IAPRD 2025 30th World Congress on Parkinson's Disease and Related Disorders is available on the Science and Technology section of the Company's website at About GT-02287Gain 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 >50% increase in glucocerebrosidase (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 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, 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 StatementsThis 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; 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 as a result of new information, future events or otherwise. Investors:Gain Therapeutics, Inc. Apaar Jammu Manager, Investor Relations and Public Relationsajammu@ LifeSci Advisors LLCChuck PadalaManaging Directorchuck@ Media:Russo Partners LLC Nic Johnson and Elio 846-9256Sign in to access your portfolio
