GBA1

Gain Therapeutics Reports Financial Results for Year End 2023 and Provides Corporate Update

Retrieved on: 
Tuesday, March 26, 2024
GAAP, Research, GBA1, Multiple, Therapy, G&A, Safety

BETHESDA, Md., March 26, 2024 (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 reports financial results for the year ended December 31, 2023 and provides a corporate update.

Key Points: 
  • BETHESDA, Md., March 26, 2024 (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 reports financial results for the year ended December 31, 2023 and provides a corporate update.
  • We believe our cash position and operational plans are strong and allow us to meet our near-term milestones,” said Matthias Alder, Gain Therapeutics’ CEO.
  • Research and development (R&D) expenses increased by $3.1 million to $11.5 million for the year ended December 31, 2023, from $8.4 million for the year ended December 31, 2022.
  • Net loss as of December 31, 2023 included non-cash compensation expenses of $3.3 million as compared to 2022 non-cash compensation expenses of $1.5 million.

Athira Pharma Presents Clinical and Preclinical Data Supporting Therapeutic Potential of Fosgonimeton in Alzheimer’s and Parkinson’s Diseases at AD/PD™ 2024 International Conference

Retrieved on: 
Friday, March 8, 2024
Central nervous system, Toxicity, Research, Pathology, ADAS, HGF, Hepatocyte growth factor, Conference, SHAPE, Doctor of Philosophy, Safety, Dementia, Survival, Athira Pharma, GBA1, Lewy body, Ageing, DSM-IV codes

BOTHELL, Wash., March 08, 2024 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, presented new clinical and preclinical data further highlighting the therapeutic potential of fosgonimeton at the AD/PD™ 2024 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders, being held in Lisbon, Portugal March 5 – 9, 2024.

Key Points: 
  • Fosgonimeton is a potentially first-in-class investigational small molecule designed to positively modulate the hepatocyte growth factor (HGF) system, which can activate neuroprotective, neurotrophic and anti-inflammatory pathways in the central nervous system.
  • “We believe safety and efficacy outcomes from the exploratory SHAPE Phase 2 clinical trial presented at this year’s AD/PD, along with previous clinical data, support the continued development of fosgonimeton.
  • These findings support the potential therapeutic benefit of modulating the neurotrophic HGF system with fosgonimeton for neurodegenerative diseases.
  • “We’re pleased to be presenting additional preclinical data highlighting the neuroprotective effects of fosgonimeton in models of both Alzheimer’s and Parkinson’s diseases at AD/PD 2024,” said Kevin Church, Ph.D., Chief Scientific Officer of Athira.

Gain Therapeutics Presents Data at the AD/PD™ 2024 Conference Demonstrating the Mechanism of Action of GT-02287, its Clinical Stage GCase Regulator for the Treatment of Parkinson’s Disease

Retrieved on: 
Tuesday, March 5, 2024
Alzheimer's disease, PD, Research, Treatment, Travel, Conference, Endoplasmic reticulum, Therapy, Poster, DSM-IV codes, Science, GBA1, Pharmaceutical industry

AD/PD™ 2024 is being held March 5th – 9th, 2024 in Lisbon, Portugal.

Key Points: 
  • AD/PD™ 2024 is being held March 5th – 9th, 2024 in Lisbon, Portugal.
  • GCase is consequently able to travel to the lysosome, resulting in enhanced lysosomal activity and efficient processing of the GCase substrate glucosylceramide.
  • Increase in GCase substrate in the lysosome was previously shown to be associated with accumulation of aggregated alpha-synuclein, a pathological hallmark of Parkinson’s disease and related disorders.
  • “These data further confirm our understanding of the mechanism of action of GT-02287, how it impacts cellular health by preventing the downstream consequences of GCase misfolding, including cellular stress and lysosomal dysfunction” commented Dr. Natalia Perez-Carmona, Senior Director of Biology at Gain Therapeutics and presenting author.

Gain Therapeutics Announces the Initiation of the Multiple Ascending Dose (MAD) Part of the Phase 1 Clinical Trial of GT-02287, a Novel GCase-targeting Small Molecule Therapy for GBA1 Parkinson’s Disease

Retrieved on: 
Tuesday, February 27, 2024
SAD, Glucocerebrosidase, Safety, Single, Therapy, AFL, Clinical trial, Plasma, GBA1, Multiple

Following the HREC approval, administration of GT-02287 in the MAD part of the study has been initiated.

Key Points: 
  • Following the HREC approval, administration of GT-02287 in the MAD part of the study has been initiated.
  • “Our Phase 1 clinical trial of GT-02287 remains on track and according to plan,” said Matthias Alder, Chief Executive Officer of Gain Therapeutics.
  • Compelling preclinical data presented at WORLDSymposium™ earlier in February 2024 demonstrated that treatment with GT-02287 completely restored motor function and reduced plasma levels of the emerging neurodegeneration biomarker NfL back to normal levels.
  • Based on these data, GT-02287 may have the potential to slow or even stop the progression of Parkinson’s disease.

Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich’s Ataxia in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment

Retrieved on: 
Monday, February 26, 2024
Traffic barrier, ALS, SOD1, Neurocrine Biosciences, Therapy, Neurocrine, FXN, GBA1, AAV, Research, FA, Vaccine

The candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform.

Key Points: 
  • The candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform.
  • Selection of the development candidate triggered a $5 million milestone payment to Voyager, which the Company expects to receive in the first quarter of 2024.
  • Voyager is eligible to receive additional future development and commercialization milestone payments based on the further advancement of this program.
  • We believe our strategy to replace the defective frataxin gene could address the underlying disease etiology of FA.

Gain Therapeutics’ GT-02287 Completely Restores Motor Function in Mouse Models of Parkinson’s Disease

Retrieved on: 
Tuesday, February 6, 2024
Neuroprotection, Poster, Pharmacokinetics, ALS, AFL, Clinical trial, Patient, Safety, Mutation, FDA, Animal, Food, MPS II, Disease, Therapy, MPS, Neurofilament, SOD1, Glucocerebrosidase, GBA1, Plasma, Pharmaceutical industry

The data was accepted as a late-breaker abstract and will be presented at the 20th Annual WORLDSymposium™ being held in San Diego this week.

Key Points: 
  • The data was accepted as a late-breaker abstract and will be presented at the 20th Annual WORLDSymposium™ being held in San Diego this week.
  • “We believe the data showing complete restoration of motor function in a therapeutic model are remarkable and further support the potential of GT-02287 to slow or stop the progression of Parkinson’s disease, a disease for which only symptomatic treatments are available to patients at this time,” said Matthias Alder, Gain Therapeutics’ Chief Executive Officer.
  • Further, animals in the most challenging treatment group – those that began treatment eight days following onset of the disease – showed motor improvement from day 14 to day 27, which suggests progressive reversal of neuronal deficit associated with continued treatment duration.
  • Further details of the study, including protocol and specific results can be found in the poster, which was presented today and can be accessed here .

Gain Therapeutics’ CEO Matthias Alder Issues Letter to Shareholders and Provides Operational Update

Retrieved on: 
Wednesday, January 31, 2024
Multiple sclerosis, Movement, Poster, GM1 gangliosidoses, Neuroprotection, Enamine, Workflow, SAD, AFL, GM1, ChromeOS, Clinical trial, Research, PLOS, Neuroinflammation, Biomarker, Patient, Safety, Ferdinand Magellan, FDA, Drug discovery, Organization, Neurodegenerative disease, AbbVie, Dopamine, Disease, Protein, Therapy, Acquisition, Merck, Letter, Glucocerebrosidase, GBA1, GLB1, University, Parkinson's disease, ALS, Plasma, Conference, Caraway, Pharmaceutical industry

In 2023, we advanced GT-02287 through preclinical development and initiated the company’s first clinical trial in September 2023 on time and on plan.

Key Points: 
  • In 2023, we advanced GT-02287 through preclinical development and initiated the company’s first clinical trial in September 2023 on time and on plan.
  • The dose escalation of the SAD phase is underway, and the MAD phase of the study is expected to begin in Q1 2024.
  • In 2023, we made several data presentations of results of our GBA1 program in preclinical models of Parkinson’s disease and Alzheimer’s disease.
  • Xavi is leaving the world of academia as a professor at the University of Barcelona and his part-time engagement with Gain to join a major pharmaceutical company.

Gain Therapeutics Announces Closing of $10.1 Million Public Offering and Concurrent Private Placement of Common Stock and Warrants, Including Full Exercise of Over-Allotment Option

Retrieved on: 
Friday, November 24, 2023
Telephone, Suite, ATTN:, Sale, Form, Therapy, S-3, GBA1, Financial adviser, Security (finance), Exercise

The warrants were sold at the rate of one warrant for every two shares of common stock.

Key Points: 
  • The warrants were sold at the rate of one warrant for every two shares of common stock.
  • In a private placement completed concurrently with the completion of the public offering, Gain issued to an accredited investor 2,500,088 shares of common stock (or pre-funded warrants in lieu thereof) and warrants to purchase up to 2,500,088 shares of common stock.
  • Newbridge Securities Corporation acted as the sole book-running manager for the public offering and as placement agent for the private placement.
  • Gross proceeds from the public and private offering were approximately $10.1 million, excluding underwriting and placement agent discounts and commissions and other offering-related expenses.

Gain Therapeutics Announces Pricing of $9.4 Million Public Offering and Concurrent Private Placement of Common Stock and Warrants

Retrieved on: 
Tuesday, November 21, 2023
Telephone, Suite, ATTN:, Sale, Form, Therapy, GBA1, Financial adviser, Security (finance), Exercise

The warrants are being sold at the rate of one warrant for every two shares of common stock.

Key Points: 
  • The warrants are being sold at the rate of one warrant for every two shares of common stock.
  • In connection with the offering, Gain has granted the underwriters a 30-day option to purchase up to an additional 15% of shares of common stock and/or warrants to purchase shares of common stock at the public offering price, less underwriting discounts and commissions.
  • In a private placement to be completed concurrently with the completion of the public offering, Gain will issue to an accredited investor an aggregate of 2,500,088 shares of common stock (or pre-funded warrants in lieu thereof) and warrants to purchase up to 2,500,088 shares of common stock.
  • Newbridge Securities Corporation is acting as the sole book-running manager for the public offering and as placement agent for the private placement.

Gain Therapeutics Announces Proposed Public Offering

Retrieved on: 
Monday, November 20, 2023
Telephone, Suite, Sale, Form, Therapy, GBA1, Prospectus, ATTN:

BETHESDA, Md., Nov. 20, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (“Gain” or the “Company”) (Nasdaq: GANX) today announced that it is proposing to offer and sell, subject to market conditions, shares of its common stock and warrants to purchase common stock in an underwritten public offering.

Key Points: 
  • BETHESDA, Md., Nov. 20, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (“Gain” or the “Company”) (Nasdaq: GANX) today announced that it is proposing to offer and sell, subject to market conditions, shares of its common stock and warrants to purchase common stock in an underwritten public offering.
  • Gain expects to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of common stock and/or the warrants offered in the offering.
  • The final terms of the offering will depend on market and other conditions at the time of pricing, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.
  • Newbridge Securities Corporation is acting as the sole book-running manager for the proposed offering.