• Two Patents on Modulator Compounds for Treatment of CNS Disorders.

• Initial Public Offering Raised $46 Million in Gross Proceeds.

• Agreement with Zentalis Pharmaceuticals for New Cancer Treatments. 

• Four New Independent BOD Members to Strengthen Leadership Team.

• Expects to Complete Proof of Concept Studies and Begin IND-enabling Trials in the Second Half of 2021. 

Gain Therapeutics, Inc. (NASDAQ: GANX) is redefining drug discovery with its SEE-Tx™ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, GANX is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding. 

GANX was established in 2017 with the support of its founders and institutional investors. It has been awarded funding support 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. 

•  Granted Two New PCT Patents Covering GBA and GLAC Modulator Compounds for Treatment of CNS Disorders 

GANX a biotechnology company focused on identifying and optimizing allosteric binding sites that have never been targeted in neurodegenerative diseases and lysosomal storage disorders, today announced its financial results as of and for the second quarter of June 30, 2021, and highlighted recent corporate accomplishments.

Eric Richman, Chief Executive Officer of Gain, added, “We are pleased with the progress in advancing our preclinical compounds to the clinic and announced important and compelling data related to our GBA Parkinson’s program. Our SEE-Tx supercomputing platform technology designed to identify novel allosteric binding sites on misfolded proteins continues to meet objectives within our internal pipeline and our external pharmaceutical partnerships. We continue to advance our pipeline towards achieving our mission in redefining drug discovery and developing novel therapies for devastating conditions with significant unmet medical need.”

Business and Recent Developments

• Expansion of Intellectual Property Portfolio. GAIN continues to expand its intellectual property portfolio and in June announced the publication of two PCT patents. PCT/IB2020/061158 and PCT/IB2020/0611156 are directed at compounds targeting misfolded beta-​glucocerebrosidase (GBA) and galactosylceramidase (GALC) respectively, addressing central nervous system (CNS) and demyelinating disorders such as Parkinson’s Disease, Lewy body dementia and Alzheimer’s Disease.

• Signed Multi-Target Drug Discovery Collaboration with Zentalis Pharmaceuticals in April to discover new product candidates for the treatment of cancer. The Company will utilize its proprietary SEE-Tx computational platform technology to identify new sites on target proteins for potential use in oncology. The intended deliverables are newly discovered targets or target protein interactions that can then be drugged for therapeutic benefit to intervene on protein misfolding.

• Presented at the 26th International Association of Parkinsonism & Related Disorders (IAPRD) World Congress in May. Awarded top 10 best abstract, the presentation highlighted both in vitro & in vivo pre-clinical data demonstrating the potential of STAR compounds for the treatment of GBA1 Parkinson’s Disease. Through partnerships with the Michael J. Fox Foundation and the Silverstein Foundation for Parkinson’s with GBA, GAIN identified two STAR lead candidates (GT-02287 and GT-02329) that can potentially help Parkinson’s patients with GBA1 gene mutations as well as patients whose GCase protein is misfolded due to ageing cellular processes.

• Expansion of the leadership team. In June, Terenzio Ignoni Pharm.D., joined the leadership team as SVP Quality and CMC overseeing all manufacturing operations, global CMC regulatory developments, and quality. Dr. Ignoni joins the Company with more than 20 years of experience in the biopharma industry, holding senior positions in multinational pharmaceutical companies including Jazz Pharmaceuticals, Gentium, Vicuron, and Zambon Group.

• Strong cash position to advance the Company’s programs. As of June 30, 2021, the Company’s cash position was $43.16 million, compared to $7.49 million as of December 31, 2020.

Upcoming 2021 & 2022 Milestones

• New Data Release – Top line data from collaboration with University of Maryland School of Medicine on GBA-associated Parkinson’s Disease in IPSC model.

• Industry Conference – Full data from collaboration with University of Maryland School of Medicine on GBA-associated Parkinson’s Disease.

• Initiate IND-enabling studies 2021 – Gaucher’s / Parkinson’s Disease.

Activity of GBA and GALC Enzymes Play Key Roles in the Neurodegenerative Diseases and Lysosomal Storage Diseases

On June 10thGANX announced the publication of two PCT patents. PCT/IB2020/061158 and PCT/IB2020/0611156 are directed at compounds targeting misfolded beta-​glucocerebrosidase (GBA) and galactosylceramidase (GALC) respectively, addressing central nervous system (CNS) and demyelinating disorders such as Parkinson’s Disease, Lewy body dementia, and Alzheimer’s.

The GANX patent PCT/IB2020/061158 covers compounds and therapeutic uses of conditions associated with the alteration of Beta-glucocerebrosidase (GBA). These conditions include Gaucher Disease, Parkinson’s Disease, and other Lewy body disorders including Alzheimer’s Disease and amyotrophic lateral sclerosis (ALS).

The GANX patent PCT/IB2020/0611156 covers compounds and therapeutic uses of conditions associated with the alteration of galactosylceramidase (GALC). These conditions include lysosomal storage diseases and other demyelinating diseases, such as Krabbe Disease, Lewy body dementia, Multiple Sclerosis, and peripheral neuropathy.

For more information on Gain Therapeutics, Inc. (NASDAQ: GANX) visit https://www.gaintherapeutics.com/

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Media Contact
Company Name: GAIN THERAPEUTICS, INC
Contact Person: CEO: Eric I. Richman
Email: Send Email
Phone: (301) 500-1556
Address:4800 Hampden Lane Suite 200
City: Bethesda
State: MD 37219
Country: United States
Website: https://www.gaintherapeutics.com/