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Gain Therapeutics

Updated: Aug 31, 2022

Gain Therapeutics  |  source -

Gain Therapeutics (NASDAQ: GANX) is a preclinical stage drug discovery and development company focused on protein misfolding diseases, like GBA1 Parkinson’s Disease (GBA1 PD). Their supercomputer-driven patented discovery platform, SEE-Tx, identifies novel high probability binding sites on misfolded proteins toward the development of new small molecule drug candidates for treating disease. They have several candidate drugs in development, two of which (GT-02287, GT-02329) have demonstrated potential in improving physiological and behavioral symptoms associated with GBA1 PD and Gaucher Disease in preclinical models. Investment risks associated with preclinical stage companies cannot be easily overstated; however, Gain’s diverse value proposition (multiple candidates and a discovery platform) and leadership’s extensive commercial experience differentiate them from competitors.


Proteins are the building blocks of life, made up of amino acids, folded into precise shapes to accomplish specific tasks. For biological systems to function properly, proteins must be (1) initially folded into the correct shape, (2) maintained in their proper conformation (shape), and (3) cleared by degradation when erroneous protein folding occurs. A complex ensemble of chaperone molecules and enzymes exist as our internal Protein Quality Control System (PQCS). When proteins misfold, or when elements of the PQCS fail, their function is almost always lost. In addition to becoming dysfunctional, misfolded proteins tend to self-associate and aggregate. The aggregation of misfolded proteins is a hallmark of several incurable diseases, including Parkinson’s and Alzheimer’s. One attractive potential solution to address protein misfolding is found in designer small molecules that bind and change the conformation of the protein, called allosteric regulators. However, therapeutics which might allosterically modulate and correct the misfolded proteins associated with disease are not currently available due to a lack of known targetable binding sites.

Gain Therapeutics (NASDAQ:GANX) SEE-Tx Discovery Platform By the Numbers  |  source -
SEE-Tx Discovery Platform By the Numbers | source -


Gain Therapeutics’ physics-based platform SEE-Tx utilizes a patented approach to identify previously unknown “druggable binding hot spots” and associated probability of binding site success. Developed over ~20 years by Chief Technology Officer Xavier Barril, Ph.D, the SEE-Tx engine generates an average hit rate (across all implementations) of ~14%, which is several orders of magnitude better than high throughput screening approaches. SEE-Tx is further differentiated from other drug discovery platforms by its ability to rapidly analyze candidate molecules - moving from screening through experimental validation in <10 weeks. This structural and computational approach to rapidly identifying allosteric targets provides numerous opportunities for drug discovery in conditions with notable unmet need, from hereditary conditions to cancers and CNS disorders. SEE-Tx has thus far enabled the development of two promising drug candidates soon to enter Phase I clinical trials: GT-02287 for GBA1 Parkinson’s Disease, and GT-02329 for Gaucher Disease. Gain’s lead drug candidates demonstrate significant improvement in clinically-relevant disease phenotypes in preclinical in vitro and in vivo models. Notably, GT-02287 reduces both total α-synuclein and phosphorylated α-synuclein in the substantia nigra in a rodent model of PD - the buildup of which is an important hallmark of the disease.

STAR Compound GT-02287 Decreases Total and Phosphorylated α-synuclein in the Substantia Nigra in a Rotenone Rat Model  |  Source -
GT-02287 Decreases α-synuclein in the Substantia Nigra in a Rotenone Rat Model | source -


  • Eric Richman (Chief Executive Officer + Board Member): Seasoned biotech exec and venture partner with >30 years of experience in research, development and commercialization across a range of indications, from rare hereditary disorders to infectious disease. Eric has been essential to multiple successful acquisitions.

  • Manolo Bellotto, PhD (General Manager + President): International pharmaceutical product expert with a PhD in molecular developmental genetics, Dr. Bellotto has >20 years of pharmaceutical product strategy in rare/orphan disease.

  • Xavier Barril, PhD (Chief Technology Officer): SEE-Tx platform developer with an impressive publication (80) and patent (10) record. Dr. Barril holds a PhD in biochemistry and has broad success as a senior investigator.

  • Matthias Alder (Chief Operations Officer): Operations veteran with >20 years of drug development and licensing success, delivering multiple strategic partnerships across orphan, rare CNS, and oncology indications.

  • Khalid Islam (Founder + Chairman of the Board): Industry giant with >30 years of demonstrated success in multiple roles across life science. Key player in Gentium's $1B all cash merger with Jazz Pharmaceuticals.

To learn more about Gain's leadership and board of directors, visit their website.


Trends: There is enormous global interest in improving treatment options for protein misfolding disorders, with Alzheimer’s and Parkinson’s Disease being two of the most prevalent, debilitating and deadly. Note that there have been no meaningful improvements in standard of care treatment for PD in several years, despite intense clinical, public and institutional interest, reflected by exceptionally good liquidity among public companies with drug candidates in pipeline for PD despite market conditions, including Denali Therapeutics (DNLI), Ionis Pharmaceutical (IONS), Sage Therapeutics (SAGE), Arvinas (ARVN), Supernus Pharmaceuticals (SUPN), and Intra-Cellular Therapies (ITCI). Notable recent advances in computational protein research have renewed incredible, warranted public interest in protein-related discovery. DeepMind’s recently released AlphaFold project marks a new generation of protein research by successfully applying artificial intelligence to rapidly predict protein structures. As 3D protein structure (previously from crystallography) is a variable fed into the SEE-Tx platform, the AlphaFold release is beneficial, not competitive, to Gain Therapeutics.

Value: Preclinical validation of GT-02287 and GT-02329, a rich library of candidates in research- and discovery-stages, and a proprietary discovery engine, together represent several opportunities for company success. The estimated prevalence of PD is approximately 1 in 200 people in the United States, with GBA1 PD accounting for roughly 10% of those diagnosed with PD. With prevalence increasing YoY, no cure, and poor available treatment strategies, a successful drug in this category likely represents a multi-billion USD opportunity. Their listed shares (NASDAQ:GANX) are trading at $3.56 USD (as of market close 8/30/22) and there are currently 11,883,368 shares outstanding (as of 7/31/22) and cash or cash equivalents of $29.1M USD (as of 6/30/22). Gain has runway through the end of 2024 at current burn, though non-dilutive financing from drug development collaborations could significantly extend runway.

Risks: Aside from the typical clinical, regulatory and manufacturing risks facing preclinical life science companies, we observe the following specific risks for Gain Therapeutics:

  • While Gain has extraordinary depth of experience in commercialization and strategy, their leadership experience in clinical trials (design, endpoint selection, etc.) is unclear. It may be prudent to bring on a clinical expert with demonstrated success bringing drugs through FDA safety and efficacy trials as their candidates enter FDA trials.

  • While they have significant cash, it is possible that Gain will need to raise capital for clinical trials or commercialization if additional drug development collaborations don’t emerge. FDA safety trials may take several months to a year, and efficacy trials are unlikely to be completed before the end of their cash runway.

To stay up to date with Gain Therapeutics, visit their website.

Integra Consulting Group logo

The above opinion is for information purposes only and is not intended to be investment advice. Always seek a licensed professional for investment advice. At the time of publication, Integra Consulting Group has no current or prior financial relationship with Gain Therapeutics (NASDAQ:GANX) or its partners. At any time, employees or advisors of Integra Consulting Group may or may not hold a position in Gain Therapeutics (NASDAQ:GANX) or its partners.



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