Protein misfolding specialist Gain nets $40m from IPO

Gain Therapeutics has priced its initial public offering, raising more than $40 million for its computational drug discovery platform and pipeline of drugs for diseases associated with protein misfolding. 

The shares began trading on the Nasdaq yesterday under the symbol ‘GANX’, closing at $11.20 and valuing the company at around $130 million.

Bethesda, Maryland-headquartered Gain, which was originally formed in Switzerland, is focusing initial on lysosomal storage disorders (LSDs), a group of metabolic disorders that involve an abnormal build-up of various toxic proteins in the body.

Gain’s focus is on LSDs that are caused by proteins that, as a result of pathological processes become misfolded, and lose their ability to function normally, contributing to this toxic built-up. Its initial targets are Gaucher disease, Morquio syndrome (also called mucopolysaccharidosis type IV), GM1 gangliosidosis (GM1) and Parkinson’s disease.

The company has developed a toolkit called Site-Directed Enzyme Enhancement Therapy or SEE-Tx, which uses computational techniques to find small-molecule drugs that are able to bind to sites on misfolded proteins, restore their shape and function and in some cases prevent them clumping together.

The platform looks for areas to bind that are located away from a protein’s main active site, so don’t compete with a protein’s natural substrate, zeroing in on areas that have never been targeted in the past. According to Gain, the computational approach can be used to target misfolded proteins that have been considered “undruggable” using other approaches.

Around $7 to $9 million from the proceeds of the IPO will be used to advance Gain’s preclinical-stage candidates for Morquio and GM1 into phase 1/2 clinical trials, according to the prospectus for the offering.

Another $11 to $14 million is earmarked to take the Gaucher and Parkinson’s disease programmes to the same stage, and $3 to $5 million will be used for preclinical development work on other candidates and other early-stage research activities.

Protein misfolding has become a hot topic in the biopharma industry, with a clutch of emerging companies targeting the broad range of diseases that feature aberrant proteins.

Wren Therapeutics for example is focusing its efforts in this area on neurodegenerative disorders like Lewy body dementia and Parkinson’s, for example, attracting the attention of Eisai which formed an alliance with the startup last year.

Yumanity and Proteostasis are also active in this area, joining forces to seek out therapies for neurodegenerative diseases, including Parkinson’s, dementia and amyotrophic lateral sclerosis (ALS), whilst other players include Orphazyme, Prothena and Proclara.

Meanwhile, in the rare disease arena, CRISPR specialist Intellia is working on a gene-editing approach to treat  hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN), which causes misfolded transthyretin to accumulate and damage nerve cells.

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