Gain Raises $40 Million in IPO, Funds Will Advance Rare Disease Pipeline

Gain Therapeutics made its debut on the Nasdaq Thursday with a $40 million initial public offering. The company will use the funds to advance the development of its pipeline of therapeutics for rare genetic diseases characterized by protein misfolding.

The newly-public stock, trades under the ticker symbol GANX, opened at $11 per share and finished out the day at $11.20. Gain’s IPO included 3,636,364 shares of its common stock at a price of $11. The offering is expected to close on March 22 and the underwriters have a 30-day option to purchase up to an additional 545,454 shares of common stock at the initial public offering price, less underwriting discounts, and commissions. The IPO comes less than one year after it raised $10 million in a Series B financing round.  

Bethesda-based Gain’s Chief Executive Officer Eric I. Richman said the proceeds raised in the IPO will be used to complete pre-clinical proof of concept and IND-enabling studies in multiple programs for patients with rare lysosomal storage diseases and neurodegenerative diseases, which includes Morquio B, GM1 gangliosidosis, Gaucher and Parkinson’s disease.

GM1 gangliosidosis and Morquio B disease are disorders characterized by the same β-galactosidase enzyme deficiency. Neuronopathic Gaucher disease and Parkinson’s disease are characterized by mutations in the GBA gene. The deficiencies interfere with the normal transport to the lysosome.

“Additionally, we look forward to leveraging our SEE-Tx platform to identify previously unknown and previously undruggable targets. The platform uses a validated-target approach to identify druggable allosteric binding sites on misfolded enzymes that when bound by our proprietary molecules, could be exploited to restore the enzyme’s functional activity and address the underlying cause of these diseases,” Richman said in an email.


The IPO comes hot on the trail of Gain’s February presentation at the lysomal disease-focused 17th Annual WorldSymposium that showed the company’s structurally targeted allosteric regulators (STAR) candidates for the treatment of Gaucher Disease enhance GCase activity in a neuronal cell model and reduce toxic accumulation of GCase substrate. Company presentations also showed that STAR candidates for the treatment of GM1 gangliosidosis and Morquio B increase β-gal maturation and delivery to the lysosome and enhance β-gal activity in a panel of fibroblast cell lines.

Gain’s small molecule drug candidates are designed to cross the blood-brain barrier and penetrate other hard-to-treat organs such as bone and cartilage, stabilize the effective enzyme to restore function and reduce toxic substrate. Gain’s STARs are expected to be capable of correcting mutant and wildtype enzyme misfolding and restoring their function, according to the company.

At the time of the announcement, Manolo Bellotto, General Manager and President at Gain, said the company continues to see evidence that structurally targeted allosteric regulators have the ability to increase enzymatic activity in proteins whose deficiency can lead to devastating diseases such as Gaucher disease and GLB1-related disorders. Data that was showcased at the conference showed the ability of STARS to successfully target regulatory allosteric sites in vitro models leading to depletion of the toxic substrate.

Additionally, Bellotto said these data “provide clear evidence that orally bioavailable STARs can penetrate hard-to-treat tissues and reduce substrate accumulation, the root cause of these diseases.”In addition to the preclinical data announced in February, the IPO also followed Gain’s research partnership with the University of Maryland School of Medicine that will investigate the company’s STARs in cellular models of neuronopathic Gaucher disease (nGD) and Parkinson’s disease (PD).

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Alex Keown is a freelance journalist who writes about a variety of subjects including the pharma, biotech, and life science industries. Prior to freelancing, Alex has served as a staff writer and editor for several publications.