Gain Therapeutics reports positive Phase 1 trial results for Parkinson’s treatment By Investing.com

BETHESDA, Md. – Gain Therapeutics, Inc. (NASDAQ: GANX), a biotech firm engaged in developing allosteric small molecule therapies, has announced the completion of a Phase 1 clinical trial for its leading drug candidate, GT-02287, intended to treat Parkinson’s disease. The trial results showed the drug was well tolerated with no serious adverse events reported among the participants.

The clinical trial, which included both single ascending dose (SAD) and multiple ascending dose (MAD) studies, assessed the safety and tolerability of GT-02287 in healthy adults. The MAD portion, which recently concluded, involved 32 volunteers who completed daily oral dosing for 14 days. According to the press release, 90% of the adverse events were mild, and no safety signals were detected up to the highest planned dose level.

Executive Chairman of Gain, Khalid Islam, Ph.D., expressed confidence in the drug’s potential based on these results, stating that the company is on schedule to begin a clinical trial with Parkinson’s disease patients in Q4 2024. The SAD part of the trial, completed in April, also showed positive outcomes with no serious adverse events across 40 healthy participants.

GT-02287 targets the lysosomal enzyme glucocerebrosidase (GCase), which is impaired in Parkinson’s due to mutations in the GBA1 gene. Preclinical data suggests that the drug can restore GCase function, potentially slowing the progression of the disease. The full safety analysis and plasma pharmacokinetics from the Phase 1 study are slated to be presented in mid-August at a future congress.

Gain Therapeutics’ approach to drug discovery utilizes AI and proprietary algorithms to identify novel allosteric binding sites on proteins implicated in diseases. The company’s platform can screen vast chemical spaces for potential small molecule modulators, accelerating the development of treatments for neurodegenerative diseases, rare genetic disorders, and oncology.

It is important to note that this article is based on a press release statement.

In other developments, H.C. Wainwright adjusted its price target for Gain Therapeutics to $8.00, following a shift in the company’s leadership. Gene Mack, the firm’s CFO, has taken on the role of interim CEO, while Khalid Islam, Ph.D., the founder and Chairman of Gain Therapeutics’ Board of Directors, will provide additional support as Executive Chairman.

The company also announced its intention to offer and sell shares of its common stock in a public offering, with Titan Partners Group acting as the sole bookrunner. The proceeds from the offering are earmarked for the clinical and nonclinical development of GT-02287, among other corporate purposes.

Finally, Gain Therapeutics has welcomed new additions to its leadership team. Gene Mack has taken on the role of CFO and interim CEO, while Jonas Hannestad, M.D., Ph.D., has been appointed as its Chief Medical Officer. Both bring extensive experience in their respective fields.

InvestingPro Insights

Amid the news of Gain Therapeutics, Inc.’s (NASDAQ: GANX) successful completion of a Phase 1 clinical trial for GT-02287, investors may be weighing the potential impact on the company’s financial health and stock performance. With a market capitalization of $28.19 million, the company is currently navigating a challenging financial landscape. According to the latest InvestingPro Data, GANX has a Price / Book ratio of 3.26 as of the last twelve months ending Q1 2024, which provides some insight into how the market values the company relative to its book value.

One of the InvestingPro Tips highlights that analysts are expecting sales growth in the current year, which could be a positive sign for the company’s future revenue streams. However, it’s also noted that the company is not anticipated to be profitable this year, and it has been quickly burning through cash. This situation underscores the importance of the company’s research and development efforts to potentially bring new products to market that could alter its financial trajectory.

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