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Gain Therapeutics Announces the Completion of Dosing in the Multiple Ascending Dose (MAD) of the Phase 1 Study of GT-02287, a Novel Small Molecule Therapy for Parkinson’s Disease

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Gain Therapeutics announced the completion of dosing in the Multiple Ascending Dose (MAD) part of their Phase 1 study for GT-02287, a novel small molecule therapy for Parkinson's Disease. Conducted without any serious adverse events or discontinuations, the study indicates that GT-02287 is well-tolerated. The topline data from this randomized, double-blind, placebo-controlled study is expected in mid-August 2024, with full safety analysis and plasma pharmacokinetics to be presented later. The company aims to initiate trials in Parkinson’s disease patients by Q4 2024. The MAD phase, begun in February 2024, involved 32 healthy volunteers completing 14 days of daily oral doses. The completion of the Single Ascending Dose (SAD) part in April 2024 also reported no serious adverse events. Preclinical data shows GT-02287 effectively restores the function of the enzyme glucocerebrosidase, which is linked to Parkinson’s disease due to GBA1 gene mutations.

Positive
  • Completion of MAD part of Phase 1 study for GT-02287 with no serious adverse events reported.
  • Topline data expected in mid-August 2024, with full safety analysis and plasma pharmacokinetics to follow.
  • Trials in Parkinson’s disease patients on track to initiate by Q4 2024.
  • GT-02287 well-tolerated up to the highest planned dose level, indicating a favorable safety profile.
  • SAD part of Phase 1 study also completed with no serious adverse events.
  • Preclinical data indicates GT-02287 may slow Parkinson’s disease progression.
Negative
  • Adverse events recorded were 90% mild and 10% moderate, though none were Grade 3 or higher.

The completion of the multiple ascending dose (MAD) phase of the Phase 1 clinical trial for GT-02287 is a significant milestone for Gain Therapeutics. With no serious adverse events reported and a favorable safety profile, the drug shows promise in its potential to become a transformative therapy for Parkinson's disease. The fact that the drug was well-tolerated across various dosage levels is crucial, as it lays a strong foundation for subsequent clinical trials involving Parkinson’s patients.

For retail investors, this development indicates a solid step forward in the drug development pipeline. The absence of severe adverse reactions in a Phase 1 trial is important because it significantly de-risks the subsequent phases of clinical testing. However, it's important to note that this is still an early stage in the drug development process and future trials will need to confirm efficacy in Parkinson’s patients and further validate these safety findings.

The announcement that GT-02287 is on track to initiate clinical trials in Parkinson’s patients by Q4 2024 is encouraging. This timeline adheres to typical drug development schedules, suggesting that Gain Therapeutics is progressing as planned without significant delays.

For investors, patience is key as the drug moves through additional phases of testing. While early results are promising, the path to regulatory approval can be long and challenging. The safety and pharmacokinetics data to be released mid-August will offer further insights into the drug’s performance and potential.

In summary, the completion of the MAD phase is a positive development, but investors should remain cautious until more comprehensive data is available.

From a financial perspective, the news represents a positive step for Gain Therapeutics, potentially increasing investor confidence and market valuation. The fact that no serious adverse events were reported in the Phase 1 trial reduces the risk profile of GT-02287, which can be attractive to investors looking for opportunities in the biotech sector. This successful milestone may also improve Gain's ability to secure additional funding, either through capital markets or strategic partnerships, which is often critical for clinical-stage biotech companies.

Investors should keep an eye on the upcoming release of the topline data and full safety analysis. These reports will provide further validation of the drug’s potential and could significantly influence stock performance. Additionally, initiating trials in Parkinson’s patients by Q4 2024 could serve as a catalyst for stock price movement, especially if the results are favorable.

It's also worth mentioning that advancements in GT-02287 might attract attention from larger pharmaceutical companies, potentially leading to strategic partnerships or acquisition opportunities. These could provide substantial financial upside for Gain Therapeutics and its investors.

However, as always in the biotech industry, the path to market approval is fraught with uncertainty. Investors should be prepared for the inherent volatility and potential setbacks that could occur as the drug progresses through clinical trials.

GT-02287 Well Tolerated With No Serious Adverse Events or Other Safety Signals Reported

Additional Topline Data To Be Presented Mid-August

GT-02287 on Track To Initiate Clinical Trial in Parkinson’s Disease Patients in Q4 2024

BETHESDA, Md., July 09, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, announces the last healthy subject in the multiple ascending dose (MAD) part of its Phase 1 study has completed the highest planned dose levels. No discontinuations or serious adverse events were reported. After database lock, topline data from this Phase 1, randomized, double-blind, placebo-controlled study is expected to be released in August, and full safety analysis and plasma pharmacokinetics are expected to be presented at a future congress.

“The successful completion of the SAD/MAD in 72 healthy subjects with no serious adverse events or discontinuations related to adverse events and the ability to achieve therapeutic plasma levels with oral dosing further confirm our belief in GT-02287’s potential to be a transformative therapy for Parkinson’s disease patients,” commented Gain’s Executive Chairman, Khalid Islam, Ph.D. “We remain on track with our recent guidance and look forward to continuing to advance GT-02287 through clinical development and initiating a trial in Parkinson’s disease patients by Q4 2024.”

The MAD part of the Phase 1 study was initiated in February, and all four MAD cohorts have completed daily oral dosing for 14 days. GT-02287 was well tolerated up to and including the highest planned dose level, and no safety signals have been detected in the 32 healthy volunteers who participated in the MAD part. Of the adverse events that occurred, 90% were mild and 10% were moderate with no Grade 3 or higher events reported. The favorable safety and tolerability profile and the appropriate range of plasma exposures achieved after oral administration further bolster GT-02287’s best-in-class potential.

The primary objective of this Phase 1 clinical trial with single- and multiple ascending doses was to evaluate the safety and tolerability of GT-02287 administered orally once daily in healthy adults. The secondary objective was to evaluate the pharmacokinetics of SAD and MAD dose levels to identify recommended doses for further clinical development in people with Parkinson’s disease. The SAD part of the Phase 1 clinical trial concluded in April with positive results and no serious adverse events. The SAD part of the Phase 1 clinical trial enrolled 40 healthy participants across five separate cohorts – all of which were completed at the planned dose levels with no premature discontinuations or safety signals.

GT-02287 has been shown to restore the function of the lysosomal enzyme glucocerebrosidase (GCase), which becomes misfolded and dysfunctional due to GBA1 gene mutations, the most common genetic risk factor for the development of Parkinson’s disease. Compelling preclinical data in mouse models of GBA1-PD, including that presented at FENS Forum 2024 in June describing improvement in cognitive performance in addition to motor performance after administration of GT-02287, suggests that GT-02287 may have the potential to slow the progression of Parkinson’s disease.

About GT-02287
Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease. The orally administered, brain-penetrant small molecule is an allosteric protein modulator that restores the function of the lysosomal protein enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to mutations in the GBA1 gene, the most common genetic abnormality associated with PD, or other age-related stress factors. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced aggregated α-synuclein, neuroinflammation and neuronal death, increased dopamine levels and improved motor function and cognitive performance. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker for neurodegeneration.

Gain’s lead program in Parkinson’s disease 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 – Swiss Innovation Agency.

About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate GT-02287 for the treatment of Parkinson’s disease, is currently being evaluated in a Phase 1 clinical trial.
Leveraging AI-supported structural biology, proprietary algorithms, and supercomputer-powered physics-based models, the company’s Magellan™ drug discovery platform can identify novel allosteric binding sites on disease-implicated proteins, pinpointing pockets that cannot be found or drugged with current technologies. Its AI and machine-learning tools and virtual screening capabilities leverage the emerging on-demand compound libraries covering vast chemical spaces of over five trillion compounds to identify and select suitable small molecule hits for experimental validation.

Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders and oncology.

Forward-Looking Statements
This release contains “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are typically preceded by words such as “believes,” “expects,” “anticipates,” “intends,” “will,” “may,” “should,” or similar expressions. These forward-looking statements reflect management’s current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct or that those goals will be achieved, and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, risks associated with market conditions and the satisfaction of customary closing conditions related to the offering and uncertainties related to the offerings and the use of proceeds from the offerings. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the Company’s business in general, please refer to the Company’s prospectus supplement to be filed with the SEC, and the documents incorporated by reference therein, including the Company’s Form 10-K for the year ended December 31, 2023 and Form 10-Q for the quarter ended March 31, 2024.
All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this release. We have no obligation, and expressly disclaim any obligation, to update, revise or correct any of the forward-looking statements, whether as a result of new information, future events or otherwise.

Investor Contacts:
Apaar Jammu and Chuck Padala
ajammu@gaintherapeutics.com
chuck@lifesciadvisors.com

Media Contacts:
Russo Partners
Nic Johnson and Elio Ambrosio
nic.johnson@russopartnersllc.com
elio.ambrosio@russopartnersllc.com
(212) 845-4242


FAQ

What is GT-02287?

GT-02287 is a novel small molecule therapy developed by Gain Therapeutics for treating Parkinson's Disease.

When did Gain Therapeutics complete the dosing in the MAD part of the Phase 1 study for GT-02287?

The dosing in the Multiple Ascending Dose (MAD) part of the Phase 1 study for GT-02287 was completed in July 2024.

Were there any serious adverse events reported in the MAD study for GT-02287?

No serious adverse events or discontinuations were reported in the Multiple Ascending Dose (MAD) study for GT-02287.

When will the topline data for GT-02287's Phase 1 study be available?

The topline data for GT-02287's Phase 1 study is expected to be released in mid-August 2024.

When is Gain Therapeutics planning to initiate clinical trials for GT-02287 in Parkinson's disease patients?

Gain Therapeutics plans to initiate clinical trials for GT-02287 in Parkinson's disease patients by Q4 2024.

What were the findings from the preclinical studies of GT-02287?

Preclinical studies indicated that GT-02287 can restore the function of the enzyme glucocerebrosidase and may slow the progression of Parkinson's disease.

Gain Therapeutics, Inc.

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