Chemistry:GT-02287

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GT-02287 is a β-glucocerebrosidase (GCase) activator, positive allosteric modulator, and/or chaperone which is under development for the treatment of Parkinson's disease, Gaucher's disease, solid tumors, and dementia.[1][2][3][4][5][6] It is taken orally.[1]

The drug is under development by Gain Therapeutics.[1][2][3] As of January 2026, it is in phase 1 clinical trials for Parkinson's disease, the preclinical research stage of development for Gaucher's disease and solid tumors, and the research stage of development for dementia.[1][2][3] GT-02287 was also under development for alpha 1-antitrypsin deficiency, globoid cell leukodystrophy, and GM1 gangliosidosis, but development for these indications was discontinued.[1]

Rexaceract (INN) structure.

The chemical structure of GT-02287 does not yet appear to have been disclosed.[1] However, Gain Therapeutics has patented GCase allosteric chaperones, notably including rexaceract (INN), which is described as a β-glucocerebrosidase positive allosteric modulator and antiparkinsonian agent.[7][8][9] Gain Therapeutics is or was also developing another GCase activator, GT-02329, to treat Gaucher's disease and Parkinson's disease, but no recent development has been reported for this candidate.[10]

See also

  • List of investigational Parkinson's disease drugs
  • Pariceract (BIA 28–6156; LTI-291)
  • Ambroxol

References

  1. ↑ 1.0 1.1 1.2 1.3 1.4 1.5 "GT 02287". AdisInsight. Springer Nature Switzerland AG. 29 January 2026. https://adisinsight.springer.com/drugs/800063603. 
  2. ↑ 2.0 2.1 2.2 "Delving into the Latest Updates on GT-02287 with Synapse". 20 December 2025. https://synapse.patsnap.com/drug/00935c3d39b64522a8ba4b8603d44da6. 
  3. ↑ 3.0 3.1 3.2 "GT-02287 Drug Profile". 1 January 1900. https://pryzm.ozmosi.com/product/23956. 
  4. ↑ "Allosteric Modulation of GCase Enhances Lysosomal Activity and Reduces ER Stress in GCase-Related Disorders". International Journal of Molecular Sciences 26 (9): 4392. May 2025. doi:10.3390/ijms26094392. PMID 40362629. 
  5. ↑ "Glucocerebrosidase Target Engagement and Therapeutic Plasma and Cerebrospinal Fluid Levels After GT-02287 Administration in Healthy Volunteers". Movement Disorders. November 2025. doi:10.1002/mds.70111. PMID 41194551. 
  6. ↑ "Early data suggest GT-02287 slows Parkinson's disease". 8 October 2025. https://www.ddw-online.com/early-data-suggest-gt-02287-slows-parkinsons-disease-37433-202510/. 
  7. ↑ "Heteroaryl compounds and therapeutic uses thereof in conditions associated with the alteration of the activity of beta-glucocerebrosidase". 25 November 2020. https://patents.google.com/patent/WO2021105908A1. 
  8. ↑ "4-amino-N-(2,3-dihydro-1H-inden-2-yl)-6-(2-fluoroanilino)pyridine-2-carboxamide". https://pubchem.ncbi.nlm.nih.gov/compound/156408376. 
  9. ↑ "Proposed INN: List 134 International Nonproprietary Names for Pharmaceutical Substances (INN)". WHO Drug Information 39 (4): 1270–1271. 2025. https://cdn.who.int/media/docs/default-source/international-nonproprietary-names-(inn)/pl134.pdf. 
  10. ↑ "GT 02329". AdisInsight. Springer Nature Switzerland AG. 28 October 2025. https://adisinsight.springer.com/drugs/800063606. 



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