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Also known as Low Dose GT-02287
GT-02287 · 2 trials · 1 indication
Change from Baseline to Week 48 in the sum of the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II score and Part III score assessed in the practically defined OFF state. MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of four parts. Part II assesses motor experiences of daily living (Range 0-52). It contains 13 questions which are to be rated by the patient and/or caregiver. Part III assesses the motor signs of PD and is rated by the investigator (Range 0-132). Part III contains 33 scores based on 18 items. For each question a numeric score is assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. A higher score indicates more severe symptoms of PD.
| Arm | Type | Description |
|---|---|---|
| Low Dose GT-02287 | ACTIVE_COMPARATOR | GT-02287 400 or 600 mg/day |
| High Dose GT-02287 | ACTIVE_COMPARATOR | GT-02287 800 or 1000 mg/day |
| Placebo | PLACEBO_COMPARATOR | Magnesium aluminometasilicate (MAS) |
| single-arm. All participants receive the active molecule (once daily GT-02287 administration) | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Low Dose GT-02287 | DRUG | Sachets containing 400 mg/day or 600 mg/day of active (GT-02287), depending on the participant's body weight. Each daily dose will be prepared as an oral suspension by mixing the content of 1 sachet with the vehicle (supplied in an amber glass bottle) and the requisite volume of tap water. |
| High Dose GT-02287 | DRUG | Sachets containing 800 mg/day or 1000 mg/day of active (GT-02287), depending on the participant's body weight. Each daily dose will be prepared as an oral suspension by mixing the content of 1 sachet with the vehicle (supplied in an amber glass bottle) and the requisite volume of tap water. |
| Placebo | DRUG | Sachets containing 420 to 1200 mg of MAS indistinguishable from the active sachets. Each daily dose will be prepared as an oral suspension by mixing the content of 1 sachet with the vehicle (supplied in an amber glass bottle) and the requisite volume of tap water. |
| GT-02287 | DRUG | Dose of 13.5 mg/kg/day (plus/minus 2 mg/kg/day based on body weight) to be administered orally once a day for 90 days. Dosage form: powder in sachet (200 mg of GT-02287 per sachet) for reconstitution with a suspending agent |
Inclusion Criteria: * Able and willing to provide written informed consent and willing to comply with the requirements and restrictions of the study * Willing to undergo PD-related genetic testing and analysis * Any sex, ≥30 and ≤85 years of age * Body mass index of ≥18 and ≤40 kg/m2 and a body wei...
Top 15 of 23 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Supernus Pharmaceuticals, Inc. | SUPN | 2 | PHASE3 | Onapgo (SPN-830) |
| Prothena Corp. Plc | PRTA | 3 | PHASE3 | Prasinezumab |
| Denali Therapeutics Inc. | DNLI | 1 | PHASE2 | BIIB122 |
| Bayer | BAYRY | 4 | PHASE3 | bemdaneprocel, AB-1005 |
| Biohaven Ltd. | BHVN | 1 | PHASE2 | BHV-8000 |
| AC Immune SA | ACIU | 2 | PHASE2 | ACI-7104.056 |
| Eli Lilly and Company | LLY | 4 | PHASE1 | LY3884961, LY4006896, LY3962681 |
| Gain Therapeutics, Inc. | GANX | 2 | PHASE2 | GT-02287 |
| Annovis Bio Inc | ANVS | 1 | PHASE2 | Buntanetap |
| Regeneron Pharmaceuticals, Inc. | REGN | 2 | PHASE1 | ALN-SNCA |
| MeiraGTx Holdings Plc | MGTX | 1 | PHASE1 | AAV-GAD |
| Serina Therapeutics, Inc. | SER | 1 | PHASE1 | SER-252 |
| Ardelyx, Inc. | ARDX | 1 | PHASE2 | Tenapanor |
| Hoffmann-La Roche | RHHBY | 1 | PHASE1 | RO7568282 |
| Merck & Co., Inc. | MRK | 1 | EARLY_PHASE1 | MK-0947 |
GT-02287 is an investigational small molecule being developed for the treatment of Parkinson disease. It is a Phase 2 candidate in the neurology therapeutic area, and it is not yet approved for any use. Gain Therapeutics is studying it in early Parkinson disease as well as in a separate safety and pharmacology study.
GT-02287 targets GCase, a lysosomal enzyme also known as glucocerebrosidase. By acting on this lysosomal enzyme, the small molecule is designed to address GCase-related biology in Parkinson disease. GCase is the molecular target named for this investigational compound.
GT-02287 is being developed by Gain Therapeutics, Inc., which trades on the Nasdaq under the ticker GANX. Gain Therapeutics is the sponsor behind the clinical program evaluating the compound in Parkinson disease, including a Phase 2 trial and a Phase 1 study.
GT-02287 is in Phase 2 development for Parkinson disease. It is an investigational drug and has not been approved by the FDA. The Phase 2 trial is not yet recruiting, while a Phase 1 safety, tolerability, pharmacokinetics, and pharmacodynamics study is currently recruiting.
GT-02287 is being studied in two trials. NCT07280299 is a Phase 2 trial evaluating efficacy, safety, tolerability, and biomarker effects in early Parkinson disease, with planned enrollment of 111 participants. NCT06732180 is a Phase 1 trial assessing safety, tolerability, pharmacokinetics, and pharmacodynamics in Parkinson disease, enrolling 20 participants in Australia.
Yes. Low Dose GT-02287 is another name used for GT-02287, so searches for either term refer to the same investigational compound. Both names point to the GCase-targeting small molecule that Gain Therapeutics is developing for Parkinson disease in Phase 2 and Phase 1 clinical trials.