Use of newly named drug leads to stable motor symptoms in Parkinson’s

Small study shows benefits at 1 year with oral therapy once called GT-02287

Written by Andrea Lobo | Oct. 6, 2026

• The oral Parkinson's therapy candidate rexaceract, formerly GT-02287, was shown to stabilize motor symptoms in patients in an early trial.
• Treatment with rexacert led to no meaningful disease progression among the participants after one year.
• Developer Gain Therapeutics is now planning to launch a new mid-stage clinical trial in early 2027.

Gain Therapeutics’ oral therapy candidate rexaceract, formerly called GT-02287, stabilized motor symptoms in a small group of people with Parkinson’s disease who were treated for one year.

That’s according to new data from an open-label extension of an early clinical trial, which showed no meaningful progression of Parkinson’s motor symptoms for any of the evaluable participants taking rexaceract. The study involved people with and without genetic factors that may increase the risk of Parkinson’s.

The findings, “Long-Term Open-Label Data on the GCase Modulator GT-02287 (Rexaceract) in People with Parkinson’s Disease,” were presented by Joanne Taylor, PhD, Gain’s chief scientific officer, at the International Congress of Parkinson’s Disease and Movement Disorders, now being held in Seoul, South Korea. Gain stated in a company press release that these data, stemming from a Phase 1b trial, offer preliminary clinical evidence supporting the drug’s potential as a disease-modifying treatment for Parkinson’s.

“We believe rexaceract has the potential to shift the treatment paradigm beyond symptom management by addressing the underlying biology of Parkinson’s disease, and we expect rexaceract to be the backbone of the next generation of treatment for Parkinson’s,” said Gene Mack, Gain’s president and CEO.

The company said it now plans to submit a new Phase 2 trial protocol to the U.S. Food and Drug Administration in the coming weeks, incorporating information from this study. That new mid-stage trial is expected to start the trial at the beginning of 2027, per the developer.

A progressive disease, Parkinson’s is marked by the buildup of toxic clumps of alpha-synuclein protein, which is thought to contribute to the loss of nerve cells that produce dopamine, a signaling molecule involved in motor function. Genetic factors, such as mutations in the GBA1 gene, may increase a person’s risk of developing Parkinson’s.

The GBA1 gene provides instructions to produce glucocerebrosidase, known as GCase, an enzyme that breaks down and recycles certain fatty molecules, such as glucosylsphingosine (GluSph), inside lysosomes, the cell’s recycling centers. GBA1 mutations reduce GCase activity, which can contribute to lysosomal dysfunction and to alpha-synuclein accumulation.