ADDED
As of February 28, 2026, 42,241,947 shares of the registrant's Common Stock were outstanding.
trade policies that may be made by the presidential administration; and other factors and assumptions described in this Annual Report.
In December 2024, we received approval in Australia to initiate a Phase 1b open-label safety and tolerability study for GT-02287 in 15-20 people with Parkinson s disease with or without a GBA1 mutation.
The study consists of a 90-day Part 1 and an optional 9-month extension (Part 2).
Enrollment was completed in September of 2025, and Part 1 was completed in November 2025.
This study s secondary and exploratory endpoints include pharmacokinetics, GCase modulation, levels of GCase sphingolipid substrates, and other biomarkers in plasma and cerebrospinal fluid.
Part 2 of the Phase 1b study is expected to be complete in the third quarter of 2026, and we are currently planning a Phase 2 study to commence during the second half of 2026.
Our Magellan Platform We use our computational target and drug discovery platform, Magellan , to discover novel allosteric binding sites on proteins implicated in a disease and to identify proprietary small molecules that bind these sites to modulate protein function and treat the underlying cause of the disease.
Allosteric Binding Site Identification Using the three-dimensional structure of proteins that have been experimentally derived or generated or predictive protein structures from AI-powered databases such as Alphafold, our Magellan platform applies various computational methods and proprietary algorithms to identify and map previously uncharacterized clusters of binding hotspots on the protein surface where a small molecule can potentially bind.
We believe our approach can be significantly less expensive, significantly faster and significantly more effective.
REMOVED
As of February 28, 2025, 27,786,952 shares of the registrant's Common Stock were outstanding.
We believe these results support the continued development of GT-02287 and its potential as a biology-modifying treatment for Parkinson s disease.
In December 2024, we received approval in Australia to initiate a Phase 1b trial for GT-02287 in people with Parkinson s disease with or without the GBA1 mutation.
We are working with local Parkinson s disease (PD) advocacy groups in Australia to support enrollment and expect enrollment to complete in the summer of 2025 with interim data from the study expected by mid-2025.
The Phase 1b open-label trial will assess the safety and tolerability of 13.5 mg/kg/day of GT-02287 for three months in patients with GBA1-PD or idiopathic Parkinson s disease.
Secondary endpoints include pharmacokinetics, GCase modulation, levels of GCase substrates, and other biomarkers in plasma and cerebrospinal fluid.
The primary goal of the Phase 1b trial is to assess the safety and tolerability of GT-02287.
Upon successful completion we expect to begin planning a Phase 2 study during the second half of 2025.
In preparation for the treatment of Parkinson s disease patients, we initiated a chronic (6 months in rodents and 9 months in non-rodents) preclinical toxicity study in July 2024, enabling the conduct of clinical studies in patients with a GT-02287 treatment duration beyond three months.
The 6- and 9-month studies will be completed in the second quarter of 2025 and the third quarter of 2025, respectively.