Gain Therapeutics (GANX) Corporate presentation summary

Platform and pipeline

• Magellan combines AI-supported structural biology, proprietary algorithms and physics-based models to identify allosteric binding sites; pipeline spans Parkinson’s, Gaucher’s, dementia with Lewy bodies, Alzheimer’s and lysosomal storage disorders.

• Rexaceract targets GCase; other pipeline programs include GT-04686 and undisclosed candidates targeting GCase, GALC and GLB1.

• Initial therapeutic indication selection for GT-04686 targeted for Q4 2026.

Rexaceract rationale and preclinical evidence

• Rexaceract binds an allosteric site and chaperones GCase to lysosomes and mitochondria; preclinical models showed restored GCase function and improved disease pathways and neuronal survival in GBA1 and idiopathic PD.

• Preclinical animal models of GBA1-PD and idiopathic PD showed improved motor performance with rexaceract, including after treatment washout.

• The presentation estimates US market potential of $4B for Parkinson’s disease and $3B for GBA1-PD; existing therapies address symptoms without slowing progression.

Clinical evidence and safety

• In Phase 1b, CSF GluSph decreased from pretreatment to Day 90 (n=13; p<0.05); participants with elevated baseline CSF GluSph had an average 81% reduction.

• After 12 months of dosing, 12 evaluable participants showed no clinically meaningful progression on MDS-UPDRS motor scores; both high- and low-baseline CSF GluSph groups showed stabilization by Day 360.

• In healthy volunteers, single and multiple doses were generally well tolerated, with no SAEs or Grade 3 adverse events; therapeutic exposure and CNS exposure were achieved.

• Five of six participants in MAD Cohort 4 had increased dried-blood-spot GCase activity; the reported increase was 53% by Day 14 (p<0.001), with no increase in placebo subjects.