A Randomized Phase 2 KINETIC Trial Evaluating SAGE-324/BIIB124 in Individuals with Essential Tremor

Area of Science:

• Neuroscience
• Pharmacology

Background:

• SAGE-324/BIIB124 is an investigational positive allosteric modulator targeting GABA_A receptors.
• Essential tremor (ET) is a neurological disorder characterized by involuntary rhythmic shaking.

Purpose of the Study:

• To evaluate the efficacy and safety of SAGE-324/BIIB124 in individuals with essential tremor (ET).
• The KINETIC study (NCT04305275) was a phase 2, double-blind, randomized, placebo-controlled trial.

Main Methods:

• 69 participants aged 18-80 years were randomized to receive oral SAGE-324/BIIB124 (60 mg) or placebo once daily for 28 days.
• The primary endpoint was the change from baseline in The Essential Tremor Rating Assessment Scale-Performance Subscale (TETRAS-PS) Item 4 (upper-limb tremor) at day 29.

Main Results:

• SAGE-324/BIIB124 demonstrated a statistically significant reduction in upper-limb tremor compared to placebo at day 29 (P=0.0491).
• The most frequent treatment-emergent adverse events were somnolence, dizziness, fatigue, and balance disorder.

Conclusions:

• The study results support the continued development of SAGE-324/BIIB124 as a potential therapeutic option for essential tremor.
• Further research is warranted to fully establish the safety and efficacy profile of SAGE-324/BIIB124 in ET patients.