GM-1020 · program

Blixeprodil (GM-1020) for Major depressive disorder

Blixeprodil (GM-1020) for MDD. Phase 2a (NCT06309277) completed; positive topline announced 6 Jan 2026: single oral dose gave -6.3 MADRS vs placebo at 24h (p=0.006); 53% remission at Day 42 (multiple-dose arm), sustained through 28-day follow-up. Company intends late-stage studies in 2026.

Development timeline

Phase 2May 2024 – Jan 2026
  1. Positive Phase 2a topline; -6.3 MADRS vs placebo at 24h (p=0.006); 53% Day-42 remission. Late-stage studies planned for 2026.
  2. Phase 2a MDD trial initiated; first subject dosed.

Clinical trials in Major depressive disorder

NCT06309277Phase 2Completedn=46

Two-Part Study of Single and Multiple Oral Doses of GM-1020 in Major Depressive Disorder

Started Feb 2024· Primary completion Mar 2025· 📍 1 site across 1 country (United Kingdom)

metprimaryMADRS change vs placebo, 24h after single 140mg oral dose — -6.3 points (effect size 0.85) (0.006)

Day 8 -5.1 (p=0.076); 53% remission at Day 42 in the multiple-dose arm, sustained through 28-day follow-up.

Formulations

FormulationRouteRegimenPharmacokinetics
Blixeprodil oral
140 mg and 210 mg oral doses studied (Phase 2a: 140 mg single dose in Part A; 140 mg or 210 mg twice weekly for 2 weeks in Part B)
OralTwice weeklyt½ 4.3 h · Tmax 1.5 h · F 60%

Mechanism of action (compound-wide)

Non-competitive NMDA-receptor antagonist binding the PCP/channel site (voltage-dependent block), non-subunit-selective across GluN2A/GluN2B. Low-micromolar affinities.

TargetActionAffinity
NMDA receptorprimaryGRIN1AntagonistKi 3.25 µM

← Full GM-1020 compound page (identity, identifiers, all indications)

Sources

  1. Blixeprodil — Wikipedia
  2. Gilgamesh Pharma Announces Positive Topline Phase 2a Results for Blixeprodil (GM-1020) in Major Depressive Disorder — Gilgamesh / PR Newswire
  3. GM-1020 Phase 2a MDD (NCT06309277) — ClinicalTrials.gov
  4. GM-1020: oral NMDA antagonist with antidepressant-like effects — Molecular Psychiatry (PMC)
  5. prnewswire.com — Reference