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Drug Development.

Howard H Feldman1,2,3, Karen Messer1,2, Jing Zhang2,1

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Varoglutamstat was safe for early Alzheimer's disease (AD) patients but did not show significant efficacy benefits in this trial. Further biomarker and PK/PD results are pending.

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Area of Science:

  • Neuroscience
  • Pharmacology

Background:

  • Varoglutamstat (PQ912) is an oral glutaminyl cyclase inhibitor targeting amyloid-beta (Aβ) and CCL2 formation.
  • Preclinical and Phase 1 data supported its evaluation in early Alzheimer's disease (AD).

Purpose of the Study:

  • To determine the optimal safe and well-tolerated dose of varoglutamstat for early AD.
  • To assess the drug's safety and efficacy over 72 weeks in a Phase 2 trial.

Main Methods:

  • Biomarker-confirmed early AD patients were randomized to varoglutamstat or placebo.
  • A sequential design tested descending doses with futility analysis at 24 weeks.
  • Primary endpoint was CDR-sum-of-boxes (CDR-SB); secondary endpoints included cognitive and functional measures.

Main Results:

  • The 600mg dose was selected based on safety in Phase 2A; however, the trial was terminated prematurely.
  • No significant differences were observed between varoglutamstat and placebo for CDR-SB or other efficacy endpoints at 72 weeks.
  • Varoglutamstat was generally safe and well-tolerated, though treatment discontinuations due to adverse events were higher than placebo.

Conclusions:

  • Varoglutamstat demonstrated a favorable safety profile in early AD patients.
  • The study did not show significant treatment benefits, potentially due to limited sample size.
  • Pending biomarker and pharmacokinetic/pharmacodynamic (PK/PD) data may offer further insights.