VarmX Receives Rare Phase 1 Waiver in Japan for VMX-C001, Advances Directly to Global Phase 3

VarmX, a biotech company focused on innovative therapies for bypassing factor Xa inhibitors (FXa DOACs) and inherited coagulation disorders, announced that Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) has waived the requirement for a local Phase 1 trial of its lead candidate VMX-C001.

This rare exemption allows Japanese patients to be included directly in the ongoing global Phase 3 EquilibriX-S study, evaluating VMX-C001 as a bypass agent to restore blood coagulation in patients receiving FXa DOACs who require urgent surgery.

Regulatory Milestone

• The PMDA recognized the robust global clinical dataset supporting VMX-C001 and the unmet medical need for therapies that enable surgery in patients on FXa DOACs.
• Typically, foreign drug candidates must undergo a Japanese Phase 1 (ethnic bridging) trial; full waivers are exceptionally uncommon.
• The PMDA has also committed to early interactions, potentially supporting an accelerated submission for approval in Japan.

Recent U.S. Progress

• July 2025: FDA cleared the Investigational New Drug (IND) application for VMX-C001.
• August 2025: FDA granted Fast Track Designation to VMX-C001.
• These milestones support initiation of EquilibriX-S, the first global Phase 3 trial of an FXa DOAC bypassing agent.
• VarmX also entered a strategic collaboration and option agreement with CSL to advance the program.

Clinical Need

• By 2030, ~30 million patients across the U.S., Europe, and Japan are expected to receive FXa DOACs for chronic anticoagulation (stroke prevention, DVT prevention).
• Each week, >30,000 patients experience severe bleeding or require emergency surgery where DOAC-related anticoagulation poses life-threatening risk.

Leadership Commentary

“Being granted a Phase 1 waiver by the PMDA is a rare and important milestone,It allows Japan to participate in our global Phase 3 program, reflecting both the urgency of the unmet need and the strength of our clinical data package. This decision de-risks our development in Japan and enables potential patient access in parallel with the U.S. and Europe.”

• John Glasspool, CEO of VarmX.

About VMX-C001

• Mechanism: Modified human factor X protein, insensitive to FXa DOACs, bypassing their anticoagulant effect and restoring coagulation.
• Advantages:
  - Universal dosing across FXa DOACs
  - Rapid, easy administration
  - Compatibility with anticoagulants such as heparin
  - No added thrombotic risk
• Status: Lead program in global Phase 3 development (EquilibriX-S).

About VarmX

Founded in 2016 as a spin-off from Leiden University Medical Center (LUMC), VarmX is advancing therapies for anticoagulation reversal and coagulation disorders. The company’s lead compound, VMX-C001, is a recombinant modified blood factor X, developed under the leadership of Professor Pieter Reitsma, a world expert in hemostasis and thrombosis.

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