Bioxodes SA announced encouraging second interim results from its BIRCH Phase 2a clinical trial of BIOX-101, a novel therapeutic candidate for treating intracerebral hemorrhage (ICH). The Belgian biopharmaceutical company reported that all 23 patients treated with BIOX-101 experienced hematoma volume reduction, with the Data Monitoring Committee unanimously endorsing progression to Phase 2b or Phase 3 efficacy trials.
"These results suggest BIOX-101 may become a disruptive novel therapeutic, offering a long-awaited breakthrough for treatment of stroke, starting with ICH," said Marc Dechamps, Chief Executive Officer of Bioxodes. The company is now in discussions with prospective investors and partners to support a potentially registrational Phase 2b trial.
Clinical Trial Results Show Multiple Benefits
The Phase 2a trial met its primary safety endpoint with no deaths reported after a mean follow-up of 8 months. Key efficacy signals included patients showing a trend toward slower perihematomal edema expansion over 10 days after the initial stroke, with hemorrhage volume decreasing more steadily in the BIOX-101 group compared to standard of care.
Functional outcomes appeared more favorable than those in the standard of care group, with more patients regaining independence on day 90 after BIOX-101 treatment, achieving Modified Rankin Scale scores of 0-2. The body's inflammatory reaction, measured by the Neutrophil-to-Lymphocyte Ratio, showed improved trends in treated patients.
"In this first study of BIOX-101 in patients suffering from acute intracranial hemorrhage the drug appears to be safe in this population," said Prof. Robin Lemmens, Principal Investigator of the BIRCH study and head of the stroke unit at the University Hospital Leuven. "Although this study was not statistically powered to show potential benefit of BIOX-101, initial findings on progression of edema support the further evaluation in larger clinical trials."
Addressing Critical Unmet Medical Need
ICH represents a devastating condition with no approved therapies, accounting for 40% of all stroke-related deaths despite comprising just 15% of stroke cases. Mortality approaches 50% at 30 days, with approximately half of all ICH-related deaths occurring within the first 24 hours. Fewer than 20% of survivors achieve functional independence after six months, often due to secondary damage from untreatable bleeding and associated inflammation.
"An effective therapy like BIOX-101 is urgently needed for the millions of patients struck by ICH each year," said Hans Warrinnier, Chief Medical Officer of Bioxodes. "ICH is a life-threatening disease, and the downstream damage from inflammation and secondary ischemia can be even more deadly or debilitating than the initial event."
Novel Mechanism of Action
BIOX-101 is a proprietary recombinant version of a small protein found in tick saliva (Ixodes ricinus). Unlike currently marketed anticoagulants that cannot be used to treat ICH because they increase bleeding risk, BIOX-101 reduces clotting without increasing bleeding by targeting Factors XIa and XIIa of the intrinsic coagulation pathway.
The therapeutic candidate also exerts anti-inflammatory effects by inhibiting neutrophil activation and their release of neutrophil extracellular traps (NETs), which can cause excessive inflammation and exacerbate brain damage. "We believe those synergistic mechanisms explain the favorable signals observed in the study," Warrinnier explained.
Regulatory Pathway and Timeline
Bioxodes received Orphan Drug Designation for BIOX-101 in both the U.S. and Europe in March 2025. The company plans to file for PRIME status with the EMA later this year and intends to pursue Fast Track designation with the FDA. The planned Phase 2b trial could be sufficient to submit BIOX-101 for conditional marketing authorizations in both regions.
If initiated by early 2027, Bioxodes believes BIOX-101 could become available to patients as early as 2030. The company also plans to develop BIOX-101 for acute ischemic stroke and an undisclosed indication, while further characterizing its anti-inflammatory mechanism and developing novel drug candidates targeting NETs for thrombo-inflammatory diseases.
The Data Monitoring Committee recommended maintaining similar subject eligibility criteria for future trials and suggested including perihematoma edema as an efficacy endpoint. Bioxodes stopped recruiting for the Phase 2a trial after treating 23 patients to accelerate the launch of the Phase 2b efficacy trial.
