The US Food and Drug Administration (FDA) and the EU’s European Medicines Agency (EMA) have granted Bioxodes’ intracerebral hemorrhagic stroke (ICH) therapy orphan drug designation.
“The granting of Orphan Drug Designation in these two jurisdictions confirms that we are on a relatively rapid and cost-effective path to market for BIOX-101. We are working hard to offer a disruptive new therapy for patients suffering from Intracerebral Hemorrhagic Stroke, an often deadly condition with a poor prognosis, for which no therapeutic is currently available,” said Marc Dechamps, CEO at Bioxodes.
BIOX-101 development plan
Bioxodes is currently evaluating BIOX-101 in a randomized open label Phase IIa ‘proof-of-concept’ trial in ICH patients. The study will enroll 32 patients, with 24 receiving BIOX-101, and 8 standard-of-care treatment. The company plans to “imminently” release the interim results from the first 16 patients.
Furthermore, Bioxodes intends to launch a Series B financing round to fund the ‘potentially registrational’ Phase IIb trial with BIOX-101 for ICH in Q2 2025. The patient recruitment for the Phase IIb trial is planned to start in 2027. Adding, “the Chemistry, Manufacturing and Controls (CMC) process required to produce BIOX-101 for Phase IIb will take approximately a year to complete, and the company is currently ramping up production to this end.”
Last month, the company secured an additional €2.7 million (approx. $2.9 million) in an extended Series A funding round. Bioxodes did not disclose the backers, noting that it was from its existing investors. The Belgian company previously raised €12 million (approx. $13 million) in Series A financing in November 2023.
The company is also planning to file for PRIME status with the EMA this year, and for Fast Track approval at the US FDA later during development of the product. Both these allow for a faster approval process and an increased interaction with the regulatory agencies.
Mechanism of action
BIOX-101 is a recombinant protein designed to inhibit the harmful secondary effects of a hemorrhagic stroke, such as secondary brain ischemia, neuroinflammation and neuronal damage. The therapy has anti-clotting effects as it targets Factors XIa and XIIa of the intrinsic coagulation pathway, without increasing the risk of bleeding.
BIOX-101 also has anti-inflammatory properties, and acts by inhibiting activation of neutrophils and their release of extracellular DNA filaments (called NETs), which can cause excessive inflammation, exacerbating brain damage and disrupting the blood-brain barrier.
ICH is a subtype of stroke where a hematoma is formed within the brain parenchyma with or without blood extension into the ventricles. Non-traumatic ICH comprises 10-15% of all strokes and is associated with high morbidity and mortality.
