Bizaxofusp

Summary

Bizaxofusp (formerly MDNA55) is an investigational EGF-targeted immunotoxin designed for convection-enhanced delivery (CED) directly into brain tumors, particularly glioblastoma multiforme (GBM). It selectively targets EGFR-overexpressing tumor cells and delivers a potent cytotoxic payload. It is in late-stage clinical development for recurrent GBM.

Mechanism of Action

Bizaxofusp is a recombinant fusion protein consisting of human epidermal growth factor (EGF) fused to a truncated form of Pseudomonas exotoxin A (PE38). It binds to EGF receptor (EGFR/ErbB1) overexpressed on tumor cells, is internalized, and the exotoxin moiety inhibits protein synthesis via ADP-ribosylation of elongation factor 2 (EF-2), leading to tumor cell apoptosis.

Routes of Administration

Goals & Uses

• Targeting EGFR-overexpressing solid tumorsOncologyLow
• Treatment of recurrent glioblastoma multiforme (GBM)OncologyModerate
• Tumor debulking prior to additional therapyOncology Combination StrategyLow

Contraindications

• Hypersensitivity to Pseudomonas exotoxin componentsImmunologicHigh
• Coagulopathy or bleeding diathesisHematologicModerate
• Active systemic infectionInfectiousHigh
• Poor performance status precluding neurosurgeryClinicalModerate

Adverse Effects

• HeadacheNeurologicCommonPain in the head or upper neck
• SeizuresNeurologicalUncommon
• FatigueGeneralCommonLow energy or tiredness
• Intracranial hemorrhageNeurologicalRare
• Cerebral edemaNeurologicalCommon
• Neurological deficits (focal)NeurologicalCommon

Drug Interactions

• Immunosuppressive agentsLow
• Corticosteroids (e.g., dexamethasone)Low

Population Constraints

• EGFR-negative tumor patientsBiomarker / Tumor BiologyRelative
• Pediatric patientsAgeRelative
• Pregnant or breastfeeding womenReproductiveRelative
• Patients with prior severe neurological compromiseClinicalRelative

Regulatory Status

• European UnionInvestigationalNot approved in the European Union; investigational use only.
• United StatesInvestigationalFDA Breakthrough Therapy Designation granted for recurrent glioblastoma. Not approved; Phase 2/3 clinical trials conducted.
• United KingdomInvestigationalNot approved by MHRA; investigational status only.

Bizaxofusp received FDA Breakthrough Therapy Designation for recurrent glioblastoma. It is not currently approved in any jurisdiction. Phase 2/3 trials have been conducted. Previously developed by Medicenna Therapeutics.

Evidence & Sources

No sources recorded yet.