Turning a Lysosomal Protein into a Drug: BXQ-350

Active agent is the Lysosomal Protein Saposin C Fusogenic to Phosphatidylserine

Sphingolipid activator protein, SapC

Sphingolipid activator protein, SapC

  • 80 amino acids, 9kDA
  • Fermentation and Purification – rDNA, e. Coli
  • Sphingolipids – contribute to  regulation of cell membrane bilayers
  • Bioactive sphingolipids (like ceramide) involved in regulation of cancer pathogenesis and therapy
Healthy and tumor cells

Lysosomes exists as a crucial regulator of cellular function and central to its own function are the Saposins —  four proteins that regulate lysosomal enzymes which are responsible for maintaining homeostasis in the cell.

Bexion’s lead compound, BXQ-350, contains one of these Saposins—Saposin C—embedded in a phospholipid, catalyzes activity that can restore the dysfunction of numerous disease pathologies creating the potential to treat a broad spectrum of diseases.

The pursuit of our first clinical indication for BXQ-350 has been in solid tumors as that is where the anti-cancer activity was first demonstrated in -vitro by a researcher exploring Saposin C’s role in lysosomal storage diseases. Additional indications in DIPG/DMG and newly diagnosed CRC/GBM patients are also being targeted due to the tumor agnostic properties of BXQ-350. Several non-oncology indications with significant market potential including Neuropathy are also part of Bexion’s clinical development plan.