Bio-Path Holdings is a clinical-stage biotech focused on developing RNAi antisense drug candidates for oncology, with early-stage work in obesity and metabolic diseases. The company has no approved products and generates no revenue. Bio-Path's entire pipeline is built on a single proprietary drug delivery platform called DNAbilize, which uses antisense DNA strands packaged in neutral-charged lipid nanoparticles to block the production of disease-causing proteins. Bio-Path argues the neutral charge distinguishes DNAbilize from competing delivery technologies by reducing toxicity. The lead candidate, prexigebersen, targets the Grb2 signaling protein and is in Phase 2 trials for AML, specifically elderly or unfit patients who cannot tolerate standard chemotherapy. Interim data from the ongoing trial show remission rates of 75% and 55% in newly diagnosed and relapsed/refractory AML cohorts, respectively. A reformulated version, BP1001-A, uses smaller nanoparticles to penetrate solid tumors and is in Phase 1 trials across ovarian, pancreatic, and breast cancers. BP1002 targets Bcl-2 and is in Phase 1 dose-escalation for AML, while BP1003 targets STAT3 in solid tumors and is in IND-enabling studies. Bio-Path's strategy is to advance candidates to clinical proof of concept and then out-license to larger pharma partners for late-stage development. The company funds operations through equity raises and ATM offerings, and was delisted from Nasdaq in early 2025, now trading on the OTCQB.
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