BioTheryX, Inc., a clinical-stage company focused on developing targeted protein degraders to create life-saving medicines, today announced dosing of the first patient in a new, first-in human Phase 1 study investigating its lead candidate BTX-1188, an oral, dual target protein degrader specifically engineered to degrade GSPT1 and IKZF1/3, in patients with advanced hematologic and solid tumor malignancies.
“BTX-1188 has demonstrated rapid, deep and durable degradation of both GSPT1 and IKZF1/3 at low nanomolar concentrations in preclinical models, and we are delighted to make this exciting advancement into the clinic,” said Zung Thai, M.D., Ph.D., Chief Medical Officer of BioTheryX. “We are grateful to our clinical trial site partners for initiating this study in these challenging times and thank them for their commitment to investigating potential new therapeutic options for people facing difficult-to-treat cancers.”
The Phase 1 clinical trial is a multicenter, open label, non-randomized, sequential dose escalation study to assess the safety, pharmacokinetics and preliminary efficacy of BTX-1188 in patients with acute myeloid leukemia, advanced lymphoid and solid tumors who have failed standard therapy. The primary objective of the Phase 1 study is to determine a recommended Phase 2 dose, which will then be carried forward into expansion cohorts where the safety and efficacy of BTX-1188 will be evaluated in both hematologic and solid tumor malignancies. More information can be found on clinicaltrials.gov (NCT05144334).
“BTX-1188 is one of many potential best-in-class protein degrader candidates our team has designed and developed leveraging our distinctive PRODEGY targeted protein degradation platform to provide new treatment approaches for people living with cancer and other diseases,” said Philippe Drouet, President and Chief Executive Officer of BioTheryX. “With our clinical trial site partners, we are working as expeditiously as possible to study BTX-1188 and validate the potential of our platform.”
BTX-1188 is the first orally bioavailable, rationally designed dual target protein degrader, engineered to specifically degrade validated oncology targets GSPT1 and IKZF1/3. By degrading GSPT1 and IKZF1/3, BTX-1188’s profile is expected to be differentiated from protein degraders that exclusively target GSPT1. Preclinical data for BTX-1188 has shown rapid, deep and durable degradation of these targets at low nanomolar concentrations. Acute myeloid leukemia (AML) xenograft experiments have also shown significant decreases in tumor volume and increased survival compared to controls and other degraders of GSPT1. BioTheryX believes BTX-1188 has broad potential applicability in both hematologic and solid tumor malignancies and is currently studying the molecule in both settings as part of a Phase 1 clinical study.