BioTheryX Receives IND Clearance from FDA to Proceed with Phase 1 Study of BTX-1188, a Molecular Glue, for the Treatment of Hematologic and Solid Malignancies

Alyssia Watkin BioTheryX, Lumira News, Portfolio News

Enrollment for Phase 1 Clinical Trial to Commence by End of 2021

BioTheryX, Inc., a clinical-stage company focused on targeted protein degradation to create life-saving medicines, today announced that the U.S. Food and Drug Administration (FDA) cleared the company to proceed with a Phase 1 clinical trial of BTX-1188 in hematologic and solid malignancies. 

BTX-1188 is a novel oral small molecule cereblon binder that has immunomodulatory properties and promotes the degradation of a neosubstrate G1 to S phase transition 1 (GSPT1), a translation termination factor, as well as IKZF1/3, zinc finger transcription factors important in hematological function. By degrading multiple proteins including GSPT1 and IKZF1/3, BTX-1188’s profile is expected to be differentiated from protein degraders that exclusively target GSPT1. BTX-1188 has demonstrated promising preclinical activity against a variety of leukemias and solid tumors.

“The FDA clearance to begin our Phase 1 study for BTX-1188 is a major milestone for BioTheryX, which represents our first molecular glue to enter clinical development, and the second IND clearance for our pipeline,” said Zung Thai, M.D., Ph.D., Chief Medical Officer of BioTheryX. “We plan to study BTX-1188 in patients with hematological and solid malignancies who have limited treatment options and for whom new therapies are desperately needed. We expect to initiate patient enrollment in the Phase I study by year end.”

The Phase I clinical trial is designed to assess the safety and tolerability of BTX-1188 and to determine the recommended Phase 2 dose. Following determination of the recommended Phase 2 dose, the company plans to further evaluate the safety and efficacy of BTX-1188 in expansion cohorts in both hematological and solid tumor malignancies.

About BioTheryX, Inc.

BioTheryX is a clinical-stage biopharmaceutical company focused on restoring protein homeostasis, including protein degradation and modulation, and multi-kinase inhibition to develop treatments intended to extend and improve the quality-of-life of patients with cancer and other diseases. Our principal technology platform centers on targeted protein degradation, ‘molecular glues’ and PROTACs, that enable the design of small molecules to regulate protein equilibrium. This technology is designed to utilize the body’s own protein disposal system to selectively degrade and remove disease-causing proteins. It has potential applicability for a broad range of diseases, including targets that have to date been considered ‘undruggable’, and BioTheryX is initially focused on treating oncology indications with high unmet need. 

For more information, please visit www.biotheryx.com and engage on LinkedIn.