Bio-Path Shares Phase 2 Prexigebersen AML Study Data at EHA Congress

Bio-Path Holdings, a biotechnology firm specializing in targeted nucleic acid cancer treatments, revealed promising interim results from its Phase 2 trial of prexigebersen (BP1001) combined with decitabine and venetoclax for acute myeloid leukemia (AML). The data was shared during the 2024 European Hematology Association (EHA) Congress in Madrid, Spain, by Dr. Jorge Cortes from the Georgia Cancer Center.

The study highlighted that prexigebersen, used in combination with other drugs, is not only well-tolerated but also shows significant clinical efficacy. Peter Nielsen, Bio-Path’s CEO, emphasized the importance of presenting these findings to a European audience, given the ongoing trials in the U.S. The aim is to broaden awareness among global oncology experts about prexigebersen’s potential in AML treatment.

Key findings were presented from two patient cohorts. Cohort 1 included 31 newly diagnosed AML patients, out of which 20 were evaluable. These patients, aged between 69 and 84, were treated with at least one cycle of the drug combination. The results showed that 75% of the evaluable patients achieved complete remission (CR), CR with partial recovery (CRh), or CR with incomplete hematologic recovery (CRi). Additionally, two patients achieved partial remission (PR) and two others had stable disease (SD).

Cohort 2 involved 38 relapsed or refractory AML patients, with 23 being evaluable. These patients, aged between 24 and 89, also underwent at least one treatment cycle. In this group, 55% of evaluable patients achieved CR/CRi/CRh, while one patient had partial remission, and eight patients achieved stable disease.

Adverse events among these patients were consistent with expectations for treatments involving decitabine, venetoclax, and AML itself. Common side effects included fatigue (72%), anemia (60%), and neutropenia (49%). Severe adverse events recorded were febrile neutropenia (26%) and sepsis (5%).

Encouraged by these promising interim results, Bio-Path plans to continue enrolling patients for both cohorts, aiming for up to 98 evaluable patients in Cohort 1 and 54 in Cohort 2.

Bio-Path Holdings is advancing its DNAbilize® technology, a novel liposomal delivery mechanism for RNAi nanoparticle drugs. Their lead product, prexigebersen, is undergoing Phase 2 trials for blood cancers, while a modified version, BP1001-A, is in Phase 1/1b trials for solid tumors. Another product, BP1002, targets the Bcl-2 protein and is being tested for blood cancers and solid tumors. The company is also preparing to file an IND for BP1003, a STAT3 antisense oligodeoxynucleotide for inhibiting STAT3.

This interim analysis underscores Bio-Path's progress in developing effective, targeted treatments for cancer, particularly AML, and highlights the potential of prexigebersen as a viable therapeutic option. With ongoing trials and further patient enrollment, the company aims to substantiate these promising results and contribute significantly to the field of oncology.