• The primary objective of the study is to determine whether the combination of prexigebersen and LDAC provides greater efficacy than would be expected with LDAC alone in this de novo patient population.  

• Prexigebersen is also in ongoing pre-clinical testing in solid tumors such as those caused by triple negative breast cancer (TNBC), inflammatory breast cancer (IBC), and ovarian cancer, and in the autoimmune indication, systemic lupus erythematosus.

Bio-Path Holdings, Inc., (NASDAQ: BPTH) announced on 8/27/18 that it has commenced Stage 2 of the company’s Phase 2 trial of prexigebersen in acute myeloid leukemia.

The open-label Phase 2 study is evaluating the efficacy and safety of prexigebersen in conjunction with LDAC, a therapeutic regimen well established in treatment of AML patients who cannot or elect not to be treated with more intensive chemotherapy.  The primary objective of the study is to determine whether the combination of prexigebersen and LDAC provides greater efficacy than would be expected with LDAC alone in this de novo patient population.

“We are delighted to announce the expansion of our ongoing Phase 2 clinical trial of prexigebersen for the treatment of acute myeloid leukemia using a dosing schedule that administers a greater amount of prexigebersen to the patient prior to commencing LDAC dosing than in the first part of the trial. Based on compelling new data, we are also including a cohort of patients who will be treated with a combination of prexigebersen and decitabine,” said Peter Nielsen, President and Chief Executive Officer of Bio-Path. “Results from the planned interim analysis of the first part of this Phase 2 study were particularly encouraging, with 47% of treated patients demonstrating a response.  Consequently, we remain enthusiastic about prexigebersen’s potential and believe these protocol changes will optimize the drug’s impact in AML cancer patients with high unmet need.”

Based on recommendations from the study’s principal investigators, the Company amended the study’s protocol to change the dosing schedule in Stage 2 to that used in the Phase 1b study in relapsed and refractory AML patients as announced in April 2018. In the Phase 1b study, a greater amount of prexigebersen was administered prior to LDAC treatment starting at day 10 versus LDAC treatment starting on day four, as was the case in Stage 1 of the current Phase 2 study.  Importantly, Stage 2 of the study includes a cohort of patients treated in combination with decitabine based on relatively new and positive data with this compound.  Bio-Path plans to perform an interim analysis of each cohort once approximately 19 evaluable patients are reached in the cohort.

A previously announced interim analysis of Stage 1 of the study was performed on 17 evaluable patients, with four patients achieving complete responses and four patients achieving stable disease, including one patient achieving a morphologic leukemia free state and one patient who showed significantly reduced bone marrow blasts.  In total, 47% of the evaluable patients showed some form of response, including stable disease, to the combination treatment. The average patient in Stage 1 of the study was 73.5 years of age.

Prexigebersen [Liposomal Grb-2 (Growth factor Receptor Bound protein-2)] suppresses the expression of Grb-2, which is an adaptor protein that links tyrosine kinases with their downstream signaling molecules. Tyrosine kinases function as "on" "off" switches in many cellular functions so interrupting their signaling may have an inhibitory effect on rapidly-growing cancer cells.
In addition to AML trials, prexigebersen is in trials for CML.

Prexigebersen is also in ongoing pre-clinical testing in solid tumors such as those caused by triple negative breast cancer (TNBC), inflammatory breast cancer (IBC), and ovarian cancer, and in the autoimmune indication, systemic lupus erythematosus.

Bio-Path is a nano-cap company with a $16.7M market cap. The company's shares are up 9.9% as of this writing.