Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilizeĀ®Ā antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, today

Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize® antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, today announces a poster highlighting the safety and preliminary efficacy data of its Phase 2 study of prexigebersen (BP1001) was presented at the 2021 American Society of Hematology (ASH) Annual Meeting, taking place from December 11-14, 2021.
 

The poster, titled, "Safety and Efficacy of Lower Intensity Induction Therapy with Intravenous Prexigebersen (BP1001) in Patients with High-Risk and Relapsed/Refractory Acute Myeloid Leukemia (AML)," was presented by Maro Ohanian, D.O., Department of Leukemia, University of Texas MD Anderson Cancer Center. The poster described the safety and preliminary efficacy of Bio-Path's lead drug candidate, prexigebersen (liposomal Grb2 antisense), from a Phase 2 study in combination with decitabine or decitabine plus venetoclax as a potential treatment for patients diagnosed with AML.

"Our study's novel clinical trial protocol was designed to adjust for the inclusion of newly approved therapies that we believed would be enhanced from combination with our DNAbilize technology," said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings. "This robust design provided for early evaluation of various combinations of prexigebersen to optimize the best potential outcomes for patients and allows for the most expeditious pathway to market. We are delighted to have these safety and preliminary efficacy data presented among an audience dedicated to bringing new cancer treatments to patients."

The Phase 2 clinical trial is a multi-center, open label study with three patient cohorts:

  • Untreated AML patients treated with prexigebersen in combination with decitabine plus venetoclax;
  • Refractory/relapsed AML patients treated with prexigebersen in combination with decitabine plus venetoclax; and
  • Refractory/relapsed AML patients, resistant or intolerant to venetoclax, treated with prexigebersen in combination with decitabine.
     

The primary objective of the study is to assess whether prexigebersen in combination with decitabine plus venetoclax provides higher response rates than decitabine plus venetoclax in AML patients and whether prexigebersen in combination with decitabine provides higher response rates than decitabine alone in AML patients. The study was amended to obtain safety run-in data for patients treated with prexigebersen + decitabine first before proceeding to safety run-in for patients treated with prexigebersen + decitabine + venetoclax. In the Poster we report the safety run-in and efficacy data of AML patients treated with prexigebersen + decitabine or prexigebersen + decitabine + venetoclax.

Data Highlights

Six patients, including four patients (67%) with de novo AML and two secondary AML patients (33%), were treated with at least one cycle of prexigebersen + decitabine combination therapy. All patients in this cohort (median age 72 years) were considered high risk due to having either adverse risk status by ELN (n=5) or treated secondary AML (n=1). Data showed that adverse events (AEs) were generally consistent with those expected with decitabine and/or AML. Three of the six patients (50%) had a response, including two de novo patients (33%) who achieved a CRi (complete remission with incomplete blood count recovery) and one secondary AML patient (17%) who achieved a partial remission (PR). Patients with these conditions generally have a less than 20% CR/CRi response rate.

Six patients were treated with at least one cycle of prexigebersen + decitabine + venetoclax combination therapy. Of the six patients, two (33%) had de novo AML and four (67%) were relapsed/refractory. All patients in this cohort were adverse-risk by ELN (n=2) or relapsed/refractory (n=4). AEs were generally consistent with decitabine and venetoclax treatment and/or for AML. Four patients (67%) achieved a complete remission (CR)/CRi/morphological leukemia free state (MLFS) (n=1/2/1) and one (17%) achieved a PR. Of these five patients, three were relapsed/refractory (75% of relapsed/refractory patients) (1 CR/1 CRi/1 MLFS) and two were de novo (1 CRi/1 PR) (100% of the de novo patients). CR rates to combination treatment with decitabine and venetoclax for relapsed/refractory AML patients are 42-52%1,2 and 0-39%1,2 for relapsed/refractory secondary AML patients.

The preliminary efficacy data are compelling and show that prexigebersen -based combination therapy was not only safely administered to high-risk and relapsed/refractory AML patients considered unsuitable for standard chemotherapy, but also demonstrated encouraging efficacy signals. This is particularly encouraging as relapsed/refractory patients are a challenging population in which current treatment options are suboptimal.