On August 26, 2019 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, reported patient dosing in Bio-Path’s amended Phase 2 trial of prexigebersen for the treatment of acute myeloid leukemia (AML), as announced in March 2019 (Press release, Bio-Path Holdings, AUG 26, 2019, View Source [SID1234539010]).
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The key change in the amended Phase 2 study is the inclusion of patients with high risk myelodysplastic syndrome (MDS) and refractory/relapsed AML patients. The restructured Phase 2 clinical trial has two cohorts of patients. The first being untreated AML patients as existed in the pre-amended trial but with the addition of high risk MDS patients, and a second cohort comprised of refractory/relapsed AML patients and high risk MDS patients.
The amended Phase 2 study will continue evaluating the safety of prexigebersen in combination with decitabine in both cohorts of patients at a dose of 60 mg/m2 in combination with decitabine. The study will include a total of six evaluable patients for a safety assessment of prexigebersen and decitabine. To date, the Company has enrolled five evaluable patients: three untreated AML patients in who received therapy prior to amending the trial, and two patients who are now being treated under the amended Phase 2 trial. Assuming a successful completion of this safety assessment, the study will then modify testing of both cohorts of patients to add venetoclax to the prexigebersen/decitabine combination treatment.
After a six-patient safety assessment of the prexigebersen/decitabine/venetoclax combination, the Company intends to commence the efficacy segment of this trial. It is anticipated that each cohort will include an interim assessment of 19 evaluable patients that would assess whether the treatment efficacy of the combination of prexigebersen/decitabine/ venetoclax exceeds the efficacy of current standard-of-care therapy with statistical significance. Upon such favorable data, Bio-Path would petition the U.S. Food and Drug Administration (FDA) for accelerated approval. The efficacy segment of the trial is expected to be conducted at up to ten clinical sites in the United States. Moving forward, the Company intends to evaluate potential clinical sites in Europe with an emphasis on patient accruals.
"We are excited to have dosed the first patient in our amended protocol of this important clinical trial, confident that the changes made to the protocol, along with the inclusion of MDS patients, will further demonstrate the potential of prexigebersen in a number of cancer indications for which there are limited treatment options," said Peter Nielsen, President and Chief Executive Officer of Bio-Path. "We are encouraged about the outcome for this study, as preclinical work showed the benefit of prexigebersen in combination with decitabine and venetoclax. We look forward to advancing this development program with the goal of bringing new therapies to cancer patients in need."