Bio-Path Holdings reported significant clinical progress across its cancer drug pipeline during a recent corporate update, highlighting promising patient responses and advancing multiple therapeutic programs targeting blood cancers and solid tumors.
AML Treatment Shows Durable Responses
The company's Phase 2 clinical trial for prexigebersen in acute myeloid leukemia (AML) has demonstrated notable treatment durability in two patients who have each received over 15 treatment cycles and remain in complete remission. The trial comprises three separate cohorts treating different patient populations with combinations of prexigebersen, decitabine, and venetoclax.
The first two cohorts are evaluating the triple combination in untreated AML patients and relapsed/refractory AML patients, respectively. The third cohort treats relapsed/refractory AML patients who are venetoclax-resistant or intolerant with a two-drug combination of prexigebersen and decitabine. These older patients, unable to receive intensive chemotherapy due to challenging side effects, typically face suboptimal outcomes with median survival of only 5 to 10 months.
Bio-Path expects to complete Cohort 2 and conduct an interim analysis for Cohort 3 during 2025, with plans to utilize an advisory panel of AML experts to assist in designing final clinical development plans through potential FDA approval.
Solid Tumor Program Advances with Tumor Reduction
The Phase 1/1b clinical trial of BP1001-A in advanced solid tumors has progressed to the second dose level, showing encouraging results. BP1001-A incorporates the same drug substance as prexigebersen but features a modified formulation designed to enhance nanoparticle properties.
A patient in the second dose cohort has demonstrated a positive response, experiencing a 15% reduction in her primary tumor through ten cycles of treatment. This patient had previously failed extensive chemotherapy and surgical treatment for gynecologic cancer and continues to maintain rigorous exercise and improved quality of life.
The study targets patients with advanced or recurrent solid tumors, including ovarian, uterine, pancreatic, and breast cancers. Following completion of three monotherapy dose cohorts, the Phase 1b portion will assess BP1001-A in combination with paclitaxel for recurrent ovarian or endometrial tumors, with additional combination studies planned for pancreatic and breast cancers.
Venetoclax-Resistant AML Program Shows Promise
Bio-Path's BP1002 program for relapsed/refractory AML patients, including those resistant to venetoclax, has completed dose escalation through 60 mg/m2 without dose-limiting toxicities. The fourth dosing cohort of 90 mg/m2 is now open for enrollment.
BP1002 targets the Bcl-2 protein, which drives cell survival in up to 60% of all cancers. Unlike venetoclax, which blocks Bcl-2 activity, BP1002 prevents the cell's ability to produce Bcl-2, potentially benefiting venetoclax-resistant patients. AML patients who fail frontline venetoclax-based therapy face very poor prognosis with median overall survival of less than three months.
Enrollment in the third dosing cohort closed faster than expected, which Bio-Path believes reflects the significant need for additional treatment options in this patient population.
Expanding into Metabolic Disease
Bio-Path is extending its pipeline beyond oncology into obesity treatment for Type 2 diabetes patients. Preclinical studies have shown that BP1001-A downregulates Grb2 expression to increase insulin sensitivity and help lower blood glucose levels.
Recent milestone achievements from preclinical studies demonstrated that BP1001-A rescues decreased AKT activity in liver cells and prevents insulin resistance, confirming its potential for treating obesity and related metabolic diseases in Type 2 diabetes patients. The company expects to complete preclinical testing and file an Investigational New Drug (IND) application in 2025.
Technology Platform and Business Model
Bio-Path's DNAbilize® liposomal delivery and antisense technology platform generates targeted nucleic acid drugs administered through simple intravenous infusion. The company's business model centers on generating new drug candidates from this platform and licensing them for final development and commercialization with partners.
"The clinical progress we are making across our pipeline is bringing us one step closer to our goal of delivering a better path for oncologic and cardiometabolic patients," said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings.
The company maintains a robust intellectual property portfolio with seven patents issued in the U.S., 61 foreign patents issued across 26 countries, and additional applications pending in multiple jurisdictions. These composition of matter patents protect the company's proprietary products and allow application of core technology to new protein targets with new 20-year patent protection.
