Bio-Path Holdings, Inc. (NASDAQ:BPTH) is a clinical and preclinical stage oncology-focused RNAi nanoparticle drug development company utilizing a novel technology that achieves systemic delivery for target-specific protein inhibition. The company's drug delivery and antisense technology, called DNAbilize®, is a platform that uses P-ethoxy, a deoxyribonucleic acid (DNA) backbone modification, incorporated inside of a lipid bilayer having neutral charge. This combination allows for high efficiency loading of antisense DNA into non-toxic, cell-membrane-like structures for delivery of the antisense drug substance into cells.
Business Overview
Using DNAbilize® as a platform for drug development and manufacturing, Bio-Path currently has four antisense drug candidates in development to treat at least five different cancer disease indications. The company's lead drug candidate, prexigebersen, targets growth factor receptor-bound protein 2 (Grb2) and is currently in a Phase 2 clinical trial for the treatment of acute myeloid leukemia (AML). Bio-Path's second drug candidate, Liposomal Bcl-2 (BP1002), targets the Bcl-2 protein and is in Phase 1 and Phase 1/1b clinical trials for the treatment of refractory/relapsed lymphoma, chronic lymphocytic leukemia (CLL), and AML. The company's third drug candidate, Liposomal STAT3 (BP1003), targets the STAT3 protein and is currently in IND-enabling studies as a potential treatment for pancreatic cancer, non-small cell lung cancer (NSCLC), and AML. Bio-Path's fourth drug candidate, BP1001-A, is a modified product from prexigebersen with enhanced nanoparticle properties and is in a Phase 1/1b clinical trial for the treatment of advanced or recurrent solid tumors.
Financials
In its most recent fiscal year ended December 31, 2023, Bio-Path reported an annual net loss of $16.1 million, no revenue, an annual operating cash flow of -$11.5 million, and an annual free cash flow of -$11.5 million. For the first quarter of 2024, the company reported a net loss of $3.2 million, no revenue, and net cash used in operating activities of $1.0 million.
Recent Developments
Despite the lack of significant revenue to date, Bio-Path has made important advancements across its clinical pipeline, corporate initiatives, and financial position. The company recently announced positive interim results from Stage 2 of its Phase 2 clinical trial of prexigebersen for the treatment of AML in combination with frontline therapy decitabine and venetoclax. The data showed that prexigebersen-based combination therapy was not only safely administered to high-risk newly diagnosed and refractory/relapsed AML patients, but also demonstrated efficacy signals better than current therapies. Based on these results, Bio-Path plans to pursue FDA expedited programs for Fast Track designation for its prexigebersen AML treatment.
In the BP1002 program, Bio-Path has completed the second dose cohort of the dose escalation portion of its Phase 1/1b clinical trial in refractory/relapsed AML, including venetoclax-resistant patients. The company also announced the completion of the first dose cohort of the dose escalation portion of its Phase 1 clinical trial of BP1002 for the treatment of refractory/relapsed lymphoma and CLL. With BP1001-A, Bio-Path has completed the first cohort of the dose escalation portion of the Phase 1/1b clinical trial in patients with advanced or recurrent solid tumors.
Regarding the BP1003 program, Bio-Path has identified a method for oligo detection in plasma that will enable the company to complete final safety testing needed to finalize an investigational new drug (IND) application for submission to the FDA. The company is particularly excited to launch its first-in-human validation of this cutting-edge therapy in the challenging cancer indication of pancreatic cancer, which has limited treatment options.
Liquidity
Bio-Path has also strengthened its balance sheet in recent weeks, raising $1.2 million through a registered direct offering and $2.3 million through its at-the-market offering agreement. These additional funds provide the financial underpinning for the company to execute its clinical development plan.
Intellectual Property
In terms of the company's intellectual property, Bio-Path's patent portfolio currently includes five issued patents in the US and 54 issued patents in foreign jurisdictions, providing protection in 21 countries. The company continues its efforts to build protection around its platform, which safeguards its technology, deters would-be competitors, and creates value around its core competencies.
Outlook
Looking ahead, Bio-Path expects to continue reporting updates on its important clinical programs while being good stewards of its resources. The company's work is crucial for patients waiting for effective treatments to halt the progression of deadly cancers, and the positive data from its various studies is encouraging.
Conclusion
Overall, Bio-Path Holdings is a promising oncology-focused RNAi nanoparticle drug developer with a diverse pipeline of clinical-stage and preclinical drug candidates targeting significant unmet medical needs. The company's recent advancements, strengthened financial position, and robust intellectual property portfolio position it well to continue advancing its innovative therapies and creating value for shareholders.