Bio-Path Holdings Profile
Bio-Path Holdings, Inc. operates as a clinical and preclinical stage oncology-focused RNAi nanoparticle drug development company.
The company utilizes a novel technology that achieves systemic delivery for target specific protein inhibition for any gene product that is over-expressed in disease.
The company's drug delivery and antisense technology, called DNAbilize, is a platform that uses P-ethoxy, which is a deoxyribonucleic acid (DNA) backbone modification that is intended to protect the DNA from destruction by the body's enzymes when circulating in vivo, incorporated inside of a lipid bilayer having neutral charge. In vivo, the DNAbilize delivered antisense drug substances are systemically distributed throughout the body to allow for reduction or elimination of target proteins in blood diseases and solid tumors. Through testing in numerous animal studies and treatment in over 130 patients, the company's DNAbilize drug candidates have demonstrated an excellent safety profile. DNAbilize is a registered trademark of the company.
Using DNAbilize as a platform for drug development and manufacturing, the company has four drug candidates in development to treat at least five different cancer disease indications. The company's lead drug candidate, prexigebersen (pronounced prex' i je ber' sen), which targets growth factor receptor-bound protein 2 (Grb2), initially started the efficacy portion of a Phase 2 clinical trial for untreated acute myeloid leukemia (AML) patients in combination with low-dose cytarabine (LDAC). As a result, Stage 2 of the Phase 2 trial in AML was amended to remove the combination treatment of prexigebersen and LDAC and replace it with the combination treatment of prexigebersen and decitabine, a DNA hypomethylating agent, for the treatment of a second cohort of untreated AML patients.
The U.S. Food and Drug Administration (FDA) granted approval of venetoclax in combination with LDAC, decitabine or azacytidine (the latter two drugs are DNA hypomethylating agents) as frontline therapy for newly diagnosed AML in adults who are 75 years or older, or who have comorbidities precluding intensive induction chemotherapy. Preclinical efficacy studies for the triple combination treatment of prexigebersen, decitabine and venetoclax in AML have been successfully completed. The company further amended Stage 2 of this Phase 2 clinical trial to add the triple combination treatment comprising prexigebersen, decitabine, and venetoclax.
The company's approved amended Stage 2 for this Phase 2 clinical trial has three cohorts of patients. The first two cohorts will treat patients with the triple combination of prexigebersen, decitabine, and venetoclax. The first cohort will include untreated AML patients, and the second cohort will include relapsed/refractory AML patients. Finally, the third cohort will treat relapsed/refractory AML patients, who are venetoclax-resistant or -intolerant, with the two-drug combination of prexigebersen and decitabine. On August 13, 2020, the company announced the enrollment and dosing of the first patient in this approved amended Stage 2 of the Phase 2 clinical trial.
On April 5, 2021, the company announced the successful completion of the safety run-in of Stage 2 of the Phase 2 clinical study. In the safety run-in of the triple combination, six evaluable patients were treated with the combination of prexigebersen, decitabine and venetoclax.
The company's second drug candidate, Liposomal Bcl-2 (BP1002), targets the protein Bcl-2, which is responsible for driving cell survival in up to 60% of all cancers. On November 21, 2019, it announced that the FDA cleared an Investigational New Drug (IND) application for BP1002 for an initial Phase 1 clinical trial to evaluate the ability of BP1002 to treat refractory/relapsed lymphoma and chronic lymphocytic leukemia (CLL) patients. The Phase 1 clinical trial is being conducted at the Georgia Cancer Center while two additional clinical trial sites are being processed for inclusion in the study, The University of Texas Southwestern and New York Medical College.
On August 24, 2021, the company announced that the FDA cleared an IND application for BP1002 for an initial Phase 1/1b clinical trial to evaluate the ability of BP1002 to treat refractory/relapsed AML patients, including venetoclax-resistant patients. The Phase 1/1b clinical trial is being conducted at several leading cancer centers in the United States, including the Weill Medical College, The University of Texas MD Anderson Cancer Center (MD Anderson Cancer Center), Scripps Health and The University of California at Los Angeles Cancer Center. On October 24, 2022, the company announced the enrollment and dosing of the first patient in the Phase 1/1b clinical trial.
The company's third drug candidate, Liposomal STAT3 (BP1003), targets the STAT3 protein and is in IND enabling studies as a potential treatment of pancreatic cancer, non-small cell lung cancer (NSCLC) and AML. Its lead indication for BP1003 is pancreatic cancer due to the severity of this disease and the lack of effective, life-extending treatments. For example, pancreatic adenocarcinoma is projected to be the second most lethal cancer behind lung cancer by 2030.
In addition, a modified product named BP1001-A, the company's fourth drug candidate, has shown to enhance chemotherapy efficacy in preclinical solid tumor models. Results of the preclinical study were published in the scientific journal Oncotarget in July 2020. On October 27, 2021, the company announced that the FDA cleared an IND application for BP1001-A for an initial Phase 1/1b clinical trial in patients with advanced or recurrent solid tumors, and on December 7, 2022, it announced the enrollment and dosing of the first patient in the Phase 1/1b clinical trial. The Phase 1/1b clinical trial is being conducted at several leading cancer centers in the United States, including the Weill Medical College, MD Anderson Cancer Center, Scripps Health, and The University of California at Los Angeles Cancer Center.
The company's DNAbilize technology-based products are available for out-licensing or partnering. It intends to apply its drug technology template to new disease-causing protein targets to develop new liposomal antisense drug candidates for inclusion in its pipeline that meet scientific, preclinical, and commercial criteria and file new patents on these targets. As the company expand its drug development programs, it will look at indications where a systemic delivery is needed and antisense RNAi nanoparticles can be used to slow, reverse or cure a disease, either alone or in combination with another drug.
The company has certain intellectual property as the basis for its drug products in clinical development, prexigebersen, BP1002, BP1003 and BP1001-A. It is developing RNAi antisense nanoparticle drug candidates based on its own patented technology to treat cancer and autoimmune disorders, where targeting a single protein may be advantageous and result in reduced patient adverse effects as compared to small molecule inhibitors with off-target and non-specific effects. It has composition of matter and method of use intellectual property for the design and manufacture of antisense nanoparticle drug products.
Strategy
The company's strategy is to develop prexigebersen, BP1002, BP1003, and BP1001-A for multiple indications where the pathways involving Grb2, Bcl-2 and STAT3, respectively, are utilized to promote cancer growth, survival, angiogenesis, and tumor surveillance evasion.
The key elements of the company's strategy are to develop prexigebersen for the treatment of AML in combination therapies; develop BP1002 for the treatment of lymphoma and CLL; develop BP1002 for refractory/relapsed AML patients, including venetoclax-resistant patients; develop BP1003 for pancreatic cancer, NSCLC and AML; develop BP1001-A for the treatment of solid tumors; expand DNAbilize to evaluate targets beyond cancer; and expand DNAbilize as the antisense drug delivery method of choice by forming partnerships with pharmaceutical and academic clinical research labs.
Intellectual Property
The company's patent portfolio includes five issued patents in the U.S. and eight issued patents in foreign jurisdictions. It has six additional pending patent applications in the U.S. and one additional allowed patent application in a foreign jurisdiction. Further, the company has pending patent applications in key foreign jurisdictions across its six families of applications.
Research and Development
During the year ended December 31, 2022, the company's research and development expenses were $9.2 million.
Government Regulation
Any drug products manufactured or distributed by the company pursuant to FDA approvals are subject to continuing regulation by the FDA, including record-keeping requirements; reporting of adverse experiences with the drug; providing the FDA with updated safety and efficacy information; drug sampling and distribution requirements; notifying the FDA and gaining its approval of specified manufacturing or labeling changes; complying with certain electronic records and signature requirements; and complying with FDA promotion and advertising requirements.
History
Bio-Path Holdings, Inc. was founded in 2007.
