Bio-Path Holdings Provides Clinical and Operational Update

Rhea-AI Summary

Bio-Path Holdings provided updates on its DNAbilize platform and clinical pipeline. The company's lead candidate prexigebersen is in Phase 2 trials for AML treatment, with two patients showing complete remission after 15+ treatment cycles. BP1001-A is advancing in Phase 1/1b trials for solid tumors, with one patient showing 15% tumor reduction. BP1002, targeting Bcl-2 protein, is progressing in Phase 1/1b trials for relapsed/refractory AML. The company also reported promising preclinical results for BP1001-A in treating obesity in Type 2 diabetes patients. Bio-Path's intellectual property portfolio includes 7 U.S. patents, 61 foreign patents across 26 countries, and multiple pending applications. The company's business model focuses on developing drug candidates and licensing them for final development and commercialization.

Positive

• Two AML patients in prexigebersen Phase 2 trial showed complete remission after 15+ treatment cycles
• BP1001-A demonstrated 15% tumor reduction in a gynecologic cancer patient through ten treatment cycles
• BP1002 Phase 1/1b trial completed third dosing cohort ahead of schedule, indicating strong demand
• Preclinical studies showed BP1001-A's potential effectiveness in treating obesity in Type 2 diabetes patients
• Strong IP protection with 7 U.S. patents, 61 foreign patents, and multiple pending applications

Negative

• Current AML treatment outcomes remain suboptimal with median survival of only 5-10 months
• AML patients failing frontline venetoclax-based therapy have poor prognosis with median survival under 3 months

Insights

Biotechnology Investment Analyst

Bio-Path's DNAbilize platform shows promise with multiple advancing clinical trials and early positive patient responses in difficult-to-treat cancers.

Bio-Path's clinical update reveals significant progress across multiple oncology programs. The company's lead candidate prexigebersen is advancing through a three-cohort Phase 2 trial for AML with two patients showing remarkable durability - both remaining in complete remission after 15+ treatment cycles. This is particularly meaningful given the poor prognosis (5-10 month median survival) for the elderly AML patients they're targeting.

Their BP1001-A program for solid tumors has demonstrated early efficacy signals, with a gynecologic cancer patient showing a 15% tumor reduction through ten cycles despite previously failing extensive treatments. The rapid enrollment in the BP1002 program for venetoclax-resistant AML patients suggests strong physician interest, likely driven by the urgent unmet need in this population where median survival is under three months.

The expansion into obesity represents a strategic diversification beyond oncology. Recent preclinical data confirms BP1001-A's mechanism in preventing insulin resistance, potentially opening a massive commercial opportunity given the obesity market's size.

Their intellectual property position appears robust with seven U.S. patents, 61 foreign patents across 26 countries, and numerous pending applications. This creates significant barriers to competition and enhances partnering/licensing potential - which aligns with management's stated business model of developing candidates and then licensing them to larger partners for late-stage development and commercialization.

While the update shows encouraging clinical signals, investors should note these are still early/mid-stage programs that face substantial development hurdles before reaching commercial potential.

Highlights Promise of DNAbilize® Platform to Produce New Drug Candidates and Potential Licensing Opportunities

HOUSTON, June 03, 2025 (GLOBE NEWSWIRE) -- Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize^® liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer and obesity drugs, today announced highlights from the recent clinical development and operational update conference call and webcast held May 26, 2025. An archived webcast of the event can be accessed here.

“Our business model centers around generating new drug candidates from our DNAbilize^® platform and licensing them for final development and commercialization with partners that have expertise and scale to successfully bring them to market,” said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings. “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.”

Clinical Program Overview

Prexigebersen Phase 2 Clinical Trial – Bio-Path’s Phase 2 clinical trial for the treatment of AML is comprised of three cohorts of patients and treatments, each separately approvable by the FDA as a new indication. The first two cohorts are treating patients with the triple combination of prexigebersen, decitabine and venetoclax. The first cohort includes untreated AML patients, and the second cohort includes relapsed/refractory AML patients. Finally, the third cohort is treating relapsed/refractory AML patients who are venetoclax-resistant or intolerant with the two-drug combination of prexigebersen and decitabine. Outcomes for these older patients who are unable to receive intensive chemotherapy due to the challenging side effect profile, remain suboptimal with a median survival of only 5 to 10 months. As previously reported, Bio-Path identified two patients who have demonstrated continued treatment durability. These patients have each received over 15 treatment cycles and remain in complete remission.

Bio-Path expects to utilize an advisory panel of AML experts to assist in the design of the final clinical development plans through potential FDA approval. Other significant milestones expected during 2025 include the completion of Cohort 2 and an interim analysis for Cohort 3.

Phase 1/1b Clinical Trial in BP1001-A in Advanced Solid Tumors – A Phase 1/1b clinical trial of BP1001-A in patients with advanced or recurrent solid tumors, including ovarian and uterine, pancreatic and breast cancer, is ongoing. BP1001-A is a modified product candidate that incorporates the same drug substance as prexigebersen but has a slightly modified formulation designed to enhance nanoparticle properties. The Phase 1 study has advanced to the second, higher dose level and the first patient in the second dose cohort continued experiencing a positive response which may signal that this analog of prexigebersen has potential as a new treatment for advanced solid tumors. The patient continues to be doing well after failing extensive chemotherapy and surgical treatment for gynecologic cancer, demonstrating a 15% reduction in her primary tumor through ten cycles of treatment. Moreover, it appears that these positive outcomes may have contributed to allowing her to continue with rigorous exercise and improved quality of life. Completion of the second and third dosing cohorts are expected later this year.

The Phase 1b portion of the study is expected to commence after successful completion of the three BP1001-A monotherapy dose level cohorts and is intended to assess the safety and efficacy of BP1001-A in combination with paclitaxel in patients with recurrent ovarian or endometrial tumors. Phase 1b studies are also expected to be initiated in combination with gemcitabine in Stage 4 pancreatic cancer and combination therapy in breast cancer.

Phase 1/1b Clinical Trial in BP1002 in Relapsed/Refractory AML – A Phase 1/1b clinical trial for BP1002 to treat relapsed/refractory AML patients, including venetoclax-resistant patients, is ongoing. BP1002 targets the protein Bcl-2, which is responsible for driving cell survival in up to 60% of all cancers. Venetoclax treats AML patients by blocking the activity of the Bcl-2 protein in AML patients. However, over time patients become resistant to venetoclax. BP1002 treats the Bcl-2 target by blocking the cell’s ability to produce Bcl-2 and could provide benefit for these venetoclax resistant patients. AML patients that fail frontline venetoclax-based therapy have very poor prognosis with median overall survival of less than three months. The first dose cohort consisted of a starting dose of 20 mg/m², the second dose cohort of 40 mg/m² and there were no dose limiting toxicities. The third dosing cohort of 60 mg/m² has been completed and the fourth dosing cohort of 90 mg/m² is open for enrollment. Enrollment in the third dosing cohort closed faster than expected which Bio-Path believes reflects the need for additional treatment options.

Prexigebersen as Potential Treatment for Obesity in Type 2 Diabetes Patients – BP1001-A downregulates Grb2 expression to increase insulin sensitivity and helps lower blood glucose level in Type 2 diabetes patients. Scientific evidence suggests that by downregulating Grb2 expression, BP1001-A could help lower blood glucose levels by affecting insulin signaling. Bio-Path conducted preclinical studies that confirmed the effectiveness of BP1001-A in affecting insulin signaling and its potential efficacy as a therapeutic treatment for obese patients who have Type 2 diabetes. In May, the Company reported the achievement of a third milestone from preclinical studies of BP1001-A that provide additional support for its potential as a treatment for obesity. These studies showed BP1001-A rescues the decrease in AKT activity in liver cells and prevents cells from becoming insulin resistant, confirming its potential as a treatment for obesity and related metabolic diseases in Type 2 diabetes patients. In 2025, Bio-Path expects to complete preclinical testing and to file an Investigational New Drug (IND) application.

Intellectual Property Protection

Bio-Path’s composition of matter patents are designed to protect encroachment from third parties on its proprietary products. These composition patents allow the Company to apply its core technology to new protein targets and receive new 20-year patents. Bio-Path’s patent portfolio is as follows:

• Composition and methods of use patents issued cover DNAbilize technology, solely owned by Bio-Path.
• Seven patents issued in the U.S. with one additional application allowed; 61 foreign patents issued across 26 countries; five additional foreign patent applications allowed; three applications pending in the U.S. along with more than 30 applications pending in foreign jurisdictions.

About Bio-Path Holdings, Inc.

Bio-Path is a biotechnology company developing DNAbilize^®, a novel technology that has yielded a pipeline of RNAi nanoparticle drugs that can be administered with a simple intravenous infusion. Bio-Path’s lead product candidate, prexigebersen (BP1001, targeting the Grb2 protein), is in a Phase 2 study for blood cancers, and BP1001-A, a drug product modification of prexigebersen, is in a Phase 1/1b study for solid tumors. The Company’s second product, BP1002, which targets the Bcl-2 protein, is being evaluated for the treatment of blood cancers and solid tumors, including acute myeloid leukemia. In addition, an IND application is expected to be filed for BP1003, a novel liposome-incorporated STAT3 antisense oligodeoxynucleotide developed by Bio-Path as a specific inhibitor of STAT3.

For more information, please visit the Company's website at http://www.biopathholdings.com.

Forward-Looking Statements

This press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws. These statements are based on management's current expectations and accordingly are subject to uncertainty and changes in circumstances. Any express or implied statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Any statements that are not historical facts contained in this release are forward-looking statements that involve risks and uncertainties, including Bio-Path’s ability to raise needed additional capital on a timely basis in order for it to continue its operations, have success in the clinical development of its technologies, the timing of enrollment and release of data in such clinical studies, the accuracy of such data, limited patient populations of early stage clinical studies and the possibility that results from later stage clinical trials with much larger patient populations may not be consistent with earlier stage clinical trials, the maintenance of intellectual property rights, that patents relating to existing or future patent applications will be issued or that any issued patents will provide meaningful protection of our drug candidates, the impact, risks and uncertainties related to global pandemics, including the COVID-19 pandemic, and actions taken by governmental authorities or others in connection therewith, and such other risks which are identified in Bio-Path's most recent Annual Report on Form 10-K, in any subsequent quarterly reports on Form 10-Q and in other reports that Bio-Path files with the Securities and Exchange Commission from time to time. These documents are available on request from Bio-Path Holdings or at www.sec.gov. Bio-Path disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Contact Information:

Investors

Will O’Connor
Stern Investor Relations, Inc.
212-362-1200
will@sternir.com

Doug Morris
Investor Relations
Bio-Path Holdings, Inc.
832-742-1369

FAQ

What are the key clinical developments in Bio-Path's (BPTH) prexigebersen Phase 2 trial?

Two patients in the prexigebersen Phase 2 trial for AML have shown complete remission after more than 15 treatment cycles. The trial includes three cohorts, with expected milestones in 2025 including completion of Cohort 2 and interim analysis for Cohort 3.

How is Bio-Path's (BPTH) BP1001-A performing in solid tumor trials?

BP1001-A has shown promising results in Phase 1/1b trials, with one gynecologic cancer patient experiencing 15% tumor reduction through ten treatment cycles and improved quality of life.

What progress has Bio-Path (BPTH) made with BP1002 in AML treatment?

BP1002 has completed its third dosing cohort of 60 mg/m2 with no dose-limiting toxicities and is now enrolling for the fourth cohort at 90 mg/m2. The trial targets relapsed/refractory AML patients, including venetoclax-resistant cases.

What is Bio-Path's (BPTH) potential in obesity and diabetes treatment?

Preclinical studies of BP1001-A showed promise in treating obesity in Type 2 diabetes patients by affecting insulin signaling. The company plans to complete preclinical testing and file an IND application in 2025.

How strong is Bio-Path's (BPTH) patent protection?

Bio-Path has robust IP protection with 7 U.S. patents, 61 foreign patents across 26 countries, 5 additional foreign patent applications allowed, and numerous pending applications in both U.S. and foreign jurisdictions.