Bio-Path Holdings Presents Data from Ongoing Phase 2 Combination Study of Prexigebersen for Treatment of Acute Myeloid Leukemia at American Society of Clinical Oncology (ASCO) Annual Meeting

Rhea-AI Summary

Bio-Path Holdings (NASDAQ:BPTH) presented interim results from its Phase 2 study of prexigebersen (BP1001) at the ASCO Annual Meeting on June 1, 2024. The study involves treating acute myeloid leukemia (AML) patients with a combination of prexigebersen, decitabine, and venetoclax. Data from two cohorts were presented: 31 newly diagnosed patients and 38 relapsed/refractory patients.

In Cohort 1, 75% of evaluable newly diagnosed patients achieved complete or partial remission. In Cohort 2, 55% of evaluable relapsed/refractory patients achieved complete or partial remission. Adverse events were consistent with those typically associated with AML treatments, including fatigue, anemia, and neutropenia. Severe adverse events included febrile neutropenia (26%) and sepsis (5%).

Given these promising interim results, Bio-Path plans to continue patient enrollment to meet study targets. The company's DNAbilize® technology shows potential in developing RNAi nanoparticle drugs for various cancers.

Positive

• 75% of evaluable newly diagnosed AML patients achieved complete or partial remission.
• 55% of evaluable relapsed/refractory AML patients achieved complete or partial remission.
• Adverse events were consistent with expectations and manageable.
• Severe adverse events were relatively low, with febrile neutropenia at 26% and sepsis at 5%.
• Bio-Path continues to enroll patients, indicating confidence in study outcomes.
• The DNAbilize® technology shows potential in developing effective cancer treatments.

Negative

• Only 54% and 32% of enrolled patients in Cohort 1 and Cohort 2, respectively, were evaluable.
• No significant improvement in overall severe adverse events compared to standard treatments.
• Relatively high frequency of fatigue (72%), anemia (60%), and neutropenia (49%) among patients.
• data on long-term outcomes and overall survival rates.

Insights

Oncology Doctor

The interim results from Bio-Path Holdings' Phase 2 study of prexigebersen for treating acute myeloid leukemia (AML) are promising. In Cohort 1, the complete remission (CR) rates are impressive at 75% for newly diagnosed patients. This is a significant outcome given that these patients have severe adverse-risk profiles or secondary AML.

In Cohort 2, relapsed/refractory patients achieved a 55% CR rate, which is notable as these patients typically have a poor prognosis and limited treatment options.

Moreover, the safety profile seems tolerable with the most frequent severe adverse events being febrile neutropenia (26%) and sepsis (5%), which are manageable in a clinical setting. These findings suggest that prexigebersen, in combination with decitabine and venetoclax, could provide a valuable new treatment option for AML patients.

Medical Research Analyst

The data presented by Bio-Path Holdings at the ASCO meeting indicates substantial clinical improvement for AML patients treated with prexigebersen in combination with decitabine and venetoclax. The high rates of complete remission in both newly diagnosed and relapsed/refractory patients suggest that this combination therapy offers significant efficacy compared to existing treatments.

Given the limited treatment options for high-risk AML patients, the continuation of this Phase 2 study is crucial. The safety profile, which shows expected adverse events consistent with AML treatments, underscores the potential for prexigebersen as part of standard AML therapy.

Additionally, the ongoing enrollment and expansion of patient cohorts will provide further insight into the drug’s efficacy and safety, which is essential for regulatory approval and future clinical use.

Financial Analyst

The interim results from Bio-Path’s Phase 2 study of prexigebersen are likely to be well-received by investors, given the high remission rates reported. These results not only validate the company’s DNAbilize® technology but also enhance the commercial potential of prexigebersen. Successful continuation and eventual completion of the study could lead to significant milestones, including potential FDA approval and market entry.

From a financial perspective, the positive interim results may boost investor confidence, leading to an appreciation in Bio-Path's stock price. It’s important to monitor the enrollment progress and upcoming data releases, as further positive outcomes could attract strategic partnerships or acquisition interest, ultimately benefiting shareholders.

However, investors should remain cautious until final study results are available, considering the inherent risks of biotech investments and the competitive landscape in AML treatments.

Positive Results from Interim Analysis Demonstrate Significant Clinical Improvement and Tolerable Safety Profile for Prexigebersen Combination in High-Risk Patients

HOUSTON, June 03, 2024 (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 drugs, presented interim results from the Company’s Phase 2 study of prexigebersen (BP1001) in combination with decitabine and venetoclax for the treatment of acute myeloid leukemia (AML) in an oral presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting, on June 1, 2024 in Chicago, IL.

Maro Ohanian, D.O., Department of Leukemia, University of Texas MD Anderson Cancer Center, presented data showing prexigebersen continues to be well-tolerated and has demonstrated compelling efficacy results in two reporting cohorts including newly diagnosed AML patients and refractory/relapsed AML patients, both of which exceeded outcomes with frontline therapy.

“We were honored to have our data selected for oral presentation at ASCO as it underscores not only the quality of our data, but also highlights the continued unmet need for these most vulnerable cancer patients,” said Peter Nielsen, Chief Executive Officer of Bio-Path. “We continue to advance this important study confident that prexigebersen can make a difference in the lives of these patients for whom there are limited treatment options.”

Data Highlights

In Cohort 1, 31 newly diagnosed patients were enrolled; 20 evaluable patients (9 male: 45%) with a median age of 75 years (range, 69 - 84), treated with at least one cycle of prexigebersen, decitabine and venetoclax, had adverse-risk (n=12, 2017 ELN guidelines) or secondary AML (sAML; n=7) evolved from myelodysplastic syndromes (n=4), chronic myelomonocytic leukemia (n=1) or treatment-related AML (n=2). Fifteen patients (75% of evaluable; 54% of enrolled) achieved complete remission (CR), CRh (CR with partial recovery of peripheral blood counts), or CRi (CR with incomplete hematologic recovery); two patients achieved partial remission (PR) and two patients achieved stable disease (SD).

In Cohort 2, 38 relapsed/refractory patients were enrolled; 23 evaluable patients (13 male: 57%) with a median age of 63 years (range, 24 - 89), treated with at least one cycle of prexigebersen, decitabine and venetoclax, had adverse-risk (n=13) or sAML (n=5). Twelve patients (55% of evaluable; 32% of enrolled) achieved CR/CRi/CRh; one patient achieved PR, eight patients achieved SD and one patient had treatment failure.

Among the evaluable patients of both cohorts, adverse events were consistent with those expected with decitabine and venetoclax and/or AML, including fatigue (72%), anemia (60%) and neutropenia (49%), while the most frequent severe adverse events were febrile neutropenia (26%) and sepsis (5%). Given these promising interim results, Bio-Path expects to continue enrollment of up to 98 and 54 evaluable patients for Cohorts 1 and 2, respectively.

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 transfusion. 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 lymphoma and acute myeloid leukemia. In addition, an IND 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 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 were the interim results of Bio-Path Holdings' Phase 2 study of prexigebersen?

The interim results showed that 75% of evaluable newly diagnosed AML patients and 55% of relapsed/refractory AML patients achieved complete or partial remission.

How many patients were involved in Bio-Path Holdings' Phase 2 study of prexigebersen?

The Phase 2 study included 31 newly diagnosed AML patients and 38 relapsed/refractory AML patients.

What adverse events were reported in the Bio-Path Holdings' Phase 2 study of prexigebersen?

Common adverse events included fatigue (72%), anemia (60%), and neutropenia (49%). Severe adverse events included febrile neutropenia (26%) and sepsis (5%).

What is the significance of the interim results from Bio-Path Holdings' Phase 2 study of prexigebersen?

The promising interim results suggest that prexigebersen, in combination with decitabine and venetoclax, may be an effective treatment for AML, warranting continued patient enrollment and study.

What technologies is Bio-Path Holdings using in its cancer treatment developments?

Bio-Path Holdings is utilizing its proprietary DNAbilize® liposomal delivery and antisense technology to develop targeted nucleic acid cancer drugs.