Biotech Company Presents Promising Data For Acute Myeloid Leukemia Treatment
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.
Disclaimer: This article is for informational purposes only. It does not constitute medical advice, diagnosis, or treatment recommendations. Readers are advised to consult with healthcare professionals for any medical concerns.
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, held on June 1, 2024, in Chicago, IL.
Presenting the Data
Maro Ohanian, D.O., from the Department of Leukemia at the University of Texas MD Anderson Cancer Center, presented data indicating that prexigebersen continues to be well-tolerated and has demonstrated compelling efficacy results in two reporting cohorts: newly diagnosed AML patients and refractory/relapsed AML patients. Both cohorts achieved outcomes that exceeded those typically seen 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.”
In the first cohort, 31 newly diagnosed patients were enrolled. Among them, 20 were evaluable (9 male: 45%) with a median age of 75 years (range, 69 - 84). These patients were treated with at least one cycle of prexigebersen, decitabine, and venetoclax. Most of the patients had adverse-risk AML (n=12, based on the 2017 ELN guidelines) or secondary AML (sAML; n=7) which evolved from myelodysplastic syndromes (n=4), chronic myelomonocytic leukemia (n=1), or treatment-related AML (n=2). The results were promising:
Fifteen patients (75% of evaluable; 54% of enrolled) achieved complete remission (CR), CR with partial recovery of peripheral blood counts (CRh), or CR with incomplete hematologic recovery (CRi).
Two patients achieved partial remission (PR).
Two patients achieved stable disease (SD).
Cohort 2: Relapsed/Refractory AML Patients
In the second cohort, 38 relapsed/refractory patients were enrolled. Among them, 23 were evaluable (13 male: 57%) with a median age of 63 years (range, 24 - 89). These patients were treated with at least one cycle of prexigebersen, decitabine, and venetoclax. They had either adverse-risk AML (n=13) or sAML (n=5). The results were encouraging:
Twelve patients (55% of evaluable; 32% of enrolled) achieved CR/CRi/CRh.
One patient achieved PR.
Eight patients achieved SD.
One patient had treatment failure.
Adverse Events: Among the evaluable patients from both cohorts, adverse events were consistent with those expected with decitabine and venetoclax and/or AML. The common adverse events included:
Fatigue (72%)
Anemia (60%)
Neutropenia (49%)
The most frequent severe adverse events were:
Febrile neutropenia (26%)
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
Bio-Path Holdings 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. The company’s lead product candidate, prexigebersen (BP1001), is in a Phase 2 study for blood cancers. Additionally, BP1001-A, a drug product modification of prexigebersen, is in a Phase 1/1b study for solid tumors.
DNAbilize® technology uses liposomal delivery and antisense technology to precisely target nucleic acids within cancer cells. This innovative approach provides a new avenue for treating various types of cancer by interfering with the genes that promote tumor growth and survival.
The Phase 2 combination study of prexigebersen for the treatment of AML is ongoing, and the next steps are crucial for further validating the effectiveness and safety of the treatment. Bio-Path Holdings will continue to monitor the progress of the enrolled patients while enrolling additional participants to reach the targeted numbers for both cohorts.
Researchers and clinicians are particularly optimistic about the potential of prexigebersen given its performance in the interim results. If the trends observed continue, prexigebersen could become a significant addition to the therapeutic arsenal against AML, especially for patients who have exhausted other treatment options. Bio-Path Holdings’ commitment to innovation in cancer treatment is evident through the development of their DNAbilize® technology and the promising results from their clinical trials. By targeting specific genetic mechanisms within cancer cells, the company aims to provide more effective and less toxic treatment options for cancer patients.
Future Implications
The implications of successful Phase 2 results extend beyond the immediate patient population. Positive outcomes could pave the way for broader regulatory approvals, potential partnerships, and further research into additional applications of prexigebersen and other DNAbilize®-based therapies. The success of this technology could encourage more investment and interest in nucleic acid-based treatments, potentially leading to new breakthroughs in cancer therapy.
Bio-Path Holdings’ presentation at the ASCO Annual Meeting marks a pivotal moment in the ongoing battle against AML. The interim results from the Phase 2 study of prexigebersen combined with decitabine and venetoclax are promising, showing significant efficacy and manageable adverse events. As the study progresses, the hope is that prexigebersen will continue to demonstrate its potential to improve the lives of AML patients, offering a new ray of hope for those battling this challenging disease.
Readers should stay tuned for more updates on the progress of this study and other developments from Bio-Path Holdings as they continue their mission to innovate and improve cancer treatment.
Disclaimer: This article is for informational purposes only. It does not constitute medical advice, diagnosis, or treatment recommendations. Readers are advised to consult with healthcare professionals for any medical concerns.
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