prexigebersen (BP1001) / Bio-Path - LARVOL DELTA

Bio-Path Holdings Provides Clinical and Operational Update (The Manila Times) - "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....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."

P2 data • Trial status • Acute Myelogenous Leukemia

BP1001-201-AML: Clinical Trial of BP1001 in Combination With With Venetoclax Plus Decitabine in AML (clinicaltrials.gov) - P2 | N=108 | Recruiting | Sponsor: Bio-Path Holdings, Inc. | Phase classification: P2a ➔ P2 | Trial completion date: Dec 2024 ➔ Dec 2028 | Trial primary completion date: Dec 2024 ➔ Dec 2027

Phase classification • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

AML Patients Demonstrate Extended Treatment Durability (GlobeNewswire) - P2 | N=108 | NCT02781883 | Sponsor: Bio-Path Holdings, Inc. | "Previously, Bio-Path reported two patients were identified in the Phase 2 clinical trial treating AML patients who demonstrated continued treatment durability. As of January 2025, both these patients are receiving treatment and are continuing to do well. The first patient is an elderly female who had received 16 cycles of treatment over 21 months when first reported on. She continues on study having received 20 cycles over 26 months and she remains in complete remission. The second patient is an elderly male who had received 12 cycles of treatment over 14 months when first reported on. He continues on study having received 16 cycles over 20 months and remains in complete remission. Both patients are being treated with the triple combination of prexigebersen, decitabine and venetoclax."

P2 data • Acute Myelogenous Leukemia

Bio-Path Holdings Reports Third Quarter 2024 Financial Results (GlobeNewswire) - "In October, Bio-Path announced completion of enrollment for the third dosing cohort of the Company’s Phase 1/1b trial of BP1002 in venetoclax-resistant AML patients....Research and development expense for the three months ended September 30, 2024 decreased to $1.3 million, compared to $2.3 million for the three months ended September 30, 2023 primarily due to decreased manufacturing expenses related to drug product releases as well as a decrease in expense related to our clinical trial for BP1001 in AML due to timing of patient enrollment during the quarter."

Commercial • Enrollment status • Acute Myelogenous Leukemia

Bio-Path Holdings Provides Clinical Update and Expansion Plans (GlobeNewswire) - "Prexigebersen Phase 2 Clinical Trial – Bio-Path’s Phase 2 clinical trial is treating Acute Myeloid Leukemia (AML) patients....Based on recent interim data for safety and efficacy, the Company plans to pursue next development steps by applying molecular biomarkers to future patient selection for enrollment into the Phase 2 clinical trial....The study is currently paused for an interim analysis, amendment preparation and U.S. Food and Drug Administration (FDA) review. Bio-Path expects to complete enrollment in cohorts 1 and 2 of the study over the next eighteen months."

Enrollment status • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Bio-Path Holdings Provides 2024 Clinical and Operational Update (GlobeNewswire) - "Based on positive interim data for safety and efficacy, the Company plans to pursue FDA Fast Track designation for the accelerated approval of prexigebersen for the treatment of fragile AML patients who are unable to tolerate intensive chemotherapy and thus experience very poor clinical outcomes....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 opened in combination with gemcitabine in Stage 4 pancreatic cancer and combination therapy in breast cancer."

Fast track • Trial status • Acute Myelogenous Leukemia • Endometrial Cancer • Fallopian Tube Cancer • Gynecologic Cancers • Hematological Malignancies • Leukemia • Oncology • Ovarian Cancer • Peritoneal Cancer • Solid Tumor

INTERIM SAFETY AND EFFICACY OF BP1001 IN A PHASE II ACUTE MYELOID LEUKEMIA STUDY (EHA 2024) - P2a | "Preclinical studies indicated that BP1001 reduced leukemia proliferation and enhanced the inhibitory effects of chemotherapy, including decitabine ( DAC ) and venetoclax ( VEN ) against AML cells. BP1001 + DEC + VEN has been safely administered to patients without drug-related toxicity. Since >5 responses are observed in both cohorts, the study will continue with enrollment up to 98 and 54 evaluable patients in cohorts 1 and 2, respectively. Efficacy data are encouraging in a challenging population of frontlineadverse-risk, sAML and R/R patients."

Clinical • P2 data • Acute Myelogenous Leukemia • Anemia • Chronic Myelomonocytic Leukemia • Fatigue • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Oncology • Septic Shock • GRB2

Interim safety and efficacy of BP1001 in a phase II acute myeloid leukemia (AML) study. (ASCO 2024) - P2a | "BP1001, a liposome-incorporated Grb2 antisense oligonucleotide,enhanced cancer cell sensitivity to chemotherapy, such as decitabine (DEC) and venetoclax (VEN). BP1001 + DEC + VEN has been safely administered to pts without drug-related toxicity. Since >5 responses are observed in both cohorts, the study will continue with enrollment up to 98 and 54 evaluable pts in cohorts 1 and 2, respectively. Efficacy data are encouraging in a challenging population of frontline adverse-risk, sAML and R/R pts."

Clinical • P2 data • Acute Myelogenous Leukemia • Anemia • Chronic Myelomonocytic Leukemia • Fatigue • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Oncology • Septic Shock

Bio-Path Holdings Presents Data from Ongoing Phase 2 Combination Study of Prexigebersen for Treatment of Acute Myeloid Leukemia at European Hematology Association Congress (GlobeNewswire) - P2a | N=108 | NCT02781883 | Sponsor: Bio-Path Holdings, Inc. | "In Cohort 1, 31 newly diagnosed patients were enrolled...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...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."

P2a data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

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 (GlobeNewswire) - P2 | N=108 | NCT02781883 | Sponsor: Bio-Path Holdings, Inc. | "Bio-Path Holdings...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...ASCO Annual Meeting....In Cohort 1, 31 newly diagnosed patients were enrolled....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....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."

P2 data • Acute Myelogenous Leukemia

Bio-Path Holdings to Present Data at 2024 European Hematology Association Congress (GlobeNewswire) - P2a | N=108 | NCT02781883 | Sponsor: Bio-Path Holdings, Inc. | "Bio-Path Holdings, Inc...announced an upcoming poster presentation at the 2024 European Hematology Association (EHA) Congress, taking place June 13-16, 2024 in Madrid, Spain. Jorge Cortes, M.D., Director of the Georgia Cancer Center, will present 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). The data show prexigebersen continues to be well-tolerated and has now demonstrated compelling efficacy results in two reporting cohorts including evaluable newly diagnosed AML patients and evaluable refractory/relapsed AML patients, both of which exceeded outcomes with frontline therapy."

P2a data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Bio-Path Holdings to Present Data at American Society of Clinical Oncology (ASCO) Annual Meeting (GlobeNewswire) - P2 | N=108 | NCT02781883 | Sponsor: Bio-Path Holdings, Inc. | "Bio-Path Holdings, Inc...announced an upcoming oral presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting, taking place May 31 - June 4, 2024 in Chicago, IL...Maro Ohanian, D.O., Department of Leukemia, University of Texas MD Anderson Cancer Center, will present 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). The data show prexigebersen continues to be well-tolerated and has now demonstrated compelling efficacy results in two reporting cohorts including evaluable newly diagnosed AML patients and evaluable refractory/relapsed AML patients, both of which exceeded outcomes with frontline therapy."

P2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Bio-Path Holdings Reports Third Quarter 2023 Financial Results (GlobeNewswire) - "Research and development expense for the three months ended September 30, 2023 decreased to $2.3 million, compared to $2.4 million for the three months ended September 30, 2022, primarily due to decreased manufacturing development expenses partially offset by an increase in expense related to our clinical trial for prexigebersen in AML due to increased patient enrollment in 2023."

Commercial • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Bio-Path Holdings to Host Virtual Key Opinion Leader Event to Discuss Prexigebersen and Advances in the Treatment Landscape for Acute Myeloid Leukemia (GlobeNewswire) - "Bio-Path Holdings, Inc...announced it will host a virtual key opinion leader event to highlight the prexigebersen program and discuss advances in the treatment landscape for acute myeloid leukemia (AML). The conference call will be held on Monday, October 30, 2023 at 9:00 a.m. ET."

Clinical data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Bio-Path Holdings Reports Second Quarter 2023 Financial Results (GlobeNewswire) - "Research and development expense for the three months ended June 30, 2023 increased to $3.1 million, compared to $1.9 million for the three months ended June 30, 2022 primarily due to manufacturing expenses related to drug product releases during the second quarter of 2023 and increased patient enrollment related to our Phase 2 clinical trial for prexigebersen in AML."

Commercial • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Bio-Path Holdings Announces Positive Results from Interim Analysis of Phase 2 Clinical Trial of Prexigebersen in Acute Myeloid Leukemia (GlobeNewswire) - P2 | N=108 | NCT02781883 | Sponsor: Bio-Path Holdings, Inc. | "In total, 100% of the evaluable patients had a response to treatment. The complete remission rate (CR/CRi) of (86%) for the evaluable patients in Cohort 1 is significantly higher than complete remission (CR/CRi) rates (62%) for newly diagnosed patients treated with the frontline combination treatment of decitabine and venetoclax....The complete remission rate (CR/CRi) of (57%) for the evaluable refractory and relapsed patients in Cohort 2 is significantly higher than complete remission (CR/CRi) rate (21%) for refractory/relapsed patients treated with the combination treatment of decitabine and venetoclax....As a result of the interim review, Bio-Path has demonstrated superiority of prexigebersen combination therapy in treating AML patients and currently plans to pursue U.S. Food and Drug Administration (FDA) expedited programs for Fast Track and Breakthrough Therapy designations."

Breakthrough therapy designation • Fast track designation • P2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Bio-Path Holdings Reports Full Year 2022 Financial Results (GlobeNewswire) - "Prexigebersen (BP1001) Phase 2 Clinical Trial in AML: Two of the three cohorts in the clinical trial already exceed the minimum efficacy required for enrollment expansion. Assess safety and efficacy of each cohort treatment combination therapy with potential to qualify for expedited program status after cohort’s initial interim analysis, which are expected to commence by cohort in the second quarter of 2023....Research and development expense for the year ended December 31, 2022, increased to $9.2 million, compared to $5.9 million for the year ended December 31, 2021, primarily due to manufacturing expenses related to drug product releases in 2022, increased enrollment in our Phase 2 clinical trial for prexigebersen in AML and start-up costs related to our Phase 1 clinical trial for BP1002 in refractory/relapsed AML patients."

Commercial • Trial status • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Bio-Path Holdings Provides Clinical and Operational Update (GlobeNewswire) - "BP1001-A Phase 1/1b Clinical Trial in Solid Tumors: Important trial with advanced or recurrent solid tumors, including ovarian and uterine, pancreatic and breast cancer with initial cohort completion and data readout expected before mid-year. BP1002 Phase 1/1b Clinical Trial in Relapsed/Refractory AML: Focus on patients who relapsed on venetoclax treatment with initial cohort completion and readout expected in the second quarter of 2023. Prexigebersen (BP1001) Phase 2 Clinical Trial in AML...Assess safety and efficacy of each cohort treatment combination therapy with potential to qualify for expedited program status after cohort’s initial interim analysis, which are expected to commence by cohort in the second quarter of 2023."

P1 data • Trial status • Acute Myelogenous Leukemia • Breast Cancer • Gastrointestinal Cancer • Gynecologic Cancers • Hematological Malignancies • Leukemia • Oncology • Ovarian Cancer • Pancreatic Cancer • Solid Tumor • Uterine Cancer

Clinical Trial of BP1001 in Combination With With Venetoclax Plus Decitabine in AML (clinicaltrials.gov) - P2a | N=108 | Recruiting | Sponsor: Bio-Path Holdings, Inc. | Trial completion date: Dec 2022 ➔ Dec 2024 | Trial primary completion date: Dec 2022 ➔ Dec 2024

Combination therapy • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Safety and Efficacy of Lower Intensity Induction Therapy with Intravenous Prexigebersen (BP1001) in Patients with High-Risk and Relapsed/Refractory Acute Myeloid Leukemia (AML) (ASH 2021) - P2a | "A Phase I/IB single center study (Ohanian, Lancet Haematol 2018) in relapsed/refractory AML patients demonstrated BP1001 safety up to 90 mg/m 2 and demonstrated CR/CRi in 3 of 6 relapsed/refractory AML patients treated with BP1001 + low dose cytarabine combination in the Phase IB portion. Preclinical studies indicated that BP1001 enhanced the inhibitory effects of decitabine (DAC), or DAC + venetoclax (VEN) against AML cells... BP1001-based combination therapy has been safely administered to high-risk and relapsed/refractory AML patients who were considered unsuitable for standard chemotherapy. Preliminary data showed the combination treatment of BP1001 + DAC or BP1001 + DAC + VEN was well-tolerated, with encouraging efficacy signals. The study will continue enrollment across all three cohorts."

Clinical • Acute Myelogenous Leukemia • Constipation • Endocrine Disorders • Fatigue • Febrile Neutropenia • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Hypotension • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Neutropenia • Oncology • Pain • Pulmonary Disease • Septic Shock • FLT3 • KRAS • NF1 • NRAS • PTPN11

Clinical Trial of BP1001 in Combination With With Venetoclax Plus Decitabine in AML (clinicaltrials.gov) - P2a; N=108; Recruiting; Sponsor: Bio-Path Holdings, Inc.; Trial completion date: Dec 2021 ➔ Dec 2022; Trial primary completion date: Dec 2021 ➔ Dec 2022

Clinical • Combination therapy • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Bio-Path Holdings Presents Data from Ongoing Phase 2 Study of Prexigebersen at 2021 American Society of Hematology Annual Meeting (GlobeNewswire) - P2, N=108; NCT02781883; Sponsor: Bio-Path Holdings, Inc;"Six patients were treated with at least one cycle of prexigebersen + decitabine + venetoclax combination therapy. Of the six patients, two (33%) had de novo AML and four (67%) were relapsed/refractory. All patients in this cohort were adverse-risk by ELN (n=2) or relapsed/refractory (n=4). AEs were generally consistent with decitabine and venetoclax treatment and/or for AML. Four patients (67%) achieved a complete remission (CR)/CRi/morphological leukemia free state (MLFS) (n=1/2/1) and one (17%) achieved a PR. Of these five patients, three were relapsed/refractory (75% of relapsed/refractory patients) (1 CR/1 CRi/1 MLFS) and two were de novo (1 CRi/1 PR) (100% of the de novo patients). CR rates to combination treatment with decitabine and venetoclax for relapsed/refractory AML patients are 42-52%1,2 and 0-39%1,2 for relapsed/refractory secondary AML patients."

P2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology

Bio-Path Holdings to Present Clinical Data at the 63rd Annual American Society of Hematology Annual Meeting (GlobeNewswire) - "Bio-Path Holdings, Inc...announced an upcoming poster presentation at the 2021 American Society of Hematology (ASH) Annual Meeting and Exposition, taking place from December 11-14, 2021 in Atlanta, GA...Dr. Maro Ohanian, Associate Professor of the Department of Leukemia at The University of Texas M.D. Anderson Cancer Center, will present the safety and preliminary efficacy data from the ongoing Phase 2 trial of prexigebersen (BP1001), the Company’s lead drug candidate, for the treatment of acute myeloid leukemia."

P2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology

Bio-Path Holdings Announces Clearance of Investigational New Drug Application for Phase 1/1b Clinical Trial of Prexigebersen-A in Solid Tumors (GlobeNewswire) - "Bio-Path Holdings, Inc...announced that the U.S. Food and Drug Administration (FDA) has reviewed and cleared the Investigational New Drug (IND) application to initiate a Phase 1/1b clinical trial of prexigebersen-A (liposomal Grb2-A or BP1001-A) in patients with solid tumors, including ovarian, endometrial, pancreatic and triple negative breast cancer...The Phase 1/1b clinical trial is anticipated to be conducted at several leading cancer centers in the United States, including The University of Texas MD Anderson Cancer Center and the Mary Crowley Research Center. Eventually, we expect to have six sites to conduct the clinical trial. Initially, a total of six evaluable patients are scheduled to be treated with prexigebersen-A monotherapy in a standard 3+3 design, with a starting dose of 60 mg/m2."

IND • Endometrial Cancer • Gastrointestinal Cancer • Gynecologic Cancers • Oncology • Ovarian Cancer • Pancreatic Cancer • Solid Tumor • Triple Negative Breast Cancer

[VIRTUAL] A phase II study of BP1001 (liposomal Grb2 antisense oligonucleotide) in patients with hematologic malignancies. (ASCO 2020) - P2a | "A Phase I/Ib single center study in patients with refractory/relapsed leukemias demonstrated the safety of BP1001 up to 90 mg/m2 dose and the efficacy of BP1001 in combination with low dose cytarabine (LDAC) in refractory/relapsed acute myeloid leukemia (AML) patients (Ohanian, Lancet Haematol 2018)...The secondary objectives of this study are to assess: Safety of BP1001 + decitabine; partial remissions and blast count reductions; overall survival, time to response and duration of response; minimal residual disease status in patients who achieve complete remission; and plasma PK profile of BP1001. The exploratory objective of this study is to evaluate the association of treatment response with cytogenetic and molecular characteristics. Research Funding: Bio-Path Holdings, Inc."

Clinical • P2 data • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology