Bio-Path Holdings Provides Update from Phase 1/1b Clinical Trial of BP1002 for Treatment of Refractory/Relapsed Acute Myeloid Leukemia
Bio-Path Holdings (NASDAQ:BPTH) has announced progress in its Phase 1/1b clinical trial of BP1002 for treating refractory/relapsed acute myeloid leukemia (AML). The trial has advanced to the fourth dose cohort of 90 mg/m2, with promising results from the third cohort where a patient showed stable disease and significant blast count reduction after one treatment cycle.
The study targets AML patients, including those resistant to venetoclax, the current standard of care. Notably, venetoclax-resistant patients typically have a median survival of less than 3 months. BP1002's unique approach targets Bcl-2 at the mRNA level, potentially overcoming venetoclax resistance mechanisms.
The trial is being conducted at major U.S. cancer centers, including Cornell University, MD Anderson Cancer Center, Scripps Health, and UCLA Cancer Center. The treatment protocol involves eight doses over 28 days, with plans to commence a combination therapy phase with decitabine after completing the monotherapy cohorts.
Bio-Path Holdings (NASDAQ:BPTH) ha annunciato progressi nel suo studio clinico di Fase 1/1b riguardante BP1002 per il trattamento della leucemia mieloide acuta (AML) refrattaria/recidivante. Lo studio è avanzato al quarto gruppo di dosaggio di 90 mg/m2, con risultati promettenti dal terzo gruppo, dove un paziente ha mostrato una malattia stabile e una significativa riduzione del conteggio dei blasti dopo un ciclo di trattamento.
Lo studio si rivolge a pazienti affetti da AML, compresi quelli resistenti al venetoclax, l'attuale standard di cura. È importante notare che i pazienti resistenti al venetoclax hanno tipicamente una sopravvivenza mediana di meno di 3 mesi. L'approccio unico di BP1002 colpisce Bcl-2 a livello di mRNA, potenzialmente superando i meccanismi di resistenza al venetoclax.
La sperimentazione è condotta presso i principali centri oncologici degli Stati Uniti, tra cui la Cornell University, il MD Anderson Cancer Center, lo Scripps Health e il UCLA Cancer Center. Il protocollo di trattamento prevede otto dosi nell'arco di 28 giorni, con piani per avviare una fase di terapia combinata con decitabina dopo aver completato i gruppi di monoterapia.
Bio-Path Holdings (NASDAQ:BPTH) ha anunciado avances en su ensayo clínico de Fase 1/1b de BP1002 para tratar la leucemia mieloide aguda (LMA) refractaria/recidivante. El ensayo ha avanzado al cuarto grupo de dosis de 90 mg/m2, con resultados prometedores del tercer grupo donde un paciente mostró enfermedad estable y una reducción significativa en el conteo de blastos después de un ciclo de tratamiento.
El estudio se enfoca en pacientes con LMA, incluidos aquellos resistentes al venetoclax, el estándar de atención actual. Es notable que los pacientes resistentes al venetoclax típicamente tienen una supervivencia mediana de menos de 3 meses. El enfoque único de BP1002 apunta a Bcl-2 a nivel de mRNA, potencialmente superando los mecanismos de resistencia al venetoclax.
El ensayo se lleva a cabo en importantes centros de cáncer de EE. UU., incluidos la Universidad de Cornell, el MD Anderson Cancer Center, Scripps Health y el UCLA Cancer Center. El protocolo de tratamiento implica ocho dosis durante 28 días, con planes para iniciar una fase de terapia combinada con decitabina después de completar los grupos de monoterapia.
Bio-Path Holdings (NASDAQ:BPTH)는 재발성 급성 골수성 백혈병(AML) 치료를 위한 BP1002의 1/1b상 임상 시험에서 진전을 발표했습니다. 이 시험은 90 mg/m2의 네 번째 용량 그룹으로 진행되었으며, 세 번째 그룹에서 한 환자가 안정된 질병 상태와 치료 주기 후 상당한 블라스트 수 감소를 보였습니다.
이 연구는 현재 표준 치료인 베네토클락스에 저항성을 가진 AML 환자를 포함하여 AML 환자를 대상으로 하고 있습니다. 특히, 베네토클락스에 저항성이 있는 환자는 일반적으로 3개월 미만의 중위 생존율을 보입니다. BP1002의 독특한 접근 방식은 mRNA 수준에서 Bcl-2를 목표로 하여 베네토클락스 저항 메커니즘을 극복할 수 있습니다.
이 시험은 Cornell University, MD Anderson Cancer Center, Scripps Health, UCLA Cancer Center 등 미국의 주요 암 센터에서 진행되고 있습니다. 치료 프로토콜은 28일 동안 8회의 용량을 포함하며, 단일 요법 그룹을 완료한 후 데시타빈과의 병용 요법 단계에 착수할 계획입니다.
Bio-Path Holdings (NASDAQ:BPTH) a annoncé des progrès dans son essai clinique de phase 1/1b concernant BP1002 pour le traitement de la leucémie myéloïde aiguë (LMA) réfractaire/récidivante. L'essai a avancé au quatrième groupe de dosage de 90 mg/m2, avec des résultats prometteurs du troisième groupe où un patient a montré une maladie stable et une réduction significative du nombre de blastes après un cycle de traitement.
L'étude cible les patients atteints de LMA, y compris ceux résistants au venetoclax, le traitement standard actuel. Notamment, les patients résistants au venetoclax ont généralement une survie médiane de moins de 3 mois. L'approche unique de BP1002 cible Bcl-2 au niveau de l'ARNm, ce qui pourrait permettre de surmonter les mécanismes de résistance au venetoclax.
L'essai est mené dans des centres de cancer majeurs aux États-Unis, notamment à l'Université de Cornell, au MD Anderson Cancer Center, à Scripps Health et au UCLA Cancer Center. Le protocole de traitement implique huit doses sur 28 jours, avec des plans pour commencer une phase de thérapie combinée avec la décitabine après avoir terminé les groupes de monothérapie.
Bio-Path Holdings (NASDAQ:BPTH) hat Fortschritte in seiner klinischen Phase 1/1b-Studie zu BP1002 zur Behandlung von refraktärer/rezidivierender akuter myeloischer Leukämie (AML) bekannt gegeben. Die Studie hat die vierte Dosierungsgruppe mit 90 mg/m2 erreicht, wobei vielversprechende Ergebnisse aus der dritten Gruppe vorliegen, in der ein Patient eine stabile Erkrankung und eine signifikante Reduktion der Blastenanzahl nach einem Behandlungszyklus zeigte.
Die Studie richtet sich an AML-Patienten, einschließlich solcher, die gegen Venetoclax resistent sind, dem aktuellen Standard der Versorgung. Bemerkenswert ist, dass Venetoclax-resistente Patienten typischerweise eine mediane Überlebenszeit von weniger als 3 Monaten haben. Der einzigartige Ansatz von BP1002 zielt auf Bcl-2 auf der mRNA-Ebene ab und könnte somit die Resistenzmechanismen gegen Venetoclax überwinden.
Die Studie wird in großen US-Krebszentren durchgeführt, darunter die Cornell University, das MD Anderson Cancer Center, Scripps Health und das UCLA Cancer Center. Das Behandlungsprotokoll umfasst acht Dosen über 28 Tage, mit Plänen, nach Abschluss der Monotherapie-Gruppen eine Phase der Kombinationstherapie mit Decitabine zu beginnen.
- Trial advanced to higher dose cohort (90 mg/m2)
- Patient in third cohort showed positive response with stable disease
- Faster than expected enrollment in third dosing cohort
- Treatment shows promise for venetoclax-resistant patients
- Still in early clinical trial phases
- patient data available so far
- Multiple competing cancer centers involved may increase operational complexity
Insights
The advancement of BP1002 to its fourth dose cohort represents a important milestone in addressing one of oncology's most challenging scenarios - venetoclax-resistant AML. The accelerated enrollment in the third cohort signals both significant unmet medical need and strong investigator interest, particularly noteworthy for a small-cap biotech company's clinical program.
The observed patient response in the third cohort is particularly meaningful given the context: patients who fail venetoclax-based therapy typically survive less than 3 months, making any signal of efficacy - especially stable disease and blast count reduction - potentially significant. The blast count reduction after just one treatment cycle is especially intriguing, as it suggests biological activity at doses below the maximum tolerated dose.
BP1002's mechanism of targeting Bcl-2 at the mRNA level represents a differentiated approach from venetoclax's protein-level inhibition. This distinction is important for three reasons:
- It may circumvent known resistance mechanisms to venetoclax
- It could potentially prevent the development of new resistance patterns
- It opens the possibility for combination therapy with existing treatments
The trial's progression to combination therapy with decitabine in the Phase 1b portion could prove particularly significant, as combination approaches often show superior efficacy in hematologic malignancies. The involvement of premier cancer centers like MD Anderson and Cornell adds credibility to the program and suggests strong investigator belief in the mechanism of action.
Study Progresses to Fourth Higher 90 mg/m2 Dose Cohort
Compelling Patient Response Highlighted by Stable Disease and Significant Reduction in Blast Count After One Treatment Cycle
HOUSTON, Feb. 12, 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 provides an update from the Company’s ongoing Phase 1/1b clinical trial evaluating BP1002 for the treatment of refractory/relapsed acute myeloid leukemia (AML), including venetoclax-resistant patients. The Company announced a meaningful patient response to treatment and the clinical trial has progressed to the fourth, higher dose cohort of 90 mg/m2.
“We were excited to learn that one patient in the third cohort had a meaningful response to just one treatment cycle, experiencing stable disease and a significant reduction in blast count, which we believe offers promise for venetoclax-resistant AML patients with limited treatment options,” said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings. “AML patients that fail or relapse from frontline venetoclax-based therapy have very poor prognosis with a median overall survival of less than three months. The third dosing cohort completed enrollment faster than expected, which we believe reflects the urgent need for additional treatment options. We look forward to quickly advancing this study through the fourth dosing cohort and into the combination therapy segment of this Phase 1/1b study with increased levels of BP1002 for the treatment of these vulnerable patients.”
The current standard of care for patients with AML not eligible for intensive chemotherapy is venetoclax, an oral Bcl-2 inhibitor that targets the BH3 domain of the Bcl-2 protein, in combination with a hypomethylating agent or with low-dose cytarabine. However, many patients become resistant to venetoclax treatment. A published study found that AML patients who had relapsed from frontline venetoclax-based treatment were refractory to salvage therapy and had a median survival of less than 3 months. By targeting Bcl-2 at the mRNA level rather than the protein, BP1002 may overcome and prevent some of the mechanisms of resistance that affect venetoclax treatment.
The Phase 1/1b clinical trial is being conducted at several leading cancer centers in the United States, including the Weill Medical College of Cornell University, The University of Texas MD Anderson Cancer Center, Scripps Health, and The University of California at Los Angeles Cancer Center. The approved treatment cycle is two doses per week over four weeks, resulting in eight doses administered over twenty-eight days. The Phase 1b portion of the study is expected to commence after completion of BP1002 monotherapy cohorts and will assess the safety and efficacy of BP1002 in combination with decitabine in refractory/relapsed AML patients.
Gail J. Roboz, M.D., is the National Principal Investigator for the Phase 1/1b trial. Dr. Roboz is a Professor of Medicine and Director of the Clinical and Translational Leukemia Program at the Weill Medical College of Cornell University and the New York-Presbyterian Hospital in New York City. Gary Schiller, M.D., The University of California at Los Angeles Cancer Center, Maro Ohanian, D.O., Department of Leukemia, University of Texas MD Anderson Cancer Center, and David Hermel, M.D., Scripps Health, are each serving as principal investigators.
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
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