Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company
leveraging its proprietary DNAbilize® antisense RNAi nanoparticle
technology to develop a portfolio of targeted nucleic acid cancer
drugs, today announced the enrollment and dosing of the first
patient in the amended Stage 2 of the Phase 2 clinical study of
prexigebersen (BP1001), a liposomal Grb2 antisense, for the
treatment of acute myeloid leukemia (AML), in combination with
frontline therapy decitabine and venetoclax.
As previously reported, Phase 2 clinical
development of prexigebersen in AML commenced with Stage 1 of the
Phase 2 clinical trial, which was open label and treated de novo
AML patients with a combination of prexigebersen and low dose
cytarabine (LDAC). The combination of prexigebersen and LDAC
was shown to be safe and more efficacious to treat this class of
patients than with LDAC alone. Despite the superior
combination treatment results with LDAC, the new drug decitabine
was favored by oncologists. As a result, Stage 2 of the Phase 2
trial in AML dropped the combination treatment of prexigebersen and
LDAC and replaced it with the combination treatment prexigebersen
and decitabine. In addition, a second cohort of
relapsed/refractory AML patients was added.
The recent approval of the frontline therapy
venetoclax provided an opportunity for adding prexigebersen to the
newly approved frontline, two-drug combination of venetoclax and
decitabine for the treatment of AML patients.
Prior to finalizing plans to include
prexigebersen with the frontline treatment combination of
decitabine and venetoclax, the Company performed preclinical
testing in AML cancer cell lines to assess prexigebersen’s
increased benefit to efficacy. Preclinical testing of
prexigebersen with the frontline treatment of decitabine and
venetoclax demonstrated the potential to enhance efficacy of the
frontline treatment combination. In the studies, four AML
cancer cell lines were treated with three different combinations of
decitabine, venetoclax and prexigebersen (BP1001). Decrease
in AML cell viability was the primary measure of
efficacy. The triple combination of decitabine,
venetoclax and BP1001 showed significant improvement in efficacy in
three of the four AML cell lines. Based on these results, the
Company believes that adding prexigebersen to the treatment
combination of decitabine and venetoclax could lead to improved
efficacy in AML patients.
|
% Decrease in Cell Viability |
AML Cell Lines |
BP1001 + decitabine |
BP1001 + decitabine + venetoclax |
Controla + decitabine + venetoclax |
KG-1 |
64 |
90 |
46 |
MOLM-13 |
86 |
100 |
88 |
MV-4-11 |
67 |
95 |
79 |
Kasumi-1 |
33 |
50 |
47 |
a Empty liposomes control
Bio-Path’s approved amended Stage 2 for this
Phase 2 clinical trial has three cohorts of patients, which the
Company believes provides for several potential regulatory
pathways. The first two cohorts will treat patients with the
triple combination of prexigebersen, decitabine and
venetoclax. The first cohort will include untreated AML
patients, and the second cohort will include relapsed/refractory
AML patients. Finally, the third cohort will treat
relapsed/refractory AML patients who are venetoclax resistant or
intolerant with the two-drug combination of prexigebersen and
decitabine.
The first step in establishing the amended Stage
2 of the Phase 2 trial in AML was demonstrating the safety of
treating patients with the two-drug combination of prexigebersen
and decitabine, which the Company previously reported has been
successfully completed. Importantly, results from patients
who were previously treated with the two-drug combination of
prexigebersen and decitabine prior to the amendment to Stage 2 of
the Phase 2 trial in AML and who meet the criteria for enrollment
in the third cohort of the amended Stage 2 of the Phase 2 trial in
AML can be included in the third cohort results. This
represents a beneficial head-start in the third cohort
enrollment. This third cohort represents a significant, unmet
opportunity in clinical treatment, as options are limited for AML
patients who fail frontline therapy.
The first six evaluable patients in the amended Stage 2 of the
Phase 2 trial in AML will be treated with the triple combination of
prexigebersen, decitabine and venetoclax to test the safety of this
treatment combination. As noted previously, the enrollment
and dosing of the first patient in the amended Stage 2 of the Phase
2 clinical study has occurred. This patient is in the
relapsed/refractory cohort being treated with the triple
combination of prexigebersen, decitabine and venetoclax.
“Despite recent advances in the field, AML
continues to be a challenging malignancy with unmet medical need as
most patients unfortunately eventually succumb to their disease.
The potential for prexigebersen in combination with new standard of
care treatments seems particularly promising. We look forward
to the execution of this trial and, hopefully, to bringing another
tool to bear in the fight against this deadly disease,” said Jorge
Cortes, M.D., Director of the Georgia Cancer Center and Chairman of
the Bio-Path Scientific Advisory Board.
“We are excited to be testing prexigebersen in
this promising triple combination. Given our preclinical
data, which support treating AML patients with this triple
combination, we believe there is strong potential for improved
outcomes for patients with AML who otherwise have limited treatment
options,” said Peter Nielsen, President and Chief Executive Officer
of BioPath Holdings.
“We believe that this unique trial design
provides us with several definable registration pathways. We
believe that prexigebersen, with its promising efficacy and safety
profile, has the potential to be an ideal combination candidate
with frontline therapy,” concluded Mr. Nielsen.
Study Design
The amended Stage 2 of this Phase 2 trial in AML
is an open label Phase 2, two-stage, multicenter study of
prexigebersen in combination with decitabine and venetoclax in two
cohorts of patients with previously untreated AML and
relapsed/resistant AML. A third cohort includes treating
relapsed/refractory AML patients who are venetoclax resistant or
intolerant with the two-drug combination of prexigebersen and
decitabine.
The full trial design-plans have approximately
54 evaluable patients for the cohort treating relapsed/refractory
AML patients with the triple combination treatment of
prexigebersen, decitabine and venetoclax and the cohort treating
AML patients who are venetoclax resistant or intolerant with the
two-drug combination of prexigebersen and decitabine, with a review
of both cohorts performed after 19 evaluable patients.
The full trial design-plans have approximately
98 evaluable patients for the cohort treating untreated AML
patients with the triple combination treatment of prexigebersen,
decitabine and venetoclax, with a preliminary review for the cohort
performed after 19 evaluable patients and a formal interim analysis
after 38 evaluable patients. The higher number of patients in
the full trial design for the untreated AML patient cohort is due
to the higher baseline response of the frontline therapy.
The primary endpoint for this study will be the
number of patients who achieve complete remission (“CR”), which
includes complete remission with incomplete hematologic recovery
(“CRi”), and complete remission with partial hematology recovery
(“CRh”).
An interim analysis will be performed on each
cohort to assess the safety and efficacy of the treatment. In
the event these results exceed the primary endpoint in a number of
patients that meets or exceeds statistically determined thresholds,
the Company plans to seek to convert the trial into a registration
trial for accelerated approval.
The study is anticipated to be conducted at ten
clinical sites in the U.S., and Gail J. Roboz, M.D., will be the
national coordinating Principal Investigator for the Phase 2
trial. Dr. Roboz is 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. For more information on the
Phase 2 study, visit www.clinicaltrials.gov.
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 prexigebersen-A, a drug product
modification of prexigebersen, is under consideration by the FDA to
commence Phase 1 studies in solid tumors. This is followed by
BP1002, targeting the Bcl-2 protein, where it will be evaluated in
lymphoma and solid tumors clinical studies.
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 the impact, risks and uncertainties
related to COVID-19 and actions taken by governmental authorities
or others in connection therewith, Bio-Path’s ability to raise
needed additional capital on a timely basis in order for it to
continue its operations, Bio-Path's ability to have success in the
clinical development of its technologies, the timing of enrollment
and release of data in such clinical studies and 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 for future
patent applications will be issued or that any issued patents will
provide meaningful protection of our drug candidates, risks
relating to maintaining Bio-Path's listing on the Nasdaq Capital
Market 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’ConnorStern Investor Relations,
Inc.212-362-1200will@sternir.com
Doug MorrisInvestor Relations Bio-Path Holdings,
Inc. 832-742-1369
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