Panbela Announces Publication of Clinical Data Titled: Phase 1 study of high-dose DFMO, celecoxib, cyclophosphamide and topotecan for patients with relapsed neuroblastoma: A New Approaches to Neuroblastoma Therapy Trial
2024年1月18日 - 10:15PM
Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage
biopharmaceutical company developing disruptive therapeutics for
the treatment of patients with urgent unmet medical needs announces
the publication of clinical data from studies of CPP-1X (also known
as α-Difluoromethylornithine (DFMO) or Eflornithine) in
neuroblastoma. According to Hogarty et al, children with relapsed
refractory neuroblastoma have dismal outcomes and new therapeutic
options are needed. Data published in the British Journal of Cancer
investigated the tolerability and activity of depleting polyamines
by high dose CPP-1X and celecoxib in combination with standard of
care chemotherapy in heavily pretreated neuroblastoma patients.
Results showed that DFMO treatment was well tolerated, and the
median time-to-progression was 19.8 months. The work reflects the
Company’s previous collaboration with New Advances in Neuroblastoma
Therapy Consortium (NANT) (https://www.nant.org/). A link to the
publication can be found here:
https://www.nature.com/articles/s41416-023-02525-2.
From the Phase 1 dose range finding study of CPP-1X in heavily
pretreated neuroblastoma patients, CPP-1X was well tolerated. The
best overall response included 2 partial responses (PR), 4 minor
responses (MR), 10 Stable disease (SD), 7 progressive disease (PD)
and 1 unevaluable. All patients with an overall response of PR or
MR sustained this response until stopping or completing protocol
therapy. The overall objective response rate (CR+PR) was 9% and
rate of any response (CR+PR+MR) was 26%. At 2 years, PFS
(progression free survival) for the entire cohort was 29.5%.
Notably, three patients completed protocol therapy and remain
without disease progression or event at >4 years from treatment
end in the absence of additional therapy.
These results build upon the recent FDA approval of CPP-1X or
DFMO to reduce the risk of relapse in adult and pediatric patients
with high-risk neuroblastoma (HRNB) who have demonstrated at least
a partial response to prior multiagent, multimodality therapy
including anti-GD2 immunotherapy. Results from these studies
suggest that CPP-1X is a safe, oral treatment option that may
improve response rates in heavily pretreated relapsed refractory
neuroblastoma patients and are the basis for the ongoing ANBL-1821
Phase 2 trial.
“We are excited about the publication of these Phase 1 trial
results in light of the recent DFMO FDA approval for patients in
maintenance therapy. From this dose escalation study, our
collaborators were able to demonstrate high dose DFMO is well
tolerated and demonstrated activity in patients with heavily
pretreated neuroblastoma,” said Elizabeth Bruckheimer, PhD, Vice
President & Chief Scientific Officer of Panbela. “Moreover,
three patients remain alive over four years from treatment end
without any additional therapy which suggests that high dose DFMO
treatment in combination with chemotherapy may be a potential
treatment option for this high unmet need population.”
"Overall, these results in addition to the recent approval of
DFMO as a maintenance therapy, suggests a role for polyamine
inhibition therapy for neuroblastoma that may impact other cancer
types such as prostate cancer. We are excited by these results and
the potential role for CPP-1X in the clinical management of
neuroblastoma and cancer as a whole,” said Dr. Bruckheimer. “These
studies were the basis for the ongoing Children’s Oncology Group
Phase II trial in relapsed refractory neuroblastoma to support the
goal of developing effective novel therapies for patients with
unmet medical needs.”
First author Michael Hogarty, MD, Professor of Pediatrics at the
University of Pennsylvania, and Children’s Hospital of Philadelphia
said, "The results from the Phase 1 study have built upon the
preclinical work performed in my laboratory demonstrating a role of
deep polyamine depletion as a potential therapeutic target for
relapsed or refractory neuroblastoma. By understanding the
underlying biology and role of MYC signaling and the polyamine
pathway in neuroblastoma, we are able to show the potential impact
of high-dose DFMO in neuroblastoma."
About PanbelaPanbela Therapeutics, Inc. is a
clinical-stage biopharmaceutical company developing disruptive
therapeutics for patients with urgent unmet medical needs.
Panbela’s lead assets are Ivospemin (SBP-101) and Flynpovi. Further
information can be found at www.panbela.com
. Panbela’s common stock is listed on The Nasdaq
Stock Market LLC under the symbol “PBLA”.
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program; (iii) the impact of the current COVID-19 pandemic on our
ability to conduct our clinical trials; (iv) our ability to
demonstrate the safety and effectiveness of our product candidates:
ivospemin (SBP-101) and eflornithine (CPP-1X); (v) our reliance on
a third party for the execution of the registration trial for our
product candidate Flynpovi ; (vi) our ability to obtain regulatory
approvals for our product candidates, SBP-101 and CPP-1X in the
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(vii) the market acceptance and level of future sales of our
product candidates, SBP-101 and CPP-1X; (viii) the cost and delays
in product development that may result from changes in regulatory
oversight applicable to our product candidates, SBP-101 and CPP-1X;
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Contact Information:
Investors:James CarbonaraHayden IR(646)
755-7412james@haydenir.com
Media:Tammy GroenePanbela Therapeutics, Inc.(952)
479-1196IR@panbela.com
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