Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage
biopharmaceutical company developing disruptive therapeutics for
the treatment of patients with urgent unmet medical needs announces
a poster presentation highlighting the results for CPP-1X (also
known as α-Difluoromethylornithine (DFMO) or Eflornithine) in
recent onset type 1 diabetes at the Endocrine Society meeting,
which was held June 15-18, 2023. The work reflects the Company’s
ongoing collaboration with Indiana University (IU) School of
Medicine.
The research is part of a multi-site clinical trial led by
Indiana University School of Medicine, supported by funding from
JDRF, the leading global type 1 diabetes (T1D) research and
advocacy organization. The preclinical data were generated by
Raghavendra Mirmira's laboratory at the University of Chicago.
Panbela Therapeutics is providing the drug at no cost to
researchers and was not involved in the design and analysis of
these studies.
“These preclinical studies examined the role of ornithine
decarboxylase (ODC) on β cell stress that occurs in T1D. Results
showed that stressed human islet cells treated with CPP-1X had
alterations in several pathways such as antigen presentation, and
reactive oxygen species.” said Jennifer K. Simpson, PhD, MSN, CRNP,
President & Chief Executive Officer of Panbela. “Together with
data from recent onset T1D patients treated with CPP-1X in the
multi-site randomized, placebo-controlled Phase I trial, these
results suggest that inhibition of ODC by CPP-1X may preserve β
cell function in response to stress.”
"These results expand on the previously presented work
identifying potential mechanisms for CPP-1X and its potential role
in the clinical management of recent onset type 1 diabetes.” said
Dr. Simpson “We are excited to support the recently initiated IU
and JDRF-funded Phase II trial in recent onset T1D and the goal of
developing effective novel therapies for patients with unmet
medical needs.”
The poster investigates the mechanism of polyamines and
polyamine inhibition by CPP-1X on β cell stress that plays a role
in the onset of type 1 diabetes in in vitro and ex vivo models.
Human islet cells were either treated with vehicle or CPP-1X and
RNA sequencing and proteomics were performed. Results showed that
CPP-1X treatment of stressed human islets cells resulted in altered
pathways related to endoplasmic reticulum-related protein
processing, antigen presentation, and reactive oxygen species.
In vivo studies in mice with β cell-specific deletion of ODC showed
improved glucose tolerance, increased β cell mass, and reduced
incidence of diabetes following streptozotocin treatment.
In the Phase 1 dose range finding study of in patients with
recent onset T1D, CPP-1X was well tolerated and a dose dependent
inhibition of ODC was observed. An exploratory secondary analysis
showed that at the two highest dose levels, treatment with CPP-1X
stabilized C-peptide areas under the curve compared to placebo.
When assessing immune cell populations, there were no differences
between the placebo and CPP-1X patients.
Results from these studies suggest that CPP-1X is a safe, oral
treatment option that may improve β cell function and/or survival
in recent onset T1D.
Details of the presentation are as follows:
Poster Presentation
Title: Deletion or inhibition of Ornithine
Decarboxylase Protects Islet β-Cell Health and is Associated with
Preserved Residual C-peptide in Persons with Recent Onset Type 1
Diabetes
Abstract Control
Number: 7860Poster Board
Number: THU-277Session
Number: P14Session
Type: Poster AbstractSession
Title: Diabetes & Vascular
Disease: Clinical Diabetes
Additional meeting information can be found on the Endocrine
Society website:
https://www.endocrine.org/meetings-and-events/endo2023/program
The poster will also be available on the Company's website at
https://panbela.com/events-presentations/
About Panbela’s
PipelineThe pipeline consists of assets currently
in clinical trials with an initial focus on familial adenomatous
polyposis (FAP), first-line metastatic pancreatic cancer,
neoadjuvant pancreatic cancer, colorectal cancer prevention and
ovarian cancer. The combined development programs have a steady
cadence of anticipated catalysts with programs ranging from
pre-clinical to registration studies.
Ivospemin (SBP-101)Ivospemin
is a proprietary polyamine analogue designed to induce polyamine
metabolic inhibition (PMI) by exploiting an observed high affinity
of the compound for pancreatic ductal adenocarcinoma and other
tumors. It has shown signals of tumor growth inhibition in clinical
studies of metastatic pancreatic cancer patients, demonstrating a
median overall survival (OS) of 14.6 months and an objective
response rate (ORR) of 48%, both exceeding what is typical for the
standard of care of gemcitabine + nab-paclitaxel suggesting
potential complementary activity with the existing FDA-approved
standard chemotherapy regimen. In data evaluated from clinical
studies to date, ivospemin has not shown exacerbation of bone
marrow suppression and peripheral neuropathy, which can be
chemotherapy-related adverse events. Serious visual adverse events
have been evaluated and patients with a history of retinopathy or
at risk of retinal detachment will be excluded from future SBP-101
studies. The safety data and PMI profile observed in the previous
Panbela-sponsored clinical trials provide support for continued
evaluation of ivospemin in the ASPIRE trial.
Flynpovi™Flynpovi is a
combination of CPP-1X (eflornithine) and sulindac with a dual
mechanism inhibiting polyamine synthesis and increasing polyamine
export and catabolism. In a Phase III clinical trial in patients
with sporadic large bowel polyps, the combination prevented >
90% subsequent pre-cancerous sporadic adenomas versus placebo.
Focusing on FAP patients with lower gastrointestinal tract anatomy
in the recent Phase III trial comparing Flynpovi to single agent
eflornithine and single agent sulindac, FAP patients with lower GI
anatomy (patients with an intact colon, retained rectum or surgical
pouch), showed statistically significant benefit compared to both
single agents (p≤0.02) in delaying surgical events in the lower GI
for up to four years. The safety profile for Flynpovi did not
significantly differ from the single agents and supports the
continued evaluation of Flynpovi for FAP.
CPP-1XCPP-1X (eflornithine) is being developed
as a single agent tablet or high dose powder sachet for several
indications including prevention of gastric cancer, treatment of
neuroblastoma and recent onset Type 1 diabetes. Preclinical studies
as well as Phase I or Phase II investigator-initiated trials
suggest that CPP-1X treatment may be well-tolerated and has
potential activity.
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”.
Cautionary Statement Regarding Forward-Looking
Statements This press release contains “forward-looking
statements,” including within the meaning of the Private Securities
Litigation Reform Act of 1995. Forward-looking statements can be
identified by words such as: “anticipate,” “can,” “continue,”
“design,” “expect,” “focus,” “intend,” “may,” “plan,” “potential,”
and “will.” All statements other than statements of historical fact
are statements that should be deemed forward-looking statements.
Forward-looking statements are neither historical facts nor
assurances of future performance. Instead, they are based only on
our current beliefs, expectations, and assumptions regarding the
future of our business, future plans and strategies, projections,
anticipated events and trends, the economy and other future
conditions. Because forward-looking statements relate to the
future, they are subject to inherent uncertainties, risks and
changes in circumstances that are difficult to predict and many of
which are outside of our control. Our actual results and financial
condition may differ materially and adversely from the
forward-looking statements. Therefore, you should not rely on any
of these forward-looking statements. Important factors that could
cause our actual results and financial condition to differ
materially from those indicated in the forward-looking statements
include, among others, the following: (i) our ability to obtain
additional funding to execute our business and clinical development
plans; (ii) progress and success of our clinical development
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
United States, the European Union or other international markets;
(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;
(ix) the rate of progress in establishing reimbursement
arrangements with third-party payors; (x) the effect of competing
technological and market developments; (xi) the costs involved in
filing and prosecuting patent applications and enforcing or
defending patent claims; (xii) our ability to maintain the listing
of our common stock on a national securities exchange; and (xiii)
such other factors as discussed in Part I, Item 1A under the
caption “Risk Factors” in our most recent Annual Report on Form
10-K, any additional risks presented in our Quarterly Reports on
Form 10-Q and our Current Reports on Form 8-K. Any forward-looking
statement made by us in this press release is based on information
currently available to us and speaks only as of the date on which
it is made. We undertake no obligation to publicly update any
forward-looking statement or reasons why actual results would
differ from those anticipated in any such forward-looking
statement, whether written or oral, whether as a result of new
information, future developments or otherwise.
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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