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 preclinical and clinical data from studies of
CPP-1X (also known as α-Difluoromethylornithine (DFMO) or
Eflornithine) in recent onset type 1 diabetes (T1D). According to
Sims et al, although therapy of T1D has improved, the morbidity,
mortality and cost continue to impact the quality of life for those
affected highlighting the need for safe and effective therapies
that address the underlying pathology. Data published in the
journal Cell Reports Medicine investigated 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. Results showed that DFMO treatment may preserve β cell
function, reflected by C-peptide levels in patients with T1D
through the modulation of urinary polyamines, in particular
putrescine. The work reflects the Company’s ongoing collaboration
with Indiana University School of Medicine. A link to the
publication can be found here:
https://www.cell.com/cell-reports-medicine/fulltext/S2666-3791(23)00438-X.
The research is part of a multi-site clinical trial led by
Indiana University (IU) School of Medicine, supported by funding
from JDRF, the leading global 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.
From the Phase 1 dose range finding study of CPP-1X 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.
“By investigating β cell-specific deletion of ornithine
decarboxylase (ODC) in preclinical models, our collaborators were
able to demonstrate the protection against toxin-induced diabetes
which suggests a role for polyamine dysregulation in T1D.” said
Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief
Executive Officer of Panbela. “This observation was extended to the
clinical setting where results from the multi-site randomized,
placebo-controlled Phase I trial in patients with recent onset T1D
showed that inhibition of ODC by CPP-1X may improve β cell
function.”
"Overall, we are excited by these results which suggest that
CPP-1X may have a role in the clinical management of recent onset
type 1 diabetes.” said Dr. Simpson. “These studies were the basis
for the ongoing IU and JDRF Phase II trial in recent onset T1D to
support the goal of developing effective novel therapies for
patients with unmet medical needs.”
First author Emily K. Sims, MD, an associate professor of
pediatrics at IU School of Medicine and a pediatric endocrinologist
at Riley Children's Health said, "With our promising early
findings, we hold hope that DFMO, possibly as part of a combination
therapy, could offer potential benefits not only to individuals
with recent-onset type 1 diabetes but could ultimately also be
tested for potential benefit to delay disease onset in those who
are at risk of developing the condition."
Indiana University School of Medicine is annually ranked
among the top medical schools in the nation by U.S. News &
World Report. The school offers high-quality medical education,
access to leading medical research and rich campus life in nine
Indiana cities, including rural and urban locations consistently
recognized for livability.
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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