Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company
developing disruptive therapeutics for the treatment of patients
with urgent unmet medical needs, today announced that
US
WorldMeds®1 (USWM), a Kentucky-based
specialty pharmaceutical company to whom it divested certain assets
in its eflornithine pediatric neuroblastoma program, received FDA
approval of its New Drug Application (NDA) for the use of
eflornithine as a maintenance therapy for high-risk neuroblastoma
patients who have achieved at least a partial response to certain
prior therapies.
The approval of USWM’s NDA for the use of eflornithine for the
treatment of patients with high-risk neuroblastoma marks the first
FDA approval of an NDA for any polyamine targeted therapy in a
cancer indication. “This approval highlights the role polyamines
can play in cancer therapy as we look forward to data from our
ongoing programs in metastatic pancreatic cancer, colorectal
cancer, non-small cell lung cancer, and prostate cancer and the
advancement of pre-clinical programs in ovarian and multiple
myeloma,” said Jennifer K. Simpson, PhD, MSN, CRNP, President &
Chief Executive Officer of Panbela.
In July 2023, Panbela divested its pediatric neuroblastoma
program to USWM in an arrangement entitling Panbela to up to
approximately $9.5 million of non-dilutive funding, including
payments upon USWM’s successful completion of milestones related to
eflornithine's clinical development, regulatory approval, and
commercial sales.
“The FDA’s approval of USWM’s eflornithine NDA for high-risk
neuroblastoma is an exciting milestone in our partnership. This
demonstrates the potential for polyamine targeted therapies in
cancer,” said Jennifer K. Simpson, PhD, MSN, CRNP, President &
Chief Executive Officer of Panbela. “This approval is a
prerequisite for considerable development milestone payments for
Panbela as US WorldMeds continues its efforts to bring eflornithine
to the market.”
“After many years investigating the role of polyamines and the
therapeutic potential of eflornithine in neuroblastoma, it is
rewarding to see USWM’s success with the FDA’s positive review of
its NDA for eflornithine,” said Elizabeth Bruckheimer, Ph.D., Vice
President & Chief Scientific Officer of Panbela, “We are
excited about how the USWM NDA approval of eflornithine should help
address this high unmet need in patients with neuroblastoma.”
Neuroblastoma, a rare cancer originating from immature nerve
cells, contributes to nearly 15% of pediatric cancer deaths.[1]
Panbela's subsidiary, Cancer Prevention Pharmaceuticals,
extensively collaborated with leading neuroblastoma research groups
such as the Neuroblastoma Medulloblastoma Translational Research
Consortium (NMTRC) (now Beat Childhood Cancer), New Advances in
Neuroblastoma Therapy (NANT), the Children’s Oncology Group (COG),
and the National Cancer Institute (NCI) in the clinical development
of eflornithine as a treatment for neuroblastoma before the program
was divested to USWM.
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 3 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 3 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.
Eflornithine (CPP-1X)CPP-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 1 or Phase 2
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
StatementsThis 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,”
“design,” “may,” “plan,” and “will.” Examples of forward-looking
statements include statements we make regarding timing of
trials and results of collaborations with third parties and future
studies. 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) our lack of diversification and
the corresponding risk of an investment in our Company; (iii) our
ability to maintain our listing on a national securities exchange;
(iv) the progress and success of our clinical development program;
(v) our ability to demonstrate the safety and effectiveness of our
product candidates: ivospemin (SBP-101), Flynpovi, and
eflornithine (CPP-1X); (vi) our ability to obtain
regulatory approvals for our product candidates, SBP-101, Flynpovi
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, Flynpovi
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, Flynpovi 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; and (xii) 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
References: [1] Giselle L. et al. Maintenance DFMO
Increases Survival in High Risk Neuroblastoma. Scientific
Reports, 2018; 8 (1) DOI: 10.1038/s41598-018-32659-w
1 US WORLDMEDS is a registered trademark of USWM, LLC.
Panbela Therapeutics (NASDAQ:PBLA)
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Panbela Therapeutics (NASDAQ:PBLA)
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