Polyamine

Panbela Therapeutics Announces US WorldMeds NDA Approval for Eflornithine (DFMO) in Pediatric Neuroblastoma

Retrieved on: 
Monday, December 18, 2023

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.

Key Points: 
  • 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.
  • 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.

Panbela Announces Publication of Abstract Titled: Evaluation of Myeloma Cell Lines Viability Following Administration of SBP-101 and DFMO Polyamine Inhibitors

Retrieved on: 
Monday, December 4, 2023

Data published in the November supplemental issue of the Journal Blood investigated the effects of polyamine inhibition by ivospemin and CPP-1X on myeloma cell lines growth and viability in vitro.

Key Points: 
  • Data published in the November supplemental issue of the Journal Blood investigated the effects of polyamine inhibition by ivospemin and CPP-1X on myeloma cell lines growth and viability in vitro.
  • Results showed that ivospemin and CPP-1X treatment significantly decreased cell proliferation and induced apoptosis in a panel of multiple myeloma cell lines.
  • When ivospemin and CPP-1X were combined an almost complete abolition of cell growth occurred.
  • “We are excited to have initiated the studies evaluating our polyamine inhibitors ivospemin and CPP-1X in models of multiple myeloma.

Panbela Announces Publication of Preclinical and Clinical Data Titled: Inhibition of Polyamine Biosynthesis Preserves β Cell Function in Type 1 Diabetes

Retrieved on: 
Thursday, November 2, 2023

A Phase 1 clinical study showed that DFMO treatment may provide metabolic benefits to preserve β cell function and health in T1D.

Key Points: 
  • A Phase 1 clinical study showed that DFMO treatment may provide metabolic benefits to preserve β cell function and health in T1D.
  • 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.
  • 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.

Panbela Therapeutics to Receive a Total Up to $9.5 Million for Divestiture of Assets within Eflornithine (DFMO) Pediatric Neuroblastoma Program to US WorldMeds

Retrieved on: 
Wednesday, July 19, 2023

Neuroblastoma, a rare cancer originating from immature nerve cells, contributes to nearly 15% of pediatric cancer deaths.

Key Points: 
  • Neuroblastoma, a rare cancer originating from immature nerve cells, contributes to nearly 15% of pediatric cancer deaths.
  • Under the terms of the agreement, Panbela is entitled to receive up to approximately $9.5 million non-dilutive funding in exchange for the sale of certain assets within its pediatric neuroblastoma program for eflornithine.
  • Panbela will receive payments upon USWM’s successful completion of milestones related to eflornithine's clinical development, regulatory approval, and commercial sales.
  • “Divesting eflornithine assets for pediatric neuroblastoma is another milestone in executing our business plan to generate long-term value for our shareholders.

Panbela Announces Poster Presentation at the Endocrine Society Meeting: Polyamine Inhibition and β-Cell Preservation in Recent Onset Type 1 Diabetes

Retrieved on: 
Monday, June 26, 2023

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.

Key Points: 
  • 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.
  • Panbela Therapeutics is providing the drug at no cost to researchers and was not involved in the design and analysis of these studies.
  • 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.
  • 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.

Panbela Announces Sponsored Research Agreement to Evaluate Polyamine Metabolic Inhibitor Therapy in Combination with CAR-T Cell Therapy

Retrieved on: 
Tuesday, June 13, 2023

The initial goal of these studies will be to ascertain if eflornithine and/or ivospemin treatment will augment CAR-T mediated cytotoxicity against CD19+ large B-cell lymphoma (LBCL) cell lines.

Key Points: 
  • The initial goal of these studies will be to ascertain if eflornithine and/or ivospemin treatment will augment CAR-T mediated cytotoxicity against CD19+ large B-cell lymphoma (LBCL) cell lines.
  • Recently, a metabolite panel primarily consisting of polyamines was identified as predictive of poor response to anti-CD19 CAR T-cell therapy in relapsed refractory LBCL.
  • Together, this suggests the potential for a polyamine targeted therapy in combination with CAR-T therapies.
  • “While the literature has demonstrated the relationship between polyamines and the immune system, recent research shows that an elevated polyamine metabolic profile correlated to poor response to CAR-T cell therapy.

Panbela Announces Poster Presentation at Immunology of Diabetes Society Meeting: Evaluating the Potential of CPP-1X (Eflornithine) in Recent Onset Type 1 Diabetes

Retrieved on: 
Thursday, June 1, 2023

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 type 1 diabetes (T1D) research and advocacy organization.

Key Points: 
  • 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 type 1 diabetes (T1D) research and advocacy organization.
  • Panbela Therapeutics is providing the drug at no cost to researchers and was not involved in the design and analysis of these studies.
  • 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:
    Additional meeting information can be found on the IDS website: https://www.idsparis2023.com/_files/ugd/d65629_717d6a377fae45dc95b0ebb26...

Panbela Announces Clinical Trial with Moffitt Cancer Center for Phase I/II Program in STK11 Mutant Non-Small Cell Lung Cancer

Retrieved on: 
Monday, May 22, 2023

MINNEAPOLIS, May 22, 2023 (GLOBE NEWSWIRE) -- Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, today announced it has entered into a clinical trial agreement with Moffitt Cancer Center for a Phase I/II program in STK11 mutant non-small cell lung cancer (NSCLC).

Key Points: 
  • MINNEAPOLIS, May 22, 2023 (GLOBE NEWSWIRE) -- Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, today announced it has entered into a clinical trial agreement with Moffitt Cancer Center for a Phase I/II program in STK11 mutant non-small cell lung cancer (NSCLC).
  • The initial goal of the Phase I trial will be to ascertain the maximum tolerated dose of eflornithine, while evaluating efficacy and then moving into a Phase II efficacy trial.
  • We anticipate data from the Phase I trial by the end of this year with a look to start the Phase II trial at the end of the year or early 2024.
  • We know that STK11 mutant tumors have reduced levels of T cells that direct immune surveillance, and they can avoid immune detection.

FUJIFILM Wako Chemicals U.S.A. Commercializes CEPT Cocktail for Research-Use iPSC Applications

Retrieved on: 
Thursday, April 20, 2023

CEPT Cocktail is intended for commercial research-use of iPSC applications including disease modeling, organoid generation, drug discovery, toxicology, and pharmacological studies.

Key Points: 
  • CEPT Cocktail is intended for commercial research-use of iPSC applications including disease modeling, organoid generation, drug discovery, toxicology, and pharmacological studies.
  • Compared to other commercially available reagents, FUJIFILM Wako Chemicals U.S.A., Corporation’s CEPT Cocktail consists of a mixture of all the components, providing comprehensive cell protection and viability.
  • Under a license granted from the National Center for Advancing Translational Sciences (NCATS) and NIH, Fujifilm commercializes CEPT cocktail as a research-use-only, ready-to-use DMSO solution.
  • “As part of a robust offering of iPSC technologies and cell culture media solutions, FUJIFILM Wako Chemicals U.S.A., Corporation is proud to commercialize the CEPT Cocktail with the hope of further advancing the role of iPSCs and their limitless potential in translational medicine,” said Toshihiro Hiramatsu, president, FUJIFILM Wako Chemicals, U.S.A., Corporation.

First Patient Enrolled in JDRF-Funded Phase II Trial of CPP-1X-T for Recent Onset Type I Diabetes, Led by Indiana University School of Medicine

Retrieved on: 
Wednesday, April 12, 2023

The multi-site clinical trial is led by researchers at Indiana University School of Medicine and funded by JDRF, the leading global type 1 diabetes research and advocacy organization.

Key Points: 
  • The multi-site clinical trial is led by researchers at Indiana University School of Medicine and funded by JDRF, the leading global type 1 diabetes research and advocacy organization.
  • The trial entitled “TArgeting Type 1 Diabetes Using POLyamines (TADPOL)” is a double-blind placebo-controlled clinical trial to evaluate CPP-1X-T vs placebo in patients with recent onset type 1 diabetes at approximately 6 academic medical centers in the United States.
  • “We’re really excited to have the first patient enrolled in the Phase II trial for CPP-1X-T led by Indiana University School of Medicine and funded by JDRF, the leading global organization advancing life-changing breakthroughs for type 1 diabetes (T1D),” said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela.
  • 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.