DFMO

Norgine B.V. submits Marketing Authorisation Application via Project Orbis for eflornithine (difluoromethylornithine [DFMO]) in high-risk neuroblastoma

Retrieved on: 
Monday, April 15, 2024
Orbis, FDA, Risk, SOC, DFMO, Patient, USWM, Standard of care, Eflornithine, Pharmaceutical industry

AMSTERDAM, April 15, 2024 /PRNewswire/ -- Norgine B.V. today announced its first marketing authorisation application submissions on 10 April 2024, seeking approval for eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom.

Key Points: 
  • AMSTERDAM, April 15, 2024 /PRNewswire/ -- Norgine B.V. today announced its first marketing authorisation application submissions on 10 April 2024, seeking approval for eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom.
  • This milestone supports Norgine's efforts to deliver patient access to eflornithine and bring a further treatment option in the field of paediatric oncology.
  • Norgine and USWM, LLC (dba US WorldMeds), a Kentucky-based specialty pharmaceutical company, have an exclusive licensing agreement by which Norgine will register and commercialise eflornithine, also referred to as DFMO, in Europe, Australia and New Zealand.
  • On 13 December 2023, the US Food and Drug Administration (FDA) approved eflornithine as the first oral maintenance therapy for high-risk neuroblastoma (HRNB), indicated to reduce the risk of relapse in adult and paediatric patients who have received certain prior therapies.1 The approval decision was based on findings from a trial comparing outcomes from Study 3b (NCT02395666; investigational arm)2,4 and Study ANBL0032 (NCT00026312; clinical-trial-derived external control arm)3,4, where use of eflornithine resulted in improved event-free survival and overall survival when compared to outcomes for  patients with high-risk neuroblastoma treated with the standard of care (SoC) without the drug.1
    Dr David Gillen, Chief Medical Officer at Norgine, added, "These submissions via Project Orbis represent an important first step in the regulatory process for eflornithine and re-emphasise Norgine's passion and commitment in attempting to secure additional treatment options for patients living with HRNB, a condition with a high level of unmet medical need."

Panbela Provides Business Update and Reports Q4 and FY 2024 Financial Results

Retrieved on: 
Tuesday, March 26, 2024
Research, Topotecan, PBLA, Neuroblastoma, Patient, Data monitoring committee, Acquisition, Celecoxib, Doctor of Philosophy, AACR, MSN, CPP, Pancreatic cancer, Therapy, DSMB, Poster, DFMO, Cyclophosphamide, Pharmaceutical industry

MINNEAPOLIS, March 26, 2024 (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 provides a business update and reports financial results for the quarter and full year ended December 31, 2023.

Key Points: 
  • MINNEAPOLIS, March 26, 2024 (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 provides a business update and reports financial results for the quarter and full year ended December 31, 2023.
  • “Looking ahead, Panbela remains unwavering in its commitment to patients and in its pursuit of maximizing value for stockholders.
  • Notes payable, plus accrued interest, on the balance sheet, the result of the acquisition of CPP, totaled approximately $5.4 million.
  • Gross proceeds from the raise, which closed on January 31, 2024, were approximately $9 million.

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

Retrieved on: 
Thursday, January 18, 2024
DFMO, Pediatrics, Patient, Therapy, Biology, MD, PBLA, Prostate cancer, University, Celecoxib, Neuroblastoma, Risk, PD, PFS, FDA, Doctor of Philosophy, MYC, Pharmaceutical industry

According to Hogarty et al, children with relapsed refractory neuroblastoma have dismal outcomes and new therapeutic options are needed.

Key Points: 
  • According to Hogarty et al, children with relapsed refractory neuroblastoma have dismal outcomes and new therapeutic options are needed.
  • The work reflects the Company’s previous collaboration with New Advances in Neuroblastoma Therapy Consortium (NANT) (https://www.nant.org/).
  • From the Phase 1 dose range finding study of CPP-1X in heavily pretreated neuroblastoma patients, CPP-1X was well tolerated.
  • “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.

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
Immune system, Research, Apoptosis, Polyamine, PBLA, Multiple myeloma, MD Anderson Cancer Center, MSN, Doctor of Philosophy, Cell proliferation, Literature, Therapy, Eflornithine, DFMO, STK11, Patient, University

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 Provides Business Update and Reports Q3 2023 Financial Results

Retrieved on: 
Thursday, November 9, 2023
Pancreatic cancer, MSN, Acquisition, Safety, Oncology Drug Advisory Committee, Therapy, Castration, Data monitoring committee, Exercise, Clinical trial, Sanofi, Patient, DSMB, Intellectual property, Research, ODAC, Patent, DFMO, Doctor of Philosophy, CPP, PBLA, Eflornithine, Pharmaceutical industry

MINNEAPOLIS, Nov. 09, 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 provides a business update and reports financial results for the quarter ended September 30, 2023.

Key Points: 
  • MINNEAPOLIS, Nov. 09, 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 provides a business update and reports financial results for the quarter ended September 30, 2023.
  • The independent Data Safety Monitoring Board (DSMB) of the ASPIRE trial completed its pre-specified review of safety data for treated patients in the trial.
  • The aggregate gross proceeds from the exercise of the existing warrants totaled approximately $1.9 million, before deducting financial advisory fees.
  • Third Quarter ended September 30, 2023 Financial Results
    General and administrative expenses were $1.1 million in the third quarter of 2023, compared to $1.3 million in the third quarter of 2022.

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
Survival, PBLA, Indiana University School of Medicine, Health, JDRF, Therapy, Hope, Mortality, MD, Pathology, Doctor of Philosophy, Disease, Research, Observation, Pediatrics, Riley Children's Foundation, News, Quality of life, Polyamine, School, Risk, Putrescine, University, U.S. News & World Report, ODC, Indiana University, Patient, MSN, Ornithine decarboxylase, DFMO, Pharmaceutical industry, Vaccine, Eflornithine, Medicine, T1D

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.

Acceptance of Polyamine Inhibitor CAR-T Combination Abstract for Online Publication

Retrieved on: 
Wednesday, October 25, 2023
ASH, Therapy, University, PBLA, MD Anderson Cancer Center, Patient, Multiple myeloma, Research, DFMO, Society, Ashes and Blood, Pharmaceutical industry, Online shopping, Blood, Eflornithine

MINNEAPOLIS, Oct. 25, 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 that an abstract about SBP-101 and CPP-1X (also known as DFMO or Eflornithine) research in multiple myeloma (cell lines), has been accepted for an online publication on the American Society of Hematology (ASH) meeting site in the November supplemental issue of the journal Blood.

Key Points: 
  • MINNEAPOLIS, Oct. 25, 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 that an abstract about SBP-101 and CPP-1X (also known as DFMO or Eflornithine) research in multiple myeloma (cell lines), has been accepted for an online publication on the American Society of Hematology (ASH) meeting site in the November supplemental issue of the journal Blood.
  • The work reflects the company’s on-going collaboration with researchers from The University of Texas MD Anderson Cancer Center and will become part of the permanent ASH and Blood abstracts archive.
  • Additional meeting information can be found on the ASH website: https://www.hematology.org/meetings/annual-meeting/abstracts
    The abstract will also be available on the company's website at https://panbela.com/events-presentations/ once the information has been released by ASH.

Panbela Provides Business Update and Reports Q2 2023 Financial Results

Retrieved on: 
Thursday, August 10, 2023
Pancreatic cancer, Abstract, JDRF, AACR, Therapy, DSMB, MSN, Diabetes, Eflornithine, Clinical trial, Advanced Materials and Processes Research Institute, American Association for Cancer Research, PBLA, FAP, Research, NSCLC, Doctor of Philosophy, IDS, MD Anderson Cancer Center, Data monitoring committee, CPP, Acquisition, Patent, Patient, Colorectal cancer, University, DFMO, Endocrine Society, Immunology, H. Lee Moffitt Cancer Center & Research Institute, Ovarian cancer, Safety, Johns Hopkins School of Medicine, Pharmaceutical industry, Life insurance, STK11

MINNEAPOLIS , Aug. 10, 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 provides a business update and reports financial results for the quarter ended June 30, 2023.

Key Points: 
  • MINNEAPOLIS , Aug. 10, 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 provides a business update and reports financial results for the quarter ended June 30, 2023.
  • Poster presentation highlighting the results for CPP-1X in recent onset type 1 diabetes at the Endocrine Society meeting in June 2023.
  • Research and development expenses were $4.2 million in the second quarter of 2023, compared to $20.0 million in the second quarter of 2022.
  • Notes payable, plus accrued interest, on the balance sheet, the result of the acquisition of CPP, totaled approximately $5.3 million.

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
Therapy, MSN, USWM, Polyamine, Eflornithine, New Drug Application, Cancer, COG, PBLA, Research, Doctor of Philosophy, Sale, National Cancer Institute, Patient, The News & Advance, DFMO, FDA, NCI, Pharmaceutical industry, Neuroblastoma

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
IU, JDRF, Indiana University, University, Therapy, Diabetes, MSN, Polyamine, Survival, PBLA, Research, Cell, RNA, Doctor of Philosophy, Proteomics, Incidence, Indiana University School of Medicine, Patient, Antigen presentation, ODC, Reactive oxygen species, DFMO, School, Endocrine Society, Mouse, Poster, Ornithine decarboxylase, Pharmaceutical industry, Vaccine, T1D, Eflornithine, 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.

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.