Eflornithine

PRISM MarketView Highlights Work of Emerging Companies on Rare Disease Day

Retrieved on: 
Thursday, February 29, 2024
Rare disease, Therapy, Regenerative Medicine Advanced Therapy, FAP, Concha bullosa, Sulindac, Chemistry, Fast track (FDA), Muscular dystrophy, FDA, Sensation, News, Eflornithine, DMD, Prader–Willi syndrome, BLA, Chromosome, RDEB, PDUFA, Skin, Voyager, PRISM, Duchenne muscular dystrophy, Safety, Neurocrine Biosciences, Rare Disease Day, Genetics, Fatigue, Walking, PWS, Patient, DSM-IV codes, Medicine, Disease, Exosome, FA, CMC, Priority review, Pharmaceutical industry

All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.

Key Points: 
  • All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.
  • Today on Rare Disease Day, PRISM MarketView highlights emerging companies working to deliver life changing treatments for those living with rare diseases.
  • RDEB is a severe rare disease characterized by extremely fragile, and extensive blistering and wounds.
  • Capricor is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases.

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

Retrieved on: 
Monday, December 18, 2023
Lung cancer, National Cancer Institute, Partnership, Neuroblastoma, Research, FDA, Colorectal cancer, Polyamine, PBLA, Multiple myeloma, NDA, Prostate cancer, MSN, Doctor of Philosophy, Therapy, COG, USWM, Eflornithine, Cancer, New Drug Application, Patient, NCI, Pharmaceutical industry

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
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.

US WorldMeds Announces FDA Approval of IWILFIN™ (eflornithine) to Strengthen Fight Against Aggressive Childhood Cancer

Retrieved on: 
Thursday, December 14, 2023
Children, FDA, Health, General Health, Pharmaceutical, Oncology, Research, Family, Consumer, Science, Parenting, Neuroblastoma, Tenderness, Urine, Pneumonia, Huber loss, Somatic symptom disorder, Patient, Otitis media, Diagnosis, Tiffany Aching, Bruise, Liver, Chills, Hospital, Hearing loss, Vomiting, Food, Chair, American Cancer Society, EFS, Nose, Mortality, Multimedia, Division, Hematology, Pennsylvania State University, Blood, Diarrhea, Kool-Aid, Bleeding, Nausea, Development, Skin infection, Shivering, Urinary tract infection, Cough, Consortium, Risk, Sinusitis, Humble, Texas, Pain, Skin, Eflornithine, Hope, Upper respiratory tract infection, Disease, Abdomen, Tablet, Pediatric Hematology and Oncology, Sneeze, MD, Death, Immunotherapy, Pharmaceutical industry, Medical device, Nursing, Birth control, Phosphorus, Fever, USWM

High-risk neuroblastoma is a challenging disease, with a high mortality rate driven primarily by the risk of relapse after achieving remission.

Key Points: 
  • High-risk neuroblastoma is a challenging disease, with a high mortality rate driven primarily by the risk of relapse after achieving remission.
  • US WorldMeds partnered with the Beat Childhood Cancer Research Consortium at Penn State University, which conducted the preclinical and clinical research to help advance this vital therapy.
  • The Consortium represents a group of over 50 hospitals that offer collaboration through a network of childhood cancer clinical trials.
  • “We are thankful for the dedication of our partners, specifically the Beat Childhood Cancer Research Consortium, who work tirelessly to improve treatment outcomes for pediatric cancer patients.

Musely Enters New Era of "Look Good, Live Better" with Rebrand and New Treatments

Retrieved on: 
Wednesday, November 15, 2023
Cream, John Foster Dulles, Woman, Rx, Looking Good, Feeling Gorgeous, Oxymetazoline, Living Better Now, DNA, Chin, Beauty, Collection, Gynaecology, Estriol, Dry, Red, Patient, Ageing, Medicine, Stanford University Medical Center, Health, Blood, Elasticity, Rosacea, Growth, Obstetrics, Skin, Eflornithine, Face, Life, YOY, Femininity, Cosmetics, Pharmaceutical industry

SILICON VALLEY, Calif., Nov. 15, 2023 /PRNewswire-PRWeb/ -- Musely, the fastest-growing telemedicine skincare company offering personalized prescription skin and hair treatments from the convenience of one's home, has officially rebranded, entering a new chapter of its brand and Rx treatments under the new banner of "Look Good, Live Better". This pivotal moment for Musely marks an expansion into a new class of treatments that go beyond traditional cosmetic treatments, aiming to redefine the concept of beauty and wellness with a more comprehensive approach.

Key Points: 
  • Since Musely's 2019 launch with two FaceRx treatments, the brand has grown exponentially now offering a product lineup of 15+ highly effective and affordable treatments.
  • We are extremely proud to present our rebrand which reflects these pillars of our brand ethos.
  • Musely's new " Life Enhancer " collection is the debut assortment of treatments under the "Look Good, Live Better" banner.
  • The Aging Repair Cream will revolutionize the way aging while allowing women to maintain their divine femininity and youthful glow.

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.

Orbus Therapeutics Announces First Patient Enrolled in Phase 1 Clinical Study in Newly Diagnosed Glioblastoma

Retrieved on: 
Wednesday, September 6, 2023
Radiation, University, Temozolomide, Neuro-Oncology (journal), PALO, Eflornithine, Cancer, Pharmacokinetics, Toxicity, Doctor of Philosophy, PFS, Glioblastoma, ORR, Huntsman Cancer Institute, MD, Patient, Jon, Entrepreneurship, Lomustine, Neuro, Therapy, FAAN, Glioma, Safety, Pharmaceutical industry, Medical imaging

PALO ALTO, Calif., Sept. 06, 2023 (GLOBE NEWSWIRE) -- Orbus Therapeutics Inc., a private, late-stage biopharmaceutical company focused on the development and commercialization of therapies that treat rare diseases, announced today that the first patient has been enrolled in its Phase 1b study combining temozolomide and eflornithine in patients newly diagnosed with glioblastoma.

Key Points: 
  • PALO ALTO, Calif., Sept. 06, 2023 (GLOBE NEWSWIRE) -- Orbus Therapeutics Inc., a private, late-stage biopharmaceutical company focused on the development and commercialization of therapies that treat rare diseases, announced today that the first patient has been enrolled in its Phase 1b study combining temozolomide and eflornithine in patients newly diagnosed with glioblastoma.
  • Orbus Therapeutics is currently conducting a Phase 3 clinical trial, referred to as the STELLAR study, to evaluate its eflornithine oral solution in patients with anaplastic astrocytoma whose cancer has recurred following radiation and adjuvant temozolomide chemotherapy.
  • The initiation of this Phase 1b study is part of Orbus’ ongoing efforts to develop a new treatment option for patients with malignant glioma.
  • Temozolomide is the current standard of care in patients with newly diagnosed glioblastoma.”
    The Phase 1b study is an open-label, single arm, dose-limiting toxicity clinical trial that will include approximately 8 leading neuro-oncology clinical trial centers in the United States.

Panbela Announces Issuance of New Patent in Chile for Claims of a Novel Process for the Production of Flynpovi

Retrieved on: 
Tuesday, September 5, 2023
Therapy, MSN, Sulindac, Eflornithine, PBLA, FAP, Doctor of Philosophy, Patent, Patient, Sanofi, Growth, Pharmaceutical industry

This product, developed in collaboration with Sanofi, claims a novel composition of a fixed dose formulation of eflornithine and sulindac, which is called Flynpovi.

Key Points: 
  • This product, developed in collaboration with Sanofi, claims a novel composition of a fixed dose formulation of eflornithine and sulindac, which is called Flynpovi.
  • This is Panbela’s lead investigational product for the treatment of patients with Familial Adenomatous Polyposis (FAP).
  • Dr. Simpson added, “We are pleased with the continued growth of our patent portfolio with this latest patent issuance.
  • The co-formulated product described in this patent has the potential to be the first pharmacotherapy approved for patients with FAP.