Osteosarcoma

Lisata Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to LSTA1 for the Treatment of Osteosarcoma

Retrieved on: 
Tuesday, April 9, 2024
Disease, Osteosarcoma, Patient, Orphan, Food and Drug Administration, Cancer, Therapy, FDA, Food, Pharmaceutical industry

LSTA1 recently received Rare Pediatric Disease Designation for osteosarcoma, as announced by the Company on March 21, 2024.

Key Points: 
  • LSTA1 recently received Rare Pediatric Disease Designation for osteosarcoma, as announced by the Company on March 21, 2024.
  • “We are thrilled to have received another favorable regulatory designation from the FDA.
  • “Osteosarcoma, while rare, is a type of bone cancer that is often associated with high morbidity, early metastasis, rapid progression, and poor prognosis.
  • In addition to the financial benefits, it also may potentially shorten clinical development due to closer collaboration with the FDA.

Lisata Therapeutics Announces U.S. FDA Rare Pediatric Disease Designation Granted to LSTA1 for the Treatment of Osteosarcoma

Retrieved on: 
Thursday, March 21, 2024
Osteosarcoma, Food and Drug Administration, NDA, Cancer, Therapy, FDA, Food, Pharmaceutical industry

BASKING RIDGE, N.J., March 21, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced that the U.S. Food and Drug Administration (the “FDA”) has granted Rare Pediatric Disease Designation (“RPDD”) to LSTA1, the Company’s lead product candidate, for the treatment of osteosarcoma, a rare cancer that can develop in children, adolescents and young adults.

Key Points: 
  • BASKING RIDGE, N.J., March 21, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced that the U.S. Food and Drug Administration (the “FDA”) has granted Rare Pediatric Disease Designation (“RPDD”) to LSTA1, the Company’s lead product candidate, for the treatment of osteosarcoma, a rare cancer that can develop in children, adolescents and young adults.
  • A substantial benefit of a RPDD is receipt of a priority review voucher, often referred to as a “golden ticket,” once the FDA approves the new drug application (“NDA”) for the product and indication having received the RPDD.
  • The voucher may be used by the sponsor or sold to another sponsor for their use.
  • Priority review vouchers have sold for as much as $350 million USD historically and, more recently, have sold for $75 to $100 million USD.

Fennec Pharmaceuticals Reports Preliminary Unaudited Net Revenue for Fourth Quarter and Full-Year 2023 Results

Retrieved on: 
Thursday, February 29, 2024
Incidence, Osteosarcoma, Fennec, FDA, TSX, Food, Neuroblastoma, Germ cell tumor, Hepatoblastoma, Risk, Literacy, Safety, Audit, Cisplatin, Hearing loss, Medulloblastoma, Kool-Aid, Ototoxicity, Food and Drug Administration, Patient, Survival, FENC, COG

RESEARCH TRIANGLE PARK, N.C., Feb. 29, 2024 (GLOBE NEWSWIRE) -- Fennec Pharmaceuticals Inc. (NASDAQ: FENC; TSX: FRX), a commercial stage specialty pharmaceutical company, today announced preliminary unaudited fourth quarter and full-year 2023 net revenues.

Key Points: 
  • ~ Company Expects to Report 2023 Fourth Quarter and Audited Full-Year Results on or about March 26, 2024 ~
    RESEARCH TRIANGLE PARK, N.C., Feb. 29, 2024 (GLOBE NEWSWIRE) -- Fennec Pharmaceuticals Inc. (NASDAQ: FENC; TSX: FRX), a commercial stage specialty pharmaceutical company, today announced preliminary unaudited fourth quarter and full-year 2023 net revenues.
  • The information in this press release is based upon preliminary unaudited information and management estimates for the fourth quarter 2023 and is subject to the completion of Fennec’s financial closing procedures and year end audit.
  • Preliminary Unaudited 2023 Fourth Quarter Revenue and Year End Performance:
    Fourth quarter 2023 net revenues are expected to be approximately $9.2 to $9.7 million, which represents approximately a 41-49% increase over the third quarter of 2023.
  • Fennec expects to report its 2023 fourth quarter and audited full-year year results of operations on or about March 26, 2024.

St. Baldrick's Foundation Allocates $1.1 Million for Fellowships to Empower Emerging Childhood Cancer Researchers

Retrieved on: 
Friday, March 8, 2024
Neoplasm, Cancer, Survivor, Toxicity, Osteosarcoma, Immunotherapy, Children's Hospital of Philadelphia, University, University of California, Canadian Aviation Regulations, Hospital, Brain, Child, Food, Vaccine, Cell adhesion, Bone marrow, Dana–Farber Cancer Institute, Leukemia, Medicine, Baylor College of Medicine, Acute myeloid leukemia, Food security, Lymphoma, Research, Baylor University, Immune system

The St. Baldrick's Foundation granted over $1.1 million in Fellow awards to nurture emerging childhood cancer researchers dedicated to overcoming pediatric cancers.

Key Points: 
  • The St. Baldrick's Foundation granted over $1.1 million in Fellow awards to nurture emerging childhood cancer researchers dedicated to overcoming pediatric cancers.
  • The $1.1 million was distributed to seven researchers making strides in discovering new cures for pediatric cancers.
  • The St. Baldrick's Foundation 2024 Fellows are:
    Dr. Vanja Cabric at Memorial Sloan Kettering Cancer Center, New York, NY
    Hepatoblastoma, common in childhood, lacks effective immunotherapy due to limited understanding of pediatric immune response to tumors.
  • Since 2005, St. Baldrick's has granted more than $342 million to support the development of childhood cancer treatments that have the potential to impact every kid diagnosed with cancer.

Genprex Collaborators to Present Positive Preclinical Data on the Use of Reqorsa® and on NPRL2 Gene Therapy Utilizing Non-Viral Oncoprex® Delivery System for the Treatment of Lung Cancers at the 2024 AACR Annual Meeting

Retrieved on: 
Wednesday, March 6, 2024
Glioblastoma, NSCLC, Diabetes, Lung, Lung cancer, Metabolism, EGFR, Osteosarcoma, MD, Neoplasm, FDA, Cancer, Glycolysis, Food, Growth, Anaplastic lymphoma kinase, Pembrolizumab, AACR, Cell, Apoptosis, Date, Osimertinib, Agonal respiration, Mitochondrion, Breast, University, Nanoparticle, Food and Drug Administration, NPRL2, Patient, ATP, Clinical trial, Gene, Disease, TUSC2, Trial of the century, Mesothelioma, Research, ALK, Vaccine

AUSTIN, Texas, March 6, 2024 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that its research collaborators will present at the upcoming 2024 American Association for Cancer Research (AACR) Annual Meeting being held April 5-10, 2024 in San Diego, California. The collaborators will present positive preclinical data from studies of its lead product candidate, Reqorsa® Immunogene Therapy (quaratusugene ozeplasmid), as well as NPRL2 gene therapy, which both utilize the Company's non-viral Oncoprex® Delivery System for the treatment of lung cancer.

Key Points: 
  • The collaborators will present positive preclinical data from studies of its lead product candidate, Reqorsa® Immunogene Therapy (quaratusugene ozeplasmid), as well as NPRL2 gene therapy, which both utilize the Company's non-viral Oncoprex® Delivery System for the treatment of lung cancer.
  • In this humanized mouse model, researchers investigated the anti-tumor immune responses to NPRL2 gene therapy in pembrolizumab resistant KRAS/STK11mt NSCLC.
  • "Today's bolus of compelling data validates the potential of REQORSA and the ONCOPREX Delivery System as innovative cancer treatments.
  • We look forward to continuing to evaluate the ONCOPREX Delivery System using both REQORSA and NPRL2 as potential treatments for lung cancer."

Novel lower-cost blood test for cancer screening in dogs validated in large clinical study by PetDx

Retrieved on: 
Friday, February 9, 2024
Carcinoma, SAN, Cancer, American Veterinary Medical Association, Liquid biopsy, Leukemia, Lymphoma, DNA, Risk, Hemangiosarcoma, Journal, Osteosarcoma, Veterinary medicine, Dog, Histiocytic sarcoma, JAVMA, Medical imaging

SAN DIEGO, Feb. 9, 2024 /PRNewswire/ -- PetDx® – The Liquid Biopsy Company for Pets™ published a clinical validation study this week showing that its new OncoK9® Screen test has a high detection rate across seven predefined, clinically relevant cancer types that represent a large proportion of all cancers found in dogs and are often difficult to detect by physical exam alone due to their anatomic location and/or lack of specific clinical signs. These cancer types are often aggressive, requiring prompt intervention, and have typically been associated with improved outcomes when detected and treated early.

Key Points: 
  • By integrating cell-free DNA quantification and next-generation sequencing using a proprietary method, the new test can be performed faster and at lower cost than the company's flagship OncoK9® Dx test, allowing for broader access to blood-based cancer screening in dogs.
  • These cancer types are often aggressive, requiring prompt intervention, and have typically been associated with improved outcomes when detected and treated early.
  • "Previously published research has shown that only around 10% of cancer cases are currently detected in dogs prior to the onset of clinical signs.
  • "The availability of comprehensively validated blood-based cancer detection tools that can be readily incorporated into preventive care protocols opens the door to the development of cancer screening guidelines for early cancer detection in dogs."

Telix Signs Agreement to Acquire QSAM Biosciences and Its Bone Cancer Targeting Platform

Retrieved on: 
Wednesday, February 7, 2024
PRV, Steroid, Prostate cancer, COGS, Contingent value rights, TLX, Palliative care, Osteosarcoma, Patient, Safety, Nephrotoxicity, QSAM, FDA, Appendix, Sarcoma, Food, Term sheet, Brain, Disease, Pain management, Acquisition, Metastasis, Samarium, ODD, Radiopharmaceutical, Aplastic anemia, Glioma, ASX, Priority review, Medical imaging

MELBOURNE, Australia, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) today announces it has entered into an agreement to acquire QSAM Biosciences, Inc. (U.S. OTC: QSAM) and its lead investigational drug Samarium-153-DOTMP (153Sm-DOTMP).

Key Points: 
  • MELBOURNE, Australia, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) today announces it has entered into an agreement to acquire QSAM Biosciences, Inc. (U.S. OTC: QSAM) and its lead investigational drug Samarium-153-DOTMP (153Sm-DOTMP).
  • QSAM is a United States (U.S.) based company developing therapeutic radiopharmaceuticals for primary and metastatic bone cancer.
  • Telix believes that 153Sm-DOTMP may benefit patients with metastatic lung and breast cancer, where many patients develop brain and bone metastases, and disease management often focusses on quality-of-life palliative care.
  • Telix will issue ordinary shares to the stockholders of QSAM at closing within its Listing Rule 7.1 placement capacity as consideration for the acquisition.

Orphan designation: vactosertib Treatment of osteosarcoma, 25/07/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, European Commission, Osteosarcoma, Committee, Patient, EMA, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - vactosertib
    - Intended use
    - Treatment of osteosarcoma
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2797
    - Date of designation
    - Sponsor
    Sirius Regulatory Consulting EU Limited
    Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Human medicines European public assessment report (EPAR): Eladynos, abaloparatide, Date of authorisation: 12/12/2022, Revision: 3, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Patient, Urine, Medical Subject Headings, Marketing, INN, Risk, Fracture, Alkaline phosphatase, Woman, Allergy, Heart rate, Vitamin D, ATC, Hypercalcaemia, Menopause, International, Diet, Parathyroid hormone, Headache, Disease, Osteosarcoma, Nausea, Blood, Language, Calcium, Osteoblast, Palpitations, Cancer, Heart, Dizziness, Abdomen, European Medicines Agency, Spine, Back pain, Hypercalciuria, Select, System, Safety, Cardiovascular disease, Anatomy, Arthralgia, Bone fracture, Female, IIA, Skin, Medical device

Human medicines European public assessment report (EPAR): Eladynos, abaloparatide, Date of authorisation: 12/12/2022, Revision: 3, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Eladynos, abaloparatide, Date of authorisation: 12/12/2022, Revision: 3, Status: Authorised

Human medicines European public assessment report (EPAR): Natpar, parathyroid hormone, Date of authorisation: 24/04/2017, Revision: 17, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Patient, Gland, Medical Subject Headings, Diarrhea, Urine, Marketing, European Commission, Agency, INN, Risk, Vitamin D, ATC, System, International, European, Headache, Disease, Needle, Osteosarcoma, Hypoesthesia, Blood, Language, Calcium, Heart, Cancer, Pseudohypoparathyroidism, European Medicines Agency, Thigh, Select, Safety, Kidney, Anatomy, Medicine, Paresthesia, Vomiting, IIA, Neck, Skin, Medical device

Human medicines European public assessment report (EPAR): Natpar, parathyroid hormone, Date of authorisation: 24/04/2017, Revision: 17, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Natpar, parathyroid hormone, Date of authorisation: 24/04/2017, Revision: 17, Status: Authorised