PTEN

In Preparation for Expected Human Clinical Trials, NurExone Signs Agreement for Large Scale Preclinical Testing for Its Spinal Cord Injury Therapy, ExoPTEN

Retrieved on: 
Friday, March 22, 2024
Drug discovery, Rat, NRX, PTEN, Patient, Contract research organization, IND, Regenerative medicine, Behavior, Spinal cord, Spinal cord injury, SCI, Pharmaceutical industry

This large-scale animal testing represents a significant step towards filing an Investigational New Drug (IND) application in the US for NurExone’s ExoPTEN therapy for spinal cord injury (SCI).

Key Points: 
  • This large-scale animal testing represents a significant step towards filing an Investigational New Drug (IND) application in the US for NurExone’s ExoPTEN therapy for spinal cord injury (SCI).
  • Under the terms of the agreement, Vivox will provide Contract Research Organization (CRO) services to NurExone, as a prerequisite to commencing Human Trials under the planned IND.
  • The testing will encompass mobility, behavior, motor and sensory recovery of rats following Spinal Cord Injury and treatment with ExoPTEN," said Dr. Lior Shaltiel, CEO of NurExone.
  • By collaborating with Vivox, NurExone aims to accelerate the development and regulatory path of ExoPTEN, to bring this potentially transformative treatment to patients in need.

Pharming Group reports fourth quarter and full year 2023 financial results

Retrieved on: 
Thursday, March 14, 2024
Patient, Health care, Epidemiology, Executive Committee, Laboratory, Microsoft Access, Lymphocyte, Clinical trial, European Commission, APDS, MHLW, AEG, Welfare, Ageing, Committee, EMA, Trial of the century, PID, CTLA4, Safety, Pharmacokinetics, Ministry, CHMP, ODD, U.S. FDA, Prevalence, PTEN, Disease, HAE, Immunodeficiency, VUS, Literature, PIK3R1, PMDA, FDA, DSM-IV codes, Diagnosis, Civil service commission, IRP, Haploinsufficiency, Genetic testing, MAA, Autoimmunity, World Games, Iberian languages, Marketing, MHRA, Euronext Amsterdam, PIK3CD, Physician, Pharmaceutical industry

The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.

Key Points: 
  • The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.
  • Revenues increased to US$7.9 million in the fourth quarter of 2023, driven by the continued increase in patients on paid therapy, and revenues were US$18.2 million for 2023.
  • Pharming made continued progress in the fourth quarter of 2023 on leniolisib regulatory filings for APDS patients 12 years of age and older in key global markets.
  • Pharming filed regulatory submissions in Canada and Australia in the third quarter of 2023, and Israel in the second quarter.

Pharming Group announces development plans for leniolisib for additional primary immunodeficiencies (PIDs)

Retrieved on: 
Wednesday, December 13, 2023
APDS, Food, FRCPA, Infection, NIAID, IND, Epidemiology, FDA, Prevalence, Immunodeficiency, Autoimmune lymphoproliferative syndrome, CTLA4, Pharmacokinetics, Primary immunodeficiency, Euronext Amsterdam, Protocol, Lymphocyte, PTEN, Gene, Bangladesh Technical Education Board, Face, NIH, Patient, Safety, FAS, PID, Pharmaceutical industry

Leiden, The Netherlands, December 13, 2023: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces today the expansion of its rare disease pipeline with plans to develop leniolisib for additional primary immunodeficiencies (PIDs) beyond activated phosphoinositide 3-kinase delta syndrome (APDS).

Key Points: 
  • Leiden, The Netherlands, December 13, 2023: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces today the expansion of its rare disease pipeline with plans to develop leniolisib for additional primary immunodeficiencies (PIDs) beyond activated phosphoinositide 3-kinase delta syndrome (APDS).
  • Pharming has engaged with the US Food and Drug Administration (FDA) and has received feedback on its plans to develop leniolisib for PID disorders with immune dysregulation.
  • The Phase 2 clinical trial will evaluate leniolisib in PIDs with immune dysregulation linked to PI3Kẟ signaling in lymphocytes, with similar clinical phenotypes to APDS.
  • Building upon the success of leniolisib for APDS, we believe that leniolisib will continue to have efficient uses in rebalancing immune dysregulation in PIDs.

Ocean Biomedical, Inc. Announces Publication of Groundbreaking Breast Cancer Research Uncovering a New Tumor Suppression Pathway for Its Proprietary Anti-Chi3L1 Antibody

Retrieved on: 
Tuesday, December 5, 2023
Cancer research, Ocean Biomedical, Immunotherapy, Neoplasm, University, IND, Breast cancer, Antibody, Research, PD-1, Glioblastoma, Apoptosis, Yale University, NETS, TKI, Cancer, CTLA4, Neutrophil extracellular traps, Colorectal cancer, Journal, PTEN, BioRxiv, Growth, McGill University, Publication, Immunity, Science, Pharmaceutical industry

PROVIDENCE, Rhode Island, Dec. 05, 2023 (GLOBE NEWSWIRE) -- Ocean Biomedical, Inc. (NASDAQ: OCEA), a biopharma company working to accelerate the development and commercialization of scientifically compelling assets from research universities and medical centers, announced today that its Scientific Co-founder, Jack A. Elias, MD, co-authored new findings in the peer-reviewed journal Immunity that detail the mechanisms behind the role of chitinase 3-like-1 (CHI3L1) in the growth of triple negative breast cancer. The groundbreaking discoveries by a team led by Dr. William Muller at McGill University and in collaboration with Dr. Elias demonstrates that CHI3L1 stimulates neutrophil elaboration of NETs which block T cells from contacting and killing the breast cancer tumor. Additionally, the study provides further evidence of the potential impact of Ocean’s anti-Chi3L1 antibody in reversing this process and suppressing breast cancer tumor growth.

Key Points: 
  • Additionally, the study provides further evidence of the potential impact of Ocean’s anti-Chi3L1 antibody in reversing this process and suppressing breast cancer tumor growth.
  • This groundbreaking paper deepens the understanding of how CHI3L1 inhibits the body’s natural ability to fight breast cancer tumors.
  • Although these findings were defined in NSCLC, they have potential effectiveness in other EGFR-mutation driven cancers including Glioblastoma and Colon cancer.
  • Prior research has established that elevated Chi3L1 levels are associated with many cancers, including glioblastoma, and may be targeted therapeutically.

NurExone Reports Third Quarter 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Friday, November 24, 2023
Private, Neurosurgery, IND, Drug development, Drug discovery, Indian stock exchange, Research, Small RNA, CAD, Spinal cord, Patent, Health, Exosome, Investigational New Drug, Advisory Committee, PTEN, Unit, Option, NRX, Therapy, Food and Drug Administration, Exercise, Pharmaceutical industry

TORONTO and HAIFA, Israel, Nov. 24, 2023 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (FSE: J90) (NRX.V) (the “Company” or “NurExone”), a pioneering biotechnology company, released a corporate update and reported financial results for the quarter ended September 30, 2023.

Key Points: 
  • TORONTO and HAIFA, Israel, Nov. 24, 2023 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (FSE: J90) (NRX.V) (the “Company” or “NurExone”), a pioneering biotechnology company, released a corporate update and reported financial results for the quarter ended September 30, 2023.
  • Research and development expenses were US$0.40 million in the third quarter of 2023, compared to US$0.42 million in the same quarter in 2022.
  • Financial (income) expenses were (US$0.01) million in the third quarter of 2023, compared to US$0.02 million in the same period in 2022.
  • Net loss was US$1.16 million in the third quarter of 2023, compared to a net loss of US$1.04 million in the same quarter in 2022.

Third Success from Astex Drug Discovery Pharma Collaboration and Licence Agreements as AstraZeneca Receives US Marketing Approval for Cancer Drug Truqap (capivasertib)

Retrieved on: 
Monday, November 20, 2023
Sale, Institute of Cancer Research, Translation, Research, AstraZeneca, Food, US Foods, PIK3CA, Mouse, Patient, PTEN, FDA, PKB, Marketing, Neoplasm, CRT, Therapy, US FDA, Drug discovery, AKT, Cancer, 3D, AKT1, Central nervous system, Recurrence, ICR, Pharmaceutical industry, Astex Pharmaceuticals

CAMBRIDGE, United Kingdom, Nov. 20, 2023 (GLOBE NEWSWIRE) -- Astex Pharmaceuticals (UK), a pharmaceutical company dedicated to the discovery and development of novel small molecule therapeutics for oncology and diseases of the central nervous system, announced today that it is in line to receive a milestone payment from AstraZeneca on first commercial sale of the drug in the US and royalties from AstraZeneca on future sales following the US Food and Drug Administration’s (FDA) approval of Truqap™ plus Faslodex® as a treatment for adult patients with HR-positive, HER2-negative locally advanced or metastatic breast cancer with one or more biomarker alterations (PIK3CA, AKT1 or PTEN). Eligible patients will have progressed on at least one endocrine-based regimen in the metastatic setting or experienced recurrence on or within 12 months of completing adjuvant therapy.

Key Points: 
  • This is the third cancer drug in the last five years to reach market approval that has been discovered and developed under, or subsequent to, an Astex drug discovery collaboration.
  • Truqap was discovered by AstraZeneca following an earlier drug discovery research collaboration between Astex, The Institute of Cancer Research, London, and Cancer Research Technology (CRT; now Cancer Research Horizons) that was signed in 2003.
  • In 2010, AstraZeneca announced its discovery of Truqap, and began to develop the drug as a potential treatment for various forms of cancer.
  • This is also a great example of UK Biotech-Academia-Pharma collaboration and underlines the strength of the UK life sciences ecosystem.”
    Graphic of the hat-trick of three marketed cancer drugs discovered and developed under, or subsequent to, an Astex drug discovery collaboration with pharma

TRUQAP™ (capivasertib) Now Available from Onco360

Retrieved on: 
Wednesday, November 29, 2023
Oncology, Health, Clinical Trials, Research, Pharmaceutical, Science, Vomiting, AstraZeneca, Neutrophil, Toxicity, Stomatitis, Incidence, National Cancer Institute, Fulvestrant, White, Specialty pharmacy, Lymphocyte, PIK3CA, Breast cancer, Fatigue, Patient, Pharmacy, PFS, PTEN, FDA, Neoplasm, Creatinine, HER2, Diarrhea, Nausea, Cancer, Diagnosis, Death, AKT1, Recurrence, HIV disease progression rates, Triglyceride, Pharmaceutical industry

Onco360®, the nation’s leading independent Specialty Pharmacy, has been selected as a pharmacy partner by AstraZeneca for Truqap™ (capivasertib).

Key Points: 
  • Onco360®, the nation’s leading independent Specialty Pharmacy, has been selected as a pharmacy partner by AstraZeneca for Truqap™ (capivasertib).
  • FDA also approved the FoundationOne®CDx assay as a companion diagnostic device to identify patients with breast cancer for treatment with capivasertib with fulvestrant.
  • “Onco360 is excited to partner with the team at AstraZeneca and become a specialty pharmacy provider for Truqap™,” said Benito Fernandez, Chief Commercial Officer.
  • “We continue to see therapeutic advancements that support our mission; to improve the lives of patients battling cancer, rare, and orphan disease.

NurExone Biologic Receives FDA Orphan-Drug Designation, Accelerating Development of ExoPTEN therapy for Acute Spinal Cord Injury Treatment

Retrieved on: 
Monday, October 30, 2023
FDA, PTEN, Food, Rare, RNA, NRX, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Drug development, ODD, MSC, FSE, EVS, Patient, Small RNA, Pharmaceutical industry

The orphan drug designation provides significant benefits to pharmaceutical companies developing drugs for rare diseases, i.e.

Key Points: 
  • The orphan drug designation provides significant benefits to pharmaceutical companies developing drugs for rare diseases, i.e.
  • These benefits include market exclusivity, financial incentives, regulatory assistance, and support with drug development.
  • Overall, the designation incentivizes and supports the development of certain treatments, increasing access to therapies for patients.
  • We appreciate the formal recognition of the potential impact of our therapy on the lives of patients suffering from acute spinal cord injuries,” said Dr. Shaltiel, CEO of NurExone Biologic, Ltd.

AI-Powered Tumor Microenvironment Analysis Predicts Treatment Outcomes in NSCLC Patients with EGFR Mutation: Groundbreaking Studies to be Presented by Lunit at SITC 2023

Retrieved on: 
Wednesday, November 1, 2023
TKI, TILS, Patient, Neoplasm, The Cancer Genome Atlas, TCGA, NSCLC, PTEN, Progression-free survival, Biomarker, Therapy, Booth, ICI, BRCA1, Data, PFS, PIK3CA, Phenotype, Inflammation, TIL, Samsung Medical Center, BRCA2, OR, TNBC, IL1A, SITC, Immunotherapy, Methylation, ITIL, Annual general meeting, Breast cancer, Triple-negative breast cancer, Gene, Tumor microenvironment, Lung cancer, Society, IL17A, Cancer, TFI, TME, STIL, Mutation, EGFR, Medical imaging

The study found that EGFR-TKI affects the immune landscape of EGFR-mutated NSCLC as higher PD-L1 expression and differential immune phenotypes.

Key Points: 
  • The study found that EGFR-TKI affects the immune landscape of EGFR-mutated NSCLC as higher PD-L1 expression and differential immune phenotypes.
  • Patients with an inflamed immune phenotype after EGFR-TKI treatment showed a more favorable response to subsequent immune checkpoint inhibitors (ICI) treatment.
  • Another study investigated the effect of tumor fragmentation index (TFI), the number of tumor fragments per total tumor area, in TME.
  • Visit Lunit's booth at SITC 2023 at Booth #227 to learn more about these pioneering studies.

Aadi Bioscience Announces Poster Presentations on Nab-Sirolimus at the International Gynecologic Cancer Society (IGCS) 2023 Annual Global Meeting

Retrieved on: 
Thursday, October 19, 2023
PTEN, Research design, Pathology, EEC, Emotional dysregulation, TSC1, Multicenter Automatic Defibrillator Implantation Trial, Letrozole, Safety, Patient, TSC2, Pharmaceutical industry, Vaccine

LOS ANGELES, Oct. 19, 2023 /PRNewswire/ -- Aadi Bioscience, Inc. (NASDAQ: AADI), a biopharmaceutical company focused on developing and commercializing precision therapies for patients with mTOR pathway alterations, today announced e-poster presentations at the International Gynecologic Cancer Society (IGCS) 2023 Annual Global Meeting, taking place November 5-7, 2023, in Seoul, Korea.

Key Points: 
  • LOS ANGELES, Oct. 19, 2023 /PRNewswire/ -- Aadi Bioscience, Inc. (NASDAQ: AADI), a biopharmaceutical company focused on developing and commercializing precision therapies for patients with mTOR pathway alterations, today announced e-poster presentations at the International Gynecologic Cancer Society (IGCS) 2023 Annual Global Meeting, taking place November 5-7, 2023, in Seoul, Korea.
  • Abstract and e-poster presentation details are below:
    This is a Phase 2 open-label, multi-institutional study to evaluate the efficacy and safety of nab-sirolimus and letrozole in patients with advanced or recurrent endometrioid endometrial carcinoma, exploring the potential for this combination to produce additive anti-tumor activity in patients with endometrioid-type endometrial carcinoma (EEC)
    Dysregulation of mTOR signaling is implicated in the pathology of EEC, in which >80% harbor PTEN or PI3K/AKT/mTOR pathway alterations
    In preclinical models, intravenous nab-sirolimus demonstrates significantly higher tumor growth inhibition, intra-tumoral drug accumulation, and greater mTOR target suppression compared with oral inhibitors
    An encore presentation titled, "Phase 2, Multicenter, Open-label Basket Trial of nab-Sirolimus for Malignant Solid Tumors Harboring Pathogenic Inactivating Alterations in TSC1 and TSC2 (PRECISION1)" will be displayed as an e-poster during the meeting.