Cystic fibrosis

Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Absorption, Lower respiratory tract infection, Exercise, Cell, Infection, DSM-IV codes, Inflammation, Patient, Committee, Cystic fibrosis, Knowledge, Digestion, Regulation, Bacteria, Food, EMA, IRIS, Anti-inflammatory, Mucus, CB2, European, COMP, Pancreas, Sponsor, Ivacaftor, European Commission, Safety, CFTR, Marketing, Medicine, Pharmaceutical industry

Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Key Points: 


Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Orphan designation: mRNA encoding the human CFTR gene Treatment of cystic fibrosis, 19/02/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
Patient, Committee, Arcturus Therapeutics, Messenger, EMA, IRIS, European, Therapy, COMP, European Commission, CFTR, Marketing, Cystic fibrosis, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of cystic fibrosis in the European Union on 19 February 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • 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:

BiomX Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, April 3, 2024
DFO, OrbiMed, NESS, Cystic Fibrosis Foundation, APT, FEV1, Part, Phage therapy, Patient, Orphan, KOL, Acquisition, Șaeș, Cystic fibrosis, Infection, Therapy, Pseudomonas aeruginosa, Pharmaceutical industry

CAMBRIDGE, Mass. and NESS ZIONA, Israel, April 03, 2024 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX” or the “Company”), a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a business update.

Key Points: 
  • and NESS ZIONA, Israel, April 03, 2024 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX” or the “Company”), a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a business update.
  • Initial top line results of the Phase 2 trial are expected in the first quarter of 2025.
  • However, our financial statements contain an explanatory paragraph regarding substantial doubt about our ability to continue as a going concern.
  • ET to discuss its fourth quarter and full year 2023 financial results and to provide a corporate update.

4DMT Announces Update on Regulatory Interactions and Development Path for 4D-710 for Treatment of Cystic Fibrosis

Retrieved on: 
Thursday, March 28, 2024
CDMO, Conference, TDN, Patient, Lung, Cohort, RNA, EMA, Technology transfer, Cystic fibrosis, European Medicines Agency, GMP, Clinical trial, ECFS, Safety, CFTR, AMD, Food, FDA, VG, Pharmaceutical industry

Phase 3 clinical endpoints include changes after 4D-710 treatment in ppFEV1, quality-of-life (Cystic Fibrosis Questionnaire Revised Respiratory Domain, CFQ-R-RD) and frequency of pulmonary exacerbations.

Key Points: 
  • Phase 3 clinical endpoints include changes after 4D-710 treatment in ppFEV1, quality-of-life (Cystic Fibrosis Questionnaire Revised Respiratory Domain, CFQ-R-RD) and frequency of pulmonary exacerbations.
  • The Company anticipates initiation of technology transfer to a commercial contract development and manufacturing organization (CDMO) in H1 2025.
  • Our goal is to initiate Phase 3 development in H2 2025 with 4D-710 suspension GMP process clinical trial material.
  • We look forward to sharing interim clinical data from the AEROW Phase 1/2 clinical trial at the ECFS conference in June 2024.”

Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Positive

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Absorption, Lower respiratory tract infection, Exercise, Cell, Infection, DSM-IV codes, Inflammation, Patient, Committee, Cystic fibrosis, Knowledge, Digestion, Regulation, Bacteria, Food, EMA, IRIS, Anti-inflammatory, Mucus, CB2, European, COMP, Pancreas, Sponsor, Ivacaftor, European Commission, Safety, CFTR, Marketing, Medicine, Pharmaceutical industry

Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Positive

Key Points: 


Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Positive

BiomX Announces Closing of the Acquisition of Adaptive Phage Therapeutics and Concurrent $50 Million Financing

Retrieved on: 
Monday, March 18, 2024
DFO, McDermott Will & Emery, NESS, Cystic Fibrosis Foundation, APT, Phage therapy, Patient, Cystic fibrosis, LLP, Acquisition, Cooley LLP, Infection, OrbiMed, RBC Capital Markets, Pharmaceutical industry

CAMBRIDGE, Mass. and NESS ZIONA, Israel, March 18, 2024 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (together with its subsidiaries and/or associates, “BiomX”), a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today announced the closing of its previously announced (March 6, 2024) acquisition (the “Acquisition”) of Adaptive Phage Therapeutics, Inc. (“APT”), a U.S.-based privately-held, clinical-stage biotechnology company pioneering the development of phage-based therapies to combat bacterial infections, and its previously announced $50 million private placement to certain institutional accredited investors, which was led by affiliates of Deerfield Management Company and the AMR Action Fund, and additional investors including the Cystic Fibrosis Foundation, OrbiMed and Nantahala Capital.

Key Points: 
  • “We are very pleased to announce the closing of the APT acquisition, which creates a leading phage company and an advanced pipeline that includes two Phase 2 assets each aimed at treating serious infections with unmet medical need,” said Jonathan Solomon, Chief Executive Officer of BiomX.
  • “We also appreciate the continued support of leading institutional healthcare investors.
  • Haynes and Boone, LLP served as legal counsel to BiomX.
  • Wilmer Cutler Pickering Hale and Dorr LLP served as legal counsel to the placement agents.

First Wave BioPharma Announces Completion of Business Combination with ImmunogenX, Adding Phase 3-Ready Latiglutenase to its Late-Stage GI-Focused Clinical Pipeline

Retrieved on: 
Thursday, March 14, 2024
Conference, Webcast, Patient, Common, Fats, GI, Acquisition, First wave, NIH, Gastrointestinal disease, Series, Marketing, Gastroparesis, FDA, Cystic fibrosis, Food, Digestion, Pharmaceutical industry

BOCA RATON, Fla., March 14, 2024 (GLOBE NEWSWIRE) -- First Wave BioPharma, Inc., (NASDAQ: FWBI), (“First Wave BioPharma” or the “Company”), a clinical-stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases, today announced it has acquired ImmunogenX in an all-stock transaction with the combined company focused on advancing a GI pipeline comprised of multiple, late-stage clinical assets, including latiglutenase, a potentially first-in-class, near Phase 3-ready, targeted, oral biotherapeutic for celiac disease. James Sapirstein will continue to serve as Chairman and Chief Executive Officer of First Wave BioPharma with Jack Syage, Ph.D., previously the Chief Executive Officer and Co-Founder of ImmunogenX, assuming the role of President and Chief Operating Officer of First Wave BioPharma. Dr. Syage and Dr. Chaitan Khosla will also join the board of directors of First Wave BioPharma.

Key Points: 
  • Dr. Syage and Dr. Chaitan Khosla will also join the board of directors of First Wave BioPharma.
  • First Wave BioPharma intends to license the commercial rights to latiglutenase in the United States and Canada to a strategic global pharmaceutical company which will commercialize latiglutenase following receipt of marketing approval.
  • First Wave BioPharma will also seek to secure financing commitments from a syndicate of institutional healthcare investors to fund the ongoing development of latiglutenase.
  • First Wave BioPharma will host a conference call and live audio webcast today, March 14, 2024, at 8:30 a.m.

Registration Opens for the National Association of Specialty Pharmacy 2024 Annual Meeting & Expo

Retrieved on: 
Tuesday, March 12, 2024
Convention center, Pharmacist, Annual, Organ transplantation, Rheumatoid arthritis, CE, Woman, Multiple sclerosis, Haemophilia, Pharmacy, Patient, Cancer, Abstract, Love, Specialty pharmacy, National Association, CSP, Cell, Data management, Nursing, Education, NASP, Consultant, Cystic fibrosis, Accreditation, Hospital

Washington, DC, March 12, 2024 (GLOBE NEWSWIRE) -- Today, the National Association of Specialty Pharmacy (NASP), the only industry association in the U.S. whose membership includes all specialty pharmacy stakeholders, opened registration for the NASP 2024 Annual Meeting & Expo, to be held October 6 - 9, 2024, at the Gaylord Opryland Resort & Convention Center in Nashville, TN.

Key Points: 
  • Washington, DC, March 12, 2024 (GLOBE NEWSWIRE) -- Today, the National Association of Specialty Pharmacy (NASP), the only industry association in the U.S. whose membership includes all specialty pharmacy stakeholders, opened registration for the NASP 2024 Annual Meeting & Expo, to be held October 6 - 9, 2024, at the Gaylord Opryland Resort & Convention Center in Nashville, TN.
  • “The NASP 2024 Annual Meeting & Expo offers everything that attendees have come to love and expect, including top-of-class educational content and networking events.
  • Workshops include Accreditation, Cell & Gene Therapy, Certified Specialty Pharmacist (CSP) Exam Prep Course, Hospital/Health System Specialty Pharmacy, Specialty Pharmacy Law, and Technology.
  • The Annual Meeting & Expo also features a showcase for specialty pharmacy clinical research with its abstract and poster program.

CF Foundation Provides Up to $8.5M to SpliSense to Support a Clinical Trial for a Potential Treatment for Splicing Mutations

Retrieved on: 
Wednesday, April 3, 2024
Science, Research, Pharmaceutical, General Health, Health, Philanthropy, Clinical Trials, Foundation, MD, Cystic fibrosis, CFTR, RNA, Investment, Clinical trial, Cystic Fibrosis Foundation, Pharmaceutical industry

The Foundation's funding will support a planned Phase 2 clinical trial to test the efficacy of SpliSense’s inhaled ASO drug as a potential treatment for the lungs of people with the splicing mutation 3849+10Kb C-to-T. A recent Phase 1a study indicated the drug was safe and well tolerated.

Key Points: 
  • The Foundation's funding will support a planned Phase 2 clinical trial to test the efficacy of SpliSense’s inhaled ASO drug as a potential treatment for the lungs of people with the splicing mutation 3849+10Kb C-to-T. A recent Phase 1a study indicated the drug was safe and well tolerated.
  • The objective of the clinical trial is to generate a proof-of-concept for ASO technology in cystic fibrosis.
  • If the clinical trial is successful, it could validate other similar therapeutic approaches in SpliSense's pipeline that address rare CF mutations.
  • In 2021, the Foundation invested $8.4 million in SpliSense to develop a therapy for splicing mutations, in addition to $400,000 the Foundation provided in 2017.

Vertex Announces FDA Clearance of Investigational New Drug Application for VX-407 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Retrieved on: 
Thursday, March 21, 2024
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, FDA, Clinical Trials, Death, Autosome, VRTX, Growth, Disease, PC1, Patient, Kidney failure, Kidney transplantation, Polycystic kidney disease, IND, Vertex Pharmaceuticals, PKD1, Vertex, ADPKD, Dialysis, Cystic fibrosis, Food, Pharmaceutical industry

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants.
  • ADPKD is the most common inherited kidney disease, with an estimated 250,000 people in the U.S. and Europe living with ADPKD; however, there are no treatments currently available that address the underlying causal biology of the disease.
  • ADPKD is a life-shortening genetic kidney disease characterized by the growth of numerous kidney-enlarging cysts that impair kidney function and can ultimately lead to kidney failure, requiring dialysis or kidney transplantation, and premature death.
  • The majority of ADPKD cases are caused by variants in the PKD1 gene, which encodes the polycystin 1 (PC1) protein.