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Arrowhead Pharmaceuticals Hosts Key Opinion Leader Webinar on ARO-ENaC for Treatment of Cystic Fibro

PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) is hosting a key opinion leader webinar today at 12:00 PM EDT on ARO-ENaC, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF). The webinar features a presentation by Marcus Mall, M.D., professor and director of the Department of Pediatric Pulmonology and Immunology at The Charité University Medical Center Berlin, who will discuss the current treatment landscape and unmet medical need in treating patients with CF. Arrowhead management is providing a review of the ARO-ENaC program, which utilizes Arrowhead’s proprietary Targeted R

AzurRx Begins Phase 2b Trial Testing MS1819 for Pancreatic Insufficiency in CF Patients

AzurRx BioPharma has launched a Phase 2b clinical trial investigating its MS1819 as a potential treatment for exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF). The first patient has now been screened in the OPTION 2 trial (NCT04375878), which has opened at three clinical sites to start. Researchers intend to enroll a total of 30 CF patients at 15 sites across the U.S. and Poland, and AzurRx anticipates providing top-line data from the study in early 2021. “The initiation of the OPTION 2 trial is a major clinical milestone for the company,” James Sapirstein, CEO of AzurRx, said in a press release. “We look forward to the successful completion of the trial and to r

Smoking, Respiratory Issues, Older Age Raise Hospital Costs, Chinese Study Finds

Older age, smoking, and having other respiratory symptoms and conditions increase the hospitalization costs of people with bronchiectasis, a large Chinese study says. The findings also suggest that avoiding overuse of antibiotics may help lower hospital expenses in this patient population. Titled “Clinical features related to hospital expenses for non-cystic fibrosis bronchiectasis in China,” the study was published in the Journal of International Medical Research. Bronchiectasis is characterized by a permanent widening of the airways of the lung, which can be caused by congenital conditions such as cystic fibrosis (CF), or can be acquired. Non-CF bronchiectasis is associated with recurrent

Positive Results from Phase 2 WILLOW Study of Brensocatib in Patients with Non-Cystic Fibrosis Bronc

--Brensocatib Shown to Reduce Time to First Pulmonary Exacerbation and Reduce Rate of Exacerbations Versus Placebo-- --New Data Demonstrate Relationship Between Neutrophil Elastase Reduction and Risk of Exacerbation in Patients Treated with Brensocatib-- BRIDGEWATER, N.J., June 24, 2020 /PRNewswire/ -- Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, today announced that final results from the Phase 2 WILLOW study of brensocatib (formerly INS1007) in patients with non-cystic fibrosis bronchiectasis (NCFBE) were presented during a virtual American Thoracic Society (ATS) session titled Breaking

World-first clinical trial raises hopes of new drug for chronic lung disease

CHRONIC lung disease sufferers could be aided by a new drug which helps reduce the need for antibiotics, according to a clinical trial. The final results from the Phase 2 WILLOW study were announced today by James Chalmers, British Lung Foundation Professor of Respiratory Research at the University of Dundee. Scientists at the University, in collaboration with biopharmaceutical company Insmed Incorporated tested patients living with non-cystic fibrosis bronchiectasis, a long-term lung condition which causes abnormal widening of the airways. The final results demonstrated that patients on the new drug, brensocatib, could potentially reduce their risk of pulmonary exacerbations by more than on

The Multidisciplinary Team Improves Cystic Fibrosis Outcomes

CYSTIC FIBROSIS (CF) is an inherited autosomal recessive disease, and it affects approximately 30,000 Americans. Chronic, progressive, and life-shortening, CF primarily targets the lungs and the pancreas, but can also affect the gastrointestinal tract, reproductive organs, and sinuses. In a virtual symposium held in conjunction with the Asembia Specialty Pharmacy Summit, 2 speakers covered the numerous care improvements developed for CF over the past 10 to 20 years. Rachel Espinosa, PharmD, described a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR maintains fluid balance across epithelial cells, but in CF, decreased chloride transport leads to dehydrati

Women With CF Use Combined Hormonal Contraceptives More Than Other Methods, Study Reports

Among women of childbearing age with cystic fibrosis (CF), the use of combined hormonal contraceptives containing both estrogen and progestin is more common than condoms or other methods of contraception, according to data from a pilot registry study. The study, which linked survey findings to patient data from the Cystic Fibrosis Foundation National Patient Registry (CFFNPR), also revealed that pregnancies were unplanned for half of the women who reported becoming pregnant over the study time period. The study, “Contraceptive use among women with cystic fibrosis: A pilot study linking reproductive health questions to the Cystic Fibrosis Foundation National Patient Registry,” was published i

Drying Does Not Clear Nebulizers of NTM Bacteria, Study Finds

Nontuberculous mycobacteria (NTM), a type of infectious bacteria, is able to survive being dried for a full day, a study found, suggesting that drying is not a sufficient way to disinfect nebulizers used by people with cystic fibrosis (CF). The study, “Susceptibility of the Mycobacterium abscessus complex to drying: Implications for nebulizer hygiene in patients with cystic fibrosis,” was published in the International Journal of Mycobacteriology. NTM refers to mycobacteria — a bacterial genus — other than Mycobacterium tuberculosis (which causes tuberculosis) and Mycobacterium leprae (which causes leprosy). NTM are increasingly being recognized as a common and worrisome infectious agent in

CHMP Gives Positive Opinion to Vertex’s Triple Combination Therapy Kaftrio

The Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) as a treatment for cystic fibrosis (CF) in people 12 and older who have either two F508del mutations or one F508del mutation and one minimal function mutation in the CFTR gene. “We are delighted to have received this positive opinion from CHMP,” Carmen Bozic, MD, executive vice president of global medicines development and medical affairs, and chief medical officer at Vertex Pharmaceuticals, said in a press release. Vertex is the developer of Kaftrio, which is approved and marketed in the U.S. as Trikafta. “If approved

Brensocatib prolongs time to first pulmonary exacerbation in patients with bronchiectasis

Treatment with brensocatib for 24 weeks significantly prolonged time to first pulmonary exacerbation in patients with non-cystic fibrosis bronchiectasis compared with placebo, according to results from the WILLOW study. Brensocatib (Insmed) is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) that interrupts neutrophilic inflammatory processes in the lung. As Healio previously reported, brensocatib received FDA breakthrough therapy designation in mid-June to treat adults with non-cystic fibrosis bronchiectasis. “Currently, bronchiectasis guidelines recommended airway clearance and mucoactive therapies to target abnormally disorderly clearance and macrolides in inh

New Combination of CFTR Mutations Found in Chinese Siblings With CF

A combination of two mutations, including one that had never been reported, were found in two Chinese siblings who developed a severe form of cystic fibrosis (CF), according to a case report. The report, “Identification of a Mutation in the Novel Compound Heterozygous CFTR in a Chinese Family with Cystic Fibrosis,” was published in the Canadian Respiratory Journal. CF is one of the most common recessive disorders in people of Northern European descent — a recessive disorder means that patients must carry mutations in both copies of the affected gene (one from the mother and the other from the father), in this case the CFTR gene resulting in a defective or absent CFTR protein. However, CF is

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