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Data from Clinical and Pre-Clinical Trials Evaluating Verona Pharma’s RPL554 in Cystic Fibrosis Pres

Pre-clinical findings show RPL554 stimulates rare class III and IV CFTR mutants Phase 2a results demonstrate RPL554 has favorable pharmacokinetic and pharmacodynamic profile in cystic fibrosis patients LONDON, Oct. 26, 2018 (GLOBE NEWSWIRE) -- Verona Pharma plc (AIM:VRP) (Nasdaq:VRNA) ("Verona Pharma" or the "Company"), a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for respiratory diseases, announce the presentation of positive data from pre-clinical and Phase 2a trials evaluating RPL554 as a potential treatment for cystic fibrosis ("CF") at the 2018 North American Cystic Fibrosis Conference in Denver, CO. Data from the trials show

Proteostasis Therapeutics' Double and Triple-Combo CF Drugs Possible Threat to Vertex Dominance

Boston-based Proteostasis Therapeutics announced positive preliminary data from its ongoing Phase I clinical trial of PTI-808 and PTI-801 in cystic fibrosis (CF). Despite it being an early-stage trial and preliminary data, company shares exploded upward by 301 percent, with more than 44 million shares trading hands. Proteostasis announced positive data from three doublet cohorts. PTI-801 is a third-generation CF transmembrane conductance regulator (CFTR) corrector. PT-808 is a novel CFTR potentiator. A third drug, PTI-428, is a novel CFTR amplifier. The clinical trials are evaluating the safety, tolerability and pharmacokinetics of the drugs in combination against placebo. The endpoints were

FDA Approves AzurRx Biopharma Request to Test MS1819-SD in CF Patients with EPI

AzurRx BioPharma is planning a multi-center Phase 2 trial to be conducted in the United States and Europe to evaluate its lead candidate MS1819-SD as a treatment for patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). This announcement follows the approval by the U.S. Food and Drug Administration (FDA) of an Investigational New Drug (IND) application for MS1819-SD submitted by the company. The trial is expected to start by the end of 2018. “We are moving forward aggressively with our plans for the Phase 2 multi-center study in cystic fibrosis, which we expect will include approximately 30 patients in a head-to-head test against the current standard of care,” Ja

Scientists Reveal New Cystic Fibrosis Treatments Work Best in Inflamed Airways

New UNC School of Medicine research led by Martina Gentzsch, PhD, and Carla Ribeiro, PhD, suggests that cystic fibrosis drugs used to correct the faulty CFTR protein would work better when the patients’ airways are inflamed. Media contact: Mark Derewicz, 984-974-1915, mark.derewicz@unchealth.unc.edu CHAPEL HILL, NC – A new UNC School of Medicine study shows that two cystic fibrosis (CF) drugs aimed at correcting the defected CFTR protein seem to be more effective when a patient’s airway is inflamed. This is the first study to evaluate the efficacy of these drugs under inflammatory conditions relevant to CF airways. This work, published in the European Respiratory Journal, offers a greater un

Corbus Awarded US Patent Covering Lenabasum as Potential CF and Fibrotic Disease Treatment

Corbus Pharmaceuticals has received a patent from the U.S. Patent and Trademark Office covering all compositions that comprise lenabasum and might be used to treat any fibrotic disease, including cystic fibrosis (CF), the company announced in a press release. This patent (No. 10,085,964) also covers lenabasum in potentially treating dermatomyositis and systemic sclerosis. Previous patents for this investigational therapy pertain to its use in various inflammatory and fibrotic conditions. Lenabasum (also known as anabasum, JBT-101, or resunab) is a synthetic oral small molecule being developed by Corbus that is aimed at resolving chronic inflammation by binding to a specific receptor (cannabi

MS1819-SD Shows Positive Results for Exocrine Pancreatic Insufficiency in Phase 2a Trial

AzurRx BioPharma, in partnership with Mayoly Spindler, announced that a recent Phase 2a clinical trial testing MS1819-SD as a potential treatment for exocrine pancreatic insufficiency (EPI) achieved its goals, namely a statistically significant improvement in fat absorption. EPI, often caused by cystic fibrosis, is a disease resulting from a lack of exocrine pancreatic enzymes, which play a role in breaking down food molecules. Consequently, patients with EPI have trouble digesting food. The current standard treatment for EPI is porcine pancreatic enzyme replacement pills, which consist of enzymes derived from pigs. MS1819-SD, an investigational treatment given as an oral biologic capsule, i

Decibel Obtains Exclusive License for ORC-13661 for Prevention of Antibiotic-related Ototoxicity

Decibel Therapeutics received worldwide, exclusive rights to develop and commercialize ORC-13661, Oricula Therapeutics’ investigational treatment to prevent hearing loss and balance disorders in patients taking high doses of aminoglycoside antibiotics, including cystic fibrosis (CF) patients. Under the terms of the agreement, Oricula will provide scientific advice and support to Decibel throughout the ORC-13661 development process. ORC-13661 was developed to preserve hearing during treatment with aminoglycosides, a class of antibiotics created to kill a specific type of bacteria called gram-negative bacteria. Aminoglycosides are known to potentially cause severe side effects, including ototo

New Approach Against Antibiotic-resistant Bacteria in CF Patients Shows Promise in Phase 1 Trial

Researchers may have found a new way of treating antibiotic-resistant bacterial infections, specifically Pseudomonas aeruginosa, in the airways of patients with cystic fibrosis (CF), a study shows. In a preliminary Phase 1 trial, a single five-day infusion with the potential treatment, called gallium nitrate, improved the lung function of CF patients with a chronic P. aeruginosa infection, showing no signs of toxicity. Trial results were published in the study, “Gallium disrupts bacterial iron metabolism and has therapeutic effects in mice and humans with lung infections,” in the journal Science Translational Medicine. Research looking for new treatments against difficult-to-treat and antibi

Sound Pharmaceuticals to present initial data on the STOP Ototoxicity Study at Cystic Fibrosis Confe

Sound Pharmaceuticals (SPI) is pleased to announce that its recent submission to the upcoming North American Cystic Fibrosis Conference (NACFC) Oct. 18-20 has been selected as a late-breaking abstract. This presentation will focus on the incidence and severity of ototoxicity in CF patients undergoing intravenous (IV) tobramycin treatment for acute pulmonary exacerbation. Ototoxicity (hearing loss, tinnitus, vertigo or dizziness) is a common side effect of tobramycin and other aminoglycoside antibiotics (amikacin, gentamycin and streptomycin). Currently, there are no FDA approved therapies for the prevention or treatment of ototoxicity or any other type of sensorineural hearing loss, tinnitus

Chronic P. Aeruginosa Infection Greatly Raises Risk of Flares in Non-CF Bronchiectasis, Study Shows

People with non-cystic fibrosis (CF) bronchiectasis are at a significantly greater risk of exacerbations if they have chronic infections caused by Pseudomonas aeruginosa, a common bacteria that produces specific virulence factors, according to researchers in China. Their study, “Presence of pldA and exoU in mucoid Pseudomonas aeruginosa is associated with high risk of exacerbations in non-cystic fibrosis bronchiectasis patients,” was published in the journal Clinical Microbiology and Infection. P. aeruginosa is often found in people with chronic lung diseases, including CF, non-CF bronchiectasis, and chronic obstructive pulmonary disease (COPD). After colonizing the respiratory tract, the ba

Understanding antibiotic resistance in patients with cystic fibrosis

A defective gene causes thick, sticky mucus to build up in the lungs of patients with cystic fibrosis (CF). There, it traps bacteria, causing patients to develop frequent lung infections that progressively damage these vital organs and impair patients' ability to breathe. Most patients with this progressive genetic disorder die by the fourth decade of life. A key to helping patients live even that long -- a vast improvement from an average lifespan of 10 years just decades ago -- is judicious use of antibiotics, explains Andrea Hahn, M.D., a pediatric infectious diseases specialist at Children's National Health System. But antibiotics are a double-edged sword, Dr. Hahn adds: Although they're

#NACFC — Sound Pharmaceuticals to Present Preliminary Data on Ototoxicity Study

Sound Pharmaceuticals (SPI) will present data on the incidence and severity of ototoxicity in cystic fibrosis (CF) patients who received tobramycin for the treatment of acute pulmonary exacerbations. The presentation, which was accepted as a late-breaking abstract, will be given at the upcoming North American Cystic Fibrosis Conference (NACFC), taking place Oct. 18-20 in Denver. Ototoxicity — a complication that can result in hearing loss, tinnitus (perception of noise or ringing in the ears), vertigo (sensation of spinning while stationary), or dizziness — is known to be a common side effect of treatment with tobramycin and similar antibiotics, such as amikacin, gentamicin, and streptomycin

MS1819-SD Shows Positive Results for Exocrine Pancreatic Insufficiency in Phase 2a Trial

AzurRx BioPharma, in partnership with Mayoly Spindler, announced that a recent Phase 2a clinical trial testing MS1819-SD as a potential treatment for exocrine pancreatic insufficiency (EPI) achieved its goals, namely a statistically significant improvement in fat absorption. EPI, often caused by cystic fibrosis, is a disease resulting from a lack of exocrine pancreatic enzymes, which play a role in breaking down food molecules. Consequently, patients with EPI have trouble digesting food. The current standard treatment for EPI is porcine pancreatic enzyme replacement pills, which consist of enzymes derived from pigs. MS1819-SD, an investigational treatment given as an oral biologic capsule, i

Vertex and Treating CF: High Hopes for Triple Combinations and Gene Therapy Possibilities

Editor’s note: This is third in a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. Here, we look at potential “next-generation” treatments that range from triple combinations in or readying for clinical trials, to gene editing that has a way to go. The research that led to Vertex being popularly known as “the CF company” spans almost 30 years and three approved disease-modifying treatments. Now, focus is on younger patients and a “new generation” of potential therapies. It’s part of Vertex’s three-part approach to treating all with CF by: running pediatric studies aiming to bring Kalydeco (ivacaftor), Orkambi(lumacaftor/ivacaftor), and Symdeko (tezacaftor/

Does Monotherapy With Correctors Have Clinical Benefit in Cystic Fibrosis?

Insufficient evidence exists to demonstrate that monotherapy with correctors has clinically important effects in individuals with cystic fibrosis (CF) who have 2 copies of the F508del mutation, according to an analysis published in the Cochrane Database of Systematic Reviews. Researchers conducted a search of the Cochrane Cystic Fibrosis and Genetic Disorders Cystic Fibrosis Trials Register, as well as a literature search of reference lists of relevant articles and online trial registries. The most recent search was conducted on February 24, 2018. They sought to assess the effects of CF transmembrane conductance regulator (CFTR) correctors on clinically important outcomes, both benefits and

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