Proteostatis stock plunges on cystic fibrosis triple combo data

Shares of Boston biotech Proteostasis Therapeutics Inc. sank roughly 60 percent Monday after the company released new data for its triple combination cystic fibrosis treatment that puts it behind competitor Vertex Pharmaceuticals. Pre-commercial company Proteostasis (Nasdaq: PTI) is hoping to gain a foothold in the cystic fibrosis field, which is currently dominated by Vertex (Nasdaq: VRTX). It is currently developing a handful of drugs for the rare lung disorder, including a combination of its…
Source: Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news

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Abstract Objectives To assess the perinatal and maternal outcomes of pregnant women with cystic fibrosis (CF) and severe lung impairment. Methods This was a series of cases aiming to review the maternal and fetal outcomes in cases of singleton pregnant women with a diagnosis of CF. We have included all of the cases of singleton pregnancy in patients with CF who were followed-up at the obstetrics department of the Medical School of the Universidade de S ão Paulo, between 2003 and 2016. The exclusion criteria were the unattainability of the medical records of the patient, and delivery at other institutions. A forced e...
Source: Revista Brasileira de Ginecologia e Obstetricia - Category: OBGYN Source Type: research
Condition:   Depression, Anxiety Interventions:   Behavioral: CF-CBT: A Cognitive-Behavioral Skills-Based Program to Promote Emotional Well-Being for Adults wtih Cystic Fibrosis;   Behavioral: Waitlist Control Sponsors:   Massachusetts General Hospital;   Cystic Fibrosis Foundation;   DeltaQuest Foundation, Inc.;   University at Buffalo;   University of Kansas Medical Center;   Stanford University;   Nicklaus Children's Hospital f/k/a Miami Children's Hospital Not yet recruiting
Source: - Category: Research Source Type: clinical trials
Emerging Knowledge for Clinical Practice Podium Presentations focusing on the Research Agenda Priority of Pediatric Research: Chronic Illness, Presented at NAPNAP's 40th National Conference on Pediatric Health Care, March 8, 2019, New Orleans, LA.
Source: Journal of Pediatric Health Care - Category: Pediatrics Authors: Source Type: research
Catherine Farrer from Dulwich, London, is pictured with her daughter Kate, 4, who is a Cystic Fibrosis sufferer. Caring for Kate has exposed her to the worrying realities of antibiotic resistance.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news
CONCLUSION: Epidemiological analysis suggested that this cluster could not always be attributed to patient-to-patient transmission. The acquisition of ICE-related virulence factors may have had an impact on its prevalence. PMID: 31210630 [PubMed - as supplied by publisher]
Source: Journal of Medical Microbiology - Category: Microbiology Authors: Tags: J Med Microbiol Source Type: research
In CF, people with higher FEV1 are less aggressively treated with intravenous (IV) antibiotics, with resultant negative impact on their health outcomes. This could be entirely clinician-driven, but patient choice may also influence IV use. In this prospective observational study, we explored IV recommendations by clinicians and IV acceptance by adults with CF to understand how clinical presentations consistent with exacerbations resulted in IV use.
Source: Respiratory Medicine - Category: Respiratory Medicine Authors: Source Type: research
Abstract High energy expenditure is reported in cystic fibrosis (CF) animal models and patients. Alterations in skeletal muscle oxidative capacity, fuel utilization, and creatine kinase-phosphocreatine system suggest mitochondrial dysfunction. Studies were performed on congenic C57BL/6J and F508del (Cftrtm1kth) mice. Indirect calorimetry was used to measure gas exchange to evaluate aerobic capacity during treadmill exercise. The bioenergetic function of skeletal muscle subsarcolemmal (SSM) and interfibrillar (IFM) mitochondria was evaluated using an integrated approach combining measurement of the rate of oxidativ...
Source: American Journal of Physiology. Endocrinology and Metabolism - Category: Physiology Authors: Tags: Am J Physiol Endocrinol Metab Source Type: research
Condition:   Bronchiectasis Adult Interventions:   Drug: Roflumilast;   Drug: Placebo oral tablet Sponsor:   University of Sao Paulo General Hospital Recruiting
Source: - Category: Research Source Type: clinical trials
The devastating inherited disease cystic fibrosis (CF) is caused by mutations of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) anion channel. The recent approval of the CFTR potentiator drug ivacaftor (Vx-770) for the treatment of CF patients has marked the advent of causative CF therapy. Currently, thousands of patients are being treated with the drug, and its molecular mechanism of action is under intensive investigation. Here we determine the solubility profile and true stimulatory potency of Vx-770 towards wild-type (WT) and mutant human CFTR channels in cell-free patches of membrane. We find that its ...
Source: eLife - Category: Biomedical Science Tags: Structural Biology and Molecular Biophysics Source Type: research
Treatment of patients with Cystic Fibrosis homozygous for the Phe508del gene, with Lumacaftor /Ivacaftor (LUM/IVA) improves outcomes in patients with FEV1  > 40% predicted. We set out to observe the most sensitive...
Source: BMC Pulmonary Medicine - Category: Respiratory Medicine Authors: Tags: Research article Source Type: research
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