Phase 1a study for drug to treat CF regardless of CF Mutation

http://www.businesswire.com/news/home/20171018005403/en/Synspira-Announces-Patient-Dosed-Phase-1a-Study

Synspira Announces First Patient Dosed in Phase 1a Study of SNSP113 in Cystic Fibrosis
— First-in-class drug candidate for treatment of cystic fibrosis regardless of genetic mutation — Continue reading Phase 1a study for drug to treat CF regardless of CF Mutation

Biopharmaceutical company developing novel small molecule medicines for genetic diseases caused by nonsense mutations

http://markets.businessinsider.com/news/stocks/Eloxx-Pharmaceuticals-Announces-Expansion-of-Intellectual-Property-Estate-for-ELX-02-with-Newly-Granted-Patent-in-Europe-1004482616

Eloxx Pharmaceuticals Announces Expansion of Intellectual Property Estate for ELX-02 with Newly Granted Patent in Europe

Sevion Therapeutics, Inc. (OTCQB:SVON) and Eloxx Continue reading Biopharmaceutical company developing novel small molecule medicines for genetic diseases caused by nonsense mutations

New Inhaled Antibiotic To Treat Respiratory Infections

https://www.europeanpharmaceuticalreview.com/news/67983/polyphor-antibiotic-murepavadin/

Polyphor launches the development of an inhaled antibiotic murepavadin

Polyphor Ltd has announced the development of an inhaled dosage form of its break­through antibiotic Murepavadin. Continue reading New Inhaled Antibiotic To Treat Respiratory Infections

New Pre-Clinical Data for Iclaprim

Motif Bio Presents New Pre-Clinical Data for Iclaprim at IDWeek 2017™

https://globenewswire.com/news-release/2017/10/06/1142163/0/en/Motif-Bio-Presents-New-Pre-Clinical-Data-for-Iclaprim-at-IDWeek-2017.html

1. Pre-Clinical Data Support the Potential Use of Iclaprim in the Treatment of Staphylococcus aureus Pneumonia in Cystic Continue reading New Pre-Clinical Data for Iclaprim

New Nitric Oxide Treatment Eradicates Emerging ‘Superbugs’

http://markets.businessinsider.com/news/stocks/New-Nitric-Oxide-Treatment-Eradicates-Emerging-Superbugs-1002996746

Novoclem Therapeutics, a biotech company focused on advancing nitric oxide therapies for the treatment of respiratory diseases, today announced that data from an in vitro study with Continue reading New Nitric Oxide Treatment Eradicates Emerging ‘Superbugs’

Antifungal Drug Candidate Receives a Second “Qualified Infectious Disease Product” Designation

Pulmatrix Antifungal Drug Candidate Receives a Second “Qualified Infectious Disease Product” (QIDP) Designation from the FDA

http://markets.businessinsider.com/news/stocks/Pulmatrix-Antifungal-Drug-Candidate-Receives-a-Second-Qualified-Infectious-Disease-Product-QIDP-Designation-from-the-FDA-1002944255 Continue reading Antifungal Drug Candidate Receives a Second “Qualified Infectious Disease Product” Designation

Phase 1b Trial of QR-010

https://globenewswire.com/news-release/2017/08/29/1101646/0/en/ProQR-Completes-Dosing-of-Cystic-Fibrosis-Patients-in-QR-010-Phase-1b-Trial.html

ProQR Completes Dosing of Cystic Fibrosis Patients in QR-010 Phase 1b Trial

Key Updates
• Last patient received their final dose in the PQ-010-001 Phase 1b clinical trial of QR-010 in CF patients with the F508del mutation.
Top-line trial data are expected to be issued in a press release Continue reading Phase 1b Trial of QR-010

Vertex Drug Gets Priority Review for Cystic Fibrosis

http://www.empr.com/drugs-in-the-pipeline/tezacaftor-ivacaftor-vertex-cystic-fibrosis-priority-review-nda/article/684284/

Tezacaftor/Ivacaftor Gets Priority Review for Cystic Fibrosis

The Food and Drug Administration (FDA) has granted Priority Review to the New Drug Application (NDA) of tezacaftor/ivacaftor (Vertex) for the treatment of patients ≥12yrs old with cystic fibrosis (CF) who have two copies of the F508del mutation or one F508del mutation and one residual function mutation.

The NDA submission was based on positive results from 2 global Phase 3 trials, which showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in patients treated with tezacaftor/ivacaftor.

The combination treatment consists of ivacaftor (marketed under the brand name Kalydeco), a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, and tezacaftor, a novel CFTR corrector. Tezacaftor is designed to address the processing defect of F508del-CFTR to enable it to reach the cell surface, where ivacaftor can further enhance the protein’s function.

The FDA has set a Prescription Drug User Fee Act (PDUFA) target date of February 28, 2018 to make a decision on the NDA.

For more information visit Vrtx.com.

Two clinical trials presented at the European Cystic Fibrosis Conference

https://globenewswire.com/news-release/2017/08/17/1088301/0/en/Fluidigm-Licenses-CFTR-Next-Generation-Sequencing-Assay-From-Baylor-Genetics-for-Use-With-the-Juno-System.html

ProQR Announces Results for the Second Quarter of 2017

Key updates
• Data from two clinical trials of QR-010 presented at the European Cystic Fibrosis Conference. Enrollment completed in the Phase 1b clinical trial in cystic Continue reading Two clinical trials presented at the European Cystic Fibrosis Conference