Positive Results for Phase 3 Studies of the Tezacaftor/Ivacaftor Combination Treatment

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced that the New England Journal of Medicine (NEJM) published two articles with results from two Phase 3 studies of the tezacaftor/ivacaftor combination treatment, a medicine in development that is designed to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Continue reading Positive Results for Phase 3 Studies of the Tezacaftor/Ivacaftor Combination Treatment

Live Stream the North American CF Conference Tomorrow for Free!

The North American CF Conference (NACFC) provides a collaborative and educational forum for all CF professionals. The educational elements of the meeting program are targeted to physicians, nurses, research scientists, respiratory therapists, physical therapists, nutritionists, social workers, and pharmacists. Continue reading Live Stream the North American CF Conference Tomorrow for Free!

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

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 III study starts AVAIL study

Savara Announces Start of Pivotal Phase III AVAIL Study of AeroVanc

http://markets.businessinsider.com/news/stocks/Savara-Announces-Start-of-Pivotal-Phase-III-AVAIL-Study-of-AeroVanc-1002569033

Savara, Inc.(NASDAQ: SVRA), a clinical-stage specialty pharmaceutical company focused on the development Continue reading Phase III study starts AVAIL study

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

Phase Two Clinical Trials to treat NTM

http://www.nasdaq.com/press-release/ait-therapeutics-announces-enrollment-of-first-patient-into-its-nontm-abscessus-phase-2-trial-in-20170807-00641

AIT Therapeutics Announces Enrollment of First Patient into Its NO-NTM abscessus Phase 2 Trial in Nontuberculous Mycobacteria (NTM)

10 patients with Mycobacterium Abscessus Complex (MABSC) are expected to enroll in the trial Continue reading Phase Two Clinical Trials to treat NTM

Rare mutation cell collection (RARE) (RARE-OB-16)

https://www.cff.org/Trials/finder/details/477/Rare-mutation-cell-collection-RARE

This study is taking place at multiple care centers across the U.S. Researchers will collect and make available for study cells from people with rare CFTR mutations.

This study will consist of a single visit where researchers will collect nasal cells and a blood sample. CFTR genotype will be confirmed and a repository of rare CFTR mutation specimens will be established and made available to other researchers for further study.

This study is for people with CF over the age of 2 who have a rare CFTR mutation. This study may require nasal swabs and/or other methods of cell collection.