Read about this interesting form of help for the Dutch CF Patients
Vanda Pharmaceuticals Inc announced that it has entered into a license agreement with UC San Francisco (UCSF), under which Vanda will acquire an exclusive worldwide license from UCSF to develop and commercialize a portfolio of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) activators and inhibitors.
New for me and added to list
Read more form CF Roundtable below:
We have completed a phase I trial with 18 healthy volunteers. alidornase alfa (PRX-110) was found to be safe and tolerable.
In July 2016, the first patient was dosed in Protalix’s phase IIa clinical trial of alidornase alfa (PRX-110). In January 2017, Protalix announced positive interim results from the phase II clinical trial for the first 13 CF patients enrolled in the study. At that time, 15 patients had been enrolled in, and were expected to complete, the study. The initial primary efficacy result shows that alidornase alfa (PRX-110) improves lung function as demonstrated by a mean absolute increase in the percent predicted forced expiratory volume in one second (ppFEV1) of 4.1 points from baseline. A commercially available small molecule CFTR modulator for the treatment of CF has reported a mean absolute increase in ppFEV1 of 2.5 from baseline in its registration clinical study. This score was achieved while 74% of the patients participating in the trial of the CFTR modulator were also treated with Pulmozyme® on top of the modulator. While this marketed CFTR addresses a certain mutation applicable to less than 50% of CF patients, alidornase alfa (PRX-110) is being developed to treat all CF patients.
Sputa available DNA samples were analyzed for approximately half of the patients. A mean reduction of approximately 60% in DNA content from baseline was observed, and a mean reduction of approximately 90% from baseline was observed for sputa visco-elasticity. This data provides further supportive evidence of improved lung function after treatment with alidornase alfa (PRX-110), as demonstrated by the increase in ppFEV1.
No serious adverse events were reported, and all adverse events that occurred during the study were mild and transient in nature.
The phase II trial is a 28-day switch-over study of 15 CF patients previously treated with Pulmozyme® to evaluate the efficacy and safety of alidornase alfa (PRX-110) in CF patients. Participation in the trial is preceded by a two-week washout period from Pulmozyme® before treatment with PRX-110 via inhalation. The main efficacy endpoint is the change of forced expiratory volume (FEV1) and forced vital capacity (FVC). Additional endpoints include safety and tolerability, immunogenicity and pharmacokinetic data. In the trial, alidornase alfa (PRX-110) is administered through Philips Respironics’ I-neb AAD Inhaler System, for which Protalix has a supply agreement for the exclusive use of the device for the development of an inhaled product based on dornase alfa for the treatment of CF. The I-neb AAD is a small, lightweight, virtually silent device that is fully portable and has a unique vibrating mesh technology that allows for faster administration than conventional jet or ultrasonic nebulizers.
“Patients in the highest dose cohort of anabasum (20 mg orally, twice per day) had a 75% reduction in the annualized rate of pulmonary exacerbations requiring IV antibiotics compared to placebo cohort.”
Looking forward for more details on these data
Corbus Pharmaceuticals Reports Positive Topline Data Demonstrating Anabasum Reduces Acute Pulmonary Exacerbations and Multiple Inflammatory Biomarkers in Phase 2 Study in Patients with Cystic Fibrosis :: Corbus Pharmaceuticals Holdings, Inc. (CRBP)
Spyryx Biosciences Expands Executive Team and advances it’s SPX-101 to Phase 1
This one is new to me, and I added it to the pipeline. Polyphor has completed a Phase 1 whit this and is now moving on to 1b in CF individuals. It is a inhibitor of hNE (human neutrophil elastase)
Vertex makes another collaboration, broadening its CF portfolio
Added to the pipeline under RNA therapies
Drug candidate added to pipeline after recently granted Orphan Drug Designation.
A novel new drug being developed to treat antibiotic-resistant lung infections shows significant promise for treating the often life-threatening mixed bacterial and fungal lung infections contracted by cystic fibrosis patients,
In the study, Mul-1867 provided 80 percent and greater protection against mortality from lung infections caused by a mix of Pseudomonas aeruginosa and Staphylococcus aureusbacteria and a bacterial-fungal mix of Pseudomonas aeruginosa and the fungi Candida albicans.
By comparison, mice treated with an aminoglycoside antibiotic widely used for the treatment of lung infections in cystic fibrosis patients had only a 50 percent survival rate in the experimental models.
ProMetic (a new company to me) begins a 90 patients wide Phase 2 of its oral drug PBI-4050. I added the drug to the list.