View Full Version : N30 phase 1b/2a trial with CFs who have 2 copies of F508del

03-14-2013, 06:12 AM
N30 has started a phase 1b/2a trial with CFs who have 2 copies of F508del. The N6022 is both anti-inflammatory and a CFTR corrector.

"N6022 is the first of a novel class of N30 Pharma's drugs aimed at conservation of S-nitrosoglutathione (GSNO) a key, endogenous, signaling molecule that has been shown to be reduced in cystic fibrosis patients. N30 Pharma's drugs restore GSNO levels by inhibiting GSNOR, the enzyme in the body that breaks down GSNO.

N30 Pharma's GSNOR inhibitors have dual effects in cystic fibrosis. First, in experimental models of cystic fibrosis, they rescue or "correct" the abnormal chloride channel function that causes the thick, tenacious secretions of the lung and other organs characteristic of cystic fibrosis. Secondly, they are potently anti- inflammatory. This is potentially important because inflammation, particularly of the lung, is the cause of major morbidity in CF patients."


Here are the study details, looks like they are recruiting in Children's Hospital Colorado, National Jewish Health and the University of North Carolina: http://www.clinicaltrials.gov/ct2/show/NCT01746784?term=N6022&rank=2

03-14-2013, 12:38 PM
Hello all. I don't post frequently though I am very active in CF Research & development as well as being a CF-Dad to a 13 year old girl. I have been following N30 for some time. I am convinced that N6022 is a VERY exciting drug but not getting the attention it deserves - I believe N6022 has even more potential to help CF-ers than anything else out there (perhaps more than...ie Kalydeco & VX809)

What's important to note is that they have 18 sites for this first trial...so way more that Colorado, National Jewish Health & UNC. If you or your loved one is a DD508, I highly urge your involvement. Their approach to treating CF has me on the edge of my seat. To learn more, the contact person at N30 is [email protected] and here's a link to their corporate web site www.n30pharma.com


03-16-2013, 07:34 PM
Where are these 18 sites? I don't see anything listed anywhere but Colorado and North Carolina.

03-26-2013, 05:49 AM
Where are these 18 sites? I don't see anything listed anywhere but Colorado and North Carolina.

Cincinnati Children's Hospital was just added to the clinical trials website. The website also says 'Up to 5 cohorts will be studied with a total of 67 patients at approximately 18 clinical sites in the United States.'

I just re-read the press release and noticed it said that in addition to the intravenous N6022 mentioned above "N30 Pharma is rapidly advancing an oral dosage form, N91115, through preclinical testing and IND submission. Clinical trials of N91115 are targeted to begin in the first half of 2014."

04-03-2013, 09:26 AM
Latest update from N30: http://www.news-medical.net/news/20130402/N30-Pharmaceuticals-presents-preclinical-data-of-GSNORi-at-European-Cystic-Fibrosis-Society-meeting.aspx and http://www.n30pharma.com/docs/news/2013-04-02-Malaga.pdf
Link to the scientific information: http://www.n30pharma.com/science/posters/

The March 21 poster includes this comment: "These data suggest that GSNOR inhibitors can modulate CFTR function in cell based systems. Together with other benefits of restoring GSNO levels in the lung such as bronchodilation, anti-inflammatory effects, and enhanced ciliary function, these inhibitors may be beneficial in the treatment of F508del CFTR patients."

04-17-2013, 10:54 AM
More sites have been listed:

Alabama: University of Alabama
Colorado: Children's Hospital Colorado, National Jewish Health
Maryland: Johns Hopkins Hospital
Minnesota: University of Minnesota
Missouri: Washington University
North Carolina: University of North Carolina
Ohio: Cincinnati Children's Hospital, Nationwide Children's Hospital


04-17-2013, 03:34 PM
VERY exciting! You only hear about kalydeco, so good to hear about other pharmaceudical studies for DDF508. :) Thanks so much for posting.

04-17-2013, 04:31 PM
I had no idea how exciting this was, I may be participating in this study!