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N30 Pharmaceuticals company

Question
Hello

Do you have information regarding the progress of research performed on 2 molecules ((n91115 et n6022) from N30 Pharmaceuticals company?
Can we have hope that this type of drug could emerge soon?
Do you think that these molecules and the miglustat, based on their mechanism of action, could be polyvalent and have an effect on all mutations?

Thank for all you do for parents and patients.

Best regards
Answer
Hello

The N30 Pharmaceuticals company has developed these 2 molecules N91115 and N6022 which are inhibitors of the S-nitroglutathione reductase (GSNOR). A decrease of intracellular GSNO levels, due to an increased activity of GSNOR (GSNO reductase), is associated with inflammatory lung disease. Inhibition of the reductase should allow a good level of intracellular GSNO and reduce inflammatory symptoms.
The company announces on ist website the beginning of a phase 1 study of orally administered N91115 in order to evaluate the safety, the tolerability in healthy volunteers.
A phase 1 trial is ongoing, to evaluate the safety of N6022, an intravenous GSNOR inhibitor, in patients who are homozygous for the F508del mutation.
A priori, these 2 molecules seem not to have an effect on all CFTR mutations, because preclinical studies have been done on the F508del mutation.
Regarding Miglustat, 2 phase II trials, conducted by the pharmaceutical company Actelion (which markets Miglustat) first in Spain then in Belgium, have failed to draw clear conclusions about the ability of Miglustat to restore the function of the CFTR protein in patients with the F508del mutation. The data showed considerable variability between patients treated by the same treatment (miglustat or placebo).
However, research does not stop and collaboration between the group of public hospitals in Paris and the pharmaceutical company Actelion should allow to perform a phase II trial. The establishment of contractual agreements and obtaining regulatory approvals take longer than expected.


Best regards,
Anna Ronayette
26.05.2014