Lynovex (Cysteamine)

Question

Dear team, while surfing in the Internet, I came across the above mentioned candidate that is acutally in Phase IIb. I was not able to find anything about it on the German CF websites. The pharmaceutical newspaper [German newspaper] reported in January 2015:
“The experimental antibacterial substance NM001 (Lynovex®) is effective against gram-positive and negative germs, that are associated with CF, like Pseudomonas aeruginosa or Burkholderia cepacia. It prohibits furthermore the formation of biofilms, that lead in CF patients to persistent and antibiotic-resistant infections of the lung. Additionally the small molecule NM001 had a mucolytic effect in vitro and ex vivo. Actually, the molecule is in phase IIa of clinical investigations (1,4).” Source: http://www.pharmazeutische-zeitung.de/index.php?id=55785
On the 38th European CF congress in the middle of the year, positive data was reported from phase IIa. Source: http://www.novabiotics.co.uk/blog/2015/06/positive-lynovex-phase-iia-data-presented-at-the-38th-european-cystic-fibrosis-symposium
Question:
Is this molecule a mucolytic substance or an antibiotic drug?
Do you have further information about this drug candidate?
Many thanks for your efforts and best regards,

Answer

Dear questioner,
Cysteamine is an aminothiol that is chemically closely related to the aminoacid L-Cysteine respecitvely L-Cystin, that is composed of two Cysteine molecules. Cysteamine is a degradation product of Cysteine and occurs in the human body as part of Coenzyme A, that is important for the metabolism.
Cysteamine is known for years as a drug for treating nephropathic cystinose, that is a kidney disease and thereofore some experience exist on the side effect profile respectively the generally good tolerance of Cysteamine.
Cysteamine is thought to have a mucolytic effect, which is not surprising as on the other hand it is chemically close to N-acetylcysteine, that is a classical mucolytic drug. The mucolytic effect of the N-acetylcysteine is based on the destruction of disulfide bridges of the mucopolysaccharides in the mucus of the airways.
Cysteamine is recently thought to have also an antibacterial effect, as it is capable itself alone or in combination with certain antibiotic drugs (e.g. Tobramycin) to reduce the germ count in the sputum of CF patients. Cysteamine is not an antibiotic in the general sense, i.a. because the mode of reaction is not clarified yet. This could be an anti-biofilm way of action, as could be shown on the example of Pseudomonas aeruginosa (destruction of the building of the biofilm respectively the integrity of the biofilm). The mode of action of germ reduction does not seem to be specific for a certain species, as effectiveness could be shown for different CF germs. The data we have so far, is based only on in vitro results.
Cysteamin has the status of a so-called “orphan drug” (a drug for rare diseases) and is actually in phase I/II studies in Italy and Great Britain for the indication of CF. Those results have not been published yet to my knowledge. About the clinical effectiveness there is therefore still too few data, so that the benefit for the CF therapy can at the moment not yet be quantified.
With best regards,
Michael Hogardt

14.01.2016