Protalix, SarcoMed USA Enter Non-binding Deal for Potential Sarcoidosis Treatment

Protalix BioTherapeutics and SarcoMed USA have entered into a non-binding agreement related to the development and commercialization of PRX-110 (alidornase alfa) for pulmonary sarcoidosis and related diseases, according to a Protalix press release.

PRX-110 is currently being developed by Protalix as a potential therapy for cystic fibrosis and other conditions, including sarcoidosis.

The therapy received orphan drug designation from the U.S. Food and Drug Administration this month for the treatment of sarcoidosis. This designation is given to medications that are intended to treat or prevent rare diseases or conditions, defined as those that affect fewer than 200,000 people in the United States.

The investigational therapy contains a recombinant form (lab-made) of the human deoxyribonuclease I (DNase I) made using plant cells — Protalix’s proprietary plant-cell based ProCellEx platform. DNase I is an enzyme that is able to cut certain kinds of DNA molecules.

In sarcoidosis patients, DNA from microbes, such as bacteria, is often identified in affected tissues, suggesting that microbial DNA plays a role in the disease.

More specifically, microbial DNA is known to trigger an immune response, as its presence is typically indicative of an infection. Sarcoidosis is characterized by increased immune system activity, leading to the formation of small clumps of inflammatory cells called granulomas in different tissues. Of note, in pulmonary sarcoidosis, the affected tissue is the lung.

Based on the fact that microbial DNA drives inflammation and that it is commonly found in sarcoidosis tissue, researchers hypothesized that microbial DNA could be a driver of the chronic inflammation that characterizes sarcoidosis.

DNase I is able to cut up microbial DNA, effectively removing it from the tissue milieu. By removing these DNA molecules, PRX-110 may eliminate a primary signal that drives inflammation, thus ultimately reducing inflammation and granuloma formation, and easing disease symptoms.

PRX-110 is also being investigated as a potential treatment for cystic fibrosis because, apart from its antimicrobial activity, it may help to loosen up the thick mucus that characterizes the disease. Preclinical data, as well as data from early clinical trials, have so far been supportive of the efficacy of PRX-110 in cystic fibrosis.

SarcoMed USA is also developing another DNase 1 compound, called SM001, as a potential treatment for chronic pulmonary inflammation in patients with pulmonary sarcoidosis.