Protalix Biotherapeutics and SarcoMed USA have entered into an exclusive worldwide license agreement to advance PRX-110 (alidornase alfa) as a treatment of pulmonary sarcoidosis and other respiratory diseases.
“Most patients with pulmonary sarcoidosis do not show symptoms and do not realize they have the disease until it has progressed to later stages. Currently available treatment options vary significantly from patient to patient, and the treatments generally either control symptoms or improve the patient’s lung function,” Dror Bashan, president and CEO at Protalix, the therapy’s developer, said in a press release.
“A treatment that both controls symptoms and improves the patient’s lung function is needed in the market today. We believe PRX-110 has the potential to be an exciting treatment option,” Bashan added.
The U.S. Food and Drug Administration designated PRX-110 an orphan drug last year as a potential sarcoidosis treatment. This designation, given to therapies aiming to treat or prevent diseases affecting less than 200,000 people in the U.S., offers financial and other incentives to support and speed development and regulatory review.
An inhaled treatment, PRX-110 is a chemically modified, recombinant (lab-made) form of human deoxyribonuclease I (DNase I) produced using plant cells via Protalix’s patented plant-cell based ProCellEx system. DNase I, an enzyme, is able to degrade some types of DNA molecules.
The presence of microbial DNA from bacteria has been identified in lung tissue of sarcoidosis patients, indicating it might have a role in disease development.
As microbial DNA is known to activate an immune response, research suggests it might trigger chronic inflammation. The DNase activity of PRX-110 may be able to degrade microbial DNA to ease or eliminate inflammatory signaling.
PRX-110 is also being investigated as a potential therapy for pulmonary fibrosis and cystic fibrosis (CF), among other potential pulmonary disorders. Its use in a proof-of-concept study in CF patients was reported to show promise in clearing sticky mucus from the lungs.
“We are pleased to partner with SarcoMed USA, a privately-held company led by professionals with a wealth of experience in the research and development of pharmaceuticals,” Bashan said.
SarcoMed will identify and choose pharmaceutical candidates under the license, and be responsible for their clinical research and development.
The agreement includes a $3.5 million upfront payment to Protalix, subject to certain conditions, plus additional milestone payments and royalties on any sales under the license agreement.
SarcoMed’s lead candidate, SM001, is also a DNase I compound being investigated as a potential treatment for chronic pulmonary inflammation in people with pulmonary sarcoidosis.