SarcoMed USA, Inc. is focused on the development of SM001 (alidornase), a therapeutic candidate aimed at improving outcomes for patients with pulmonary sarcoidosis. Established in 2017, the company has assembled a team of drug development experts and has made significant progress, including receiving orphan drug status from the FDA following a successful Pre-IND meeting in 2021. The company recognizes the substantial market potential for SM001, estimating that it could generate over a billion dollars annually while addressing a critical unmet need for patients suffering from pulmonary sarcoidosis. Additionally, SarcoMed is investigating other potential indications for SM001, including Adult Respiratory Distress Syndrome, Idiopathic Pulmonary Fibrosis, and Cystic Fibrosis.

The clinical development of SM001 has included rigorous testing, with two studies conducted to assess its biosimilarity to the reference drug Pulmozyme. A Phase 1 open-label randomized trial demonstrated that SM001 and Pulmozyme had comparable safety, tolerability, and pharmacokinetic profiles in healthy volunteers. Furthermore, a Phase 2 trial involving cystic fibrosis patients showed similar efficacy in improving lung function, as measured by changes in FEV1, between the two treatments. Throughout these studies, no significant differences in adverse events or pharmacokinetic parameters were observed, indicating that SM001 is a viable alternative to Pulmozyme for patients requiring treatment for respiratory conditions.

United States

SarcoMed USA was formed in Texas in 2017 to determine if Pulmozyme could improve Pulmonary Sarcoidosis. The company is nearing a final license for the treatment of pulmonary sarcoidosis and related respiratory diseases via inhaled delivery.

SarcoMed USA

Company Details