SarcoMed USA was formed in 2017 to investigate if a novel DNase 1 compound (SM001) could influence the chronic pulmonary inflammation seen in Pulmonary Sarcoidosis patients.
Sarcoidosis is a systemic granulomatous disease characterized by a T-helper-1 response with accumulation of CD4+ lymphocytes and activated macrophages in the lungs and affected organs, resulting in characteristic granuloma formation. The lungs are involved in more than 90% of patients with sarcoidosis, thus abnormal pulmonary function is characteristic for many patients. Sarcoidosis affects approximately 200,000 people in the United States and at present corticosteroids are the only approved treatments.
A reduction in the use of steroids is a significant unmet need for sarcoidosis patients
The presence of microbial DNA (mDNA) from mycobacteria, propionibacteria and borrelia have been identified in sarcoidosis tissues suggesting their role in sarcoidosis granuloma formation
- Bacterial amyloid curli act as a carrier for such DNA to elicit an autoimmune response
- Serum amyloid A (SAA) derived from the host may participate in the granuloma formation by regulating inflammation through Toll-like receptor-2 (TLR2)
- SAA localized to macrophages and giant cells within sarcoidosis granulomas correlate with CD3(+) lymphocytes, linking expression to local Th1 responses
- Additional antigens implicated in initiating the inflammatory cascade leading to granuloma formation include beta-actin, intermediate filament vimentin and pathogen-associated molecular patterns (PAMPs)
The DNase activity of SM001 is expected to degrade the microbial DNA within the protein/DNA complex present in lungs of patients with pulmonary sarcoidosis (extracellular red X marks). The reduction of the burden of bacterial DNA could then reduce or eliminate the antigenic stimulation responsible for the persistent immunological stimulation causing granuloma formation (See intracellular orange X marks).
Lead product candidate, SM001, is a recombinant form of human deoxyribonuclease I (DNase I), with a proven mechanism of action and a differentiated clinical profile.
Clear development and regulatory pathway along with FDA Orphan status designation granted for the treatment of sarcoidosis.
Phase 1 healthy subject clinical study completed (SAD, MAD with 18 subjects). SarcoMed is planning to initiate a Phase 1b Randomized, Double-Blind, Placebo-Controlled Multiple Ascending Dose Study of Inhalation SM001 in Patients with Pulmonary Sarcoidosis.
Please contact us should you have an interest in learning more about our efforts.