SARCOMED SM001
SarcoMed USA is focused on developing DNase I of an independent source (Protalix) for treating unmet medical need not currently addressed by existing marketed products. The lead product candidate, SM001 is a modified recombinant human DNase I (chemically amidated post expression in plant cells, INN alidornase alfa) presented as a drug product formulated at 5 mg strength which has been used with the Phillips nebulizer. SM001, alidornase alfa, has a similar mechanism of action to dornase alfa, the active ingredient in Pulmozyme, registered for the management of cystic fibrosis (CF) patients to improve daily function. Based on prior experience with SM001 in early clinical studies in CF and success of DNase I in a FDA-recognized animal model, SarcoMed USA aims to leverage a developed drug product presentation and device for inhalation to address the unmet medical need of pulmonary sarcoidosis.
ABOUT SM001
SarcoMed USA is focused on developing DNase I of an independent source (Protalix) for treating unmet medical need not currently addressed by existing marketed products. The lead product candidate, SM001 is a modified recombinant human DNase I (chemically amidated post expression in plant cells, INN alidornase alfa) presented as a drug product formulated at 5 mg strength which has been used with the Phillips nebulizer. SM001, alidornase alfa, has a similar mechanism of action to dornase alfa, the active ingredient in Pulmozyme, registered for the management of cystic fibrosis (CF) patients to improve daily function. Based on prior experience with SM001 in early clinical studies in CF and success of DNase I in a FDA-recognized animal model, SarcoMed USA aims to leverage a developed drug product presentation and device for inhalation to address the unmet medical need of pulmonary sarcoidosis.
SM001 BACKGROUND
- Recombinant human DNase I has superior activity in breaking down extracellular DNA.
- Originally developed for Cystic Fibrosis by Generium Pharma.
- Pre-clinical and clinical studies showcase safety and efficacy.
- Finalizing license agreement of Tigerase from Generium.
- They have extensive drug discovery and development experience.
- Have back-end loaded term sheet and no milestones.
- Finalizing pilot study in Kazakhstan. ($1million).
- Plan to transfer tech from Generium to a U.S. or European manufacturer after POC study.
SM001’S MECHANISM OF ACTION
Systemic Inflammatory Disorder characterized by:
- Microbial(mDNA) & Mitochonrial DNA(mtDNA) along with Amyloid like protein aggregates are found in sarcoidosis tissues.
- Microbial DNA (mDNA) and Serum Amyloid A (SAA)aggregates cause inflammation mediated by TLR2 transport to TLR9.
- DNA stimulate Th1, Th17, TNF, IL6 Cytokines, which are critical to sarcoidosis inflammation seen in the lungs.
- DNase I minimizes or prevents granuloma.
Formation by breaking down the DNA before it can hit TLR9.
- DNA stimulate Th1, Th17, TNF, IL6 Cytokines, which are critical to sarcoidosis inflammation seen in the lungs.
- Chen et al. 2010. 'Serum amyloid A regulates granulomatous inflammation in sarcoidosis through Toll-like receptor-2', Am J Respir Crit Care Med, 181: 360-73.
- Drake et al. 2013. 'Effects of broad-spectrum antimycobacterial therapy on chronic pulmonary sarcoidosis', Sarcoidosis Vasc Diffuse Lung Dis, 30: 201-11.
- Eishi et al. 2002. 'Quantitative analysis of mycobacterial and propionibacterial DNA in lymph nodes ...', J Clin Microbiol, 40: 198-204.
- Gupta et al. 2007. 'Molecular evidence for the role of mycobacteria in sarcoidosis: a meta-analysis', Eur Respir J, 30: 508-16.
- TursiS, et al. (2017) Bacterial amyloid curli acts as a carrier for DNA to elicit an autoimmune response via TLR2 and TLR9. PLoS Pathog 13(4): e1006315.
TESTING SM001 (Tigerase)
The benefit aspects for patients participating in the present clinical study include free test drug product as well as complex of diagnostic procedures not included into the routine clinical practice which are also free under the present protocol to the study subjects. During the study, patients will be closely supervised by the medical institution personnel participating in the study and will be able to obtain more complete information on their health condition. The potential benefit for the patients may include effect of SM001 on pulmonary sarcoidosis.
