Pharmaceuticals, LLC

Aut​oImmune Disease Treatments

Genotype Marker - ROT1

There is a quest to identify biomarkers that enable physicians to choose treatments that will work in a specific patient.  In addition there is a quest to use biomarkers to identify patients who will have an adverse reaction to a treatment.  This is often referred to as “personalized medicine.”  The price of these bio-marker tests vary depending on the complexity of the test and the disease.

ROT1 has been identified as a predictor of patient’s response to oral immune tolerance induction.  Specifically it has been identified in the use of ARG201 to treat Systemic Scleroderma patients.  There are an estimated 100,000 Scleroderma patients in the US and approximately the same number in the EU.

ROT1 is an identified single nucleotide polymorphism (SNP) that indicates whether a patient will respond to immune tolerance.  If ROT1 is present, the patient will not respond to the oral tolerance treatment, hence it is designated “Resistance to Oral Tolerance” or ROT1. 

During the Phase IIa clinical trail for ARG201, the investigators detected that approximately 30% of the patients who should have responded to the treatment did not respond.  Upon further research, they identified a specific SNP that correlated highly with this response.  Research had indicated that this SNP is applicable to other diseases being treated by oral immune tolerance induction.  There are patents pending for ROT1.