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 varies depending on the complexity of the test and the disease.

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. 

ROT1 has been identified as a predictor of patient’s response to ASeRT.  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.

During the Phase IIa clinical trial 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 that ROT1 correlated highly with this response. 

The method of action by orally administrated ARG201 is Antigen-Specific Regulatory T-Cell  induction (ASeRT Induction).
  • ROT1 identifies patients responsive to ASeRT in autoimmune indications, such as Scleroderma, RA, IPF, food allergies, Crohn’s and others.
  •  ROT1 located in the noncoding/regulatory region proximal to the gene for Interferon-γ.
  •  ROT1-AA (ROT1+) patients do not develop ASeRT induction (~33% of population).
  •  ROT1-GA or GG (ROT1-) patients develop ASeRT induction (~67% of population).
  •  Stratification of patients using ROT1 genotyping will reduce the risk and costs in clinical studies.
  •  ROT1 has independent US & UE IP coverage.
  •  Development of an FDA approved companion diagnostic kit
  •  Commercial use as diagnostics in other oral tolerance and autoimmune indications
  •  Kit-based ROT1 genotyping has independent commercial value.

Research indicates that ROT1 is applicable to other diseases being treated by oral immune tolerance induction.  There are US and EU patents issued for ROT1.

Aut​oImmune Disease Treatments

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