arGentis
Pharmaceuticals, LLC

Aut​oImmune Disease Treatments

Scleroderma - ARG201


Scleroderma is a chronic autoimmune disease in which the skin and connective tissue tightens and hardens. Median survival from diagnosis is approximately 11 years. Patients experience a deteriorating quality of life and typically die from one of the many complications of the disease.   There is no cure or effective treatment for the disease. The most popular treatments control the inflammatory phase of scleroderma by the use of immunosuppressive therapy, which can also lead to a number of side effects, which mirror those associated with chemotherapy treatments.  

arGentis has targeted the diffuse version of Scleroderma to treat as it is the most devasting of the different types of Scleroderma.  Additionally, the reatement is targeted toward late stage patients (patients with 3 years or more suffering from the disease.

ARG201 will be an effective treatment which will transform the lives of patients, redefine the treatment standards, and be widely accepted by patients and physicians. The size of the addressable dcSSc market is expected to be over US$ 1B and EU€ 1B annually in the US and EU, respectively.  ARG201 has been designated an Orphan Drug in the US and the EU and has been designated a biologic in the US. There are patents issued in the US and pending in the US and EU.



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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.  The researchers believe this SNP is applicable to other diseases being treated by oral immune tolerance induction.  There are patents pending for ROT1.



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Rheumatoid Arthritis- ARG301


Rheumatoid arthritis is a chronic inflammatory disorder that typically affects the small joints in your hands and feet. Unlike the wear-and-tear damage of osteoarthritis, rheumatoid arthritis affects the lining of your joints, causing a painful swelling that can eventually result in bone erosion and joint deformity.


An autoimmune disorder, rheumatoid arthritis occurs when your immune system mistakenly attacks your own body's tissues. In addition to causing joint problems, rheumatoid arthritis sometimes can affect other organs of the body — such as the skin, eyes, lungs and blood vessels.


Although rheumatoid arthritis can occur at any age, it usually begins after age 40. The disorder is much more common in women than in men. Treatment focuses on controlling symptoms and preventing joint damage.


arGentis' treatment, ARG301, utilized a modified peptide ligand to treat the disease.  The first in man, Phase I clinical trail, is currently being conducted with results expected in 2015.  There are patents pending in the US and EU.

Treatments Under Development