Toleranzia is introducing Re-Tolerogen – a targeted immunotolerance platform for the treatment of autoimmune diseases
Autoimmune diseases are a diverse group of diseases caused by an abnormal immune response attacking tissues normally present in the body. This feature makes them difficult to treat since the disease causing agent can not be removed from the body. Today’s treatments are unspecific and only symptomatic and associated with several problems:
• They do not cure the disease, hence lifelong treatment is required.
• Although the symptoms are reduced the tissue destruction progress.
• They are aimed at unspecific components of the immune system, leading to high risks of severe side effects, such as increased susceptibility to infections.
• There is a high unmet need for new medications due to patient dissatisfaction.
Toleranzia is developing Re-Tolerogen, a platform technology, with the potential to revolutionize the treatment of autoimmune disease. The Re-Tolerogen technology provides targeted, disease specific, treatments that re-educate the immune system by inducing immune tolerance, thereby acting on the cause of the disease and not only the symptoms.
Fusion proteins developed using the Re-Tolerogen platform consists of three different parts that together form a unique treatment:
• Induction of tolerance
• Targeting directly to specific cells, allowing for a low treatment dose, and few side effects
• Specific treatments for each disease
These three features in combination with the fact that Re-Tolerogens are given as a nasal spray makes the Re-Tolerogens unique compared to other tolerance inducing approaches developed by competitors. Once on the market Re-Tolerogens will therefore be the first choice therapy.
The Re-Tolerogen platform is highly flexible and can be adapted to host peptide sequences that are relevant to a variety of autoimmune diseases and Proof of Concept in animal models have been achieved for the following diseases:
• Myasthenia Gravis
• Multiple Sclerosis
• Rheumatoid Arthritis
• Diabetes type1
• Undisclosed Orphan indications
Focus on Orphan diseases: Myasthenia Gravis
Toleranzia is focusing its efforts on developing a therapy for the treatment of the Orphan disease Myasthenia Gravis (MG). MG is a neuromuscular autoimmune disease with a prevalence of 0,02% that lacks specific treatment today. Due to the fact that rare diseases historically have been neglected by the large pharmaceutical companies the governments of several major markets provide special legislation to promote the development of treatments towards these diseases which have made them more attactive to the pharmaceutical market recently.
Toleranzia have proof of Concept in an animal model of MG for the MG:Re-Tolerogen and humanization is ongoing. Orphan Drug Designation will be applied for during 2014.
Customer benefits of Re-Tolerogen – The only targeted non-injectable tolerance treatment
Patient & care providers will find that Re-Tolerogen:
• prevents disease progression
• provides disease specific treatment
• has few, if any, side effects, such as general impairment of immune function
• is targeted, requiring only low doses and intranasal administration (”nasal spray”)
Pharmaceutical partners will find that Re-Tolerogen:
• is the first non-injectable tolerance inducer that targets the root cause of disease
• offers a fast and efficient platform for development of new therapies
• Orphan Drug Designation will allow for regulatory alleviations and a shorter time to market
• prolonged IP protection once on the market (Orphan indication)
Toleranzia own all IP connected to the platform. Patents are granted in some countries and in national phase in other.
• To develop products for treatment of autoimmune diseases
up to clinical trials/phase II, to be further exploited by pharmaceutical industry
• To offer development services and licenses to pharmaceutical industry
In-house, co-development and license model:
• Re-Tolerogen can be developed both in-house and in collaboration with other researchers and pharmaceutical companies, as well as by licensees.
• Toleranzia has extensive competence and its own lab facilities.