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Commercialization of catalytic enhancers targeting IDO1 for multiple sclerosis

Project description

A novel drug against multiple sclerosis

Multiple sclerosis (MS) is a chronic neurodegenerative disease characterised by debilitating relapses associated with loss of neurological function. MS affects millions of individuals worldwide, but existing treatment options are primarily palliative. The EU-funded ENHANCIDO project aims to evaluate novel small molecules potentiating the activity of the immunosuppressive enzyme IDO1 known to reduce MS severity in animal models. By evaluating the efficacy, pharmacokinetics and pharmacodynamics of these compounds, scientists hope to deliver an innovative first-in-class drug that will improve the lives of MS patients. More importantly, this drug has the potential to be used to treat other autoimmune diseases.

Objective

ENHANCIDO will assess the commercial and technical feasibility of novel immune tolerance-inducing small molecules as superior treatments for multiple sclerosis (MS). MS is a chronic neurodegenerative disease affecting 2.3 million people worldwide. 85% of MS cases belong to the relapsing-remitting subtype (RRMS) characterised by bouts of recurring and debilitating relapses followed by unstoppable loss of neurological functions. Unfortunately, current treatments for RRMS are ineffective, poorly tolerated, and expensive. They cannot prevent disease progression and only provide symptomatic relief. Furthermore, severe adverse effects and inconvenient administration routes contribute to high rates of treatment discontinuation amongst patients (30-80%). To tackle these issues, Prof. Ursula Grohmann has developed a set of novel, orally administered, effective, and safe small molecules that induce immune tolerance to alter the previously inexorable course of the disease. These first-in-class molecules are positive allosteric modulators (PAMs) of the immunoregulatory enzyme IDO1. Prof. Grohmann’s PAMs increase IDO1 activity, reduce disease severity in mouse models of acute MS and show no off-target effects. Within ENHANCIDO, we will assess whether IDO1 PAMs are feasible as breakthrough treatments for RRMS. Firstly, we will validate their technical feasibility by evaluating their efficacy, pharmacokinetics and pharmacodynamics in RRMS mouse models. Subsequently, commercial feasibility will be determined by verifying IP position and strategy, performing in-depth market and competitor analyses, and finally consolidating these into a business plan to establish the best path to commercialisation. Successful commercialisation of IDO1 PAMs could reduce the profound socioeconomic burden of MS, extend and improve the lives of patients, and provide the pharmaceutical industry with a profitable, first-in-kind drug that can potentially be used for other autoimmune diseases.

Host institution

UNIVERSITA DEGLI STUDI DI PERUGIA
Net EU contribution
€ 150 000,00
Address
PIAZZA DELL UNIVERSITA 1
06123 Perugia
Italy

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Region
Centro (IT) Umbria Perugia
Activity type
Higher or Secondary Education Establishments
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Total cost
No data

Beneficiaries (1)