Project description
Breakthrough treatment for acute pancreatitis targeting Store-operated calcium entry
Store-operated calcium entry (SOCE), or capacitative calcium entry, is a mechanism by which the emptying of endoplasmic reticulum calcium stores causes calcium influx across the plasma membrane. Calcium overload associated with SOCE hyper-activation has been implicated as a trigger in a cluster of diseases, including acute pancreatitis (AP), which affects over 10 million people globally. Early preclinical data identified a drug candidate targeting the SOCE process, which might lead to a breakthrough in the pharmacological treatment of AP. The EU-funded RAPID project is supporting the completion of the preclinical studies, aiming to prove that the new compound is suitable for systemic application. The results will pave the way for Phase I and II clinical trials.
Objective
ChemICare is a high-tech spin-off of the University of Piemonte Orientale and has patented and preclinically developed small molecules that are unique in their capacity to modulate calcium ions uptake in cellular processes. Calcium overload associated with Store-Operated Calcium Entry hyper-activation has been proved to be to be the key trigger in a cluster of diseases, including Acute Pancreatitis, which affects over 10 million people globally and is the most common gastroenterology discharge diagnosis in Europe and the US. AP is associated with significant disease burden, emergency room visits, frequent hospitalizations, and patients often undergo endoscopic and surgical procedures and are admitted to intensive care, costing more than 20 B€ yearly to National Healthcare Services. Preclinical data obtained so far demonstrate that drug candidate 39 represents a breakthrough innovation in the field of pharmacological therapies for AP. We are now in the process of generating the last data to complete the preclinical regulatory dossier, which will pave the way for the Phase I and II clinical trials which will lead to an out-license agreement to a large pharma company prior to the initiation of Phase III. The revenues will therefore be reinvested to finance development of the same technology for other therapeutic indications associated with calcium overload (e.g. Duchenne Muscular Dystrophy and Tubular Aggregate Myopathy). This Phase 1 is of utmost importance to validate the market position of ChemICare molecules, apply for orphan designation, and plan the preclinical study necessary to prepare the clinical validation Phases in the frame of the Phase 2. Our goal is to have a solution ready for the market by 2024, which will be cost-effective for health providers and deliver optimal care to patients with AP. Such rapid time-to-market is possible because both EMA and FDA have accelerated paths for rare disease solutions that we will take advantage of.
Fields of science
Programme(s)
Funding Scheme
SME-1 - SME instrument phase 1Coordinator
28100 NOVARA
Italy
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.