LUCIOL project illustrates the global approach endorsed by AVEROA by developing a breakthrough treatment (AVA2206) and a diagnostic test (AVA2065) for a rare renal disease with large unmet medical needs worldwide.
The disease is a rare autosomal recessive disorder that affects young patients, and which results in painful lithiasis. The induced stones can cause painful symptomatology or even obstruction, requiring surgical interventions, infection, and ultimately chronic kidney disease.
CKD can be due to multiple causes: High blood pressure, diabetes, high cholesterol, kidney infections, glomerulonephritis, polycystic kidney disease, genetic conditions, autoimmune diseases, kidney stones, smoking, age, use of certain medicines etc.
There is no reliable marker of the disease activity that could predict the stone formation. An early and correct diagnosis is essential for the successful treatment of this renal disease.
Effective therapeutic option by preventing stone forming Well tolerated drug and Need for an advanced diagnosis and reliable biomarker
AVA2206 is a natural thiolhistidine that may play a major role in the early process of crystallization of renal stone. This has been demonstrated in preclinical model of cystinuria and AVA2206 has the potential to become the first line treatment for cystinuria targeting the cause leading to crystal formation.
Beyond Cystinuria, additional preclinical developments are planned to explore the full range of potential effects of AVA2206. As an inhibitor of crystal formation, AVA2206 novel mechanism of action may have high potential to be effective in various type of renal lithiasis.
AVEROA signed a research collaboration and license agreement with IdiBell, CIBER and University of Barcelona for the development to develop and exploit the product in the field of kidney and/or metabolic diseases.
AVA2206 is a product that could play a major role in renal lithiasis and has the potential to become the first line treatment as targeting the cause leading to crystal formation and ultimately to the symptomatology.
Animal proof of concept has confirmed the potential of AVA2206. Preclinical studies and clinical studies are planned in evaluating safety and pharmacokinetic in healthy volunteers
AVA2206 has been granted with an Orphan Drug Designation (ODD) by the European Medicine Agency (EMA) and the US FDA
AVA2065 is a new cystine lithiasis biomarker that was validated in a mouse model of the disease. This biomarker is very promising and its validation in human is already planned. The use of AVA2065 as a predictive and reliable biomarker of activity able to discriminate patients who meet diagnostic criteria for the disease could be an enormous added value to assess the response of the treatment and to predict outcomes.
