Renal lithiasis is the medical term for the presence of stones, i.e. an agglomerate of crystals that obstruct kidney or urinary tracts.
Renal lithiasis is a common pathology, affecting 5-9% of the population in Europe and is recurrent. If nothing is done, the recurrence rate for urinary lithiasis is estimated at 50% at 5 years.
They are frequently associated with Chronic Kidney Disease and are responsible for 2 to 3% of end-stage renal diseases.
There are multiple types of kidney stones which are composed of different minerals.
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.
Current diagnosis is achieved via stone analysis (infrared spectroscopy, electron microscopy coupled with X-Ray diffraction crystallography, microscopic examination) and/or quantitative analysis of cystine in urine.
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.
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.
AVA2206 is a natural thiolhistidine that may play a major role in the early process of crystallization of renal stone. Its activity has been demonstrated in preclinical model of cystinuria and AVA2206 has the potential to become a 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.
Co-founder of Advicenne in 2007 and Panntherapi in 2020, he is an expert in the clinical development of innovative products for pharmaceutical and biotech industry. He has conducted many clinical studies and has successfully conducted numerous drug development programs in the field of the central nervous system (CNS) and kidney diseases. In particular, he successfully conducted the proof-of-concept study of the « Blockbuster » duloxetine (Cymbalta, Xeristar, Yentreve), approved by the FDA in 2004. His long history of interactions with the U.S. FDA and European (EMA) regulatory agencies provides Averoa with a strategic regulatory vision that is essential to its development.
