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 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.
