What is cystinuria and what are its symptoms?

Cystinuria is a genetic disease characterized by a buildup of the amino acid cystine in the kidneys and bladder.

These high levels of cystine lead to the formation of cystine stones in the kidneys, ureter and bladder. These cystine stones generate various complications, such as hypertension, intense abdominal pain, recurrent urinary tract infections, renal function impairment in up to 70% of patients and chronic renal insufficiency. These complications can ultimately lead to renal failure.

Signs and symptoms of the condition are related to the presence of stones and may include nausea, hematuria (blood in the urine), flank pain and/or frequent urinary tract infections.

There are estimated to be 70,000 and 25,000-35,000 patients with cystinuria in Europe and the United States, respectively.

  How is cystinuria treated?

In order to prevent cystine stones, cystinuric patients require around-the-clock control of their urine pH. Treatment of cystinuria involves the combination of 1) alkalizing treatments to increase urine pH for higher cystine solubility, 2) hyperdiuresis, which reduces cystine concentration by increasing urine production and excretion, and 3) a protein-poor diet, since proteins are high in cystine.

Of the available alkalizing therapies for the management of cystinuria, citrate is the intervention of choice as citrate excretion in urine potentially prevents calcium salt crystallization in alkali conditions (over a pH of 7.5). Potassium salts do not lead to the adverse consequences observed with sodium salts, which had been historically used as an alkalizing therapy. Moreover, it is recommended that potassium deficiency be avoided in cystinuric patients since this condition may decrease citrate excretion and cause renal acidosis, a buildup of acids in the blood.

There are currently no approved drugs, including potassium citrate/potassium bicarbonate, specifically indicated for the treatment of cystinuria. Advicenne’s multi-particulate formulation provides prolonged release and absorption of potassium citrate and potassium bicarbonate along the gastrointestinal tract while avoiding abdominal pain. The formulation maintains a sustained-release over 12 hours for a twice-a-day administration.

Since its founding in 2007, Advicenne has been dedicated to developing and commercializing innovative treatments that address unmet medical needs in the area of nephrology. Our lead drug candidate, ADV7103, is in late-stage clinical trials for two indications: distal renal tubular acidosis (dRTA) and cystinuria. Listed on both the Euronext Paris and Euronext Brussels stock exchanges, the company is headquartered in Nîmes, France.

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