A phase 1/2 study of the drug lumasiran for the treatment of primary hyperoxaluria Type 1 has produced encouraging results, reports Alnylam Pharmaceuticals. The full article can be read here, at Business Wire.
Primary hyperoxaluria Type 1 (PH1) is a rare condition that affects the kidneys and is caused by an overproduction of oxalate. In patients with PH1, excess oxalate combines with calcium to form a hard substance that accumulates to cause damage. Accumulations of this substance, known as calcium oxalate, can cause kidney stones, kidney damage, and in some cases kidney failure, as well as blood in the urine, urinary tract infections, and damage to other areas of the body. There are three types of the condition; type 1 is the form proposed to be treated with lumasiran. It makes up about 80% of cases of PH and typically leads to kidney stones by early adulthood and end-stage renal disease can occur at any age. Types 2 and 3 both account for an estimated 10% of cases each. Combined, all three forms of PH are thought to affect one in every 58,000 people worldwide.
Lumasiran, previously known as ALN-GO1, is an experimental drug being developed for PH1. It is an RNAi therapy, which means that it uses gene silencing techniques to treat conditions. Lumasiran is designed to reduce levels of the enzyme glycolate oxidase in the body. Since this specific enzyme is involved in the production of oxalate, reductions in the amount of it are thought to also reduce the level of oxalate. This is intended to alleviate the effects of PH1. This experimental treatment has already been granted Orphan Drug Designation in the US and EU, as well as Breakthrough Therapy Designation by the FDA, and a Priority Medicines designation from the EMA. These designations are designed to help the developers investigate the drug, and, following successful results and approval, bring it to market.
The results of the Phase 1/2 clinical trial of lumasiran were presented in Italy this year at the OxalEurope, European Hyperoxaluria Consortium. Three cohorts comprised of a total of twelve patients took part in a single-blind, placebo-controlled study. Another eight patients were given the drug as an expansion of the first two cohorts. In total, twenty patients took part. It was found that in the first three cohorts, those given lumasiran showed a reduced level of urinary oxalate (on average a 64% reduction). These results were also found to be long-lasting. Pritesh Gandhi, PharmD. Vice President and General Manager of the Lumasiran program, says,
“We are pleased to present data that signal hope to patients with PH1.”
