Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) recently published the data collected from its ILLUMINATE-B phase 3 clinical trial evaluating an investigative therapy called lumasiran.
Lumasiran is an RNAi-based therapy designed to target the gene encoding glycolate oxidase known as hydroxyacid oxidase 1 (HAO1). The therapy has been in development as a potential treatment for children and adult patients suffering from primary hyperoxaluria type 1 (PH1). Alnylam presented the ILLUMINATE-B phase 3 clinical trial results at the American Society of Nephrology (ASN) Kidney conference held from October 22 to October 25. ASN conferences are held annually but this year’s conference was held virtually due to the coronavirus pandemic.
“We are delighted to present these positive data from ILLUMINATE-B that reinforce previously reported clinical study findings for lumasiran,” stated Pritesh J. Gandhi, general manager and vice president of Alnylam’s Lumasiran Program.
Gandhi also noted that the findings highlight lumasiran’s potential to become a vital treatment option for PH1 patients especially since there are currently no other approved treatment options. Its approval will thus be a welcome relief for PH1 patients since the disease is potentially fatal. If it receives the marketing approval, Alnylam will market lumasiran under the trademark OXLUMO.
Understanding the mechanism behind PH1 and how lumasiran works
PH1 occurs when oxalate is overproduced by the liver, thus leading to morbidity, as well as mortality. Dr. Jeffrey M. Saland stated that the research findings from the clinical trials indicate that urinary oxalate levels correlate with the clinical results in patients that have PH1. He also excitedly announced that the study findings show that there was significant decline in urinary oxalate levels. Dr. Saland expressed excitement about the published findings because they represent hope for patients living with PH1.
The data presented at the ASN conference features nephrocalcinosis, exploratory renal stone, and long-term treatment data. Dr. Saland believes that the findings from the clinical studies are a good indicator of the benefits that it can potentially deliver to PH1 patients. The findings revealed that the pipeline treatment had a good safety profile with no adverse effects or deaths reported. There was also discontinuation of any subjects enrolled in the study.