Alnylam Initiates Phase 2 Study of Lumasiran in Patients with Recurrent Kidney Stone Disease
- Category: DNA RNA and Cells
- Published on Tuesday, 21 December 2021 10:24
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– Phase 2 Study will Evaluate the Safety and Efficacy of Lumasiran in Patients with Recurrent Kidney Stone Disease and Elevated Urinary Oxalate Levels –
CAMBRIDGE, MA, USA I December 20, 2021 IAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the company has initiated a global Phase 2 study to evaluate the safety and efficacy of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in patients with recurrent kidney stone disease and elevated urinary oxalate levels. Lumasiran is approved by the U.S. Food and Drug Administration (FDA) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients and by the European Medicines Agency (EMA) for the treatment of PH1 in all age groups, in each region under the brand name OXLUMO®.
“Recurrent kidney stone disease is a prevalent disease, associated with significant clinical burden, including pain, infection, hospitalizations, and an increased risk of developing chronic kidney disease and kidney failure. The majority of kidney stones in adults are formed from calcium oxalate crystals and are accompanied by elevated urinary oxalate,” said Jeroen Valkenburg, General Manager, Lumasiran program at Alnylam. “To that end, we believe the reductions in urinary oxalate due to silencing of hepatic HAO1 we see with lumasiran in patients with primary hyperoxaluria type 1 may potentially apply to other diseases characterized by oxalate overproduction, such as recurrent kidney stone disease, where despite standard of care, stones continue to recur. We look forward to testing this hypothesis in this Phase 2 study.”
About the Phase 2 Study in Patients with Recurrent Kidney Stone Disease
Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of advanced primary hyperoxaluria type 1 (PH1) and recurrent kidney stone disease. HAO1 encodes glycolate oxidase (GO). Thus, by silencing HAO1 and depleting the GO enzyme, lumasiran inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1 and recurrent kidney stone disease. Lumasiran utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. Lumasiran has received regulatory approvals from the U.S. Food and Drug Administration (FDA) for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients and from the European Medicines Agency (EMA) for the treatment of PH1 in all age groups under the brand name OXLUMO®.
OXLUMO® (lumasiran) INDICATION AND IMPORTANT SAFETY INFORMATION
OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
For additional information about OXLUMO, please see the full U.S. Prescribing Information.
About Recurrent Kidney Stone Disease
It is estimated that one in eleven people will experience a kidney stone at some point in their lifetime. Approximately 80 percent of kidney stones in adults are formed from calcium oxalate crystals with up to 2.5 million Americans having recurrent calcium oxalate stone disease with elevated urinary oxalate. Kidney stones can cause painful urination and blood in the urine, flank pain, urinary tract infections, and can require hospitalizations and surgery for removal. They are associated with a greater risk of developing chronic kidney disease (CKD) and end stage kidney disease (ESKD). There are limited effective treatment options for recurrent kidney stone disease and despite best standard of care – including dietary and lifestyle changes, citrate supplementation, and thiazide diuretics – recurrent stones still occur.
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO® (lumasiran), as well as Leqvio® (inclisiran), which is being developed and commercialized by Alnylam’s partner Novartis. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam, on LinkedIn, or on Instagram.
SOURCE: Alnylam Pharmaceuticals