Alnylam Initiates ILLUMINATE-C Phase 3 Study of Lumasiran for the Treatment of Advanced Primary Hyperoxaluria Type 1 and Presents New Positive Results from Phase 2 Open-Label Extension Study
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Nov. 9, 2019 -- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the Company has initiated ILLUMINATE-C, a new global Phase 3 study of lumasiran, an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1). The study will enroll patients of all ages with advanced renal disease, and the primary study endpoint is the percent reduction in plasma oxalate from baseline to six months. Alnylam expects to report initial ILLUMINATE-C results in late 2020.
The Company also announced new positive efficacy results from the ongoing Phase 2 open-label extension (OLE) study of lumasiran, which were presented at the American Society of Nephrology (ASN) 2019 Annual Meeting on Saturday, November 9 in Washington, DC.
“We are pleased to announce the start of the ILLUMINATE-C trial designed to assess the safety and efficacy of lumasiran in a PH1 patient population with advanced renal disease, including patients of all ages and those on dialysis. This study complements our comprehensive clinical development plan for lumasiran, led by our ILLUMINATE-A pivotal study with results expected later this year and our ILLUMINATE-B study in young pediatric patients. Given the heterogeneity of the PH1 population, the ILLUMINATE trials collectively address PH1 patients across the spectrum of age and disease onset and severity,” said Pritesh J. Gandhi, PharmD, Vice President and General Manager, Lumasiran program at Alnylam. “We are also pleased to report new results from our Phase 2 OLE study, and are encouraged by the sustained reductions in urinary oxalate and by the overall safety profile of lumasiran observed to date.”
The Phase 2 OLE results were reported as of the data cut-off date of September 12, 2019 and demonstrated a 76 percent mean maximal reduction (range: 43-91 percent) in urinary oxalate excretion relative to Phase 1/2 baseline values in all cohorts (N=19)*. In the study, all patients achieved a urinary oxalate level at or below 1.5 times the upper limit of normal (less than or equal to 0.69 mmoL/24hr/1.73m2), and 68 percent of patients achieved a urinary oxalate level within the normal range (less than or equal to 0.46 mmol/24hr/1.73m2). Patients also experienced an 82 percent mean maximal reduction in urinary oxalate:creatinine ratio (range: 62-94 percent) after lumasiran dosing across all cohorts (N=20).
The Phase 2 OLE safety results were based on a median study duration of 10.4 months (range: 7-17 months) since the first dose administered in the OLE study. As of the data cut-off date, there were no discontinuations from treatment. A single patient (1/20; 5 percent) reported two serious adverse events (SAEs) of traumatic brain injury and bone contusion sustained in a car accident; neither was assessed as related to study drug. There were no other reported SAEs in the OLE study. Adverse events (AEs) were reported in 19/20 (95 percent) patients; most were mild in severity and assessed as unrelated to study drug by the investigators. Injection site reactions, which were reported in 4/20 (20 percent) patients, were mild and did not affect dosing. Other AEs reported in more than one patient were: headache, oropharyngeal pain (N=3); gastroenteritis, viral gastroenteritis, pyrexia, and vomiting (N=2). There were no clinically significant laboratory changes.
To view the results presented by Alnylam at ASN 2019 Annual Meeting, please visit www.alnylam.com/capella.
*Patients who had a valid 24-hour urinary oxalate assessment. （Article from : www.drugs.com)