Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) announced new positive results from its phase I/II study on its experimental RNAi therapeutic candidate, lumasiran, which targets glycolate oxidase (GO). The data was presented at the OxalEurope, European Hyperoxaluria Consortium, in Naples, Italy.
The candidate is being evaluated for the treatment of Primary Hyperoxaluria Type 1 (PH1).
The data presented was from part B of the study with a cut-off date of Mar 29, 2018. The part B of the study is a randomized (3:1 drug:placebo), single-blind, placebo-controlled evaluation of lumasiran in patients with PH1.
While cohorts 1 and 2 received three monthly doses of lumasiran at 1 mg/kg or 3 mg/kg, respectively, cohort 3 received two quarterly doses at 3 mg/kg. The study also enrolled an additional eight patients who received open-label lumasiran in expansions of each of the first two cohorts, totaling 20 patients enrolled. In addition, patients randomized to the placebo group also received subsequent subcutaneous administration of lumasiran, following administration of placebo.
The results showed that lumasiran demonstrated a mean maximal reduction in urinary oxalate of 64% in patients enrolled in cohorts 1-3 (N=12), while all lumasiran-treated patients experienced a lowering in urinary oxalate below 0.7 mmol/24 hrs/1.73m2, a threshold level associated with a reduced rate of progression to end-stage renal disease.
Meanwhile, patients receiving lumasiran (N=9) maintained a mean reduction in urinary oxalate of 63% on day 85.
Consequently, Alnylam believes that the potent and durable reductions in urinary oxalate support a once quarterly, subcutaneous dose regimen.
The results also indicate that GO inhibition has the potential to reduce and possibly normalize levels of hepatic oxalate production, thus potentially halting PH1 disease progression.
The company continues to dose patients in Part B of the phase I/II study. Eligible patients are being transitioned into an open-label extension (OLE) study.
Alnylam expects to present additional data from all cohorts as well as from the OLE study in late 2018.
The candidate was generally well tolerated in all patients in the study. We remind investors that lumasiran has received Breakthrough Therapy Designation from the FDA and Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The candidate also enjoys the orphan drug status both in the United States and Europe.
Alnylam and the FDA have recently agreed on a pivotal study design for lumasiran including a primary endpoint at six months, based on reduction of urinary oxalate and a study size of approximately 25 patients with PH1. The company plans to initiate the phase III trial in mid-2018.
Alnylam’s share price has lost 21.8% in the last six months compared with a 6.8% decline for the industry.
Alnylam is developing novel therapeutics based on RNA interference. Alnylam filed its first new drug application (NDA) and marketing authorisation application (MAA) for lead candidate, patisiran.
The European Medicines Agency (EMA) has accepted the MAA and initiated its review. In February 2018, Alnylam announced that the FDA has accepted its NDA and granted priority review with an action date of Aug 11, 2018. A potential approval and successful commercialization will be a significant boost for the company, as it will begin generating product revenues in 2018.
Meanwhile, Alnylam has entered into several collaborations for the development and commercialization of its broad pipeline of RNAi therapeutic candidates. These include collaborations with Sanofi’s (SNY - Free Report) Genzyme and The Medicines Co. (MDCO - Free Report) among others. In March 2018, Alnylam announced a discovery collaboration with Regeneron (REGN - Free Report) for identification of RNAi therapeutics for nonalcoholic steatohepatitis (NASH), chronic liver disease and other related diseases.
Alnylam is a Zacks Rank #3 (Hold) stock. You can see the complete list of today’s Zacks #1 Rank stocks here.
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