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Fly News Breaks for May 7, 2019
May 7, 2019 | 05:22 EDT
Piper Jaffray analyst Edward Tenthoff reiterates an Overweight rating on Alnylam Pharmaceuticals (ALNY) with a $142 price target after surveying 16 physicians treating 490 hereditary transthyretin amyloidosis patients with polyneuropathy. Doctors have a "clear preference" for Alnylam's Onpattro over Onpattro and Ionis Pharmaceuticals' (IONS) Tegsedi, with efficacy, safety, and experience being cited, Tenthoff tells investors in a research note. Roughly two-thirds of existing and new gene silencer patients are prescribed Onpattro, says the analyst. Based on the survey results, the analyst is increasingly confident in his 2019 Onpattro sales estimates of $127M.
News For ALNY;IONS From the Last 2 Days
Jun 17, 2019 | 07:14 EDT
Alnylam announced that it has achieved full patient enrollment in its ILLUMINATE-A Phase 3 study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase for the treatment of adults and children with primary hyperoxaluria type 1, or PH1. The study enrolled patients across 16 sites in eight countries. Alnylam is on track to report topline results from ILLUMINATE-A expected in late 2019 and, if positive, to submit filings for global regulatory approvals starting in early 2020. The company also announced complete results from its Phase 1/2 clinical study and reiterated positive results from its ongoing Phase 2 open-label extension study of lumasiran. Results will be presented at the 2019 Oxalosis & Hyperoxaluria international workshop. In final Phase 1/2 study results, lumasiran demonstrated a mean maximal reduction in urinary oxalate of 75% relative to baseline across all cohorts. At 28 days post the last dose, the mean reduction relative to baseline was 66%. All patients achieved oxalate lowering to less than 1.5 times upper limit of normal. Among patients receiving 3 mg/kg monthly or quarterly doses of lumasiran, 92% achieved urinary oxalate levels within the normal range. Furthermore, lumasiran-treated patients across all cohorts experienced a mean maximal decrease of 77% in the ratio of urinary oxalate to creatinine. Lumasiran results showed an acceptable safety and tolerability profile, with PH1 patients on study for a median of 9.8 months, there were no study discontinuations. Serious adverse events, or SAEs, were reported for one patient receiving placebo and four patients receiving lumasiran; none were related to study drug. The placebo patient experienced acute pyelonephritis and kidney stones. The lumasiran patients with SAEs included one patient with vomiting, one patient with abdominal pain, fever and vomiting, one patient with gastroenteritis and one patient with kidney stones. Adverse events, or AEs, were reported in two patients during placebo dosing and 20 patients after lumasiran dosing. The majority of AEs were mild or moderate in severity and were assessed as unrelated to study drug. Severe AEs were reported in one patient during placebo dosing and one patient after lumasiran dosing; none were considered related to study drug by investigator. AEs reported in more than three patients receiving lumasiran were pyrexia; vomiting, cough, abdominal pain, headache and rhinitis and nephrolithiasis. Self-limiting injection site reactions were reported in three patients receiving lumasiran; all mild or moderate and with none affecting dosing. Lumasiran was not associated with any clinically significant adverse laboratory findings, including liver function tests.