Alnylam Pharmaceuticals: Phase 1/2 ALN-CC5 Trial Underway


This morning Alnylam Pharma (NASDAQ:ALNY) announced that it initiated a Phase 1/2 clinical trial for ALN-CC5 for complement-mediated diseases. Initial data is expected in mid-15 with additional data in late 2015. ALN-CC5 is a SC-administered, ESC-GalNAc–conjugated, siRNA therapeutic that targets complement component C5. Complement-mediated diseases represent a $6B market opportunity by analyst estimates. If approved, ALN-CC5 would directly compete with Alexion’s (NASDAQ:ALXN) Soliris, which currently generates greater than $2B in revenues annually.

The Phase 1/2 trial will be conducted in normal human volunteers, and in patients with PNH (paroxysmal nocturnal hemoglobinuria). The trial is being conducted in the UK in three parts. Parts A and B will be randomized (3:1, drug:placebo), doubleblind, placebo-controlled, single-dose (Part A) and multi-dose (Part B), dose-escalation studies, enrolling up to a total of 60 adult volunteers. Patients will receive fixed SC doses administered once per week for up to five weeks. The primary endpoints are safety and tolerability. Secondary endpoints include knockdown levels of serum C5 and inhibition of C5 complement activity. Additionally, Part C will be an open-label multidose study in up to eight patients with PNH with administration of multiple doses up to 13 weeks. Primary endpoints of Part C include safety, tolerability, and clinical activity, as well as levels of LDH, a measure of red blood cell hemolysis.

Shares of Alnylam closed yesterday at $93.83 . ALNY has a 1-year high of $111.49 and a 1-year low of $47.03. The stock’s 50-day moving average is $96.61 and its 200-day moving average is $83.07.

On the ratings front, Alnylam has been the subject of a number of recent research reports. In a report issued on February 2, Piper Jaffray analyst Edward Tenthoff maintained a Buy rating on ALNY, with a price target of $155, which reflects a potential upside of 65.19% from last closing price. Separately, on December 30, Goldman Sachs’ Terence Flynn maintained a hold rating on the stock.