Rodman & Renshaw Maintains Outperform on Alnylam Pharmaceuticals after Phase I ALB-TTR02 Data

Rodman & Renshaw has published a research report on Alnylam Pharmaceuticals ALNY after the company reported Phase I data for ALN-TTR02 that showed robust knockdown of the transthyretin (TTR) gene. In the report, Rodman & Renshaw writes, "Recall, this trial and the previous Phase I trial of ALN-TTR01 evaluated the effects of escalating doses of the respective drugs on TTR protein levels based on a single administration of drug. The earlier Phase I trial of ALN-TTR01 demonstrated a 41% mean knockdown in serum TTR levels at nadir at a dose of 1.0mg/kg. ALN-TTR02 utilizes a proprietary second-generation lipid nanoparticle technology, based on the “MC3” lipid, which has shown increased potency of the siRNA. As an example, making use of the MC3 lipid nanoparticle, ALN-TTR02, at doses of 0.30mg/kg, led to 87% knockdown in TTR levels at nadir, and a durable 67% knockdown at day 28. It is important to note that a 50% reduction in TTR levels is thought to result in disease improvement or stabilization. Further, two of the three volunteers treated in the 0.30mg/kg cohort had TTR levels that were below measurable limits by assay, again highlighted the potency of the drug. Results by cohort are described in further detail in Figure 1 and Figure 2. These results could support dosing once every four weeks, or potentially, as indicated by management, once every six to eight weeks." Rodman & Renshaw maintained its Market Outperform rating and $19 price target on Alnylam Pharmaceuticals, which is currently trading up around 47 percent from Friday's $12.51 closing price.