Background The $11 billion market cap, ALNY is an undisputed leader in the field of ribonucleic acid interference (RNAi) therapeutics. The company has built a strong product pipeline around both construction and delivery technology using liver-targeted approach. The company's current clinical programs, particularly FAP and FAC, are seen as extremely promising with high probability of success. It also has strong development and commercialization partnership with Genzyme (owned by Sanofi) in orphan drug for rare genetic diseases. The company has ridiculously long pipeline, many of which are seen as significant blockbuster drugs in the future.
Catalyst For the purposes of this trade, I am specifically following ALN-PCSsc (hypercholesteremia) Phase 1 data scheduled to be presented at the European Society of Cardiology (ESC) between August 29 - September 2. This will be a potential significant catalyst for the stock because based on my research this compound is of significant interest to analyst community as well as medical professionals. The compound works by degrading mRNA inside the cell, thus preventing the production of protein, thus lowering LDLR receptor, thus lowering levels of bad cholesterol. Two reasons why this compound is unique:
1) possibility of only one per month or even one per quarter drug usage with minimal adverse events (which the FDA and medical professionals are going to love)
2) the compound is partnered with The Medicines (MDCO) which has already showed with 32 patient study about 70% PCSK9 protein reduction and 40% reduction in LDL (bad cholesterol) vs. placebo. Nonhuman primates actually showed 96% knockdown in protein and up to 77% reduction in LDL-C. It is this point what I am after with September calls. I think at the ESC Meeting in Aug 29 - Sep 2 the company could demonstrate fantastic data.
Lastly, if Amgen (AMGN) was smart enough, who is developing its own PCSK9 drug with less promising future, they would acquire ALNY right now while the market cap is still only $11 billion. That's just my opinion.