Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY)

The near-term hopes of Alnylam (and RNA interference in general) to be taken seriously as part of the future armentarium against infectious disease were dealt a severe blow today when the company reported failure of ALN-RSV01 to significantly reduce the incidence of bronchiolitis in RSV-infected patients in a phase IIb trial. The stock seems to have escaped serious punishment today by applying generous amounts of lipstick to their press release pig, but I believe this development will drain market cap from the company as institutional investors edge quietly towards the exits. If this was an early proof-of-concept trial, the "near-miss" angle might hold water but it doesn't really make the grade for a phase IIb study. I suspect that Alnylam will choose not to expend further resources on phase III development of ALN-RSV01, and that eventual announcement will punch the hole in their sail that they averted today.

RNAi therapeutics for transthyretin-mediated amyloidosis remain in very early development and anti-neoplastic RNAi compound ALN-VSP won't be advanced into phase II without a partner. ALN-VSP has had an empty dance card for nine months so far. I've profited before from volatility in Alnylam, but that was at a lower price entry point with more cash in the coffers. This time round, I'm seeing a lot more tunnel before any light appears.

Lots of buying by several insiders.

RNA interference is a very interesting and potentially powerful tool for gene therapy. It has proven very difficult to exploit; thus, all the major pharmaceutical companies have given up on pursuing development of RNAi based therapies -in house. ALNY is very good at keeping its "runway" long and healthy so it has been able to focus on developing this technology for some time. It manages to generate income from the "Majors" who do not want to be left out when RNAi technology is finally ready. This leaves ALNY as the nexus of RNAi technology. I expect that RNAi can be made to work and that ALNY will be the company that does it. As long as ALNY survives it has enormous potential; and yes I have some understanding of the phenomenon.

Company just cut ~25% of staff. Never in the history of biotechs has this been a good omen.

There are currently 16900 ALNY shares in my "fund" with break-even of around 9.37 USD.
http://caps.fool.com/Blogs/fund-trades/678775.

On December 23, 2010 portefeuille (99.94) wrote:

“Roche has elected to make a number of broad-based portfolio decisions across their entire R&D organization that appear to affect their ability for continued and sustained resource commitment to RNAi therapeutics,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “While we are disappointed and surprised to hear of their portfolio decision given the progress being made in the RNAi field, we remain more confident than ever in our efforts to advance RNAi therapeutics as a whole new class of innovative medicines to patients. In particular, we have achieved major breakthroughs in the safe and effective delivery of RNAi therapeutics and have also advanced our RNAi therapeutic efforts to patients in several clinical programs. We expect our delivery progress and clinical pipeline to continue to grow. Moreover, we believe we are on the frontier of demonstrating human proof-of-concept for these and other programs in the near term, and we are confident that these data will provide a strong foundation for continued execution on our business model and value creation strategy.”

http://finance.yahoo.com/news/Alnylam-Comments-on-Recent-bw-4192054651.html?x=0&.v=1

Alnylam has done an amazing job at keeping RNAi hyped as a legitimate technology, but the jig seems to be nearly up. Deal flow has been declining for a few years, marquis partners are starting to throw in the towel, and anyone who has ever worked with this technology in a lab is wondering how the hell anyone was able to get this far with something that is dirty even in a dish and can't be delivered.

RNAi has been dumped by the major drug companies. I also personally think that RNA-interference is not a feasible drug technology in humans, and was just a bunch of early 2004-2006 buzz. Already not as important in biology labs as was predicted to be in 2004.

Priced at near cash on hand, with bright prospects in the coming years.

Alnylam Pharmaceuticals is developing a new class of medicine called RNA Interface. The company is considered the leader in the technology, has an excellent R&D pipeline with clinical trials emerging, and an excellent balance sheet. As of Dec'10, the company's stock is valued at less than 1/3 of its 2008 value. The stock price depressed after news of one of its large partners pulling the plug on RNAi technology. The stock is very well priced because of investor fear and has excellent opportunity for growth.

Risky, but considering how much it's fallen, it's worth the risk.

If they ever make this happen, and there is no assurance that it will be done, RNAi can potentially end disease as we know it.

why not

I don't know much about biotechnology. However, this firm has some products in the pipeline, and they're already generating revenue through licensing deals. So this is speculative but not so much as most smaller biotech stocks, which are IMO just lottery tickets.

Have been watching Alnylam for a few years and am throwing it in the caps portfolio. The risk is very high with technology like this but the price seems right and think it will out perform in the mid term.

RNAi will transform medicine. With over 600 million in cash-very little premium is baked into this cake

Low debt/equity (0.02) enables price compeititve products (01/03/2009)

Great tech, great partnerships, plenty of cash on hand for the short term.

MAJOR PATENTS IN RNA INTERFERENCE WITH STRONG PARTNERSHIP WITH MULTIPLE MAJOR PHARMACEUTICAL COMPANIES.