Seeking Alpha: Alnylam Strengthens Its Clinical Case Once Again

I can understand why many investors may be skeptical of Alnylam Pharmaceuticals (NASDAQ:ALNY). With a $10 billion market cap (as of last night's close), I believe this is the most highly-valued biotech without an actual approved drug. That creates considerable downside risk when (and I do believe it's "when", not "if") a clinical disappointment occurs.

The thing is, though, that Alnylam continues to make its case stronger and stronger. Not only has the company been very active in filling out a robust clinical pipeline, but the incremental data from those drugs has also been quite strong. Alnylam's latest announcement, positive 12-month data from an open-label extension study of patisiran, only serves to highlight that this biotech may in fact be developing a collection of medicines that deliver clear and significant benefits in a variety of undertreated conditions.

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Alnylam Strengthens Its Clinical Case Once Again

Seeking Alpha: Alnylam Pharmaceuticals Has Plenty On Its Plate

Investor sentiment, not to mention cash, has been draining out of the biotech sector since the spring of this year, leading to unimpressive performance for leading RNAi companies Alnylam Pharmaceuticals (NASDAQ:ALNY) and Isis Pharmaceuticals (NASDAQ:ISIS). I won't try to argue that valuations didn't get overheated when news broke that Alnylam and Sanofi (NYSE:SNY) agreed to a large-scale partnership, but the correction since then has gone too far. Interest (and potential competition) in RNAi is picking up again, but I believe Alnylam has more than enough in its own pipeline to remain an attractive story for some time to come.

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Alnylam Pharmaceuticals Has Plenty On Its Plate

Seeking Alpha: PTC Therapeutics Looks Like A High-Risk Play On Rare Diseases

With money and momentum flowing out of the biotech sector, there are going to be a lot of beaten-down diamonds in the rough amidst the wreckage. I'm not entirely convinced PTC Therapeutics (PTCT) is a diamond, or at least not yet. While the company's lead compound ataluren has a lot of promise, there some serious questions and concerns about the drug. Bulls are right about the high-end potential here, but prior trial failures shouldn't be ignored.

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PTC Therapeutics Looks Like A High-Risk Play On Rare Diseases

Seeking Alpha: In A Shaky Biotech Market, Alnylam's Prospects Are Getting Stronger

Biotech investing is not where you go if you want an easy investing life, but the profits of patience and good stock selection can be significant. Investors are clearly nervous about biotech now, and there is a threat that sector-wide selling in these notoriously fickle stocks will put them in hibernation until the cycle turns around again.

Specific to Alnylam (ALNY), though, I would argue the company has never been stronger. The company is on pace to exceed its target of having five compounds in human studies by the end of 2015 ("5 x 15"), and a recent deal with Sanofi (SNY) gives Alnylam development funding, a motivated commercial partner, and the perception that it has been vetted by a large pharmaceutical company with a significant presence in rare diseases.

Alnylam is absolutely a risky pick, and investors should not ignore the risk that not only may the company's drugs fail in the clinic or in the marketplace, but that investors indiscriminately bailing out of the sector could weigh on the share price at times. On the flip side, I cannot ignore that Alnylam has multiple potential billion dollar-plus drugs in the clinic and a very strong R&D position in what could prove to be one of the next major therapeutic alternatives. With that, I see almost 50% upside in Alnylam shares today.

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In A Shaky Biotech Market, Alnylam's Prospects Are Getting Stronger

Seeking Alpha: Silence Therapeutics A Quieter Name In RNAi

By now most biotech investors know about RNAi companies like Alnylam Pharmaceuticals (ALNY) and Isis Pharmaceuticals (ISIS), and many probably know Tekmira (TKMR) and Arrowhead Research (ARWR) as well. Alnylam and Isis have excited investors with the potential multiple billion-dollar drugs in their pipeline, while Tekmira and Arrowhead have solid delivery technologies and emerging therapeutic pipelines.

If you'll pardon the obvious pun, it has been a lot quieter where London-based Silence Therapeutics (OTCPK:SLNCF) (SLN.L) is concerned. This small biotech is barely followed on the Street and isn't exceedingly liquid even on its home stock exchange (the LSE). Silence's corporate history is shakier than many others and the company does not have a particularly thick book of clinical data in humans, but what the company does have is a three-pronged delivery platform that could, like Arrowhead's delivery technology, open up a wider range of treatment targets for RNAi therapy.

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Silence Therapeutics A Quieter Name In RNAi

The Motley Fool: Alnylam Offers an Exciting Rare Disease Pipeline, but Expectations Are High

"Biotechs are risky" is a standard boilerplate warning when writing about the sector, but some situations are riskier than others. In the case of Alnylam Pharmaceuticals (NASDAQ: ALNY  ) , the sell-side seems so eager to make a bullish case that uncommonly high approval odds are already being applied to the company's early stage pipeline.

There is also still the prospect of competition from Isis Pharmaceuticals (NASDAQ: ISIS  ) and its partner GlaxoSmithKline (NYSE: GSK  ) , as well as other large players like Sanofi (NYSE: SNY  ) , Novo Nordisk (NYSE: NVO  ) , and Baxter (NYSE: BAX  ) . Against that is the possibility that Alnylam is following a path that could closely resemble that of other rare disease biotechs like Genzyme, Alexion (NASDAQ: ALXN  ) , or BioMarin (NASDAQ: BMRN  ) .

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Alnylam Offers an Exciting Rare Disease Pipeline, but Expectations Are High

Seeking Alpha: Tekmira's Transition Worth Watching

RNA interference (RNAi) went through a painful stretch of doubt a few years back, but two of the sector's leaders, Alnylam (ALNY) and Isis Pharmaceuticals (ISIS) have come roaring back on the strength of both solid clinical data and a "risk on" bullish biotech market. Tekmira (TKMR), too, has come back into its own as a settlement with Alnylam has cleared away some legal and IP uncertainties and the company repositions itself as a product-focused biotech.

It's difficult to call any RNAi biotech cheap at this point, and I can't help but note that a great deal of valuation is being pinned to early-stage clinical programs, which history shows is often a very dangerous move. Even so, if investors consider what Tekmira could be in line to receive in the form of royalties and from its own pipeline, this shares could still offer some relative value.

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Tekmira's Transition Worth Watching

Seeking Alpha: Alnylam Far From Primetime, But Still An Intriguing Prospect

Here at the midpoint, 2012 is looking like a strong year for biotech and some risky names have done quite well. Although not coming close to the gains seen in Arena Pharmaceuticals (ARNA), Amylin Pharmaceuticals (AMLN), or Lexicon Pharmaceuticals (LXRX), RNA interference specialist Alnylam Pharmaceuticals (ALNY) has come on strong with encouraging early-stage data and a greater risk tolerance on the part of investors.

All of the standard warnings apply to Alnylam, as the company is indeed many years from potential product approvals and none of the company's programs are in advanced trials. Upping the risk even more is the fact that RNA interference is still an unproven approach and many Big Pharma companies appear to have backed away from the technology. All that said, this company has a broad clinical program, numerous high-value targets, deep IP, and a healthy balance sheet.

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Alnylam Far From Primetime, But Still An Intriguing Prospect

Seeking Alpha: Sanofi May Just Have It All

Although I don't own it, I'm wondering if Sanofi (SNY) may just be the model of what a Big Pharma ought to look like. The company is exceptionally well-balanced geographically (with roughly 30% exposure to the U.S., EU, and emerging markets) and has a complimentary array of businesses that includes generics, vaccines, OTC, and branded drugs. Sanofi also strikes a solid balance between drug types (biologics, etc.) and pipeline risk/reward.

While Sanofi still has some patent expirations to digest, this company actually has one of the better growth profiles in the space right now. It also happens to look meaningfully undervalued.

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Sanofi May Just Have It All

Seeking Alpha: Alnylam Looks Nearly Left For Dead

By no means is it easy to try to launch an entirely new class of pharmaceuticals, but that is essentially what Alnylam Pharmaceuticals (ALNY) has set out to do. Despite some encouraging (albeit very early) signs of success, investors have very much taken note of Big Pharma's increasing skepticism towards what this company is trying to do. Consequently, the stock trades for little more than the cash on its books despite a promising pipeline that is only getting started in human studies.

The Long March
Investors who have high hopes for Alnylam's RNA interference (RNAi) technology may want to take a page from the history books. Although Amgen (AMGN), Biogen Idec (BIIB), Johnson & Johnson (JNJ), and Roche (RHHBY.PK) have all had success with recombinant protein and monoclonal antibody drugs, the reality is that the science underpinning these drugs really started early in the 20th century and it took more than a decade for promising (and Nobel Prize-winning) research in the 1970's to actually result in approvable drugs.

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Alnylam Looks Nearly Left For Dead

Investopedia: XOMA Rearranges The Deck Chairs One More Time

XOMA (Nasdaq:XOMA) really is a remarkable story in many ways. Few companies have survived so long, and raised capital so many times, and done so little with it. While the company has actually approached $100 million in reported sales, and not many biotechs do even that much, the company has never had any real success in the clinic and has basically strung along successive generations of investors on hype and hope. Now that the company is changing up management yet again, investors should be asking themselves if there is really a good reason to stick by a company that has floundered around for more than 20 years to no real purpose.


A Change at the Top
XOMA announced Wednesday that it's CEO Steve Engle was bailing on the company. Engle said, "I am looking forward to applying my expertise building life science companies in the future."

Hmmm, expertise? Engle became XOMA's CEO in August of 2007. In the those four years, XOMA has gone from a market cap north of $350 million to a current market cap of just under $64 million, and the company really is not a great deal closer to having a winning product in its portfolio.


Read the complete article at the link below:
http://stocks.investopedia.com/stock-analysis/2011/XOMA-Rearranges-The-Deck-Chairs-One-More-Time-XOMA-REGN-PFE-ABT-ISIS-VICL-CELG0901.aspx

Investopedia; Amarin's Magic Pill

Sometimes good ideas are just there staring scientists in the face. A great deal of energy has been spent finding drugs that can lower cholesterol and triglycerides and counteract the coronary side-effects of the typical American lifestyle. All the while, part of the answer may have just been swimming around our oceans.

Data released by Amarin (Nasdaq:AMRN) on Monday morning indicates strong triglyceride-lowering potential for its purified EPA omega-3 pill, and a potential blockbuster for this small company.

Strong Data Should Drive Approval 

Amarin's results in a Phase 3 study of AMR101 were quite good. The study showed that a daily dose of four or two grams reduced triglyceride levels by 21.5% and 10.1%, respectively. Arguably even more important, though, was that the data showed no meaningful increase in LDL levels (the so-called "bad cholesterol"). (For more, see Measuring The Medicine Makers.)

Here's why that matters: GlaxoSmithKline (NYSE:GSK) has done quite well with its Lovaza pill, a mixture of DHA and EPA ethyl ester omega-3 that also lowers triglyceride levels. While Lovaza is quite efficacious in lowering triglycerides (and maybe better than AMR101), it does lead to higher LDL levels. That combination is worrisome for some patients, and could give AMR101 a real shot at blockbuster status. (For more, see Pharmaceutical Phenoms: America's Best-Selling Medicines.)

To read the full piece, please click below:
http://stocks.investopedia.com/stock-analysis/2011/Amarins-Magic-Pill-AMRN-GSK-PFE-AZN-ISIS-SNY0419.aspx

Investopedia: A Deal At Last For Sanofi And Genzyme

Ultimately it looks like two major drug companies are getting what they both think they need. After months of posturing, Sanofi-Aventis (NYSE:SNY) and Genzyme (Nasdaq:GENZ) found common ground on the value and structure of a deal, and Genzyme will become part of Sanofi. Though this deal was long in the making, only time will tell whether shareholders on both sides of the deal really benefit. 

The Deal

Sanofi-Aventis agreed to pay $74 a share in cash up front for Genzyme, a price that on its own virtually matches the all-time high set back in 2008. At that price, Sanofi is paying over four-times trailing revenue and over 26-times trailing EBITDA - a pretty generous premium compared to larger biotechs like Amgen (Nasdaq:AMGN) and Gilead (Nasdaq:GILD) as well as other growth names like Celgene (Nasdaq:CELG).

In response to charges of opportunism from Genzyme's management, Sanofi agreed to sweeten the pot with so-called contingent value rights (CVR). If Genzyme's business reaches certain milestones after the deal, Genzyme shareholders will get additional payments. There are six different hurdles laid out for Genzyme, worth up to $14 per share in total (or about $3.8 billion), but only the first three (production levels for Cerezyme, approval of Lemtrada and Lemtrada sales in excess of $400 million in certain territories) seem highly likely to be reached. If those three are reached, it will cost Sanofi about $4 per Genzyme share, while the remaining hurdles are all tied to ever-higher levels of sales. 

Please find the full piece here:
http://stocks.investopedia.com/stock-analysis/2011/A-Deal-At-Last-For-Sanofi-And-Genzyme-SNY-GENZ-AMGN-GILD-CELG-SHPGY-ISIS0218.aspx

Note: I realize how out of date this is now. My apologies for that ... I submitted it Wednesday morning, but it got held up in the queue. 

Alnylam Takes Another Hit

The trouble with being a trailblazer is that whenever there are brambles or gopher holes along the way, you're the first to blunder into them. Alnylam (Nasdaq: ALNY) is unquestionably one of the pioneers of RNAi drug technology, but this is a technology that is far from ready for primetime. While RNAi might very well take its place alongside Amgen's (Nasdaq: AMGN) recombinant proteins or Roche's (Nasdaq: RHHBY) monoclonal antibodies and became a major therapy class, it may also go the way of antisense or gene therapy – a promising theoretical approach that produces little in the way of marketable drugs.

The latest blow to Alnylam comes from Roche's decision to cut costs by firing part of its research staff and curtailing some development programs, including RNAi research. Alnylam and Roche signed a development deal over three years ago, a deal that was originally advertised as a $1 billion alliance – assuming, of course, that preclinical programs developed to full fruition. 

Now, though, there is a real risk that those product milestones and royalties never come and the deal is instead just the $331 million upfront payment and 2M share investment.That would take out a sizable chunk of Alnylam's potential revenue over the next few years (development milestones) and substantially bump up the dilution that investors can expect (since the company will have to raise the money from the markets instead of getting its from Roche's development progress).

This follows Novartis's (NYSE: NVS) decision a little while ago to not expand its product development agreement with Alnylam and end its research collaboration. That, then, is two major partners and two globally-respected top-tier drug companies that have decided that they do not need to rush forward in RNAi development.

This is not the end of the line for Alnylam by any stretch. First, the company has an exceptional amount of cash on the balance sheet today. Moreover, Roche has not stated that they are abandoning RNAi research for good. While they are ending RNAi research in Germany and Wisconsin, those programs could be moved to other locations and resumed at a later date. Moreover, if Alnylam can get a few compounds into Phase 2 testing and show strong clinical efficacy, it would not be at all surprising to see companies try to jump back into the space.

Now, Alnylam's stock...

Clearly, I was way too eager to get into this name when I bought a few months ago. You would think I would have learned my lesson with Isis (Nasdaq: ISIS) quite a few years ago, but I look at Alnylam (and early stage biotech in general) as my substitute for gambling or playing the lottery. Relatively few of these plays will ever pay off, but the ones that do will pay off well and I find that these investments stimulate my intellectual curiosity and feed my inner life sciences geek.

Moreover, I still do actually believe that Alnylam can be a leader in RNAi technology and that RNAi-based treatments can be a major new category of pharmaceutical compounds in the next decade. The company has over $5 nearly $9 a share in cash on the balance sheet (or about $7.50 on a fully diluted basis). Granted, that cash is going to get whittled away by ongoing R&D demands, but paying a little more than 2x 1.25x cash for potentially blockbuster technology is not ridiculous from a valuation perspective.

Alnylam's stock is almost certain to take another beating today, and there's no obvious near-term catalyst. As with most biotechs, the odds are that any incremental news will be negative not positive. Still, for those with a strong stomach, a lot of patience, and a long-term horizon, this is one of the most interesting biotech names today. 

Disclosure: I own shares of Alnylam

Update: I mistakenly went with just the cash and short-term securities on the balance sheet in the original posting, excluding the securities categorized as long-term investment assets. I was in a rush and I apologize for the sloppy error. ss