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It's dog-and-pony show week for prominent biotech and pharmaceutical companies. Isis Pharmaceuticals (NASDAQ: IONS ) joined the crowd of presenters at two investor conferences this week -- the Morgan Stanley Global Healthcare Conference, and the Stifel Nicolaus Wiesel Healthcare Conference. Here are three key takeaways from what Isis had to say.
1. Biogen Idec (NASDAQ: BIIB ) relationship
As you might expect, one of the top topics of discussion was related to the recently announced deal with Biogen Idec to use Isis' antisense technology in developing drugs to treat neurological diseases. Members of Isis' management team emphasized the strategic nature of this fourth collaboration with Biogen in the past two years. And, of course, they highlighted the nice financial aspects of the deal -- $100 million upfront, with a potential for over $3 billion if all goes well.
Isis officials particularly focused on its current collaboration with Biogen for spinal muscular atrophy, or SMA, drug ISIS-SMN(RX). Two mid-stage trials are under way for the drug, with initial results expected later this year, or possibly in early 2014.
If the findings are positive, the antisense drug could ultimately prove to be a winner for Isis and Biogen. There are currently no approved treatments for SMA, which, although rare, is still the leading genetic cause of infant deaths.
2. Triglyceride trials trucking along
A lot of attention was also given to Isis' ISIS-APOCIII(RX) drug, which targets treatment of very high triglyceride levels. In August, Isis announced positive preliminary results from a mid-stage study that showed the drug lowered triglyceride levels by up to 75% in patients with very high, and severely high, triglycerides. Results from another phase 2 study were announced the previous month that showed reduction of triglyceride levels by up to 64% in patients with high triglyceride levels.
Management didn't provide many more details than were announced with the clinical results. However, when asked about the company's strategy going forward at the Morgan Stanley conference, Isis Chief Operating Officer Lynne Parshall said that the plans were to move forward with a phase 3 study targeting severely high triglyceride levels above 880 mg/dL. She indicated that the company could also conduct larger outcomes studies for patients with high, but not as severe, triglyceride levels in the future.
Isis seems headed toward a possible duel with Amarin (NASDAQ: AMRN ) down the road. Amarin gained U.S. Food and Drug Administration approval last year for fish-oil drug Vascepa in treating patients with severe triglyceride levels above 500 mg/dL. FDA approval was granted based on clinical studies that showed Vascepa lowered triglyceride levels by 33%.
3. One of its least-appreciated drugs
Wade Walke, Isis VP of Corporate Communications and Investor Relations, briefly mentioned the company's familial amyloid polyneuropathy drug, ISIS-TTR(RX), which is being developed with GlaxoSmithKlein (NYSE: GSK ) , during his comments at the Stifel Nicolaus Weisel conference. COO Lynne Parshall talked about the drug as well -- but only when asked about it.
Parshall described ISIS-TTR(RX as one of the company's "least appreciated assets." Isis advanced the drug directly to phase 3 after solid results were obtained from a phase 1 study. Glaxo has an option to license the drug after the findings from the phase 3 study are known. According to Parshall, Isis will move forward with another phase 2 study of ISIS-TTR(RX) in treating a related indication, familial amyloid cardiomyopathy, or FAC.
Not enough time
We don't get to use the word "cornucopia" a lot, but that's exactly what Isis has with its pipeline -- an overflowing abundance of drugs in development. There are actually too many for the company to fully discuss at any of these investor conferences.
For example, while Isis officials talked a little about a phase 3 study under way for Kynamro, no one brought up its other drug in phase 3 studies. Teva Pharmaceuticals (NYSE: TEVA ) licensed cancer drug custirsen in 2009. The Israel-based drugmaker, along with OncoGeneX, are conducting late-stage trials of custirsen targeting treatment of prostate cancer and non-small cell lung cancer. And there are quite a few other pipeline drugs not as far along in development that the Isis representatives didn't address.
Isis looks to be one stock that investors should keep on their radar screens, even if they don't get to attend the Morgan Stanley and Stifel Nicolaus Wiesel shindigs. The growing company puts on a good dog-and-pony show at those investment conferences, but the stock is certainly no dog, and Isis absolutely isn't a one-trick pony.
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