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Biotech Stock Mailbag: Keryx, Sangamo, Threshold and Amarin

Stocks in this article: KERX SGMO THLD AMRN AEZS CELG

Sangamo shares are up about 60% since the start of the year, not quite a double but still very strong. I saw CEO Ed Lamphier present at the J.P. Morgan Healthcare Conference earlier this month. He tells a compelling investment story about Sangamo's research into "zinc-finger" proteins -- customized drugs that can turn genes on or off in order to treat and cure disease.

Zinc-finger proteins work in plants and in the lab. Sangamo has yet to prove the technology works effectively enough in people. A first stab at a therapy designed to regrow nerves in patients with diabetic neuropathy was unsuccessful. Sangamo has now turned its attention to SB-278. The therapy is designed to genetically alter a patient's T cells to eliminate a protein known as CCR5 used by HIV to invade and infect cells. Without CCR5, cells are much more resistant to HIV infection. Sangamo refers to SB-278 as a potential "functional cure" for HIV because if it works, HIV-infected people might be able to halt treatment with anti-retroviral drugs like those marketed by Gilead Sciences. For newly infected people, treatment with SB-278 could delay or prevent the need for HIV therapy.

Cool stuff. Sangamo has presented some interesting, early data on SB-278 but nothing that demonstrates definitive proof. What we've seen so far from SB-278-treated patients is short-term increases in CD4+ T-cell counts, but is that enough to justify stopping or delaying anti-retroviral therapy? Sangamo's challenge is complicated by the potency of existing HIV drugs, which are capable of reducing viral loads to undetectable levels.

Sangamo is conducting phase II studies of SB-278 with interim results expected in the first half of the year and full results before the end of the year. Anticipation for these important data is probably what's driving investor interest in the stock right now.

There's a possibility that Sangamo snags a late-breaker presentation slot at the 2013 Conference on Retroviruses and Opportunistic Infections (CROI), scheduled for March 3-6. This is the most important HIV research meeting of the year, so presentation of SB-278 phase II data would be a big deal for Sangamo. Expect to hear something about a CROI presentation (or not) at the end of January.

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