SouthShore

Isis Pharmaceuticals' Obesity Buster an Enormous Finding

Re: Bioinvestor. Actually, big pharma invests in most promising new platforms in a rather unique way - in a press release, with a transfer of money, rights to regional distribution, and in exchange for a percentage of the company they're investing in. So they do share in the profits of the successful ventures, and they also lose their investment in those platforms that are not viable. Even the big pharma's were taken during dot.com bubble.

As you correctly said, upstart biotechs must partner away early products in order to build a revenue stream that will power growth of their future pipeline. Most fail. Also, AMGN and DNA are now struggling as they've picked most of the low hanging fruit.

A non-oral form of Mipomersen will discourage its wide spread use, but if the formulation can be produced in an extended release form or packaged in a pen-style injection similar to AMLN's Byetta, then this might change.
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Specifically regarding Mipomersen, a reversal of plaque would be a significant benefit and would provide ISIS with an expanded use. Prevention of cardiac disease surgeries would be a significant savings to the HMO system (not to mention the impact on patients and families).
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Generally regarding ISIS, they have a potential platform that applies a new drug type to old and new disease targets. The old targets are the most promising since these are already validated, have probably come off patent, and are abundant. Plus, ISIS will not have to spend much money or time on early-phase discovery or legal. Targeting PTP-1b with small molecules for instance was tried to death by every pharma and is the poster child for each new drug platform. The platform for ISIS is the bull case for investing here. Mipomersen is the proof-of-principle product.

Toxic side effects, drug-delivery issues, competition, and production costs are the bear points.

Long-term, ISIS is a speculative buy - to me. IMO.