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At Long Last, A Win Against Lupus: HGSI Succeeds in Phase III

Lupus is like the Black Knight in Monty Python and the Holy Grail: it's been telling drug developers for decades that "none shall pass" -- and when they insist on trying to cross that Phase III bridge, well, then they must die.

Until this week, when Human Genome Sciences came along on lobbed the Black Knight's arms off--or cut his kidneys out--whatever, it was more than a flesh wound.

The best part of the story is that no one thought HGSI would succeed. OK, maybe that's the second-best part. The best part would have to be that lupus patients, who have to endure the toxic side effects of chemotherapy and high-dose steroids, may finally have a new option after half a century of disappointments.

There was plenty of reason to think HGSI's Benlysta (belimumab, formerly LymphoStat-B) would disappoint as well. Lupus is a complex autoimmune disease that manifests itself through a variety of symptoms that wax and wane in severity. It's tough to diagnose and tougher to treat. Failures have included Genelabs Technologies' Prestara (prasterone), Aspreva Pharmaceuticals' CellCept (oral mycophenolate mofetil), Biogen Idec and Roche/Genentech's Rituxan (rituximab), and La Jolla Pharmaceutical Co.'s Riquent (abetimus sodium) on numerous occasions.

In fact Benlysta itself failed its Phase II trial. But a subgroup of patients experienced a statistically significant improvement in lupus signs and symptoms, so HGSI focused on that subgroup and that endpoint in Phase III.

It was still a risky retrospective subset analysis, and most folks didn't think it would work, as evidenced by the fact that HGSI's stock was under a buck earlier this year and trading around $3 before the data on Monday.

But despite the long odds, HGSI's Phase III trial succeeded. Benlysta met its primary endpoint, achieving a patient response rate of 57.6 percent for drug versus 43.6 for placebo (p=0.0006). The stock rocketed up 277 percent.

It's not the end of the road: a second Phase III trial will read out in November. But it's the farthest anyone has gotten yet â€" and that in itself is an achievement.

Monty Python photo by FanPop