Biotech Values

[DewDiligence]

GENZ Reports 5-Year Data for Campath in MS

[These data come from a phase-2 study of Campath vs Rebif. 4-year data from the same study were reported in Apr 2010 (#msg-48971681).]

http://www.reuters.com/article/idCNN1324222020101014

›Thu Oct 14, 2010 9:30am EDT
By Toni Clarke

BOSTON, Oct 14 (Reuters) - Genzyme Corp presented new data from a trial of its experimental multiple sclerosis drug alemtuzumab on Thursday that continued to show strong efficacy with no new or worsening side effects.

Genzyme believes the drug, if approved, could be the most effective available and gain a significant portion of a market the company estimates will be worth roughly $13 billion by the end of this year.

Alemtuzumab, also known as Campath, is a key pawn in the battle between Genzyme and French drugmaker Sanofi-Aventis, which has made a hostile, $18.5 billion bid for the company… Sanofi Chief Executive Chris Viehbacher has tentatively forecast peak annual sales of the drug at $700 million, a figure he said is in line with market expectations, based on forecasts from seven brokerages.

But independent market research group BioMedTracker predicts sales more than double that, at $1.6 billion in 2019. And some analysts predict peak sales closer to $2 billion.

Five-year follow-up data from a mid-stage trial of the drug, presented at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) on Thursday, showed that 87 percent of patients experienced no worsening of disability.

By comparison, only 62 percent of patients taking Rebif, a rival drug made by EMD Serono, an affiliate of Germany's Merck KGaA and Pfizer Inc, experienced no worsening of symptoms. The alemtuzumab group, moreover, on average saw an improvement in disability scores as opposed to a worsening among those on Rebif.

Patients taking Campath had an annualized relapse rate of 0.11, compared with a relapse rate of 0.35 for Rebif, which indicates that on average, approximately one in every 10 patients who received alemtuzumab had a relapse in any given year, compared with about one in every three patients taking Rebif.

"The most remarkable thing about this data is that it hasn't changed much from the four-year data," said Michael Panzara, who oversees clinical development of Genzyme's multiple sclerosis and immune system drugs. "This is unique and starts us thinking, are these people in remission? Is this what remission in MS looks like?"

Four-year data released in April showed that 71 percent of patients had no relapse or worsening of disability. The company did not present a comparison to that combined figure, saying it will do so at the American Academy of Neurology conference next April.

For investors, the key questions revolve around the drug's safety.

Initial three-year results, reported in 2008, showed that six patients developed immune thrombocytopenic purpura (ITP), an autoimmune disease in which low blood platelet levels can lead to bruising and bleeding. One patient in the trial died of a cerebral hemorrhage. The remainder were diagnosed more quickly and successfully treated. Since then, no new cases of the condition have been reported.

Another potential side effect is Goodpasture syndrome, a rare disease that can quickly cause kidney failure. In the initial clinical trial, one patient developed the condition. That patient was treated and has recovered normal kidney function, Panzara said.

Two other patients who were given the drug outside the confines of a clinical trial also developed the syndrome. Campath is currently approved to treat cancer, but physicians are allowed to prescribe it for conditions for which it has not been approved on a so-called "off-label" basis. No additional cases of the condition were reported in the latest data.

Panzara said it is unclear why no new cases been observed, and said the company is still working to fully understand the drug's mechanism of action. But one theory is that the drug resets the body's immune system in some fundamental way, allowing it to function more normally even when the drug is no longer being taken.

Genzyme is co-developing the drug in multiple sclerosis with German drugmaker Bayer. Late-stage, or Phase III, data, will be presented around the middle of next year.‹