Genzyme's MS Therapy on the Fast Track: Competition Heats Up

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by: EP Vantage

The competitive landscape for current first-line treatments for MS, particularly beta interferons, looks increasingly inhospitable. Following last week’s FDA advisory committee recommendation to approve Novartis’ (NYSE:NVS) Gilenia in early use and potentially the first oral MS drug (Gilenia clears major regulatory hurdle with surprising ease, June 11, 2010), the threat posed by a launch in 2012 of Campath in the MS market was highlighted again Monday with FDA fast track status for the antibody which Genzyme (GENZ) acquired from Bayer (OTCPK:BAYRY) last year.

While Gilenia demonstrated superior efficacy over Biogen Idec’s (NASDAQ:BIIB) Avonex, Campath is being tested directly against another beta interferon, Merck KGaA (OTCPK:MKGAY) and Pfizer’s (NYSE:PFE) Rebif, with results from two phase III trials expected in the second half of 2011. Although Genzyme did not pay anything upfront for global rights to Campath in MS, the troubled biotech clearly sees huge potential in MS with potential payments to Bayer reaching $1.2bn.

Big deal

Already marketed since 2001 as a monotherapy treatment for B-cell chronic lymphocytic leukaemia (B-CLL), Genzyme expanded its long-standing deal with Bayer over Campath to include rights in MS, as well as rights to two relatively small cancer drugs, Fludara and Leukine.

The complex deal for all three products, including various buy-out clauses, actually totals a staggering $2.8bn which could be realized over the next ten years. The lack of an upfront payment at the time means the deal was largely over-looked in terms of major licensing transactions last year but in terms of bio-dollars it would rank as the biggest in 2009 (AstraZeneca tops the product deal charts in 2009, February 16, 2010).

Genzyme’s ensuing manufacturing woes then largely buried this deal as an important event last year, so Monday’s news throws the spotlight back on to the potential of Campath in MS. Many analysts at the time claimed the drug had blockbuster sales potential in this indication, especially if it manages to gain approval as a first-line therapy.


Both pivotal trials involve two-year treatment with either Campath or Rebif. Campath will be administered as an annual dose cycle, once a day intravenous injection for five consecutive days at the start and then once a day for three consecutive days after 12 months, while Rebif will be injected subcutaneously three times a week for 2 years.

The first trial, CARE-MS I, will be as a first line therapy in 580 patients and is expected to complete in May 2011. The second, CARE-MS II, will treat 840 MS patients who have relapsed while on either beta interferon or Teva’s (NASDAQ:TEVA) Copaxone, with results due by the end of 2011.

Phase II data so far has been very encouraging, although concerns about autoimmune adverse events, including immune thrombocytopenic purpura and thyroid effects, could ultimately restrict use of the drug to a second or third line setting, negating somewhat the clear dosing advantage over beta interferons.

Nevertheless, Tysabri achieved blockbuster global sales last year despite being used as a second or third line drug and highly publicized PML safety concerns.

So with fast track status in the bag, meaning the results from pivotal trials can be filed as they emerge and a high chance of receiving a six-month priority review as well, Campath could yet become another important new MS therapy and a silver lining perhaps to the black clouds currently hanging over Genzyme.