Genzyme Corp. (GENZ) recently announced that it will be publishing two-year follow-up data on its Gaucher disease candidate, eliglustat tartrate, in the journal Blood. 52-week phase II data on the candidate, presented last year, showed that the candidate met its primary endpoint. The two year follow-up data showed continued improvement across all endpoints.
Eliglustat tartrate is currently in two phase III studies. While one study, ENCORE, is comparing eliglustat tartrate to Cerezyme in previously treated patients, the second study, ENGAGE, is evaluating the candidate in patients who are yet to receive enzyme replacement therapy within 9 months or substrate reduction therapy within 6 months of participation in the study.
A third study, EDGE, was initiated recently. This study will compare a once-daily dosing regimen of eliglustat tartrate with a twice-daily dosing regimen of the candidate.
Genzyme is looking to expand its Gaucher disease franchise through the successful development of eliglustat tartrate. Eliglustat tartrate could have an advantage over existing treatments as it is taken orally. Currently available treatments for Gaucher disease require intravenous infusion. Cerezyme, Genzyme’s lead product, is the market leader in the Gaucher disease market.
Unfortunately, Genzyme has been plagued by manufacturing issues which cropped up in 2009 and led to a reduction in supply of Cerezyme. We remain concerned that a further delay in resuming full supply of Cerezyme could lead to loss of share to Shire plc’s (SHPGY) Vpriv and Protalix BioTherapeutics Inc.’s (PLX) Uplyso. While Vpriv was launched earlier this year, Uplyso may receive approval in Feb 2011. Both products were made available under the US Food and Drug Administration’s (FDA) expanded access program while Cerezyme supply was affected.
Neutral on Genzyme
We currently have a Neutral recommendation on Genzyme, which is supported by a Zacks #3 Rank (short-term “Hold” rating). We are pleased to see that Genzyme is taking steps to emerge from the impact of the temporary shutdown of its Allston manufacturing plant.
We view the recent approval of Lumizyme as a major positive and are pleased to see the company working on expanding its product portfolio and pipeline so as to reduce its dependence on a handful of products for growth. The company has also been in the news lately with rumors of a possible takeover by Sanofi-Aventis (SNY).
GENZYME-GENERAL (GENZ): Free Stock Analysis Report
PROTALIX BIOTHR (PLX): Free Stock Analysis Report
SHIRE PLC-ADR (SHPGY): Free Stock Analysis Report
SANOFI-AVENTIS (SNY): Free Stock Analysis Report
Zacks Investment Research
