Genzyme is upbeat on Myozyme data

02 May 2005 00:01  [Source: ICB Americas]

Genzyme’s corner on the niche market for drugs to treat lysosomal storage disorders (LSD) appears to be strengthening. Last week, the company unveiled promising new data for Myozyme (alglucosidase alfa), a treatment for a rare muscle disease, solidifying analysts’ expectations of a late-2005 launch for the drug.

Myozyme is an enzyme replacement therapy for Pompe disease, which is caused by deficiency in acid alpha-glucosidase and results in weakness of the respiratory muscles. Though fewer than 10,000 people worldwide are afflicted with the inherited disease, the need for a treatment is critical—those with infantile-onset generally die by 12 months of age, and the life span of late-onset patients is usually shortened because of respiratory failure.

Interim data from a pivotal clinical trial, known as AGLU01602, in patients with infantile-onset Pompe disease, showed that Myozyme was effective at reversing cardiomyopathy, or enlargement of the heart, and improved patients’ motor development.

The trial also appears to be on track to meet its primary endpoint, or the number of patients that have survived and do not need to be on invasive ventilator support at 18 months of age. One year into the trial, 89 percent of the patients treated with Myozyme were free of ventilator support, compared to 17 percent of untreated patients alive at that stage, according to historical data.

“Most importantly from a commercial standpoint in our view, 1602 showed gains in cognitive skills in all Myozyme-treated patients, providing increased potential that Myozyme may ultimately confer a more normal life along with increased survival,” said R.W. Baird analyst Christopher Raymond in a research note last week.

Genzyme says it is on track to file for US regulatory approval mid-year, leaving analysts confident the drug will gain approval in late 2005 or early 2006. European regulatory agencies are already reviewing the drug, with approval expected late this year. R.W. Baird expects the drug to bring in $8 million this year and $59 million in 2006.

“All in, we are very encouraged by the status of the Myozyme program thus far, and in particular the clinical data we have seen from the drug’s pivotal program,” added Raymond.

The positive data for Myozyme builds on Genzyme’s growing cache of drugs to treat LSD. Though the patient population for these rare diseases is limited, Genzyme enjoys a tidy cash flow from the drugs.

The company’s recently released first-quarter results came in ahead of analyst expectations, driven in part by the performance of the LSD drugs Cerezyme (imiglucerase) and Fabrazyme (agalsidase beta). Sales of Fabrazyme soared 84 percent to $70 million in the first quarter, while Cerezyme brought in $220 million in the quarter, up 11 percent.

Overall sales at Genzyme increased 28 percent in the first quarter to $630 million. Underlying net income came in at $131 million, compared to $86 million in the first quarter of 2004.

Based on strong first-quarter sales, the company raised its full-year EPS guidance to $2.12 to $2.18 from its previous estimate of $2.08 to $2.06.

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