Analysis: Street betting on Amicus' drugs

WASHINGTON, July 11 (UPI) -- Wall Street is betting on Amicus' candidates for treating lysosomal storage disorders, with analysts saying the oral compounds could provide a significant advantage over Genzyme's $1.5 billion infused enzyme replacement therapies.

Amicus, which first went public in May, is developing three compounds known as oral chaperones that are intended to counteract the problems in lysosomal storage disorders, which include damage to the heart, kidneys and other organs.

Amicus' therapies are designed to bind to misfolded enzymes and transport them into the lysosome where they can break down proteins. Due to a genetic mutation in people with lysosomal storage disorders, the enzymes don't function properly and fail to break down proteins, which then accumulate and lead to organ damage.

"We like the company," Pacific Growth Equities analyst Greg Wade told United Press International. "It's got an interesting technology that would appear to have broad utility in lysosomal storage disorders and potentially other diseases."

Wade noted the lysosomal storage disorders market alone could be worth more than $1 billion in sales for Amicus' products.

However, the company's most advanced compound -- Amigal for Fabry disease -- is in phase 2 with data expected later this year, so it's difficult to predict with any certainty how the candidates will fare as they get further down the clinical pipeline.

Wade said the phase 2 data anticipated for the end of the year will give investors a better indication of Amigal's potential, but so far, preclinical and early clinical work has suggested the agents may be effective in treating these diseases.

"It remains to be seen whether or not in actual people this results in changes that are deemed meaningful," he said. But he noted the company has "done excellent research and science and have an excellent chance of success."

Wade currently rates the stock a buy.

Lazard Capital Markets analyst Matthew Osborne also thinks Amicus' oral chaperones show great potential.

"It's certainly a game-changing technology for the treatment of lysosomal storage disorders," Osborne told UPI.

"There's a lot ahead for the company in terms of what they need to prove clinically, but they've shown they have the proof of concept down," he added.

Osborne said the phase 2 data for Amigal should help give a better indication of the potential of the drug, but he thinks their chances of success are lower for this indication.

"We think there's more risk for the chaperone technology in Fabry disease, but they've shown early evidence of clinical activity so far," he said.

The best opportunity for Amicus, in Osborne's view, resides in Gaucher disease, and phase 2 data is expected to be released in the second half of next year for Plicera in that indication. In addition, Amicus is expected to release phase 1 data for another compound -- AT2220 in Pompe disease -- in the fourth quarter of this year.

By 2014, Osborne projects Plicera could generate $400 million in sales, with Amigal coming in at $100 million.

If the drugs do pan out, it could mean a hit for Genzyme and their monopoly on treating lysosomal storage disorders.

Genzyme's enzyme replacement therapies -- which include Cerezyme for Gaucher disease, Fabrazyme for Fabry disease and Myozyme for Pompe disease -- dominate the market and generated $1.5 billion in worldwide sales last year.

Osborne said Genzyme is developing its own oral drug for Gaucher disease that could help it retain its hold on the market. Final phase 2 results are anticipated to be released in the middle of 2008, so they're roughly at the same stage of development as Amicus.

Shire is also developing a drug for treating lysosomal storage disorders, but it's an infused agent, so it would probably pale in comparison to Amicus' or Genzyme's oral agents.