Replies to post #61430 on Biotech Values

[ghmm]

Amicus had said they were doing some preclinical work looking at the possibility of administering their chaperone with ERT (I don't recall which of the three but think either in Fabry or Gaucher). While it would open up a very interesting opportunity this doesn't make a lot of sense to me as wouldn't this imply the replaced enzyme also has the mutation?


The Shire time line may not have changed my comment was from when TKT was a standalone company and they were looking at possible commericialization in 2008 here is a PR after their Phase I/II.
http://phx.corporate-ir.net/phoenix.zhtml?c=95926&p=irol-newsArticle&ID=698247&highlight
I think initially they had hoped to get approval based on just that one trial (Cerezyme may have needed just 1 trial to go from Ceredase)

[corpstrat]

Amicus - Shire: Thank you for jogging the memory. It's interesting strategy to invest in a quasi-rival product, though, as you say, perhaps they see it a s product that can work for pts for whom ERT does not work. I hope they don't plan to smother Plicera, a cynical strategy that is not inconceivavble in this game.

Genzyme's price umbrella is outrageously high, making for an attractive market for Shire with its own candidate and Plicera?

[genisi]

I never had much faith in their chaperone technology:

Amicus Therapeutics Announces Preliminary Results of Phase 2 Study with Plicera(TM) for Gaucher Disease

http://finance.yahoo.com/news/Amicus-Therapeutics-Announces-prnews-1330118971.html?x=0&.v=1

CRANBURY, N.J., Oct. 2 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD - News) today announced preliminary results from its Phase 2 randomized, open-label study to assess the safety, tolerability and preliminary efficacy of its investigational drug, Plicera(TM) (afegostat tartrate), in treatment-naive adult patients with type 1 Gaucher disease. Two dose regimens of Plicera (225 mg three days on/four days off and seven days on/seven days off) were studied during this six month trial. While all patients enrolled experienced an increase in the level of the target enzyme (GCase) as measured in white blood cells, clinically meaningful improvements in key measures of disease were observed in just one of the eighteen patients who completed the study. The preliminary results suggest that treatment with Plicera was generally well tolerated, with no serious adverse events (SAEs) reported. Nineteen subjects were enrolled and 18 subjects completed the study. One subject discontinued treatment because of an adverse event (conjunctivitis-related symptoms).

Once the data are final, the Company plans to further analyze and evaluate the results in collaboration with its partner, Shire Human Genetic Therapies, Inc. (Shire HGT), and, based on this work, will determine the appropriate next steps for the Plicera program. However, based on these preliminary results, the Company does not expect to advance Plicera into Phase 3 development at this time.

John F. Crowley, President and CEO of Amicus, stated, "The preliminary results of this Phase 2 study certainly do not meet our expectations, but we believe they do provide additional insights into the biological activity of Plicera in Gaucher disease and the pharmacological chaperone technology platform. We plan a detailed analysis to ensure we have a complete understanding of the data."

The Company is also today announcing a change to its financial guidance for 2009. Based on its current projections of net operating expense, the Company now expects to end 2009 with approximately $70-$80 million in cash.

Conference Call and Webcast

Amicus will host a conference call to discuss the Plicera Phase 2 preliminary results, today, October 2, 2009, at 5 p.m. EDT.
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