We’ve listened to most of the webcasts this morning and most were eh-eh, so-so. Isis was good, 2009 will be a pivotal year for them for sure. Amicus is one of our personal favorites. On a fundamental basis, Amicus has solid molecules for great unmet needs (lysosomal storage diseases), and made good regulatory progress in 08. Here’s the lowdown in quicknotes – sorry for any typos :

Amigal (migalastat hydrochloride) for the treatment of Fabry Disease

*FDA said it supports a Phase 3 clinical trial comparing Amigal to placebo based on a surrogate primary endpoint (EP) of the change in the amount of kidney GL-3, the substrate that accumulates in the cells of Fabry patients

*SPA agreed with FDA, need to determine how EP will be measured (i.e. assay)

*additional study under EMEA vs. ERT

Plicera(TM) (isofagomine tartrate) for the treatment of Gaucher Disease

*Phase 2 clinical trial of Plicera in Gaucher patients is ongoing

*6 month study to evaluate safety as well as demonstrate trends of efficacy as measured by the standard endpoints in Gaucher disease

*target enrollment of 16 patients

*results expected 3Q09

AT2220 (1-deoxynojirimycin HCl) for the treatment of Pompe Disease

*Phase 2 clinical trial of AT2220 in adult Pompe patients ongoing

*11 week treatment period with an optional extension study

*measure safety and pharmacodynamics of multiple doses and regimens

*results expected 1Q09

Financial Health

*expects to spend about $70M on 2009 cash operating expenses (to be offset by $50M in program cost-sharing reimbursements and clinical milestone payments from partner Shire)

*$100M cash on hand by end 2009

Read more here.

Image from cambridgesoft

Related Posts