Friday, February 5, 2010

Repligen and HDAC-3

This is potentially very very exciting
This came from Pam  of FAPG

I listened to Repligen's 3rd quarter financial report this morning and
transcribed what they said about the HDAC inhibitors. Here is what was
said:

"We are also developing inhibitors of HDAC-3 for the treatment of
Friedreich's Ataxia. We have completed the evaluation of one of our
lead compounds in a 2 species toxicology study which is required by the
FDA prior to the initiation of clinical trials. Manufacturing of
clinical drug supply is in progress and we expect to file an IND to
initiate Phase I clinical trials by mid-2010. These studies are being
partially supported by the MDA which provided us with a second research
grant in December. Our clinical goal is to cross over from normal
volunteer studies to Friedreich's patients as soon as possible to enable
us to get an early read on the potential efficacy of our drug by
monitoring changes in the blood levels of frataxin, the protein missing
in Friedreich's patients. Recent data suggests that this biomarker is
expressed at a consistent level in patients which will facilitate the
observation of a potential increase in frataxin levels following drug
treatment. We believe that there are 15,000 Friedreich's Ataxia
patients worldwide which represents a market opportunity of more than
$300,000,000 even with very modest pricing assumptions."

This is very exciting news!
Pam

No comments: