Press Release from FARA (US)

This is regarding the HDAC inhibitor we have been watching so
closely....
WONDERFUL

FOR IMMEDIATE RELEASE
New Clinical Trial in Friedreich's Ataxia in Italy

FARA, January 5, 2012 -- The Italian Health Ministry and the Ethics
Committee of the San Luigi Hospital in Torino, Italy, have given
investigators at the hospital approval to initiate a Phase I clinical
trial of a new drug designed specifically to treat Friedreich's ataxia.
After additional study site preparations, this Phase I trial will test
the Repligen drug, known as RG2833, in patients with Friedreich's
ataxia. The investigators will aim to find out whether the drug is safe,
and to learn more about its effects in Friedreich's ataxia patients. In
particular, they will determine if RG2833 increases production of
frataxin, a key protein that is diminished in people with Friedreich's
ataxia. Because lower frataxin levels are the base cause of Friedreich's
ataxia symptoms, it is hoped that, if RG2833 acts to increase frataxin
production, it could be beneficial to Friedreich's ataxia patients.
RG2833 is a type of drug called a Histone Deacetylase (HDAC) inhibitor
and is being developed by Repligen working with an international team of
scientists. HDAC inhibitors may provide a way to increase frataxin
protein production by "switching on" the frataxin gene, which is too
often "switched off" in people with Friedreich's ataxia. RG2833 has been
awarded orphan drug designation by the U.S. Food and Drug Administration
and the EMA. The development of HDAC inhibitors for Friedreich's ataxia
has been supported by FARA and other patient advocacy groups including
the Muscular Dystrophy Association and GoFAR. Repligen has indicated
that when the final preparatory steps are complete and the first patient
is enrolled in the Italian trial, more details will be made available on
the status of this study and global plans for the drug's development.
About FA
FA is a rare, degenerative, life-shortening neuro-muscular disorder that
affects children
and adults and involves the loss of strength and coordination usually
leading to wheelchair use; diminished vision, hearing and speech;
scoliosis (curvature of the spine); increased risk of diabetes, and a
life-threatening heart condition. There are currently no effective
treatments. About FARA The Friedreich's Ataxia Research Alliance (FARA)
is a 501(c)(3), non-profit, charitable organization dedicated to
accelerating research leading to treatments and a cure for Friedreich's
ataxia. www.CureFA.org Contact: Jennifer Farmer Executive Director,
Friedreich's Ataxia Research Alliance (484) 879 6160 info@curefa.org