Letter from Ron Bartek re press release

Hello all,

We know we do not want to put all our research eggs in any one basket -
we want as many really promising eggs as we can incubate in as many
baskets (FA disease mechanisms) as we can identify.

The disease baskets we are carrying right now include:
1. The mitochondrial dysfunction basket - the shortage of frataxin
protein in FA patients leads to their mitochondria producing far less
energy and far more oxidative stress that kills their cells. The eggs
we have been incubating in this basket include CoQ10, Idebenone, A0001
and EPI-743,as well as Pioglitazone and Resveratrol, each of which has
been aimed at improving mitochondrial function in cells with reduced
frataxin protein. The CoQ10 and Idebenone eggs have not hatched
successfully. We continue to support advancement of the other
mitochondrial agents in hopes that they will hatch by showing in
"pivotal" (phase 2B/phase 3) trials that they are safe and capable of
increasing energy and reducing oxidative damage in our patients so they
can be approved as treatments for FA. The FDA has awarded both A0001
and EPI-743 "orphan drug designation" which means that the FDA judged
that:

a. FA is an orphan disease (fewer than 200,000 U.S. patients), and
b. the applications for both A0001 and EPI-743 presented sufficient
evidence/data that the drug holds potential for benefitting FA patients.

Orphan drug designation also carries significant incentives for the drug
company. For example, if an application for drug approval is submitted
to the FDA following the pivotal trial, the review of the application is
expedited (the review might take six months rather than 18 months). If
the drug is approved, the company would receive seven years of
exclusivity in the market for that drug for treatment of that
disease. The company would also be given tax breaks amounting to up to
50 percent of the funds it expended in developing the drug.

The FDA's approval of additional "Expanded Access" for the treatment of
"seriously ill" patients with mitochondrial disorders refers to the
emergency treatment of about forty patients so far with various late- or
final-stage conditions (two with FA, the others seriously ill
with different mitochondrial dysfunction disorders). Today's
announcement indicates that some additional patients that are seriously
ill with such late- or final-stage conditions will be eligible for such
emergency treatment with EPI-743. FARA is seeking guidance as to how,
when and where such treatments are to be managed.

2. The other disease mechanism baskets we are all carrying together
right now include the efforts to increase the frataxin protein levels
available to FA patients (HDAC inhibitor, EPO, CEPO, TAT-Frataxin,
gene-replacement, etc.), and the efforts to reduce the amount of toxic
iron in the mitochondria (for example, the iron chelator Deferiprone).

With everybody's continued support and participation in the clinical
research, FARA's plan is to keep filling the pipeline with promising
drug discoveries and to keep pushing the clinical development of those
drugs until we all achieve the treatments and cure to which the FA
family is committed.

Hope that helps,
Ron

Ronald J. Bartek
President
Friedreich's Ataxia Research Alliance (FARA)
P. O. Box 1537
Springfield, VA 22151
Tel (703) 426-1576
FARA website: http://www.CureFA.org
Email: fara@CureFA.org
Please register in the FARA Patient Registry at
http://www.curefa.org/registry/ and for e-news at
http://visitor.constantcontact.com/email.jsp?m=1101190303489