FDA Approves Clinical Trial of TDI-132 (Gilenya™) in ALS Patients

      FDA Approves Clinical Trial of TDI-132 (Gilenya™) in ALS Patients

PR Newswire

CAMBRIDGE, Mass., Feb. 11, 2013

CAMBRIDGE, Mass., Feb. 11, 2013 /PRNewswire-USNewswire/ --The ALS Therapy
Development Institute (ALS TDI) announced today that it has received FDA
approval to conduct a clinical trial of TDI-132 (aka: fingolimod, Gilenya™) in
ALS. Fingolimod is currently being marketed by Novartis AG as Gilenya™ as a
treatment for some forms of multiple sclerosis.

This clinical trial is being launched as a Phase IIA with the primary purpose
of determining the safety and tolerability of TDI-132/Gilenya in people with
ALS. ALS TDI, a non-profit biotech, is the sole funding sponsor of this
clinical trial, thanks to the support it received from the ALS community.
Current enrollment sites include Massachusetts General Hospital in Boston;
University of California, Irvine in Orange; Georgia Health Sciences University
in Augusta; and Methodist Neurological Institute in Houston, TX. More
information can be found online here: http://www.als.net/TDI-132.

"It was exciting to see how expeditious the FDA reviewed our application to
test Gilenya in ALS patients," comments Steve Perrin, Ph.D., CEO and CSO of
ALS TDI. "We are eager to start enrolling patients in the clinical trial of
TDI-132 and take this important step toward understanding whether or not
Gilenya is a potential treatment for ALS."

"Seeing TDI-132 enter into clinical trial for ALS gives me hope that people
living with ALS may soon be able to fight back," says Augie Nieto, an ALS
patient and chairman of the board at ALS TDI. Nieto, through his involvement
as head of the Muscular Dystrophy Association's "Augie's Quest," has
contributed greatly in funding the research leading to the advancement of this
clinical trial.

ALS TDI researchers first began preclinical testing of TDI-132 in 2011 for its
ability to block certain immune cells from entering the brain and spinal cord
where they can cause activities that result in damage to motor neurons. The
Institute has confirmed that TDI-132 significantly reduces the circulation of
these immune cells through the bloodstream, resulting in fewer of them
infiltrating into the central nervous system. Further experiments at ALS TDI
showed treatment with TDI-132 resulted in positive outcome based on several
disease measures in preclinical studies. In February 2012, the Institute
officially announced TDI-132 as a clinical candidate.

ALS TDI worked with the Northeast ALS Consortium (NEALS) on the clinical trial
design. NEALS will oversee the execution of the clinical trial.

For more information about TDI-132 (fingolimod/Gilenya) and the clinical
trial, please visit: http://www.als.net/TDI-132.

A formal listing of the clinical trial can be found here:

An FAQ about the Phase IIA clinical trial can be found here:

Information about why TDI-132 (fingolimod/Gilenya) might be helpful for ALS
can be found here:

About ALS

Amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease) is a progressive
neurodegenerative disease that leads to paralysis, due to the death of motor
neurons in the spinal cord and brain. There is no known cure for the disease.
About 5,000 people in the US are diagnosed with ALS each year; the incidence
is similar to multiple sclerosis. However, with no effective treatment, the
average patient survives only 2-5 years following diagnosis. There are about
30,000 people in the US diagnosed with ALS today. The worldwide population of
ALS patients is estimated at 450,000.

About the ALS Therapy Development Institute (ALS TDI)

The mission of the ALS Therapy Development Institute (ALS TDI) is to develop
effective therapeutics that slow or stop amyotrophic lateral sclerosis (ALS,
Lou Gehrig's disease) as soon as possible for patients today. Focused on
meeting this urgent unmet medical need, ALS TDI executes a robust target
discovery program, while simultaneously operating the world's largest efforts
to preclinically validate potential therapeutics; including a pipeline of
dozens of small molecules, protein biologics, gene therapies and cell-based
constructs. The world's first nonprofit biotech institute, ALS TDI has
developed an industrial-scale platform, employs 30 professional scientists and
evaluates dozens of potential therapeutics each year. ALS TDI collaborates
with leaders in both academia and industry to accelerate ALS therapeutic
development, including Biogen Idec, UCB, Aestus Therapeutics, MDA and RGK
Foundation. ALS TDI is a 501(c)3 registered charitable nonprofit organization.
For more information, please visit us online at www.als.net.

Media Contact:
Mari Sullivan, ALS TDI, msullivan@als.net, 617-441-7220

SOURCE ALS Therapy Development Institute

Website: http://www.als.net/
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