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

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

February 11, 2013 – CAMBRIDGE, MA – 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.

A formal listing of the clinical trial can be found here: http://www.als.net/ALS-Research/166/ClinicalTrials/

An FAQ about the Phase IIA clinical trial can be found here: http://www.als.net/docs/uploads/General_Information_about_ALS_TDI_Phase_IIA_Clinical_Trial_of_TDI-132_Gilenya_FAQs.pdf

Information about why TDI-132 (fingolimod/Gilenya) might be helpful for ALS can be found here: http://www.als.net/docs/uploads/Why_might_Gilenya_be_helpful_for_ALS.pdf

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, [email protected], 617-441-7220

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