The ALS Therapy Development Institute to Launch Phase II Clinical Trial of TDI 132 in ALS Patients

CAMBRIDGE, Mass., Feb. 14, 2012 /PRNewswire-USNewswire/ -- The ALS Therapy Development Institute (ALS TDI) will launch a Phase II clinical trial on TDI 132 (aka: fingolimod/Gilenya®) as a potential treatment for ALS (Lou Gehrig's disease).  Fingolimod is currently being marketed by Novartis AG as Gilenya® as a treatment for some forms of multiple sclerosis.

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"Getting to this point was only possible because ALS patients, families and the community stood up and took ownership over funding the search for potential treatments for ALS.  We are excited to announce this trial and will be working with the ALS community to ensure the funding is there to move as quickly as possible from starting enrollment to reporting results," said Steve Perrin, Ph.D., CEO & CSO of ALS TDI.  Perrin will make the official announcement during a company presentation as part of the 14th Annual BIO CEO Investor Conference taking place in New York City (www.bio.org) later this morning.

ALS TDI researchers first began preclinical testing TDI 132 in 2011 for its ability to block certain immune cells from entering the central nervous system (CNS) where they can cause activities that result in damage to motor neurons.  The Institute has confirmed that TDI 132 significantly alters the trafficking of these immune cells through the bloodstream, resulting in fewer of them infiltrating into and damaging the nervous system.  Further experiments at ALS TDI showed treatment with TDI 132 resulted in positive outcomes on several disease measures in preclinical studies in the SOD1 mouse model.  A key partner of ALS TDI, the Muscular Dystrophy Association, provided significant funding for some of that preclinical work, as did numerous other private donors to ALS TDI.

"We are encouraged by these early findings and look forward to the implementation of this clinical trial.  This work further exemplifies the effective outcomes from the long-term partnership between MDA and ALS TDI," said MDA Interim President and Medical Director Valerie Cwik, M.D.

The primary purpose of this Phase II clinical trial will be to determine the safety and tolerability of TDI 132 (fingolimod/Gilenya®) in people living with ALS today.  ALS TDI is a nonprofit biotech and is working with the Northeast ALS Consortium (NEALS) to assist with clinical trial design and to oversee the execution of the clinical trial.

"We are thrilled to work with ALS TDI on this important Phase II trial in ALS," said Merit Cudkowicz, M.D., Director of the Neurology Clinical Trials Unit at Massachusetts General Hospital, one of the principal investigators of the clinical trial and member of MDA's medical advisory committee. "We hope to learn from this study whether or not TDI 132 is safe to test in people living with ALS.  Participants will be evaluated over a month-long exposure to the drug, and we'll be monitoring their response closely." This initial study will provide important information on dosing and safety for a subsequent efficacy study.

Perrin and Cudkowicz will conduct an informational webinar for the ALS community on a date to be determined in March, 2012.  The focus of this webinar will be to review the scientific rationale for this clinical trial and to describe the current clinical trial design. With current funding available, the Phase II will be divided into two stages.  The date and time of that webinar will be announced within the next few days online at www.als.net.

About ALS Therapy Development Institute (www.als.net)
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, it 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 biotechnology institute, ALS TDI was founded in 1999. It has developed an industrial-scale platform, employs 30+ professional scientists and evaluates dozens of potential therapeutics each year. Built by and for ALS patients today, the Cambridge, Massachusetts based biotechnology institute works with leaders in both academia and industry to accelerate ALS therapeutic development, including Biogen Idec, UCB, Aestus Therapeutics, MDA and RGK Foundation.

About MDA
MDA (mda.org) is the world's leading nongovernmental provider of ALS services and funder of ALS research. Over the years, MDA has led the fight against ALS, investing more than $290 million in its ALS research, services and information programs. In addition, MDA operates some 200 clinics across the country, 41 of which are designated as ALS‐specific research and care centers. The Association's unparalleled research, health care services, advocacy and education programs provide help and hope to more than 1 million Americans affected by ALS and the more than 40 other progressive neuromuscular diseases.

About the Northeast ALS Consortium (www.alsconsortium.org)
The Northeast ALS Consortium (NEALS) is an international, independent, non-profit group of researchers who collaboratively conduct clinical research in Amyotrophic Lateral Sclerosis (ALS) and other motor neuron diseases. The mission of NEALS is to translate scientific advances into new treatments for people with ALS and motor neuron disease as rapidly as possible.  NEALS performs high quality clinical trials of potentially therapeutic agents to treat ALS and motor neuron disease and supports other projects, such as studies related to natural history, biomarkers, and the effects of ancillary treatment on disease progression. For more information on NEALS and ALS clinical research, please visit us online at www.alsconsortium.org.

Media Contact: Robert A. Goldstein, ALS TDI, [email protected], 617-441-7295

SOURCE ALS Therapy Development Institute

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