LIBERTYVILLE, Ill. and ATLANTA, Aug. 28, 2007 (PRIME NEWSWIRE) -- Families of Spinal Muscular Atrophy (FSMA) and Tikvah Therapeutics, Inc. today announced that they have established a collaboration to help advance and accelerate the clinical development of Tikvah Therapeutics, Inc.'s new formulation of sodium phenylbutyrate, TIK-201, for the treatment of Spinal Muscular Atrophy (SMA). SMA is the leading genetically inherited cause of death of children under the age of two years. This collaboration is focused on utilizing the existing Project Cure SMA clinical network and protocols to clinically test Tikvah Therapeutics' non-viscous, concentrated solution formulation.
Spinal Muscular Atrophy is an often-fatal genetic disorder resulting from the loss of both copies of the Survival Motor Neuron (SMN1) gene. This causes a chronic deficiency in the production of the SMN protein, which is essential to the proper functioning of the motor neurons in the spinal cord and to the control of muscles in the limbs, neck and chest. Early ex-vivo and other clinical results suggest that sodium phenylbutyrate, amongst other HDAC inhibitors, may be effective in treatment of SMA by increasing production of the remaining SMN2 protein. However such trials have been complicated by difficulties in administering currently available tablet and powder preparations of sodium phenylbutyrate due to its unpleasant taste and smell and other properties. Tikvah's proprietary formulation overcomes these limitations and is optimized for delivery of drugs to infants, toddler and others with swallowing difficulties by conventional oral, nasogastric and g-tube routes of administration.
Specific details on the trial design and dates will be available later this year, with the goal of beginning enrollment in the first half of 2008.
"Testing of Sodium Phenylbutyrate has been hampered by the unpleasant taste, smell and large bulk of poorly soluble preparations," said Lou Barbato, M.D., V.P. Clinical Research & Development and Chief Medical Officer of Tikvah Therapeutics, Inc. "We believe that this formulation has the potential to enhance patient compliance and to facilitate the treatment of infants and children with SMA."
Kenneth Hobby, Executive Director of Families of SMA said "We applaud Tikvah Therapeutics for their insight and commitment to helping bring hope to families living with SMA, and look forward to a successful collaboration. Building networks between clinicians and companies to advance SMA research is a part of the Families of SMA research strategy, and we are excited to have Tikvah Therapeutics as a new partner is the effort to find an effective treatment and cure for SMA."
"We are very excited by the opportunity to work closely with FSMA and its network of clinical investigators. These dedicated individuals have not only significantly advanced our knowledge of SMA, but they have importantly developed the critical techniques and systems allowing consistent and well controlled clinical studies to be conducted in very young patients with SMA," said Dr. Harold H. Shlevin, President and CEO of Tikvah Therapeutics, Inc. "This is a critical first step toward demonstrating the efficacy and safety of new agents for the treatment of SMA. It is an honor and a privilege for us to work with the dedicated individuals at FSMA, its clinical collaborators as together we seek to help the patients and their parents who suffer from the devastating impacts of SMA."
About FSMA: FSMA is dedicated to eradicating SMA by promoting and supporting research, helping families cope through informational programs and support, and educating the public and the medical community about SMA. The organization, originally founded in 1984 by a small group of parents, has grown to more than 32 chapters and affiliates worldwide and more than 5,000 member families. FSMA receives the majority of its funding through volunteer efforts, funding over $30 million to date, and continues to increase its funding commitments each year with $15 million in new research planned over the next three years. In addition, Families of SMA has funded more than $3 million in patient support efforts. Since its founding, FSMA-sponsored research has made significant contributions to better understanding SMA and advancing new therapies towards human clinical testing. These accomplishments include:
* Identification of a mutation in the SMN1 gene as the cause of
the disease. A second copy of the gene called SMN2 produces
reduced amounts of SMN protein due to a defect in splicing.
* The funding of two leading-edge drug discovery programs
designed to increase functional protein production from the
SMN2 gene to compensate for the loss of the SMN1 gene.
* The establishment of Project CURE SMA, a 7 center clinical
trial network, which is currently testing two medications for
their possible impact on treating SMA patients. This network
will also serve as the conduit for future human drug trial
For more information visit the website www.curesma.com or call 1-800-886-1762.
About Tikvah Therapeutics, Inc.: Tikvah Therapeutics, Inc., Atlanta, Ga., focuses on exploring new uses for late-stage pharmaceutical compounds in selected therapeutic indications of Central Nervous System diseases -- neurology and psychiatry. Its focus is on new therapeutic uses which have been confirmed in multiple, clinical proof-of-concept studies. This strategy shortens product development timelines and substantially decreases the risk associated with the research and development efforts. A second prong of its strategy is to focus on specialized products with multiple stepping-stone indications and strong patent protections, thus helping to ensure long product life cycles and manageable commercial risk. For further information, please see www.tikvahtherapeutics.com
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