Healthcare Industry News:  histone deacetylase 

Biopharmaceuticals FDA

 News Release - August 20, 2007

FDA Grants Tikvah Therapeutics, Inc. Orphan Drug Status for Sodium Phenylbutyrate for the Treatment of Spinal Muscular Atrophy

ATLANTA, Aug. 20, 2007 (Healthcare Sales & Marketing Network) -- Tikvah Therapeutics, Inc., a biopharmaceutical company focused on new treatment options to better manage central nervous system diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Tikvah Therapeutics orphan drug status for sodium phenylbutyrate for the treatment of spinal muscular atrophy (SMA). Orphan drug designation entitles Tikvah to seven years of market exclusivity for phenylbutyrate for the treatment of patients with spinal muscular atrophy. Additional incentives for orphan drug development include tax credits related to certain development expenses, an exemption from the FDA user fee and FDA assistance in clinical trial design.

SMA, the leading genetic killer of children under the age of two, is a group of inherited and often fatal diseases that destroy the nerves controlling voluntary muscle movement, which affects crawling, walking, head and neck control, and even swallowing. There currently are no approved therapies for the treatment of SMA.

``We are pleased that the FDA has awarded us orphan drug status for the use of phenylbutyrate in the treatment of SMA. To accelerate our clinical programs, we plan to take full advantage of all means afforded by the Orphan Drug Act to establish and maintain an extremely close working relationship with the FDA and the Office of Orphan Products Development throughout the entire clinical and regulatory programs. We will also continue to build relationships with external research and clinical experts and various SMA advocacy groups to help accelerate the clinical development and eventual approval of this novel therapy,'' said Dr. Harold Shlevin, President and Chief Executive Officer of Tikvah Therapeutics. ``We are hopeful that our research will progress and offer some meaningful hope for a higher quality of life to both children with SMA and their parents.''

Bringing Hope to Life...(sm)

About the Orphan Drug Designation

The Orphan Drug Act (Public Law 97-414, enacted in 1983 and amended) provides for economic incentives to encourage pharmaceutical companies to develop drugs for rare diseases defined as those affecting fewer than 200,000 people in the United States. Orphan drug designation entitles Tikvah Therapeutics, Inc. to seven years of market exclusivity for phenylbutyrate for the treatment of patients with spinal muscular atrophy. Additional incentives for orphan drug development include tax credits related to certain development expenses, an exemption from the FDA user fee and FDA assistance in clinical trial design. Importantly, the Orphan Drug Act facilitates a close working relationship between regulatory agencies and companies aimed at accelerating the drug development and approval processes for treatment of rare diseases. For further information, please see: http://www.fda.gov/orphan/oda.htm.

About Spinal Muscular Atrophy (SMA)

Spinal muscular atrophy (SMA) is a genetic, motor neuron disease characterized by wasting of the skeletal muscles caused by progressive degeneration of the anterior horn cells of the spinal cord. The disease causes weakness and atrophy of the voluntary muscles, more commonly in the legs than in the arms. Patients with SMA either do not acquire, or progressively lose the ability to walk, stand, sit and, eventually, move. Cognition and intellect, emotional development and sensory nerves are unaffected.

SMA is one of the most prevalent genetic disorders. It can strike anyone of any age, race or gender. One in every 40 people carries the gene that causes SMA. The child of two carriers has a 25 percent chance of developing SMA.

There are four types of SMA generally recognized; however, they are rare, occurring in one in 8,000 to 10,000 births in the United States. SMA type I, also called Werdnig-Hoffman disease, is the most severe form which strikes infants between birth and six months old. Babies affected with type I cannot sit without support and 50 percent will die before their second birthday. SMA type II affects infants between seven and 18-months old. Children affected by SMA type II may be unable to stand or walk and are at increased risk for complications from respiratory infections. SMA type III, also known as Kugleberg-Welander disease, is the least deadly form of childhood-onset SMA. It strikes children as early as 18 months, but can surface as late as adolescence. SMA-type III patients are able to walk, but weakness is prevalent. Most patients eventually need to use a wheelchair. Adult SMA, SMAtype IV may begin between 40 and 60 years of age and progresses rapidly. Symptoms may include paralysis and atrophy of the leg muscles, arm muscle weakness and respiratory problems.

Further information on SMA maybe found at http://www.fsma.org , http://www.smafoundation.org , http://www.fightsma.org , amongst others.

About Sodium Phenylbutyrate

Sodium phenylbutyrate, a histone deacetylase (HDAC) inhibitor, currently approved to treat urea cycle deficiency, has been identified in in vitro systems and in various animal models as an agent that can increase the level of SMN protein. About 95 percent of individuals with SMA have genetic mutations that cause a deficiency of the SMN protein. This protein deficiency causes a loss of motor neurons which leads to the characteristic muscular atrophy and other signs and symptoms of SMA.

Findings from in vitro studies as well as pilot clinical work suggest that phenylbutyrate treatment in SMA patients may improve motor function.

Tikvah Therapeutics will be working in conjunction with the FDA, and collaborative clinical trial groups focused on SMA to develop well-controlled multicenter trials to fully evaluate sodium phenylbutyrate in the treatment of SMA. Tikvah Therapeutics is also supporting research on new diagnostic approaches and new therapeutic approaches to the treatment of SMA and allied neurodegenerative diseases such as ALS (amyotropic lateral sclerois) and MS (multiple sclerosis).

About Tikvah Therapeutics, Inc.

Atlanta-based Tikvah Therapeutics, Inc. focuses on exploring new uses for late-stage pharmaceutical compounds in selected therapeutic indications of Central Nervous System diseases, including 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. Tikvah was founded by Paramount BioSciences, LLC. For additional information, please see http://www.tikvahtherapeutics.com

About Paramount BioSciences

Paramount BioSciences, LLC is a global drug development and healthcare development firm that conceives, nurtures, and supports new biotechnology companies. For additional information about Paramount BioSciences, please visit http://www.paramountbio.com.

Forward Looking Statement

This press release contains certain forward-looking information that is intended to be covered by the safe harbor for ``forward-looking statements'' provided by the Private Securities Litigation Reform Act of 1995. Forward- looking statements are statements that are not historical facts. Words such as ``expect(s),'' ``feel(s),'' ``believe(s),'' ``will,'' ``may,'' ``anticipate(s)'' and similar expressions are intended to identify forward-looking statements. These statements include, but are not limited to, financial projections and estimates and their underlying assumptions; statements regarding plans, objectives and expectations with respect to future operations, products and services; and statements regarding future performance. Such statements are subject to certain risks and uncertainties, many of which are difficult to predict and generally beyond the control of Tikvah Therapeutics Inc., that could cause actual results to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include: those generally associated with developmental stage biopharmaceutical companies; the progress or likelihood of success of our product research and development programs; potential benefits from obtaining orphan drug designation; the status of our preclinical and clinical development of potential drugs; the likelihood of success of our drug products in clinical trials and the regulatory approval process; our drug products' efficacy, abuse and tamper resistance, onset and duration of drug action, ability to provide protection from overdose, ability to reduce the development of tolerance, ability to improve symptomatology or otherwise improve patients' symptoms; the incidence of adverse events; the ability to develop, manufacture, launch and market our drug products; our projections for future revenues, profitability and ability to achieve certain sales targets; our estimates regarding our capital requirements and our needs for additional financing; the likelihood of obtaining favorable scheduling and labeling of our drug products; the likelihood of regulatory approval under Section 505(b)(2) and other applicable Sections under the Federal Food, Drug, and Cosmetic Act; our ability to develop safer and improved versions of widely-prescribed drugs using our technology; and our ability to obtain favorable patent claims. Readers are cautioned not to place undue reliance on these forward- looking statements that speak only as of the date hereof. Tikvah Therapeutics Inc. does not undertake any obligation to republish revised forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.

``Tikvah Therapeutics, Inc.'', ``Bringing Hope to Life'', and the above logo design are trademarks of Tikvah Therapeutics, Inc., Atlanta, Georgia USA


Source: Tikvah Therapeutics

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