[SEOUL & FAIR LAWN, N.J.=Whowired] April 20, 2012—SK Biopharmaceuticals, a South Korean pharmaceutical company, announced today that it was granted special status by the U.S. Food and Drug Administration (FDA) for carisbamate for the management of patients with infantile spasms (IS), a form of epilepsy associated with increased risk of death or mental retardation.

Dr. Christopher Gallen, CEO of SK Biopharmaceuticals, said, “Carisbamate is an important new anti-seizure therapy. Experimental evidence demonstrates its utility in a model of IS. (1) The designation of carisbamate as an Orphan Drug by the FDA facilitates our progression of this vital medication to yet another area of great unmet medical need.”

The FDA grants Orphan Drug designation to products that are intended to treat rare or orphan diseases or conditions such as infantile spasms. For IS patients, as with patients fighting other rare diseases, new treatments are few and far between, making this designation by the FDA of significant importance.

“IS, or West Syndrome, is a devastating and difficult to treat form of childhood epilepsy that often leads to some form of mental retardation and other behavioral problems over time,” said Dr. Jeongwoo Cho, Vice President of SK Biopharmaceuticals’ Drug Development Business. “Carisbamate is being developed to provide physicians with an additional treatment option for patients with IS for whom there are very few safe and effective treatments.”

Carisbamate has been designated as an orphan drug by the FDA under the auspices of the Orphan Drug Act. The Act, which will mark its 30th anniversary on January 4, 2013, provides incentives to sponsors that develop therapies intended to treat orphan conditions, which are classified as any condition or disease that affects less than 200,000 patients per year in the U.S. It is estimated that there are more than 6,000 rare diseases affecting 25 million Americans. (2, 3)

Carisbamate has previously been shown to have efficacy in clinical trials for adult forms of epilepsy, such as partial onset seizures. (4-6)

About Infantile Spasms

 An infantile spasm (IS) is a specific type of seizure that occurs in a chronic epilepsy syndrome of infancy and childhood known as West Syndrome. West Syndrome is characterized by infantile spasms, developmental regression, and a specific pattern on electroencephalography. The onset of infantile spasms is usually in the first year of life, typically between 4-8 months. The seizures primarily consist of a sudden bending forward of the body with stiffening of the arms and legs; some children arch their backs as they extend their arms and legs. Spasms tend to occur upon awakening or after feeding, and often occur in clusters of up to 100 spasms at a time. Infants may have dozens of clusters and several hundred spasms per day. Infantile spasms usually stop by age five, but may be replaced by other seizure types. (7) Infantile spasms are the major cause of cognitive deterioration in the first year of life. (8) An estimated 8,500 infants in the United States have been diagnosed with IS (9, 10), and each year approximately 2,500 new cases of IS are reported in the U.S.

About Carisbamate

 Carisbamate is a chlorophenyl carbamate that has a distinctive chemical structure compared to other currently available antiepilepsy drugs. It was initially developed to reduce the adverse effects associated with other antiseizure medications while still maintaining efficacy. The exact mechanism of action of carisbamate’s therapeutic benefit has not been yet been defined.

Though not currently approved for any use in the U.S., SK Biopharmaceuticals plans to seek FDA approval for carisbamate in the management of patients with IS.

Carisbamate is one of the key products in SK Biopharmaceuticals’ central nervous system (CNS) development pipeline. The company is advancing its near-term pipeline on other fronts as well. In January 2010, the company completed a pilot study of the antiepileptic drug YKP3089 in patients with photosensitive epilepsy. A Phase 2b study in patients with treatment-resistant partial onset seizures is ongoing.

About SK Biopharmaceuticals

 SK Biopharmaceuticals is a newly-independent development-stage pharmaceutical company as of April 2011, after being spun out from SK Holdings, the third-largest conglomerate in South Korea. SK Biopharmaceuticals focuses on discovery and development of new drugs as well as providing custom services for the manufacture and sales of active pharmaceutical ingredients in addition to intermediates of drugs for other major pharmaceutical companies.

SK Biopharmaceuticals, through its affiliate SK Life Science, leads all Korean pharmaceutical companies with 13 authorized INDs in the U.S. Among compounds in current clinical development are YKP3089 in Phase 2b trials for the treatment of epilepsy and neuropathic pain, SKL11197 in a Phase 2a trial for the treatment of neuropathic pain, and YKP10811 in a Phase 2a trial for chronic constipation. In February, YKP3089 was selected for funding by the Korea Drug Development Fund. SK Biopharmaceuticals intends to file INDs later this year for two additional compounds, one for the treatment of Alzheimer’s disease and one for neuropathic pain; both of these compounds are first-in-class candidates acting via new mechanisms of action.

In parallel with its internal development, SK Biopharmaceuticals has adopted a strategy of collaboration with partners to accelerate drug discovery and development. Its current partners include Neuronex with diazepam nasal spray for the treatment of acute repetitive seizures, Aerial Biopharma with SKL-N05 for the treatment of narcolepsy, Medicilon with SKL-PSY for bipolar disorder, and the Mayo Clinic to identify molecules that interact with the TDP-43 protein in amyotrophic lateral sclerosis (ALS, Lou Gehrig’s disease).

The Drug Development Business division of SK Biopharmaceuticals has a preclinical research and development center located in Daejeon, South Korea, and SK Life Science, its business development and clinical research center in New Jersey, for the clinical development of its assets in the areas of epilepsy, neuropathic pain, depression, irritable bowel syndrome, and Parkinson’s disease.

The Custom Manufacturing Service (SK CMS) is a second business division of SK Biopharmaceuticals; it generated $40 million last year in the production of Active Pharmaceutical Ingredients for other pharmaceutical company customers. These customers have recognized the quality of the products and the operational efficiency provided by the Korean FDA-approved manufacturing facility in Daejeon, Korea. This facility is fully automated and is used to conduct technologies proprietary to SK Biopharmaceuticals, such as continuous flow synthesis, low temperature reactions, and catalytic conversions, that differentiate the company from other chemical suppliers.

For more information, visit www.skbp.com