Primary Sclerosing Cholangitis – SC404

Primary Sclerosing Cholangitis (PSC) is characterized by progressive inflammatory fibrotic destruction of the biliary ducts and patients with PSC often have chronic jaundice, hepatomegaly and pruritus. The median time from diagnosis to death or liver transplant is 9-18 years. In most patients, PSC progresses slowly and can lead to liver failure, repeated infections, and tumors of the bile duct or liver.It is estimated that approximately 35,000 adults in the United States suffer from PSC, according to a report published by the Orphanet Journal of Rare Diseases. In addition, according to a report of a researcher with the Mayo Clinic, the estimated prevalence of PSC in Western populations is six to eight cases per 100,000 persons.

Problem: There are no approved medical therapies for PSC, and high dose ursodeoxycholic acid, which was a common treatment, is no longer recommended due to significant adverse events.

Current Treatment: Liver transplant is the only known treatment for PSC.

ALT™ Solution:  Using ALT™, we believe our therapy may reduce the patient’s inflammatory response and thereby slow, or potentially reverse, the progression of PSC. We believe this treatment may reduce the patient’s inflammatory response and will slow the progression of PSC by preventing the scarring of the bile ducts in the liver.

In October 2014, we submitted an application to the Federal Drug Administration requesting Orphan Drug designation for SC404 for the treatment of PSC and our application was granted in December 2014. This designation may provide seven years of market exclusivity in the United States, subject to certain limited exceptions. However, the orphan drug designation does not convey any advantage in, or shorten the duration of, the regulatory review or approval process. The FDA has requested a brief progress report of our development of SC404 within 14 months of the grant date and annually thereafter until we receive marketing approval.

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