Antisense ALS treatment
(Original articles available below)

"Antisense ALS treatment announced by Isis:- ISIS 333611 Granted Orphan Drug Status for Treatment of ALS"

Isis Pharmaceuticals, Inc. (Nasdaq: ISIS)announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISIS 333611 for thetreatment of an inherited form of amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig's disease. ALS is aprogressive neurodegenerative disease resulting in the loss of muscle control that can lead to death. ISIS 333611, a secondgenerationantisense drug, is Isis' first drug treating neurodegenerative diseases to enter development and will be administereddirectly into the cerebral spinal fluid."ALS is a terrible disease in need of innovative and efficacious new therapies. We are pleased that ISIS 333611 has beengranted orphan drug status for this small but important patient population. In our preclinical studies, we demonstrated thatdirect delivery of an antisense drug onto the spinal fluid via an implantable pump effectively reduced SOD1, a proteinassociated with the inherited form of ALS," said C. Frank Bennett, Ph.D., Senior Vice President of Research at IsisPharmaceuticals, Inc. "Our preclinical success with ISIS 333611 demonstrates the applicability of antisense technology to treata variety of diseases that affect the central nervous system. In addition, with funding from CHDI, we recently initiated a separatedrug discovery program in Huntington's Disease, another fatal neurodegenerative disease."ABOUT ISIS 333611 ( Antisense ALS treatment )ISIS 333611 is a second-generation antisense drug in development for the treatment of ALS through the inhibition of Cu/Znsuperoxide dismutase (SOD1), a molecule that is associated with an inherited, aggressive form of ALS. In preclinical rodentstudies, inhibition of SOD1 extended survival of rats that show many features of ALS. The ALS Association and the MuscularDystrophy Association are providing funding for IND-enabling studies of ISIS 333611.

About Orphan Drug Designation

The Orphan Drug Act provides for economic incentives to encourage the development of drugs for diseases affecting fewerthan 200,000 people in the United States. Orphan drug designation entitles Isis Pharmaceuticals to seven years of U.S. marketexclusivity for ISIS 333611 for the treatment of patients with familial ALS.

SOURCE Isis Pharmaceuticals, Inc.

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