Other

We have been successful in developing novel drugs and licensing them to highly focused satellite companies that have the specific expertise and resources to continue developing these drugs. Together with our partners we continue to advance new drugs into development and move antisense drugs into clinical studies that are outside of our core therapeutic areas.

• Vitravene^® (CMV Retinitis)
• ACHN-490 (Severe Bacterial Infection)
• iCo-007 (Diabetic Macular Edema, Diabetic Retinopathy)
• ATL1103 (Acromegaly, Diabetic Retinopathy)
• EXC 001 (Local Fibrosis)

Vitravene^®

Vitravene, approved by the FDA in 1998, is an antisense drug that we discovered and developed, to treat cytomegalovirus, or CMV retinitis in AIDS patients. Novartis Ophthalmics AG, our worldwide distribution partner for this drug, launched Vitravene in November 1998. New anti-HIV drugs, particularly protease inhibitors and combination treatment regimens, have prolonged survival in HIV-infected individuals. This has resulted in a decline in mortality from AIDS, accompanied by a decline in the incidence of many opportunistic infections, including CMV retinitis. As a result, Novartis no longer markets Vitravene. Vitravene demonstrates our ability to meet FDA and European regulatory requirements for safety and efficacy, and for the commercial manufacture of antisense drugs.

ACHN-490

ACHN-490 is a next-generation aminoglycoside, or neoglycoside, drug that Achaogen is developing for the treatment of multi-drug resistant gram-negative bacterial infections. Aminoglycosides are a group of antibiotics that inhibit bacterial protein synthesis and that clinicians use to treat serious bacterial infections. ACHN-490 incorporates aminoglycoside technology that we licensed to Achaogen. In earlier studies, ACHN-490 displayed broad-spectrum activity against multi-drug resistant gram-negative bacteria that cause systemic infections, including E. coli and methicillin-resistant staphylococcus aureus, or MRSA. In preclinical studies, ACHN-490 demonstrated an acceptable safety profile and the potential for once-daily dosing. In September 2009, Achaogen reported the successful completion of a Phase 1 study on ACHN-490. In August 2010, Achaogen initiated a Phase 2 study on ACHN-490 and we achieved a milestone for this advancement.

iCo-007

iCo-007 is an antisense drug that targets c-Raf kinase. In preclinical studies, antisense inhibition of c-Raf kinase was associated with a reduction in the formation and leakage of new blood vessels in the eye, suggesting inhibiting c-Raf kinase can improve treatment for both diabetic macular edema and diabetic retinopathy. Diabetic retinopathy is one of the leading causes of blindness in people in the U.S., and nearly 100 percent of type 1 diabetics by age 20 have evidence of retinopathy. Additionally up to 21 percent of people with type 2 diabetes have retinopathy when they are first diagnosed with diabetes, and most will eventually develop some degree of retinopathy. We discovered iCo-007 and licensed it to iCo for the treatment of various eye diseases that occur as complications of diabetes.

In May 2010, investigators evaluating iCo-007 in patients with diffuse Diabetic Macular Edema presented positive results from the Phase 1 study showing that treatment with iCo-007 was well tolerated. In July 2010, iCo received approval to initiate a Phase 2 study on iCo-007 in patients with Diabetic Macular Edema.

ATL1103

ATL1103 is an antisense drug that targets growth hormone receptor, or GHr, a receptor that, when inhibited, reduces the level of circulating insulin-like growth factor-1, or IGF-1, produced in the liver. IGF-1 is a hormone that contributes to various diseases including acromegaly, which is characterized by abnormal growth of organs, face, hands and feet, as well as for diabetic retinopathy, a common disease of the eye and a leading cause of blindness. In preclinical studies, ATL1103 demonstrated significant reductions in IGF-1 levels in the blood. ATL has recently completed preclinical toxicity studies on ATL1103.

EXC 001

EXC 001 is an antisense drug that targets connective tissue growth factor (CTGF), a growth factor that is over-expressed in damaged skin or tissue following a traumatic event. We co-discovered and licensed EXC 001 to Excaliard for the local treatment of fibrotic diseases, including scarring. Fibrosis represents a significant and expanding area of unmet medical need where antisense drugs could offer a unique advantage for anti-fibrotic agents. Excaliard reported the successful completion of a Phase 1 study on EXC 001 and has initiated three Phase 2 studies on EXC 001. In August 2010, Excaliard reported positive data from the first of the three Phase 2 studies demonstrating that treatment with EXC 001 reduced scarring in patients following elective abdominal surgery.