"In addition to our pipeline advances, we continue to support our satellite companies by participating in their financings.  And, we continue to see a strong interest in antisense technology from large pharmaceutical companies as evidenced by OncoGenex' licensing of OGX-011 to Teva Pharmaceuticals and Regulus' expanded alliance with GSK.  As you can see, 2009 was a productive year and we believe that we will continue this momentum through 2010. With a pipeline of 22 drugs, we expect to have a steady stream of accomplishments as drugs move into the clinic, complete clinical studies and advance into Phase 2 and Phase 3 development.  In summary, we are making excellent progress across all areas of our business.  Our financial position remains strong and it enables us to invest in our business and our technology in ways that we believe will continue to generate value for our shareholders," concluded Ms. Parshall.

2010 Goals

In 2010, Isis is planning to achieve the following goals itself and with partners:

Prepare for filing new drug applications for mipomersen in the United States (NDA) and Europe (MAA) in the first half of 2011 with Genzyme   Complete and report remaining Phase 3 studiesReport data on the following drugs: ISIS-CRPRx (Phase 1)ISIS-SGLT2Rx (Phase 1)iCo-007 (Phase 1 - iCo Therapeutics)AIR645 (Phase 2 - Altair Therapeutics)Continue to advance its pipeline.  Together with partners initiate the following clinical studies: Phase 3 study on OGX-011 (OncoGenex and Teva)Phase 2 studies on 5 drugsPhase 1 studies on 2 drugsBroaden its pipeline by: Expanding further into new therapeutic areas including neurodegenerative diseases and cancerAdding 3 to 5 new drugs to its pipelineAdvance antisense technology by: Selecting the next generation antisense chemistryAdd new partnership and/or satellite company relationships

Ortho-McNeil Collaboration

Isis' collaboration with Ortho-McNeil Janssen Pharmaceuticals (OMJP) has ended and Isis has regained the rights to drugs from both the glucagon receptor and glucocorticoid receptor programs.  Isis reported positive Phase 1 data on ISIS-GCGRRx.  Antisense inhibition of GCGR with ISIS-GCGRRx improved blood glucose levels following a glucagon challenge with statistical significance at a dose of 400 mg/week and resulted in positive trends at lower doses.  The drug was well tolerated at all doses.  Isis intends to move a more potent inhibitor for its GCGR program forward that was identified as part of its collaboration with Ortho-McNeil.  A more potent drug should enhance the therapeutic profile of the GCGR program and provide much greater commercial value.  Isis also intends to move forward the GCCR program, which reduced blood glucose and demonstrated a dramatic and favorable effect on lipid levels including cholesterol and triglycerides, and reduced body fat in preclinical studies.  

Drug Development Highlights

Mipomersen continues to advance in clinical development and move closer to the market for patients who cannot adequately control their cholesterol levels with current therapies and who need new treatment options.  Isis and Genzyme reported positive data from two Phase 3 studies evaluating mipomersen in patients with familial hypercholesterolemia (FH). In a Phase 3 study evaluating mipomersen in patients with homozygous FH, Isis and Genzyme reported that the study met its primary endpoint with a 25% reduction in LDL-C after 26 weeks of treatment compared to a decrease of 3% for placebo (p<0.001), which constitutes an average reduction of LDL-C>100 mg/dL, and also met all of its secondary and tertiary endpoints.  In a Phase 3 study evaluating mipomersen in patients with heterozygous FH, Isis and Genzyme reported that the study met its primary endpoint with a 28% reduction in LDL-C after 26 weeks of treatment compared to an increase of 5% for placebo (p<0.001) and also met all of its secondary endpoints.  Patients treated with mipomersen had an average LDL-C level of 104 mg/dL at the end of the study and 45% of the mipomersen-treated patients achieved LDL-C levels of less than 100 mg/dL.Isis reported positive Phase 2 data on ISIS 113715 in patients with type 2 diabetes on stable doses of sulfonylurea.   In this study, patients treated with 200 mg per week of ISIS 113715 for 13 weeks achieved consistent and statistically significant reductions in multiple short and intermediate measures of glucose control.  Isis reported positive Phase 1 data in which ISIS-GCGRRx produced a significant improvement in glucagon-induced blood glucose levels and was well tolerated.  In addition, Isis and its partners continued to advance the drugs in Isis' pipeline and reported encouraging clinical results in a broad range of diseases. Isis and its partners presented positive Phase 1 data on six drugs including, LY2181308, OGX-427, ISIS-GCGRRx, AIR645, iCo-007 and ACHN-490, and positive Phase 2 data on OGX-011.  Isis and its partners added four new drugs to its pipeline including ISIS-FXIRx to treat thrombosis, ISIS-SMNRx to treat spinal muscular atrophy, ISIS-APOCIIIRx to treat cardiovascular disease, and ACHN-490 to treat severe bacterial infections.  Isis and its partners initiated Phase 1 clinical studies on four drugs and initiated Phase 2 studies on two drugs including AIR645 and EXC 001.

Corporate Highlights

Isis executed its business strategy by successfully monetizing a key asset. Isis sold its Ibis subsidiary to AMI.  Isis benefited financially as its partners advanced drugs in development.  Isis received $11 million in milestone and sublicensing fees. Isis also benefited from its partnerships focused on developing and advancing certain RNA-based therapeutic technologies, receiving nearly $13 million in total.  Isis supported its satellite company partners who are developing antisense drugs discovered by Isis for the treatment of a broad range of diseases by participating in the financings of iCo Therapeutics, Altair Therapeutics, Excaliard Pharmaceuticals, and Regulus Therapeutics.Isis strengthened its intellectual property assets by obtaining patent grants and allowances that expand Isis' fundamental patents and increase protection of Isis' drugs in development. Isis also exclusively licensed certain intellectual property from the University of Massachusetts to develop drugs to treat spinal muscular atrophy (SMA).  Funding support for the University of Massachusetts' research program responsible for creating this intellectual property was provided in part by Families of SMA.    Regulus formed a new alliance with GSK to develop and commercialize microRNA therapeutics targeting miR-122 for hepatitis C viral infection.

SOURCE Isis Pharmaceuticals, Inc.