Inflammation and Other Diseases

The broad applicability of our antisense technology allows us to create promising drugs and we have successfully developed novel drugs designed to treat many different diseases. In therapeutic areas that are outside of our core areas of development, we have licensed our drugs to highly focused satellite companies that have the specific expertise and resources to continue developing the drugs. Together with our partners we continue to advance drugs in clinical development that are outside of our core therapeutic areas, such as the ocular and antiviral drugs we and GSK are developing under our preferred partner collaboration.


Plazomicin, formerly ACHN-490, is a next-generation aminoglycoside drug that Achaogen, Inc. 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. Achaogen discovered plazomicin based on technology licensed from us.

Plazomicin has displayed broad-spectrum activity in animals against multi-drug resistant gram-negative bacteria that cause systemic infections, including E. coli, and against methicillin-resistant staphylococcus aureus, or MRSA. In preclinical studies, plazomicin demonstrated an acceptable safety profile and the potential for once-daily dosing. Achaogen has completed a Phase 1 study of plazomicin in healthy volunteers and a Phase 2 study. In the Phase 2 study, Achaogen evaluated plazomicin compared to levofloxacin for the treatment of complicated urinary tract infections and acute kidney infections in adults. In this study, patients treated with plazomicin tolerated the drug well and patients demonstrated favorable activity of plazomicin as compared to levofloxacin.

Achaogen is currently evaluating plazomicin in a Phase 3 study in patients with serious multi-drug resistant, or MDR, gram-negative bacterial infections. The Phase 3 study is designed as a superiority study to evaluate the efficacy and safety of plazomicin compared to colistin in patients with bloodstream infections and nosocomial pneumonia caused by carbapenem-resistant Enterobacteriaceae, or CRE. Achaogen announced last year that it has reached a special protocol assessment, or SPA, with the U.S. Food and Drug Administration for this Phase 3 study.

EXC 001

EXC 001 is an antisense drug that targets connective tissue growth factor, or CTGF, a growth factor that is over-expressed in damaged skin or tissue following a traumatic event. We co-discovered EXC 001 with Excaliard Pharmaceuticals, Inc. and exclusively licensed it 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 as anti-fibrotic agents. In November 2011, Pfizer Inc. acquired Excaliard. Pfizer continues to evaluate EXC 001 in a Phase 2 program designed to provide information, including the optimization of the dose, for the design of the Phase 3 program for EXC 001.


ATL1102 is an antisense drug that ATL is developing for the treatment of multiple sclerosis, or MS. ATL1102 inhibits CD49d, a subunit of Very Late Antigen-4, or VLA-4. Studies in animal models have demonstrated that inhibiting VLA-4 positively affects a number of inflammatory diseases, including multiple sclerosis, or MS. We licensed ATL1102 to ATL in December 2001. In 2008, ATL reported Phase 2a results of ATL1102 showing significantly reduced disease activity in patients with relapsing remitting MS. In 2014, ATL completed a chronic toxicology study in primates to support a potential Phase 2b trial of ATL1102 in patients with MS.


RG-101 is a preclinical drug that Regulus is developing. RG-101 is an anti-miR, antisense drug designed to target microRNA-122, or miR-122. Researchers believe that miR-122 is essential for the replication of HCV suggesting that an anti-miR-122 drug may reduce HCV infection and improve HCV-associated pathologies like fatty liver.

MicroRNAs are small RNA molecules that do not encode proteins, but instead work as natural antisense sequences that scientists believe regulate the expression of approximately one-third of all human genes.

In February 2015, Regulus reported data from the Phase 1/2 study of RG-101 in patients with HCV. Regulus reported results from this study demonstrating that treatment with a single subcutaneous dose of 2 mg/kg or 4 mg/kg of RG-101 as monotherapy resulted in significant and sustained reductions in HCV RNA in a varied group of patients, including difficult to treat genotypes and patients who experienced viral relapse after a prior interferon-containing regimen. Additionally, RG-101 was well tolerated and has demonstrated a favorable pharmacokinetic profile to date, which Regulus believes may allow RG-101 to be combined with oral direct-acting antiviral agents to treat HCV. Regulus expects to initiate two Phase 2 studies evaluating RG-101 as a single agent and in combination with direct acting antivirals in patients with HCV in 2015.


ISIS-HBVRx, formerly ISIS-GSK3Rx, is an antisense drug we designed to reduce the production of viral proteins associated with hepatitis B virus (HBV) infection and replication. Hepatitis B virus infection is a serious health problem that can lead to significant and potentially fatal health conditions. Chronic HBV infection is one of the most common persistent viral infections in the world. Currently available therapies, including oral antiviral agents or injectable interferons, do not clear HBV and do not effectively clear HBV antigens from these patients. As a result, patients are unable to fully control HBV infection and achieve sustained disease remission. Many of these patients are at elevated risk for severe liver complications such as cirrhosis and primary liver cancer

In preclinical studies, an antisense compound targeting HBV produced dose-dependent reductions of HBV-associated antigens, including hepatitis B surface antigen, which is present in both acute and chronic infections and is associated with a poor prognosis in patients with chronic HBV infection. In addition, other measures of viral infection were reduced in both the liver and serum following treatment with the ISIS-HBVRx. We have completed a Phase 1 study of ISIS-HBVRx in healthy volunteers.


ISIS-GSK4-LRx is an antisense drug we designed to an undisclosed target to treat an ocular disease. We are developing ISIS-GSK4-LRx as part of our collaboration with GSK. We are eligible to receive milestone payments from GSK as ISIS-GSK4-LRx advances in development, and we are responsible for developing the drug up to Phase 2 proof-of-concept, at which time GSK has the option to pay us a license fee and license ISIS-GSK4-LRx from us. We are also eligible to receive double-digit royalties on sales of ISIS-GSK4-LRx.


ISIS-GSK6-LRx is in preclinical development. We are developing ISIS-GSK6-LRx as part of our collaboration with GSK.

About Isis

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Mission & Values

Our mission is to provide antisense drugs to patients with significant unmet medical needs.

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