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Severe and Rare

Our severe and rare disease franchise is the largest franchise in our pipeline. We believe that our antisense technology could offer effective therapies for patients with severe and rare diseases and neurological disorders that are life-threatening or fatal and for which there are limited treatment options. According to the National Institutes of Health, or NIH, there are approximately 5,000 to 8,000 rare diseases, many life-threatening or fatal. Unfortunately, patients with many of these severe and rare diseases have few effective therapies available. Since most severe and rare diseases are genetic or have a genetic component, parents often pass the disease to their children, creating a legacy of the disease and resulting in profound effects on the family. ISIS-SMNRx, the most advanced neurological drug in our pipeline, is now in two Phase 3 studies for the treatment of infants and children with SMA.

We are discovering and developing antisense drugs to treat severe and rare and neurological diseases for which there is a need for new treatment options. We have established strategic alliances in drug development areas that are high risk or in which our partners have significant expertise and resources to allow us to expand our drug discovery and development efforts beyond what we would choose to do internally. For example, our strategic partnerships with Biogen Idec and Roche have supported advancing five drugs for the treatment of neuromuscular or neurological diseases in our pipeline.

Due to the severe nature of these diseases and the lack of available treatments, there is an opportunity for more flexible and efficient development paths to the market. This means that, in some cases, the studies necessary for us to demonstrate proof-of-concept with a particular drug may also be the studies that complete our marketing registration package, thereby providing us with a relatively rapid path to market for potential new treatments for devastating and often fatal diseases.

KYNAMRO® (mipomersen sodium)

NOW APPROVED

Kynamro

KYNAMRO® (mipomersen sodium) is an oligonucleotide inhibitor of apolipoprotein B-100 synthesis indicated as an adjunct to lipid-lowering medications and diet to reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B (apo B), total cholesterol (TC), and non-high density lipoprotein-cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).

KYNAMRO is approved for use in patients with HoFH in the United States, Mexico, Argentina and South Korea.

U.S. Product Web Site

U.S. Prescribing Information (PDF)

Alicaforsen

Alicaforsen, now under license to Atlantic Pharmaceuticals Limited, is an antisense drug that targets intercellular adhesion molecule 1, or ICAM-1. ICAM-1 is over-expressed in a wide variety of inflammatory disorders, including ulcerative colitis and pouchitis. Ulcerative colitis, or UC, is an inflammatory bowel disease, or IBD, of the colon, a part of the large intestine, and pouchitis is an inflammation of the surgically constructed internal pouch created in UC patients who have had their diseased colons removed.

Atlantic Pharmaceuticals Limited is developing and selling alicaforsen through a named patient program. A named patient program allows Atlantic to sell alicaforsen in response to physicians' requests under international named patient supply regulations for patients with pouchitis and other indications.

In 2007, we licensed alicaforsen to Atlantic Pharmaceuticals for pouchitis, UC and other inflammatory diseases. The FDA and European Medicines Agency, or EMA, have since granted alicaforsen Orphan Drug Designation for the treatment of pouchitis in the United States and Europe, respectively. We are eligible to receive royalties on product sales, including product sales under the named patient supply program from Atlantic Pharmaceuticals. Atlantic Pharmaceuticals is currently pursuing opportunities to fund further development of alicaforsen.

ISIS-TTRRx

ISIS-TTRRx is an antisense drug we designed to treat TTR amyloidosis, a severe and rare genetic disease in which the patient inherits a mutant gene that produces a misfolded form of TTR, which progressively accumulates in tissues. In patients with TTR amyloidosis, both the mutant and normal forms of TTR can build up as fibrils in tissues, such as the heart, peripheral nerves, and the GI tract. The presence of TTR fibrils interferes with the normal functions of these tissues, and as the TTR protein fibrils enlarge, more tissue damage occurs and the disease worsens.

We are evaluating ISIS-TTRRx to treat two types of TTR amyloidosis, familial amyloid cardiomyopathy, or FAC, which affects more than 40,000 patients worldwide, and familial amyloid polyneuropathy, or FAP, which affects more than 10,000 patients worldwide. Patients with FAC have TTR build up in the heart muscle and succumb to heart failure approximately five to six years after symptom onset. Patients with FAP have TTR build up in peripheral nerve tissue leading to the loss of nerve function and wasting.

ISIS-TTRRx is the first drug to enter development under our preferred partner alliance with GSK. We designed ISIS-TTRRx to inhibit the production of all forms of TTR, and to offer an alternative approach to treat all types of TTR-related amyloidosis. We completed a Phase 1 study evaluating the safety and activity of ISIS-TTRRx in healthy volunteers. In this study, ISIS-TTRRx produced rapid, dose-dependent reductions in plasma TTR protein with an average of 75 percent reduction in TTR protein, with some subjects achieving approximately 90 percent reduction. In addition, there were several subjects that reached TTR protein levels that were below the limit of assay detection. Subjects treated with ISIS-TTRRx generally tolerated the drug well.

In February 2013, we initiated a randomized, double-blind, placebo-controlled 15-month Phase 3 study of ISIS-TTRRx in patients with FAP. In this study, we are evaluating the efficacy of ISIS-TTRRx by measuring neurological dysfunction and quality of life in patients with FAP. We already have patients who have completed all fifteen months of treatment and are currently receiving ISIS-TTRRx in an open-label extension study. Our partner, GSK, also plans to evaluate ISIS-TTRRx in patients with FAC and is in the planning stages of a Phase 3 study for this indication.

Visit our TTR Study website »

ISIS-SMNRx

ISIS-SMNRx is an antisense drug we discovered in collaboration with Dr. Adrian R. Krainer at Cold Spring Harbor Laboratory. We designed ISIS-SMN Rx to treat SMA, a severe motor-neuron disease that is the leading genetic cause of infant mortality. SMA affects approximately 30,000 to 35,000 patients in the United States, Europe and Japan. One in 50 people, approximately six million people in the United States, carry the gene mutation that causes SMA. Carriers experience no symptoms and do not develop the disease. When both parents are carriers, however, there is a one in four chance that their child will have SMA. SMA is caused by a loss of, or defect in, the survival motor neuron 1, or SMN1, gene leading to a decrease in the protein, survival motor neuron, or SMN. SMN is critical to the health and survival of nerve cells in the spinal cord that are responsible for neuro-muscular growth and function. The severity of SMA correlates with the amount of SMN protein. Infants with Type I SMA, the most severe life-threatening form, produce very little SMN protein and have a significantly shortened life expectancy. Children with Type II and Type III SMA have greater amounts of SMN protein and have less severe, but still life-altering, forms of SMA. The FDA granted Orphan Drug Designation with Fast Track Status to ISIS-SMNRx for the treatment of patients with SMA.

In January 2012, we and Biogen Idec entered into a preferred partner alliance that provides Biogen Idec an option to develop and commercialize ISIS-SMNRx. We designed ISIS-SMNRx to potentially treat all types of childhood SMA by altering the splicing of a closely related gene, SMN2, which leads to the increased production of fully functional SMN protein. We developed a biomarker assay to measure levels of SMN protein in the cerebral spinal fluid of children and infants with SMA. In February 2014, we reported the first set of data using this biomarker assay. Using this assay, we observed dose-dependent increases in SMN protein levels in children with SMA treated with ISIS-SMNRx from both the single- and multiple-dose studies. In the single-dose study, SMN protein levels more than doubled in the two highest dose cohorts, 6 mg and 9 mg, with average increases of approximately 120 percent and 160 percent compared to baseline, respectively, approximately nine to 14 months after dosing. Similarly, in the multiple-dose study, we observed substantial increases in SMN protein levels in the 9 mg cohort of 115 percent compared to baseline approximately three months after the first dose. In October 2014, we reported results from an analysis of spinal cord tissue samples from autopsies showing that ISIS-SMNRx is distributed throughout the central nervous system. The results of these analyses also showed greater levels of full length SMN2 mRNA and full length SMN protein in tissues in ISIS-SMNRx-treated SMA infants compared to the levels of SMN2 mRNA and full length SMN protein in the tissues analyzed from untreated SMA infants.

We are evaluating ISIS-SMNRx in a Phase 2 open-label, multiple-dose, dose-escalation study in children with SMA. We reported an update on this study in October 2014 at the World Muscle Society Congress with a data cut off of September 2, 2014. In this study, we measured changes in muscle function scores using the Hammersmith Functional Motor Scale-Expanded, or HFMSE, in children treated with multiple doses of ISIS-SMNRx. We reported that children in the 3 mg, 6 mg and 9 mg cohorts achieved mean increases in muscle function scores from baseline of 1.7, 3.2 and 2.3, respectively, eight to 13 months after last dose. These data are consistent with previously reported HFMSE scores for these children nine months after their last dose, which were reported in April 2014. We also reported that increases in muscle function scores were observed eight to 13 months after last dose in the six-minute walk test, or 6MWT, and the upper limb mobility, or ULM, test. In the 6MWT, performed with 10 ambulatory children, a mean increase of 24.4 meters was observed 12 to 16 months after the patients' baseline visits, compared to the previously reported increase of 22.7 meters at nine months. In the ULM test, a mean increase of 3.1 points was observed 11 to 16 months after the patients' baseline visits, compared to the previously reported increase of 2.3 points at nine months.

We are also evaluating ISIS-SMNRx in a Phase 2 open-label, multiple-dose, dose-escalation study in 20 infants who have been diagnosed with SMA. We reported an update on this study in October 2014 with a data cut off of September 2, 2014. We reported a median event-free age of 16.3 months in the infants in the 6 mg cohort. For the infants in the 12 mg cohort, which began dosing five months after the initiation of dosing for the 6 mg cohort, we reported a median event-free age of 11.6 months. These data compared favorably to the natural history of infants with SMA as published by the Pediatric Neuromuscular Clinical Research Network, or PNCR, in the journal Neurology. As reported in October 2014, there had been four events (one permanent ventilation and three deaths, all related to respiratory infections) in the 16 infants in the 12 mg cohort and two events (one permanent ventilation and an accidental death) in the four infants in the 6 mg cohort. We also observed increases in muscle function scores in infants from both dose cohorts.

The safety and tolerability profile of ISIS-SMNRx to date supports continued development. The lumbar puncture procedure in SMA infants and children has been well tolerated and shown to be feasible. Furthermore, as of September 2014, we had administered a total of 250 intrathecal doses of ISIS-SMNRx, and the procedure was well tolerated. In all infants and children dosed, there have been no drug-related serious adverse events, or SAEs. Most of the adverse events, or non-SAEs, have been mild or moderate in severity and not related to drug. There were no changes in the safety profile with repeated doses of ISIS-SMNRx.

We are evaluating ISIS-SMNRx in two Phase 3 studies in infants and children with SMA. We designed these studies to support marketing registration for ISIS-SMNRx in the United States and Europe. The Phase 3 study, ENDEAR, is a randomized, double-blind, sham-procedure controlled 13-month study in approximately 110 infants with SMA. In this study, we are evaluating the efficacy of ISIS-SMNRx by measuring the time to permanent ventilation or survival. The Phase 3 study, CHERISH, is a randomized, double-blind, sham-procedure controlled 15-month study in approximately 117 children with SMA. In this study, we are evaluating the efficacy of ISIS-SMNRx by measuring changes in muscle function scores. We initiated both of these studies in 2014.

We acknowledge support from the following organizations for this program: Muscular Dystrophy Association, SMA Foundation, and Families of Spinal Muscular Atrophy. We have licensed intellectual property from Cold Spring Harbor Laboratory and the University of Massachusetts Medical School.

More information on SMA can be found at:
SMA Foundation: www.smafoundation.org
Cure SMA: www.curesma.org

ISIS-APOCIIIRx

ISIS-APOCIIIRx is an antisense drug we designed to reduce apoC-III protein production and lower triglycerides. ApoC-III regulates triglyceride metabolism in the blood and is an independent cardiovascular risk factor. The fact that people who have certain mutations in the gene for apoC-III that result in lower levels of apoC-III have lower levels of triglycerides and lower instances of cardiovascular disease supports our approach. Also, people with elevated levels of apoC-III have increased dyslipidemia associated with multiple metabolic abnormalities, such as insulin resistance and/or metabolic syndrome. In addition, people with elevated triglycerides are at increased risk for type 2 diabetes, and people with severely elevated triglycerides are at high risk for acute pancreatitis and other serious conditions. Results from our studies support our continued advancement of ISIS-APOCIIIRx.

ISIS-APOCIIIRx is the most advanced drug in our lipid franchise. We plan to transition the development of ISIS-APOCIIIRx to Akcea, our wholly owned subsidiary, which is also responsible for conducting commercial activities. ISIS-APOCIIIRx is in development to treat patients with partial lipodystrophy and patients with FCS. Both partial lipodystrophy and FCS are rare orphan diseases, and each one affects approximately one to two out of a million people. Patients with partial lipodystrophy have diabetes and other metabolic abnormalities, including elevated triglycerides, which increases their risk of pancreatitis. We believe that the robust triglyceride reduction and the improvements in glucose control we observed in our Phase 2 program support our evaluation of ISIS-APOCIIIRx in this patient population. FCS patients often have triglyceride levels higher than 2,000 mg/dL and experience a number of health problems such as recurrent acute pancreatitis that often requires hospitalization, abdominal pain, and enlargement of the liver and spleen. We believe that the significant unmet medical need for an effective triglyceride-lowering drug for patients with FCS and partial lipodystrophy and the robust, consistent effects we observed with ISIS-APOCIIIRx should enable us to rapidly move this program forward toward the market.

In preclinical studies, ISIS-APOCIIIRx diminished signs of metabolic syndrome and reduced atherosclerosis in mice. In a Phase 1 study in healthy volunteers, ISIS-APOCIIIRx produced rapid, dose-dependent median reductions in blood of up to 78 percent in apoC-III protein levels and up to 44 percent in triglyceride levels.

We completed a broad Phase 2 program evaluating ISIS-APOCIIIRx in patients with high, very high, and severely high triglycerides, in patients with type 2 diabetes and in patients with FCS. We also evaluated ISIS-APOCIIIRx both as a single agent and in combination with fibrates. Patients in our Phase 2 program entered with baseline triglyceride levels ranging from moderately high to severely high. In all patient groups treated with ISIS-APOCIIIRx, irrespective of their incoming triglyceride levels, we observed consistent reductions in apoC-III, triglycerides and apoC-III-associated very low-density lipoprotein, or VLDL, complexes, and increased HDL, with a positive effect on non-HDL. Data from the 300 mg/week dose from each of these studies are summarized in the table below.

Table 1: Comparison of the effects on key lipids for patients treated with 300 mg/week of ISIS-APOCIIIRx

  Single Agent in Diabetics with High TG Single Agent in Very High TG In Addition to Fibrates in Very High TG Single Agent in FCS
Baseline Mean mg/dL (range)
ApoC-III 14 (9-20) 23 (14-33) 18 (12-30) 25 (19-35)
Triglycerides 260 (167-361) 559 (291-952) 394 (224-932) 1844 (1406-2083)
HDL-C 41 (29-55) 34 (22-52) 34 (14-53) 13 (8-16)
Non-HDL-C 172 (128-300) 175 (76-312) 185 (118-243) 262 (214-327)
Mean % Change from Baseline (Standard Deviation)
ApoC-III -88% (5.4) -80% (9.3) -71% (13.0) -81% (9.8)
Triglycerides -69% (10.1) -71% (14.1) -64% (8.9) -69% (15.6)
HDL-C +42% (32.2) +46% (24.0) +52% (23.7) +78% (74.6)
Non-HDL-C -22% (18.5) -11% (38.3) -19% (28.8) -58% (14.3)

The safety and tolerability profile of ISIS-APOCIIIRx supports continued development. The most common adverse event was injection site reactions, which were predominantly mild and typically resolved rapidly. There were no flu-like symptoms, no treatment-related elevations in liver enzymes greater than three times the upper limit of normal, no abnormalities in renal function and no clinically meaningful changes in other laboratory values.

In December 2014, we published results from the Phase 2 study of ISIS-APOCIIIRx in patients with FCS in the New England Journal of Medicine.

We are evaluating ISIS-APOCIIIRx in a Phase 3 study in patients with FCS. The Phase 3 study, APPROACH, is a randomized, double-blind, placebo-controlled 52-week study in approximately 50 patients with FCS. In this study, we are evaluating the efficacy of ISIS-APOCIIIRx by measuring the percent change in fasting triglycerides from baseline after three months of dosing. We plan to initiate a Phase 3 study in patients with partial lipodystrophy in 2015. We are designing this study to support a regulatory filing for ISIS-APOCIIIRx in patients with partial lipodystrophy. In early 2015, we initiated a second Phase 3 study, COMPASS, in patients with triglycerides greater than 500 mg/dL. We are designing this study to provide additional clinical experience and safety data to support our regulatory filings for FCS and partial lipodystrophy.

ATL1103

ATL1103 is an antisense drug that targets the 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, an abnormal growth disorder of organs, face, hands and feet. IGF-1 also contributes to diabetic retinopathy, a common disease of the eye and a leading cause of blindness, diabetic nephropathy of the kidney and certain forms of cancer. In preclinical studies, ATL1103 demonstrated significant reductions in IGF-1 levels in the blood and inhibition of neovascularization, or new blood vessels, in the eye in a mouse retinopathy model.

Antisense Therapeutics Limited, or ATL is developing ATL1103 and has completed a Phase 1 study in healthy volunteers demonstrating that ATL1103 was safe and well tolerated. ATL has also completed a Phase 2 study of ATL1103 in patients with acromegaly. In September 2014, ATL reported results from this study showing a statistically significant average reduction in serum IGF-I levels of 26 percent from baseline at week 14 with the 400 mg per week dose, the highest dose tested. ATL reported that ATL1103 was generally well tolerated in the study. The most common adverse event was injection site reactions, which were predominantly mild and typically resolved within days. ATL plans to initiate a small study at a higher dose than 400 mg per week.

ISIS-DMPK-2.5Rx

ISIS-DMPK-2.5Rx, formerly ISIS-DMPKRx, is a generation 2.5 antisense drug we designed to correct the underlying genetic defect that causes Myotonic Dystrophy Type 1, or DM1. DM1 is a rare genetic neuromuscular disease primarily characterized by progressive muscle atrophy, weakness and myotonia. DM1 is the most common form of muscular dystrophy in adults and affects approximately 150,000 patients in the United States, Europe and Japan. Patients with DM1 have a genetic defect in their DMPK, or dystrophia myotonica-protein kinase, gene in which a sequence of three nucleotides repeats extensively, creating an abnormally long RNA, which becomes toxic as it accumulates in the nucleus of cells and prevents the production of proteins needed for normal cellular function. The number of triplet repeats increases from one generation to the next, resulting in the possibility of more severe disease in each subsequent generation. There are currently no disease-modifying therapies that address the disease. The FDA granted Orphan Drug Designation to ISIS-DMPK-2.5Rx for the treatment of patients with DM1.

In 2012, we and Biogen Idec entered into an alliance that provides Biogen Idec an option to develop and commercialize ISIS-DMPK-2.5Rx. We designed ISIS-DMPK-2.5Rx to target DMPK and reduce the toxic DMPK RNA in the cells. In preclinical studies, we showed that an antisense compound targeting the DMPK messenger RNA, or mRNA, entered muscle cells and significantly reduced the toxic RNA. Effective reduction of toxic RNA led to a reversal of the disease symptoms that was sustained for up to one year after treatment in a mouse model of DM1. By removing toxic RNA, ISIS-DMPK-2.5Rx could be an effective approach to treating patients with DM1.

We are evaluating ISIS-DMPK-2.5Rx in a randomized, placebo-controlled, dose-escalation Phase 1/2 clinical study in patients with DM1.

ISIS-GCCRRx

ISIS-GCCRRx is an antisense drug we designed to target the glucocorticoid receptor, or GCCR. Glucocorticoid hormones affect a variety of processes throughout the body, and excessive levels of glucocorticoid hormones can have a detrimental effect on many of the tissues and organs in the body. Cushing's Syndrome is an orphan disease caused by prolonged exposure to high levels of glucocorticoids. If untreated, patients with Cushing's Syndrome can develop hypertension, diabetes and impaired immune functions and have an increased risk of early death. Although there are approved treatments for Cushing's Syndrome, current medicines are associated with significant side effects, such as hypertension and diabetes, and there remains a high unmet medical need for new therapies for these patients. We have already demonstrated that subjects tolerated ISIS-GCCRRx well in a Phase 1 study in healthy volunteers.

ISIS-PKKRx

ISIS-PKKRx is an antisense drug we designed to inhibit the production of prekallikrein, or PKK, a protein produced in the liver that plays an important role in the activation of inflammatory mediators associated with acute attacks of hereditary angioedema, or HAE. HAE is a rare genetic disease that is characterized by rapid and painful attacks of inflammation in the hands, feet, limbs, face, abdomen, larynx and trachea. HAE affects approximately 20,000 patients in the United States and Europe and can be fatal if swelling occurs in the larynx. In patients with frequent or severe attacks, doctors may use prophylactic treatment approaches to prevent and reduce the severity of HAE attacks. However, current prophylactic treatment approaches are very limited and have major tolerability issues due to challenging administration requirements leaving patients with few therapeutic options. By inhibiting the production of PKK, ISIS-PKKRx could be an effective prophylactic approach to preventing HAE attacks.

We have completed a Phase 1 study evaluating ISIS-PKKRx in healthy volunteers in which we observed up to 95% reduction of PKK. In this study, ISIS-PKKRx was generally well tolerated.

ISIS-HTTRx

ISIS-HTTRx is an antisense drug we designed to correct the underlying genetic defect that causes Huntington’s disease (HD). HD is an inherited genetic brain disorder that results in the progressive loss of both mental faculties and physical control. It is caused by the expansion of the CAG trinucleotide sequence in the huntingtin (HTT) gene. The resulting mutant HTT protein is toxic and gradually damages neurons. Symptoms usually appear between the ages of 30 and 50, and worsen over a 10 to 25 year period. Ultimately, the weakened individual succumbs to pneumonia, heart failure or other complications. Presently, there is no effective treatment or cure for the disease, and current available medicines only mask the patient’s symptoms but do not slow down the underlying loss of neurons.

In April 2013,we and Roche entered into an alliance to develop treatments for HD based on our antisense technology and utilizing Roche’s scientific expertise in developing neurodegenerative therapeutics. Under the agreement, we are responsible for all research and development through the first Phase 1 study. Roche has the option to license the program through the completion of the first Phase 1 study and thereafter assume worldwide development, regulatory and commercial responsibilities. We will receive milestone payments from Roche as ISIS-HTTRx advances through development.

ISIS-HTTRx is designed to block production of all forms of HTT protein. As such, ISIS-HTTRx has the potential to treat all HD patients. In preclinical studies, we demonstrated an antisense compound targeting HTT mRNA produced a significant reduction in HTT mRNA and protein, delayed disease progression and prolonged the survival in a mouse model of HD. By reducing the expression of HTT protein, ISIS-HTTRx represents effective potentially comprehensive approach to treating patients with HD.

ISIS-BIIB3Rx

ISIS-BIIB3Rx is an antisense drug to an undisclosed target designed to treat a neurodegenerative disease. ISIS-BIIB3Rx is the third clinical program from our partnership with Biogen Idec. We are eligible to receive milestone payments from Biogen Idec as ISIS-BIIB3Rx advances in development. Biogen Idec has the option to license ISIS-BIIB3Rx from us for a license fee. We are also eligible to receive double-digit royalties from sales of ISIS-BIIB3Rx.

ISIS-BIIB4Rx

ISIS-BIIB4Rx is an antisense drug to an undisclosed target designed to treat a neurodegenerative disease. ISIS-BIIB4Rx is the third clinical program from our partnership with Biogen Idec. We are eligible to receive milestone payments from Biogen Idec as ISIS-BIIB4Rx advances in development. Biogen Idec has the option to license ISIS-BIIB4Rx from us for a license fee.

RG-012

RG-012 is a preclinical drug candidate Regulus is developing, which is an anti-miR that targets microRNA-21, or miR-21. Regulus reports that subcutaneous administration of RG-012 significantly decreased the rate of renal fibrosis and increased the lifespan of mice treated with RG-012 up to 50 percent in a mouse model of Alport Syndrome. Alport Syndrome is a life-threatening disease in which patients experience progressive loss of kidney function.

Regulus currently plans to develop RG-012 to proof-of-concept. At that stage of development, Regulus’ partner Sanofi has an exclusive option to license. We are eligible to receive a portion of all milestone payments and royalties Regulus receives from Sanofi if Sanofi chooses to exercise its option to license RG-012 from Regulus and RG-012 advances in development.

ISIS-RHO-2.5Rx

ISIS-RHO-2.5Rx is an antisense drug designed to treat autosomal dominant retinitis pigmentosa. ISIS-RHO-2.5Rx is the second drug in our collaboration with GSK to enter development to treat an ocular disease (ISIS-GSK4-LRx is the first). We are eligible to receive milestone payments from GSK as ISIS-RHO-2.5Rx advances in development. GSK has the option to license ISIS-RHO-2.5Rx from us for a license fee. We are also eligible to receive double-digit royalties on sales of ISIS-RHO-2.5Rx.

ISIS-GHR-LRx

ISIS-GHR-LRx is in preclinical development. We are currently conducting studies on ISIS-GHR-LRx to support clinical development.

About Isis

We know that sick people depend on us, and we have the technology to change the way diseases are treated.

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Mission & Values

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

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TTR Study

Isis Pharmaceuticals is currently enrolling a Phase 3 study that is designed to evaluate the safety and efficacy of ISIS-TTRRx in patients with Familial Amyloid Polyneuropathy (FAP), the form of TTR amyloidosis that causes nerve damage.

Learn More About the Study