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Jim Cramer mention ISIS as "Buy" at the top of "Mad Money" show today!
Antisense therapy for hepatitis C virus infection -Commentary
Download the PDF here
http://www.natap.org/2013/HCV/011314_07.htm
Journal of Hepatology January 2014
Ype P. de Jong1,2, Ira M. Jacobson1,
1Division of Gastroenterology and Hepatology, Center for the Study of Hepatitis C, Weill Cornell Medical College, New York, NY, USA; 2Laboratory of Virology and Infectious Disease, Center for the Study of Hepatitis C, The Rockefeller University, New York, NY, USA
COMMENTARY ON:
Treatment of HCV infection by targeting microRNA. Janssen HL, Reesink HW, Lawitz EJ, Zeuzem S, Rodriguez-Torres M, Patel K, van der Meer AJ, Patick AK, Chen A, Zhou Y, Persson R, King BD, Kauppinen S, Levin AA, Hodges MR. N Engl J Med. 2013 May 2;368(18):1685-94. doi: 10.1056/NEJMoa1209026. Copyright 2013. Abstract reprinted by permission from the Massachusetts Medical Society.......http://www.ncbi.nlm.nih.gov/pubmed/23534542
NEJM- Treatment of HCV Infection by Targeting
MicroRNA......http://www.natap.org/2013/HCV/033013_04.htm
3 commentaries/ background on microRNA Miravirsen.
(1) Current and Future Therapies for Hepatitis C Virus Infection, T. Jake Liang, M.D., and Marc G. Ghany, M.D., M.H.Sc., N Engl J Med 2013; 368:1907-1917May 16, 2013DOI: 10.1056/NEJMra1213651
(2) Editorial, Micromanaging Hepatitis C Virus, Judy Lieberman, M.D., Ph.D., and Peter Sarnow, Ph.D., N Engl J Med 2013; 368:1741-1743May 2, 2013DOI: 10.1056/NEJMe1301348
(3) Correspondence, HCV Infection and Miravirsen, N Engl J Med 2013; 369:877-878August 29, 2013DOI: 10.1056/NEJMc1307787
Abstract. Background: The stability and propagation of hepatitis C virus (HCV) is dependent on a functional interaction between the HCV genome and liver-expressed microRNA-122 (miR-122). Miravirsen is a locked nucleic acid-modified DNA phosphorothioate antisense oligonucleotide that sequesters mature miR-122 in a highly stable heteroduplex, thereby inhibiting its function. Methods: In this phase 2a study at seven international sites, we evaluated the safety and efficacy of miravirsen in 36 patients with chronic HCV genotype 1 infection. The patients were randomly assigned to receive five weekly subcutaneous injections of miravirsen at doses of 3 mg, 5 mg, or 7 mg per kilogram of body weight or placebo over a 29-day period. They were followed until 18 weeks after randomization.
Results: Miravirsen resulted in a dose-dependent reduction in HCV RNA levels that endured beyond the end of active therapy. In the miravirsen groups, the mean maximum reduction in HCV RNA level (log10 IU per milliliter) from baseline was 1.2 (P = 0.01) for patients receiving 3 mg per kilogram, 2.9 (P = 0.003) for those receiving 5 mg per kilogram, and 3.0 (P = 0.002) for those receiving 7 mg per kilogram, as compared with a reduction of 0.4 in the placebo group. During 14 weeks of follow-up after treatment, HCV RNA was not detected in one patient in the 5-mg group and in four patients in the 7-mg group. We observed no dose-limiting adverse events and no escape mutations in the miR-122 binding sites of the HCV genome.
Conclusions: The use of miravirsen in patients with chronic HCV genotype 1 infection showed prolonged dose-dependent reductions in HCV RNA levels without evidence of viral resistance. (Funded by Santaris Pharma; ClinicalTrials.gov number, NCT01200420.)
MicroRNAs are small non-coding RNAs encoded by the human genome that transcriptionally and post-transcriptionally modify gene expression. The microRNA-122 (miR-122) forms the dominant microRNA in the liver and is exclusively expressed in hepatocytes. It has been implicated in multiple different processes, including lipid metabolism, cell differentiation, iron metabolism and hepatic circadian regulation [1]. In 2005 Jopling and colleagues identified miR-122 as an essential co-factor for hepatitis C virus (HCV) replication [2]. The 5' untranslated region (UTR) of HCV is highly conserved across genotypes and contains two miR-122 binding sites, disruption of which blocks HCV replication through unknown mechanisms [3]. Miravirsen, a 15 nucleotide long oligonucleotide complementary to miR-122, can form stable heteroduplexes with miR-122 and interfere with HCV replication. Whether miravirsen exerts its antiviral effects predominantly through sequestration of available miR-122, indirectly through disrupting lipid pathways essential to the viral lifecycle, or through other mechanisms remains under active investigation. Its efficacy against chronic HCV infection was first shown in studies in chimpanzees, the only natural HCV animal model. Chimpanzees that received the highest, 5 mg/kg, dose through a weekly infusion had a marked decrease in plasma and liver HCV RNA [4], which led to clinical testing of miravirsen. Now Janssen and colleagues report on their findings from a phase 2a study in treatment naive non-cirrhotic patients chronically infected with HCV genotype 1 [5]. They enrolled 36 patients who were randomized to 5 weekly subcutaneous injections with three different doses of miravirsen (3, 5 or 7 mg/kg) or placebo, with the possibility of pegylated interferon (PegIFN) and ribavirin (RBV) rescue at defined time points after miravirsen completion and at the investigator's discretion. After the last injection of miravirsen, patients were followed for an additional 14 weeks for viral kinetics and adverse events. They found that HCV RNA showed a dose-dependent decline, with 1 (11%) patient in the 5 mg/kg and 4 (44%) patients in the 7 mg/kg groups reaching undetectable HCV RNA levels, all after the fifth dose of miravirsen. Notably, the individual response curves shown by the authors were quite variable, even with the highest dose. Three of the patients whose HCV RNA became undetectable relapsed 4-5 weeks later and one patient went on to be treated with PegIFN/RBV. The long term outcome in the remaining patient who achieved an undetectable HCV RNA at study week 14 and remained undetectable through week 18 was not reported. Adverse events were generally mild with only injection site reactions being likely related to miravirsen administration.
Treatment for chronic HCV, which until recently was plagued by poor tolerability and suboptimal response rates, is undergoing a profound paradigm shift with the development of HCV protease, polymerase and NS5A inhibitors and the demonstration of extremely high rates of sustained virologic response with interferon-free combination regimens. Although DAAs currently under clinical investigation generally have high potency, all but nucleotide polymerase inhibitors have low barriers to resistance and therefore combination therapy appears necessary for most HCV genotypes. In contrast to the rapid decline in HCV RNA level within days after starting potent DAAs, there was a <2 log decline at week 4 even with the highest dose of miravirsen. Given our lack of understanding how HCV utilizes miR-122 in its life cycle [6] and by what mechanisms miravirsen blocks HCV replication, it is difficult to account for the different kinetics. Apart from being amongst the first successful antisense oligonucleotide therapies in man, the current study by Janssen and colleagues is notable for additional reasons. First, the strategy of targeting miR122 is distinct from that underlying the three major DAA classes under investigation; in theory, it can be used complementary to DAAs. Similar to strategies that interfere with cyclophilin A [7], miravirsen is an example of the capacity for an inhibitor of a host factor essential for the HCV life cycle to abrogate viremia with no evidence of viral escape. Although theoretical escape mutants can be engineered in vitro [8], the high barrier to resistance and the duration of post-treatment viral suppression in some patients (to the end of study in one patient), imply the possibility that miravirsen could lead to viral eradication with a monotherapy. To address this ambitious proposition, the authors mention that longer duration studies with 12 weeks of miravirsen monotherapy are ongoing. Finally, given the highly conserved 5' UTR and miravirsen's ability to block replication of all HCV genotypes in vitro [8], it is likely that the current results with genotype 1 patients will hold true across genotypes. This may make therapy with miravirsen a potential approach for genotypes that may be less susceptible to some of the DAAs in development.
Its attractive features and favorable short-term safety profile notwithstanding, antisense therapy warrants a note of caution. MiR-122 modulates the expression of an estimated 200 hepatocyte proteins, some of which have been implicated in cholesterol metabolism and cancer development. Although not confirmed in the setting of HCV infection, low miR-122 levels expressed in hepatocellular carcinomas (HCCs) appear to predict a poor prognosis [9], as acknowledged by the authors, and decrease susceptibility to sorafenib in cell culture[10]. Furthermore germline deletion of miR-122 was recently shown to lead to steatohepatitis and spontaneous HCC development in mice [11], [12]. The lower serum cholesterol levels observed in patients after miravirsen administration [5] illustrate that other miR-122 targets are also affected during therapy. In a population at increased risk for developing HCC, these experimental findings warrant careful scrutiny during further clinical development.
The potential benefits of miravirsen must be weighed in the context of the very high rates of sustained virologic response in recent studies of oral DAA combination regimens. The requirement for parenteral administration is a potential drawback, but this may be mitigated if the authors prove to be correct in their suggestion that the pharmacokinetic profile of the drug makes once monthly dosing feasible. Overall, the results of Janssen et al. represent an intriguing proof of concept for a new class of host factor antagonists that combine antiviral potency with a high barrier to resistance. The formulation of a developmental pathway for miravirsen may prove to be challenging in the current environment, but it deserves further study and could have a therapeutic role, particularly if DAA combinations leave unmet needs in some patient populations.
... agreed these guys are hacking the healthcare industry ...
Isis is about to go big
7:08AM ISIS Pharm earns $10 mln milestone payment from Biogen Idec (BIIB) for advancement of ISIS-DMPK Rx to treat myotonic dystrophy (ISIS) 32.41 : Co announces that it has earned a $10 mln milestone payment from Biogen (BIIB) related to the selection and advancement of ISIS-DMPKRx to treat myotonic dystrophy type I.
Strategic Alliance Update:
In June 2012, Isis entered into an alliance with Biogen Idec to discover and develop an antisense drug targeting DMPK for the treatment of DM1. Under the terms of the agreement, Isis received an upfront payment of $12 mln and is eligible to receive up to $59 mln in milestone payments associated with the clinical development of ISIS-DMPKRx, including this $10 mln milestone payment.
Biogen Idec has the option to license ISIS-DMPKRx from Isis up through completion of the Phase 2 study. Isis could receive up to another $200 mln in a license fee and regulatory milestone payments plus double-digit royalties on sales of ISIS-DMPKRx. Isis is responsible for global development of ISIS-DMPKRx through completion of Phase 2 clinical studies, with Biogen Idec providing advice on the clinical study design and regulatory strategy. If Biogen Idec exercises its option, it will assume global development, regulatory and commercialization responsibilities.
7:01AM ISIS Pharm earns $7 mln in milestone payments from GSK for advancement of ISIS-GSK3 Rx to treat viral infection (ISIS) 36.07 : Co announced that GlaxoSmithKline has added a development candidate ISIS-GSK3Rx, to its collaboration with Isis. Isis earned $7 mln in milestone payments associated with the advancement of this program. ISIS-GSK3Rx is an antisense drug designed to inhibit the production of an undisclosed target to treat a common viral infection. Isis will develop ISIS-GSK3Rx to Phase 2 proof-of-concept, after which GSK has an exclusive option to in-license the program and further develop and commercialize the asset.
ISIS @ Leerink-Swann-Rare-Disease-Roundtable
transcript here
http://www.earningsimpact.com/Transcript/83912/ISIS/Isis-Pharmaceuticals%2c-Inc----2013-Leerink-Swann-Rare-Disease-Roundtable
Isis advances Phase 2 Study For Infants With Spinal Muscular Atrophy http://www.biotechpicklist.com/isis-advances-phase-2-study-for-infants-with-spinal-muscular-atrophy/
1:29AM ISIS Pharm reports Phase 2 data on ISIS-APOCIII Rx Showing substantial reductions of triglycerides and Apoc-III in patients with familial chylomicronemia (ISIS) 36.06 : Co announces data from a Phase 2 study of ISIS-APOCIIIRx as a monotherapy in patients with familial chylomicronemia syndrome, or FCS. FCS is a rare orphan disease characterized by severely high triglyceride levels that affects an estimated 3,000 to 5,000 patients worldwide.
In this study, three patients with FCS treated with ISIS-APOCIIIRxachieved substantial reductions in triglycerides with all three patients achieving a triglyceride level below 500 mg/dL, which substantially reduces the risk of an acute pancreatic event. Patients also achieved substantial reductions in apoC-III and apoCIII-associated very low-density lipoprotein-cholesterol particles.
7:05AM ISIS Pharm reports follow-up data from iSIS-SMN Rx Phase 1 study in children with spinal muscular atrophy; improvements in muscle function continue to be observed up to fourteen months after a single dose (ISIS) 31.80 : Co announced today that follow-up preliminary data from a single dose, open-label Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy, show that most SMA children receiving the two highest doses of the drug continued to show improvements in muscle function tests up to 14 months after a single injection of the drug. The Phase 1 data, including these preliminary follow-on data, will be presented at the International Congress of the World Muscle Society by Dr. Kathy Swoboda on Oct. 3, 2013. SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality. ISIS-SMNRx is an antisense drug designed to treat all types of SMA.
The preliminary data reported today is from a follow-up analysis of 24 children with SMA who participated in a Phase 1 single-dose, open-label study of ISIS-SMNRx. Analysis of motor function was performed in these children nine to 14 months following a single dose of ISIS-SMNRx using the Hammersmith Functional Motor Scale-Expanded. The improvements in HFSME scores were dose dependent with the largest improvements observed in children in the highest dose cohort. Most children in the 9 mg dose cohort showed continuing improvements during follow up, with no children declining.
Q2-2013-Earnings-Call updates
received $30 million from new collaboration with Roche, $16 million from AstraZeneca and $3.5 million from Biogen Idec
http://www.earningsimpact.com/Transcript/82661/ISIS/Isis-Pharmaceuticals%2c-Inc----Q2-2013-Earnings-Call
7:04AM ISIS Pharm announces patients treated with ISIS-CRPRx achieved rapid, dose-dependent mean reductions of up to 67% in C-reactive protein (CRP) in a Phase 2 study in patients with rheumatoid arthritis (ISIS) 29.23 : Co announced that patients treated with ISIS-CRPRx achieved rapid, dose-dependent mean reductions of up to 67% in C-reactive protein (CRP) in a Phase 2 study in patients with rheumatoid arthritis (RA).
Patients treated with ISIS-CRPRx showed improvements in signs and symptoms of RA; however, these improvements were not statistically significant when compared to those observed in patients in the placebo group, which demonstrated a higher than expected response in both symptom score and CRP reduction. A Phase 2 study of ISIS-CRPRx in patients with atrial fibrillation is currently ongoing with data anticipated in the first half of 2014.
7:06AM ISIS Pharm earns $2 mln from GSK for the advancement of ISIS-TTR Rx (ISIS) 27.79 : Co announced that it has earned a $2 mln milestone payment from GlaxoSmithKline (GSK) related to the advancement of the ongoing Phase 2/3 study of ISIS-TTRRx in patients with familial amyloid polyneuropathy (FAP). Isis has earned $20 million in upfront and milestone payments prior to first patient dosed in the ongoing Phase 2/3 study. The $2 million milestone payment announced today is the first milestone of the $50 million in milestone payments Isis is eligible to earn as the Phase 2/3 study progresses. In addition, if GSK elects to exercise its option to exclusively license the ISIS-TTRRx program, Isis is eligible to receive a license fee, regulatory and sales milestone payments and double-digit royalties on sales of ISIS-TTRRx.
Insightful -- Annual-Meeting-of-Stockholders
read here
http://www.earningsimpact.com/Transcript/82485/ISIS/Isis-Pharmaceuticals%2c-Inc----2013-Annual-Meeting-of-Stockholders
ISIS
Imp updates at Business Update Call
Observation from current study is that ISIS-APOCIIIRx is additive to fibrates
Read here
http://www.earningsimpact.com/Transcript/82416/ISIS/Isis-Pharmaceuticals%2c-Inc----Business-Update-Call
But, Isis gets in bed with Glaxo... and the Chinese just arrested a bunch of monkeys from Glaxo. (I suspect industrial espionage because that just sounds better than “kick backs”).
I seek a cure for amyloids (for my cats). You guys get on that one, cause I think we're going to need it. (it's in man too).
1:31AM ISIS Pharm reports Phase 2 data on ISIS-APOCIII Rx showing significant reductions in triglycerides and APOC-III in patients with high triglycerides and Type 2 diabetes (ISIS) 21.99 : Co reports that data from the Phase 2 study of ISIS-APOCIIIRx in patients with high triglycerides and type 2 diabetes. In this study, patients treated with ISIS-APOCIIIRx experienced an 88 percent reduction in apolipoprotein C-III, a 72% reduction in triglyceride levels, a 40 percent increase in high-density lipoprotein cholesterol, the 'good' cholesterol, and improvements in other atherogenic lipid parameters. In addition, patients dosed with ISIS-APOCIIIRx showed consistent trends toward enhanced insulin sensitivity with improvements in multiple measures of glucose control. Isis is also evaluating ISIS-APOCIIIRx in a separate Phase 2 study in patients with moderate to severe high triglycerides and plans to report data from this study this summer.
7:01AM ISIS Pharm earns $6 mln payment from AstraZeneca (AZN) (ISIS) 20.91 : Co announced that it has earned a $6 mln payment from AstraZeneca related to continuation of the research collaboration between ISIS and AZN to discover and develop novel antisense therapeutics to treat cancer. In addition to the research collaboration, which includes three cancer targets, Isis and AstraZeneca are developing two antisense drugs, ISIS-ARRx and ISIS-STAT3Rx, discovered by Isis and licensed to AstraZeneca for the treatment of cancer.
In December 2012, Isis entered into a collaboration with AstraZeneca to discover and develop antisense drugs to treat cancer. The collaboration combines AstraZeneca's experience and expertise in developing anti-cancer agents with Isis' antisense technology platform to broaden Isis' cancer franchise. With the $6 million payment, Isis has earned $41 million in upfront and milestone payments from AstraZeneca and is eligible to earn additional milestone payments as the collaboration progresses and royalties on sales of drugs resulting from the collaboration.
7:04AM ISIS Pharm earns $10 mln milestone payment from AstraZeneca (AZN) for ISIS-AR Rx to treat prostate cancer (ISIS) 21.69 : Co announces that AstraZeneca has added a second development candidate, ISIS-ARRx, to its collaboration with Isis. Isis earned a $10 mln milestone payment associated with this decision. ISIS-ARRx is an antisense drug designed to treat patients with prostate cancer by inhibiting the production of the androgen receptor. AstraZeneca is planning to develop ISIS-ARRx broadly to treat patients under a variety of settings during the course of prostate cancer treatment, including both as a single agent and in combination therapy.
1:09AM ISIS Pharm prices 9 mln share offering at $19 per share (ISIS) 19.65 :
9:03AM ISIS Pharm: Roche and Isis Pharmaceuticals form alliance for Huntington's disease; Roche will make an upfront payment of $30 million to Isis (ISIS) 17.37 : Roche (RHHBY) and ISIS announced that they have formed an alliance to develop treatments for Huntington's disease (HD) based on Isis' antisense oligonucleotide (ASO) technology. In addition, Isis and Roche will be collaborating to combine Isis' ASOs and Roche's proprietary "brain shuttle" program with the objective of increasing the brain penetration of ASOs with systemic administration.
Under the terms of the agreement, Roche will make an upfront payment of $30 million to Isis, with total payments related to license fee and pre- and post-licensing milestone payments reaching potentially $362 million, including up to $80 million in potential commercial milestone payments.
In addition, Isis will receive tiered royalties on sales of the drugs. Roche has the option to license the drugs from Isis through the completion of the first Phase 1 trial. Prior to option exercise, Isis is responsible for the discovery and development of an antisense drug targeting HTT protein.
Roche and Isis will work collaboratively on the discovery of an antisense drug utilizing Roche's "brain shuttle" program. If Roche exercises its option, it will be responsible for global development, regulatory and commercialization activities for all drugs arising out of the collaboration.
ISIS is halted
Isis Pharmaceuticals initiates Phase 1 study of ISIS-APOARx
Theflyonthewall.com – 1 hour 31 minutes ago
Isis Pharmaceuticals announced that it has initiated a Phase 1 clinical study for ISIS-APOARx, an antisense drug targeting apolipoprotein(a) for the treatment of atherosclerosis. Apolipoprotein contributes to the formation of plaque in arteries through its attachment to an LDL-C particle in a complex termed lipoprotein. High levels of lipoprotein are associated with an increased risk of atherosclerosis, coronary heart disease, heart attack and stroke. ISIS-APOARx is designed to reduce lipoprotein by inhibiting the production of apolipoprotein. Isis plans to develop ISIS-APOARx to treat patients with high Lp(a) levels who are at high risk of experiencing cardiovascular events.
7:02AM ISIS Pharm publishes data demonstrating antisense targeting of ApoC-III significantly reduces ApoC-III and Triglycerides (ISIS) 16.94 : Co announced today the publication of new data in the journal Circulation Research demonstrating that antisense targeting of apolipoprotein C-III resulted in significant reductions in apoC-III and triglycerides, each an independent risk factor for cardiovascular disease. Hypertriglyceridemia is a serious medical condition associated with premature coronary artery disease and an increased risk for pancreatitis.
In the published data, treatment with an antisense compound targeting apoC-III produced a variety of potential cardio-protective effects including significant dose-dependent reductions of apoC-III and triglycerides in all models and species, including man.
1:23AM ISIS Pharm presents data from ISIS-SMN Rx Phase 1 study in children with spinal muscular atrophy (ISIS) 17.84 : Co announces that data from the Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy were presented. In the presentation, ISIS-SMNRx was well tolerated in children with SMA at all dose levels tested and that improvements were observed in Hammersmith scores, a measure of muscle function, in a number of the children.
Co that ISIS-SMNRx was well tolerated when administered intrathecally as a single dose directly into the spinal fluid and that no safety concerns related to the drug were identified. In addition, the intrathecal injection procedure was well tolerated in children with SMA. Concentrations of ISIS-SMNRx measured in cerebral spinal fluid were consistent with levels predicted from preclinical studies, indicating that the drug half-life in nervous system tissues is very long and that dosing once every six to nine months is feasible. Although the study was not designed to provide evidence of functional activity, clinically significant, dose-dependent improvements in the Hammersmith Functional Motor Scale-Expanded, a measure of muscle function, were observed in these children.
8:36AM ISIS Pharm reports narrower than expected loss, beats on revs (ISIS) 14.23 : Reports Q4 (Dec) loss of $0.03 per share, $0.20 better than the Capital IQ Consensus Estimate of ($0.23); revenues fell 38.6% year/year to $19.9 mln vs the $15.29 mln consensus.
"We expect 2013 to be a year of substantial growth and maturation for Isis. We believe that the initial commercial launch of KYNAMRO will be successful and we support Genzyme's ongoing development efforts for KYNAMRO with the FOCUS FH study, which Genzyme is conducting under a special protocol assessment with the FDA... Beyond KYNAMRO, we have a number of drugs in our pipeline that will complete later-stage clinical studies, which are designed to provide clear evidence that these drugs have the potential to work in patients in numerous different diseases. In addition, we plan to advance many other drugs in our pipeline, creating additional opportunities for significant long-term revenue. And finally, we expect to explore partnering opportunities that are the right fit for Isis and designed to provide the most benefit to our programs and drugs, the best balance of risk and commercial participation, while allowing us to continue to do what we do best, drug discovery and early drug development."
8:03AM ISIS Pharm initiates Phase 2 study Of ISIS-CRP Rx In patients with atrial fibrillation (ISIS) 14.02 : Co announced the initiation of a Phase 2 study evaluating ISIS-CRPRx in patients with paroxysmal atrial fibrillation.
7:08AM ISIS Pharm initates Phase 2/3 study Of ISIS-TTR Rx and earns $7.5 million milestone payment from GlaxoSmithKline (GSK) (ISIS) 14.43 : Co announced today that it has earned a $7.5 million milestone payment from GlaxoSmithKline (GSK) related to the initiation of a Phase 2/3 clinical study for ISIS-TTRRx. ISIS-TTRRx is an antisense drug in development with GlaxoSmithKline (GSK) for the treatment of transthyretin amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues. The Phase 2/3 study of ISIS-TTRRx is a randomized, double-blinded, placebo-controlled, international study designed to support an application for marketing approval of ISIS-TTRRx in patients with FAP. The fifteen month study will enroll approximately 200 patients randomized 2:1 to receive 300 mg/week of ISIS-TTRRx or placebo and will measure the effects of ISIS-TTRRx on neurological dysfunction and on quality-of-life. The United States Food and Drug administration granted ISIS-TTRRx fast track designation and orphan drug status for the treatment of FAP.
ISIS PDUFA
Tue, January 29, 12am – Wed, January 30, 12am
Calendar
FDA Calendar
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7:30AM ISIS Pharm and Genzyme, a Sanofi co (SNY), announced that the Committee for Medicinal Products for Human Use of the EMA has adopted a negative opinion for its marketing authorization application for KYNAMRO (ISIS) 9.61 : Isis Pharmaceuticals and Genzyme, a Sanofi co (SNY), announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion for its marketing authorization application (MAA) for KYNAMRO (mipomersen) for the treatment of patients with Homozygous Familial Hypercholesterolaemia (HoFH). Genzyme plans to request a re-examination of the CHMP Opinion. Isis will review the CHMP opinion on KYNAMRO in a conference call and webcast today at 8:30 a.m. Eastern Time.
8:02AM ISIS Pharm and Biogen (BIIB) announce collaboration for antisense programs to treat neurological disorders (ISIS) 8.99 : ISIS and BIIb have entered into a global collaboration agreement under which the cos will discover and develop antisense drugs against three undisclosed targets to treat neurological or neuromuscular disorders. Biogen Idec and Isis are also developing antisense drugs to treat spinal muscular atrophy and myotonic dystrophy type 1 under previously established collaborations. Isis will receive an upfront payment of $30 million and is responsible for the discovery of a lead antisense drug for each of the three undisclosed targets. Isis is eligible to receive substantial development milestone payments to support research and development of each program prior to the exercise by Biogen Idec of its option to license each program. Biogen Idec has the option to license a drug from each of the three programs through the completion of Phase 2 trials. Isis could receive up to another $200 million in a license fee and regulatory milestone payments per program. In addition, Isis will receive double-digit royalties on sales of drugs. Isis will be responsible for development of the drugs through the completion of the initial Phase 2 clinical trial, with Biogen Idec providing advice and assistance on research and the clinical trial design and conduct and regulatory strategy for each program. If Biogen Idec exercises its option, it will assume global development, regulatory and commercialization responsibilities.
8:01AM ISIS Pharm reports 'positive' Phase 1 data on three drugs in development to treat metabolic disorders (ISIS) 9.00 : Co announces positive results from Phase 1 studies of three drugs, ISIS-PTP1BRx, ISIS-GCGRRx and ISIS-GCCRRx, in development to treat metabolic disorders, including type 2 diabetes. In all three Phase 1 studies, the drugs were safe and well tolerated with early encouraging data that supports the unique mechanism of each drug. All three drugs are part of Isis' metabolic franchise and are designed to act through distinct mechanisms to improve insulin sensitivity and/or reduce glucose production. In the Phase 1 study of ISIS-PTP1BRx in obese healthy volunteers, Isis observed encouraging data in measures of insulin sensitivity and in a biomarker associated with weight loss. Previous clinical experience with the PTP-1B inhibitor ISIS 113715 demonstrated that PTP-1B inhibition improved glucose control and reduced LDL-cholesterol without causing weight gain. Additionally, co's Phase 1 study ISIS-GCGRRx displayed a good safety profile with no clinically significant increases in blood pressure or lipids and with no hypoglycemia
Will the Golden Cross do anything?
What do you think? It's about to cross over the 50/200, unless I'm reading the charts wrong. I got in at 8.75 and am thinking I should have waited a bit, but hindsight is 20/20.
Everytime this stock is about to TANK they put out worthless news to get Longs to cover there losses... its pathetic... Im waiting for a major dip to $6 any day now..possibly today.
8:02AM ISIS Pharm to receive $1.25 mln payment as Pfizer (PFE) continues development of EXC 001 (ISIS) 8.92 : Co announced that it will receive a $1.25 mln contingent payment from Pfizer (PFE) triggered by Pfizer's decision to advance EXC 001 into a Phase 2 study. In 2011, Isis received $4.4 mln for its equity ownership of Excaliard from Pfizer's acquisition of Excaliard Pharmaceuticals and, excluding the $1.25 mln, is eligible to receive an additional $8.35 mln in contingent payments upon achievement of various milestones associated with the clinical and commercial progress of EXC 001, also referred to as PF-06473871. Isis also remains eligible to receive milestone and royalty payments under its licensing agreement with Excaliard for EXC 001.
8:08AM ISIS Pharm reports 'encouraging' interim Phase 1 data on ISIS-STAT3Rx in patients with cancer (ISIS) 8.90 : Co announced that preliminary data from the Phase 1 study of ISIS-STAT3Rx in patients with cancer were presented today at the Oligonucleotide Therapeutics Society in Boston. Interim results of the initial dose escalation study in patients with cancer showed that ISIS-STAT3Rx treatment resulted in clear responses in patients with advanced cancer who were refractory to prior chemotherapy treatment, with an acceptable safety profile. Based on these results, Isis has initiated a Phase 2 study in focused patient populations with advanced cancer.
Now that they got a small run theyll DUMP shares on you. They are pumping this stock on mad money & on a measly 1million dollar press release.
They are proping this stock up to unload on the public.
This will quicly hit $6 in 2 weeks........ Buy around $5-6 dollars.
MARKET TALK: Slim Backing for Isis Raises Approval ConcernsFont size: A | A | A
9:25 AM ET 10/19/12 | Dow Jones
9:25 EDT - ISIS may have beat back some naysayers with a narrow win before an FDA advisory panel Thursday for its cholesterol treatment, but ultimate approval still seems far from certain. The 9-6 vote in favor of the drug--for a rare cholesterol disease--came a day after a rival treatment from Aegerion (AEGR) easily won over its panel. Amid concerns about efficacy and potential carcinogenicity, "some panelists admitted their votes were a 'weak yes' and provided multiple reasons why they almost voted no," says Leerink Swann about the ISIS poll. The investment bank now sees $9/share as a fairer valuation for ISIS, down from $12 previously. It's down slightly premarket at $9.58. (mia.lamar@dowjones.com)
Hows that money mande douche? LOL
This stock is Going SouTH-%. Daytraders will dump there position before the weekend.
The vote was a funny vote. Wasnt good enough for the fda..they will require more information.
,,,,,,,A win is a win! SEE YOU IN TEENS--MONEYMADE
1 off.lol.Not convincing enough for FDA. They will need more info to pass..
This is going to Tank
9 yes -6 no
Not very strong IMO
Preliminary ISIS panel vote 10-5 vote AGAINST
INSIDERS ARE DUMPING CAREFUL...
Yesterday's trading wasnt good for most of us but there is still hope for ISIS... at lower levels... 1.2m shares were sold after market. Seem like ISIS wants to see $8 or $9.
ISIS AdCom Thu, October 18, 12am – Fri, October 19, 12am
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Ionis Pharmaceuticals, Inc., through its drug discovery platform based on antisense technology, discovers and develops drugs that bind to RNA antisense technology. The company has commercialized its antisense drug and has 19 drugs in development. Ionis' drug development programs are focused on treating cardiovascular, metabolic, and severe neurodegenerative diseases and cancer. It licenses its drugs to partners prior to late-phase development and commercialization. The company and Alnylam Pharmaceuticals jointly own Regulus Therapeutics Inc., a company focused on the discovery, development, and commercialization of microRNA therapeutics. Ionis is the owner or exclusive licensee of approximately 1,600 issued patents worldwide. The company was founded in 1989 and is based in Carlsbad, California.
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