ionis pharmaceuticals
ionis pharmaceuticals™ is a trademark of ionis pharmaceuticals, inc. akcea therapeutics™ is a trademark of ionis pharmaceuticals, inc .
see what the ihs markit score report has to say about ionis pharmaceuticals inc .
here' s why the best is yet to come for ionis pharmaceuticals, inc .
ionis pharmaceuticals revises 2015 financial guidance and provides pipeline update in conjunction with j. p. morgan conference
, the company changed its name from isis pharmaceuticals, inc. to ionis pharmaceuticals, inc. in this press release, unless the context requires otherwise ,
ionis ,
company ,
we ,
our ,
and
us
refers to ionis pharmaceuticals and its subsidiaries .
here' s why the best is yet to come for ionis pharmaceuticals, inc. - - the motley fool
, chief financial officer of ionis pharmaceuticals .
we expect to end 2015 with a pro forma nol in the low
brian orelli has no position in any stocks mentioned. the motley fool owns shares of and recommends alnylam pharmaceuticals, biogen, and ionis pharmaceuticals. the motley fool has a disclosure policy .
ptc therapeutics spiked wednesday after an analyst suggested it and roche could rout biogen and ionis pharmaceuticals in treating spinal muscular atrophy .
here' s why the best is yet to come for ionis pharmaceuticals, inc. @ themotleyfool # stocks $ ions, $ nvs
the companies, alnylam pharmaceuticals and isis pharmaceuticals, expect to announce today an equally owned joint venture to develop drugs that work by blocking so - called micro - rnas .
there' s also ionis education group based in paris; the ionis art hotel on the island of zakynthos, greece; and ionis international, provider of pacific rim intercultural business training programs .
isis pharmaceuticals has changed its name to ionis pharmaceuticals, the carlsbad biotech company announced friday. with the new name - - free of any similarities to the widely used acronym for the islamic state terrorist group, comes a new stock ticker - - ions .
brian orelli has no position in any stocks mentioned. the motley fool owns shares of and recommends biogen and ionis pharmaceuticals. the motley fool has a disclosure policy .
kastle therapeutics has acquired global rights to develop and commercialize the marketed drug kynamro ® (mipomersen sodium) injection from ionis pharmaceuticals for up to $ 95 million - plus .
brian orelli has no position in any stocks mentioned. the motley fool owns shares of and recommends ionis pharmaceuticals and johnson & johnson. the motley fool has a disclosure policy .
the companies had partnered on nusinersen for sma— one of several collaborations by the companies —since 2012, when ionis was known as isis pharmaceuticals and the sma candidate was called isis - smn rx .
ionis pharmaceuticals (ions) has developed many drugs for the treatment of various life - threatening diseases through its broadly applicable drug discovery platform. as mentioned in the previous part, ionis’s revenue grew 24. 7% yoy (year - over - year) in q1 2018. the chart below shows ionis’s revenue since q1 2017, and analysts’ estimate for q2 2018 .
ionis pharmaceuticals (ions), an rna1 - targeted therapeutics development leader, commercializes products in collaboration with other pharmaceutical companies. in q1 2018, ionis’s revenue grew 24. 7% yoy (year - over - year) to $ 144. 4 million from $ 115. 8 million .
ionis - dmpk - 2. 5rx was being tested in myotonic dystrophy type 1, which is caused by the toxic dmpk rna. like most of ionis' drugs, ionis - dmpk - 2. 5rx was designed to bind to the rna and degrade it. while ionis and its partner biogen saw some signs that the drug was working, it wasn' t enough to justify taking ionis - dmpk - 2. 5rx further into development .
several cure sma grants totaling over $ 500, 000 were given to dr. ravi singh while at u massachusetts, to help develop the intellectual property needed to investigate asos for sma. the intellectual property generated with our funding was then licensed to ionis pharmaceuticals for an sma drug called spinraza (originally called ionis - smnrx, then nusinersen). ionis partnered with biogen to continue developing this drug .
about ionis pharmaceuticals, inc. ionis is the leading company in rna - targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. using its proprietary antisense technology, ionis has created a large pipeline of first - in - class or best - in - class drugs, with over a dozen drugs in mid - to late - stage development. drugs currently in phase 3 development include volanesorsen, a drug ionis is developing and plans to commercialize through its wholly owned subsidiary, akcea therapeutics, to treat patients with familial chylomicronemia syndrome and familial partial lipodystrophy; ionis - ttr rx, a drug ionis is developing with gsk to treat patients with all forms of ttr amyloidosis; and nusinersen, a drug ionis is developing with biogen to treat infants and children with spinal muscular atrophy. ionis' patents provide strong and extensive protection for its drugs and technology. additional information about ionis is available at urltoken .
biogen has exercised its option to develop and commercialize nusinersen following positive interim phase iii results, agreeing to advance the spinal muscular atrophy (sma) candidate in a deal that could generate $ 225 million - plus for ionis pharmaceuticals .
in 2015, ionis added nine new drugs to its pipeline. already in 2016, the company has added two new drugs to its pipeline: ionis - biib5 rx and ionis - biib6 rx. both new drugs are being developed together with biogen and are designed to address undisclosed targets for the treatment of patients with neurodegenerative diseases. ionis currently has a total of 38 drugs in its pipeline .
carlsbad, calif. , june 18, 2018 / prnewswire / - - ionis pharmaceuticals, inc. (ions) today announced that damien mcdevitt, ph. d. has joined the company as chief business officer. just this year, ionis has seen its first commercial drug for a neurological disease, spinraza, reach blockbuster status .
biogen (biib) completed a pact to collaborate with ionis pharmaceuticals (ions), and bought 11. 5 million ionis shares for $ 625 million, paying $ 54. 34 each. biogen also invested $ 375 million as an upfront payment to ionis to
jointly discover and develop novel antisense drugs for a broad range of neurological diseases ,
as stated in a press release. in total, biogen laid out $ 1 billion in cash .
ionis—which changed its name in december 2015 from isis pharmaceuticals—had been seeking a commercialization alternative since january, when it terminated its license agreement with sanofi, a genzyme company, to co - market kynamro: “we’ve been disappointed with the performance of genzyme for quite a while, ” ionis cbo sarah boyce told the san diego union - tribune at the time .
as discussed earlier, ionis pharmaceuticals’ (ions) revenue grew 24. 7% yoy (year - over - year) in q1 2018, and analysts expect it to grow 30. 6% yoy to $ 136. 0 million in q2 2018. the graph below shows ionis’s commercial and r & d (research and development) revenue since q1 2017 .
mid - caps stocks, like ionis pharmaceuticals inc (nasdaq: ions) with a market capitalization of us $ 5. 85b, aren’t the focus of most investors who prefer to direct their investments towards either large - cap or small - capread more ...
the rest of the revenue came from the usual mix of milestone payments and licensing fees from ionis' various partners .
in addition, ionis announced that results from the phase 1 / 2 study with ionis - ar - 2. 5 rx in heavily pretreated, late - stage prostate cancer patients demonstrated encouraging data, including several durable reductions in psa levels. the drug also exhibited a good safety and tolerability profile supportive of continued development. data from this study will be presented later in the year. ionis plans to continue the development of ionis - ar - 2. 5 rx independent of astrazeneca .
the deal marks kastle’s first acquisition since it was formed last year. kastle focuses on acquiring, developing, and commercializing pharmaceuticals targeted toward diseases with high unmet medical needs .
nusinersen is the first antisense drug from ionis’ neurological disease franchise to advance to regulatory review: “it illustrates the potential of our antisense technology to address severe diseases that other therapeutic modalities are unable to address adequately, ” added ionis coo b. lynne parshall .
however, the name has been used in other contexts. ionis is
a level 53 npc ,
( non - player character) according to a world of warcraft fan page. and in another fantasy game, final fantasy xi, ionis is a beneficial magic spell .
next up in the late - stage pipeline, investors will get to see data from ionis - ttrrx, which treats familial amyloid polyneuropathy, sometime this quarter. assuming the data from the phase 3 trial are positive, ionis - ttrrx should be able to get onto the market soon after volanesorsen. positive data could also convince ionis' marketing partner, glaxosmithkline, to test the drug against the cardiomyopathy version of the disease .
biogen has paid ionis a $ 75 million license fee after exercising its option to develop and commercialize nusinersen globally. biogen is now responsible for all nusinersen development, regulatory and commercialization activities, and costs, while ionis will complete the phase iii studies and work with biogen on regulatory filings .
basel, january 06, 2017 - novartis announced today a collaboration and option agreement with ionis pharmaceuticals, inc. and its affiliate akcea therapeutics, inc. , to license two novel treatments with the potential to significantly reduce cardiovascular risk in patients suffering from high levels of lipoproteins known as lp (a) and apociii. the two investigational antisense therapies developed by ionis - called akcea - apo (a) - l rx and akcea - apociii - l rx - have the potential to lower both lipoproteins up to 90% and significantly reduce cardiovascular risk in high - risk patient populations. in addition novartis entered into a stock purchase agreement with ionis .
carlsbad, calif. , june 5, 2018 / prnewswire / - - ionis pharmaceuticals, inc. (ions) announced today that it closed its expanded strategic collaboration with biogen (biib) to discover and develop novel antisense drugs for a broad range of neurological diseases following receipt of clearance under the hart - scott - rodino (hsr) antitrust improvements act of 1976. biogen paid ionis $ 1 billion in cash, which included $ 625 million for the purchase of 11, 501, 153 shares of ionis common stock at a price of $ 54. 34 per share and a $ 375 million upfront payment. on april 20, 2018, ionis and biogen announced they would expand their strategic collaboration to develop novel antisense drugs for a broad range of neurological diseases through a new ten - year collaboration .
kastle agreed to pay ionis $ 15 million upfront, another $ 10 million due may 3, 2019, and up to $ 70 million in payments tied to achieving sales milestones. beginning in 2017, kastle will pay ionis royalties on global sales of kynamro in the mid to low teen percentage range .
the new technology has gotten the backing of ionis' partners. astrazeneca and johnson & johnson are developing drugs with ionis that use the combination of both technologies. bayer recently exercised its option to develop a lica - based drug and the aforementioned deal with novartis was for lica - based drugs as well .
these drugs work through the antisense technology pioneered by ionis. this technology blocks diseases at the genetic level, by blocking or changing the production of proteins .
and ionis recently got approved for spinal muscular atrophy, side effects aren' t an issue because there aren' t any other treatment options at the moment .
a decade of improvements, modifications, and collaboration with antisense therapeutics company isis (now ionis) pharmaceuticals resulted in the development of nusinersen—a drug delivered via spinal injection that is expected to become the first therapeutic for sma. in august, ionis halted its phase 3 trial in sma patients after interim analyses showed that infants receiving a spinal injection of nusinersen were better able to kick, stand, and walk than infants undergoing a sham procedure. “obviously, we’re extremely excited, ” says krainer. “it’s sort of beyond anyone’s most optimistic expectations. ”
however, 3% of the cash payments that ionis receives from kastle—up to $ 2. 85 million—plus a 3% royalty on kynamro sales—will be earned by genzyme .
ionis recently announced plans to spin off its cardiovascular drugs, including volanesorsen and the novartis - partnered drugs, into a separate company called akcea. ionis will get royalties ranging from the mid - teens to the mid - 20s on the drug as well as a 50% cut of the milestone payments and royalties from the novartis partnership .
the two companies said they will also work together to transition the clinical programs that ionis is conducting to biogen. in addition to the license fee, ionis could receive tiered royalties on potential sales of nusineren up to a percentage in the mid - teens, in addition to up to $ 150 million in milestone payments based on regulatory approvals .
for isis pharmaceuticals, a name change was not in consideration as recently as last year. but a company spokesman told cbs news this week that the tragedy “weighed heavily on us, and we don’t want to have that association. ”
and volanesorsen. any statement describing ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward - looking statement and should be considered an at - risk statement. such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. ionis' forward - looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward - looking statements. although ionis' forward - looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by ionis. as a result, you are cautioned not to rely on these forward - looking statements. these and other risks concerning ionis' programs are described in additional detail in ionis' annual report on form 10 - k for the year ended
“the promise is already here, ” john rossi, an rna biologist and cofounder of dicerna pharmaceuticals, wrote in an email to the scientist, adding that he sees “the next few years as becoming the era [ of oligonucleotide therapeutics ]. ”
it seemed to me that everybody came together and decided that ionis was a nice - sounding, feeling name, as soon as someone hit on it ,
crooke said .
in july, but it still owns 70% of the shares after the ipo, so ionis will have to incorporate ackcea' s financials into its own for the foreseeable future .
ionis has two drugs on the market, two more in late - stage development, and a large number of phase 2 drugs that could have proof of concept data shortly. to pay for those clinical trials, ionis phamraceuitcals has partnered many of its drugs while keeping some of the ones for smaller orphan indications where smaller clinical trials can be run relatively cheaply .
alnylam seemingly outperformed rival ionis in tests of drugs to treat a progressive rare disease, an analyst said thursday. both biotech companies are making treatments for a condition known as hereditary attr amyloidosis .
since we know the antisense technology works, it' s likely that the issue is that ionis - dmpk - 2. 5rx isn' t getting into the muscles, where the toxic rna lies. as noted on the slide, ionis and biogen are exploring a ligand conjugated antisense (lica) strategy to target a drug directly to the muscle, which should increase the potency .
in related chmp news, the committee also advised against approval of vanda pharmaceuticals' ($ vnda) fanaptum for treating schizophrenia, with the chmp raising concerns about evidence of marginal efficacy of the drug compared with placebo in studies and heart risks associated with the treatment .
ionis' newest drugs use generation 2. 5 chemistry that can provide up to a 10 - fold increase in potency over the generation 2. 0 version. ionis has also developed a ligand - conjugated antisense (lica) technology that can target drugs to specific tissue. the combination of generation 2. 5 and lica makes for very potent drugs, which can decrease doses and reduce side effects .
ionis still has five unpartnered drugs in its pipeline that it could license to generate more cash and has plans to add three to five drugs this year (although some of those drugs could already be partnered under research collaborations with partners). and, to top it off, royalties income will ramp up as the launch of spinraza continues and volanesorsen and ionis - ttrrx are hopefully launched in the years to come .
over the years - - many, many years - - ionis has improved the technology to increase the drugs' potency through increasing the binding of the drug to the rna and by decreasing degradation of the drug .
ionis is an empty vessel name, crooke said, without any inherent meaning other than what is created by what the company does. patent and other checks indicated that the name was free of legal problems or unwanted associations .
amazingly, despite having about two dozen drugs in the clinic and essentially no royalties from drugs yet, ionis was able to cover all of its expenses through up - front and milestone payments from partners during the fourth quarter .
stanley crooke, the ceo of ionis, is bluntly enthusiastic. “the babies [ with the severest disease ] are alive and the longer we treat the better they seem to get, ” he says. “it’s a miracle. ”
as discussed previously, analysts expect ionis pharmaceuticals’ (ions) revenue to grow yoy (year - over - year) in 30. 6% yoy to $ 136. 0 million in q2 2018. they expect the company to report an adjusted net loss of $ 24. 3 million due to higher sg & a (selling, general, and administrative) and r & d (research and development) expenses, resulting in a loss per share of $ 0. 09. the chart below shows analysts’ recommendations for pfizer stock over the last 12 months .
companies called isis have been forced to confront how attached they are to the name. isis was once the name of a mobile payment technology and a new condo building in florida. they both changed their names last year. isis pharmaceuticals, a california biotech company founded in 1989, told cnn it is now discussing the same move .
while that' s fine for clinical trials, it may make it difficult for ionis' drugs to compete with drugs that work as well but don' t have side effects that need to be monitored. of course, for spinraza, which
the technologies are more similar than they are different, notes c. frank bennett, senior vice president of research at isis pharmaceuticals .
at the end of the day, they' re both catalytic mechanisms and antisense mechanisms, in that the antisense strand is what binds to the target and causes cleavage of the message ,
he says .
despite considerable progress in oligonucleotide delivery, clinical successes have been balanced with setbacks that serve as reminders that the path to commercialization is never guaranteed. in august, for example, biopharmaceutical company and ionis partner oncogenex announced that its antisense drug custirsen failed to improve survival of patients with prostate cancer in late - stage clinical trials. and earlier this year, ionis reported dangerous reductions in platelets among patients in phase 3 trials for two unconjugated antisense therapies, one for a rare cardiac condition, and one for elevated triglycerides. although most drugs in ionis’s pipeline are now conjugated, which the company hopes will avoid such side effects, the announcement triggered a 40 percent plunge in share prices in may. (shares are now recovering as the company makes progress with nusinersen. )
ionis is built on a platform technology called antisense where the drug binds to a specific rna and, for most of the drugs, signal for their degradation. lower levels of the rna results in lower levels of the disease - causing protein the rna codes for .
akcea therapeutics, inc. (akca), an affiliate of ionis pharmaceuticals, inc. , focused on developing and commercializing drugs to treat people with serious and rare diseases, today announced completion of a new study to assess the burden of people living with familial chylomicronemia syndrome (fcs) and its impact on their daily lives. results from the study known as re - focus were published on june 11, 2018 in the peer - reviewed journal expert review of cardiovascular therapy. the web - based global study was conducted among people with fcs treated with volanesorsen, akcea’s investigational therapy currently under regulatory review in the us, eu and canada .
akcea therapeutics, inc. (akca), an affiliate of ionis pharmaceuticals, inc. , focused on developing and commercializing drugs to treat people with serious and rare diseases, today announced completion of a new study to assess the burden of people living with familial chylomicronemia syndrome (fcs) and its impact on their daily lives. results from the study known as re - focus were published on june 11, 2018 in the peer - reviewed journal expert review of cardiovascular therapy. the web - based global study was conducted among people with fcs treated with volanesorsen, akcea 's investigational therapy currently under regulatory review in the us, eu and canada .
but it wasn' t just the unsavory association that caused isis to become ionis, (pronounced “eye - oh - nis
) said stanley t. crooke, the company' s chairman and chief executive. it was the potential problems the confusion could cause for isis employees .
that pipeline contains dozens of medicines for severe or rare diseases such as various cancers, diabetes, spinal muscular atrophy and cardiovascular diseases. the phase 3 drugs are nusinersen, for spinal muscular atrophy; volanesorsen, for high triglycerides; and ionis - ttrrx, for ttr cardiomyopathy amyloidosis .
last may, ionis announced that it had discovered cases of thrombocytopenia - - low blood platelet levels - - in patients taking two of its drugs. the clinical trial protocols were amended to increase testing of platelet levels, which has eliminated the occurrence of
serious platelet events .
the main difference between the two approaches is that the mechanism of first - and second - generation oligonucleotide - based drugs is fairly well - characterized, whereas rnai is still in its early days. in fact, alnylam pharmaceuticals, a leader in rnai, only recently showed that short interfering rnas could have a systemic effect, a feat achieved long ago by earlier generations of antisense molecules .
reported $ 203 million in sales of their spinal muscular atrophy drug, spinraza, during its second full year on the market, netting ionis $ 22. 4 million in royalties on sales of the drug. the royalties are tiered and should increase as a percentage of sales as spinraza sales increase .
more recently, alnylam has conjugated its sirnas to the sugar molecule n - acetylgalactosamine (galnac) to give the drugs even greater affinity for hepatocytes, improve potency, and reduce off - target effects. early - stage testing of fitusiran, a galnac - conjugated sirna targeting a liver - produced protein associated with hemophilia, is ongoing, the company says. meanwhile, ionis has already adopted a similar method to conjugate galnac with antisense oligonucleotides destined for the liver—an advance that ionis claims improves delivery and provides a more than 30 - fold increase in potency over unconjugated approaches currently in trials. rnai company arrowhead pharmaceuticals is also using galnac to help deliver oligonucleotides to the liver, but instead of conjugating it directly to the sirna, the company’s dynamic polyconjugate (dpc) technology links the sugar to the sirna via a polymer that helps protect the cargo until it enters a cell. a recent clinical study on hepatitis b therapeutic arc - 520 found that the drug inhibits the production of viral protein in infected patients by up to 99 percent after a single dose .
on a rocky road toward hoped - for approvals, sanofi' s ($ sny) mipomersen has hit a serious rut with european regulators. a european medicines agency panel handed down a negative opinion late this week on the antisense drug from isis pharmaceuticals ($ isis) for treating a genetic disease that causes blood cholesterol to jump, citing safety concerns such as signs of liver toxicity and cardiovascular risks in patients in clinical trials .
it took several glancing blows and one heck of a sucker punch to fully shake the confidence of the investment community in oligonucleotide - based antisense therapeutics. a major hit came as early as 1998, when gilead sciences, a pioneer in the antisense space, abandoned the technology after its gene - code - blocking development pact with glaxo wellcome fell apart. gilead subsequently sold its antisense patent estate, which had been more than a decade in the making, to isis pharmaceuticals .
even with a relatively small number of patients, ionis was able to show beyond any doubt that volanesorsen could reduce triglyceride levels better than the placebo. sure, management is gloating a little, listing all of those zeros after the decimal point, but it has a right to be excited about the data - - and volanesorsen' s potential .
participants enrolled in the sham - controlled arm of embrace, a phase ii study that also included infantile - onset patients, will have the opportunity to receive nusinersen, biogen and ionis said. other studies in the nusinersen program, including cherish (later - onset consistent with type 2) and nurture (presymptomatic infants), will continue as planned .
, chief operating officer at ionis .
we have also demonstrated in 2015 that the advances we are making in our technology continue to make better and better medicines, which allows us to expand the application of our drugs to new tissues, new targets and new diseases. all of this sets us up for an exciting and event - filled 2016 .
ionis' antisense technology is currently the most effective way to inhibit synthesis of both lipoproteins in the liver, according to data published in the lancet [ 1 ]. the galnac3 - conjugated antisense oligonucleotide technology is 30 times more potent than the parent antisense oligonucleotide, leading to a lower dose and the potential for highly effective therapeutic targeting and much improved tolerability .
on the basis of those findings, biogen and ionis said, the endear study will be stopped and participants will be able to join the shine open - label study in which all patients receive nusinersen. data from the other endpoints of endear will be analyzed when the full data set is available, with results to be presented at future medical congresses, the companies said .
the arrival of vitravene, a short strand of 21 dna molecular units that blocks viral replication, was hailed as a major milestone for the biotech industry and was widely anticipated to usher in a new era of antisense products. but no more came. and by the middle of the last decade, isis pharmaceuticals, the carlsbad, california–based company behind vitravene, ended up pulling the therapy from the market because improvements in other antiretrovirals had effectively eliminated the drug’s target market. boni’s cautionary words proved all too prescient .
sanofi' s genzyme unit, leading the regulatory charge for mipomersen, which the company hopes to market under the name kynamro, plans to request the ema give its application another look through a re - examination process. yet the fortunes of the program appear to be waning with every safety red flag reviewers have raised about the drug. and aegerion pharmaceuticals ($ aegr) has a rival oral therapy for the same disease that has already gained wider support from the fda panel that narrowly recommended approval of mipomersen months ago .
researchers, pharmaceutical companies, and investors now anxiously await results from several late - stage trials, anticipated in 2017, as well as nusinersen’s approval, for confirmation that oligonucleotide therapeutics are finally reaching maturity. “every year it seems like we’ve gotten closer, ” says phillip zamore, director of the rna institute at the university of massachusetts school of medicine and cofounder of rnai therapeutics company alnylam pharmaceuticals. even rnai drugs, which joined the game later than antisense, are catching up, he adds. “now there are phase 3 clinical trials. ”
it appears from the phase 3 data that volanesorsen should be able to get approved. it' s not a perfect drug - - it has some side effects - - but the risk - benefit profile looks strong enough to gain approval, especially given the unmet need in patients with rare diseases - - familial partial lipodystrophy and familial chylomicronemia syndrome - - that cause severely high triglyceride levels, which ionis is going after .
in 2003, adrian krainer and his postdoctoral colleague luca cartegni, biochemists at the cold sping harbor laboratory in new york, engineered a synthetic, rna - like molecule that in the test tube changed how the transcripts from smn2 are spliced into the mature messenger rnas that then are translated into protein—in principle, this could boost production of the normal, full - length smn protein. three thousand miles away, at isis—now renamed ionis—a scientist named frank bennett noticed the work, published in nature structural biology, and called krainer. the spinal muscular atrophy foundation provided funding for krainer to hire researcher yimin hua. hua’s extensive work in krainer’s lab, in collaboration with ionis, led to the identification and preclinical development of nusinersen, a snippet of modified rna called an antisense oligonucleotide that intervenes in the splicing process to ensure that smn2 expresses the full - length smn protein .
even with the inevitable failures, ionis has plenty of additional drug candidates to take their place. check out the chart in the lower right; that' s a list of drugs expected to enter the clinic in the next 18 months. it' s hard to read the details, but it' s the volume of drug candidates, not necessarily what diseases the new crop of drugs will treat, that investors should be excited about .
in may, the company reported positive results for its neuro - ttr phase 3 trial testing inotersen in patients with hereditary ttr amyloidosis. glaxosmithkline handed back rights to the drug, but this may be a it' s - not - you - it' s - me kind of breakup, since glaxosmithkline is undergoing a strategic review of its assets. ionis is shooting to file marketing applications for inotersen in the u. s. and eu by the end of the year .
while that' s good news, and ionis has next - generation technologies that are even more potent - - generation 2 - plus - - where it thinks the lower doses aren' t causing thrombocytopenia side effects, the lack of serious events doesn' t mean the current late - stage drugs aren' t reducing platelet levels; it' s just that patients with low - but - not - serious platelet levels can be taken off the drug, avoiding major problems .
novartis is building a robust cardiovascular portfolio of targeted therapies to address unmet medical need of high - risk patients ,
said vasant narasimhan, global head, drug development and chief medical officer, novartis .
lp (a) and apociii are potent, genetically validated cardiovascular risk reduction targets. the importance of predictive biomarkers in achieving successful cardiovascular outcomes will also be essential in the future payer environment. we look forward to working with ionis and akcea to develop both treatments .
although just published in the lancet, those findings have since been overtaken by strikingly positive results from the larger phase iii trials. in their november announcement about the second trial’s halt, ionis and biogen, which has licensed nusinersen and has dubbed it spinraza, said the treated children experienced a “highly statistically significant improvement” in motor function compared to untreated children. the companies had used an established, widely used test of motor movements that monitors actions like sitting, standing, putting the hands on the head and taking steps .
after extensive preclinical development and testing, the drug began trials in sma patients in late 2011. in a phase ii trial described in the lancet today, ionis and academic collaborators report that it extended the life spans and improved the motor function of babies with severe sma, compared with the disease courses in natural history studies of untreated babies. to receive nusinersen, the babies had to have developed sma symptoms between 3 weeks and 6 months of age; the drug was injected into the cerebrospinal fluid surrounding the spinal cord .
while ionis' approved drugs and those in phase 3 development will dictate the biotech' s near - term future, long - term results will come from the success of its next - generation technologies. adding lica to the molecules as well as chemistry changes in the structure between generation 2 - plus and generation 2. 5 lower the effective dose required to treat 50% of the population (ed50). with increased potency will hopefully come decreased side effects and the ability to target new diseases in different parts of the body .
about novartis novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. headquartered in basel, switzerland, novartis offers a diversified portfolio to best meet these needs: innovative medicines, eye care and cost - saving generic pharmaceuticals. novartis is the only global company with leading positions in these areas. in 2015, the group achieved net sales of usd 49. 4 billion, while r & d throughout the group amounted to approximately usd 8. 9 billion (usd 8. 7 billion excluding impairment and amortization charges). novartis group companies employ approximately 118, 000 full - time - equivalent associates. novartis products are available in more than 180 countries around the world. for more information, please visit urltoken .
“these [ infant - onset ] sma kids are going to die. and not only are they now not dying, you are essentially on the path to a true cure of a degenerative [ neurological ] disease, which is unheard of, ” says jeffrey rothstein, a neurologist at the johns hopkins school of medicine in baltimore, maryland, who was not affiliated with the trials of the drug and is not connected with either of the two companies involved in its development: ionis of carlsbad, california, and biogen of cambridge, massachusetts .
consequently, most oligonucleotide therapeutics companies have focused on the liver. ionis has antisense oligonucleotide therapeutics in more than 10 clinical trials targeting diseases in the organ, from thrombosis to hepatocellular carcinoma. and alnylam’s most advanced therapeutic is an intravenous, nanoparticle - carried drug called patisiran, an rnai treatment that knocks down the translation of a mutated protein made in the livers of patients with a rare disease called hereditary amyloidosis. results from phase 3 trials of the drug are expected in 2017 and, if successful, could pave the way for the first approval of an rnai therapeutic .
pipeline update ionis management will present an update today on its drug development pipeline at the j. p. morgan conference, including an update on its ongoing open - label phase 2 clinical study of nusinersen in infants with type i spinal muscular atrophy (sma). previously the company reported data from this study on event - free survival, measures of muscle function and assessments of developmental milestones. the data reported today show continued increases in median event - free survival and muscle function scores. the safety and tolerability profile of nusinersen to date continues to support further development .
here, we present retrospective and prospective examples of how early analysis of headroom may aid in decisions along a translational continuum. the first example illustrates the need for analysis of future market prospects—technologies may fail close to clinical adoption if the expected health benefit shrinks at the time of market entry. vitravene (fomivirsen), an antisense therapy developed at the national institutes of health and licensed by isis pharmaceuticals, inc, was approved by the fda in august 1998 to treat cytomegalovirus retinitis (cmv - r). during its development in the 1990s, aids had transformed cmv - r from a rare disease into one of the most common ocular infections in the united states. when approved for use, vitravene was a biomedical breakthrough as the first fda - approved antisense therapy. while vitravene was in phase 3 clinical trials, highly active anti - retroviral therapy (haart) regimen, the “3 - drug cocktail”, was developed to suppress hiv replication and allow the immune system to recover. as a consequence, the number of new cases of cmv - r declined by nearly 75% . when vitravene reached the market, distributed by novartis ophthalmics ag, haart had been standard therapy for about a year. new cases of cmv - r were less common, and many existing cases no longer needed treatment; in other words, the health crisis that vitravene was designed to address had receded and sales of the product were much lower than predicted. as a result, novartis no longer markets vitravene, but isis pharmaceuticals still cites vitravene as evidence of its “ability to meet fda and european regulatory requirements for safety and efficacy, and for the commercial manufacture of antisense drugs. ”
although targeting other tissues is challenging, some companies are now making strides to do just that. ionis, for example, has developed modified antisense oligonucleotides that show effective targeting to cells in the kidney. arrowhead, meanwhile, is working on dpcs that will target cells in non - liver tissues such as tumors by incorporating ligands that target cell type–specific surface proteins. “it’s a matter of finding the right receptor - ligand combinations and to figure out how to conjugate the proper ligands, ” explains krainer. “down the line, i think one could have a set of conjugates that would allow you to target particular organs. ”
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mipomersen, to which sanofi gained rights through its 2011 buyout of genzyme, narrowly passed muster with an fda panel in october with a rems plan to track the safety of the therapy in patients taking it for the genetic disease, homozygous familial hypercholesterolaemia (hofh). the ema' s committee for medicinal products for human use (chmp), however ,
was not convinced that the company had proposed sufficient measures to prevent the risk of irreversible liver damage ,
the ema stated in a document .
we are disappointed by the committee' s recommendation. patients with hofh carry extreme, ongoing cardiovascular risk with significantly elevated ldl - c levels despite use of currently available therapies ,
said david meeker, president and ceo, genzyme .
this is a rare disease patient population, with a life - threatening condition, in need of new therapies. we will work closely with the chmp during the re - examination process to address the committee' s concerns, with the goal of making this important medication available to hofh patients in europe .
isis certainly has a lot riding on mipomersen too, as the company has been prolific in securing partnerships, notably with with biogen idec ($ biib) and astrazeneca ($ azn), and slow to advance its antisense therapies to the market .
the european commission gets the final say on drug approvals and often follows the advice of the chmp .
sanofi' s cholesterol drug shot down by ema. is aegerion' s lomitapide a better bet for approval? with input from @ ryanmfierce
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two biotechnology companies are joining forces in commercial pursuit of an emerging scientific field that could lead to treatments for numerous diseases .
micro - rnas, virtually unheard of a few years ago, are tiny snippets of rna — the chemical cousin of the genetic material dna — that have been found to play a major role in controlling biological processes. scientists have identified about 500 different micro - rnas that are made by human cells, and these snippets in turn appear to influence the activity of thousands of genes .
studies have already linked micro - rnas to cancer, viral infections, immune disorders and other diseases. so blocking specific micro - rnas — or perhaps stimulating them — could theoretically provide a powerful way to treat diseases .
“it’s as if we’ve discovered a new kingdom of regulation in biological metabolism that we didn’t know was there, ” said david baltimore, a nobel laureate and the president emeritus of the california institute of technology, who will be a director of the new company as well as chairman of its scientific advisory board .
the new company, called regulus therapeutics, will be equally owned by isis and alnylam but will have its own management and board. the management team has not been identified yet .
both alnylam and isis are pioneers in developing drugs that are made of rna and seek to suppress particular genes. the companies hope that combining their skills — and their patents — will allow them to dominate the micro - rna therapeutics business .
“this is the first time i’ve seen a biological advance as broad as this where the intellectual property is as concentrated in a single company as with regulus, ” said stanley crooke, chief executive of isis .
other companies are also starting to focus on micro - rna. one is rosetta genomics, an israeli company that is already collaborating with isis .
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so far, however, rna - related drugs have been difficult to develop. isis, based in carlsbad, calif. , has had many setbacks developing drugs using a technology called antisense. but its stock has nearly doubled in the last year on hopes for a drug that has sharply lowered cholesterol in clinical trials .
alnylam, based in cambridge, mass. , is trying to develop drugs that turn off specific genes by harnessing a natural phenomenon called rna interference. two of the discoverers of the phenomenon were awarded the nobel prize last year .
micro - rnas are related to rna interference. but john maraganore, chief executive of alnylam, said that while his company aims to shut off specific genes, regulus might be able to inactivate entire networks of genes. the broader effect, he conceded, could raise the risk of side effects .
to block a micro - rna, regulus will probably use the antisense approach — synthesizing short strands of an rna - like molecule that would bind to the micro - rna and inactivate it .
both companies are contributing intellectual property rights to regulus. alnylam is also contributing $ 10 million, since it has fewer patents to offer .
after this agreement, the companies will fund the venture equally. mr. crooke said that regulus, which is likely to be based in the san diego area, is expected to have about 20 people employed next year and to spend up to $ 20 million over its first two years. regulus has no drugs in clinical trials yet .
victor ambros, a professor at dartmouth who in the early 1990s discovered the first micro - rna, in the roundworm, said it was exciting to see companies being formed. “there’s a lot of excitement, and i think justifiably so, for the prospect of targeting the micro - rnas, ” said professor ambros, who has no ties to the companies .
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the owners of a bookstore in colorado that was vandalized earlier this week want the islamic state to be referred to as daesh .
a woman’s facebook account was disabled. a bookstore was vandalized. and one company, moved by the attacks on paris, just doesn’t want the association anymore .
while the name isis has become synonymous with a terrorist organization, the islamic state of iraq and syria, it is also the name of a well - known egyptian goddess .
many women are named isis, including 396 babies born in the united states last year .
for the world’s many isises, each act of horror carried out by the islamic state has been accompanied by the unsettling and repeated use of their name in news reports. an online petitioner has even gathered almost 57, 000 supporters to call on the news media to stop using the acronym .
“i named my daughter isis because it’s strong and meaningful, ” wrote one commenter on the petition, shani allman of florida. “to have it degraded and have her jeopardized by the media’s insensitivity is completely disturbing. ”
a woman in britain wrote that her 7 - year - old daughter, who was named after the goddess, went to bed crying “because people were being horrible to her about her name. ”
part of the river thames in britain remains known by the name, as does a feminist club at fordham university and the labrador in “downton abbey. ” the dog has been killed off from the show — though not because of its name. the cartoon “archer” also had to adjust the name of its spy agency. (fans of comics and 1970s television may recall isis as the name of a superhero. )
ionis pharmaceuticals ( nasdaq : ions ) ( known as isis pharmeceuticals until december 2015 ) is a publicly traded pharmaceutical company based in carlsbad , california , united states . the company was founded in 1989 by stanley crooke , a former glaxosmithkline head of research , with a goal to commercialize antisense therapy . the company received its first fda approval for an investigational new drug application in 1992 , marking the first time any antisense drug was approved to be tested in humans ; the ind was for isis ' genital warts drug candidate . the company and others founded to bring antisense drugs to market faced early failures ; isis ' lead compound for genital warts failed in clinical trials and isis terminated development in 1995 ; by that time two other companies working on antisense , gilead sciences and genta had left the field , leaving isis , hybridon , and lynx therapeutics working in the field . gilead sold the patents it had developed around antisense to isis . in december 2015 isis changed its name to ionis , driven in part by the negative image of " isis " generated by the terrorist group , islamic state of iraq and the levant , commonly known as isil or isis . 
ionis pharmaceuticals (nasdaq: ions) (known as isis pharmeceuticals until december 2015) is a publicly traded pharmaceutical company based in carlsbad, california, united states. the company was founded in 1989 by stanley crooke, a former glaxosmithkline head of research, with a goal to commercialize antisense therapy. the company received its first fda approval for an investigational new drug application in 1992, marking the first time any antisense drug was approved to be tested in humans; the ind was for isis' genital warts drug candidate. the company and others founded to bring antisense drugs to market faced early failures; isis' lead compound for genital warts failed in clinical trials and isis terminated development in 1995; by that time two other companies working on antisense, gilead sciences and genta had left the field, leaving isis, hybridon, and lynx therapeutics working in the field. gilead sold the patents it had developed around antisense to isis. in december 2015 isis changed its name to ionis, driven in part by the negative image of " isis " generated by the terrorist group, islamic state of iraq and the levant, commonly known as isil or isis.
ionis pharmaceuticals (nasdaq: ions) (known as isis pharmeceuticals until december 2015) is a publicly traded pharmaceutical company based in carlsbad, california, united states. the company was founded in 1989 by stanley crooke, a former glaxosmithkline head of research, with a goal to commercialize antisense therapy. the company received its first fda approval for an investigational new drug application in 1992, marking the first time any antisense drug was approved to be tested in humans; the ind was for isis' genital warts drug candidate. the company and others founded to bring antisense drugs to market faced early failures; isis' lead compound for genital warts failed in clinical trials and isis terminated development in 1995; by that time two other companies working on antisense, gilead sciences and genta had left the field, leaving isis, hybridon, and lynx therapeutics working in the field. gilead sold the patents it had developed around antisense to isis. in december 2015 isis changed its name to ionis, driven in part by the negative image of " isis " generated by the terrorist group, islamic state of iraq and the levant, commonly known as isil or isis.