Novartis's Experimental Meningitis Vaccine Meets Goals in Late-Stage Study

Novartis AG said an experimental meningitis B vaccine met the goals of a late-stage clinical study.

The vaccine, called 4CMenB, achieved a “robust immune response” in the study of more than 3,600 infants, the Basel, Switzerland-based company said today in an e-mailed statement. The drug also had “an acceptable” side-effect profile, the company said.

Novartis expects the results of additional clinical trials over the next few months and plans to seek European regulatory approval for the vaccine by the end of 2010. The compound may be used in different age groups and alongside other vaccines, the Swiss drugmaker said. Infants are particularly vulnerable to the meningitis B virus which can kill within 48 hours of the first symptoms appearing, according to Novartis.

“Meningitis B can be devastating for affected families and is a major concern for pediatricians who care for children with this serious illness,” said Andrew Pollard, Professor of Pediatric Infection and Immunity at the University of Oxford. “Many cases of meningitis are prevented today by the vaccines we give to our children, but the more complex meningitis B remains as a major threat.”

Novartis pioneered the technology known as “reverse vaccinology’’ to develop the shot after decoding the genetic makeup of a pathogenic meningitis strain.

read more» Read more...

Promise Seen in Drug for Fragile X Syndrome

An experimental drug succeeded in a small clinical trial in bringing about what the researchers called substantial improvements in the behaviors associated with retardation and autism in people with fragile X syndrome, the most common inherited cause of these mental disabilities.

The surprising results, disclosed in an interview this week by Novartis, the Swiss pharmaceutical giant that makes the drug, grew out of three decades of painstaking genetic research, leaps in the understanding of how the brain works, the advocacy of families who refused to give up, and a chance meeting between two scientists who mistakenly showed up at the same conference.

“Just three years ago, I would have said that mental retardation is a disability needing rehab, not a disorder needing medication,” said Dr. Thomas R. Insel, director of the National Institute of Mental Health, who was told of the Novartis trial results. “Any positive results from clinical trials will be amazingly hopeful.”

Dr. Mark C. Fishman, president of the Novartis Institutes for BioMedical Research, cautioned against too much optimism. The trial involved only a few dozen patients, only some of whom benefited from treatment. The drug is likely to be years away from being commercially available and could fail in further clinical trials, he said.

“We have been reluctant to make this public because we still need to do more experiments, do them correctly and in a bigger way,” Dr. Fishman said. “But our group feels pretty good about the data.”

If authenticated in further, larger trials, the results could also become a landmark in the field of autism research, since scientists speculated that the drug may help some patients with autism not caused by fragile X, perhaps becoming the first medicine to address autism’s core symptoms.

One child in five thousand is born with fragile X syndrome, with mental effects ranging from mild learning disabilities to retardation so profound that sufferers do not speak, and physical effects that include elongated faces, prominent jaws, big ears, and enlarged testes. It mostly affects boys and earned its name because, under a microscope, one arm of the X chromosome seems nearly broken, with part hanging by a thread.

The gene for fragile X was discovered in 1991. Work since then has found that fragile X patients seem to experience an overload of unchecked synaptic noise — synapses being the junctions between brain neurons. The Novartis drug and others like it are intended to lower the volume of this noise so memory formation and high-level thinking can take place, allowing children to develop normally.

The Novartis trial, which began in 2008 in Europe with data analysis completed this year, was too brief to observe effects on basic intelligence. Instead, researchers measured a range of aberrant behaviors like hyperactivity, repetitive motions, social withdrawal and inappropriate speech. They gave one set of patients the drug and another a placebo, and after a few weeks switched treatments, with both doctors and patients unaware of which pill was which.

The results of the trial were something of a jumble until Novartis scientists noticed that patients who had a particular, undisclosed biological trait improved far more than others. “The bottom line is that we showed clear improvements in behavior,” Dr. Fishman said.

Told of the results, two parents of a fragile X patient were euphoric.

“This is what we have been working for and hoping for since our son was diagnosed with fragile X 17 years ago,” said Katie Clapp, president and co-founder of the Fraxa Research Foundation, a nonprofit organization dedicated to financing fragile X research. “This may be the key to solving the mystery of autism and other developmental disorders.”

Geraldine Dawson, chief science officer at Autism Speaks, the world’s largest autism advocacy organization, said that a growing body of research suggests that the many genetic causes of autism all seem to affect synapses, suggesting that a treatment for one form of the disease might help others.

“The exciting thing about these results is that it is our hope that these same medications may have similar positive benefits for people with autism who don’t have fragile X syndrome,” Dr. Dawson said.

Between 10 percent and 15 percent of autism cases result from fragile X syndrome or some other known genetic defect. While fragile X is the most common inherited cause of mental retardation, Down syndrome — which also causes retardation — is more common but is not inherited.

The Novartis trial results were not published or peer reviewed, and for commercial reasons Dr. Fishman refused to divulge many details. Dr. Luca Santarelli, head of neuroscience at Roche, confirmed that Roche is in the midst of testing a similar medicine in fragile X patients at four sites in the United States.

“So far we like what we see,” Dr. Santarelli said in his only characterization of their study.

One reason for the euphoria surrounding the Novartis trial is that it was seen as an especially difficult test of the drug’s effects. For ethical reasons, Novartis tested the drug only in adults. But the company and outside researchers believe that such compounds may prove most effective in young children, whose brains are far more likely to respond rapidly when barriers to learning are removed.

“This is perhaps the most promising therapeutic discovery ever for a gene-based behavioral disease,” said Dr. Edward M. Scolnick, former research chief at Merck and now director of the Stanley Center for Psychiatric Research at the Broad Institute at Harvard and the Massachusetts Institute of Technology.

Dr. Scolnick has not seen the results of the Novartis trial, but was told of them and concluded that if the drugs work in fragile X, “there’s nothing to say that they won’t work in some cases of broader autism-spectrum disorders.”

An Unlikely Beginning

The roots for the Novartis results began in 1982 when Stephen T. Warren, then a graduate student in genetics at Michigan State University, was looking for a job and something to research. A friend told him about fragile X and, with the same reflection he might use to pick a novel for a long flight, he decided that he wanted to find the gene that caused it.

“I had no idea how hard this would be,” Dr. Warren said. Nine years later, Dr. Warren, then at Emory University, was part of an international team that won a fierce competition by isolating the gene. The discovery was front-page news around the world, and experts predicted that widespread fetal testing and therapies were in the offing.

The predictions were premature because, like most of genetic research, discovering how the flawed gene caused disease was far harder than anticipated and required multiple leaps in neurology and biology. And even with those, much remains mysterious.

Fragile X is caused by a genetic stutter in which a portion of the gene gets repeated like a scratched album. With each subsequent generation, the number of repeats tends to rise. So if a mother has 10 repeats, her child might have 11 or 12. For reasons that are not well understood, however, this process of repeat amplification can suddenly go haywire. So mothers who have 55 or more repeats tend to have children with hundreds.

In anyone with 200 or more repeats, the body shuts off the gene. Since genes are used to make proteins, this genetic silencing means the encoded protein is never made. The absence of this protein in cells causes the wide-ranging effects of fragile X syndrome. Those with 55 to 200 repeats are considered carriers, and recent research shows they can have severe neurological declines late in life that mimic Alzheimer’s and Parkinson’s.

Many geneticists would have moved on to other research topics after finding a disorder’s underlying gene. But Dr. Warren met affected children and their parents. Instead of family pictures, Dr. Warren’s desk displays a framed photo of a fragile X chromosome.

“I could not imagine telling someone like Katie Clapp that we were not going to pursue this research anymore,” he said.

So he kept on. Years of work by him and others found that the protein missing in those with fragile X normally seems to act as a sort of traffic cop at brain synapses, helping to stop or slow brain signaling at crucial intervals. It does this by sopping up the genetic instructions needed to produce proteins that encourage brain signaling. Regulating this flow of electronic pulses across the brain is crucial for the brain’s ability to learn and mature.

Dr. Warren was puzzling over how to recreate that synaptic traffic cop when, because of a scheduling conflict, he showed up in 2001 at the wrong scientific conference and happened to sit next to Mark F. Bear, a neuroscience professor at M.I.T. who had just given a presentation about compounds that seemed to work in synapses to speed the creation of proteins — including the one missing in fragile X patients.

The two got to talking and decided to collaborate. They found that if Dr. Bear reverse-engineered his compounds, they seemed to slow brain transmissions. Instead of a traffic cop, the brain would get speed bumps. Not ideal, but perhaps adequate in lowering the synaptic noise enough to encourage learning and the moderation of the kind of synaptic traffic jams that in fragile X children can lead to seizures.

Sure enough, mice, fish and fruit flies that through genetic engineering were made to have fragile X seemed to become normal when given Dr. Bear’s compound. The Novartis compound is a member of the same drug family.

“We have been promising for a long time that unlocking the molecular basis for hereditary diseases would lead to dramatic therapeutic advances, and that promise is finally coming true,” said Dr. Francis S. Collins, director of the National Institutes of Health, in discussing the science leading up to the trial. “But it has not been easy.”

A Search for Treatment

A hundred years ago, Katie Clapp would have died giving birth to Andy, her child with fragile X.

“Andy’s head was too big to get out without a C-section, he would have killed me, and that would have taken care of the fragile X gene,” she said.

But Ms. Clapp and Andy did survive. And despite going to some of the best hospitals in the country, four years would pass before Andy’s condition was properly diagnosed.

When a doctor finally thought to do a fragile X test, Ms. Clapp and her husband, Dr. Michael Tranfaglia — both Harvard graduates with post-graduate degrees — researched the disease and came to two conclusions: fragile X was potentially treatable; and only about five researchers in the world were working toward a cure.

“And I thought, what if all five walk across the street at the same time and get hit by a Mack truck?” Ms. Clapp said. “That is not going to get us there.”

So the two started the Fraxa Research Foundation. Remarkably, their efforts seem to be paying off and may finally offer hope not only to those who with fragile X but to carriers like Andy’s sister, Laura.

“I’ve always known my kids have a chance of having it,” Laura, 18, said in a recent visit to the family’s house. “But I’m not going to have kids for at least 10 years anyway, and they’ll have a cure for by then.”

She paused, looked at her mother and said: “You’ve got 10 years.”

read more» Read more...

Novartis biological drug Ilaris® approved in EU to treat Cryopyrin-Associated Periodic Syndrome

The new biological medicine Ilaris® (canakinumab) has been approved in the European Union (EU) to treat adults and children as young as four years old with cryopyrin-associated periodic syndrome (CAPS), a rare life-long auto-inflammatory disease with debilitating symptoms and few treatment options.

The accelerated EU decision follows approvals in the US and Switzerland, where Ilaris was granted priority review in view of the significant unmet medical need. Ilaris is the only medicine approved in the EU for CAPS patients as young as four years old, and for patients with the most debilitating form of CAPS, neonatal-onset multisystem inflammatory disease (NOMID)[4]. It is a fully human monoclonal antibody given by injection under the skin once every two months - the longest dosing interval of any available treatment .

"We are excited by the latest approval because Ilaris represents a significant therapeutic advance for patients with this debilitating and sometimes fatal disease," said Joe Jimenez, CEO of the Novartis Pharmaceuticals Division. "Ilaris is the outcome of our pathways-driven search for innovative medicines that are tailored to the needs of patients. Initially we studied Ilaris in a very rare disease with a well-understood genetic profile, and now that its efficacy has been proven, we are able to move ahead rapidly with development in other diseases characterized by the same inflammatory process."

The regulatory submission was supported by data showing that Ilaris produced rapid and sustained remission of symptoms in up to 97% of CAPS patients, with most of them responding within hours of the first injection .

Ilaris, previously known as ACZ885, targets and normalizes the production of a protein within the immune system called interleukin-1 beta (IL-1ß). In CAPS patients, IL-1ß is overproduced causing widespread inflammation and tissue damage . Symptoms, such as debilitating fatigue, fever, joint pain and conjunctivitis, can be present from infancy and continue throughout the patient's life .

If left untreated, CAPS may have serious consequences such as deafness, bone deformities, erosive joint destruction, and central nervous system damage leading to loss of vision . Around 25% of patients develop amyloidosis, a condition in which the build-up of proteins can cause vital organs to fail, resulting in renal failure and requiring kidney transplantation. Approximately 20% of patients with NOMID, the most severe form of CAPS, die before reaching adulthood .

CAPS is believed to occur in around one in 2,500 people in the EU[3],[11], but fewer than 1,000 cases have been reported worldwide due to poor diagnosis[1],[3]. CAPS includes three distinct autoinflammatory disorders of increasing severity: familial cold auto-inflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS), and NOMID[2],[3]. Ilaris is the only treatment indicated in the EU and Switzerland to treat all three disorders .

Studies with ACZ885 are ongoing in other diseases in which IL-1ß plays an important role, such as chronic obstructive pulmonary disease (COPD), type 2 diabetes, systemic juvenile idiopathic arthritis (SJIA), and gout - one of the most painful forms of arthritis. Not all potential patients with these diseases would be eligible for treatment with ACZ885, if approved.

The CAPS filing was based on a clinical trial program involving more than 100 patients. Data from a pivotal study published in The New England Journal of Medicine show that Ilaris produced a rapid, complete and sustained response in most patients.

None of the patients treated with Ilaris (0 out of 15) experienced a disease outbreak or 'flare' compared to 13 of the 16 patients who received placebo (0% vs. 81% respectively, p<0.001).>"In CAPS studies, symptoms improved within 24 hours after patients received a single dose of Ilaris. The disease was barely detectable in the blood after two weeks and the remission of symptoms was sustained for six months," said Helen J. Lachmann, MD of the UK National Amyloidosis Centre at the Royal Free and University College Medical School in London, UK. "By effectively turning off the disease activity, Ilaris has the potential to transform patients' lives by offering long-term control of their disease."

Ilaris was generally well tolerated and there was no consistent pattern of adverse events apart from a slight increase in infections . Two patients experienced serious adverse events, namely a lower urinary tract infection and vertigo .

The most common adverse events were nasopharyngitis, diarrhea, influenza, headache and nausea . Ilaris was not associated with any severe injection-site reactions and those that did occur were classified as mild-to-moderate .

The EU approval was granted under exceptional circumstances, a common practice with so-called orphan drugs. This reflects a need for additional data due to factors such as the rarity of the disease or lack of scientific knowledge. The situation is reviewed every year until the European Medicines Agency (EMEA) is able to grant a normal approval. In addition to its orphan drug status for CAPS, Ilaris has been designated as an orphan drug for treating SJIA, the most severe form of arthritis in children, in the US, EU and Switzerland, and has fast-track status for SJIA in the US. Orphan drugs are those developed to treat diseases affecting fewer than 200,000 people (in the US) or fewer than five out of 10,000 people (in the EU).

Ilaris was approved in Switzerland in July 2009 to treat all three forms of CAPS in adults and children over four years old, and in the US in June 2009 to treat two forms of CAPS, namely FCAS and MWS, while a study in NOMID patients is under way. Priority reviews are ongoing in other countries including Australia, Brazil and Canada.

read more» Read more...

Novartis gets worldwide rights to antibiotic

Swiss drugmaker Novartis AG said Thursday it bought worldwide marketing rights to an antibiotic that is being developed to treat infections caused by drug-resistant bacteria, potentially including the "superbug" MRSA.

Novartis did not disclose the payments it will make to the drug's developer, Paratek Pharmaceuticals of Boston. The deal includes an upfront payment to Paratek, potential milestone payments as the drug candidate advances through clinical testing and regulatory review, and royalties on sales if the drug is approved.

Novartis and privately held Paratek will share responsibility and costs of developing the drug.

Paratek is running a late stage clinical trial of PTK 0796, evaluating the drug's effectiveness against complex skin infections. Novartis said other trials are planned, and studies have shown evidence the drug works against drug-resistant and hospital-acquired infections like MRSA.

The drug is being developed in both oral and intravenous forms. PTK 0796 could be the first broad-spectrum antibiotic that can be given both orally and by IV, the company said. The options could make it easier for patients to continue treatment after they leave the hospital.

Novartis stock rose 53 cents to $50.09 in morning trading.

read more» Read more...

Novartis says new version of Tekturna approved

The pharmaceutical company Novartis announced that the FDA has approved a newer version of one of its previously approved blood pressure medications, Tekturna. The new drug, Tekturna HCT, combines Tekturna with hydrochlororthiazide, a diuretic; Novartis claims that in combining the two drugs, blood pressure is treated on two different fronts.

Per its website, the company explains that Tekturna, or aliskeren, works on reducing blood pressure by keeping the blood vessels open. The diuretic helps eliminate sodium and excess water from the body, which also works to lower blood pressure.

Novartis has continued a trend among pharmaceuticals to combine two generic drugs that have already been approved and using them in tandem to create a newer, more effective medication. As Alexis Borisy, who has been touted as the industry "matchmaker," told the New York Times, such a revamping of drugs already approved is better business than creating one from scratch. Additionally, the safety standards of the FDA are met more swiftly, as the drugs used have already undergone clinical trials that underscore their safety.

Other "cocktail" pharmaceuticals: Advair, which combines two asthma medications, and Vytorin, which combines cholesterol lowering drugs developed by both Merck and Schering Plough. Contrave, which has just cleared efficacy standards testing with the FDA, is a weight loss drug that combines an anti-depressant and a drug used to fight opiate and alcohol addiction.

Certainly, combining drugs in such a fashion is a cost effective move that will ultimately help the patient. On its website, Novartis claims that more than half of all adults who have high blood pressure need more than one drug as part of a complete treatment plan.

Source : www.foodconsumer.org

read more» Read more...

New biological therapy Ilaris® approved in US

The US Food and Drug Administration (FDA) has approved Ilaris® (canakinumab) for the treatment of children and adults with cryopyrin-associated periodic syndrome (CAPS), which includes a number of rare but life-long auto-inflammatory disorders with debilitating symptoms and limited treatment options. The FDA granted priority review to Ilaris based on its potential to meet an important clinical need for patients with CAPS.

Ilaris is the first approved treatment for patients as young as four years old suffering from two forms of CAPS: familial cold auto-inflammatory syndrome (FCAS) and Muckle-Wells syndrome (MWS).

CAPS is caused by a single gene mutation that leads to overproduction of interleukin-1 beta (IL-1), which causes sustained inflammation and tissue damage. Symptoms, such as debilitating fatigue, rash, fever, headaches, joint pain and conjunctivitis, can be present from birth or infancy, and can occur daily throughout patients' lives. Long-term consequences may be serious and potentially fatal, including deafness, bone and joint deformities, central nervous system damage leading to visual loss, and amyloidosis resulting in renal failure and early death.

Ilaris, previously known as ACZ885, is a fully human monoclonal antibody that rapidly and selectively blocks IL-1. The dosing schedule for Ilaris is once every eight weeks, which is less frequent than the current approved therapy. Also, more than 90% of patients studied did not experience any injection site reactions and those that did occur were of a mild-to-moderate nature.

"Until now, treatments for CAPS patients have been limited to traditional inflammatory-disease medications that work by suppressing the entire immune system, and newer therapies that control the disease better but require more frequent injections," said Hal Hoffman, MD, Associate Professor of Pediatrics and Medicine at University of California, San Diego, USA.

He added: "CAPS is a life-long disease and the convenience of administration of medicine is of the utmost importance for these patients. With rapid and sustained response, good tolerability, and a significantly less-frequent dosing schedule, Ilaris represents an important treatment advance for children and adults with CAPS."

CAPS comprises three disorders of increasing severity: FCAS, MWS and neonatal-onset multisystem inflammatory disease (NOMID). There are believed to be approximately 300 cases in the US, but many patients may remain undiagnosed due to poor disease recognition. A clinical study is ongoing to evaluate the potential of Ilaris to treat patients with NOMID. There are currently no approved therapies for the treatment of NOMID.

"Children and adults affected by these inflammatory diseases have to cope daily with distressing and debilitating symptoms," said Trevor Mundel, MD, Head of Global Development at Novartis Pharma AG. "We are excited about this first Ilaris approval which reflects our commitment to developing innovative treatments that address unmet medical needs, regardless of the size of the patient population."

In addition to ongoing studies in CAPS, clinical trials are also under way with Ilaris in systemic juvenile idiopathic arthritis (SJIA), and more common disorders such as some forms of gout, chronic obstructive pulmonary disorder (COPD) and type 2 diabetes. Further development in rheumatoid arthritis (RA) is not planned.

The approval of Ilaris is based on a three-part, one-year Phase III study involving 35 patients aged nine to 74 years old with varying degrees of disease severity. Results published in The New England Journal of Medicine on June 4, 2009 show that Ilaris produced a rapid, complete and sustained response in the majority of patients.

Part two of the study included the primary endpoint, a comparison between the number of patients treated every two months with Ilaris who experienced disease outbreaks or 'flares' vs. those on placebo. Results showed that none of the patients in the Ilaris group (0 out of 15) experienced a disease flare compared to 13 out of 16 patients in the placebo group (0% vs. 81% respectively, p<0.001).>

Source : www.worldpharmanews.com

read more» Read more...

New Novartis ‘Niche Drug’ Approved for Rare Genetic Disorder

Drug maker Novartis AG has gained Food and Drug Administration approval for a new drug that treats a rare genetic disorder which afflicts just a few thousand people in the world.

The drug, Ilaris, represents a change in course of sorts for drug companies, who have begun developing more new drugs that are designed to correct one or two genetic triggers which cause rare diseases rather than cranking out medications designed to treat millions of people afflicted with more common disorders.

It may sound counter intuitive to market drugs for so-called “niche diseases” that affect fewer people than other conditions, but by focusing on specific genetic-based disorders that can be easier to unravel and develop drugs to treat, Novartis and other drug companies are gambling on the belief that such an approach will pay off in the long run, according to a report in The Wall Street Journal.

Novartis hopes the approach to developing new drugs will one day blossom into a market as big as today’s blockbuster drugs. Ilaris is Novartis’ first venture into genetic trigger drugs.

The new way of developing drugs certainly doesn’t come cheap. In 2002, Novartis spent $4 billion to build a new research center in Cambridge, Mass. and hired hundreds of scientists to work there, according to the Journal report.

Novartis won’t say how much it will cost to produce Ilaris, but industry analysts say it is likely to be among the world’s most expensive drugs.


Rare Genetic Syndrome is Treated
Ilaris is delivered via a shot and treats a rare, genetic disorder called cryopyrin-associated periodic syndrome (CAPS). The syndrome is caused by a mutation in a single gene and there are just 300 known cases in the entire United States. Worldwide, there are maybe 7,000 world-wide, medical experts say.

People with CAPS produce too much interleukin-1, a protein that is associated with the immune system, and develop inflammation, chronic rash, joint pain, and fever. In the most serious cases, CAPS can lead to kidney failure, deafness, and even death. Ilaris and its rival drug, Arcalyst, prevent the disease by stopping production of the protein.


Broader Uses Are Possible
The makers of Ilaris and Arcalyst are considering asking for FDA approval to treat gout, a more common inflammatory condition that causes chronic joint pain. Clearing the drugs for broader uses would increase profits for the drugs.

Novartis also is working on other drugs that genetically target disease. Those drugs currently in development include AIN457 for psoriasis and an inflammatory disorder of the eye that could hit pharmacy shelves as early as 2010 and AFQ056, a Parkinson’s disease drug the company plans for a 2012 launch.

Source : www.attorneyatlaw.com

read more» Read more...

Novartis, Roche unveil promising cancer drugs data

Swiss drugmaker Novartis AG said late stage data showed its Sandostatin LAR drug helped fight tumuors of the mid-gut, while other drugs showed benefits for treating advanced melanoma.

Novartis' rival Roche Holding AG also said on Thursday a new drug helped shrink the tumours of 25 percent of women with HER2-positive breast cancer, according to data from a mid-stage clinical trial.

Cancer drugs are seen as a key growth area for pharmaceutical makers like the two Swiss companies, which along with many others are presenting data on cancer drugs at an American Society of Clinical Oncology (ASCO) meeting.

Novartis said a Phase III study showed Sandostatin LAR more than doubled time without tumour growth and reduced the risk of disease progression by 67 percent.

It also said mid stage data on two of its drugs showed potential as treatments for advanced melanoma. Favourable results from a study into the drug Glivec mean the study will continue to a second expanded stage of enrolment.

The Swiss company also presented preliminary results which showed that 72 percent of melanoma patients treated with Afinitor combined with Roche's Avastin experienced a clinical benefit.

Afinitor, one of Novartis' most important new drugs, also helped patients with advanced liver cancer in an early stage trial.

Data from a mid stage trial of the new treatment from Roche, a combination of its Herceptin and chemotherapy known as trastuzumab-DM1, showed about 35 percent of a total 112 patients had their tumours shrink, or their disease stabilise for at least six months.

Source : www.reuters.com

read more» Read more...

Novartis says new drug to improve lung functions

The Swiss pharmaceutical company Novartis announced Thursday that it has a new a drug which will offer better help to sufferers of chronic obstructive pulmonary diseases, such as chronic bronchitis and emphysema.

The QAB149 drug, after phase III trials, showed it could deliver improved lung function within five minutes of the first dose, lasting for 24 hours. This would mean suffers would only need one dose a day.

Chronic obstructive pulmonary diseases can be life-threatening respiratory diseases that affect some 210 million people worldwide, mostly as a result of smoking cigarettes or inhalation of other fumes.

The World Health Organization estimates the diseases are becoming one of the leading causes of deaths worldwide.

QAB149 is currently undergoing regulatory review in the European Union and the United States.

Source : www.monstersandcritics.com

read more» Read more...

Novartis Gets Full Rights To New Respiratory Drug

Novartis AG Tuesday said it had acquired the full rights to an experimental drug to treat asthma and smoker's cough, in an asset swap with Schering-Plough Corp .

The move underlines the Swiss drugmaker's intention to build a portfolio of drugs to treat respiratory diseases, to lessen dependence on its blockbuster heart drug Diovan which will lose patent protection in two or three years.

Novartis, based in Basel, said it has assumed worldwide rights to develop and sell experimental drug QMF149, a combination of Novartis' own smoker's cough drug QAB149 and Schering-Plough's anti-inflammation drug mometasone. QAB was filed for regulatory approval in the U.S. and Europe late last year. A decision is expected by mid-2010.

In return, Schering-Plough gets the exclusive rights to develop and sell a fixed combination of its own mometasone and Novartis' asthma and smoker's cough drug Foradil, which is already approved for sale.

The companies have agreed to share royalties on sales of the respective products. Should it win regulatory approval, Novartis will book worldwide sales of QMF149. The drug will probably be filed for approval in 2013, said Karl-Heinz Koch, analyst in Zurich with broker Helvea.

Novartis will be presenting detailed late-stage study data on QAB149 at the American Thoracic Society Meeting in San Diego, which runs until May 20. The data include a direct comparison to market leading drug Spiriva, sold by privately-held German company Boehringer Ingelheim and Pfizer Inc.

At 0730 GMT, Novartis shares were unchanged at CHF44.06. The shares have lost 15% in value so far this year, underperforming the European healthcare sector, down 7.6% year-to-date, and the broader Swiss market, which is 1.8% lower in 2009.

Source : money.cnn.com

read more» Read more...

Novartis Drug Heralds Multiple Sclerosis Advances

The first in a new wave of multiple sclerosis medicines, from drugmakers led by Novartis AG and Merck KGaA, may reach the market next year, making therapy more convenient for patients and boosting profits.

In Seattle this week, the drugmakers will present results from human trials of two oral drugs. If the products win U.S. approval, patients for the first time will be able to take pills, rather than shots or transfusions, to slow the nerve- damaging condition. Five more pills and two infusion therapies may join the $7.5 billion a year MS market by 2014, according to the National Multiple Sclerosis Society in New York.

Progress against MS is coming on three fronts, said John Richert, a doctor who leads research at the society. The new drugs may reduce the frequent relapses most patients face. Improved detection may allow earlier treatment before symptoms emerge. Stem-cell therapies now being tested may go even further, reversing disability in patients with advanced disease.
“In terms of stopping disease activity, there are more therapies coming onstream than ever before and many of these also appear to be more potent,” Richert said in a telephone interview. “While the whole idea of repairing the nervous system really seemed like science fiction five to six years ago, advances in the last year suggest this may well be within our grasp.”
Novartis was unchanged at 42.46 Swiss francs in Zurich trading. Merck KHaA, which today reported a decline in first- quarter profit and said “the bottom” had been reached, advanced 1.2 euros, or 1.8 percent, to $67.70 euros in Frankfurt trading.

Rogue Cells

First described 140 years ago, multiple sclerosis causes the body to attack itself through the immune system. Rogue immune cells travel to the brain and spinal cord and enter the nervous system. There the cells assault and destroy myelin, the fatty substance that surrounds nerve-cell fibers. That interferes with the nerve cells’ ability to transmit electrical impulses and move muscles.

MS generally starts in early adulthood, disrupting people’s coordination and balance and sometimes leading to damaged vision and paralysis. For Rhonda McHenry, a fall in 1994 while chasing her then 9-month-old son, and the resulting nerve pain, led to an MS diagnosis.
McHenry, now 44, was diagnosed with the “relapsing- remitting” form of MS that makes symptoms flare and recede and that 85 percent of patients have initially, according to the MS Society.

Drugs Against Relapse

In one way, she was fortunate. Around the time of her diagnosis, three new drugs were approved to prevent MS relapses. They included two made from proteins called beta interferons -- Avonex, made by Biogen Idec Inc. of Cambridge, Massachusetts, and Betaferon, made by Bayer AG of Levrkusen, Germany. The third treatment is Copaxone, made by Teva Pharmaceutical Industries Ltd., of Petah Tikva, Israel, from a synthetic protein.

The treatments included Avonex, made by Biogen Idec Inc. of Cambridge, Massachusetts; Betaferon, made by Bayer AG of Levrkusen, Germany; and Copaxone, a product from Teva Pharmaceutical Industries Ltd., of Petah Tikva, Israel.

These products, along with Rebif, made by Merck KGaA, of Darmstadt, Germany, inhibit the overactive immune response found in MS, reducing damage to nerves and slowing disability, Richert said. Rebif was approved by the U.S. Food and Drug Administration in 2002.
The four, known by their initials as the ABCR drugs, now dominate the MS marketplace, with more than $1 billion each in sales annually. Biogen’s Avonex led the pack with $2.2 billion last year. Geoffrey Porges, an analyst with Sanford C. Bernstein & Co. in New York, projected in a report last year that the MS market will grow to $10.8 billion in 2013.

Blocking Cell Movement

As researchers have learned more about how the immune system attacks nerves, they have been able to design drugs that interfere with that process, Richert said.

The newest MS treatment on the market, Biogen’s Tysabri, blocks the movement of immune cells from the bloodstream into the nervous system.

The experimental Novartis drug FTY720 disrupts the movement of immune cells at an earlier stage than Tysabri, keeping them from leaving lymph nodes and entering the bloodstream, according to Richert.

German Merck is attempting to turn a cancer drug, cladribine, into an MS medicine because it suppresses the immune system, blunting its attack on nerve cells. The product was approved to treat leukemia more than a decade ago.

Results Next Week

Novartis, based in Basel, Switzerland, and Merck will release results this week at a Seattle meeting of the American Academy of Neurology, a professional association based in St. Paul, Minnesota. Preliminary findings already released by both companies suggest that the drugs limit relapses.

The data on side effects may determine if and when the products will take revenue from the Bayer, Biogen and Teva products, said Jack Scannell, a London-based analyst with Bernstein.
Merck plans to submit its FDA application in June or July, said Bruno Musch, the company’s head of global clinical development for neurodegenerative diseases. Novartis intends to make its submission by the end of the year, said Joseph Jimenez, head of the pharmaceutical division, in a conference call last week.

MS Pills Advance

Other companies are also racing to bring MS pills to market. Acorda Therapeutics Inc., based in Hawthorne, New York, said on April 23 it had reformatted and resubmitted an application for a pill called Fampridine-SR. The drug improved the walking speed of MS patients in a study, according to the company.

Biogen, the leading maker of MS therapies; Teva; and Sanofi-Aventis SA, of Paris are conducting late-stage trials of their own MS pills. An injectable drug called Campath, being developed by Cambridge, Massachusetts-based Genzyme Inc. and Bayer, may be the most effective therapy for people with severe MS, according to Porges. He projects Campath revenue will top $1 billion by 2013.

Data on side effects of all the new therapies will be closely watched because drugs that disrupt immune system activity can increase the risk of infections, Richert said.

Tysabri was pulled from the market in 2005 after three patients developed a life-threatening brain illness and two of them died. The FDA allowed Tysabri sales to resume in July 2006 after deciding its effectiveness outweighed the risk. The company has since reported six new cases, with one death.

Side Effects

If the new pills cause side effects, that may negate their advantages, said Al Sandrock, senior vice president of neurology for Biogen, whose drug BG-12, also taken by mouth, is in late- stage testing.

“The safety profile is never fully appreciated until a drug has been in thousands of patients for many years -- we certainly learned that,” Sandrock said.

Elizabeth Morrison, a neurologist at the Cascadia Multiple Sclerosis Center, a medical practice in Bellingham, Washington, has MS herself. She said she will probably keep taking Copaxone, as it controls her symptoms without causing side effects such as chest pain or anxiety, which patients can experience.

She also said she won’t rush to prescribe the new drugs for other patients.
“I think people with MS may think, ‘Oh, an oral medication -- that will be safer or easier for me,’” she said in a telephone interview. “But that’s not necessarily the case.”

Doctors will be cautious in prescribing the new pills until their safety is clear, said Bernstein’s Scannell.

Convenience vs. Risk

“The fact that they’re convenient and oral doesn’t trump the fact that they might give you a horrible brain infection or cancer,” he said in a telephone interview.

In addition to having more and better drugs, clinicians can now find MS lesions using MRI scans earlier than ever before, said Peter Wade, medical director for neurology at the Mandell Center for Multiple Sclerosis, at Mount Sinai Rehabilitation Hospital in Hartford, Connecticut.
“Now there is an urgency to diagnose the disease as quickly as possible because the medications we have currently available work much more effectively early on,” Wade said in a telephone interview.

For patients with advanced disease, early detection is no longer relevant.

For most of the past 15 years, daily injections of Copaxone have controlled Rhonda McHenry’s relapses and when they didn’t, five days of intravenous steroids usually cut them short.
Today, the relapses come more often, last longer and cause more weakness, making walking more difficult for McHenry, who is today the chief operating officer for a Kansas City bank. Now she hopes she will be lucky again and benefit from new therapies.

Clinical Trial

She qualified for a clinical trial that will use patients’ own stem cells to reset their immune systems. Patients will have their blood-forming stem cells extracted before chemotherapy drugs kill the immune cells in their bone marrow. The stem cells will then be returned to rebuild their marrow, creating a fresh batch of immune cells.

The immunologist leading the study, Richard Burt of Northwestern University’s Feinberg School of Medicine in Chicago, reported in January that the procedure reduced disability in 21 patients in a pilot trial. He and his colleagues aim to show by the end of 2012 that the therapy can roll back symptoms in a 110-patient trial.

McHenry, who is scheduled to start the stem-cell treatment in July, said she hopes it will halt the worsening of her disease before it causes more-severe disability.

“If it were to stop the progression, even for five years, that gives me five more years to live life and enjoy it,” she said. “If not, I hope they learn from me what it’s going to take to make it work for someone else.”

read more» Read more...

FDA Approves New Drug for Deadly Kidney Cancer

A drug from Novartis has won US approval as a treatment for patients with kidney cancer that has returned after treatment with older drugs.

The US Food and Drug Administration approved the pill to treat renal cell cancer, the most common form of kidney cancer.

The agency said patients tested with Novartis' Afinitor lived more than twice as long without tumor growth as those who didn't receive the drug. The study by the Swiss drugmaker showed Afinitor delayed tumor growth nearly five months, compared with less than two months for patients not taking the drug.

Afinitor works by blocking a protein that helps cancer cells divide and grow. The drug was approved for patients who have already taken Pfizer Inc.'s Sutent and Bayer's Nexavar.

Kidney cancer is resistant to chemotherapy and radiation therapy, and the most effective remedy is usually to surgically remove the kidney. When the cancer is isolated to the kidney, a majority of patients will survive at least five years. If the cancer has spread to other parts of the body, the survival rate is much shorter.

About 13,000 patients died from the disease last year, according to the American Cancer Society.

Basel, Switzerland-based Novartis has submitted the drug for approval in the European Union, Japan, and elsewhere.

Source : www.dddmag.com

read more» Read more...