Archive for the ‘Medicinal Chemistry’ Category
Genome Maps Solve Medical Mystery For Calif. Twins
Last Updated on Thursday, 16 June 2011 10:51 Written by admin Thursday, 16 June 2011 10:51
Ever since scientists began to sequence the entire genomes of individuals —beginning with those of Nobelist James Watson and scientific entrepreneur J. Craig Venter in 2007 — skeptics have wondered just how useful this elegant and expensive trick would become.
A pair of 14-year-old twins, Alexis and Noah Beery, now provide a compelling answer, even if it’s not yet clear how generalizable their case is to others with genetic disorders.
Whole-genome sequencing has enabled doctors to provide the Beery twins with a simple, highly effective treatment for a rare condition called DRD, or dopa-responsive dystonia. The tale of their cure appears in this week’s issue of the journal Science Translational Medicine.
The twins were diagnosed with cerebral palsy at age two. But their mother, Retta Beery, didn’t think that was correct. For one thing, Alexis’s contorted posture and jerky movements always seemed to be better in the morning and increased as the day went on.
Turns out DRD is known for these diurnal variations, as Retta found out through dogged research. That led to a diagnosis of DRD when the twins were five. Since DRD was thought to be a deficiency of the neurotransmitter dopamine, low doses of a drug called L-dopa (also used for Parkinson’s disease) rather miraculously made the twins’ “cerebral palsy” go away within days.
But other symptoms persisted and worsened. At age 14, Noah had hand tremors, awkwardness and attentional problems. More alarmingly, Alexis had breathing problems due to spasms in her larynx. But when doctors probed for an explanation of these symptoms, the twins tested negative for known mutations of two genes known to be involved in DRD.
As it happens, the twins’ father, Joe Beery, works for a California biotech company that makes DNA sequencing machines. So the parents wondered if a deep dive into their twins’ DNA might explain the nature of their particular genetic defect.
Scientists at Baylor College of Medicine, a pioneer in whole-genome sequencing of individuals, thought it was worth a go. They sequenced the genomes of the twins, their older brother, their parents and their grandparents.
Comparing the results, the researchers found that the twins both inherited a gene variant from each parent that, together, led them to have low levels of not just dopamine but two other neurotransmitters, serotonin and noradrenalin.
The twins’ neurologist, Jennifer Friedman of Rady Children’s Hospital in San Diego, suggested giving the teenagers a supplement called 5-HTP that’s a precursor for serotonin.
Together with the L-dopa, the additional supplement has improved Alexis’s breathing point to the point that she’s now running track again. Noah’s handwriting and athletic performance have improved, and he’s better able to focus in school.
And there’s an intriguing bonus. Scientists think the gene mutation that the Beery twins inherited from their mother may be responsible for a pattern of a neuromuscular disease called fibromyalgia in her family. Fibromyalgia sometimes responds to anti-depressants called SSRIs that raise serotonin levels.
If that hypothesis pans out, it would suggest that rare genetic disorders such as DRD are just the most dramatic manifestation – in people who inherit a double dose of certain gene variants – of much more common disorders such as fibromyalgia among people who have a single copy of the mutation.
Study authors say the Beerys’ case shows how genomics will ultimately revolutionize medicine by making diagnosis more precise and pointing toward life-changing treatments. Other cases are beginning to pop up, such as a Wisconsin boy whose rare disease was diagnosed by whole-genome sequencing and subsequently treated with a bone marrow transplant. (His story appeared in a Pulitzer Prize-winning series by the Milwaukee Journal-Sentinel.)
Cost is still a big obstacle. At the time the Beery family’s genomes were sequenced, it cost around $100,000 per person. Dr. Richard Gibbs of Baylor says now, less than two years later, it would cost about half as much – less than $10,000 for the actual sequencing, plus the cost of computer processing of the results and validation.
The skeptics also point out that not all genetic insights from sequencing will lead to such cheap, simple and effective treatments as the Beery twins got.
Source: http://www.npr.org/blogs/health/2011/06/16/137204964/genome-maps-solve-medical-mystery-for-calif-twins
Posted under Cell Analysis, Discoveries, Innovations and Patents, DNA Reasearch, Genetics & Pharmacogenetics, Medicinal Chemistry, North America, Research Projects, USA and Canada | Comments Off
State changes immunization schedule
Last Updated on Thursday, 16 June 2011 10:40 Written by admin Thursday, 16 June 2011 10:40
FRANKFORT Kentucky has changed its child immunization schedule to comply with national recommendations, adding a couple of childhood vaccines and recommending second “booster” immunizations for three other diseases.
“In a nutshell, the changes are to require vaccines to protect children from preventable diseases,” said Dr. William Hacker, M.D., commissioner of the Kentucky Department of Public Health. “Those diseases don’t occur as often as they once did, but they’re still serious but preventable diseases.”
Immunization requirements have been updated to better align the state’s immunization schedule with the recommendations of the Center for Disease Control and the American Academy of Pediatricians.
Hacker said many children are already taking the vaccines because physicians have recommended them.
One of the two new vaccines is pneumococcal vaccine to prevent pneumonia, ear infections and a type of meningitis. It is recommended for children under 5 years of age. The other is meningococcal vaccine to prevent meningitis and blood-borne infections and is recommended for children entering the sixth grade.
The new guidelines also call for three “booster” vaccinations: one for chicken pox for children from kindergarten age to those entering second grade; one for tetanus, diphtheria, and pertussis for those entering sixth grade; and one for measles, mumps and rubella at 6 years old.
“We’re seeing these diseases in kids who have previously been given vaccinations,” Hacker said.
The new requirements take place July 1. Certificates for school or day care entry which are submitted after that date should meet the new vaccine requirements.
Hacker said the additional cost for the vaccines is insignificant compared to the costs of illnesses they prevent.
“These diseases can cause life-threatening infections,” Hacker said. He said local health departments and some other providers offer vaccines priced on the ability to pay.
Source: http://thetimestribune.com/local/x947029853/State-changes-immunization-schedule
Posted under Medicinal Chemistry, North America, USA and Canada | Comments Off
Safety Dooms Novel Staph Vaccine
Last Updated on Wednesday, 8 June 2011 11:23 Written by admin Wednesday, 8 June 2011 11:23
A phase II/III trial of V710, an investigational vaccine for the prevention of Staphylococcus aureus infection, has been stopped for good following a risk-benefit analysis, according to Merck and Intercell AG.
In April, the Data Monitoring Committee recommended suspending enrollment even though a pre-specified interim analysis showed that the trial did not meet futility criteria. No explanation was given. But a decision about whether to continue the trial was put off until an analysis of the risks and benefits could be completed.
After further analysis, the Data Monitoring Committee unanimously recommended termination of the study, according to a statement from the vaccine makers.
The analysis revealed that the vaccine was unlikely to have a significant clinical benefit and may increase the risk of death and multiorgan dysfunction.
“In the additional analyses that were performed, this safety difference was not found to be statistically significant and was also determined not to warrant any action beyond routine safety follow?up,” the statement read.
The trial was designed to evaluate a single injection of V710 for preventing serious S. aureus infections in adult patients who were scheduled to undergo cardiothoracic surgery.
Merck will present the final results of the trial at an upcoming medical meeting.
Source: http://www.medpagetoday.com/InfectiousDisease/Vaccines/26921
Posted under Discoveries, Innovations and Patents, Drug Development, Medicinal Chemistry, New Drugs, North America, Press Releases, USA and Canada | Comments Off
Cholesterol study shows niacin no good at cutting heart risk
Last Updated on Thursday, 26 May 2011 04:36 Written by admin Thursday, 26 May 2011 04:36
(CBS/AP) Niacin seems to be no good, at least when it comes to helping prevent heart attacks. On Thursday the NIH stopped a study because people taking high doses of niacin saw no benefit.
LDL cholesterol is the main cause of clogged arteries. Statin drugs, including brand names like Zocor and Lipitor as well as generic forms – are mainstays in lowering LDL. But many statin users have heart attacks, because LDL isn’t the whole story. Low levels of HDL, as well as high levels of fatty substances known as triglycerides, also increase heart risk.
So scientists are testing whether adding various drugs to statins would increase HDL enough to protect the heart.
The study tested Niaspan, an extended-release form of the B-vitamin niacin that is a far higher dose than is found in dietary supplements. The drug has been sold for years, and previous studies showed that it boosts HDL levels.
More than 3,400 statin users in the U.S. and Canada – people still at risk for heart attack because of low HDL levels – were given Niaspan or a dummy pill in addition to their usual medication. As expected, HDL levels rose and triglyceride levels dropped in the Niaspan users – more than in people who took a statin alone. But the combination treatment didn’t reduce heart attacks, strokes or the need for artery-clearing procedures such as angioplasty, the NIH said.
The University of Washington’s Dr. Jeffrey Probstfield, who helped lead the study, said the finding “is unexpected and a striking contrast to the results of previous trials.”
There was a small rise in stroke risk in the Niaspan users – 28 among 1,718 people compared with 12 among the 1,696 placebo users. The NIH said it was unclear whether that small difference was a coincidence, as previous studies have shown no stroke risk from niacin. In fact, some of the strokes occurred after the Niaspan users quit taking that drug.
Researchers said patients shouldn’t stop taking their Niaspan without talking to a doctor first.
The Mayo Clinic has more on niacin and cholesterol.
Source: http://www.cbsnews.com/8301-504763_162-20066487-10391704.html
Posted under Discoveries, Innovations and Patents, Drug Development, Medicinal Chemistry, North America, Reports, USA and Canada | Comments Off
Researchers Find Cousin of Hepatitis C Virus in Dogs
Last Updated on Tuesday, 24 May 2011 02:45 Written by admin Tuesday, 24 May 2011 02:45
MONDAY, May 23 (HealthDay News) — Researchers report that they’ve discovered a virus similar to the human hepatitis C virus in dogs, a finding that might provide insight into how the germ evolved in people and perhaps lead to better treatments.
About 200 million people around the world are thought to suffer from hepatitis C, including an estimated 3.2 million chronically infected people in the United States. Many don’t know they’re infected with the liver-damaging virus that causes the disease, which means they can spread it to others without realizing it.
The new findings suggest that hepatitis C may have “jumped” from dogs to humans more than five centuries ago, the researchers said.
“Considering the origin of HIV, we expected to find the closest homologs, or genetic relatives, of [hepatitis C virus] in non-human primates,” study author Dr. Amit Kapoor, an investigator with Columbia University’s Mailman School of Public Health’s Center for Infection and Immunity, said in a news release.
“However,” Kapoor added, “while we were analyzing samples from dogs involved in outbreaks of respiratory disease, we came upon a virus that was more similar to HCV than other viruses of the same family. So far, we have only detected [the virus] in sick animals, a few of which had died of unknown causes. Because of its close genetic similarity to HCV, we suggested the name of canine hepacivirus.”
Study co-author Dr. Charles Rice, scientific and executive director of the Center for the Study of Hepatitis C at The Rockefeller University, said in the news release that the beginnings of hepatitis C “remain a mystery. These findings underscore the need to look beyond primates for clues to the origins.”
Scientists say there’s no risk of modern-day dogs infecting people with either human hepatitis C or the canine form.
Hepatitis C is a liver disease that’s typically spread through contact with infected blood. It can also spread through sex with an infected person and from mother to baby during childbirth, according to the U.S. National Institutes of Health.
The study appears in this week’s issue of the Proceedings of the National Academy of Sciences.
Source: http://health.usnews.com/health-news/family-health/digestive-disorders/articles/2011/05/23/researchers-find-cousin-of-hepatitis-c-virus-in-dogs
Posted under Discoveries, Innovations and Patents, Medicinal Chemistry, North America, Research Projects, USA and Canada, Veterinary Research | Comments Off
FDA Okays New HIV Drug
Last Updated on Friday, 20 May 2011 01:56 Written by admin Friday, 20 May 2011 01:56
WASHINGTON — The FDA has approved rilpivirine (Edurant) for the treatment of HIV patients who have not yet begun therapy.
The drug, a non-nucleoside reverse transcriptase inhibitor, is intended to be used as part of highly active anti-retroviral therapy (HAART) with at least two other medications, the agency said.
Rilpivirine, also known as TMC278, is to be taken once a day with food and offers another medication alternative for physicians and patients, the FDA said in a release.
The agency approved the drug on the basis of safety and effectiveness data from two phase III clinical trials with 1,368 adult participants who had not received prior HIV therapy, as well as an extension trial.
They were randomly assigned to treatment with rilpivirine or efavirenz (Sustiva), as well as other anti-HIV drugs.
In the two trials, the proportion of patients who were able to suppress their virus to undetectable levels was 84.3% for rilpivirine and 82.3% for efavirenz.
Those who started the trials with a high viral load and were assigned to rilpivirine were more likely to fail therapy than those who got efavirenz and the converse was true for those who started with a low level of virus, the researchers reported.
On the other hand, patients were more likely to drop out because of side effects if they were taking efavirenz, leading some observers to call the medication a trade-off.
Posted under FDA News, HIV Research, Medicinal Chemistry, New Drugs, New Products | Comments Off
Five New Alzheimer’s Genes Double Total as Doctors Unravel Disease Cause
Last Updated on Monday, 4 April 2011 10:01 Written by admin Monday, 4 April 2011 10:01
Five new genes have been definitively linked to Alzheimer’s disease, doubling the total confirmed by scientists and opening new areas for research into an illness that affects 35 million people globally.
The genetic pathways were reported in two studies involving more than 50,000 people worldwide. Some of the connections found involve systems that control inflammation and cholesterol in the brain, while others affect how brain cells remove toxic proteins, the researchers wrote in reports published yesterday in the journal Nature Genetics.
The newest confirmed genes raise risks for Alzheimer’s by 15 percent or less, not strong enough to be used as a marker for the disease, researchers said. Their worth is in suggesting new areas of study that may one day help speed creation of therapies for a malady that progressively destroys brain cells and makes it difficult for people to think, remember and function.
“We are beginning to piece together the jigsaw and gain new understanding,” said lead researcher Julie Williams, a professor from Cardiff University’s Centre for Neuropsychiatric Genetics and Genomics in Wales. “We still have a long way to go, but the jigsaw is beginning to come together.”
Inflammation, cholesterol and the build-up of beta amyloid protein have long been thought to play a critical role in the degradation of nerve cells in the brain, the researchers said. If treatments can prevent the detrimental effects of the genes, doctors may be able to one day reduce the number of people with Alzheimer’s disease, Williams said.
Important Combinations
Researchers are trying to determine which gene variations, and which combinations, are most important. Identifying how genes work together may speed studies of experimental drugs, said David Bennett, director of the Alzheimer’s Disease Center at Rush University Medical Center in Chicago, in a statement.
“These findings add key information needed to understand the causes of Alzheimer’s disease and should help in discovering approaches to its treatment and prevention,” Bennett said.
The U.S. National Institute on Aging helped fund the studies, which used information gleaned from five different groups, including the Rush Religious Orders Study, to analyze the genetic makeup of more than 54,000 people.
Alzheimer’s is characterized by the formation of plaque in the brain from amyloid and tau proteins. Scientists don’t know why the proteins accumulate or become twisted, whether they cause the illness or if they are an end-product resulting from a different process altogether.
“We know from our studies there are going to be dozens of these genes that will be significant when the collective data is analyzed,” said Rudolph Tanzi, professor of neurology at Harvard Medical School in Boston, and an author of one paper.
Most Exciting
While the combined genes point at several pathways that may play a role in Alzheimer’s disease, Tanzi says he is most excited about CD33, a gene he discovered that is tied to the brain’s primitive innate immune system. In some cases, it might not be eliminating as much beta amyloid as it should. In others, it may be too active and trigger inflammation, he said in a telephone interview.
“Once we figure out what’s going on, CD33 will be a good target because it sits on the cell surface,” he said. “But it will be at least 10 years before we could turn these targets into drugs,” said Tanzi, who is also director of the genetics and aging research unit at Massachusetts General Hospital’s Institute for Neurodegenerative disease.
The number of Alzheimer’s sufferers is predicted to reach 115.4 million by 2050, according to a 2009 report from Alzheimer’s Disease International.
Inevitable Deterioration
No treatment is yet available to slow or stop deterioration of the brain in patients with Alzheimer’s. Drugs aiming to slow the symptoms include Namenda from New York-based Forest Laboratories Inc. (FRX), and Aricept from New York-based Pfizer Inc. (PFE) and Tokyo-based Eisai Co.
As much as 80 percent of a person’s chance of developing Alzheimer’s is inherited, doctors say. About 400 genes have been identified that scientists believe may play a role in the condition, named for the German doctor Alois Alzheimer who described it in 1906.
The greatest inherited risk comes from the APOE gene, discovered in 1993 by a team led by Allen Roses, now director of the Deane Drug Discovery Institute at Duke University Medical Center in Durham, North Carolina. A person who inherits that gene from one parent has a 400 percent increased risk of getting the disease.
Source: http://www.bloomberg.com/news/2011-04-04/five-new-alzheimer-s-genes-double-total-in-new-push-for-cure.html
Posted under Alzheimer's disease, Discoveries, Innovations and Patents, Medicinal Chemistry, North America, USA and Canada | Comments Off
Iona Chemistry Professor Researches Cure For ALZ
Last Updated on Monday, 20 December 2010 03:17 Written by Editor Monday, 20 December 2010 03:17
New Rochelle, NY – Do curry spice, wine and apple skins hold the answer for finding a cure for Alzheimer’s disease, Parkinson’s disease and other neurological disorders?
The results of a laboratory research project, recently published in the Journal of Neurochemistry, show that a chemical compound derived from these natural products may be used in neutralizing the toxic effects of chemicals associated with some debilitating and life-threatening neurological diseases.
The findings are the result of a four-year study undertaken by Terrence Gavin, Ph.D., a chemistry professor at Iona College and Richard M. LoPachin, Ph.D, a neurochemist and director of research in the Department of Anesthesiology at Montefiore Medical Center and the Albert Einstein College of Medicine.
In lab experiments it was found that the compound, called 2-ACP, completely protects nerve cells from the harmful effects of type-2 alkenes. There is growing evidence that exposure to type 2-alkenes, which are found in the smoke inhaled from cigarettes, the exhaust of automobiles and even in French fried potatoes, can increase the chances of developing Alzheimer’s and otherneurological conditions. In addition, studies
have shown type-2 alkenes are being produced within the nerve endings during the disease process that presumably initiates Alzheimer’s.
Dr. Gavin said: “The research Dr. LoPachin and I undertook is promising because chemical compounds extracted from curry spice, red wine and apple skins, which are widely used natural products, have already been clinically demonstrated to have neuroprotective properties. This suggests it would be safe and effective to treat humans with the 2-ACP compound.”
He added: “But, these molecular findings worked in laboratory cultures. We now need to confirm the effects of 2-ACP in animal studies. That will be the focus of our efforts in the coming months.”
In addition, Dr. Gavin and some of his students
at Iona will be looking for new compounds that will be as good or better than 2-ACP in combating the effects of type 2-alkenes. “Our goal is to have new compounds ready for testing in six months. This is a very exciting scientific exploration,” Dr. Gavin stated.
Dr. Gavin has been a chemistry professor at Iona since 1982. He holds a doctoral degree in chemistry from the State University of New York at Stony Brook and attended the State University of New York at New Paltz where he earned a B.A. degree. He and his family live in New Paltz.
Source: westchester.com
Posted under Alzheimer's disease, Clinical Trials, Discoveries, Innovations and Patents, Drug Development, Medicinal Chemistry, Natural Products, North America, Reports, Research Projects, USA and Canada | Comments Off
Consumers Union Raises Concerns About Mercury in Tuna
Last Updated on Monday, 13 December 2010 05:15 Written by Editor Monday, 13 December 2010 05:15
Younger women and children should limit the amount of tuna they eat and pregnant women should not eat tuna at all, because of mercury levels found in the canned and packaged fish, says new report in the January 2011 issue of Consumer Reports.
Albacore or white tuna usually contains far more mercury than light tuna, according to Consumer Reports , and canned tuna is the most common source of mercury in our diet.
In order to test current levels, investigators for the periodical tested 42 samples from cans and pouches of tuna bought mostly in the New York City area. They found all the samples contained measurable levels of mercury, ranging from 0.018 to 0.774 parts per million. Samples of white tuna had 0.217 to 0.774 ppm of mercury and averaged 0.427 ppm. According to Consumer Reports, if a woman of childbearing age ate about half a can of any of the tested samples, she would exceed the daily mercury intake the Environmental Protection Agency considers safe.
The Food and Drug Administration and the EPA guidelines indicate women of childbearing age and young children may eat up to 12 ounces a week of light tuna or other “low in mercury” seafood, including, within that limit, up to 6 ounces per week of white tuna. But Consumer Reports believes that may be too much. Because mercury can cause defects to the nervous system during fetal development, Consumers Union, the nonprofit publisher of Consumer Reports, advises pregnant women, as a precaution, to avoid eating tuna. It also advises that children who weigh more than 45 pounds limit their intake to 12.5 ounces of light tuna or 4 ounces of white tuna per week, and children who weigh less than 45 pounds consume no more than 4 ounces of light tuna or 1.5 ounces of white tuna.
Some critics, including fish producers, say Consumer Reports is overblowing the research.
“Consumer Reports’ suggestion that pregnant women limit the amount of fish they eat, outside of the FDA’s four fish to avoid, is reckless and has the potential to harm public health,” says the National Fisheries Institute in a statement “Peer-reviewed science shows that pregnant women who limit or avoid seafood may actually be introducing risks from omega-3 deficiency. Advising pregnant women to cut canned tuna out of their diet and for others to limit their consumption based merely on a magazine’s editorial opinion is irresponsible.”
But Consumer Reports is asking for change. Due to the results of the investigation, the publication’s editors are asking the FDA strengthen its current guidance and advise pregnant women to avoid tuna altogether. The group also believes the FDA should continue to test for mercury in seafood across the country, and provide U.S. consumers updated information on mercury levels in fish, as well as diet alternatives
“Fortunately, it’s easy to choose lower-mercury fish that are also rich in healthful omega-3 fatty acids,” says Dr. Urvashi Rangan, director of Technical Policy, at Consumers Union. “That’s especially important for women who are pregnant or might become pregnant, nursing mothers, and young children, because fetuses and youngsters are still developing their nervous systems and are therefore at particular risk from methylmercury’s neurotoxic effects.”
Lower mercury seafood includes shrimp, crab and cod.
Source: CNN.com
Posted under FDA News, Medicinal Chemistry | Comments Off
Optimer Pharmaceuticals Receives Milestone Payment From Cempra for Next-Generation Macrolide Antibiotic
Last Updated on Saturday, 24 July 2010 04:26 Written by admin Thursday, 8 July 2010 11:02
SAN DIEGO, July 8 /PRNewswire-FirstCall/ – Optimer Pharmaceuticals, Inc. today announced it has received a $500,000 milestone payment from Cempra Pharmaceuticals, Inc. pursuant to the terms of a licensing agreement between the companies. Â The milestone payment was made as a result of Cempra’s continuing development of a next-generation macrolide (CEM-101) for the treatment of respiratory infections. Cempra licensed CEM-101 from Optimer and has successfully completed a Phase 1 study.
“The successful completion of the CEM-101 Phase 1 study further validates our OPopSâ„¢ drug discovery platform. We are pleased to see this compound progress into the next phase of development by Cempra,” said Pedro Lichtinger, Optimer’s President and CEO.
CEM-101 is a next-generation oral macrolide entering Phase 2 clinical development for the treatment of community-acquired bacterial pneumonia (CABP). Cempra has exclusive worldwide rights, except in Association of Southeast Asian Nations, or ASEAN, countries, from Optimer to discover, develop and commercialize macrolides from a library of more than 500 compounds, including CEM-101, originated from Optimer’s OPopS drug discovery platform.
In vitro and in vivo studies of CEM-101 have shown potent activity against S. pneumoniae as well as an extended spectrum of activity against community-associated methicillin-resistant Staphylococcus aureaus (CA-MRSA), M. avium and in animal models of malaria. These studies have also indicated that CEM-101 is active against atypical bacteria, such as Legionella, Mycoplasma and Ureaplasma and against gonococci and other organisms that cause genitourinary tract infections.
About OPopSâ„¢
Optimer’s OPopS drug discovery platform allows the development of potential drug candidates through carbohydrate mediated medicinal chemistry and enables the rapid synthesis of a wide variety of proprietary molecules. It includes GlycoOptimization, which enables the modification of a carbohydrate group on an existing drug to improve its properties, and De Novo Glycosylation, which introduces new carbohydrate groups on existing drugs to create new patentable compounds with improvement of pharmacokinetics.
About Optimer Pharmaceuticals
Optimer Pharmaceuticals, Inc. is a biopharmaceutical company focused on discovering, developing and commercializing innovative anti-infectives to treat serious infections and address unmet medical needs. Optimer has two late-stage anti-infective product candidates under development. Fidaxomicin is a narrow spectrum antibiotic being developed for the treatment of Clostridium difficile infection (CDI).  In two Phase 3 trials completed by Optimer to study the safety and efficacy of fidaxomicin for the treatment of CDI, fidaxomicin was statistically superior to vancomycin in global cure rate (defined as cure with no recurrence within four weeks of completing therapy) as well as statistically superior in reducing recurrences of CDI by up to 50% when compared with vancomycin, the only FDA approved product for CDI. Pruvel™ is a prodrug in the fluoroquinolone class of antibiotics being developed as a treatment for infectious diarrhea. Optimer has also successfully completed two Phase 3 trials with Pruvel. Additional information can be found at http://www.optimerpharma.com.
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Sanford-Burnham robots speed up path to discovery
Last Updated on Saturday, 24 July 2010 04:26 Written by admin Thursday, 8 July 2010 10:40
The work force at Lake Nona’s Sanford- Burnham Institute includes 90 scientific staffers, 60 administrators and support personnel — and a team of three robots. The robots are part of a $15 million investment from the National Institutes of Health and occupy a large, glass-enclosed room on the bottom floor of Burnham’s Lake Nona campus.
Although they resemble the robotic arms used in automobile assembly lines, these robots have a far different task: filling test tubes faster than a human being. And that, scientists say, will speed up the process of scientific discovery.
“The robots are from the automobile industry,” said Greg Roth, director of medicinal chemistry at Burnham’s Conrad Prebys Center for Chemical Genomics. “We use them because of the accuracy and precision of their movements.”
The robotic arms can fill miniature test-tube “wells” so tiny that 1,536 of them fit on a plate the size of an index card. Using such small test tubes allows researchers to save money on chemicals and compounds.
After filling the wells, the robotic arms take a plate of the tubes and subject them to numerous tests such as varying temperatures and pressures. The robots are connected to a main computer that compiles the data from the millions of tests.
Pharmaceutical companies developed the ultra-high throughput screening technology in the mid-1990s, but universities and nonprofit research institutions such as Burnham did not have access to the robots until recently.
Funded by NIH, the Burnham center and eight others around the country form the institute’s Molecular Libraries Production Centers Network. This resource library and network assists scientists in research and allows results to be shared across the public and private sectors.
The reason for the federal government’s interest? As the results of the Human Genome Project began pouring in, scientists found themselves with so many avenues to explore that they needed faster research procedures.
“My scientists like to say they can do more than a robot, but they can only do it for a day,” said Michelle Palmer, director of screening for the Broad Institute at Harvard and Massachusetts Institute of Technology.
“The humans can do it faster and better, but they can’t sustain it. We need to sustain this over months of effort and get it done exactly the same way every time,” Palmer said.
Posted under Business and Investment, Education, Equipment & Supplies, HT Screening, Medicinal Chemistry, North America, Press Releases | Comments Off
TimTec’s Contract Research Services
Last Updated on Thursday, 8 July 2010 10:18 Written by Editor Thursday, 8 July 2010 03:22
High Quality Organic Synthesis and Medicinal Chemistry Contract Research Services at an Outstanding Value
TimTec brings 15 years of expertise in organic chemistry design and supply for drug discovery closer to your supplemental bench-top requirements by launching The Organic Synthesis and Medicinal Chemistry Contract Research Services Division. TimTec scientists have a proven track record of providing high quality services and showing outstanding integrity to their clients.
TimTec remains flexible in delivering practical chemistry solutions molded to custom research requirements and budgets. All Contract Research Services are delivered with an emphasis on speed, real-time feedback, ongoing communication, superior compound quality, and the protection of our clients’ knowledge resources and interests. We are dedicated to providing these services at exceptionally competitive prices.
The Head of Contract Research Services has over 20 years of research experience in the US pharmaceutical industry as a hands-on medicinal chemist and project Team Leader at large companies and smaller, start-up operations. We are keenly aware of all the intricate details and chemistry challenges that go into bench-top research before a qualified lead molecule emerges. Your TimTec contract research team consists of experienced, bench level scientists who have access to state of the art synthesis, analytical and quality control facilities.
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Potent Novel Tool For Combating Autoimmune Diseases And Leukemia
Last Updated on Thursday, 28 January 2010 06:10 Written by Editor Thursday, 28 January 2010 06:10
A study carried out by the scientists at the Scripps Research Institute illustrated a novel, highly practical strategy for identifying molecules that avert a particular form of immune cells from launching assaults on their host. These findings have added a potent new-fangled tool to the ongoing investigation for probable treatments for autoimmune diseases like MS or multiple sclerosis
, as well as for the treatment of types of leukemia like myeloid leukemia.
The study conducted by Thomas Kodadek, a professor in the Chemistry and Cancer Biology Departments, Scripps Florida, and associates was printed in the ‘Chemistry & Biology’ Journal.
In the novel study, Kodadek and his associates utilised samples taken from an animal model of MS for screening for T cells – a kind of white blood cell that dons fundamental role in the immune system – with an increasing presence in the ailment. The screen additionally recognized molecules that interfered with such T cells’ auto-reactive nature or their assault on the body itself instead of a foreign intruder like a virus or bacteria.
Kodadek stated that their method concurrently unearths and separates auto-reactive T cells along with inhibitors to them. A dual achievement at the core of which is a relative screening procedure of healthy T cells vs. Disease-causative T cells. Even as the process is technically complex and intricate, the thought behind it is not. The scientists intended to make the process of recognizing compounds simpler that could hinder auto-reactive T cells with outstanding specificity and the scientists were able to accomplish their objective.
The scientists employed a model of MS – an autoimmune inflammatory condition that affects the brain and the spinal cord for the study. MS is a condition wherein the immune system assaults the myelin sheath coating and defensive nerve cells that lead to a host of symptoms dependent on what component of the nervous system has been affected. Prevalent signs of the condition involve weariness, numbing sensation; difficulties experienced in walking balancing and co-ordination; dysfunctional bladder and bowel; ocular problems; giddiness and vertigo; sexual dysfunction problems; pain, mental problems; emotional variations and spastic behaviours.
Simplification of the Process
Kodadek and his associates set up the novel method for shedding light on these autoimmune diseases and other kinds of disorders and produced a vast assortment of peptoids –molecules linked to, though more constant as compared to the peptides which made up the proteins. By organizing thousands of the peptides microscopically, the prototype of binding antibodies (a form of autoimmune molecule) and peptoids could be pictured. By observing samples drawn from animal models of an identified disease such as MS, peptoids which exhibited binding to antibodies closely linked with that disease could be easily identified.
Even better, peptoids which showed binding to the autoreactive T cells could be spotted with no awareness of the particular antigen (molecules that elicit the immune assault), offering an impartial approach with which to explore potentially beneficial compounds.
Kodadek stated that they had made a breakthrough where they set up a system that identifies T cell receptors which are copious in an ailing animal and in sapped levels in a healthy animal.
Potential for Curative Breakthrough
The novel process created a novel potential for curative finding. Molecules that targeted auto-reactive T cells in a direct way, while overlooking those T cells that identify foreign antigens, could provide the basis for a new drug development program intended at elimination of autoreactive cells with no affect on the normal functioning of the immune system.
Kodadek stated that the novel study is not the ultimate solution as it employed a model of MS elicited by a sole antigen whereas in human beings there could be 2 to even more antigens that trigger an autoimmune disease like MS that needs further investigation. The method could be applicable with ease to blood cancers, although as the disease-causative T cells have been completely characterized and there being quite a few of them.
Source: simplehealthguide.com
Posted under Medicinal Chemistry, Press Releases, R & D | Comments Off
3rd International Symposium on Advances in Synthetic and Medicinal Chemistry
Last Updated on Sunday, 10 May 2009 10:19 Written by admin Sunday, 10 May 2009 10:19
Kiev, Ukraine, August 23 – 27, 2009
ASMC09 in Kiev is being prepared by EFMC (European Federation for Medicinal Chemistry) and ChemBridge Corporation, in the series of events which started with ASCMC04 Moscow followed by ASMC07 St. Petersburg.
Prof. Erick Carreira, ETH Zurich, Switzerland and Dr Scott Biller, Novartis Institutes for BioMedical Research, Cambridge, USA, have kindly accepted to be the Chairmen of the Symposium.
The scientific program of the International Symposium on Advances in Synthetic and Medicinal Chemistry will include:
* 28 invited plenary lectures, presented by highly recognized scientists from academia and the pharmaceutical and biotech industry from Europe, USA and former USSR countries.
* 10 short oral communications which will be selected from submitted abstracts
* poster sessions
The scientific program will be complemented by an attractive cultural program in Kiev.
The topics to be covered during this symposium include:
* New Synthetic Methodologies, Total Synthesis of Natural Products and Heterocyclic Chemistry
* Diversity- and Target-Oriented Synthesis and Chemical Biology
* Medicinal Chemistry and Drug Discovery & Development
The program will also include a commercial exhibition and a half-day Business Mini-Symposium “Small Molecule Screening Libraries in Drug Discovery: Experience and Trendsâ€.
The official language of the symposium is English.
http://www.ldorganisation.com
Posted under Compound Libraries, Diversity Libraries, Europe, Europe, Heterocyclic Chemistry, Medicinal Chemistry, Press Releases | Comments Off
KINAXO’s Cellular Target Profiling® reveals mTOR as a new target of Celebrex
Last Updated on Sunday, 10 May 2009 08:46 Written by admin Sunday, 10 May 2009 08:46
Martinsried, Germany, April 29, 2009 / b3c newswire / - Kinaxo Biotechnologies GmbH has successfully applied its Cellular Target Profiling® technology to identify the protein kinase mTOR as a new cellular target of celecoxib (Celebrex®, Pfizer). Celecoxib is a non-steroidal, anti-inflammatory Cox-2 inhibitor approved for the treatment of osteoarthritis, rheumatoid arthritis and acute pain. In addition, celecoxib’s anti-proliferative effect has earned it a place in numerous clinical trials against several malignancies.
The discovery that celecoxib targets mTOR contributes to a better understanding of the drug’s mode of action and efficacy in cancer patients. mTOR (mammalian target of rapamycin) acts as a central regulator of cell proliferation, cell survival, angiogenesis and cell metabolism. Moreover, mTOR is a key intracellular convergence point for a number of signaling pathways that are abnormally activated in many types of cancer.
As traditional drug development approaches become more and more expensive, drug repositioning has been widely recognized as an opportunity to expand a drug’s therapeutical applications. Here, Cellular Target Profiling® provides a powerful approach to revealing new targets that indicate additional or alternative medical uses for clinically validated or approved drugs. Further information on celecoxib reprofiling (AN3) and similar studies with other small molecules (e.g. profiling of the natural product geldanamycin, AN2) can be downloaded from our website at www.kinaxo.com.
Link to the news release
http://www.b3cnewswire.com/popup.php?id=149
About KINAXO
Kinaxo Biotechnologies GmbH is a privately-held biotechnology company based in Munich/Martinsried, Germany. We offer advanced chemical proteomics and phosphoproteomics services to support lead compound selection, target deconvolution, drug reprofiling, and off-target toxicity assessment. Kinaxo has several ongoing collaborations, including with Boehringer Ingelheim, Johnson & Johnson and Takeda.
Posted under Cancer Research, Drug Development, Europe, Medicinal Chemistry, Press Releases, Proteomics, Targeted Libraries | Comments Off
Richter-Helm and Athera Biotechnologies Partner in Development of Recombinant Protein to Treat Cardiovascular Disease
Last Updated on Friday, 23 January 2009 10:19 Written by admin Friday, 23 January 2009 10:19
Hamburg, Germany and Stockholm, Sweden, January 21st, 2009 — Richter-Helm BioLogics GmbH & Co. KG and Athera Biotechnologies AB have signed an agreement for the development and manufacturing of Athera’s novel product for prevention of plaque rupture and athero-thrombosis through binding of the protein, Annexin A5, to endothelium. The recombinant protein Annexin A5 is intended for the treatment of patients with Acute Coronary Syndrome and who are at imminent risk for Myocardial Infarction.
This agreement secures a highly cost efficient long-term development and manufacturing plan for Athera, including possible future large volume commercial production. Annexin A5 is a biotechnology product produced using Richter-Helm’s proprietary E. coli based expression system. Richter-Helm will initiate strain and process development of the new process, reaching a 1000 litre production scale.
“The Annexin A5 project fits well with the competence and experience of our company. We are confident that our collaboration will be as efficient and constructive as our negotiations were. Our state-of-the-art facilities are ideal for producing the high-quality Annexin A5 required to comply with the standards of the regulatory agencies.â€, stated Dr. Bert Behnke, Managing Director of Richter-Helm.
“We are very pleased to get Richter-Helm as our development partner, they have an excellent reputation. We have now secured cost competitive and high quality production for clinical and commercial useâ€, said Carina Schmidt, CEO of Athera Biotechnologies.
Posted under Collaborations, Europe, Medicinal Chemistry, Press Releases | Comments Off
International symposium “Stem Cell Transplantation in Multiple Sclerosis, October 5th, 2009 Moscow, Russia
Last Updated on Friday, 23 January 2009 11:42 Written by admin Tuesday, 13 January 2009 06:19
International symposium “Stem Cell Transplantation in Multiple Sclerosis: Sharing the Experience” will be held on October 5th, 2009 in Moscow, Russia. Symposuim Organizers are Pirogov National Medical Surgical Center, National Center for Research and Treatment of Autoimmune Diseases, Russian Cooperative Group for Cellular Therapy, New Jersey Center for Quality of Life and Health Outcomes Research.
The symposium will be the first special meeting fully meant to discuss the state-of-the-art and perspectives of the new and quite promising method of multiple sclerosis treatment – high dose immunosuppressive therapy + autologous hematopoietic stem cell transplantation. It intends to share the newly acquired knowledge in the field, to discuss challenges and perspectives of the method, and to develop collaborative projects. The topics to be covered within the symposium include:
“Â Â Â Regimens of conditioning: Immunoablation or immunosupression?
“Â Â Â Types of transplantation: autologous or allogenic?
“Â Â Â Posttransplant immunological reconstitution
“Â Â Â Side effects
“Â Â Â Outcome measures: clinical, imaging, patient-reported outcomes
“Â Â Â Posttransplant neurorehabilitation
“Â Â Â Long-term follow-up results
“Â Â Â Proposals for cooperative studies
Neurologists, immunologists, transplantologists, hematolosits, specialists in stem cell research are invited to participate in the Symposium.
Key dates:
1 March 2009 -Â Â Deadline for abstract submission
1 April 2009Â Â -Â Â Deadline for early registration
Symposium Organizers Contact Information:
Tel: +7 495 463 4923 or +7 962 710 17 11
Web-site: http://www.stemcellms.ru
Posted under Clinical Trials, Europe, Europe, Medicinal Chemistry, North America, Press Releases | Comments Off
Vanderbilt University Announces Partnership with Janssen Pharmaceutica N.V. for Schizophrenia Drug Research
Last Updated on Monday, 12 January 2009 03:46 Written by Editor Monday, 12 January 2009 03:46
The licensing and research agreement provides for a total of $10 million in upfront payment and committed research funding to the laboratory of Jeffrey Conn, Ph.D., Director of Vanderbilt’s Program in Drug Discovery. Additional payments will be made based on meeting certain milestones and through royalties on product sales.
Vanderbilt will use its state-of-the-art drug discovery infrastructure, including high throughput screening, medicinal chemistry, molecular biology, and pharmacology testing, to create novel compounds with properties compatible with becoming schizophrenia drugs. In addition to carrying selected compounds from the collaboration into clinical development and through commercialization, Janssen Pharmaceutica N.V. will bring its expertise to the partnership through input on compound design, synthesis and later -stage safety and pharmacokinetic studies.
“This is another milestone for our Program in Drug Discovery and we are excited to be teaming up with one of the world’s leading developers of drugs for treatment of schizophrenia, said Conn. “It is also a testament to Vanderbilt’s commitment to new approaches to drug discovery and developing new models by which academic institutions work closely with industry partners to deliver new breakthrough medicines that have a fundamental impact on human health.â€
Stef Heylen, M.D., Chief Medical Officer, CNS Research and Early Development at Janssen, said, “Academic collaborations are an important part of our drug discovery strategy. This collaboration underscores the synergies between industry and academia that can help to create solutions for addressing unmet medical needs. It is a great example of how we can work together with academia to better understand complex diseases and hopefully bring improved treatments to patients.”
Posted under Collaborations, Europe, Medicinal Chemistry, North America, Press Releases | Comments Off
CytRx Unveils Clinical Development Plan for Pipeline Assets
Last Updated on Friday, 12 December 2008 02:47 Written by Editor Friday, 12 December 2008 02:47
Names World-Renowned Cancer Drug Expert Dr. Joseph Rubinfeld as Chief Scientific Advisor
LOS ANGELES–(BUSINESS WIRE)–CytRx Corporation (NASDAQ: CYTR) today unveiled its corporate strategy to focus its internal resources on the clinical development of oncology drug candidates tamibarotene and INNO-206, which the Company believes offer the greatest mix of near-term and medium-term revenue potential among its clinical assets. CytRx will pursue partnerships to advance the clinical development of INNO-406 (bafetinib) and its clinical molecular chaperone portfolio, where it continues to see significant future revenue potential. The Company further intends to use its proprietary high-throughput, high-content drug screening Master Chaperone Regulator Assay (MaCRA) platform to discover additional molecular chaperone drug candidates, including those that may inhibit cancer growth, which will support internal efforts to build an oncology drug franchise or future out-licensing possibilities.
CytRx also announced that Board of Directors’ member Dr. Joseph Rubinfeld has accepted the additional responsibility of Chief Scientific Advisor, and will consult on all aspects of the Company’s oncology development programs while serving as an important interface between the Company and investors, clinicians and industry thought leaders. Dr. Rubinfeld brings substantial expertise in oncology and drug development through his distinguished career. Dr. Rubinfeld was employed at Bristol-Myers Company International Division as Vice President and Director of Research and Development. While at Bristol-Myers, Dr. Rubinfeld was instrumental in licensing the original anticancer line of products, including Mitomycin and Bleomycin. Among other accomplishments, he was among the four co-founders of Amgen, Inc., and founded SuperGen, Inc., where he previously served as CEO, President and Chief Scientific Officer. In his career he has been instrumental in the development of several blockbuster cancer drugs including cisplatinum, etoposide, erythropoietin, decibitene and pentostatin, and the antibiotics amoxicillin and cefadroxil.
Steven A. Kriegsman, CytRx President and CEO said, “We feel that our stockholders are best served by a focus on potential therapeutics for cancer. We believe tamibarotene has strong potential as a revenue generator with a high likelihood for rapid U.S. approval as a third-line treatment for acute promyelocytic leukemia (APL). Our view is based on the substantial clinical history of tamibarotene as an approved treatment of relapsed APL, in Japan and the existing special protocol assessment (SPA) in place with the U.S. Food and Drug Administration (FDA) for our ongoing U.S. registration clinical trial. We are accelerating enrollment in this clinical trial, with the expectation of filing an NDA with the FDA as early as 2010. We are also taking steps to move into a Phase 2 clinical trial with INNO-206, our highly promising targetable pro-drug for the commonly prescribed chemotherapeutic doxorubicin. We believe that INNO-206 could be effective in a wide variety of cancers, including small cell lung cancer, sarcoma, breast and ovarian cancer and Non-Hodgkins Lymphoma.
“Importantly, we expect that we have ample financial resources with our current cash position and investment in RXi Pharmaceuticals Corporation to support this strategy,†according to Mr. Kriegsman. “We have strong oncology expertise within CytRx and are delighted that Dr. Joseph Rubinfeld, our long-time board member who has enjoyed an illustrious career developing cancer drugs, will be taking a leadership role in our oncology programs.â€
Dr. Rubinfeld said, “Having reviewed the extensive data on tamibarotene and INNO-206, I am excited about the potential for these two cancer drug candidates and look forward to working closely with the CytRx management team to advance their clinical development to potential commercialization. I am also encouraged by the Phase 1 data we announced earlier this month with INNO-406, now known as bafetinib, which demonstrated positive, clinical responses in 35% of patients with refractory chronic myeloid leukemia. I believe these results will be instrumental in our search for a partnership for bafetinib.â€
Mr. Kriegsman added, “We also stand behind our view that our orally administered molecular chaperone drug candidates, arimoclomol and iroxanadine, provide enormous potential in addressing large, underserved markets and are convinced that the prudent course to maximize stockholder value in this economic climate is to pursue pharmaceutical partners to share additional development costs for these longer-term programs. We intend to complete our ongoing arimoclomol animal toxicology studies and work aggressively toward lifting the current clinical hold in order to enable this drug candidate to move back into the clinic. At that point, we will seek partners for further development of arimoclomol as a therapeutic treatment for both ALS and stroke recovery. Additionally, iroxanadine has shown significant potential as a therapeutic treatment for diabetic foot ulcers and other diabetic complications, and based on Phase 2 data, we will pursue potential partnerships in cardiovascular conditions.â€
CytRx’s drug portfolio includes the following:
Oncology Drug Candidates:
Tamibarotene: CytRx holds the North American and European rights to tamibarotene, a rationally designed, synthetic retinoid compound designed to potentially avoid toxic side effects of the current first-line APL treatment trans-retinoic acid (ATRA). CytRx is actively enrolling patients in a Phase 2 registration clinical trial, known as STAR-1, with tamibarotene to evaluate its efficacy and safety as a third-line treatment for APL. The registration study is being conducted under a Special Protocol Assessment. The FDA has granted Orphan Drug Designation and Fast Track Designation for the use of tamibarotene in patients with relapsed or refractory APL following treatment with ATRA and arsenic trioxide.
There are currently no approved third-line treatment options for refractory APL patients. CytRx estimates the U.S. market opportunity for tamibarotene in refractory APL at approximately $20 million annually. CytRx scientists are also evaluating clinical strategies for developing tamibarotene as a first-line or second-line APL therapy. The estimated annual market potential in the U.S. and Europe for an expanded label including refractory, maintenance and front-line therapy is $150 million. CytRx also retains an option to expand its licenses for the use of tamibarotene in other cancers including multiple myeloma, myelodysplastic syndrome and certain solid tumors in the U.S., and multiple myeloma, myelodysplastic syndrome and solid tumors, other than hepatocellular carcinoma, in Europe.
INNO-206: This pro-drug derivative of the commonly prescribed chemotherapeutic agent doxorubicin is designed to reduce adverse events by controlling drug release and preferentially targeting the tumor. In a Phase 1 study, INNO-206 was administered in doses at up to six times the standard dosing of doxorubicin without an increase in observed side effects over those historically seen with doxorubicin. Objective clinical responses were seen in patients with sarcoma, breast and lung cancers. The Company plans to evaluate further clinical development of INNO-206 in a wide variety of cancers, including sarcomas, breast and ovarian cancer, and Non-Hodgkins Lymphoma.
INNO-406 (bafetinib): INNO-406 (bafetinib), a potent, orally available, rationally designed, dual Bcr-Abl and Lyn-kinase inhibitor, is being evaluated for the treatment of patients with chronic myeloid leukemia (CML) and other leukemias that have a certain mutation called the Philadelphia Chromosome (Ph+) and are intolerant of or resistant to imatinib (Gleevec®) and second-line tyrosine kinase inhibitors (i.e. dasatinib (Sprycel®) and nilotinib (Tasigna®)). In November 2008, CytRx announced that bafetinib demonstrated positive, clinical responses in 35% of patients with CML in Phase 1 clinical testing. The Phase 1 clinical trial was used to determine the optimal dose prior to Phase 2 clinical efficacy testing.
CML is a type of cancer that starts in blood-forming cells of the bone marrow and invades the blood. In 2007, the American Cancer Society estimated that approximately 4,600 new cases of CML were diagnosed in the U.S. and that the number will increase as the population ages. Current estimates are that worldwide CML prevalence will increase by 10,000 patients a year, reaching a population of 110,000 in 2010. The global market will grow to an estimated $5.5 billion by 2012.
Molecular Chaperone Regulation
CytRx is a leader in molecular chaperone regulation technology. The Company currently has two orally administered, clinical-stage, drug candidates and recently discovered a series of additional compounds that may provide a pipeline for additional drug candidates. The Company’s drug candidates are believed to function by regulating a normal cellular protein repair pathway through the activation or inhibition of “molecular chaperones.” Because damaged proteins are thought to play a role in many diseases, activation of molecular chaperones that help to reduce the accumulation of misfolded proteins may have therapeutic efficacy in a broad range of disease states. Similarly, CytRx believes that the inhibition of molecular chaperones that normally help protect cancer cells from toxic misfolded proteins may result in the selective destruction of cancer cells.
- Arimoclomol: This molecular chaperone regulator drug candidate is being considered as a treatment for amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and stroke recovery. Arimoclomol has been studied in seven Phase 1 and two Phase 2 clinical trials without any significant adverse events. CytRx’s Phase 2b clinical trial with arimoclomol as a treatment for ALS was placed on clinical hold by the FDA in January 2008, unrelated to any data generated by human studies, and additional preclinical toxicology studies are underway to resolve this issue.
- Iroxanadine: CytRx believes that this orally available small molecule compound represents a potentially powerful breakthrough in the treatment of vascular diseases that are caused in part by damage to “vascular endothelium” that lines the inside of blood vessels. CytRx believes that endothelial dysfunction plays a key role in the development of various vascular diseases or their complications including diabetic ulcers, thrombosis, retinopathy, and peripheral artery disease. Preclinical and clinical studies with iroxanadine indicate that it has therapeutic potential for the treatment of cardiovascular atherosclerosis. According to the National Heart, Lung & Blood Institute, atherosclerosis is a leading cause of illness and death in the U.S. and affects approximately 4.6 million people annually.
CytRx San Diego Laboratory: The CytRx San Diego Laboratory is using the Company’s proprietary Master Chaperone Regulator Assay (MaCRA), a cell image-based screening tool that enables the rapid and quantifiable screening of large numbers of small molecule compounds. This technology is used to identify potential drug candidates that modify the activity of a protein known as heat shock transcription factor 1 (Hsf1) and consequently control entire groups of molecular chaperone proteins that repair or degrade toxic misfolded proteins present in diseased cells. Evaluation of the compounds identified in the screen has shown that they exhibit cytoprotective properties in cell culture models of disease. This platform has broad applicability to a range of therapeutic areas, through its ability to identify drug candidates that can either inhibit or amplify molecular chaperone activity. Information related to the development of MaCRA for compound screening was published in the November 2008 issue of the peer-reviewed Journal of Biomolecular Screening.
CytRx Oncology Expertise
Collectively, CytRx’s management and its Board of Directors have brought numerous cancer drugs to market. In addition to Dr. Rubinfeld, the senior managers and directors of CytRx who hold significant oncology experience include: Max Link, Ph.D., Chairman of the Company’s Board of Directors since 1996, who served for a number of years as Chairman and CEO of Sandoz Pharma as well as a director of Alexion Pharmaceuticals, Inc., Celsion Corporation and Discovery Laboratories, Inc.; Jack R. Barber, Ph.D., Chief Scientific Officer, who has significant R&D experience in oncology at Immusol and Viagene, where he most recently served as Head of Oncology; and Shi Chung Ng, Ph.D., Senior Vice President of Research and Development, who has substantial R&D experience at companies such as Abbott and ArQule, Inc., and most recently served as Vice President of Molecular Oncology at Ligand Pharmaceuticals.
About CytRx Corporation
CytRx Corporation is a biopharmaceutical research and development company engaged in the development of high-value human therapeutics. The CytRx drug development pipeline includes programs in clinical development for cancer indications, including tamibarotene in a registration study for the treatment of acute promyelocytic leukemia (APL). CytRx is developing two drug candidates based on its industry-leading molecular chaperone technology, which aims to repair or degrade misfolded proteins associated with disease. The Company owns and operates a research and development facility in San Diego. CytRx also maintains a 45% equity interest in publicly traded RXi Pharmaceuticals, Inc. (NASDAQ: RXII). For more information on the Company, visit www.cytrx.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. Such statements involve risks and uncertainties that could cause actual events or results to differ materially from the events or results described in the forward-looking statements, including risks relating to the outcome or results of any pre-clinical or clinical testing of CytRx’s potential oncology or molecular chaperone drug candidates, including tamibarotene as a third-line treatment for APL, risks related to CytRx’s ability to enter into partnerships to advance the clinical development of INNO-406 and its clinical molecular chaperone portfolio, uncertainties related to the impact of the FDA’s clinical hold on the Company’s arimoclomol clinical trial for ALS on the timing and ability to resume clinical testing at the desired dosage of arimoclomol, the risk that any requirements imposed on the Company’s planned clinical trial designs for ALS or stroke recovery by the FDA as a result of the concerns expressed in their clinical hold of the Company’s ALS program might adversely affect the Company’s ability to demonstrate that arimoclomol is efficacious in treating ALS or stroke patients or cause the Company to cancel one or both of those trials, risks related to CytRx’s need for additional capital or strategic partnerships to fund its ongoing working capital needs and development efforts, risks related to the future market value of CytRx’s investment in RXi and the liquidity of that investment, and the risks and uncertainties described in the most recent annual and quarterly reports filed by CytRx with the Securities and Exchange Commission and current reports filed since the date of CytRx’s most recent annual report. All forward-looking statements are based upon information available to CytRx on the date the statements are first published. CytRx undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
MorphoSys and Galapagos Enter Alliance to Co-develop Novel Therapeutic Antibodies in Bone and Joint Disease
Last Updated on Monday, 1 December 2008 12:59 Written by Editor Monday, 1 December 2008 12:59
Combination of Proprietary Drug Targets and Unique Technologies to Create Range of New Therapeutic Antibodies
MUNICH, GERMANY — (Marketwire) — 11/26/08 — MorphoSys AG (FSE: MOR; Prime StandardSegment, TecDAX) and Galapagos NV (Euronext: GLPG) announced today the launch of a long term co-development alliance aimed at discovering and developing antibody therapies based on novel modes of action in bone and joint disease, including rheumatoid arthritis, osteoporosis and osteoarthritis.
The alliance spans all activities from target discovery through to completion of proof of concept clinical trials of novel therapeutic antibodies. Both companies will contribute their core technologies and expertise to the alliance. Galapagos will provide antibody targetsimplicated in bone and joint disease in addition to its adenoviral target discovery platform to discover further targets for antibody development.MorphoSys will contribute its HuCAL antibody technologies to generate fully human antibodies directed against these targets. The initial goal is to further validate the targets through disease-specific in vitro and in vivotesting of the antibodies. After successful validation, the alliance will select antibody programs for pre-clinical and clinical development.Following proof of concept in human clinical trials, programs will be partnered for subsequent development, approval and marketing.
Under the terms of the agreement, Galapagos and MorphoSys will share the research and development costs, as well as all future revenues equally.Decisions will be made by a Joint Steering Committee comprising members of both companies. An initial set of three targets implicated in bone and joint disease has been selected for the collaboration, and Galapagos isalready commencing with production of these proteins for the alliance.Generation of antibodies directed against these targets will start in2009. More targets will be selected using Galapagos’ target discovery platform to fuel the alliance in the coming years. If successful, the first antibody programs based on these novel targets could enter the clinic within four to five years.
“With this alliance, we are adding a biologics strategy to our small molecule drug discovery. Galapagos is the world leader in discovery ofnovel targets, and this alliance with MorphoSys enables us to explore the potential of proprietary antibody targets. Antibody approaches have provento be successful in developing new therapies for major diseases, including rheumatoid arthritis. Having both approaches, small molecules andantibodies, to fill our product pipeline in bone and joint disease willfurther establish Galapagos as the leader in this field,” said Onno van deStolpe, Chief Executive Officer of Galapagos. “With our cash position and revenue streams from both BioFocus DPI and our pharma alliances, we are in a good financial position to enter into this alliance to create value for our shareholders.”
“This alliance represents a major step in our efforts to gain access to novel antibody targets for proprietary drug development in disease areas with a high unmet medical need. The partnership with Galapagos combines both the scientific and financial strength of two leading companies in their space,” said Dr. Simon Moroney, Chief Executive Officer of MorphoSys. “We are excited to combine our broad antibody expertise with Galapagos’ target discovery capabilities and disease know-how to form a successful partnership. The access to novel disease-related target molecules from a renowned partner accelerates the expansion of our proprietary antibody pipeline. This alliance also complements our development efforts in the field of inflammation and arthritis includingour lead program MOR103.”
With this strategic alliance, MorphoSys gains access to a proven target discovery engine as well as to Galapagos’ expertise in bone and joint disease, to support its therapeutic antibody pipeline expansion. The threemain indications of bone and joint disease – rheumatoid arthritis,osteoporosis and osteoarthritis – all represent very significant marketopportunities with several million people affected worldwide and combinedsales of drug treatments of more than US$ 15 billion in 2006.
Through the alliance with MorphoSys, Galapagos enters the rapidly growingmarket for therapeutic antibodies. In 2007, total sales for the 20antibody drugs on the market amounted to more than US$ 25 billion andantibody sales are forecast to increase to approximately US$ 50 billion in 2013. Fully human antibodies are recognized as the next generation and the majority of therapeutic antibodies currently in development are humanized or fully human. The average industry timescale from discovery to pre-clinical development of antibody therapies is only two to three years, considerably shorter than the average six years for small molecules.Antibodies also incur lower attrition rates than small molecules.
Galapagos and MorphoSys will conduct a conference call and live audio webcast today at 02:00 p.m. CET (8:00 a.m EST) to provide detailed information on the alliance.Dial-in number for the Conference Call (listen-only):Germany & U.K. residents: +32 2 401 53 06For U.S. residents: +1 866 931 1567 Please dial in 10 minutes before the beginning of the conference.Approximately two hours after the press conference, the archived webcast will be available for replay of the conference on http://www.morphosys.comand http://www.glpg.com.
For further information please contact: Dr. Claudia Gutjahr-Löser, Head ofCorporate Communications & Investor Relations, Tel: +49 (0) 89 / 899 27-122, gutjahr-loeser@morphosys.com or Mario Brkulj, Manager CorporateCommunications & Investor Relations, Tel: +49 (0) 89 / 899 27-454,brkulj@morphosys.com
About Galapagos:
Galapagos (Euronext Brussels: GLPG; Euronext Amsterdam: GLPGA; OTC: GLPYY)is a drug discovery company with pre-clinical programs in bone and jointdiseases and bone metastasis. Its BioFocus DPI division offers a fullsuite of target-to-drug discovery products and services to pharmaceuticaland biotech companies, encompassing target discovery and validation,screening and drug discovery through to delivery of pre-clinicalcandidates. BioFocus DPI also provides adenoviral reagents for rapididentification and validation of novel drug targets, compound libraries fordrug screening as well as chemogenomics and ADMET database products toselect targets and compounds. Galapagos currently employs about 450 peopleand operates facilities in six countries, with global headquarters inMechelen, Belgium. More information about Galapagos and BioFocus DPI canbe found at www.glpg.com and www.biofocusdpi.com.
About Galapagos’ target discovery technology:
Galapagos’ target discovery engine is based on adenoviruses thatefficiently introduce human gene sequences into a wide variety of humancells to knock-down specific proteins. High-throughput assays thatrepresent a selected human disease state are then used to functionallyselect for those proteins that have a causative effect in those models ofhuman disease. After rigorous validation of these protein targets, theyform the basis for the development of novel drugs.
About MorphoSys:
MorphoSys is a publicly traded biotechnology company focused on thegeneration of fully human antibodies as a means to discover and developinnovative antibody-based drugs against life-threatening diseases.MorphoSys’s goal is to establish HuCAL as the technology of choice forantibody generation in research, diagnostics and therapeutic applications.The Company currently has therapeutic and research alliances with themajority of the world’s largest pharmaceutical companies includingBoehringer Ingelheim, Centocor/Johnson & Johnson, Novartis, Pfizer andRoche. Within these partnerships, more than 50 therapeutic antibodyprograms are ongoing in which MorphoSys participates through exclusivelicense and milestones payments as well as royalties on any end products.Additionally, MorphoSys is active in the antibody research market throughits AbD Serotec business unit. The business unit has operations in Germany(Munich), the U.S. (Raleigh, NC) and U.K. (Oxford). For further informationplease visit http://www.morphosys.com/
HuCAL® and HuCAL GOLD® are registered trademarks of MorphoSys AG
Posted under Business and Investment, Clinical Trials, Collaborations, Discoveries, Innovations and Patents, Drug Development, Europe, Industry News, Medicinal Chemistry, New Drugs, News by Region, News by Subject, North America, Press Releases | Comments Off