Neurocrine Biosciences Inc. Awarded an STTR Grant From the NIH to Develop Hypocretin Treatment for Narcolepsy SAN DIEGO, Dec. 18 /PRNewswire/ --
Neurocrine Biosciences Inc. (Nasdaq: NBIX) announced
today that it has received a Phase I Small Business
Technology Transfer (STTR) grant from the National
Institutes of Health (NIH) to develop a treatment for
narcolepsy. This grant will help fund research in a
collaborative effort between Neurocrine and the
laboratory of Dr. Emmanuel Mignot, M.D., Ph.D., Associate
Professor of Psychiatry and Behavioral Sciences,
Director, Center for Narcolepsy, Stanford University
School of Medicine/Sleep Research Center. Dr. Mignot and
his colleagues were the first to discover the link
between the hypocretin system and narcolepsy, a sleep
disorder. The development of an orally active, small
molecule agonist to the hypocretin-2 receptor may provide
a suitable therapy for patients with narcolepsy. Lack of Brain Protein Causes Sleeping Disorder "Narcolepsy" in Humans STANFORD - A year after finding the defect that causes narcolepsy in dogs, Stanford researchers have made the leap from canines to humans to show that people with the sleep disorder have a breakdown in the same molecular pathway. The finding sets the stage for a new form of treatment for the disabling condition. "Narcolepsy is a very debilitating disease with massive social consequences," said Juliette Faraco, PhD, one of the primary authors of the new study, which will appear in the September 1 issue of Nature Medicine. There is now a specific drug target to work on, Faraco said, which gives new hopes for a cure for narcolepsy. The Stanford team, led by Emmanuel Mignot, MD, PhD, associate professor of psychiatry and director of the Stanford Center for Narcolepsy, searched for defects in the brains and genes of narcolepsy patients from around the world. The team found that a small protein - or peptide - present in the brain cells of normal people was absent in every narcoleptic brain that they studied "We think this is the cause of most human cases of narcolepsy - that they don't have this peptide in the brain," said Mignot. "Now we need to develop a drug that can go into the brain to replace it.". The peptide, called hypocretin, was first identified as a culprit for narcolepsy when Mignot and his colleagues found that dogs with narcolepsy had a problem in one of the hypocretin genes. Hypocretin was being produced in the animals but there was a breakdown in the communication system that allowed hypocretin-generated messages to be transmitted into the cell. Surprisingly, the researchers found that people with narcolepsy generally have intact hypocretin genes. But they found that there is a breakdown further along the hypocretin production line. The peptide is not being produced where it is needed - in the brain - where hypocretin seems to be responsible for promoting wakefulness. The cells that make hypocretin were either completely missing in the brains of narcolepsy patients, or the few cells that remained were not making the peptide. According to Faraco, there are 10,000-15,000 of these cells in normal brains and none in narcolepsy brains. "We think that there's something that specifically kills the cells that make hypocretin," said Mignot. "We don't know how or why, but it's most likely an autoimmune disease." Mignot likens the loss of hypocretin-producing neurons in narcolepsy patients to other disorders such as the loss of insulin-producing cells that leads to diabetes and the loss of dopamine-producing neurons associated with Parkinson's disease. Faraco is a postdoctoral fellow in the laboratory of Emmanuel Mignot. Christelle Peyron, PhD, is co-lead author of the study. Researchers from Leiden University Medical Center, Netherlands; H.U.G., Belle-idee, Division de Neuropsychiatrie, Geneva, Switzerland; Charles University,Prague,Czech Republic; University of Michigan and Geriatrics, Educational and Clinical Center VAMC, Ann Arbor, Michigan; Albert Einstein college of Medicine, New York; and Neurocrine Biosciences Inc, San Diego, California, also contributed to the study. Funding was provided by the National Institutes of Health. Media Contact: Kristin Weidenbach For commentary on the various reactions to this news, please read "There Is No Cure For Narcolepsy" Cephalon Reports That a Recent Study Identifies a Possible Mechanism of Action Of PROVIGIL-Induced Wakefulness WEST
CHESTER, Pa., Nov. 29 /PRNewswire/ -- Cephalon, Inc.
(Nasdaq: CEPH) reports today that a preclinical study
published in the November 15 issue of the Journal of
Neuroscience provides further evidence that PROVIGIL(R)
(modafinil) activates specific neuronal pathways that are
believed to regulate normal wakefulness. In this study,
modafinil strongly activated two well- defined groups of
nerve cells located in the hypothalamus that have been
implicated in the control of wakefulness - the
tuberomammillary nucleus (TMN) and orexin-containing
nerve cells. This mechanism of action is different from
the pathways affected by traditional stimulants. |
ORPHAN MEDICAL ANNOUNCES FDA APPROVAL OF XYREM® July 17, 2002 MINNEAPOLIS – July 17, 2002– Orphan Medical, Inc. (Nasdaq: ORPH) announced today that the U.S. Food and Drug Administration (FDA) has approved Xyrem® (sodium oxybate) oral solution to treat cataplexy, a sudden loss of muscle tone associated with narcolepsy. Xyrem is the first approved medication indicated for the treatment of cataplexy. Narcolepsy is a chronic neurological disorder affecting approximately 140,000 Americans. An estimated 60-90 percent of narcolepsy patients experience cataplexy. Cataplexy is a debilitating symptom of narcolepsy usually triggered by strong emotions such as laughter, anger, or surprise. In its most severe form, cataplexy can cause a person to collapse during waking hours. Xyrem (sodium oxybate) is a central nervous system (CNS) depressant and should not be used in conjunction with alcohol or other CNS depressants. Sodium oxybate is GHB (gamma hydroxybutyrate), a known drug of abuse. The abuse of GHB has been associated with a number of important CNS adverse clinical events, including seizure, respiratory depression, and profound decreases in level of consciousness, with instances of coma and death. Even at recommended doses, use has been associated with confusion and other neuropsychiatric events. Reports of respiratory difficulties occurred in clinical trials. Xyrem is a Schedule III controlled substance. In addition, its distribution is governed by the FDA’s Subpart H regulations. To comply with these regulations, the Company has developed a rigorous system that makes Xyrem available to patients from a single, specialty pharmacy. Both physicians and patients must receive an education program from the Company before obtaining Xyrem. Orphan Medical has worked closely with the FDA, DEA and law enforcement agencies to develop strict distribution and risk-management controls designed to restrict access to Xyrem to the intended patient population. The Company has begun the hiring and training of approximately 35 additional sales employees who will call on accredited sleep centers, and other physician specialists treating those with cataplexy. The Company plans to launch Xyrem at the beginning of the fourth quarter. “We have worked with physicians, patients, and FDA for nearly eight years to bring Xyrem to patients with cataplexy,” said John H. Bullion, Orphan Medical Chairman and Chief Executive Officer. “We commend the FDA’s action that recognizes the therapeutic value of this important product. We are very pleased with this long-sought approval, and we are continuing work to understand fully Xyrem’s mechanism of action. We also expect to complete by the end of 2002 the clinical portion of the Phase III(b) trial designed to assess the efficacy of Xyrem in incrementally reducing excessive daytime sleepiness as a supplement to stimulant therapy.” Please join the management of Orphan Medical at 9 am Eastern time for a conference call to discuss today’s news announcement regarding Xyrem. The dial in numbers are 877-626-0595 (Domestic) and 706-634-1208 (International). The pass code to enter the call is 4839700. This call is also available on the Internet at: http://www.firstcallevents.com/service/ajwz362841482gf12.html On July 23, 2002, Orphan Medical will hold a conference call to discuss its second quarter results and provide guidance for upcoming quarters. Orphan Medical acquires, develops, and markets pharmaceuticals of high medical value for inadequately treated and uncommon diseases treated by specialist physicians. Orphan Medical's Internet Web site address is www.orphan.com Also be sure and visit the narcolepsy chats and message boards for more news and reactions to the FDA's approval of Xyrem at: FDA Has Accepted Orphan Medical's Response To Approvable Letter For Xyrem (May 2002)Orphan Medical, Inc. (Nasdaq: ORPH) announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's complete response to the Approvable Letter for Xyrem® (sodium oxybate) oral solution of April 9, 2002. The Agency stated that it would take action on the Xyrem NDA by July 17, 2002. The response made by the Company includes clarification of respiratory data and revisions to labeling as requested by the FDA. In its Approvable Letter of April 9, the FDA also indicated that it would conduct additional clinical trial site review. The FDA has completed such site review with no issued findings. While Orphan Medical believes that additional clinical site investigations are unlikely at this time, the FDA has the right to perform any additional clinical site investigations they deem necessary."We are pleased that the FDA has promptly accepted our response of May 17th for review, said John Bullion, Orphan Medical Chief Executive Officer. We are confident that our response addresses the issues raised in the recent FDA Approvable Letter and we expect approval of Xyrem within sixty days to finally make this important treatment for cataplexy available to those with narcolepsy." Narcolepsy is a chronic neurological disorder affecting approximately 140,000 Americans. The main symptoms of narcolepsy are excessive daytime sleepiness and cataplexy. Cataplexy is a debilitating symptom characterized by the sudden partial or total loss of muscle control in response to strong emotions such as laughter, anger, or surprise. Orphan Medical acquires, develops, and markets pharmaceuticals of high medical value for inadequately treated and uncommon diseases treated by specialist physicians. Orphan Medical's Internet Web site address is http;//www.orphan.com Barr Receives Approval for Generic Dextrostat(R) Tablets POMONA, N.Y., Feb. 1 /PRNewswire/ -- Barr Laboratories, Inc. (NYSE: BRL) today announced that it has received approval from the U.S. Food & Drug Administration for Dextroamphetamine Sulfate Tablets, USP 5 mg and 10 mg the generic equivalent of Shire Richwood Inc.'s Dextrostat(R) tablets. The Company expects to be the first to market generic Dextrostat and anticipates launching the product this month. "With this approval the Company expands its traditional generic business focus to include a new therapeutic category and demonstrates our ability to accelerate product development to ensure that we reach the marketplace before competitors," said Bruce L. Downey, Barr's Chairman and CEO. Dextrostat is indicated for Narcolepsy and in Attention Deficit Disorder with Hyperactivity, as an integral part of a total treatment program which typically includes other remedial measures for a stabilizing effect in pediatric patients. Current annual sales are approximately $20 million. CeNeS Says Tests for Sleep-Disorder Drug Positive London, May 18 (Bloomberg) -- CeNeS Pharmaceuticals Plc, a U.K. drugmaker specializing in central nervous system treatments, said second-phase drug trials for its sleep-disorder drug showed it could improve sleep patterns. By delaying the start of rapid eye movement, or REM, the compound CEE 03-310 could provide longer, more stable sleep to patients with conditions such as depression and narcolepsy, it said. CeNeS shares rose 4 pence, or 4.9 percent, to 85.5p. CeNeS said REM sleep patterns are affected in several disorders, including schizophrenia. With the completion of the Phase II trials -- the second of three stages normally required -- CeNeS will conduct further studies on the compound, especially in older patients. Dreaming occurs within REM periods. The company also said it started second-phase studies of Sipatrigine, a stroke treatment, in France and Germany last year, and is in talks with other trials centers to expand the number of patients enrolled. Back to: Narcolepsy - A Sleep Disorder
I have added a message board to my site. It is called the "NARCOLEPSY MESSAGE BOARD AND HELP FORUM". Please come, visit, post a message or answer a question or two...or three... |
