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Positive data from multiple dose phase1 trial of CTP-354(14/10/14)

Concert Pharmaceuticals Inc. announced Phase 1 data of CTP-354, a novel, potentially first-in-class, non-sedating, once-daily oral treatment for spasticity. In this multiple ascending dose trial, no sedation or ataxia was observed with CTP-354 and the drug was generally well tolerated across all dose cohorts.

Concert expects to initiate a Phase 2 clinical trial evaluating CTP-354 in patients with spasticity associated with spinal cord injury by the end of 2014.

"We are very pleased to have completed our clinical evaluation of CTP-354 in this Phase 1 trial. We remain on track to advance the program into Phase 2 testing later this year, initially targeting spasticity in patients with spinal cord injury followed by the start of a Phase 2 trial in multiple sclerosis patients in early 2015," said Roger Tung, President and Chief Executive Officer.

The Phase 1 multiple ascending dose clinical trial was a randomized, double-blind, placebo-controlled study in 30 healthy volunteers. The primary objective of the trial was to evaluate the safety, tolerability and pharmacokinetics of 10-day repeat dosing of 2 mg, 6 mg and 12 mg of CTP-354.

Clinical highlights include: CTP-354 was generally well tolerated. There were no serious adverse events and no treatment discontinuations. The most common adverse events were dose-related mild and moderate dizziness and somnolence (drowsiness). No sedation or ataxia was observed at the doses evaluated. Across all doses, plasma half-life was approximately 20 hours at steady state. CTP-354 exposure was generally dose-proportional across daily doses ranging from 2 mg to 12 mg. Administration of CTP-354 under both fed and fasted conditions provided similar exposure, indicating that it can be dosed without regard to meals.

The company previously reported positive results from both a Phase 1 single ascending dose trial and a Phase 1 brain imaging trial as measured by positron emission tomography, or PET imaging. The long half-life and pharmacokinetic profile observed in the single ascending dose support once-daily dosing of CTP-354. In addition, CTP-354 provided high and sustained brain GABAA receptor occupancy levels in the brain imaging trial in both single and repeat doses.

Based on the results of the Phase 1 trials, Concert intends to advance CTP-354 into Phase 2 clinical evaluation later this year. The Phase 2 program is projected to include two trials: one evaluating spasticity associated with spinal cord injury and one evaluating spasticity associated with multiple sclerosis.

Source: NASDAQ © NASDAQ OMX Group, Inc (14/10/14)

Prospective drug for Progressive MS, Glialogix receives extra funding(02/10/14)

San Francisco Bay Area-based Glialogix, Inc., a biopharmaceutical company that specializes in developing novel treatments for multiple sclerosis (MS), announced yesterday that they have closed a Sponsored Research Agreement with Fast Forward, a non-profit organization that aims to accelerate MS treatment development. Glialogix will receive funding for one of their pipeline products for neuroprotection, GLX1112, which has shown efficacy in slowing disability progression and potentially repairing neuronal damage — one of the main priorities of the National Multiple Sclerosis Society (NMSS).

Fast Forward, founded by the NMSS, will be supporting Glialogix through cutting-edge pharmacokinetic testing, preclinical models and mechanistic research on GLX1112. According to Thad Reeder, Ph.D., the company’s CSO and lead researcher for the drug, it has already shown promising results in progressive forms of MS.

Glialogix CEO, Mark Moore, Ph.D., explained that there is a great need for effective treatments for progressive MS, as this type tends to cause the most disability and does not respond to treatments indicated for relapsing MS. At present, while there are several drugs being tested for treating progressive forms of MS, most patients are treated with RRMS therapies, as these are the only drugs approved by the FDA for treating progressive MS in the United States. In other countries, progressive MS patients often have no access to therapies, and are forced to cope with symptoms and disabilities on their own.

Dr. Moore concluded by stating that he looks forward to collaborating with Fast Forward on the development of a viable progressive MS therapy, and is confident this new agreement is the boost GLX1112 is waiting for.

Source: Multiple Sclerosis News today © Copyright 2014 BioNews Services (02/10/14)

Successful phase 2a clinical study reinforces novel approach to treat Multiple Sclerosis(04/09/14)

GeNeuro SA announced today positive results from a one-year, open-label extension of a Phase IIa study. GeNeuro tested its monoclonal antibody GNbAC1 in 10 multiple sclerosis (MS) patients, nine of which had primary or secondary progressive disease. GNbAC1 targets the MSRV-Env protein expressed by genes of endogenous retroviral origin, which GeNeuro believe plays a critical role in the pathogenesis of MS.

The long-term safety profile of GNbAC1 was confirmed, with excellent tolerability among patients following repeat administration, verifying results of the earlier Phase I study. With respect to clinical endpoints, the average Expanded Disability Status Score (EDSS), which measures disease progression, remained stable over one year. MRI analysis revealed that the brain images also remained stable and biomarkers associated with the target showed a consistent decline. These results confirm the safety and tolerability of GNbAC1 and support a positive pharmacodynamic response to the treatment. The positive results of this clinical study represent the successful first step in GeNeuro’s Phase II program. A proof-of-concept clinical study to test the efficacy of GNbAC1 in MS will follow in 2015.

Francois Curtin, CEO of GeNeuro stated: “We are very excited by the potential that GNbAC1 offers as a new avenue to treat MS patients. In addition to confirmation of long-term safety of the monoclonal antibody in patients, we have seen that the cohort of progressive MS patients was stable over one year from both clinical and MRI standpoints. Moreover, there is a clear decrease in the associated biomarkers supporting a positive pharmacodynamic response. This reinforces our conviction that GNbAC1 can completely transform the MS therapeutic landscape.” Curtin adds: “Preliminary results of this study presented at the American Academy of Neurology earlier this year generated a high level of enthusiasm among clinicians as they see that this treatment could bring new hope to patients as a safe therapy addressing a causal factor of the disease, breaking with the immunosuppressive logic of existing treatments.”

Source: PipelineReview.com © La Merie Publishing (04/09/14)

Researchers investigating new treatment for multiple sclerosis(28/08/14)

A new treatment under investigation for multiple sclerosis (MS) is safe and tolerable in phase I clinical trials, according to a study published August 27, 2014, in Neurology® Neuroimmunology & Neuroinflammation.

The phase I studies were the first to test the drug candidate in humans. Studies with animals showed that the drug, which is called anti-LINGO-1, or BIIB033, may be able to reverse the demyelination of the nerves. Anti-LINGO-1 blocks LINGO-1, a central nervous system protein that prevents myelination. Current treatments for MS work to reduce new damage to the brain, but do not repair new or past damage.

In MS, the body's immune system begins to attack the myelin that acts as insulation around the nerves in the central nervous system. This makes it more difficult for the nerves to send messages to and from the brain and spinal cord.

In the study, 72 healthy people without MS and 47 people with either relapsing-remitting MS or secondary progressive MS were given the drug or a placebo. The healthy participants received either a placebo or one dose of the drug by an infusion or an injection. The people with MS received either placebo or two intravenous doses of the drug two weeks apart. In both groups, participants received varying amounts of the drug, ranging from 0.1 mg/kg to 100 mg/kg.

The occurrence of side effects was similar for people who received the drug and those who received the placebo. Most side effects were mild to moderate and were not related to the drug. Side effects included headaches, upper respiratory infections and urinary tract infections. There were no serious side effects or deaths.

There were no significant changes in vital signs, EKGs or other safety tests of the drug.

Intravenous doses of 10 mg/kg and higher resulted in concentrations of the drug in the blood that were similar to or higher than the concentration that was associated with 90 percent of the maximum remyelination effect in studies with rats.

"With these results we have been able to start phase II studies to see whether this drug can actually repair the lost myelin in humans and have any effect on restoring physical and cognitive function and improving disability," said study author Diego Cadavid, MD, of Biogen Idec in Cambridge, Mass., which developed the drug. Cadavid is a member of the American Academy of Neurology.

Source: EurekAlert! Copyright ©2014 by AAAS (28/08/14)

Phase I trial of ALKS 8700 to treat multiple sclerosis begins(21/07/14)

Ireland-based biopharmaceutical firm Alkermes has started a Phase I clinical trial of ALKS 8700, a novel monomethyl fumarate (MMF) molecule being developed for the treatment of multiple sclerosis (MS).

The trial will assess the safety, tolerability and pharmacokinetics of several oral formulations of ALKS 8700 compared to both placebo and active control groups in approximately 125 healthy volunteers.

ALKS 8700 is designed to rapidly and efficiently convert to MMF in the body and provide differentiated features compared to the currently marketed dimethyl fumarate, Tecfidera.

Alkermes chief medical officer Elliot Ehrich said: "We expect the results of this study to be highly informative and determine the therapeutic utility and differentiating features of ALKS 8700.

"ALKS 8700 leverages Alkermes' expertise in prodrug chemistry and oral controlled-release formulations to offer potential differentiated tolerability and dosing for patients with MS."

The randomized, double-blind trial will investigate the pharmacokinetics and pharmacodynamics of multiple formulations and doses of ALKS 8700 and is designed to determine those suitable to progress into advanced clinical testing.

The start of the Phase I trial follows the company's filing of an Investigational New Drug (IND) application with the US Food and Drug Administration (FDA), and the issuance of a composition of matter patent for ALKS 8700 from the US Patent and Trademark Office (USPTO) in March 2014, which is expected to provide patent protection into 2033.

Source: PBR Contract Research & Services © PBR 2010. Part of Progressive Digital Media Group Plc (21/07/14)

Could existing common medications help restore myelin in MS patients?(07/07/14)

A common cold treatment and seven other drugs already approved for other conditions could help restore a protective coating eroded around neurons in multiple sclerosis patients, according to researchers led by a team at the University of California, San Francisco.

UCSF is spearheading a 50-patient clinical trial of the most promising drug — an over-the-counter antihistamine branded by Novartis as Tavist — that is expected to be completed by the end of the year.

Researchers warn MS patients not to rush out to buy Tavist, which also is sold generically as clemastine fumarate, because its safety and effectiveness is unknown in MS patients. What’s more, they don’t know what the proper dosage or treatment regimen of the drug might be for multiple sclerosis.

Still, the emergence of Tavist and the seven other drugs is a huge potential win for MS patients and researchers who only 14 months ago launched a new method for quickly screening 1,000 drugs already approved by the Food and Drug Administration for other conditions.

“A major unmet need in the development of therapeutics for repair in MS has been the ability to screen compounds in a high-throughput manner,” Jonah Chan, a neurology professor and senior author of a paper that appeared Sunday in the Journal Nature, said in a press release.

The research group includes scientists from Third Military Medical University in Chongqing, China, the University of Cambridge in the United Kingdom, and Trianja Technologies, north of Dallas.

Multiple sclerosis is a central nervous system disease in which the immune system attacks healthy nerve tissue, destroying the fatty myelin sheaths that are meant to protect the cells. By disrupting the electrical signals from the central nervous system to the body, MS leads to muscle weakness, worsening vision, poor balance or coordination, memory problems and other symptoms.

About 2.3 million worldwide have MS, according to the Multiple Sclerosis International Federation.

Researchers for years have focused potential treatments on soothing the inflammation caused by the intermittent and progressively worsening immune system attacks. Over the past decade, however, they have focused much of their work on stopping the erosion of myelin and, potentially, restoring myelin and protecting neurons.

Only last month drug maker Roche, the parent company of South San Francisco-based Genentech Inc., and Menlo Park-based venture capital firm Versant Ventures formed a company focused on regrowing myelin in MS patients. The basis for that company is a screening technology developed by Chan and his colleagues at UCSF.

The new automated system crafted by Chan’s research group, supported by donations to UCSF's MS Research Group, UCSF's Clinical and Translational Science Institute and the National Multiple Sclerosis Society, quickly tested if 1,000 FDA-approved drugs had any effect on oligodendrocyte precursor cells. So-called OPCs are the cells from which oligodendrocytes are derived in the brain and spinal cord, and it is those specialized oligodendrocytes that myelinate extensions of neurons that transmit signals to the brain. All eight drugs identified by the research team have a common mechanism of action — blocking a specific receptor — but clemastine was the most effective, according to UCSF. But there are five types of that receptor expressed in the nervous system and researchers want to know if clemastine blocks a single receptor of a combination.

In the ongoing Phase II trial — the second of the typically three-stage FDA drug-approval process — researchers mainly want to see if clemastine has an effect on MS patients’ vision at one, three and five months of treatment.

Patients in the trial will receive one four-milligram tablet of clemastine or a placebo twice a day for three months.

Source: San Francisco Business Times © 2014 American City Business Journals (07/07/14)

Research may lead to new drug for neurological diseases(27/02/14)

A casual conversation in a hockey locker room prompted a business partnership that may eventually help people suffering from multiple sclerosis and other neuro-degenerative diseases.

Joseph S. Erlichman, a biology professor at St. Lawrence University, and Mark L. Brackett, president of the Kinney Drug Inc. Foundation, are working with others to develop a drug to treat diseases such as MS and Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s Disease.

After playing hockey one evening, the two men discussed research that Mr. Erlichman and a few of his colleagues were conducting on live brain slices from mice.

The study focuses on observing how the compound cerium oxide affects neuro-damage that’s taken place in the mice. The rodents are observed after they’re placed on a motor-skill apparatus like a treadmill or balance beam to see if they show improvement after taking the cerium oxide.

For the past several years, students from SLU have been involved in that part of the research.

Mr. Erlichman said the results have been promising so far.

“We’ve been accruing an enormous amount of data,” he said. “We’ve found this has the potential to mitigate or decrease tissue damage or disease progression in a wide variety of pathologies.”

Essentially, the compound is effective at neutralizing free radicals that are associated with many pathological diseases, Mr. Erlichman said.

For multiple sclerosis, the compound appears to be as effective as Gilenya, a drug now used to treat the disease in humans, he said.

“We have also used the nanoparticles in the treatment of a mouse model of ALS with promising results,” he said.

In 2009 Mr. Brackett and Mr. Erlichman created the company Neuroredox, with Mr. Brackett serving as its chief executive officer.

Later they teamed up with Cerion Enterprises, a Rochester-based company, to create a new joint venture company, erion NRx, which will continue the necessary steps involved with developing the drug.

Other SLU professors have also been involved in the research, including William DeCouteau, Ana Y. Estevez, Karin Heckman and Matthew C. Skeels.

Upcoming steps include receiving approval from the Food and Drug Administration to test the drug’s effectiveness on humans. Determining whether the drug is toxic to humans will be a main factor in determining if it can be marketed to a pharmaceutical company and then sold to the public, Mr. Brackett said.

Finding a pharmaceutical company to invest in the product and applying for federal funding are other hurdles.

“You have to get all the ducks in a row,” Mr. Brackett said. “We have hired a consultant who has taken other drugs to market.”

Mr. Brackett, a former pharmacist, said he has been interested in neurological diseases for many years.

“These diseases cause a great deal of devastation and pain to families. We think we can help that,” he said.

Source: Watertown Daily Times (27/02/14)

Potential new treatment for multiple sclerosis to be tested(26/02/14)

Potential 'cure' for multiple sclerosis to be tested The National Center of Neurology and Psychiatry has developed a drug that it says might provide a cure to multiple sclerosis.

The center announced Monday that it will start a three-month clinical trial from March on nine patients. If the drug’s efficacy is confirmed, it will move on to a large-scale trial.

“We are hopeful as preliminary studies have produced very good results,” said Takashi Yamamura, head of the immunology department responsible for the drug’s development. About 2.5 million people around the world are estimated to suffer from the disease, which causes symptoms such as numbness, motion problems and vision loss. In Japan, there are an estimated 15,000 sufferers, including many young women, and the number is growing.

The autoimmune disease occurs when lymphocyte immune cells misidentify the body’s own cells as foreign. They attack nerve cells, causing inflammation and destroying them.

The drug developed by Yamamura stimulates a type of immune cell that softens the attacks by lymphocytes and creates a protein that suppresses inflammation. In the clinical trial, the drug will be drunk in powder form dissolved in water, according to the center.


Could hypertension drug stimulate repair in Multiple Sclerosis?(28/01/14)

Quite often, the development of novel therapies for one disease indication later gives rise to its application for other diseases as well. In the case of Multiple Sclerosis treatment, the non-profit Myelin Repair Foundation (MRF) may have identified a proven drug for hypertension that could be used to stimulate repair and protect the brain in MS patients.

The discovery will be further explored in a new clinical trial that will be facilitated through a Cooperative Research and Development Agreement (CRADA) between the MRF and the National Institutes of Health’s National Institute of Neurological Disorders and Stroke (NINDS) at the NIH Clinical Center, which will seek to assess the use of hypertension drug MRF-008 as a potential neuroprotective therapeutic for Multiple Sclerosis.

The collaboration between the MRF and NIH is a unique one, since the Myelin Repair Foundation is a true non-profit research group that directs its research goals from a completely practical, patient-driven perspective, seeking to develop tangible Multiple Sclerosis treatments that can be realistically developed and commercialized. The Foundation’s commitment to publishing provable, reproducible results, together with the vast talent and resources at the NIH, bode well for fully exploring the efficacy of MRF-008 patients.

Scott Johnson, CEO, President and Founder of the Myelin Repair Foundation and himself a Secondary Progressive MS patient seeking viable, effective treatments in the near future, commented on the Foundation’s new collaboration with the NIH and what it could mean to future MS treatment options, stating, “As a non-profit organization beholden to patients, not profits, we are uniquely positioned to advance MRF-008, a generic drug identified by the MRF academic consortium, forward as a novel therapeutic candidate to stimulate repair for multiple sclerosis,” adding that, “With the NIH’s eminent expertise in MS clinical trials, we have found an exemplary partner to conduct the research necessary to assess MRF-008. With world-class advisors, academic scientists, industry partners and this opportunity to collaborate with NIH scientists, we remain on track to develop and deliver the next generation of MS therapeutics for patients.”

The MRF delivers substantial results from its research through the Foundation’s Accelerated Research Collaboration™(ARC™) model, which is designed to accelerate promising therapeutics to market through streamlining the research and development of promising compounds from the lab to the clinic, and ultimately to Multiple Sclerosis patients. MRF leveraged its ARC model to forge its new relationship with the NIH on studying the viability of MRF-008, and will continue to utilize the platform to facilitate partnerships between top academic scientists, pharmaceutical partners, and other key biotech and life sciences influencers.

The Myelin Repair Foundation will work closely with the NIH to assess MRF-008 as a therapeutic candidate in an MS clinical trial. MRF-008 is a generic FDA-approved compound for the treatment of hypertension identified by the Myelin Repair Foundation’s academic research consortium as a novel drug repurposing candidate for neuroprotection to stimulate MS repair. Dr. Irene Cortese, M.D. and Dr. Daniel Reich, M.D., Ph.D. will lead the research study at the NIH.

Visit the Myelin Repair Foundation to learn more about MRF-008.

Source: Bionews Texas (28/01/14)

Siponimod study in secondary-progressive multiple sclerosis(27/01/14)

A new clinical trial is underway for individuals who have secondary progressive multiple sclerosis (SPMS). Patients with SPMS generally have had relapsing-remitting MS, but now are having attacks that are less frequent and less well defined. Patients don’t generally recuperate from these attacks and disabilities begin set in. It is reported that 80% of patients with relapsing-remitting MS eventually develop secondary progressive Multiple Sclerosis, and this process tends to begin 15 or so years after being diagnosed with MS.

A Phase 3 trial will be held worldwide and is recruiting 1,530 individuals who have secondary progressive multiple sclerosis (SPMS) to test the safety and effectiveness of an experimental oral therapy siponimod (BAF312) manufactured by Novartis Pharmaceuticals AG versus an inactive placebo.

The drug, siponimod, is an immune system-modulating treatment designed to be a more selective sphingosine 1-phosphate receptor modulator than Gilenya (fingolimod, Novartis). Gilenya was approved in 2010 for adults with relapsing forms of MS to decrease the frequency of relapses to delay accumulation of physical disabilities.

In a Phase 2 trial, siponimod demonstrated safety and efficacy on MRI scans in individuals with relapsing-remitting MS. Information on this trial is available in The Lancet Neurology, Early Online Publication, June 11, 2013 . Siponimod is believed to act by retaining specific white blood cells in lymph nodes keeping then out of circulation and from getting into the central nervous system. This oral drug can also enter the central nervous system where it may have a direct anti-inflammatory or neurobiological effect. The secondary progressive multiple sclerosis (SPMS) study is currently recruiting participants and is sponsored by Novartis Pharmaceuticals under clinical trial identifier NCT01665144. The purpose of this phase 3 trial is to evaluate the safety and efficacy of Siponimod (BAF312) versus placebo in a variable treatment duration in patients with SPMS.

Patients will be randomized to receive Siponimod (BAF312) for a duration of 23 to 42 months with a maximum of 60 months. Patients who meet all inclusion and none of the exclusion criteria will be treated with BAF312 daily. Eligibility age runs 18 to 60 years and will include both sexes.

Inclusion criteria includes a prior history of relapsing remitting MS, SPMS defined as progressive increase of disability over a least 6 months, EDSS score of 3.0 to 6.5 and relapse of corticosteroid treatment within 3 months.

Of the 1,530 participants, 1,050 will take siponimod and 510 will take the placebo every day for up to 60 months. The primary outcome will look at the delay in to confirmed disability progression as measured by the EDSS scale. This scale has been in MS studies for more than 4 decades and is based on the presence of certain symptoms in a typical neurological exam. Secondary outcomes include disease activity as observed on MRI scans, scales that measure mobility, relapse rates, adverse events and abnormalities on lab tests.

Source: Bionews Texas (27/01/14)

Antioxidant drug may treat multiple sclerosis(02/01/14)

An antioxidant designed by scientists more than 12 years ago to fight damage within human cells can treat symptoms in mice that have a multiple sclerosis-like disease, a new study led by an Indian-origin researcher has found.

The antioxidant - called MitoQ - has shown some promise in fighting neurodegenerative diseases. But this is the first time it has been shown to significantly reverse an MS-like disease in an animal.

The discovery by Oregon Health & Science University could lead to a new way to treat multiple sclerosis, which affects more than 2.5 million people worldwide.

Multiple sclerosis occurs when the body's immune system attacks the myelin, or the protective sheath, surrounding nerve fibres of the central nervous system. Some underlying nerve fibres are destroyed.

Resulting symptoms can include blurred vision and blindness, loss of balance, slurred speech, tremors, numbness and problems with memory and concentration.

The antioxidant research, published in the journal Biochimica et Biophysica Acta Molecular Basis of Disease, was led by P Hemachandra Reddy, an associate scientist in the Division of Neuroscience at OHSU's Oregon National Primate Research Center.

In the study, researchers induced mice to contract a disease called experimental autoimmune encephalomyelitis, or EAE, which is very similar to MS in humans.

They separated mice into four groups: a group with EAE only; a group that was given the EAE, then treated with the MitoQ; a third group that was given the MitoQ first, then given the EAE; and a fourth "control" group of mice without EAE and without any other treatment.

After 14 days, the EAE mice that had been treated with the MitoQ exhibited reduced inflammatory markers and increased neuronal activity in the spinal cord - an affected brain region in MS - that showed their EAE symptoms were being improved by the treatment.

The mice showed reduced loss of axons, or nerve fibres and reduced neurological disabilities associated with the EAE.

The mice that had been pre-treated with the MitoQ showed the least problems. The mice that had been treated with MitoQ after EAE also showed many fewer problems than mice who were just induced to get the EAE and then given no treatment.

"The MitoQ also significantly reduced inflammation of the neurons and reduced demyelination. These results are really exciting. This could be a new front in the fight against MS," Reddy said.

"It appears that MitoQ enters neuronal mitochondria quickly, scavenges free radicals, reduces oxidative insults produced by elevated inflammation, and maintains or even boosts neuronal energy in affected cells," said Reddy.

The hope has been that MitoQ might help treat neurodegenerative diseases like Alzheimer's and Parkinson's. Studies evaluating its helpfulness in treating those diseases are ongoing.

Source: Business Standard Copyrights © 2014 Business Standard Ltd (02/01/14)

Phase IIa study of RNS60 in multiple sclerosis initiated(11/11/13)

Revalesio Corporation has announced a new collaboration with noted neurologist Dr. Roland Martin, Head of Neuroimmunology and MS Research at the Department of Neurology, University Hospital Zurich. Dr. Martin will be conducting a Phase IIa clinical trial of RNS60 in patients with relapsing-remitting multiple sclerosis (RRMS or MS). With 30 years experience, Dr. Martin is a recognized leader in the field of neuroimmunology and multiple sclerosis research.

The study will compare measurements of disease activity and progression in patients with RRMS during 6-months treatment with RNS60. The open label study will enroll 15 patients and evaluate the use of RNS60 by intravenous administration compared to patient's pre-treatment disease activity. The main outcome of the study will be the reduction of inflammatory activity in the brain as measured by Gd contrast-enhacing MRI lesions. Additional measures include progression of disability and biomarkers for RNS60 and general inflammation. The study will be conducted at the University Hospital Zurich in Switzerland and at Innsbruck Medical University in Innsbruck, Austria.

"Our MS development work with RNS60 has been an extraordinary process of discovery and pioneering research," said Dr. Richard Watson, Revalesio's Chief Science Officer. "We have published much of our data regarding RNS60's mechanisms in the pre-clinical models and are pleased to have Dr. Martin as a collaborator and to see RNS60 advance into the clinic for MS. We are hopeful this study will provide an efficacy signal that demonstrates RNS60's therapeutic potential and informs future clinical research."

RNS60 has been tested in numerous pre-clinical models of MS and has shown the ability to halt disease progression by limiting glial infiltration (inflammation in the brain), protecting myelin and enriching the regulatory T-cell (TREG) populations in animals treated with RNS60. Data from these pre-clinical models can be found in the academic journal PLOS ONE.

About RNS60 Revalesio has pioneered the use of RNS60 as a therapeutic that alters whole cell conductance through effects on voltage-sensing membrane-bound proteins, thereby modulating the activity of G protein-coupled receptors and the secretion of cytokines, chemokines resulting in decreased inflammation and cell death. RNS60 contains charge-stabilized nanostructures (CSN) that are created by subjecting normal saline to Taylor-Couette-Poiseuille (TCP) flow. RNS60 has demonstrated a reduction in inflammatory responses that are linked to numerous diseases, including neurodegenerative, respiratory and cardiovascular diseases.

Source: The Sacramento Bee Copyright © The Sacramento Bee 2013 (11/11/13)

Phase 2a GNbAC1 mAb study in MS completes(04/11/13)

GeNeuro announced today that its GNbAC1 humanized monoclonal antibody was found to have a very good safety profile when administered to patients with relapsing and progressive forms of Multiple Sclerosis as part of a Phase 2a study. GNbaC1 is a first-in-class monoclonal antibody targeting a toxic protein of endogenous retroviral origin that has been identified as a potential key factor in the onset and development of multiple sclerosis. Enrollment of patients into a multinational Phase 2b study is expected to begin during the first half 2014.

“The safety data achieved in this Phase IIa study is excellent and supports the future development of GNbaC1 in both relapsing remitting and progressive MS,” commented François Curtin, CEO of GeNeuro. “While presenting at the recent ECTRIMS1 congress in Copenhagen last month, GeNeuro’s approach was highlighted as one of the most innovative and promising new treatments in clinical development against MS as it specifically targets a potential causal factor of the disease.”

In the completed Phase 2a clinical study including a six-month extension, conducted to establish safety and pharmacokinetics, GNbAC1 demonstrated very good safety following repeated administration at 2 mg/kg and 6 mg/kg. The repeated administrations did not affect the immune system, the TLR4 function was preserved and no signs of induction of immunogenicity were observed.

About the ENV toxic Protein and its role in Multiple Sclerosis and other pathologies

The sequencing of the human genome revealed human endogenous retroviruses (HERV) represent more than 8% of the human genome and result from the integration of exogenous retroviruses DNA during the primate evolution.

The Multiple Sclerosis associated retrovirus (MSRV) is a member of the HERV-W family and was initially isolated in cell cultures from patients affected with Multiple Sclerosis in the 90’s. MSRV is normally latent in the genome of individuals, but it can be re-activated by certain co-factors to expresses a pathogenic protein, MSRV-Env. Recent evidence has demonstrated that this ENV-protein is expressed in MS lesions from an early stage, is pro-inflammatory and inhibits remyelination.

Source: Rock Hill Herald Online Copywrite Rock Hill Herald 2013 (04/11/13)

Financial support given for development of NRP2945 as potential therapy for MS progression(14/10/13)

CuroNZ, an Auckland biotechnology start-up company developing treatments for progressive multiple sclerosis, has been awarded funding of US$ 540,000 from the National Multiple Sclerosis Society through Fast Forward to support preclinical studies needed to develop CuroNZ’s NRP2945 candidate as a potential therapy to protect the nervous system from MS damage.

Current therapies available for treating multiple sclerosis do not adequately treat progressive stages of the disease or directly protect the nervous system from that damage that leads to progression.

The funding will enable CuroNZ to undertake pre-clinical proof of concept, pharmacokinetic and toxicity studies to bring the drug candidate lead NRP2945 closer to an investigational new drug (IND) application. As part of the project, CuroNZ will collaborate with leading research organisations including the University of Auckland and Monash University in Melbourne. With its unique mechanism of action and a pristine safety profile to date, NRP2945 has the potential to act as a neuroprotective agent which might eventually be developed into a product that can help patients with progressive MS, worldwide.

Finding ways to stop MS progression is a strategic priority of the National MS Society’s research program. A key aspect of the Society’s comprehensive approach to driving research progress is the support of promising research discoveries – such as NRP2945 - toward commercial drug development.

About CuroNZ: CuroNZ was founded in 2009 by Dr. Frank Sieg who discovered the drug candidate portfolio called Neural Regeneration Peptides (NRPs) in Germany. He subsequently brought them to New Zealand with the hope of being able to eventually develop products that could benefit patients suffering from the debilitating effects of progressive MS.

Source: Scoop Health News © Scoop Media 2013 (14/10/13)

Positive top-line phase II results of Ofatumumab in multiple sclerosis(11/10/13)

Genmab A/S announced top-line results from a Phase II study of the subcutaneous formulation of ofatumumab in relapsing-remitting multiple sclerosis (RRMS).

A total of 232 subjects with RRMS were randomized in the study. There was a clear separation from placebo on the cumulative number of new gadolinium enhancing lesions (active brain lesions) over a period of 12 weeks in subjects treated with all doses of ofatumumab compared to subjects treated with placebo [p < 0.001]. For the primary endpoint, analysis of data from weeks 0-12 estimated a 65% reduction in the cumulative number of new T1 gadolinium enhancing lesions for all doses [p < 0.001]. In weeks 4-12, analyses of data estimated a >= 90% reduction in the cumulative number of new T1 gadolinium enhancing lesions for all cumulative doses of ofatumumab >= 30 mg [p < 0.001].

There were no unexpected safety findings in the study. From weeks 0-12, injection related reactions were the most common adverse reaction and were observed in 52% of subjects receiving ofatumumab compared to 15% of subjects receiving placebo. There were five serious adverse events (SAEs) reported, all subjects received a 60 mg dose of ofatumumab and none of these subjects withdrew from the study. Twelve subjects withdrew during this time period; 10 of these subjects were receiving ofatumumab. To date, no cases of progressive multifocal leukoencephalopathy (PML) or opportunistic infections have been observed.

"We are encouraged by the results from this study, which we believe underline the potential of subcutaneous ofatumumab for treatment of relapsing-remitting multiple sclerosis," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

About the study
This multi-center, randomized, double-blind, placebo controlled Phase II study, conducted by GlaxoSmithKline (GSK), included subjects who had RRMS. The primary objective of the study was to determine whether 3, 30 or 60 mg of ofatumumab given subcutaneously reduces the number of new T1-weighted gadolinium-enhancing brain lesions (active brain lesions) over a period of 12 weeks, as compared with placebo, in subjects with RRMS.

Subjects in the study were randomized to one of the following treatment arms: 3 mg, 30 mg, or 60 mg of subcutaneous ofatumumab every 12 weeks or 60 mg of subcutaneous ofatumumab every 4 weeks, or placebo followed by 3 mg of subcutaneous ofatumumab at week 12. The treatment period for all subjects was 24 weeks; subjects were then followed until B-cell repletion for at least an additional 24 weeks. Currently, all subjects have completed the 24-week treatment period; some subjects continue to be followed as per protocol.

About ofatumumab 
Ofatumumab is a human monoclonal antibody which targets an epitope on the CD20 molecule encompassing parts of the small and large extracellular loopsi. Ofatumumab is being developed under a co-development and collaboration agreement between Genmab and GSK. Under the companies' agreement, GSK is solely responsible for development of ofatumumab in autoimmune indications and all related costs.

Source: The Wall Street Journal Copyright ©2013 Dow Jones & Company, Inc (11/10/13)

Set of compounds to treat multiple sclerosis in new way identified(10/10/13)

Scientists at The Scripps Research Institute (TSRI) have identified a set of compounds that may be used to treat multiple sclerosis (MS) in a new way. Unlike existing MS therapies that suppress the immune system, the compounds boost a population of progenitor cells that can in turn repair MS-damaged nerve fibres.

One of the newly identified compounds, a Parkinson's disease drug called benztropine, was highly effective in treating a standard model of MS in mice, both alone and in combination with existing MS therapies.

"We're excited about these results, and are now considering how to design an initial clinical trial," said Luke L. Lairson, an assistant professor of Chemistry at TSRI and senior author of the study, which is reported online in Nature on October 9, 2013.

Lairson cautioned that benztropine is a drug with dose-related adverse side effects, and has yet to be proven effective at a safe dose in human MS patients. "People shouldn't start using it off-label for MS," he said.

A New Approach

An autoimmune disease of the brain and spinal cord, MS currently affects more than half a million people in North America and Europe, and more than two million worldwide. Its precise triggers are unknown, but certain infections and a lack of vitamin D are thought to be risk factors. The disease is much more common among those of Northern European heritage, and occurs about twice as often in women as in men.

In MS, immune cells known as T cells infiltrate the upper spinal cord and brain, causing inflammation and ultimately the loss of an insulating coating called myelin on some nerve fibers. As nerve fibers lose this myelin coating, they lose their ability to transmit signals efficiently, and in time may begin to degenerate. The resulting symptoms, which commonly occur in a stop-start, "relapsing-remitting" pattern, may include limb weakness, numbness and tingling, fatigue, vision problems, slurred speech, memory difficulties and depression, among other problems.

Current therapies, such as interferon beta, aim to suppress the immune attack that de-myelinates nerve fibers. But they are only partially effective and are apt to have significant adverse side effects.

In the new study, Lairson and his colleagues decided to try a complementary approach, aimed at restoring a population of progenitor cells called oligodendrocytes. These cells normally keep the myelin sheaths of nerve fibers in good repair and in principle could fix these coatings after MS damages them. But oligodendrocyte numbers decline sharply in MS, due to a still-mysterious problem with the stem-like precursor cells that produce them. "Oligodendrocyte precursor cells (OPCs) are present during progressive phases of MS, but for unknown reasons don't mature into functional oligodendrocytes," Lairson said.

A 100,000-Molecule Screen

Using a sophisticated small-molecule screening laboratory that TSRI manages in conjunction with the California Institute of Regenerative Medicine and in collaboration with the California Institute for Biomedical Research (Calibr), Lairson and his team screened a library of about 100,000 diverse compounds for any that could potently induce OPCs to mature or "differentiate."

Several compounds scored well as OPC differentiation-inducers. Most were compounds of unknown activity —but one, benztropine, had been well characterized and indeed was already FDA-approved for treating Parkinson's disease. "That was a surprise, and it meant that we could move forward relatively quickly in testing it," said graduate student Vishal A. Deshmukh, first author of the paper who performed most of these experiments.

With the help of Brian R. Lawson, a senior author of the paper and assistant professor of immunology at TSRI, and his colleague Research Associate Virginie Tardif, Deshmukh set up tests of benztropine in mice with an induced MS-like autoimmune disease—a model commonly used for testing prospective MS drugs.

In these tests, benztropine showed a powerful ability to prevent autoimmune disease and also was effective in treating it after symptoms had arisen—virtually eliminating the disease's ability to relapse. Although benztropine on its own worked about as well as existing treatments, it also showed a remarkable ability to complement these existing treatments, in particular two first-line immune-suppressant therapies, interferon-beta and fingolimod.

"Adding even a suboptimal level of benztropine effectively allowed us, for example, to cut the dose of fingolimod by 90%—and achieve the same disease-modifying effect as a normal dose of fingolimod," said Lawson. "In a clinical setting that dose-lowering could translate into a big reduction in fingolimod's potentially serious side effects." In further analyses, the researchers confirmed that benztropine works against disease in this mouse model by boosting the population of mature oligodendrocytes, which in turn restore the myelin sheaths of damaged nerves—even as the immune attack continues. "The benztropine-treated mice showed no change in the usual signs of inflammation, yet their myelin was mostly intact, suggesting that it was probably being repaired as rapidly as it was being destroyed," said Lawson.

Benztropine is known to have multiple specific effects on brain cells, including the blocking of activity at acetylcholine and histamine receptors and a boosting of activity at dopamine receptors. But Lairson and his colleagues found evidence that the drug stimulates OPCs to differentiate mainly by blocking M1 or M3 acetylcholine receptors on these cells.

In addition to setting up initial clinical trials, Lairson and his team hope to learn more about how benztropine induces OPC maturation, and how its molecular structure might be optimized for this purpose. "We're also looking at some of the other, relatively unknown molecules that we identified in our initial screen, to see if any of those has better clinical potential than benztropine," he said.

"This work, like our previous studies with hematopoietic and mesenchymal stem cells, illustrates the power of small molecules to control stem and precursor cells in ways that may ultimately lead to a new generation of drugs for regenerative medicine," said Peter G. Schultz, the Scripps Family Chair Professor in the Department of Chemistry at TSRI and one of the study's senior authors.

Source: News-Medical.Net (10/10/13)

Karo Bio receives funding from the National MS Society to develop ERbeta Agonists for treatment of progressive MS(13/09/13)

Karo Bio AB has been awarded $0,5 million from the National Multiple Sclerosis Society through Fast Forward to develop a novel treatment – ERbeta agonists -- for progressive forms of Multiple Sclerosis (MS). The funds will finance the further preclinical development of Karo Bio’s advanced compounds.

Karo Bio’s ERbeta agonist has the potential to slow disease progression in multiple sclerosis as preclinical models show that it protects neurons and restores myelin. Damaged myelin leads to symptoms of illness and disability in MS patients. This intended profile differs dramatically from currently approved MS therapies, which are used for the treatment of relapsing MS and reduce inflammation, but do not restore myelin or directly protect neurons.

“We are very pleased to have received this funding from the National MS Society to support our efforts to address important unmet medical needs among MS patients” says Per Bengtsson, CEO of Karo Bio. “Our aim is to achieve a significant breakthrough in the treatment of MS.”

This investment by the National MS Society is a continuation of a strategic research focus on gender differences in MS. That effort helped drive basic research into the role of sex hormones, and estrogen in particular. Karo Bio’s development of ERbeta agonists reflects the potential of this approach, and fits the Society’s comprehensive strategy to stop the progression of disability in MS and to restore function.

About Karo Bio

Karo Bio is a pharmaceutical company focused on the research and development of innovative drugs for large medical needs. The world-leading knowledge of nuclear receptors as target proteins for the development of pharmaceuticals and the related mechanisms of action, are utilized as a foundation for developing novel, more effective and safer pharmaceuticals. The company runs drug development projects mainly within the therapeutic areas neuropsychiatry, inflammation, autoimmune diseases and cancer. The company has a number of strategic agreements and collaborations with international pharmaceutical companies. Karo Bio is based in Huddinge, Sweden, has 42 employees.

Source: PipelineReview.com © by La Merie Publishing (13/09/13)