FDA Extends Indacaterol Review

FDA Extends Indacaterol Review
Drug Discovery & Development - March 24, 2011

NEW YORK (AP) - The Food and Drug Administration is extending a review of a lung disease drug made by Novartis AG, the Swiss drugmaker said Wednesday.
Novartis said the FDA now expects to complete its review in July instead of April. It said the FDA needs more time to review data from clinical trials, but has not asked for any new information. The agency is reviewing Novartis' application on a drug candidate called QAB149, or indacaterol, which is a once-per-day treatment for patients with chronic lung conditions like bronchitis or emphysema.
Indacaterol is a bronchodilator, or a drug that opens up airway passages in the lungs to ease breathing.
In February, an FDA advisory panel recommended the drug be approved. Novartis is seeking approval to market the drug in strengths of 75 micrograms and 150 micrograms per day. The company said the drug is approved under the name Onbrez Breezhaler in more than 50 countries. Onbrez Breezhaler is marketed in strengths of 150 micrograms and 300 micrograms per day.
Date: March 23, 2011
Source: Associated Press

Gene Therapy Improves Parkinson's Symptoms

Gene Therapy Improves Parkinson's Symptoms
Maria Cheng
Drug Discovery & Development - March 24, 2011

LONDON (AP) - An experimental treatment improved symptoms of Parkinson's disease in a mid-stage test, echoing results of an earlier pilot study.
The new research is the first to show positive results in a test of gene therapy against a sham operation in about three dozen U.S. Parkinson's patients.
After six months, those who got the gene therapy scored 23 percent better on a standard test to measure motor skills while those who got the sham operation did about 13 percent better.
"Gene therapy is no longer just a theory," said Michael Kaplitt, a neurosurgeon at New York-Presbyterian Hospital and Weill Cornell Medical Center, and one of the study authors. "We are getting much closer to a reality where this treatment can be offered to patients." Kaplitt said the results might spur similar treatments for other brain disorders like Alzheimer's, epilepsy and depression.
Kaplitt and colleagues tested the gene therapy on 16 people while 21 others received a sham surgery. The patients were aged 30 to 75 and all were taking Parkinson's medication.
In patients with Parkinson's disease, their brains get overactive after losing the normal supply of a chemical called GABA. The new treatment, gene therapy, works by inserting billions of copies of a gene into patients' brains that helps them produce more GABA.
For patients who got the gene therapy, doctors drilled a hole into their brains while they were still awake. Doctors then slipped in a virus engineered to bring in billions of copies of a gene to help the brain pump out more GABA. Patients who didn't get the gene therapy had holes drilled halfway into their skull - enough to trick them they were getting the therapy but apparently not enough to do any harm.
The study was published online Thursday in the journal, Lancet Neurology. It was paid for by Neurologix Inc., the biotechnology company that devised the therapy. Kaplitt is a company cofounder and holds stock options and many of the other authors reported ties to Neurologix and other pharmaceuticals.
Parkinson's disease is a degenerative brain illness that causes problems including tremors, rigidity and slow movements. It affects about one in every 500 people. There is no cure, but some drugs help control symptoms.
"This is promising research but we need to know how long these benefits of gene therapy might last," said Michelle Gardner, research development manager at Parkinson's U.K. She was not linked to the study. "We don't know if there could be long-term consequences of introducing viruses into the brain."
In an accompanying commentary in the Lancet, Michael Hutchinson of New York University School of Medicine questioned whether gene therapy offers any advantages over deep brain stimulation, which has been used to treat Parkinson's disease for about a decade.
Walter Liskiewicz, a former oral surgeon in Michigan with the disease, could barely move before receiving the gene therapy in 2009 as part of the experiment.
Now, he plays jazz music and recently returned from a holiday in Brazil. "Everything was taken away from me and to just have them handed back is pretty special," he said. "It's like a miracle."
Date: March 23, 2011
Source: Associated Press

Symphogen Receives U.S. Patent on Lead Cancer Compound

Symphogen Receives U.S. Patent on Lead Cancer Compound
Drug Discovery & Development - March 07, 2011

Symphogen, a private biopharmaceutical company developing antibody therapeutics, has been granted a U.S. patent for Sym004 as well as other antibody compositions containing at least two distinct anti-EGFR antibodies having certain binding characteristics. The U.S. Patent and Trademark Office on February 15, 2011 issued to the company U.S. Patent No. 7,887,805, titled “Recombinant anti-epidermal growth factor receptor antibody compositions.” The US patent provides protection through 2028. Patent applications are pending in additional countries.
In addition, Symphogen said that preclinical data demonstrating the superiority of the now proprietary Sym004 to the reference monoclonal antibody (mAb) was highlighted  in Paris, France in a Plenary Session of the 9th International Symposium on Targeted Anticancer Therapies (TAT Symposium). Sym004 is a novel drug candidate based on a 1:1 mixture of two anti-EGFR monoclonal antibodies (mAbs) directed against distinct non-overlapping epitopes in EGFR extracellular domain III.
Preclinical data showed Sym004, a novel and proprietary candidate for the treatment of solid tumors, exhibited more pronounced growth inhibition in vitro and superior efficacy in in vivo models as compared to the reference anti-EGFR mAb cetuximab. The rate at which Sym004 induced rapid and efficient removal of the ligand-binding receptor from the cancer cell surface by triggering EGFR internalization and degradation was reported to have exceeded that of cetuximab. Like anti-EGFR mAbs in current clinical use, Sym004 has been previously shown to inhibit cancer cell growth and survival by blocking ligand-binding receptor activation and downstream signaling.
The preclinical safety and pharmacokinetic data for Sym004 has been accepted for a poster presentation at the upcoming 102nd Annual meeting of the American Association for Cancer Research in Orlando, Florida April 2-6, 2011.
Later this year, Symphogen plans to present safety data from its first clinical study of Sym004. Sym004 is currently being evaluated for the treatment of advanced solid tumors with particular focus in patients with KRAS wildtype advanced metastatic colorectal cancer (mCRC) that have previously progressed to standard chemotherapy and marketed anti-EGFR mAbs. A phase I-II, first-in-human study is presently ongoing at centers in USA and Spain. The main objective of the phase I component of the trial is to evaluate the safety and tolerability of multiple ascending doses of Sym004 in patients with solid tumors. The phase II part of the trial will include patients with mCRC and begin in the first quarter of 2011.

Date: March 7, 2011
Source: Symphogen  

Primary Endpoints Met In Phase 3 Trial of Tofacitinib

Primary Endpoints Met In Phase 3 Trial of Tofacitinib
Drug Discovery & Development - March 07, 2011

Pfizer Inc. announced that the ORAL Sync Phase 3 study (A3921046) of tofacitinib (development code: CP-690,550), formerly known as tasocitinib, an investigational, novel, oral JAK inhibitor, being studied in moderate-to-severe rheumatoid arthritis (RA), met its primary endpoints by showing statistically significant changes versus placebo in reducing signs and symptoms of RA, as measured by ACR20 response rates at six months; in improving physical function, as measured by mean change in HAQ DI at three months; and in reaching DAS28-4(ESR) <2.6 at six months. The safety profile of tofacitinib was consistent with that seen previously in the clinical program, and no new safety signal was detected. A full analysis of efficacy and safety data will be submitted to a future scientific meeting.
ORAL Sync evaluated the efficacy and safety of tofacitinib doses 5 mg and 10 mg given twice daily compared to placebo in patients with moderately to severely active RA who had a previous  inadequate response to a DMARD and who continued to receive background traditional DMARD therapy throughout the study.
Tofacitinib is a novel, oral Janus kinase (JAK) inhibitor that is being investigated as a targeted immunomodulator and diseasemodifying therapy for RA. More than 4,000 RA patients have been treated with tofacitinib in clinical trials to date. Unlike current therapies for RA, which are directed at extracellular targets such as pro-inflammatory cytokines, tofacitinib takes a novel approach, targeting the intracellular signaling pathways that operate as hubs in the inflammatory cytokine network. The Phase 3 ORAL Trials clinical program includes six studies with more than 350 locations in 35 countries worldwide.
Date: March 4, 2011
Source: Pfizer 

NeuroVive Conducting Late-Stage NeuroSTAT Trial

NeuroVive Conducting Late-Stage NeuroSTAT Trial
Drug Discovery & Development - March 08, 2011

NeuroVive has signed an agreement with the European Brain Injury Consortium (EBIC) to conduct a European multi-center Phase 2/3 clinical trial of NeuroSTAT for neuroprotection in patients with traumatic brain injury (TBI). The goal of the collaboration is to secure the highest possible study quality and compliance with clinical standards to support NeuroSTAT‘s rapid progress toward commercialization. To this end, NeuroVive will work closely with EBIC’s key European experts in the field of neurotrauma and acute brain injuries.
NeuroVive will manage the study in close collaboration with the EBIC group and a clinical research organization (CRO) to be selected. The collaboration with EBIC includes development of the study protocol, site selection, patient recruitment, and statistical support. This adaptive design Phase II/III study will determine the safety and efficacy of NeuroSTAT in moderate to severe TBI.
TBI is a leading cause of death and disability among a predominantly young male population, mostly victims of motor vehicle accidents. TBI is composed of two stages: a first stage of initial brain damage; and a second stage of mitochondrial collapse inside brain cells leading to additional cell death, brain damage and disability. The active agent in the NeuroSTAT formula is a proven mitochondrial protector that in animal studies is a potent neuroprotectant against TBI. There are over 10 million TBI cases per year worldwide. Annually in the U.S., head trauma sends two million people to the emergency room and a half a million have injuries severe enough to be admitted to the hospital. TBI kills 50,000 people and leaves 80,000 people disabled. The annual market for an effective TBI drug is estimated to exceed US$1 billion in the U.S. and Europe.
NeuroVive CEO Mikael Bronnegard comments: ”We are very pleased to collaborate on the NeuroSTAT Phase II/III clinical trial with a highly respected organization like EBIC, comprising key opinion leaders and medical experts in the field of neurotrauma in Europe. Through this collaboration, we have an excellent opportunity to advance the development of NeuroSTAT toward clinical use in treating millions of TBI patients worldwide.”
Professor Andrew Maas, Chairman of EBIC, Professor and Chairman Department of Neurosurgery at University Hospital Antwerp, Belgium comments: “This clinical trial is of great interest to us. The need for international collaboration in the conduct of research in the clinical management of head injury has never been greater, but rigorous evaluation must be carried out on a range of approaches that include new pharmacological agents. Therefore, the collaboration with NeuroVive in conducting a clinical trial in TBI patients with their product NeuroSTAT offers a unique way to address all critical issues in study design, selection of end-points, patient recruitment, study monitoring and, finally, study analysis.”
Date: March 3, 2011
Source: NeuroVive 

Toxikon Completes Kevetrin Toxicity Studies

Toxikon Completes Kevetrin Toxicity Studies
Drug Discovery & Development - March 08, 2011

Toxikon Corp., a pre-clinical contract research organization with facilities in the US and in Leuven, Belgium, has completed toxicity studies in two species for Cellceutix Corp.’s Kevetrin, a compound the Beverly, Mass.-based firm is developing for the treatment of resistant lung and breast cancers.  Toxikon also performed safety pharmacology studies of Kevetrin in the central nervous system as well as the cardiovascular and respiratory systems.
The toxicity studies, required for an Investigational New Drug (IND) filing, involved administering Kevetrin to two species of animals weekly over a 29-day period. In each study, there was a 14-day recovery period after the dosing. Parameters measured were hematology, clinical chemistries, weight change, food consumption, survival, urinalysis, ophthalmic examination and histopathology. The studies are designed to confirm that Kevetrin meets FDA safety requirements for clinical studies in humans.
 “We are proud to have led these important toxicology studies and help Cellceutix achieve its goal of getting Kevetrin to human trials and, ultimately, to market,” said Dr. Laxman Desai, president and CEO of Toxikon, adding that “Toxikon is equipped to meet the industry’s anticancer and antiviral drug discovery demands.”
“Along with safety studies, we can assist with synthesis of intermediates from milligrams to hundreds of grams,” said Dr. Desai, the former director of anti-cancer drug research at Boston’s Dana Farber Cancer Institute. “We are also poised to develop small novel molecules for the treatment of cancer and viral diseases.”
“We are very delighted with the results of the toxicity studies performed at Toxikon,” said Dr. Krishna Menon, Chief Scientific Officer at Cellceutix.  “While we were confident that the results would be positive, it is always reassuring to see the actual results.  We are also grateful to Toxikon for the high quality of their work and excellent customer focus.  We have now taken a major step forward toward our phase 1 clinical trial, to be conducted at a major cancer center in the Boston area.”
In animal studies, Kevetrin was found to significantly delay tumor growth in multi-drug resistant lung, breast and colon cancer cell lines. In data presented in April, 2010 at the American Association for Cancer Research, the efficacy of Kevetrin in a mouse model of drug resistant lung cancer was shown to increase with increasing dose. The data presented at the AACR also show that the administration of a second cycle of therapy with Kevetrin in the same animal model continues to delay tumor growth without the development of resistance.
Date: November 2, 2010
Source: Toxikon Corp. 

FDA Reviewing Carbinoxamine XR NDA

FDA Reviewing Carbinoxamine XR NDA
Drug Discovery & Development - March 09, 2011

Tris Pharma, a specialty pharmaceutical company that develops innovative drug delivery technologies, announced that the US Food and Drug Administration (FDA) has accepted its Extended Release Carbinoxamine Oral Suspension NDA for the treatment of allergies in kids two years and older. If approved, Tris Pharma's Extended Release Suspension will provide an alternative to the currently available immediate release formulations.
Carbinoxamine is a mildly sedating antihistamine with years of proven safety and efficacy. Prior to 2006 carbinoxamine was widely used, with more than 100 marketed Carbinoxamine containing products including extended release solid dose and combination products. However, nearly all of these were older products which hadn't gone through the FDA's rigorous approval process. Following the 2006 DESI review, the FDA removed all carbinoxamine based products with the exception of two immediate release formulations which had been reviewed, creating a void for patients and doctors who valued the benefits associated with an extended release formulation.
A leading New York based pediatrician, Dr. Laura Garabedian, who often prescribes Carbinoxamine said, "I've always found Carbinoxamine extremely effective in treating allergy symptoms in children but don't typically prescribe it as first line therapy because the existing immediate release formulations need to be dosed multiple times per day including a dose while the child is in school thus making compliance poor.  I am looking forward to a true 12 hour formulation that tastes good.  The new product profile should translate to improved compliance and better outcomes."
Ketan Mehta, Tris President and CEO added, "This is the fourth NDA submission based on Tris' OralXR+ technology platform including Nexiclon XR, a recently launched first ever 24-hour liquid Extended Release Suspension. Tris' pipeline includes several additional products targeted for the respiratory market, i.e. cough, cold, allergy therapies and are slated for submission over the next two years. Tris plans to launch Carbinoxamine Extended Release Suspension in 2012 season and is currently evaluating its options including partnering with other specialty Pharma companies. "
Date: March 9, 2011
Source: Tris Pharma 

New Leukemia Treatment Given Orphan Drug Status

New Leukemia Treatment Given Orphan Drug Status
Drug Discovery & Development - March 09, 2011

Stemline Therapeutics, Inc. announced that SL-401, the Company's lead compound, has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML).
SL-401 has completed a multi-center Phase 1/2 clinical trial in AML where it has demonstrated single agent efficacy, including two durable complete responses (CRs), multiple blast reductions and disease stabilizations, and an overall survival (OS) benefit in heavily pre-treated patients. SL-401 was well-tolerated and bone marrow-sparing. The trial results were presented at the 52nd Annual Meeting of the American Society of Hematology (ASH) in December 2010. SL-401 is now poised for Phase 3 clinical trials in patients with advanced AML.
Stemline's CEO, Ivan Bergstein, MD, noted, "Receipt of Orphan Drug designation represents another key milestone in the advancement of SL-401 through the regulatory process. We are also very encouraged by the potential of SL-401 to benefit patients with advanced stage AML, an unmet medical need, as well as additional hematologic malignancies including MDS, CML and potentially certain lymphomas."
Date: March 9, 2011
Source: Stemline Therapeutics, Inc.

Sildenafil Reduces Raynaud's Frequency in lcSSc

Sildenafil Reduces Raynaud's Frequency in lcSSc
Drug Discovery & Development - March 09, 2011

Researchers in Europe reported that treatment with modified-release sildenafil significantly reduced the frequency of attacks of Raynaud's phenomenon in patients with limited cutaneous systemic sclerosis (lcSSc), also known as scleroderma. The double-blind, placebo-controlled trial found that sildenafil was well tolerated with only some subjects experiencing minor or moderate side effects. Full findings are available in the March issue of Arthritis & Rheumatism, a journal published by Wiley-Blackwell on behalf of the American College of Rheumatology (ACR).
Raynaud's phenomenon (RP) is a major feature of systemic sclerosis and causes the blood vessels supplying the skin surface to spasm in response to cold temperatures or stress. These vasospasms can affect the fingers, toes, ears and other skin surfaces and can lead to serious complications in patients with systemic sclerosis such as digital ulceration, soft tissue or bone infection, or gangrene. According to the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) this phenomenon occurs in 90% of all scleroderma cases.
The multicenter trial of sildenafil was conducted in 57 men and women (ages18-75) with Raynaud's secondary to lcSSc between January and June 2003. For one to two weeks prior to the start of treatment, patients recorded the number of RP attacks. To be eligible for the study, subjects had to report at least 7 RP attacks per week. RP was defined as an episode where fingers or toes turned white (pallor) followed by blue (cyanosis) and/or red (erythema) in response to cold or emotion. At the onset of the treatment phase, participants were randomized in a one-to-one ratio, receiving either 100 mg of modified-release sildenafil for three days followed by a 200-mg modified-release dose daily for 25 days, or placebo for 28 days. Researchers used a two-step dosing to enhance tolerability, and a modified-release formulation to permit once daily dosing.
"Our findings indicate that modified-release sildenafil reduced the frequency of Raynaud's attacks in patients with systemic sclerosis," confirmed lead author, Ariane Herrick, M.D., from the University of Manchester in the U.K. Results showed a 44% reduction in attacks per week (from baseline to day 28) for subjects receiving sildenafil treatment compared with 18% in the placebo group. Researchers also reported that the mean number of attacks per week improved from 30.5 at baseline to 18.7 after sildenafil treatment, compared with 25.0 at baseline to 19.3 after placebo treatment.
Secondary endpoints, including Raynaud's Condition Score, duration of attacks, and RP pain score, were not significantly different between the two groups. The most frequent adverse events reported were headache and indigestion with the majority of these events being mild or moderate. Researchers did not observe any serious adverse events. Dr. Herrick concluded, "Modified-release sildenafil was well tolerated and reduced the attack frequency in patients with Raynaud's secondary to lcSSc. Our results, coupled with existing medical evidence of the favorable safety profile of sildenafil in non-SSc patient populations, may offer a beneficial treatment option in lcSSc patients with secondary Raynaud's."
Date: March 8, 2011
Source: Arthritis & Rheumatism

No Clear Evidence of Increased Efficacy In RA Treatment

No Clear Evidence of Increased Efficacy In RA Treatment
Drug Discovery & Development - March 09, 2011

A recent trial of rituximab in combination with a tumor necrosis factor (TNF) inhibitor and methotrexate (MTX) in patients with active rheumatoid arthritis (RA) found the safety profile to be consistent with other RA trials with TNF inhibitors. While the trial reported no new safety risks, clear evidence of an efficacy advantage in RA patients receiving the combination therapy was not observed in this study sample. Results of the trial are published in the March issue of Arthritis & Rheumatism, a peer-reviewed journal of the American College of Rheumatology.
The National Arthritis Data Workgroup estimates that 1.3 million U.S. adults have RA which is characterized by systemic joint inflammation that often leads to joint damage, functional impairment and significant disability. While MTX is successfully used to treat many RA patients, the severity of the disease in some patient populations requires the use of additional disease-modifying antirheumatic drugs (DMARDs). A specific group of biologic DMARDs, called tumor necrosis factor (TNF) inhibitors and includes such therapies as etanercept and adalimumab, block the immune system response, and have been shown to be safe and effective in clinical trials.
Prior studies, however, have found that up to 40% of RA patients exhibit an inadequate response, intolerance, or inadequate slowing of the rate of joint damage with biologic therapies, and require additional treatment options. "Our objective was to assess the safety of the biologic DMARD, rituximab, in combination with a TNF inhibitor and MTX in patients with active RA," said lead study author Maria Greenwald, M.D., from Desert Medical Advances in California. This was a small exploratory study to evaluate safety with this combination due to the prolonged effect of rituximab, and the fact that RA patients may switch to an alternate biologic such as a TNF inhibitor before the effects of rituximab may have resolved.
The controlled trial enrolled 51 patients with active RA (more than 5 swollen and tender joints) who were receiving a stable dose of MTX (10-25 mg/week) and either etanercept or adalimumab for more than 12 weeks. Participants were randomized 2:1 to receive one course of rituximab or placebo (two 500-mg doses intravenously or placebo). The primary study end point was the proportion of patients developing more than one serious infection through week 24.
Researchers reported one serious infection (pneumonia) in the rituximab group compared with none in the placebo group (TNF only) at week 24. Nonserious infections were reported in 18 patients (55%) and 11 patients (61%) in the rituximab and placebo groups, respectively. Infections requiring intravenous antibiotics occurred in three patients administered rituximab and in none of the patients receiving placebo. No life-threatening, opportunistic, fungal or tuberculosis infections were observed. Dr. Greenwald noted, "The incidence of serious infections was low."
During the 24-week trial period the overall proportion of patients in the placebo and rituximab cohorts who experience an adverse event (AE) was 83% and 94%, respectively. The most common AEs reported in the rituximab group included nausea, pruritus (itching sensation), and fatigue. In the placebo group the common AEs included upper respiratory tract infections, sinusitis, headache, and exacerbation of RA. Two serious adverse effects (SAEs)—pneumonia and coronary artery occlusion—were observed in two rituximab-treated patients; no SAEs were reported in patients receiving placebo.
The research team also determined the percentage of patients achieving an ACR201 improvement response at week 24 was 30% in the rituximab group compared with 17% in the placebo group. ACR50 responses achieved in the rituximab and placebo groups were 12% and 6%, respectively. "The safety of rituximab in combination with a TNF inhibitor and MTX was consistent with other RA trials of rituximab and MTX that did not include TNF inhibitors," concluded Dr. Greenwald. The authors noted that a larger study examining efficacy of treatment of RA using multiple biologic DMARDs is underway.
Date: March 8, 2011
Source: Arthritis & Rheumatism

Biotron Completes First Part of Phase 2 Trial

Biotron Completes First Part of Phase 2 Trial
Drug Discovery & Development - March 10, 2011

Australian drug development company Biotron Limited has completed stage one of a landmark human trial of its lead Hepatitis C drug candidate, BIT225.
Twelve patients have been dosed in the first half of the trial being undertaken by ACLIRES, an international contract research organisation (CRO) that specialises in running antiviral drug clinical trials.  All are infected with the most common strain of the Hepatitis C virus, genotype 1.
The second half of the trial - which also involves 12 patients - is now underway and expected to be completed in May, with results anticipated to be analysed and released in June.  The trial is blinded, so no results are available until samples are analysed at the conclusion of the trial and the data is unblinded.
Biotron CEO, Dr Michelle Miller, said the trial was proceeding as expected and the results would be "of international interest".
She said BIT225 was a first-in-class drug candidate which specifically targeted the p7 protein, a viral protein essential to virus production and replication.
"We are happy with how the trial is progressing.  We achieved the necessary ethics and drug import approvals to move to the second stage of the trial, which is now underway."
Twelve further patients are now being recruited and dosed over four weeks with BIT225.
As in stage one of the study, one-third of patients are being given a placebo, one-third a dose of 400mg BIT225 and another one -third are being dosed at 200mg BIT225.
This Phase 2 trial is examining how BIT225 works in combination with current approved treatments for HCV, Interferon and Ribavarin.
Existing drugs have limited effectiveness and can be toxic.  Doctors say fifty per cent of sufferers do not respond to current therapies, signalling a need for new treatments that directly target and halt replication and reproduction of the virus.
Date: March 9, 2011
Source: Biotron Limited www.biotron.com.au/

Lucentis Trial Shows Greatly Improved Vision in DME

Lucentis Trial Shows Greatly Improved Vision in DME
Drug Discovery & Development - March 10, 2011

Genentech, a member of the Roche Group, announced that two-year results from a pivotal Phase 3 trial (RISE) showed patients with diabetic macular edema (DME) who received Lucentis (ranibizumab injection) experienced rapid and sustained improvement in vision compared to those who received a placebo (sham) injection. According to the data, there was:
• A significantly greater number of people able to read at least 15 additional letters on the eye chart compared to baseline after 24 months (primary endpoint),
• Significant improvement in average eye chart reading scores at 24 months,
• Significant improvement in average eye chart reading scores demonstrated as early as seven days,
• Significantly decreased retinal swelling.
“There are no FDA-approved medicines to treat DME and these new data add to evidence showing that Lucentis can help improve vision soon after initiation of treatment,” said Dennis M. Marcus, M.D., Southeast Retina Center in Augusta, Ga., who presented the data today. “In the case of the RISE study, the significant improvements on day seven were maintained for two years.”
DME is an eye condition characterized by swelling of the retina, which can occur in patients with type 1 or type 2 diabetes and can cause blurred vision, severe vision loss and blindness. DME is a leading cause of blindness among the working-age population in most developed countries. The safety results were consistent with previous experience and no new adverse events related to Lucentis were observed in the study.
The data from RISE, the first of two pivotal Phase 3 studies of Lucentis in DME, were presented at the 34th Annual Macula Society Meeting in Boca Raton, Fla.
Date: March 10, 2011
Source: Genentech 

BioTox Using eStudy for Preclinical Studies

BioTox Using eStudy for Preclinical Studies
Drug Discovery & Development - March 10, 2011

iAdvantage Software, Inc., a leading solutions provider for managing development and pre-clinical life science studies, announced that BioTox Sciences (BTS), a GLP Contract Research Organization, headquartered in San Diego, California, is deploying the eStudy hosted solution to manage its GLP and Non-GLP preclinical/nonclinical studies.
In 2010, BioTox Sciences expanded its resources from people to facility to accommodate growing Sponsors' needs. The final challenge was selecting a software platform that would handle the different preclinical study types BTS conduct's today, yet flexible enough to meet the ever-changing needs they may encounter as they continue to grow.
"After weighing our options and careful due diligence, BioTox Sciences chose to implement eStudy, by iAdvantage Software," commented Tareq Abu-Nadi, BioTox Sciences, Business Development/Study Director. "eStudy will complement and streamline the study processes conducted at BTS by increasing productivity and speed of reporting while maintaining 21 CFR parts 58 and 11 compliance."
"eStudy's flexibility ensures that BTS Study Directors would be able to use eStudy on various projects and multiple species. Whether a typical 28 day toxicity study in rats or dogs, or a long term surgery study in NHPs, eStudy will be used to manage the study, capture the data and allow for on-demand reporting of results upon Sponsor request. This significant investment gives us the ability to produce complete study books in mere hours, and help Sponsors make key decisions on development of compounds and initiate more projects," concluded Abu-Nadi.
eStudy is a 100% web-based study management platform that iAdvantage Software hosts for BTS at Cegidem/Dendrite's world-class network operating center in Chesapeake, VA. "eStudy will continually grow with BioTox Sciences, easily adapt to new study-types, marry to BTS' operational work-flow and interface with 3rd party instruments and tools," stated Diana Michelotti, Director of Marketing and Sales for iAdvantage. "eStudy implemented for BTS, provides BTS with a custom fit solution without the start-from-scratch price."
Date: March 9, 2011
Source: iAdvantage Software, Inc. 

FDA Approves Taxotere Generic

FDA Approves Taxotere Generic
Drug Discovery & Development - March 10, 2011

LAKE FOREST, Ill. (AP) - Drug and medical device maker Hospira Inc. said the Food and Drug Administration approved the company's generic version of Sanofi-Aventis' blockbuster cancer drug Taxotere.
The company said its generic Taxotere, or docetaxel, is a single-vial formulation as opposed to the two-step process for the branded Taxotere.
Hospira said Taxotere, which is approved to treat breast, lung, and other types of cancer, had U.S. sales of about $1.2 billion in 2010. It lost patent protection in November, opening the market up to generic competitors.
Shares of Hospira have traded between $48.69 and $60.49 over the last 52 weeks.
Date: March 9, 2011
Source: Associated Press

FDA Approves First Lupus Treatment in Half-Century

FDA Approves First Lupus Treatment in Half-Century
Matthew Perrone
Drug Discovery & Development - March 10, 2011

WASHINGTON (AP) - The Food and Drug Administration approved the first new drug to treat lupus in over 50 years, a milestone that medical experts say could prompt development of other drugs that are even more effective in treating the debilitating immune system disorder.
Known as Benlysta, the injectable drug is designed to relieve flare-ups and pain caused by lupus, a little-understood and potentially fatal ailment in which the body attacks its own tissue and organs.
Biotech drugmaker Human Genome Sciences Inc. spent 15 years developing Benlysta and will co-market it with GlaxoSmithKline PLC.
The companies estimate there are at least 200,000 lupus patients in the U.S. who could benefit from the drug.
But experts stress that Benlysta is not a miracle drug: It only worked in 35 percent of North American patients tested and was not effective for patients with the deadliest form of the disease. Additionally, it did not show positive results in African-Americans, who are disproportionately affected by lupus.
FDA said in its news release it would require the drug developers to conduct another study exclusively on African-Americans.
Dr. Betty Diamond, who has studied lupus for 30 years, said Benlysta should provide encouragement to researchers and drug developers.
"It will send out the message that it's possible to conduct a successful clinical trial in lupus and that's tremendously important to keep the pharmaceutical industry interested in this disease," said Diamond, a researcher at the Feinstein Institute in New York.
Janice Fitzgibbon of McLean, Virginia has been taking Benlysta for two years as part of the drug's clinical trial program.
"It's given me my life back," she said, after being so crushed by pain that she couldn't take her dog for a walk or drive her children to school.
"It's a bittersweet thing for me because I have friends with lupus for whom this drug won't work," said Fitzgibbon, who is 54. "There's no one-size-fits-all for lupus and I'm just extremely fortunate that my lupus is mild and is helped by Benlysta."
FDA approved the drug for systemic lupus erythematosus, the most common form of the disease. Ten-year survival for patients diagnosed with the illness is more than 85 percent, according to the National Institutes of Health.
Lupus patients have long struggled to draw attention to their disease, which affects women nine times more than men. African-Americans are three times more likely to have the disease.
"I don't think there's a conspiracy here, but it just hasn't gotten a lot of funding and it hasn't gotten a lot of attention from the media," said Dr. Abby Abelson, chair of the Department of Rheumatologic and Immunologic Disease at the Cleveland Clinic.
Lupus causes fibrous tissue and inflammation of internal organs, skin rashes and joint pain. Most of Benlysta's benefit came from relieving muscle inflammation versus organ problems, as measured on a comprehensive checklist of lupus symptoms.
The disease occurs when the body's protector cells, known as antibodies, stop differentiating between foreign invaders, like bacteria, and healthy cells. The cause of this malfunction is not understood.
Currently most patients treat their disease with a variety of drugs that help ease inflammation, including painkillers, steroids and antimalarial drugs - which were first approved for lupus in the 1950s. Many patients say the side effects of those treatments are nearly as uncomfortable as the disease itself. Steroids can cause bone fractures, weight gain and infection.
Wednesday's approval completes a remarkable turnaround for Maryland-based Human Genome Sciences which has been developing Benlysta since 1996 and has no other products on the market. The company originally tested Benlysta, known generically as belimumab, as a treatment for rheumatoid arthritis.
When a mid-stage trial in lupus patients failed to meet researchers' goals in 2006, many analysts wrote the drug off and downgraded the company's stock. But when scientists reanalyzed the data they found that the drug helped block the antibodies that cause lupus symptoms in a subset of patients.
Analysts estimate the drug could reach annual sales exceeding $3 billion within five years.
Date: March 9, 2011
Source: Associated Press