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Aveo and Astellas Trial Fails to Impress Advisory Panel

The weekly round-up of failed trials, missed targets, and other business mishaps.

The Burrill Report

A U.S. Food and Drug Administration Oncologic Drugs Advisory Committee panel voted 13 to 1 against recommending approval for Astellas Pharma and Aveo Oncology's experimental drug for the treatment of advanced kidney cancer. Although patients taking tivozanib had statistically significant improvement in progression-free survival compared to those taking sorafenib, a drug marketed by Bayer and Onyx Pharmaceuticals as Nexavar, the improvement was slight and patients taking sorafenib had better overall survival, at 29.3 months versus 28.8 months for those taking tivozanib. The FDA panel also had concerns with part of the trial design. Tivozanib is a long half-life, non-specific VEGF-receptor tyrosine kinase inhibitor and this was the first trial designed for superiority in progression-free survival. In 2011 Aveo and Astellas agreed to develop and commercialize tivozanib outside of Asia for the treatment of a broad range of cancers.

The U.S. Food and Drug Administration has limited the use and duration of Otsuka Pharmaceutical’s drug for the treatment of abnormally low blood sodium levels, or hyponatremia, because it can cause liver injury, potentially leading to liver transplant or death. The FDA has warned healthcare providers that the drug, tolvaptan, should not be used for longer than 30 days and should not be used in patients with underlying liver disease. Back in January 2013 the FDA and Otsuka Pharmaceutical originally notified healthcare professionals of the potential for the drug to cause irreversible and potentially fatal liver injury after detecting liver enzyme problems in patients on the medication for 18 months. The FDA also required Otsuka Pharmaceutical to revise the drug label to reflect the new limitation of one month of use. Sold under the name Samsca, tolvaptan treats the condition of too little sodium in the tissues that is most commonly caused by congestive heart failure, liver cirrhosis, or syndrome of inappropriate antidiuretic hormone.

The compounding pharmacy Nora Apothecary & Alternative Therapies voluntarily recalled all of its sterile drug products on the market that have not reached their expiration dates. During a recent U.S. Food and Drug Administration inspection, agency inspectors observed defective quality control processes and became concerned about sterility of products prepared by the pharmacy. All products prepared and dispensed by the pharmacy on or before April 19 are affected by the national recall. Specifically, the recall includes 95 units of compounded products that the pharmacy supplied to offices of twelve licensed medical professionals in Indiana and approximately 400 prescriptions compounded for patients in Indiana, Illinois, Ohio, Florida, and Tennessee.

The U.S. Food and Drug Administration is warning the public and health professionals that the anti-seizure medication ezogabine can cause patients’ skin to turn blue and the pigment in their retina to change. Pigment changes in the retina are known to potentially cause serious eye disease with loss of vision. To date several patients have reported impaired visual acuity. The FDA states that it does not know whether the particular retinal pigment changes caused by the drug will lead to visual impairment nor does it know if the retina and skin changes are reversible. Valeant Pharmaceuticals developed the drug and GlaxoSmithKline markets and distributes its under the name Potiga. The FDA is working with GSK to gather and evaluate all available information to better understand the adverse events and will update the public when more information is available.

Seaside Therapeutics’ lead experimental drug for the treatment of social-cognitive impairments in autism spectrum disorder failed to meet its primary endpoint of significantly improving social withdrawal. The mid-stage trial of arbaclofen compared it to a placebo to evaluate its safety, tolerability, and efficacy in 150 young patients, aged 5 to 21, over 12 weeks. While the primary endpoint was not met, there were enough significant improvements in subjective Clinical Global Impression of Severity scores and a measure of social function for some patients for Seaside Therapeutics to initiate another trial of the drug. Arbaclofen is an oral medication that works as a selective gamma-aminobutyric acid type B (GABA-B) receptor agonist. Increasing GABA levels in turn helps dampen levels of the excitatory amino acid that contributes to the symptoms of autism spectrum disorders.

Biotechnology investor David Blech received a four-year prison sentence and a $1.3 million forfeiture in a U.S. district court from a judge, who labeled him a “serial stock manipulator,” according to The Wall Street Journal. Blech pleaded guilty to two counts of securities fraud related to attempts to manipulate the stock price of Pluristem Therapeutics in late 2006 to 2007 and the price of Intellect Neurosciences from 2005 to early 2008. He received probation in 1999 for similar biotech investment crimes in the early- to mid-1990s, according to the Journal.

Allergan chief executive David Pyott said in a conference call that a mid-stage trial of Darpin for the treatment wet age related macular degeneration showed some differentiation from Lucentis but did not support directly moving to late-stage development. DARPins, or designed ankyrin repeat proteins, are antibody-like proteins with high-affinity for their target. In this case, VEGF, which causes blood vessel growth and leakiness of the macular region of the eye. The trial was designed to assess the safety and tolerability of the long-acting inhibitor developed with the DARPin technology from Molecular Partners. Allergan will perform additional mid-stage trials to assess the drug in a greater number of patients, and that will delay its potential approval by one to two years, giving Regeneron’s Eylea time to potentially gain traction in the market.

May 03, 2013

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