Inside This Issue: Lysosomal Storage Disorders
Top News Alerts: Product Updates, Deals & Financings
Featured Topic: Opportunities and emerging strategies in lysosomal storage disorders
Featured Company: StemCells Inc. (STEM)
Subscribe now to access over 30 articles covering developments in drugs, devices and diagnostics for the brain and nervous system.
Market Highlights: August 2008
Orexigen (OREX) was the top gainer this month after investors were reminded that four Phase III trials for Contrave have completed enrollment and several new programs are expected to enter Phase II testing before the end of the year. Catalyst Pharmaceutical Partners (CPRX) got a boost after publishing results indicating that CPP-109, their formulation of vigabatrin, may have efficacy in obesity (see story, page 4). The drug has shown promising results for the treatment of stimulant addiction and is currently in several Phase II studies with Fast Track. At long last the FDA approved Prestwick Pharmaceu-ticals’ Xenazine (tetrabenazine) for chorea associated with Huntington’s disease (see story, page 3). The NDA for the drug, which had Fast Track status, was submitted in April 2005 and the FDA advisory committee recommended approval in December 2007..
Lysosomal Storage Disorders: Big Opportunities in Small Numbers
Lysosomal storage disorders (LSDs) encompass approx-imately 40 rare genetic disorders many of which have a neurological component, including Batten disease, Gaucher disease, Neimann-Pick, Tay Sach’s, Fabry, Hunter Syndrome and Krabbe among others. The disorders are caused by mutations in genes for cellular digestive enzymes that play a key role in removal of cellular waste and infection fighting. Despite their rarity – the most common LSDs have an incidence of about 1 in 50,000 people – LSDs have significant revenue potential. The most successful treatment on the market, Genzyme’s Cerezyme for Gaucher disease currently has annual revenue over $1 billion and Frabrazyme for Fabry disease has reached over $500 million. Drug regimens for LSDs can cost upwards of $150,000-$300,000/ year....
StemCells, Inc.: A novel approach to Batten Disease
StemCells, Inc. (STEM), based in Palo Alto CA, was founded in 1989 by Dr.’s Sharp and Laci. It was originally called CytoTherapeutics (CTII). The company's original technology was cell encapsulation (ECT) for a variety of applications, many of which were CNS focused. In December of 1993, the company sold its diabetes program to Neocrin Company (later Novocell Inc.) in return for an equity position. Novocell continues to develop the technology to this day, and it recently received FDA approval for a Phase I/II trial using encapsulated human pancreatic islets. The ECT technology was later licensed to Neurotech SA. CytoTherapeutics made its first move towards stem cells in 1994, when it licensed neural stem cell technology. In 1997, it acquired a San Diego start-up, called StemCells Inc. Since 1997, StemCells has been developing a method for growing human central nervous system stem and progenitor cells in culture as clusters called neurospheres. The company conducted several preclinical studies using these neurospheres in collaboration with Dr. Anders Bjorklund of Lund University in Sweden, another pioneering site...
Companies Covered In This Issue Include: Biogen Idec, Medavante, CoLucid, Bayhill Therapeutics, Theragenics, Neurogen, Map Pharmaceuticals, NeuroDiscovery, Sound Pharmaceuticals, Genaera, Northstar Neuroscience, Neuropharma AS, CoMentis, Targacept, Durect, Adolor, Evotec, BrainStorm Cell Therapeutics, Titan, Teva Pharmaceuticals, Antisense Therapeutics, Roche, Abbott, Epix, Memory, GlaxoSmithKline, Avera, Synosia, Ceregene, NsGene, Cortex, Allon Therapeutics, Merck, Amgen, Synosia, Cara Therapeutics, Probiodrug, NuPathe, Sierra Neuropharmaceuticals, CVRx, Vanda, Actelion, Collegium, NeuroHealing, Genentech, Peptimmune, Greatbatch, Integrated Sensing Technologies, Orexigen, UCB
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