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NeuroPerspective Reviews Huntington’s and Epilepsy

The March/April issue of NeuroPerspective for 2017 has been released, offering detailed reviews of important, under-served therapeutic areas, as well as coverage and commentary regarding significant developments in the CNS therapeutics space.

The first therapeutic sector review is on Huntington’s, which offers more mechanistic clarity than its neurodegenerative peers, though the route from genotype to phenotype remains to-be-defined, and the mHtt target grows more complex and multi-faceted the more that it comes into focus. Operating against the genetic anomaly would require the selective sparing of necessary wild-type Htt, and the delivery of large molecules through the BBB and deep into the brain. Which means that upstream clarity does not easily translate into downstream impact. The size of the Huntington’s-yet-to-emerge patient universe is in the 150-200,000 range in the US, making this far more than a minor market for any company that can develop a disease-slowing treatment. Such a drug would be administered early, and perhaps chronically. The advent of a disease-modifier would also invert the risk-benefit calculations involved in genetic testing: The diagnosis would finally be more than an incontrovertible harbinger of personal doom. Some of the companies whose programs are discussed in the review include: Annexon Biosciences, Proclara, Roche/Ionis, Wave Life Sciences, Spark Therapeutics, Teva, UniQure, Voyager, Prana, Evotec/CHDI, and Vaccinex.

The second therapeutic sector review is on Epilepsy: The current armamentarium of anti-epileptic drugs offer numerous variations on a few main themes; none of which have proven superior across the board; and which singly or in combination, provide adequate control for just 70-80% of epilepsy patients. For a patient formerly refractory to drug treatment, achieving seizure control, via monotherapy or adjuncts, can be a life-saving event, literally and figuratively. Improving tolerability, and reducing teratogenicity and/or weight gain, would also improve treatment compliance and outcome.The most dramatic advances on tap for the next few years involve relatively small, but severely-impacted subpopulations. Severe Refractory Status Epilepticus appears solvable for many via SAGE-547, while significant (though far from ideal) inroads are now being made with Dravet Syndrome and Lennox-Gestaut, generally refractory to intervention, by companies like GW Pharma and Zogenix. Big Pharma has largely withdrawn from the development of epilepsy drugs, as exemplified by Lilly‘s outlicensing LY3130418 to Cerecor, and JNJ/Janssen not pursuing its own promising TARP/AMPA inhibitors. In contrast, UCB Pharma has made epilepsy the centerpiece of its research and commercial strategy, although recent partnerships have been turned to neurodegeneration as they gradually broaden their focus. Of the other companies tracked in NeuroLicensing 2016-17, only Dainippon/Sunovion and Otsuka cited Epilepsy as an area where they have licensing interests. This means that drug development for Epilepsy has become the province of small and mid-sized specialty companies, and funding has not been easily found, in spite of the unmet needs that continue to be high-profile in this population. The companies whose programs are discussed in this review include: SAGE Therapeutics, GW Pharma, UCB Pharma, Marinus Pharmaceuticals, Zogenix, Epalex, Cerecor, Tansna Therapeutics, Zynerba, Catalyst Pharma, SK Biopharma, Neurona Therapeutics, and Adamas Pharmaceuticals.

The March/April issue also includes discussion of recent clinical findings from Trevena, Merck, Acorda, and Celgene, as well as commentary on the pricing debacle at Marathon Pharmaceuticals and questions regarding Alcobra’s revised strategy. Company Spotlight Reviews are included for Wave Life Sciences and Trevena.

NI Research is the leading publisher of independent research on the neurotherapeutics industry, and has developed an unmatched information base regarding both publicly and privately held CNS companies. NeuroPerspective is the authoritative, independent, monthly review of the neurotherapeutics area, providing critical analyses of therapeutics-in-development. 

 A one-year (1-5 user) subscription to NeuroPerspective is $2600.  Special startup, nonprofit, and academic pricing is available. The March/April issue of NeuroPerspective is available as a single-issue purchase for $500. 

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