Auspex Pharmaceuticals, Inc. (ASPX)

Huntington's disease (HD) is a progressive, inherited, degenerative brain disorder that produces physical, mental and emotional changes. Every child of a parent who carries the HD gene has a 50% chance of inheriting the abnormal gene. Symptoms of Huntington's disease usually develop between ages 30 and 50, but they can appear as early as age 2 or as late as 80.

The most visually prominent symptom of Huntington's disease is chorea, which can appear as involuntary, excess movements that can impair a patient's quality of life and be incredibly debilitating. At first, the movements may be subtle and be mistaken as twitching, but may develop into more pronounced movements over time. Approximately 90% of people with Huntington's disease will experience chorea at some point.

Huntington's disease brain changes lead to alterations in mood, especially depression, anxiety, and uncharacteristic anger and irritability. Another common symptom is obsessive-compulsive behavior, leading a person to repeat the same question or activity over and over.

Huntington's disease slowly diminishes the affected individual's ability to walk, talk and reason. Eventually, the person with HD becomes totally dependent upon others for his or her care. Huntington's Disease profoundly affects the lives of entire families -- emotionally, socially and economically.

ASPX reported positive topline efficacy and safety results from its Phase 3 registration trial evaluating SD-809 for the treatment of chorea associated with Huntington's disease, called First-HD.

In addition to meeting the primary efficacy endpoint, significant improvements in both patient and clinical global impressions of change and quality of life were observed. Importantly, the study showed a favorable safety and tolerability profile, including low rates of depression, somnolence, akathisia/restlessness and anxiety.

ASPX also announced results from an analysis of the completed four-week Switch portion of the ARC-HD study, which also has an ongoing long-term safety component. Data from the study show that patients who switched from the current standard of care, tetrabenazine, to SD-809 maintained chorea control at both week one and week four.

ASPX is committed to rapidly advancing SD-809 along its regulatory pathway and submitting an NDA by mid-2015.



ASPX is a biopharmaceutical company dedicated to developing innovative medicines for hyperkinetic movement disorders and other rare diseases.

More about Auspex Pharmaceuticals, Inc. (ASPX) at www.auspexpharma.com.

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Keep a close eye on CERS. The company said that the U.S. Food and Drug Administration (FDA) has approved the INTERCEPT Blood System for plasma.

CERS' INTERCEPT plasma system is approved for ex vivo preparation of plasma in order to reduce the risk of transfusion-transmitted infection (TTI) when treating patients requiring therapeutic plasma transfusion. It is comprised of single-use plasma processing sets and an ultraviolet (UVA) illumination device for the ex vivo preparation and storage of pathogen-reduced, whole blood-derived or apheresis plasma.

CERS' INTERCEPT Blood System inactivates a broad spectrum of enveloped viruses, non-enveloped viruses, Gram-positive and Gram-negative bacteria, spirochetes and parasites.

Platelets, plasma and red blood cells do not require functional DNA or RNA for therapeutic efficacy. However, pathogens (bacteria, viruses and parasites) and white blood cells do require these nucleic acids in order to replicate. The INTERCEPT Blood System targets this basic biological difference between the therapeutic components of blood, compared to harmful pathogens and donor white blood cells. The system uses a proprietary molecule that when activated, binds to and blocks the replication of DNA and RNA, preventing nucleic acid replication and rendering the pathogen inactive.



CERS is a biomedical products company focused in the field of blood safety. CERS currently markets and sells the INTERCEPT Blood System for both platelets and plasma in Europe, the Commonwealth of Independent States, the Middle East and selected countries in other regions around the world. In the United States, the INTERCEPT Blood System for plasma has received FDA approval, and the INTERCEPT Blood System for platelets is under regulatory review.

More about Cerus Corporation (CERS) at www.cerus.com

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