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Zafgen Announces Initial Results from Phase 2a Study of Beloranib in Patients with Prader-Willi Syndrome
Date:1/15/2014

ed by an additional four weeks of open label treatment, the results of which are not yet available.  The trial enrolled 17 patients with genetically confirmed PWS, including 11 women and 6 men, with a mean age of 33.9 years, and mean body mass index (BMI) of 31.4 kg/m2.  Beloranib appeared to be safe and very well-tolerated.  All 17 patients completed the randomized treatment part of the trial and all opted to continue into and completed the four-week open label extension.

About Prader-Willi Syndrome
Prader-Willi syndrome (PWS), the most common known genetic cause of life-threatening obesity, causes constant hunger that drives PWS patients to gain more weight on fewer calories than the average person.  As a result, many of those affected become morbidly obese before the age of five.  There is currently no cure for this disease.  Although the cause is complex, it results from a deletion or loss of function of a cluster of genes on the 15th chromosome.  PWS typically causes low muscle mass and function, short stature, incomplete sexual development, and a chronic feeling of hunger that, coupled with a metabolism that utilizes drastically fewer calories than normal, can lead to excessive eating and life-threatening obesity.  PWS occurs in males and females equally and in all races, with the same incidence around the world.  Prevalence estimates have ranged from 1:8,000 to 1:50,000 with the most likely figure being approximately 1:40,000.  To the best of our knowledge, prevalence is about 5,000-7,000 people in the United States needing treatment.  You can learn more through the Prader-Willi Syndrome Association website at www.pwsausa.org.

About Beloranib
Beloranib is the first compound in its class that works by targeting a key enzyme called MetAP2 that controls the production and utilization of fatty acids.  Inh
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SOURCE Zafgen, Inc.
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