Hyperion Therapeutics Announces Results for Phase II Study in Urea Cycle...( - Company preparing to initiate phase...),Health

- Company preparing to initiate phase III trial -

SOUTH SAN FRANCISCO, Calif., March 30 /PRNewswire/ -- Hyperion Therapeutics, Inc. today announced top-line results from their phase II study of HPN-100 for the treatment of urea cycle disorders. The data was presented on March 27th at the 16th Annual Clinical Genetics Meeting of the American College of Medical Genetics by Brendan Lee, M.D., Ph.D., Howard Hughes Medical Institute Investigator and Professor Department of Molecular and Human Genetics Baylor College of Medicine. The abstract is entitled "Phase 2 Study of A Novel Ammonia Scavenging Agent In Adults With Urea Cycle Disorders (UCDs)."

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The phase II study was an open-label, fixed sequence, switch-over study [BUPHENYL(R) (sodium phenylbutyrate) to HPN-100] of ten adult subjects with UCDs. The primary endpoint was safety; secondary outcome measures included pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy as measured by time-normalized area under the curve (TNAUC) for ammonia. Patients were enrolled on their stable dose of BUPHENYL and 24-hour PK, ammonia measurements, amino acids, and urine collections were completed in a study unit after seven days of on-study treatment. Each patient was then switched to HPN-100 at a dose providing the same amount of the active ingredient (PBA). After seven days of HPN-100 dosing, patients were re-admitted for the same battery of assessments that were completed at day seven. Both BUPHENYL and HPN-100 were administered TID with meals.

Twenty-one adverse events (AEs) occurred in seven subjects during BUPHENYL treatment and fifteen AEs occurred in five subjects with HPN-100. There w
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