Hyperion Therapeutics Initiates Clinical Study to Further Evaluate RAVICTI(R)

Brisbane, CA — Hyperion Therapeutics, Inc. (Nasdaq:HPTX) today announced the dosing of the first patient in its HPN-100-009 study in pediatric subjects under two years of age with urea cycle disorders (UCDs). This open-label study and post-marketing requirement is designed to assess the efficacy, safety and pharmacokinetics of RAVICTI and will enroll UCD patients who are newly diagnosed or being switched from sodium phenylbutyrate. Hyperion estimates that patients in the first two years of life account for 15-20% of all patients diagnosed with UCDs and approximately 10% of the total volume of phenylbutyrate used in the U.S.

“The initiation of protocol HPN-100-009 reflects our continuing commitment to the UCD community,” said Donald J. Santel, president and chief executive officer of Hyperion. “This study is designed to generate data which allow future label expansion and provide families with an alternative to sodium phenylbutyrate. We look forward to the day when even newborn patients can potentially benefit from RAVICTI.”

Eligible subjects will range in age from newborn to under 2 years, have a confirmed or suspected UCD diagnosis and will be treated for six to 24 months, depending on age at enrollment. The study’s primary objective is the successful initiation of RAVICTI as assessed by control of blood ammonia, and both initiation of dosing and subsequent dose adjustment will be informed by measurement of blood ammonia as well as plasma and urine metabolites. Secondary outcome measures include the rate of hyperammonemic crises during the first six months on RAVICTI and the rate of adverse events. More information about the clinical trial is available by visiting www.clinicaltrials.gov (identifier NCT02246218).

RAVICTI (glycerol phenylbutyrate) Oral Liquid was approved by the U.S. FDA on February 1, 2013, for use as a nitrogen-binding agent for chronic management of adult and pediatric patients ≥2 years of age with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI is a nearly odorless, nearly tasteless liquid that does not contain sodium. A little over 1 teaspoon given 3 times daily is equivalent to 40 tablets of sodium phenylbutyrate.

About Urea Cycle Disorders

Urea cycle disorders, or UCDs, are a collection of inherited metabolic disorders in which affected patients suffer from high levels of systemic ammonia, a potent neurotoxin. Ammonia is produced via normal protein ingestion; the body normally detoxifies it by converting it through a series of enzymatic steps to urea, which is excreted in urine. Throughout their lives, UCD patients may experience recurrent hyperammonemic crises in which ammonia levels rise, and can develop complications ranging from nausea, vomiting and headache to coma and death.

About Hyperion Therapeutics

Hyperion Therapeutics, Inc. is a commercial-stage biopharmaceutical company committed to developing and delivering life-changing treatments for orphan diseases. The company’s first commercial product, RAVICTI (glycerol phenylbutyrate) Oral Liquid, was approved in February 2013 and is currently being marketed in the United States. The company also owns worldwide rights to BUPHENYL (sodium phenylbutyrate) Tablets and Powder, which it markets in the U.S. The compound is also marketed internationally through business partners. In addition, the company is developing glycerol phenylbutyrate for the potential treatment of hepatic encephalopathy. For more information, please visit www.hyperiontx.com.

Forward-Looking Statements

To the extent that statements contained in this press release are not descriptions of historical facts regarding Hyperion, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as “may,” “will,” “expect,” “anticipate,” “estimate,” “intend,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements contained in this press release include the potential label expansion of RAVICTI to include patients under two years of age and the percentage of patients with UCDs under two years of age and the percentage of phenylbutyrate volume those patients may consume. Hyperion undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties relating to the business of the company in general, see Hyperion’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 6, 2014, and any subsequent filings with the Securities and Exchange Commission.

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