HALT-HE Study Underscores Role of Ammonia in Hepatic Encephalopathy
Hyperion Reports Phase 2 Study Results at AASLD Plenary Session
SOUTH SAN FRANCISCO, Calif., Nov. 12, 2012 (GLOBE NEWSWIRE) -- Hyperion Therapeutics, Inc. (Nasdaq:HPTX) announced today that the results of the Phase 2 HALT-HE Study were presented at the Presidential Plenary Session I of the 63rd Annual Meeting of the American Association for the Study of Liver Diseases (AASLD) (The Liver Meeting(R)), in Boston, MA.
Principal Investigator Don Rockey, Chief of Medicine at the Medical University of South Carolina delivered an oral presentation entitled, Randomized, controlled, double-blind study of glycerol phenylbutyrate in patients with cirrhosis and episodic hepatic encephalopathy.
Dr. Rockey explained, "The study results provide new insight into the importance of ammonia in the pathogenesis of hepatic encephalopathy (HE). Based on its safety profile, Hyperion's investigational drug, glycerol phenylbutyrate (GPB), or "Ravicti", shows promise as a novel therapeutic agent."
Dr. Rockey explained,
-- The study met its primary endpoint: significantly fewer patients
in the
active arm experienced HE events -- 21% vs. 36% (p = 0.021).
-- Compared to placebo, patients randomized to GPB also had
significantly
fewer total HE events (35 vs. 57 p=0.035), significantly lower
ammonia
values (46 vs. 58 umol/L p=0.036), and significantly fewer
symptomatic
days (13 vs. 27; p=0.015).
-- There were non-statistically significant trends in favor of
fewer
HE-hospitalizations and fewer total HE-hospital days.
-- Safety appeared satisfactory (76% and 79% of subjects in the
placebo and
GPB groups, respectively, reported AEs) and the adverse event
profile
was consistent with that expected for the study population.
-- Ammonia was significantly lower on GPB (45.7 vs. 58.15 umol/L, p
=
0.036) and correlated with the likelihood of HE events whether
measured
at baseline or during the study (p < 0.01)
The study enrolled 178 total patients, of whom 59 were on Rifaximin
at baseline, including 29 in the placebo arm and 30 in the active
arm.
Among the 119 patients not on Rifaximin at baseline, there was a
highly statistically significant reduction among GPB treated
patients in both the percentage of patients with events (10% vs
32%; p=0.003) as well as the total number of HE events (7 vs. 31;
p<0.001). Among patients on Rifaximin at baseline, there was no
difference in the number of patients with events or total events.
Ammonia was similarly lowered on GPB among patients taking (67 vs.
91 umol/L; p=0.13) or not taking (36 vs. 43 umol/L; p=0.08),
although the differences were not statistically significant due to
the smaller sample size. Patients on Rifaximin at baseline had
higher ammonia levels than those not on Rifaximin.
The abstract is published online at
https://www.aasld.org/lm2012. The HALT-HE study was a Phase II,
multi-center, randomized, double-blind trial of glycerol
phenylbutyrate vs. placebo in 178 patients with episodic HE
recruited from 28 sites in the United States, 9 sites in Russia and
7 sites in Ukraine. 88 patients were randomized to the placebo
group and 90 to the GPB arm. The 178 patients enrolled constituted
the intention to treat and safety populations. 55 patients in the
GPB arm and 67 patients in the placebo arm completed the
study.
The population was well balanced with respect to lactulose and
Rifaximin use, prior HE history and MELD score.
Hyperion also indicated that it was hosting and webcasting an analyst and investor briefing to recap the Plenary Session presentation tonight, November 12, 2012, in Boston, MA at from 6:30 p.m. -- 7:30 p.m. ET in the Commonwealth Room of the Sheraton Hotel, connected to the Boston Hynes Convention Center. The webcast can be accessed from Hyperion's website www.hyperiontx.com in the Investors tab, under Events.
About Hepatic Encephalopathy
HE is a serious but potentially reversible neurological disorder
that can occur in patients with cirrhosis of any etiology or acute
liver failure. HE comprises a spectrum of neurological signs and
symptoms ranging from mild (e.g. minimal disorientation) to severe
(e.g. coma,
death) and is believed to occur when the brain is exposed to
gut-derived toxins such as ammonia that are normally removed from
the blood by a healthy liver. Based on the current epidemiological
literature, Hyperion estimates that there are approximately one
million patients in the United States with cirrhosis, of whom
approximately
140,000 have overt HE.
About Glycerol Phenylbutyrate
Glycerol phenylbutyrate, an investigational drug, is a
pre-pro-drug of phenylacetic acid, the active moiety of
BUPHENYL(R), the only branded therapy currently FDA-approved as
adjunctive therapy for the chronic management of patients with the
most prevalent urea cycle disorders.
Glycerol phenylbutyrate holds orphan product designations in the
United States and Europe for the maintenance treatment of patients
with urea cycle disorders (UCD) and in the United States for the
intermittent or chronic treatment of patients with cirrhosis and
any grade of hepatic encephalopathy.
About Hyperion Therapeutics
Hyperion Therapeutics is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat disorders in the areas of orphan diseases and hepatology. Hyperion Therapeutics is developing Ravicti(TM) (glycerol phenylbutyrate) for two orphan indications: UCD and HE.
BUPHENYL(R) is a registered trademark of Ucyclyd Pharma, Inc.
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. Examples of
forward-looking statements contained in this press release include,
among others, statements regarding our expectations regarding the
timing of the presentation of the results of the HALT-HE study in
November. 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
7 and any subsequent filings with the Securities and Exchange
Commission.
CONTACT: Shari Annes, Investor Relations
Telephone: 650 888 0902
sannes@annesassociates.com
Posted: November 2012
