Horizon Pharma plc
HZNP, a specialty biopharmaceutical company with a
portfolio of products in arthritis, inflammation and orphan diseases,
announced today the presentation of data from a Phase 2 clinical
study of ACTIMMUNE(R) (interferon gamma-1b) treatment in children
with Friedreich's ataxia (FA). An abstract of the data has been
published in a supplement to the Annals of Neurology and is being
presented as a poster during the 139th Annual Meeting of the American
Neurological Association in Baltimore, MD on Monday, October 13.
This single center, Phase 2 open-label study of 12 children ages five
to 17 years with genetically confirmed FA with treatment of up to 12
weeks, evaluated the safety, tolerability and efficacy of ACTIMMUNE.
The study measured frataxin levels, a biomarker of disease and
clinical measures (Friedreich's Ataxia Rating Scale [FARS] score and
other neurological evaluations) as efficacy markers. Additional
details on the study design may be found at www.clinicaltrials.gov.
The results, as noted in the published abstract, showed ACTIMMUNE was
well tolerated with no serious adverse events and two subjects
reporting severe events and subsequent dose reductions. The safety
findings generally reflected the labeled safety profile for
ACTIMMUNE. Changes in frataxin protein levels, the primary study
endpoint, were statistically significant in red blood cells, white
blood cells and platelets. The magnitude of change observed was small
and varied between tissues. Mean improvement in the FARS score, a
clinically validated measurement of patient performance and secondary
endpoint, was statistically significant (p=0.008) and was equivalent
to two years of extension of disease progression. No other changes
were observed.
"The highly significant improvement in the FARS score, a key measure
of improvement in disease progression, supports further study of
ACTIMMUNE in Friedreich's ataxia," said Timothy P. Walbert, chairman,
president and chief executive officer, Horizon Pharma plc. "We plan
to work with the Collaborative Clinical Research Network in
Friedreich's Ataxia (CCRN in FA) and the Friedreich's Ataxia Research
Alliance (FARA) to rapidly move into a registration program for
ACTIMMUNE in FA. We plan to meet with the FDA this quarter regarding
the clinical development and regulatory pathway for ACTIMMUNE in FA."
The study was sponsored by FARA and conducted by David Lynch, M.D.,
Ph.D., professor of neurology at Children's Hospital of Philadelphia
(CHOP) and principal investigator of the CCRN in FA. Investigational
product was provided by Vidara Therapeutics Research Limited, now
part of Horizon Pharma plc.
"The FA community has been working toward a viable treatment option
and we were proud to be able to sponsor this initial pilot study. We
are encouraged that ACTIMMUNE was well tolerated and there was a
significant improvement observed in a clinical outcome measure," said
Ronald Bartek, president, director and co-founder of FARA. "We are
very pleased to partner with Horizon Pharma and look forward to
working together to explore the potential for ACTIMMUNE in FA."
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