PRIME designation granted by European Medicines Agency for RG6042 for treatment of Huntington's disease
August 03 2018 - 1:00AM
F. Hoffmann-La Roche Ltd / PRIME designation granted by European
Medicines Agency for RG6042 for treatment of Huntington's disease .
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is solely responsible for the content of this announcement.
-
European Medicines Agency PRIME
(PRIority MEdicines) status is granted to medicines that may offer
a major therapeutic advantage over existing treatments, or benefit
patients without treatment options
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RG6042 has the potential to be
the first therapy targeting the underlying cause of Huntington's
disease, a fatal neurodegenerative rare disease
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Third PRIME designation for a
Roche medicine
Basel, 3 August 2018 - Roche (SIX: RO, ROG; OTCQX:
RHHBY) today announced that the European Medicines Agency (EMA) has
granted PRIME (PRIority MEdicines) designation for the company's
investigational medicine RG6042 (formerly known as IONIS-HTTRx) for
the treatment of people with Huntington's disease (HD). RG6042 has
demonstrated its ability to reduce the toxic mutant huntingtin
protein (mHTT), which is believed to be the underlying cause of HD,
in a Phase I/IIa study.[1] PRIME is a
designation implemented by the EMA to support data generation and
development plans for promising medicines, providing a pathway for
accelerated evaluation by the agency, and thus potentially enable
them to reach patients earlier.[2]
"We are very pleased that the European
Medicines Agency has granted PRIME designation for RG6042, as there
is an urgent medical need to find treatment options for families
affected by Huntington's disease," said Sandra Horning, MD, Roche's
Chief Medical Officer and Head of Global Product Development.
"Preliminary data on RG6042 were the first to show that levels of
toxic mutant huntingtin protein can be lowered in adults with
Huntington's disease, and we are working closely with the EMA and
other health authorities to initiate a global phase III study as
soon as possible."
PRIME designation for RG6042 is primarily based on the data from an
exploratory Phase I/IIa trial of RG6042 that demonstrated a
significant reduction in mHTT, which breaks down the nerve cells in
the brain.[1] The study
demonstrated a mean 40% (up to 60%) reduction of the specific HD
protein in the cerebrospinal fluid (CSF) of adult patients treated
with RG6042 for three months at the two highest doses. Furthermore,
levels of mHTT measured in the CSF were still declining in the
majority of treated patients (~70%) as of the last measurement in
the study.[3] RG6042 was
well tolerated in this short initial study.[1] These
data were shared at the CHDI 13th Annual HD Therapeutics Conference
in March 2018,[3] and updated
results were presented at the American Academy of Neurology (AAN)
Annual Meeting in April 2018.[4]
Roche will initiate a pivotal phase III
study to evaluate RG6042 in a larger patient population to further
characterise the safety profile and determine if it can slow the
progression of HD in adults.
About RG6042
RG6042 is a
second-generation modified antisense oligonucleotide (ASO) designed
to reduce the production and levels of mHTT protein by targeting
human HTT mRNA.[5] RG6042 is
the result of a comprehensive drug discovery programme between
Roche and Ionis Pharmaceuticals focused on optimising the potency,
specificity and tolerability of an ASO targeting human HTT mRNA.
RG6042 is the most advanced compound in clinical development to
target toxic mutant huntingtin protein (mHTT), which is believed to
be the underlying cause of HD. Treatment with RG6042 has the
potential to slow or stop disease progression in all people with
HD.[1]
About Huntington's disease
Huntington's disease is a rare genetic, progressive condition that
causes the nerve cells in the brain to break down, which severely
affects a person's everyday functions such as mobility and
thinking.[6] It has a
devastating impact on people living with the disease, and the
hereditary nature of HD means it profoundly affects entire
families.[6] As the
disease progresses, people with HD may develop personality changes,
difficulty walking and swallowing, as well as having a significant
cognitive impact.[6] Survival
ranges from approximately 10-20 years following motor onset of the
disease.[6]
There is no known cure for HD and no
approved therapies that treat the underlying cause. The estimates
for the number of people affected by Huntington's vary between
geographic regions. Huntington's disease is the most common
monogenic neurological disorder in the developed world, with an
estimated prevalence of ~3.5-7/100,000 in North America, Western
Europe, and Australia.[7]
About Roche in
neuroscience
Neuroscience is a major focus of
research and development at Roche. The company's goal is to develop
treatment options based on the biology of the nervous system to
help improve the lives of people with chronic and potentially
devastating diseases. Roche has more than a dozen investigational
medicines in clinical development for diseases that include
multiple sclerosis, Alzheimer's disease, spinal muscular atrophy,
Parkinson's disease, Huntington's disease and autism spectrum
disorder.
About Roche
Roche is a
global pioneer in pharmaceuticals and diagnostics focused on
advancing science to improve people's lives. The combined strengths
of pharmaceuticals and diagnostics under one roof have made Roche
the leader in personalised healthcare - a strategy that aims to fit
the right treatment to each patient in the best way
possible.
Roche is the world's largest biotech company, with truly
differentiated medicines in oncology, immunology, infectious
diseases, ophthalmology and diseases of the central nervous system.
Roche is also the world leader in in vitro diagnostics and
tissue-based cancer diagnostics, and a frontrunner in diabetes
management.
Founded in 1896, Roche continues to search for better ways to
prevent, diagnose and treat diseases and make a sustainable
contribution to society. The company also aims to improve patient
access to medical innovations by working with all relevant
stakeholders. Thirty medicines developed by Roche are included in
the World Health Organization Model Lists of Essential Medicines,
among them life-saving antibiotics, antimalarials and cancer
medicines.
Roche has been recognised as the Group Leader in sustainability
within the Pharmaceuticals, Biotechnology & Life Sciences
Industry nine years in a row by the Dow Jones Sustainability
Indices (DJSI).
The Roche Group, headquartered in Basel, Switzerland, is active in
over 100 countries and in 2017 employed about 94,000 people
worldwide. In 2017, Roche invested CHF 10.4 billion in R&D and
posted sales of CHF 53.3 billion. Genentech, in the United States,
is a wholly owned member of the Roche Group. Roche is the majority
shareholder in Chugai Pharmaceutical, Japan. For more information,
please visit www.roche.com.
All trademarks used or mentioned in this release are protected by
law.
References
[1] Ionis
Pharmaceuticals. 2018. Press release: IONIS-HTT Rx (RG6042)
top-line data demonstrate significant reductions of disease-causing
mutant Huntingtin protein in people with Huntington's disease.
[Internet; cited 2018 July]. Available from:
http://ir.ionispharma.com/node/23401/pdf
[2] European Medicines Agency. PRIME Designation. [Internet; cited
2018 July]. Available from:
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/
general_content_000660.jsp&mid=WC0b01ac05809f8439.
[3] CHDI 13th Annual HD Therapeutics Conference 2018. Press
Release: Ionis Pharmaceuticals Licenses IONIS-HTT Rx to Partner
Following Successful Phase 1/2a Study in Patients with Huntington's
Disease. [Internet; cited 2018 July]. Available from:
https://chdifoundation.org/ionis-pharmaceuticals-licenses-ionis-htt-rx-to-partner-following-successful-phase-12a-study-in-patients-with-huntingtons-disease/.
(Data on file)
[4] Ionis Pharmaceuticals. 2018. Press release: New Data from
IONIS-HTT Rx Phase 1/2 Study Demonstrates Correlation Between
Reduction of Disease-causing Protein and Improvement in Clinical
Measures of Huntington's Disease. [Internet; cited 2018 July].
Available from: http://ir.ionispharma.com/node/23661/pdf.
[5] Leavitt B, Tabrizi S, Kordasiewicz H et al. Discovery and early
clinical development of ISIS-HTTRx, the first HTT-lowering drug to
be tested in patients with Huntington's disease (PL01.002).
Neurology 2016;86(Suppl. 16):PL01.002. [Internet; cited 2018 July].
Available from:
http://n.neurology.org/content/86/16_Supplement/PL01.002.
[6] NHS Choices. Huntington's disease: Overview. [Internet; cited
2018 July]. Available from:
https://www.nhs.uk/conditions/huntingtons-disease/.
[7] Rawlins M, et al. The Prevalence of Huntington's Disease.
Neuroepidemiology 2016;46:144-153.
Roche Group Media Relations
Phone: +41 61 688 8888 / e-mail:
media.relations@roche-global.com
- Nicolas Dunant (Head)
- Patrick Barth
- Ulrike Engels-Lange
- Simone Oeschger
- Anja von Treskow
20180803_Roche_MR_ASO_HTT_PRIME
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Source: F. Hoffmann-La Roche Ltd via Globenewswire
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