Alnylam Pharmaceuticals - News Release
Alnylam, Medtronic, and CHDI Foundation Form Collaboration to Advance RNAi
Therapeutics for the Treatment of Huntington's Disease
- Commitment from CHDI to Fund up to 50% of Development Efforts toward IND Filing
to Advance ALN-HTT, a Novel Drug-Device
Combination -
CAMBRIDGE, Mass. & MINNEAPOLIS & NEW YORK, Nov 03, 2010 (BUSINESS WIRE)
--
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), Medtronic Inc. (NYSE:MDT), and
CHDI Foundation, Inc., announced today that they have formed a collaboration
to dvance ALN-HTT, a novel drug-device combination for the treatment of Huntington's
disease. ALN-HTT consists of an RNAi therapeutic targeting huntingtin, the gene
responsible for Huntington's disease, that is being developed for delivery to
the central nervous system (CNS) using an implantable infusion system developed
by Medtronic. CHDI is a not-for-profit virtual biotech company that is exclusively
dedicated to rapidly discovering and developing therapies that slow the progression
of
Huntington's disease.
"Alnylam and Medtronic have shown leadership and encouraging progress
in developing a novel drug-device combination for the treatment of Huntington's
disease," said Robi Blumenstein, President of CHDI Management. "Their
collaborative approach demonstrates a combined commitment to tackle this devastating
disease and this
program is closely aligned with CHDI's mission. We welcome the opportunity to
accelerate this program and look forward to continued advancement toward clinical
testing in patients." Alnylam and Medtronic have worked collaboratively
to advance ALN-HTT for the treatment of Huntington's disease. Under this new
collaboration, CHDI has agreed to initially fund up to 50% of the investigational
new drug (IND) application-enabling activities. The agreement between Alnylam
and Medtronic will remain as a 50-50 partnership in the United States. Medtronic
will commercialize the therapy consisting of the RNAi compound and delivery
device. In the
United States, Alnylam has the opportunity to invest in clinical development
through product launch in return for a proportional share of the profits. In
Europe, Medtronic is solely responsible for development and commercialization,
and Alnylam is eligible to receive milestones and royalties.
"We are very pleased to add CHDI as a partner in our Huntington's disease
program; they bring a tremendous amount of disease area expertise that will
contribute to advancing ALN-HTT towards the clinic with Medtronic," said
Dinah Sah, Ph.D., Vice President, Research, at Alnylam. "Our pre-clinical
data provide a strong
rationale for this program, and we remain encouraged by the potential that ALN-HTT
may offer to patients."
Over the course of the existing collaboration, pre-clinical data have been generated supporting the continued development of ALN-HTT for the treatment of Huntington's disease, including:
• demonstration that an siRNA targeting the huntingtin gene achieves sufficient distribution for coverage of brain regions affected in Huntington's disease;
• data showing that direct delivery to the CNS results in robust silencing
of the huntingtin gene mRNA which was achieved at substantial distances from
the infusion site, an important step towards translating this delivery approach
from pre- clinical models to the larger human brain; and,
• results showing that ALN-HTT is well tolerated following continuous
direct CNS administration over a period of approximately one month.1
"The ALN-HTT program represents an exciting opportunity to combine innovative
medicines with our drug delivery technology in an area of extreme unmet medical
need," said Gregory Stewart, Ph.D., Director of CNS Drug Therapy R&D
in the Neuromodulation Business at Medtronic. "With no effective disease-modifying
therapies available currently for patients afflicted with Huntington's disease,
the collaboration between Alnylam and Medtronic, and now support from CHDI,
will work to develop a novel treatment strategy for this devastating neurodegenerative
disease."
About Huntington's Disease
Huntington's disease is an autosomal dominant neurodegenerative genetic disorder
that causes motor, cognitive, and behavioral dysfunction. It is estimated that
120,000 people in the U.S. have the genetic mutation that causes Huntington's
disease and will experience symptoms during a normal lifetime. The average lifespan
for patients after onset of motor dysfunction is approximately 10 to 20 years.
There are currently no disease-modifying therapies available to slow the progression
of Huntington's disease.
About RNA Interference (RNAi)
RNAi (RNA interference) is a revolution in biology, representing a breakthrough
in understanding how genes are turned on and off in cells, and a completely
new approach to drug discovery and development. Its discovery has been heralded
as "a major scientific breakthrough that happens once every decade or so,"
and represents
one of the most promising and rapidly advancing frontiers in biology and drug
discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine.
RNAi is a natural process of gene silencing that occurs in organisms ranging
from plants to mammals. By harnessing the natural biological process of RNAi
occurring in our cells, the creation of a major new class of medicines, known
as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the
molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform,
target the cause of diseases by potently silencing specific mRNAs, thereby preventing
disease-causing
proteins from being made. RNAi therapeutics have the potential to treat disease
and help patients in a fundamentally new way.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel therapeutics based
on RNA interference, or RNAi. The company is applying its therapeutic expertise
in RNAi to address significant medical needs, many of which cannot effectively
be addressed with small molecules or antibodies, the current major classes of
drugs. Alnylam is leading the translation of RNAi as a new class of innovative
medicines with peer-reviewed research efforts published in the world's top scientific
journals including Nature, Nature Medicine, and Cell. The company is leveraging
these capabilities to build a broad pipeline of RNAi therapeutics for the treatment
of a wide
range of disease areas, including respiratory syncytial virus (RSV), liver cancers,
TTR-mediated amyloidosis (ATTR), hypercholesterolemia, and Huntington's disease.
In addition, Alnylam formed Alnylam Biotherapeutics, a division of the company
focused on the development of RNAi technologies for application in manufacturing
processes for biotherapeutic products, including recombinant proteins and monoclonal
antibodies. The company's leadership position in fundamental patents, technology,
and know-how relating to RNAi has enabled it to form major alliances with leading
companies including Medtronic, Novartis, Biogen Idec, Roche, Takeda,
Kyowa Hakko Kirin, and Cubist. Alnylam and Isis are majority owners of Regulus
Therapeutics Inc., a company focused on the discovery, development, and commercialization
of microRNA therapeutics. Founded in 2002, Alnylam maintains headquarters in
Cambridge, Massachusetts.
For more information, please visit www.alnylam.com.
About Medtronic
Medtronic, Inc. (www.medtronic.com), headquartered in Minneapolis, is the global leader in medical technology - alleviating pain, restoring health and extending life for millions of people around the world.
Any forward-looking statements are subject to risks and uncertainties such
as those described in Medtronic's periodic reports on file with the Securities
and Exchange Commission. Actual results may differ materially from anticipated
results.
About CHDI Foundation, Inc.
CHDI Foundation, Inc. is a privately funded, not-for-profit, virtual biotech
company that is exclusively dedicated to rapidly discovering and developing
therapies that slow the progression of Huntington's disease. CHDI's scientists
work closely with a network of more than 600 researchers in academic and industrial
laboratories around the world in the pursuit of these novel therapies, providing
project management to ensure that their common goals remain in focus. This helps
bridge the translational gap that often exists between academic and industrial
research pursuits that adds costly delays to therapy development. In its role
as a collaborative enabler, CHDI seeks to bring the right partners together
to identify and address critical scientific issues and move drug candidates
to clinical evaluation as rapidly as possible. CHDI's activities extend from
exploratory biology to the identification and validation of therapeutic targets,
and from drug discovery and development to
clinical studies and trials. More information about CHDI can be found at www.chdifoundation.org.
Alnylam Forward-Looking Statement
Various statements in this release concerning Alnylam's future expectations,
plans and prospects, including without limitation, expectations regarding the
development of effective and efficient approaches for the development of a combination
RNAi therapeutic and device for the treatment of Huntington's disease and expectations
regarding the potential funding for such a program from CHDI Foundation, Inc.,
constitute forward-looking statements for the purposes of the safe harbor provisions
under The Private Securities Litigation Reform Act of 1995. Actual results may
differ materially from those indicated by these forward-looking statements as
a result
of various important factors, including but not limited to: Alnylam's ability
to discover and develop novel drug candidates, such as ALN-HTT, a combination
drug-device for the treatment of Huntington's disease, and successfully demonstrate
efficacy and safety of ALN-HTT in pre-clinical IND-enabling studies; results
from
pre-clinical testing of a product candidate, including ALN-HTT, that may not
predict the results that will be obtained in subsequent pre-clinical or human
clinical trials; and Alnylam's dependence on collaborators for funding, development,
manufacture, marketing, sales and/or distribution of products; as well as those
risks more
fully discussed in the "Risk Factors" section of its most recent quarterly
report on Form 10-Q on file with the Securities and Exchange Commission. In
addition, any forward-looking statements represent Alnylam's views only as of
today and should not be relied upon as representing its views as of any subsequent
date. Alnylam
does not assume any obligation to update any forward-looking statements.