Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, today announced that it has completed
enrollment in its ALN-VSP Phase I multi-center, multinational, open
label, dose escalation clinical trial. The study’s objectives were
to evaluate safety, tolerability, pharmacokinetics, and
pharmacodynamics in patients with advanced solid tumors with liver
involvement. ALN-VSP was administered to over 40 patients at doses
ranging from 0.1 to 1.5 mg/kg, with multiple patients continuing to
receive therapy on the study. Alnylam will present data from its
ALN-VSP program at the American Society of Clinical Oncology (ASCO)
Annual Meeting being held June 3 – 7, 2011.
“We are pleased to have completed enrollment in our Phase I
trial with ALN-VSP,” said Jared Gollob, M.D., Senior Director,
Clinical Research at Alnylam. “The data from this program have been
encouraging to date, and the results are important not only for the
continued advancement of ALN-VSP toward Phase II studies but also
for the overall advancement of systemically delivered RNAi
therapeutics. We will be presenting updated data from this Phase I
trial at the ASCO Annual Meeting in June.”
“Both primary liver cancer and metastatic disease of the liver
are associated with poor prognosis for patients, and new therapies
are clearly needed,” said Josep Tabernero, M.D., Chairman of the
Medical Oncology Department and Phase I Program at Vall d’Hebron
University Hospital in Barcelona, Spain. “This study represents an
important step in the development of a novel therapeutic strategy
to treat this devastating disease, and we look forward to
evaluating the full data from this trial.”
About ALN-VSP Clinical Program
ALN-VSP is Alnylam’s first systemic RNAi program and represents
the company’s first clinical program in oncology. ALN-VSP is a
systemically delivered RNAi therapeutic comprising two siRNAs
designed to target two genes critical for the growth and survival
of cancer cells: vascular endothelial growth factor (VEGF) and
kinesin spindle protein (KSP), also known as eglin 5 (Eg5). The
drug is formulated in a first-generation lipid nanoparticle
developed by Tekmira Pharmaceuticals Corporation. The ALN-VSP Phase
I trial is a multi-center, multinational, open label, dose
escalation study designed to enroll patients with advanced solid
tumors with liver involvement who have failed to respond to or have
progressed after standard treatment. The primary objective is to
evaluate safety and tolerability of up to eight potential dose
levels ranging from 0.1 to 1.7 mg/kg. Secondary objectives include
characterization of pharmacokinetics, and assessment of
pharmacodynamic effects and tumor response through Response
Evaluation Criteria for Solid Tumors (RECIST), a set of published
guidelines that define when cancer patients’ disease improves,
stabilizes, or progresses during treatment; change in tumor blood
flow or vascular permeability as measured by Dynamic Contrast -
Enhanced Magnetic Resonance Imaging (DCE-MRI); change in plasma
biomarkers of angiogenesis; and, molecular and cellular analyses of
tumor biopsy samples. Alnylam will present data from its ALN-VSP
program at the American Society of Clinical Oncology (ASCO) Annual
Meeting being held June 3 – 7, 2011. The company expects to partner
its ALN-VSP program prior to initiating a Phase II clinical study,
with the goal of initiating this study in 2012.
About Liver Cancers
Cancer affecting the liver, known as either primary or secondary
liver cancer, is associated with one of the poorest survival rates
in oncology and represents a major unmet medical need affecting a
large number of patients worldwide. Primary liver cancer, or
hepatocellular carcinoma (HCC), is one of the most common cancers
worldwide, with more than 600,000 people diagnosed each year.
Secondary liver cancer, also known as metastatic liver cancer, is
cancer that spreads to the liver from another part of the body due
to other common cancers like colon, lung, or breast cancer.
Worldwide, more than 500,000 people are diagnosed with secondary
liver cancer each year.
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
leading the translation of RNAi as a new class of innovative
medicines with a core focus on RNAi therapeutics for the treatment
of genetically defined diseases, including ALN-TTR for the
treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for
the treatment of severe hypercholesterolemia, and ALN-HPN for the
treatment of refractory anemia. As part of its “Alnylam 5x15TM”
strategy, the company expects to have five RNAi therapeutic
products for genetically defined diseases in advanced stages of
clinical development by the end of 2015. Alnylam has additional
partner-based programs in clinical or development stages, including
ALN-RSV01 for the treatment of respiratory syncytial virus (RSV)
infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT
for the treatment of Huntington’s disease. The company’s leadership
position on RNAi therapeutics and intellectual property have
enabled it to form major alliances with leading companies including
Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko
Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus
Therapeutics Inc., a company focused on discovery, development, and
commercialization of microRNA therapeutics; Regulus has formed
partnerships with GlaxoSmithKline and sanofi-aventis. Alnylam has
also formed Alnylam Biotherapeutics, a division of the company
focused on the development of RNAi technologies for application in
biologics manufacturing, including recombinant proteins and
monoclonal antibodies. Alnylam scientists and collaborators have
published their research on RNAi therapeutics in over 100
peer-reviewed papers, including many in the world’s top scientific
journals such as Nature, Nature Medicine, Nature Biotechnology, and
Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge,
Massachusetts. For more information, please visit
www.alnylam.com.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future
expectations, plans and prospects, including without limitation,
Alnylam’s expectations with respect to the presentation of data
from its Phase I clinical trial of ALN-VSP, as well as its
expectations with respect to its “Alnylam 5x15” product strategy,
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, without limitation, Alnylam’s
ability to discover and develop novel drug candidates, successfully
demonstrate the efficacy and safety of its drug candidates in human
clinical trials and establish and maintain strategic business
alliances and new business initiatives, as well as those risks more
fully discussed in the “Risk Factors” section of its most recent
annual report on Form 10-K 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.
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