May 07, 2009 Press Release for Alnylam
Alnylam Pharmaceuticals Reports First Quarter 2009 Financial Results
May 07, 2009
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May. 7, 2009--
“This past quarter has been very productive, as we have executed across
all dimensions of our company’s key value drivers – scientific
leadership, clinical pipeline, intellectual property, and business
development,” said
“Especially notable during the period was our pipeline progress, as we
initiated a Phase I trial for ALN-VSP in liver cancers and completed
enrollment for the Phase II trial with ALN-RSV01 in lung transplant
patients. As we committed to as part of our 2009 goals, we are on track
to initiate our third clinical development program this year,” said
Cash, Cash Equivalents, and
At
Net Loss
The net loss according to accounting principles generally accepted in
the U.S. (GAAP) for the first quarter of 2009 was
Revenues
Revenues were
Research and Development Expenses
Research and development (R&D) expenses were
General and Administrative Expenses
General and administrative (G&A) expenses were
Regulus Therapeutics
Alnylam incurred
Interest Income
Interest income was
Interest Expense
Interest expense was zero for the first quarter of 2009, as compared to
$0.2 million for the first quarter of 2008. Interest expense in the
first quarter of 2008 was related to borrowings under the company’s
lines of credit used to finance capital equipment purchases. In
Income Tax Expense
Primarily as a result of the company’s sale of its German operations to
2009 Financial Guidance
Alnylam continues to expect that its cash, cash equivalents, and
marketable securities balance will be greater than
“We continue to maintain one of the strongest net cash positions across
the entire biotechnology industry, ending the first quarter with
First Quarter 2009 and Recent Corporate Highlights
Product Pipeline and Scientific Leadership Highlights
- Advanced ALN-VSP for Liver Cancer into the Clinic. Alnylam initiated a Phase I multi-center, open label, dose escalation trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ALN-VSP in patients with advanced liver cancers, including hepatocellular carcinoma and other solid tumors with liver involvement. The Phase I trial is being conducted in the U.S., and represents Alnylam’s first clinical program with a systemically delivered RNAi therapeutic.
-
Completed Enrollment in Phase II Trial for ALN-RSV01. Alnylam
completed enrollment in its Phase II double-blind, randomized clinical
trial to assess the safety and tolerability of aerosolized ALN-RSV01
versus placebo in adult lung transplant patients naturally infected
with respiratory syncytial virus (RSV). Data from this trial, which
remains blinded, are expected in the second half of 2009. The ALN-RSV
program is partnered with
Kyowa Hakko Kirin Co., Ltd. inAsia , and Cubist worldwide except forAsia . - On Track to Expand Clinical Pipeline in 2009. Alnylam plans to file a new investigational new drug (IND) application from its development pipeline in 2009, with candidates including ALN-PCS for the treatment of hypercholesterolemia, ALN-HTT for the treatment of Huntington’s disease, and ALN-TTR for the treatment of transthyretin (TTR) amyloidosis. By the end of 2009, Alnylam expects to have a total of three RNAi therapeutic programs in clinical development, on track to meet its ‘RNAi 2010’ goal of four or more RNAi therapeutic programs in clinical development by the end of 2010.
-
Augmented Platform for RNAi Therapeutics. Alnylam formed a new
collaboration with
Isis Pharmaceuticals focused on the development of single-stranded RNAi (ssRNAi) technology. ssRNAi comprises chemically modified, single-stranded, RNA-like oligonucleotides that may have improved properties for systemic administration while harnessing advantages of the RNAi mechanism. As part of the collaboration, Isis has co-exclusively licensed its ssRNAi technology to Alnylam in exchange for upfront payments, research and development milestone payments, and royalties. Upon further development of the technology, the alliance also provides for both companies to have the opportunity to discover and develop drugs employing this approach. In addition to the new collaboration, Isis and Alnylam also agreed to extend their broad cross-licensing arrangement regarding double-stranded RNAi that was established in 2004. -
Continued Scientific Leadership. Alnylam continued to
demonstrate its scientific leadership through publication and
presentation of peer-reviewed research, including:
- pre-clinical research demonstrating that topical administration of an RNAi therapeutic resulted in robust and durable protection from the transmission of the herpes simplex virus-2 (HSV02) in mice (Wu et al., Cell Host & Microbe 5, 84-94; 2009);
-
pre-clinical research demonstrating that RNAi silencing of the
claudin-3 protein results in the suppression of ovarian tumor
growth and metastases (Huang et al., Proc. Natl Acad. Sci.
USA , 10.1073/pnas.0813348106); -
further characterization of “lipidoids,” a novel biomaterials
approach for systemic delivery of RNAi therapeutics, as part of a
research collaboration with scientists at
MIT (Akinc et al., Molecular Therapy advance online publication3 March 2009 ; doi:10.1038/mt .2009.36); -
structure determination of 2’-SMe containing oligonucleotide demonstrates
that this sugar modification can adopt an RNA-like conformation
suitable for siRNA applications (Allan et al.,
Chemical Communications , (Cambridge, United Kingdom ) (2009), (15), 2017-2019); -
in vitro data using RNAi demonstrating the role of FAPP2 as
a potential therapeutic target in cancer (Tritz et al.,
Biochemical and Biophysical Research Communications ,
doi:10.1016/j.bbrc.2009.03.126); and, - data from Alnylam and Regulus scientists presented at the “Therapeutic Modulation of RNA Using Oligonucleotides” Keystone Symposium from multiple RNAi and microRNA therapeutic pre-clinical and clinical programs, as well as delivery approaches.
Business Execution Highlights
-
Established Global Partnerships for Further Advancement of ALN-RSV
Program. Alnylam formed a strategic collaboration with Cubist for
the development and commercialization of the company’s ALN-RSV
program, which includes ALN-RSV01, currently in Phase II clinical
development for the treatment of RSV infection in adult lung
transplant patients, as well as several other potent and specific
second-generation RNAi-based inhibitors in pre-clinical studies.
Alnylam’s partnership with Cubist is a 50-50 co-development and profit
share arrangement in
North America , and a milestone- and royalty-bearing license arrangement in the rest of the world outside ofAsia , where ALN-RSV is partnered withKyowa Hakko Kirin . Combined, Alnylam’s ALN-RSV partnerships withKyowa Hakko Kirin and Cubist included$35.0 million in upfront payments and greater than$160.0 million in potential development and sales milestones payments. -
Regulus Raised
$20 Million in Series A Financing. Alnylam and Isis continued their investment in Regulus with a$20 million Series A preferred equity financing. Regulus is focused on the discovery, development, and commercialization of microRNA-based therapeutics, an entirely new frontier of pharmaceutical research. -
Novartis Purchased Additional Shares of Alnylam Common Stock.
In accordance with the terms of the 2005 Investor Rights Agreement
between Alnylam and
Novartis ,Novartis has elected to fully exercise its current and sole right and purchased approximately 66,000 unregistered shares of Alnylam’s common stock at a purchase price of$17.50 per share, which is equal to the average of the closing prices for Alnylam’s common stock for the 20 trading-day period ending onMarch 30, 2009 .
Intellectual Property (IP) Highlights
- European Patent Office Granted Tuschl I Patent. Alnylam received notification from the European Patent Office (EPO) of an intent to grant a patent (EP1309726) in the Tuschl I patent series. The Tuschl I patent includes claims broadly covering the use of double-stranded RNAs (dsRNAs) for RNAi.
-
Chinese Patent Office Granted Tuschl II Patent. A key
fundamental patent from Alnylam’s exclusively held Tuschl II patent
series was given an intent to grant in
China , one of the fastest growing markets in the world. The Tuschl II patent (Application No. 01820900.9) includes broad claims covering compositions, methods, uses, and systems for siRNAs. -
Australian Patent Office Granted Kay & McCaffrey Patent. The
Kay & McCaffrey patent (AU 2002326410) was granted in
Australia . The newly granted patent includes 38 claims that broadly cover methods and compositions of RNAi therapeutics. Alnylam has an exclusive license to this patent through an agreement withStanford University . -
New Patents Issued or Granted. Alnylam announced today the
issuance or grant of the following patents owned, controlled, or
licensed by Alnylam in the RNAi therapeutics field:
-
the “Glover” patent, which is exclusively licensed to Alnylam from
Cancer Research Institute (CRT) was granted by the Mexican Patent Office (Application No. 005013) and the Polish Patent Office (Application No. 356698); and, - new target-related patents were allowed by the U.S. Patent Office (7,473,525 and 7,507,809) and the Canadian Patent Office (2,513,809).
-
the “Glover” patent, which is exclusively licensed to Alnylam from
-
Provided Update on
Kreutzer-Limmer Patent Estate . The European Patent Office granted a new patent (EP 1550719) in the Kreutzer-Limmer I patent series covering siRNAs comprising 15-21 nucleotides in length stabilized by chemical linkages. In addition, the European Patent Office overturned the ‘945 patent (EP 1214945) and the German Patent Office overturned the ‘167 patent (DE 10080167), both in oral proceedings before respective Opposition Boards. Alnylam intends to appeal these initial rulings.
Organizational Highlights
- Strengthened Management Team with Key Promotion. Alnylam promoted Akshay Vaishnaw, M.D., Ph.D. to the role of Senior Vice President, Clinical Research of Alnylam. In this role, Dr. Vaishnaw will be responsible for continued leadership of the company’s pre-clinical, clinical, and regulatory activities.
Conference Call Information
Management will provide an update on the company, discuss first quarter
2009 results, and discuss expectations for the future via conference
call on
A live audio webcast of the call will also be available on the “Investors” section of the company’s website, www.alnylam.com. An archived webcast will be available on the Alnylam website approximately two hours after the event and will be archived for 14 days.
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. RNAi therapeutics target the cause of diseases by potently silencing specific messenger RNAs (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 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; its most advanced program is in Phase II human
clinical trials for the treatment of respiratory syncytial virus (RSV)
infection and is partnered with Cubist and
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future
expectations, plans and prospects, including without limitation, its
expectations with respect to the timing and success of its research,
clinical and pre-clinical trials and regulatory filings, including the
ability to invest significantly in its pipeline and delivery technology,
its cash position at the end of 2009, and its non-GAAP cash net
operating loss during 2009, constitute forward-looking statements for
the purposes of the safe harbor provisions under
For Media Inquiries, please contact:
Christine Akinc
Chief Corporate Communications Officer media@alnylam.com 617-682-4340
For Investor Inquiries, please contact:
Josh Brodsky
VP, Investor Relations & Corporate Communications investors@alnylam.com 617-551-8276
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