Nov 04, 2009 Press Release for Alnylam
Alnylam Pharmaceuticals Reports Third Quarter 2009 Financial Results
Nov 04, 2009
"Activities this quarter reflect the substantial progress we are making
as we continue to lead the industry's efforts in the discovery and
development of RNAi therapeutics," said
"Over the last several months, we demonstrated the continued strength of
our existing partnerships having advanced our collaboration with
Cash,
At
Net Loss
The net loss according to accounting principles generally accepted in
the U.S. (GAAP) for the third 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 third quarter of 2009, as compared to
$0.2 million for the third quarter of 2008. Interest expense in the
third quarter of 2008 was related to borrowings under the company's
lines of credit used to finance capital equipment purchases. In
Income Tax Benefit
Income tax benefit, primarily as a result of increased legal activities
in 2009, was
2009 Financial Guidance
Alnylam is continuing to provide guidance with respect to its cash, cash
equivalents, and marketable securities balance, which it now expects
will be greater than
"We continue to have a leading net cash position across the
biotechnology industry, ending the quarter with over
Third Quarter 2009 and Recent Corporate Highlights
Product Pipeline and Scientific Leadership Highlights
-
Advanced Development of ALN-RSV Program for the Treatment of RSV Infection.- Alnylam and Cubist presented complete data from a Phase II randomized, double-blind study of inhaled ALN-RSV01 or placebo in RSV-infected lung transplant patients. This study achieved its primary objective of demonstrating the safety and tolerability of ALN-RSV01. In addition, while the study was not powered for effects on viral or clinical endpoints and results should be considered exploratory due to the small sample size, ALN-RSV01 treatment was associated with improved recovery of lung function (forced expiratory volume in the first second, or FEV1) and a statistically significant reduction in the incidence of new or progressive bronchiolitis obliterans syndrome (BOS), a life-threatening complication of RSV infection and an irreversible disease of the transplanted lung resulting in approximately 50% mortality within three to five years of onset.
- After evaluating the clinical data for ALN-RSV01, Alnylam and Cubist have agreed to develop ALN-RSV01 in the adult lung transplant setting in parallel with the development of a second-generation compound, ALN-RSV02, which will be focused on the pediatric patient population. Alnylam plans to advance ALN-RSV01 in a new Phase IIb clinical trial in RSV-infected adult lung transplant patients, and expects to begin enrolling in this study in early 2010. The objective of this new study is to repeat and extend the clinical results observed in the original Phase II study. Alnylam will fund the continued advancement of ALN-RSV01, and Cubist will retain an opt-in right for the product in the adult transplant indication. Cubist will take the lead in advancing ALN-RSV02 in the pediatric setting in continued collaboration and 50-50 funding with Alnylam.
- During the period, Alnylam and Cubist also completed additional Phase I studies with ALN-RSV01 in healthy adult volunteers, exploring different dose regimens.
-
The ALN-RSV program is partnered with
Kyowa Hakko Kirin Co., Ltd. inAsia , and Cubist worldwide exceptAsia .
-
Continued Development of ALN-VSP for Liver Cancers. Alnylam continues to enroll patients in 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 solid tumors with liver involvement, including hepatocellular carcinoma (HCC). A significant number of patients have been enrolled across multiple dose cohorts, and Alnylam expects to present preliminary data from the Phase I trial in mid-2010. This is the company's first systemic RNAi program, and represents Alnylam's first clinical program in an oncology indication. - Advanced ALN-TTR for Transthyretin (TTR)-Mediated Amyloidosis (ATTR) Toward the Clinic. The company is on track to file regulatory applications for its ALN-TTR program by the end of this year, with the goal of initiating the Phase I trial in early 2010. ALN-TTR is a systemically delivered RNAi therapeutic targeting the TTR gene for the treatment of TTR-mediated amyloidosis. Pre-clinical data with ALN-TTR have demonstrated potent silencing of both the normal and mutated TTR gene in rodent and non-human primate studies. Further, pre-clinical studies recently presented at The Liver Meeting® demonstrated the ability of a single dose of ALN-TTR to silence TTR mRNA levels for a period of several weeks.
-
Presented New Pre-Clinical Data from ALN-HTT for Huntington's
Disease.
Alnylam andMedtronic, Inc. presented new pre-clinical research findings from their Huntington's disease program at the 2009World Congress on Huntington's Disease heldSeptember 12-15, 2009 inVancouver, British Columbia . In these advanced pre-clinical studies, therapeutic silencing of the disease-causing huntingtin gene was demonstrated in a manner supportive of scaled effects for the human brain, and continued infusion over approximately one month of ALN-HTT in the central nervous system (CNS) was found to be well tolerated.
-
Continued to Advance Platform for Delivery of RNAi Therapeutics.
Tekmira Pharmaceuticals Corp. and Alnylam are participating in a new research collaboration formed with scientists at TheUniversity of British Columbia andAlCana Technologies, Inc. , focused on the discovery of novel cationic lipids and lipid nanoparticles for the systemic delivery of RNAi therapeutics. -
Participation in Major Cardiovascular Research
Grant Awarded toUniversity of Pennsylvania . TheUniversity of Pennsylvania was awarded a$2.4 million grant under the "American Recovery and Reinvestment Act of 2009," which will fund research over two years in the labs ofDaniel Rader , M.D. of theUniversity of Pennsylvania School of Medicine , andSekar Kathiresan , M.D. ofMassachusetts General Hospital (MGH) andHarvard Medical School , and at Alnylam. The goal of the initiative is to mechanistically evaluate the metabolic and molecular effects of 38 novel genes implicated by human genetic studies in prevention of cardiovascular disease, and to discover and develop RNAi therapeutics toward these candidate genes. -
Continued Scientific Leadership. Alnylam continued to
demonstrate its scientific leadership through publication of
peer-reviewed research papers that total 13 thus far in 2009. During
the third quarter and the recent period alone, these include:
-
pre-clinical research characterizing the role of chemical
modifications and drug delivery formulations in modifying
immunostimulatory properties of double-stranded RNAs (dsRNAs)
(Nguyen et al., Molecular Therapy advance online
publication
7 July 2009 . Doi:10.1038/mt .2009.147); -
pre-clinical research demonstrating the silencing of FAPP2 gene
results in increased tumor cell sensitivity to FAS-induced
apoptosis (Tritz et al., Biochem Biophys Res Commun. 2009
May 29 ;383(2):167-71. Epub 2009 Mar 31); -
pre-clinical research demonstrating a role for miR-196 in
patterning the chick axial skeleton through Hox gene regulation
(McGlinn et al.,
Proc Natl Acad Sci USA . 2009 Oct. 21); -
pre-clinical research demonstrating antisense inhibition of miR-21
and miR-221 arrests cell cycle, induces apoptosis, and sensitizes
the effects of gemcitabine in pancreatic adenocarcinoma (Park et
al., Pancreas 2009
Sep 2 ); and, -
pre-clinical research demonstrating that the target site of
ALN-RSV01 is highly conserved across clinical isolates of RSV, and
that ALN-RSV01 has the ability to achieve substantially improved
anti-viral potency with multi-dose administration (Alvarez et
al., Antimicrob Agents Chemother. 2009
Sep;53(9):3952-62 Epub 2009
Jun 8 ).
-
pre-clinical research characterizing the role of chemical
modifications and drug delivery formulations in modifying
immunostimulatory properties of double-stranded RNAs (dsRNAs)
(Nguyen et al., Molecular Therapy advance online
publication
The company believes that it is on track to meet its scientific publication goal of 15 or more peer-reviewed papers in 2009.
In addition, Alnylam presented data from its clinical and pre-clinical programs at the following scientific meetings:
-
BIT Life Sciences' 2nd Annual World Summit of Antivirals
held on
July 18-25, 2009 inBeijing, China ; -
American Chemical Society (ACS) Fall 2009National Meeting & Exposition held onAugust 16-20, 2009 inWashington D.C. ; -
European Molecular Biology Organization (EMBO) Meeting 2009 held onAugust 29-September 1, 2009 inAmsterdam ; -
2009
World Congress on Huntington's Disease held onSeptember 12-15 , 2009 inVancouver, British Columbia ; -
Interscience Conference on Antimicrobial Agents and Chemotherapy
(ICAAC) held onSeptember 12-15, 2009 inSan Francisco ; -
Society for Neuroscience 2009 Meeting held onOctober 17-21, 2009 inChicago ; and, -
American Association for the Study of Liver Diseases (AASLD) held onOctober 30-November 3, 2009 inBoston .
Business Execution Highlights
-
Advanced RNAi Therapeutic Collaboration Phase of Landmark 2007
Alliance with
Roche . Alnylam announced today thatRoche and Alnylam will jointly collaborate on the discovery and development of certain RNAi therapeutic products and each will contribute key delivery technologies in the effort which is focused on specific disease targets. New delivery technologies include Alnylam lipid nanoparticles and Roche Madison dynamic polyconjugate delivery technologies, and also can include additional delivery technologies such as SNALP technology from Tekmira. Alnylam andRoche will co-develop and co-commercialize RNAi therapeutic products in the U.S. market and Alnylam is eligible to receive additional milestone and royalty payments for products developed in the rest of world. -
Extended Novartis Collaboration for Fifth and Final Planned Year.
Novartis elected to extend the company's RNAi therapeutics collaboration for a fifth and final planned year, throughOctober 2010 , resulting in continued R&D funding to Alnylam. - Updated Guidance on 2009 Business Development Goal. Alnylam announced today revised guidance regarding its 2009 goal of two or more new major alliances. While discussions remain ongoing regarding new business partnerships, the company now believes that the timing for completion of such new major alliances could extend beyond the 2009 calendar year.
-
Granted KOKEN License to Kreutzer-Limmer Patents for the RNAi
Research Products Market. Alnylam has granted
KOKEN Co., Ltd. a non-exclusive worldwide license to manufacture and provide RNAi research products and services under the Kreutzer-Limmer patent family.KOKEN is now Alnylam's 17th research reagent licensee.
Intellectual Property (IP) Highlights
-
Joined
GlaxoSmithKline (GSK) in Donating IP toPatent Pool for Neglected Tropical Diseases. Alnylam is donating its RNAi IP, technology, and know-how to the patent pool established by GSK earlier this year. Alnylam's patent estate, which includes more than 1500 patents and patent applications, will be provided on a royalty-free, non-profit basis to qualified third parties who want to conduct research on new treatments for neglected tropical diseases (NTDs), as defined by theFood and Drug Administration (FDA ). The patent pool was formed by GSK to aid in the discovery and development of new medicines for the treatment of NTDs in the world's least developed countries. - European Patent Office Granted Kreutzer-Limmer III Patent. The European Patent Office (EPO) issued a notification of intent to grant for a patent in the Kreutzer-Limmer III patent series (EP1349927). The new patent includes 22 claims covering methods and medicaments for use of an RNAi therapeutic in oncology applications.
-
Kreutzer-Limmer II Patent Upheld in European Opposition Proceedings.
The EPO upheld the Kreutzer-Limmer II '061 (EP1352061) patent in oral
proceedings held before the European Opposition Board. The '061 patent
is the first to be granted in
Europe from the Kreutzer-Limmer II patent family, and includes broad claims covering methods of silencing approximately 130 disease genes with small interfering RNAs (siRNAs), the molecules that mediate RNAi. -
First microRNA Patent Grant in
Japan from Tuschl III Patent Series. Alnylam, Isis, and Regulus announced that the Japanese Patent Office (JPO) has notified theMax Planck Society of its intent to grant a patent from the Tuschl III patent series (JP Application Number 2003-532675). This patent series pertains to the discovery of over 120 novel mammalian microRNAs, including miR-122, which is a leading pre-clinical program at Regulus.
-
Received Notice of Allowance for Key New Fundamental Patents
Covering microRNA Therapeutics.
- Alnylam and Regulus were notified that the USPTO has awarded a notice of allowance for the Manoharan patent (US 7,582,744), which covers antagomirs, a chemical class of anti-miRs, oligonucleotide inhibitors of microRNAs.
- Alnylam, Regulus, and Isis received notification that the USPTO allowed a patent application within the Esau patent family (Application No. 10/909,125), which covers methods of inhibiting miR-122, a liver-specific microRNA that has been shown to facilitate replication of hepatitis C virus (HCV) infection.
-
USPTO Allows Patent Under Isis'
Crooke Patent Estate . The USPTO has allowed an Isis patent application (Application No. 10/281,349) that further expands the scope of the Crooke patent estate. The newly allowed patent broadly covers methods of cleaving a target RNA via a double-stranded ribonuclease mechanism, including the RNAi mechanism, with chemically modified single-stranded RNA-containing drugs such as those under development by Isis and Alnylam in their single-stranded RNAi (ssRNAi) research collaboration. -
Additional New Patents Issued or Granted. Alnylam announced
today the issuance or grant of the following new patents owned,
controlled, or licensed by Alnylam in the RNAi therapeutics field:
- the Stoffel patent, which is non-exclusively licensed to Regulus Therapeutics, was issued by the USPTO (7,585,969) and covers isolated DNA or RNA molecules having the nucleobase sequence of miR-375, or the nucleobase sequence of a miR-375 precursor, with or without a variety of chemical modifications; and,
- a new target-related patent was allowed by the USPTO (Application No. 11/944,961).
The company has obtained greater than 35 new patent grants thus far in 2009, exceeding its goal of greater than 15 new patent grants in 2009.
Organizational Highlights
-
Selected as Technology Fast 500™ Award Winner by Deloitte.
Alnylam was ranked number 80 in the Deloitte Technology Fast 500, an
award that recognizes 500 of the fastest growing technology, media,
telecommunications, life sciences, and clean technology companies in
North America based on percentage of fiscal year revenue growth over five years. - Departure of Chief Scientific Officer at Alnylam. John A. Schmidt, Jr., M.D., former Alnylam Chief Scientific Officer, left the company to pursue other interests.
-
Regulus Therapeutics Expands Management Team. Regulus
Therapeutics appointed
Hubert C. Chen , M.D., Vice President of Translational Medicine. -
Director Not to Stand for Re-election in 2010.
Edward Scolnick , M.D., will not stand for re-election as a Director at the company's 2010 annual meeting. Dr. Scolnick will focus his activities on his scientific research at theBroad Institute .
"I am grateful for the time I have spent as a Director at Alnylam and
have a great deal of respect for the science taking place there," said
Dr.
"We are saddened by Ed's decision to not stand for re-election next
year, although we understand and greatly admire his commitment to his
scientific research studies. Certainly, we will miss his valuable
expertise and insight," said
Conference Call Information
Management will provide an update on the company, discuss third 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.
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, as well its expectations regarding
clinical trials, business execution, intellectual property matters and
legal activities, 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 risks related to: Alnylam's approach to
discover and develop novel drugs, which is unproven and may never lead
to marketable products; the pre-clinical and clinical results for its
product candidates, which may not support further development of product
candidates; obtaining, maintaining and protecting intellectual property;
Alnylam's ability to enforce its patents against infringers and to
defend its patent portfolio against challenges from third parties;
Alnylam's ability to obtain additional funding to support its business
activities; Alnylam's dependence on third parties for the development,
manufacture, marketing, sale and distribution of products; obtaining
regulatory approval for products; competition from others using
technology similar to Alnylam's and others developing products for
similar uses; Alnylam's dependence on current and future collaborators;
and Alnylam's short operating history; 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
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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