– Advanced Clinical Pipeline, Achieved Major Progress in Delivery, Demonstrated Strength of Partnerships, and Expanded Leadership in Intellectual Property –
– Ends Quarter with $419 Million in Cash and Total Marketable Securities –
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter ended March 31, 2010, and company highlights.
“The first quarter and recent period has been very productive as we continued to execute on our mission of building a top-tier biopharmaceutical company founded on RNAi,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “Just recently we presented new pre-clinical data from our transthyretin-mediated amyloidosis program suggesting that treatment with ALN-TTR01 could potentially reverse complications of this devastating disease. In addition, we presented important progress in our efforts with Alnylam Biotherapeutics, an opportunity that leverages our RNAi technologies to transform manufacturing processes for biotherapeutic drugs, including their quality, quantity, and attributes. All told, the opportunity for RNAi therapeutics as a potential new class of innovative medicines is stronger than ever, and we believe Alnylam will continue to lead the advancement of this innovative technology towards patients. We look forward to continuing our progress across all key value drivers of the business throughout the year.”
“We made notable achievements during the period, including the advancement of our clinical pipeline with the initiation of our ALN-RSV01 Phase IIb clinical trial in adult lung transplant patients. In addition, our ongoing ALN-VSP Phase I clinical trial in liver cancers is progressing, with preliminary data being presented at the ASCO Annual Meeting in June, and we are on track to initiate a Phase I clinical trial for our ALN-TTR program in the first half of this year,” said Barry Greene, President and Chief Operating Officer of Alnylam. “We also made continued progress in the delivery of RNAi therapeutics, such as demonstrating enhanced potency, showing broadened systemic delivery beyond the liver, and defining key mechanisms for in vivo efficacy. Further, we saw important progress with our collaborators during this period, including the completion of key technology transfer objectives with Takeda resulting in a $20 million payment to Alnylam, as well as continued commitment from Novartis as evidenced by their recent equity purchase. We are also pleased with the new partnership Regulus formed with GSK on microRNA therapeutics targeting miR-122 for treatment of HCV infection. Combined, we believe that these successes, amongst others, continue to position us as an industry leader in RNAi therapeutics.”
Cash, Cash Equivalents and Total Marketable Securities
At March 31, 2010, Alnylam had cash, cash equivalents and total marketable securities of $419.3 million, as compared to $435.3 million at December 31, 2009.
The net loss according to accounting principles generally accepted in the U.S. (GAAP) for the first quarter of 2010 was $12.3 million, or $0.29 per share on both a basic and diluted basis (including $5.3 million, or $0.13 per share of non-cash stock-based compensation expense), as compared to a net loss of $7.9 million, or $0.19 per share on both a basic and diluted basis (including $5.1 million, or $0.12 per share of non-cash stock-based compensation expense), for the same period in the previous year.
Revenues were $24.6 million for the first quarter of 2010, as compared to $25.1 million for the same period last year. Revenues for the first quarter of 2010 included $14.0 million of collaboration revenues related to the company’s alliance with Roche, $5.4 million of revenues from the company’s alliance with Takeda Pharmaceuticals Company Limited and $5.2 million of expense reimbursement and amortization revenues from Novartis, the National Institutes of Health (NIH), Cubist Pharmaceuticals, Inc., Biogen Idec Inc., InterfeRx™, research reagent and services licenses, and other sources.
Research and Development Expenses
Research and development (R&D) expenses were $24.7 million in the first quarter of 2010, which included $3.2 million of non-cash stock-based compensation, as compared to $25.3 million in the first quarter of 2009, which included $3.0 million of non-cash stock-based compensation. The decrease in R&D expenses in the first quarter of 2010 as compared to the prior year period was due primarily to license fees and milestone payments in the prior year period related to the Cubist alliance and the initiation of our ALN-VSP Phase I clinical study, partially offset by an increase in compensation and related expenses due to additional research and development headcount to support our alliances and expanding product pipeline.
General and Administrative Expenses
General and administrative (G&A) expenses were $11.2 million in the first quarter of 2010, which included $2.1 million of non-cash stock-based compensation, as compared to $7.7 million for the same period in 2009, which included $2.1 million of non-cash stock-based compensation. The increase in G&A expenses for the first quarter of 2010 was due primarily to higher professional service fees in association with business activities, primarily legal activities.
Equity in loss of joint venture (Regulus Therapeutics Inc.) was $1.6 million and $1.5 million for the first quarter of 2010 and 2009, respectively, related to Alnylam’s approximate 49% share of the net losses incurred by Regulus.
Interest income was $0.6 million for the first quarter of 2010, as compared to $2.0 million for the first quarter of 2009. The decrease in interest income was due primarily to significantly lower average interest rates as well as lower average cash, cash equivalents and marketable securities balances as compared to the prior year.
Income tax expense was $18,000 for the first quarter of 2010, as compared to $0.7 million for the first quarter of 2009. The decrease in income tax expense was due primarily to taxable income in 2009 as a result of our alliances with Roche and Takeda.
2010 Financial Guidance
Alnylam expects that its cash, cash equivalents and total marketable securities balance will be greater than $325 million at December 31, 2010, which excludes a potential payment from Novartis should they decide to execute their adoption license later this year.
“We continue to maintain a solid financial position, ending the first quarter with $419 million in cash and zero debt,” said Patricia Allen, Vice President, Finance and Treasurer of Alnylam. “This enables us to continue to invest in our scientific platform and our multiple RNAi therapeutic programs in development. We expect to finish this year with greater than $325 million in cash, excluding the potential adoption license payment from Novartis.”
First Quarter 2010 and Recent Significant Corporate Highlights
Product Pipeline and Scientific Leadership Highlights
Business Execution Highlights
Intellectual Property (IP) Highlights
In addition, significant progress was also made in the Regulus IP estate related to microRNA therapeutics, including:
-- a new grant from the Japanese Patent Office for a patent (JP 4371812) from the Tuschl III patent series pertaining to the discovery of mammalian microRNAs; and,
-- an allowance from the USPTO of a new patent (Application No. 11/977,506) owned by Stanford University and licensed exclusively to Regulus, covering methods of antagonizing miR-181a to regulate immune response.
Conference Call Information
Management will provide an update on the company, discuss first quarter 2010 results, and discuss expectations for the future via conference call on May 5, 2010 at 4:30 p.m. ET. To access the call, please dial 800-320-2978 (domestic) or 617-614-4923 (international) five minutes prior to the start time and provide the passcode 15254895. A replay of the call will be available beginning at 7:30 p.m. ET on May 5, 2010. To access the replay, please dial 888-286-8010 (domestic) or 617-801-6888 (international), and provide the passcode 20108825.
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. 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 joint 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.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including without limitation, the need for novel RNAi therapeutics, Alnylam’s views with respect to the potential for RNAi therapeutics, including ALN-RSV, ALN-VSP, ALN-TTR, ALN-PCS, and ALN-HTT, and its expectations with respect to the timing and success of its clinical and pre-clinical trials, including its recently initiated Phase IIb trial for ALN-RSV01 and its plan to initiate clinical trials for ALN-TTR01 and ALN-PCS, the timing of regulatory filings, its expectations regarding the continued development and recent advances relating to the efficient delivery of RNAi therapeutics, the formation of new alliances, the establishment of its internal Alnylam Biotherapeutics effort and the potential of this effort to generate new business opportunities, its cash position at the end of 2010, its ongoing legal activities, and its ability to continue to generate revenue through existing and new alliances, 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, including through the establishment of new alliances; Alnylam’s dependence on third parties for development, manufacture, marketing, sales and distribution of products; obtaining regulatory approval for the clinical development and commercialization of 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 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.
Alnylam Pharmaceuticals, Inc.
Unaudited Condensed Consolidated Statements of Operations
(In thousands, except per share amounts)
|Net revenues from research collaborators||$24,564||$25,057|
|Research and development (1)||24,700||25,321|
|General and administrative (1)||11,170||7,716|
|Total operating expenses||35,870||33,037|
|Loss from operations||(11,306)||(7,980)|
|Other income (expense):|
|Equity in loss of joint venture (Regulus Therapeutics Inc.)||(1,578)||(1,470)|
|Other (expense) income||(11)||178|
|Total other income (expense)||(999)||756|
|Loss before income taxes||(12,305)||(7,224)|
|Provision for income taxes||(18)||(665)|
|Net loss per common share - basic and diluted||$(0.29)||$(0.19)|
|Weighted average common shares used to compute basic and diluted net loss per common share||41,870||41,399|
|(1) Non-cash stock-based compensation expenses included in operating expenses are as follows:|
|Research and development||$3,229||$3,034|
|General and administrative||2,098||2,103|
|Alnylam Pharmaceuticals, Inc.|
|Unaudited Condensed Consolidated Balance Sheets|
|(In thousands, except share amounts)|
|March 31,||December 31,|
|Cash, cash equivalents and total marketable securities||$419,267||$435,316|
|Prepaid expenses and other current assets||5,109||4,151|
|Property and equipment, net||18,304||18,324|
|Intangible assets, net||578||622|
|Total deferred tax assets||10,611||10,493|
|Investment in joint venture (Regulus Therapeutics Inc.)||5,032||6,435|
|Accounts payable and accrued expenses||$15,742||$22,322|
|Income taxes payable||98||5,644|
|Total deferred revenue||272,141||271,813|
|Total deferred rent||3,414||3,447|
|Other long-term liabilities||181||194|
Total stockholders' equity (41.9 million and 41.8 million common
|Total liabilities and stockholders' equity||$463,406||$481,385|
This selected financial information should be read in conjunction with the consolidated financial statements and notes thereto included in Alnylam’s Annual Report on Form 10-K which includes the audited financial statements for the year ended December 31, 2009.
Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Senior Director, Investor Relations and
Patricia Allen, 617-551-8362
Vice President, Finance and Treasurer
KEYWORDS: United States North America Massachusetts
INDUSTRY KEYWORDS: Health Biotechnology Pharmaceutical General Health