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Alnylam Pharmaceuticals Reports First Quarter 2016 Financial Results and Highlights Recent Period ProgressAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter 2016, and highlighted recent progress in advancing its pipeline. "At Alnylam, we continue to advance a broad pipeline of investigational RNAi therapeutics - including 10 programs in clinical development and 2 programs in Phase 3 - across a broad range of disease indications. A major milestone in the first quarter was completion of enrollment in our APOLLO Phase 3 trial for patisiran, and we're on track to view results in mid-2017. If positive, we expect to file our first regulatory applications for approval later that same year. We're also making strong progress in our ENDEAVOUR Phase 3 trial of revusiran, where we now expect completion of enrollment in late 2016 and data readout in mid-2018. In our fitusiran program in hemophilia, we look forward to presenting important new results in July, including initial results in patients with inhibitors, and are on track to start our two Phase 3 studies shortly thereafter," said John Maraganore, Ph.D., Chief Executive Officer at Alnylam. "We're also making progress with our earlier stage clinical programs. In our ALN-CC5 program, initial results in PNH patients, which will be presented at EHA next month, point to an optimal development path forward in PNH for eculizumab poor responders and for eculizumab sparing, with parallel efforts in other complement-mediated diseases. Finally, we also filed Clinical Trial Applications for ALN-HBV and ALN-TTRsc02, and initiated our Phase 1 study for our ALN-GO1 program in primary hyperoxaluria. We look forward to sharing our continued progress throughout the course of a very data rich 2016." First Quarter 2016 and Recent Significant Corporate Highlights
Upcoming Events in Mid- and Late 2016
Financials "Alnylam continues to maintain a strong balance sheet, ending the first quarter of 2016 with approximately $1.2 billion in cash," said Michael Mason, Vice President, Finance and Treasurer. "Our financial strength allows us to continue to invest in a broad pipeline of investigational RNAi therapeutics across our three STArs, aligned with achievement of our 'Alnylam 2020' goals. As for financial guidance this year, we are updating cash guidance today to end 2016 with greater than $1.0 billion in cash, including $150.0 million of restricted marketable securities that we received from new credit agreements - related to the build out of our new manufacturing facility - entered into in April 2016." Cash, Cash Equivalents and Total Marketable Securities At March 31, 2016, Alnylam had cash, cash equivalents and total marketable securities of $1.21 billion, as compared to $1.28 billion at December 31, 2015. In April 2016, the company entered into credit agreements described below with proceeds of $150.0 million of restricted marketable securities. Credit Agreements In April 2016, Alnylam entered into credit agreements, related to the build out of the Company's new manufacturing facility, that provide for a $150.0 million term loan facility, and mature in April 2021. Interest on the borrowings will be calculated based on LIBOR plus 0.45 percent. The obligations under the credit agreements are secured by cash collateral in an amount equal to, at any given time, at least 100 percent of the principal amount of all term loans outstanding under the credit agreements at such time. GAAP Net Loss The net loss according to accounting principles generally accepted in the U.S. (GAAP) for the first quarter of 2016 was $103.0 million, or $1.21 per share on both a basic and diluted basis (including $23.5 million, or $0.28 per share of non-cash stock-based compensation expense), as compared to a net loss of $50.8 million, or $0.62 per share on both a basic and diluted basis (including $8.2 million, or $0.10 per share of non-cash stock-based compensation expense), for the same period in the previous year. Revenues Revenues were $7.3 million for the first quarter of 2016, as compared to $18.5 million for the same period last year. Revenues for the first quarter of 2016 included $4.4 million from the company's alliance with Sanofi Genzyme, $2.7 million from the company's alliance with The Medicines Company and $0.2 million from other sources. The decrease in revenues in the quarter ended March 31, 2016 as compared to the prior year period was due primarily to the completion of the company's performance obligations under the Monsanto agreement in February 2015 and the completion of its revenue amortization under the Takeda agreement in May 2015, partially offset by higher revenue from its agreement with Sanofi Genzyme. The company expects net revenues from collaborators to increase during the remainder of 2016 due to expected increases in expense reimbursement and an expected milestone payment under its agreement with Sanofi Genzyme. Research and Development Expenses Research and development (R&D) expenses were $96.3 million in the first quarter of 2016 which included $14.4 million of non-cash stock-based compensation, as compared to $58.0 million in the first quarter of 2015, which included $5.3 million of non-cash stock-based compensation. The increase in R&D expenses for the quarter ended March 31, 2016 as compared to the prior year period was due primarily to higher clinical trial and manufacturing and external services expenses resulting from the significant advancement of the company's Genetic Medicine pipeline. In addition, compensation and related expenses and non-cash stock-based compensation expenses increased during the quarter ended March 31, 2016 as compared to the quarter ended March 31, 2015 due primarily to a significant increase in headcount during the period as the company expands and advances its development pipeline, as well as the vesting of certain performance-based stock option awards during the quarter ended March 31, 2016. The company expects that on a quarterly basis in 2016 R&D expenses will increase from the first quarter as it continues to develop its pipeline and advance its product candidates into clinical trials. General and Administrative Expenses General and administrative (G&A) expenses were $21.1 million in the first quarter of 2016, which included $9.1 million of non-cash stock-based compensation, as compared to $12.7 million in the first quarter of 2015, which included $2.9 million of non-cash stock-based compensation. G&A expenses for the quarter ended March 31, 2016 as compared to the prior year period increased due primarily to an increase in non-cash stock-based compensation expense due to an increase in headcount, as well as the vesting of certain performance-based stock option awards during the quarter ended March 31, 2016. The company expects that on a quarterly basis in 2016 G&A expenses will remain relatively consistent with the first quarter of 2016. Conference Call Information Management will provide an update on the company, discuss first quarter 2016 results, and discuss expectations for the future via conference call on Monday, May 2, 2016 at 4:30 p.m. ET. To access the call, please dial 877-312-7507 (domestic) or 631-813-4828 (international) five minutes prior to the start time and refer to conference ID 99665704. A replay of the call will be available beginning at 7:30 p.m. ET on May 2, 2016. To access the replay, please dial 855-859-2056 (domestic) or 404-537-3406 (international), and refer to conference 99665704 Sanofi Genzyme Alliance In January 2014, Alnylam and Sanofi Genzyme, the specialty care global business unit of Sanofi, formed an alliance to accelerate and expand the development and commercialization of RNAi therapeutics across the world. The alliance is structured as a multi-product geographic alliance in the field of rare diseases. Alnylam retains product rights in North America and Western Europe, while Sanofi Genzyme obtained the right to access certain programs in Alnylam's current and future Genetic Medicines pipeline in the rest of the world (ROW) through the end of 2019, together with certain broader co-development/co-commercialization rights and global rights for certain products. In the case of patisiran, Alnylam will advance the product in North America and Western Europe, while Sanofi Genzyme will advance the product in the ROW. In the case of revusiran, Alnylam and Sanofi Genzyme will co-develop/co-commercialize the product in North America and Western Europe, while Sanofi Genzyme will advance the product in the ROW. In the case of fitusiran, Sanofi Genzyme has elected to opt into the program for its ROW rights, while retaining its further opt-in right to co-develop and co-promote fitusiran with Alnylam in North America and Western Europe, subject to certain restrictions. About 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 (News - Alert) 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 RNA (siRNA), 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 LNP Technology Alnylam has licenses to Arbutus LNP intellectual property for use in RNAi therapeutic products using LNP technology. 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. Alnylam's pipeline of investigational RNAi therapeutics is focused in 3 Strategic Therapeutic Areas (STArs): Genetic Medicines, with a broad pipeline of RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics toward genetically validated, liver-expressed disease targets for unmet needs in cardiovascular and metabolic diseases; and Hepatic Infectious Disease, with a pipeline of RNAi therapeutics that address the major global health challenges of hepatic infectious diseases. In early 2015, Alnylam launched its "Alnylam 2020" guidance for the advancement and commercialization of RNAi therapeutics as a whole new class of innovative medicines. Specifically, by the end of 2020, Alnylam expects to achieve a company profile with 3 marketed products, 10 RNAi therapeutic clinical programs - including 4 in late stages of development - across its 3 STArs. The company's demonstrated commitment to RNAi therapeutics has enabled it to form major alliances with leading companies including Ionis, Novartis, Roche, Takeda, Merck, Monsanto, The Medicines Company, and Sanofi Genzyme. In addition, Alnylam holds an equity position in Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 200 peer-reviewed papers, including many in the world's top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, Cell, New England Journal of Medicine, and The Lancet. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information about Alnylam's pipeline of investigational RNAi therapeutics, 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 views with respect to the potential for RNAi therapeutics, including patisiran, revusiran, fitusiran, ALN-CC5, ALN-AS1, ALN-AAT, ALN-GO1, ALN-PCSsc, ALN-HBV, ALN-TTRsc02, and ALN-F12, its expectations regarding its STAr pipeline growth strategy, its "Alnylam 2020" guidance for the advancement and commercialization of RNAi therapeutics, its expectations for the timing of filing of regulatory documents, including but not limited to submission of an MAA and NDA for patisiran, its expectations regarding the timing of the start of clinical studies and presentation of clinical data, including for studies with patisiran, revusiran, fitusiran, ALN-CC5, ALN-AS1, ALN-AAT, ALN-GO1, ALN-PCSsc, ALN-HBV, and ALN-TTRsc02, its expected cash position as of December 31, 2016, and its plans regarding the pursuit of pre-clinical programs and commercialization of RNAi therapeutics, 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 and delivery approaches, successfully demonstrate the efficacy and safety of its drug candidates, the pre-clinical and clinical results for its product candidates, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all, actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, Alnylam's ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties, obtaining regulatory approval for products, competition from others using technology similar to Alnylam's and others developing products for similar uses, Alnylam's ability to manage operating expenses, Alnylam's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Alnylam's dependence on third parties for development, manufacture, marketing, sales and distribution of products, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC (News - Alert)) and in other filings that Alnylam makes with the SEC. 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 explicitly disclaims any obligation to update any forward-looking statements.
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, 2015. View source version on businesswire.com: http://www.businesswire.com/news/home/20160502005867/en/ |