SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): November 7, 2018
Alnylam Pharmaceuticals, Inc.
(Exact Name of Registrant as Specified in Charter)
(State or Other Juris-
300 Third Street, Cambridge, MA
|(Address of Principal Executive Offices)||(Zip Code)|
Registrant’s telephone number, including area code: (617) 551-8200
|(Former Name or Former Address, if Changed Since Last Report)|
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
⃞ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
⃞ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
⃞ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
⃞ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934(§240.12b-2 of this chapter).
Emerging growth company ⃞
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ⃞
Item 2.02. Results of Operations and Financial Condition
On November 7, 2018, Alnylam Pharmaceuticals, Inc. (the “Company”) announced its financial results for the quarter ended September 30, 2018. The full text of the press release issued in connection with the announcement is furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The information in Item 2.02 of this Form 8-K (including Exhibit 99.1) shall not be deemed "filed" for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.
Item 9.01. Financial Statements and Exhibits
The following exhibit relating to Item 2.02 shall be deemed to be furnished, and not filed:
Pursuant to the requirements of the Securities Exchange Act of 1934, the
Registrant has duly caused this report to be signed on its behalf by the
undersigned hereunto duly authorized.
Date: November 7, 2018
ALNYLAM PHARMACEUTICALS, INC.
/s/ Manmeet S. Soni
Manmeet S. Soni
Senior Vice President, Chief Financial Officer
Alnylam Pharmaceuticals Reports Third Quarter 2018 Financial Results and Highlights Recent Period Activity
− Obtained FDA and EMA Approvals of ONPATTRO™ (patisiran) – the First-Ever RNAi Therapeutic – and Launched in U.S. and EU −
− Received 125 U.S. Patient Start Forms in First Seven Weeks of ONPATTRO Launch −
− Reported Positive Topline Interim Analysis Results from ENVISION Phase 3 Study of Givosiran in Patients with Acute Hepatic Porphyrias −
− Advanced Lumasiran into ILLUMINATE Phase 3 Program −
− Maintained Strong Balance Sheet with $1.27 Billion in Cash and Expects to End 2018 with Approximately $1.0 Billion in Cash −
CAMBRIDGE, Mass.--(BUSINESS WIRE)--November 7, 2018--Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter 2018 and reviewed recent commercial and R&D highlights.
“The third quarter and recent period were truly revolutionary for Alnylam with the approval of ONPATTRO in both the U.S. and EU, heralding the arrival of RNAi therapeutics as a whole new class of medicines. With these approvals and the subsequent launches, we have begun to realize the promise of RNAi therapeutics on a global scale,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “Our recent regulatory and commercial launch experiences will be leveraged for our entire portfolio including late-stage programs such as givosiran, where we will initiate a rolling NDA submission this year; lumasiran, which we have advanced into late-stage development with the recent initiation of the ILLUMINATE-A Phase 3 study; and ALN-TTRsc02, which will enter Phase 3 later this year in the HELIOS-A study. We believe these accomplishments put us well on our way toward achieving our Alnylam 2020 goal of building a multi-product, global, commercial-stage company with a deep and sustainable clinical pipeline by the end of 2020.”
“With the approval and launch of ONPATTRO, Alnylam is now a global commercial-stage company. With only seven weeks of results for the third quarter, we’re encouraged by the number of U.S. patient start forms, and emerging prescriber base, highlighting what we believe is strong demand for ONPATTRO for adults with polyneuropathy caused by hATTR amyloidosis. Moreover, we believe our regional presence in North America, Europe, Asia, and, soon, Latin America, along with established medical affairs and supply chain capabilities, positions us to expand our efforts in markets around the world,” said Barry Greene, President of Alnylam. “We look forward to continuing our work toward strong commercial execution, with a focus on raising disease awareness, improving diagnosis, and bringing ONPATTRO to patients in need.”
Third Quarter 2018 and Recent Period Significant Corporate Highlights
Additional Business Updates
In late 2018, Alnylam intends to:
Financial results for the quarter ended September 30, 2018
“We are pleased to have recognized initial revenue for an Alnylam product for the first time in the Company’s history, following the August FDA approval of ONPATTRO,” said Manmeet Soni, Chief Financial Officer of Alnylam. “With cash and investments on our balance sheet standing at $1.27 billion at September 30, and expectations to finish the year with approximately $1.0 billion, we believe Alnylam is in a strong position to continue executing on global commercial operations while advancing our pipeline programs through the clinic.”
Cash and Investments
At September 30, 2018, Alnylam had cash, cash equivalents and marketable debt securities, and restricted investments, excluding equity securities, of $1.27 billion, as compared to $1.73 billion at December 31, 2017.
GAAP and Non-GAAP Net Loss
The net loss according to accounting principles generally accepted in the U.S. (GAAP) for the third quarter of 2018 was $245.3 million, or $2.43 per share on both a basic and diluted basis, as compared to a net loss of $122.9 million, or $1.34 per share on both a basic and diluted basis, for the same period in the previous year.
The non-GAAP net loss for the third quarter of 2018 was $157.3 million, or $1.56 per share on both a basic and diluted basis, as compared to a non-GAAP net loss of $97.0 million, or $1.06 per share on both a basic and diluted basis, for the same period in the previous year.
The non-GAAP net loss for the third quarter of 2018 and 2017 excludes stock-based compensation expense. See “Use of Non-GAAP Financial Measures” below for a description of non-GAAP financial measures and a reconciliation between GAAP and non-GAAP net loss appearing later in this press release.
ONPATTRO Revenues, Net
Net product revenues from sales of ONPATTRO were $0.5 million in the third quarter of 2018 following its approval by the FDA in August 2018.
Net Revenues from Collaborators
Net revenues from collaborators were $1.6 million in the third quarter of 2018 as compared to $17.1 million in the third quarter of 2017.
GAAP and Non-GAAP Research and Development Expenses
GAAP research and development (R&D) expenses were $139.9 million in the third quarter of 2018 as compared to $95.3 million in the third quarter of 2017.
Non-GAAP R&D expenses were $94.2 million in the third quarter of 2018 as compared to $80.2 million in the third quarter of 2017. Non-GAAP R&D expenses exclude stock-based compensation expense. A reconciliation between GAAP and non-GAAP R&D expenses appears later in this press release.
GAAP and Non-GAAP Selling, General and Administrative Expenses
GAAP selling, general and administrative (SG&A) expenses were $116.5 million in the third quarter of 2018 as compared to $47.6 million in the third quarter of 2017.
Non-GAAP SG&A expenses were $74.4 million in the third quarter of 2018 as compared to $36.8 million in the third quarter of 2017. Non-GAAP SG&A expenses exclude stock-based compensation expense. A reconciliation between GAAP and non-GAAP SG&A expenses appears later in this press release.
2018 Financial Guidance
Alnylam reiterates its expectations to end 2018 with approximately $1.0 billion of cash, cash equivalents and marketable debt securities, restricted cash and restricted investments, excluding equity securities.
The Company reiterates its expectations for 2018 annual non-GAAP R&D expenses to be in the range of $420 million to $460 million and non-GAAP SG&A expenses to be in the range of $280 million to $320 million. Both non-GAAP R&D and non-GAAP SG&A expenses exclude stock-based compensation expenses.
Use of Non-GAAP Financial Measures
This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses and non-recurring gains outside the ordinary course of the Company’s business. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies.
The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in the press release are stock-based compensation expense and the gain on litigation settlement. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards. The Company has excluded the impact of the gain on litigation settlement because the Company believes this item is a one-time event occurring outside the ordinary course of the Company’s business.
The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between GAAP and non-GAAP measures is provided later in this press release.
Conference Call Information
Management will provide an update on the Company and discuss third quarter 2018 and recent period results as well as expectations for the future via conference call on Wednesday, November 7, 2018 at 8:30 am ET. To access the call, please dial 800-667-5617 (domestic) or 334-323-0509 (international) five minutes prior to the start time and refer to conference ID 7650424. A replay of the call will be available beginning at 11:30 am ET on the day of the call. To access the replay, please dial 888-203-1112 (domestic) or 719-457-0820 (international) and refer to conference ID 7650424.
Alnylam – Sanofi Genzyme Alliance
Alnylam and Sanofi Genzyme, the specialty care global business unit of Sanofi, established an alliance to accelerate the advancement of RNAi therapeutics as a potential new class of medicines for patients around the world with rare genetic diseases. The alliance enables Sanofi Genzyme to expand its rare disease pipeline with Alnylam’s novel RNAi technology and provides access to Alnylam’s R&D engine, while Alnylam benefits from Sanofi Genzyme’s proven global capabilities to advance late-stage development and, upon commercialization, accelerate market access for these promising genetic medicines products.
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
About LNP Technology
Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology.
About ONPATTRO™ (patisiran)
Patisiran, based on Nobel Prize-winning science, is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. Patisiran blocks the production of transthyretin in the liver, reducing its accumulation in the body’s tissues in order to halt or slow down the progression of the disease. In August 2018, patisiran received U.S. Food and Drug Administration (FDA) approval for the treatment of the polyneuropathy of hATTR amyloidosis in adults, as well as European Medicines Agency marketing authorization for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy.
Important Safety Information
Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. In a controlled clinical study, 19 percent of ONPATTRO-treated patients experienced IRRs, compared to 9 percent of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.
To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, paracetamol, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.
Reduced Serum Vitamin A Levels and Recommended Supplementation
ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.
Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).
The most common adverse reactions that occurred in patients treated with ONPATTRO were respiratory-tract infection (29 percent) and infusion-related reactions (19 percent).
About Alnylam Pharmaceuticals
Alnylam (Nasdaq:ALNY) is leading the translation of RNA interference (RNAi) into a new class of innovative medicines with the potential to improve the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS) diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust discovery platform. ONPATTRO™ (patisiran) lipid complex injection, available in the U.S. for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults, is Alnylam’s first U.S. FDA-approved RNAi therapeutic. In the EU, ONPATTRO is approved for the treatment of hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy. Alnylam has a deep pipeline of investigational medicines, including three product candidates that are in late-stage development. Looking forward, Alnylam will continue to execute on its “Alnylam 2020” strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam employs over 1,000 people worldwide and is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.
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 givosiran, ALN-TTRsc02, lumasiran, inclisiran, ALN-AAT02 and cemdisiran, its plans for additional regulatory filings and product launches for ONPATTRO, including in Japan and Latin America, its expectations regarding the timing for initiation of a rolling NDA submission with the FDA for givosiran and the reporting of complete topline results, its expectations regarding the potential timing of ILLUMINATE-A Phase 3 study results for lumasiran in late 2019 and a possible filing of an NDA in early 2020 if such results are positive, its plans to initiate the ILLUMINATE-B Phase 3 study of lumasiran, its plans to initiate a Phase 3 study for ALN-TTRsc02 in 2018 in hATTR amyloidosis and its plans to initiate additional Phase 3 studies of ALN-TTRsc02, including in hereditary and wild-type ATTR amyloidosis cardiomyopathy, in 2019, its plan to initiate the Phase 1/2 study for ALN-AAT02 in alpha-1 liver disease and to file a CTA application for ALN-HBV02 in partnership with Vir Biotechnology, its plans to select its first CNS-targeted DC program, its expected cash, cash equivalents and marketable debt securities, restricted cash and restricted investments balance, excluding equity securities, as of December 31, 2018, its expected range of 2018 annual non-GAAP R&D expenses and non-GAAP SG&A expenses, and expectations regarding its "Alnylam 2020" guidance for the advancement 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 and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other 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 product 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 or advice of regulatory agencies, which may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional pre-clinical and/or clinical testing, delays, interruptions or failures in the manufacture and supply of its product candidates, obtaining, maintaining and protecting intellectual property, Alnylam's ability to enforce its intellectual property rights against third parties and defend its patent portfolio against challenges from third parties, obtaining and maintaining regulatory approval, pricing and reimbursement for products, progress in establishing a commercial and ex-United States infrastructure, successfully launching, marketing and selling its approved products globally, Alnylam’s ability to successfully expand the indication for ONPATTRO in the future, competition from others using technology similar to Alnylam's and others developing products for similar uses, Alnylam's ability to manage its growth and operating expenses, 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 and distribution of products, the outcome of litigation, the risk of government investigations, and unexpected expenditures, as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) 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, except to the extent required by law, to update any forward-looking statements.
With the exception of ONPATTRO (patisiran), none of Alnylam’s investigational therapeutics have been approved by the U.S. Food and Drug Administration, European Medicines Agency, or any other regulatory authority and no conclusions can or should be drawn regarding the safety or effectiveness of such investigational therapeutics.
ALNYLAM PHARMACEUTICALS, INC.
UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
(In thousands, except per share amounts)
Three Months Ended
Nine Months Ended
|Product revenues, net||$||460||$||—||$||460||$||—|
|Net revenues from collaborators||1,609||17,096||53,415||51,988|
|Cost and expenses:|
|Cost of goods sold||137||—||137||—|
|Research and development||139,945||95,252||374,384||272,863|
|Selling, general and administrative||116,545||47,644||273,671||131,910|
|Total costs and expenses||256,627||142,896||648,192||404,773|
|Loss from operations||(254,558)||(125,800)||(594,317)||(352,785)|
|Other income (expense):|
|Other income (expense)||2,925||(433)||5,468||(3,863)|
|Gain on litigation settlement||—||—||20,564||—|
|Total other income||9,721||2,863||44,723||4,138|
|Loss before income taxes||(244,837)||(122,937)||(549,594)||(348,647)|
|Provision for income taxes||(445)||—||(462)||—|
|Net loss per common share - basic and diluted||$||(2.43)||$||(1.34)||$||(5.48)||$||(3.93)|
|Weighted-average common shares used to compute basic and diluted net loss per common share||100,783||91,828||100,430||88,672|
|Unrealized gain (loss) on marketable securities, net of tax||415||218||1,041||(2,194)|
|Reclassification adjustment for realized loss on marketable securities included in net loss||—||—||—||1,894|
ALNYLAM PHARMACEUTICALS, INC.
RECONCILIATION OF SELECTED GAAP MEASURES TO NON-GAAP MEASURES
(In thousands, except per share amounts)
Three Months Ended
Nine Months Ended
|Reconciliation of GAAP to Non-GAAP Research and development:|
|GAAP Research and development||$||139,945||$||95,252||$||374,384||$||272,863|
|Less: Stock-based compensation expenses||(45,784)||(15,090)||(67,537)||(37,035)|
|Non-GAAP Research and development||$||94,161||$||80,162||$||306,847||$||235,828|
|Reconciliation of GAAP to Non-GAAP Selling, general and administrative:|
|GAAP Selling, general and administrative||$||116,545||$||47,644||$||273,671||$||131,910|
|Less: Stock-based compensation expenses||(42,170)||(10,865)||(62,242)||(28,667)|
|Non-GAAP Selling, general and administrative||$||74,375||$||36,779||$||211,429||$||103,243|
|Reconciliation of GAAP to Non-GAAP Operating costs and expenses:|
|GAAP Operating costs and expenses||$||256,627||$||142,896||$||648,192||$||404,773|
|Less: Stock-based compensation expenses||(87,954)||(25,955)||(129,779)||(65,702 )|
|Non-GAAP Operating costs and expenses||$||168,673||$||116,941||$||518,413||$||339,071|
|Reconciliation of GAAP to Non-GAAP Net loss:|
|GAAP Net loss||$||(245,282)||$||(122,937)||$||(550,056)||$||(348,647 )|
|Add: Stock-based compensation expenses||87,954||25,955||129,779||65,702|
|Less: Gain on litigation settlement||—||—||(20,564)||—|
|Non-GAAP Net loss||$||(157,328)||$||(96,982)||$||(440,841)||$||(282,945 )|
|Reconciliation of GAAP to Non-GAAP Net loss per common share-basic and diluted:|
|GAAP Net loss per common share - basic and diluted||$||(2.43)||$||(1.34)||$||(5.48)||$||(3.93)|
|Add: Stock-based compensation expenses||0.87||0.28||1.29||0.74|
|Less: Gain on litigation settlement||—||—||(0.20)||—|
|Non-GAAP Net loss per common share - basic and diluted||$||(1.56)||$||(1.06)||$||(4.39)||$||(3.19)|
ALNYLAM PHARMACEUTICALS, INC.
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except share amounts)
|September 30,||December 31,|
|Cash, cash equivalents and marketable debt securities||$||1,221,830||$||1,704,537|
|Accounts receivable, net||3,362||34,002|
|Prepaid expenses and other assets||121,904||44,291|
|Property, plant and equipment, net||272,652||181,900|
|Accounts payable, accrued expenses and other liabilities||$||119,671||$||104,905|
|Total deferred revenue||5,067||84,780|
|Total deferred rent||42,797||8,614|
|Total stockholders’ equity (101.0 million and 99.7 million common shares issued and outstanding at September 30, 2018 and December 31, 2017, respectively)||1,478,119||1,766,431|
|Total liabilities and stockholders' equity||$||1,675,654||$||1,994,730|
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, 2017.
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom
(Investors and Media)