Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2017 Financial Results and Highlights Recent Period Activity
– Reported Final Results from APOLLO Phase 3 Study and Completed
Filings of New Drug Application (NDA) and Marketing Authorisation
Application (MAA) for Patisiran, with an Expected Commercial Launch in
– Advanced Three Additional RNAi Therapeutics in Phase 3 Development, Including Givosiran in ENVISION, Inclisiran in ORION-9, -10, and -11, and Restart of Fitusiran in ATLAS –
– Maintained Strong Balance Sheet with $1.7 Billion in Cash and Expects to End 2018 with Approximately $1.0 Billion in Cash –
“2017 was a defining inflection point in Alnylam’s history, with
positive results from the APOLLO Phase 3 study culminating in regulatory
filings for patisiran, a first-ever milestone for RNAi, with the
potential to bring a whole new class of innovative therapeutics to the
forefront of medicine. With newly acquired rest-of-world rights, we
anticipate making patisiran available to hATTR amyloidosis patients
around the world starting with the U.S. in mid-2018, followed by
European countries in late 2018, and then
Fourth Quarter 2017 and Recent Significant Corporate Highlights
Advanced patisiran, an investigational RNAi therapeutic for the
treatment of patients with hereditary ATTR amyloidosis.
- Presented positive, final results from the APOLLO Phase 3 pivotal study.
Completed the rolling submission of a New Drug Application (NDA)
U.S. Food and Drug Administration( FDA) and submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency(EMA), with the agencies recently accepting both applications.
Received Breakthrough Therapy Designation and Priority Review
status from the
FDA, and expansion of patisiran’s Orphan Drug Designation to the “treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis).” In addition, patisiran received Accelerated Assessment status from the EMA, and has been designated as a Promising Innovative Medicine (PIM) by the Medicines and Healthcare Products Regulatory Agency(MHRA) in the U.K.
- In response to the urgent need for treatment of patients living with hATTR amyloidosis, the Company is fulfilling requests from treating physicians for early access or compassionate use of patisiran; the Company announces today that, to date, more than 100 eligible patients have begun treatment with patisiran under these programs in the U.S. and EU.
Advanced ALN-TTRsc02, a once-quarterly, subcutaneously administered
investigational RNAi therapeutic in development for the treatment of
- Reaffirmed guidance to initiate a comprehensive Phase 3 program for ALN-TTRsc02 in late 2018.
Advanced givosiran, an investigational RNAi therapeutic in development
for the treatment of acute hepatic porphyrias (AHPs), with initiation
of the ENVISION Phase 3 study.
The Company reached alignment with global regulatory authorities
on the design of the ENVISION study, including with the
FDAon an interim analysis based on reduction of urinary aminolevulinic acid (ALA), a biomarker that the FDAconsiders reasonably likely to predict clinical benefit.
- The Company announces today that the interim analysis is also designed to conduct a blinded assessment of the porphyria attack rate for the purpose of a study sample size adjustment from approximately 75 patients to up to approximately 94 patients.
The Company has guided that it expects interim analysis results in
mid-2018 and, pending
FDAreview of the program at the time of interim analysis and assuming positive results, it expects to submit an NDA at or around year-end 2018.
- The Company reached alignment with global regulatory authorities on the design of the ENVISION study, including with the
Advanced fitusiran, an investigational RNAi therapeutic in development
for the treatment of hemophilia A and B with or without inhibitors,
and reached alignment with the
FDAon safety measures and a risk mitigation strategy resulting in a lift of the temporary hold on clinical studies.
Alnylam’s partner, The
Medicines Company, initiated the ORION-9, -10, and -11 Phase 3 clinical studies for inclisiran in patients with heterozygous familial hypercholesterolemia (HeFH), atherosclerotic cardiovascular disease (ASCVD) or ASCVD-risk equivalents, and completed enrollment in the approximately 1,500 patient ORION-11 study ahead of schedule.
Advanced lumasiran (formerly known as ALN-GO1), an investigational
RNAi therapeutic in development for the treatment of primary
hyperoxaluria type 1 (PH1), with new
positive data from the Phase 1/2 study presented at the
American Society of Nephrology Kidney Week2017 Annual Meeting.
Announced a strategic restructuring of the Company’s rare disease
Sanofi, originally formed in 2014, with Alnylam obtaining global rights to its ATTR amyloidosis programs – patisiran and ALN-TTRsc02 – and Sanofiobtaining global rights to fitusiran.
- Joined a research consortium with the UK Biobank, Regeneron, and four major pharmaceutical companies aimed at generating 500,000 human exome sequences linked to medical records by the end of 2019.
- Announced a licensing agreement with Vir Biotechnology for the development and commercialization of RNAi therapeutics for infectious diseases, including HBV.
Upcoming Events in Early 2018
Alnylam announces today that it will present additional data from the
APOLLO Phase 3 study of patisiran at the International Symposium on
Amyloidosis (ISA), being held
March 26-29, 2018in Kumamoto, Japan.
The Company also announces that it will present additional data from
the Phase 1 and Phase 2 OLE studies of givosiran at the
European Association for the Study of the Liver(EASL) 53rd Annual International Liver Congress, being held April 11-15, 2018in Paris, France, in an oral presentation on Saturday, April 14at 10:00 amCentral European Time ( 4:00 am ET).
Medicines Companyhas guided its intention to complete enrollment in the ORION-9 and -10 pivotal studies of inclisiran in early 2018.
Sanofiexpect to enroll patients in the ATLAS Phase 3 program of fitusiran in patients with hemophilia A or B with and without inhibitors throughout the year, with resumption of dosing expected in early 2018.
Financial results for the quarter and year ended
“Alnylam’s robust balance sheet and overall financial position allow for
the staged build of commercial capabilities and the execution of
anticipated product launches in the U.S. and
Cash and Investments
GAAP and Non-GAAP Net Loss
The net loss according to accounting principles generally accepted in the U.S. (GAAP) for the fourth quarter of 2017 was
The non-GAAP net loss for the fourth quarter of 2017 was
The non-GAAP net loss 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.
GAAP research and development (R&D) expenses were
Non-GAAP R&D expenses were
GAAP and Non-GAAP General and Administrative Expenses
GAAP general and administrative (G&A) expenses were
Non-GAAP G&A expenses were
2018 Financial Guidance
Alnylam expects that its cash, cash equivalents and fixed income
marketable securities, restricted cash and restricted investments
balance will be approximately
The Company expects its 2018 annual Non-GAAP R&D expenses to be in the
Use of Non-GAAP Financial Measures
This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses. 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 item included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in the press release is stock-based compensation expense. 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 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. 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 fourth quarter and year end 2017 results as well as expectations for the future via conference call on
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 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, 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.
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, and hepatic infectious 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 and deep pipeline of investigational medicines, including four 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 700 people in the U.S. and
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, givosiran, fitusiran, inclisiran, ALN-TTRsc02, lumasiran and cemdisiran, its expectations regarding the review, potential regulatory approval and commercial launch of patisiran in
None of Alnylam’s investigational therapeutics have been approved by the
ALNYLAM PHARMACEUTICALS, INC.
Three Months Ended
|Net revenues from collaborators||$||37,924||$||17,454||$||89,912||$||47,159|
|Research and development||117,772||105,011||390,635||382,392|
|General and administrative||67,455||27,876||199,365||89,354|
|Total operating expenses||185,227||132,887||590,000||471,746|
|Loss from operations||(147,303)||(115,433)||(500,088)||(424,587)|
|Other income (expense):|
|Other income (expense)||841||300||(3,022)||6,171|
|Total other income||5,076||2,499||9,214||14,479|
|Net loss per common share - basic and diluted||$||(1.48)||$||(1.32)||$||(5.42)||$||(4.79)|
|Weighted-average common shares used to compute basic and diluted net loss per common share||96,139||85,843||90,554||85,596|
|Unrealized loss on marketable securities, net of tax||(692)||(5,502)||(2,886)||(30,833)|
|Reclassification adjustment for realized (gain) loss on marketable securities included in net loss||—||(161)||1,894||(6,977)|
ALNYLAM PHARMACEUTICALS, INC.
Three Months Ended
|Reconciliation of GAAP to Non-GAAP Research and development:|
|GAAP Research and development||$||117,772||$||105,011||$||390,635||$||382,392|
|Less: Stock-based compensation expenses||(14,837)||(9,972)||(51,872)||(42,946)|
|Non-GAAP Research and development||$||102,935||$||95,039||$||338,763||$||339,446|
|Reconciliation of GAAP to Non-GAAP General and administrative:|
|GAAP General and administrative||$||67,455||$||27,876||$||199,365||$||89,354|
|Less: Stock-based compensation expenses||(12,280)||(10,679)||(40,947)||(32,582)|
|Non-GAAP General and administrative||$||55,175||$||17,197||$||158,418||$||56,772|
|Reconciliation of GAAP to Non-GAAP Operating expenses:|
|GAAP Operating expenses||$||185,227||$||132,887||$||590,000||$||471,746|
|Less: Stock-based compensation expenses||(27,117)||(20,651)||(92,819)||(75,528)|
|Non-GAAP Operating expenses||$||158,110||$||112,236||$||497,181||$||396,218|
|Reconciliation of GAAP to Non-GAAP Net loss:|
|GAAP Net loss||$||(142,227)||$||(112,934)||$||(490,874)||$||(410,108)|
|Add: Stock-based compensation expenses||27,117||20,651||92,819||75,528|
|Non-GAAP Net loss||$||(115,110)||$||(92,283)||$||(398,055)||$||(334,580)|
|Reconciliation of GAAP to Non-GAAP Net loss per common share-basic and diluted:|
|GAAP Net loss per common share - basic and diluted||$||(1.48)||$||(1.32)||$||(5.42)||$||(4.79)|
|Add: Stock-based compensation expenses||0.28||0.24||1.02||0.88|
|Non-GAAP Net loss per common share - basic and diluted||$||(1.20)||$||(1.08)||$||(4.40)||$||(3.91)|
ALNYLAM PHARMACEUTICALS, INC.
|December 31,||December 31,|
|Cash, cash equivalents and fixed income marketable securities||$||1,704,537||$||942,601|
|Billed and unbilled collaboration receivables||34,002||23,334|
|Prepaid expenses and other assets||44,291||32,303|
|Property, plant and equipment, net||181,900||114,572|
|Accounts payable, accrued expenses and other liabilities||$||104,905||$||99,650|
|Total deferred revenue||84,780||82,932|
|Total deferred rent||8,614||10,007|
|Total stockholders’ equity (99.7 million and 85.9 million common shares issued and outstanding at December 31, 2017 and December 31, 2016, respectively)||1,766,431||920,221|
|Total liabilities and stockholders' equity||$||1,994,730||$||1,262,810|
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
Alnylam Pharmaceuticals, Inc.
(Investors and Media)
Christine Regan Lindenboom, 617-682-4340
Josh Brodsky, 617-551-8276