Alnylam Pharmaceuticals Reports First Quarter 2018 Financial Results and Highlights Recent Period Activity
– Presented Positive New Clinical Results from APOLLO Phase 3 Study of Patisiran, and Phase 1 and Phase 1/2 Open-Label Extension (OLE) Studies of Givosiran –
– Advanced Two Additional RNAi Therapeutics in Phase 3 Development: Inclisiran in ORION-9, -10, and -11 Studies, and Fitusiran in the ATLAS Program –
– Company Provides Positive New Development Updates on Phase 3 Programs for Givosiran and Lumasiran –
– Maintained Strong Balance Sheet with
“We had a very productive first quarter of 2018 and recent period. We’re
working in close coordination with global regulatory authorities on
bringing investigational patisiran to patients around the world. In
parallel, we are actively preparing for our U.S. launch of patisiran,
following an anticipated mid-2018
First Quarter 2018 and Recent Significant Corporate Highlights
Advanced patisiran, an investigational RNAi therapeutic for the
treatment of patients with hereditary ATTR amyloidosis.
Presented new data from the APOLLO Phase 3 study, including:
- Data at the International Symposium on Amyloidosis (ISA), including results on the effects of patisiran on cardiomyopathy manifestations; and,
data from a post-hoc analysis presented at the
American Academy of Neurology(AAN) meeting on the effects of patisiran on the composite rate of all-cause hospitalization and mortality.
Received acceptance from the
United States Food and Drug Administration( FDA) and the European Medicines Agency(EMA) of patisiran’s New Drug Application (NDA) and Marketing Authorisation Application (MAA), respectively.
- The Company is continuing to fulfill requests from treating physicians for early access or compassionate use of patisiran, and to date, more than 150 eligible patients have begun treatment with patisiran under these programs in the U.S. and EU.
- Presented new data from the APOLLO Phase 3 study, including:
Advanced ALN-TTRsc02, a subcutaneously administered investigational
RNAi therapeutic in development for the treatment of ATTR amyloidosis,
data from the Phase 1 study presented at ISA, and receipt of a
positive opinion from the EMA Committee for Orphan Medicinal Products
(COMP) for Orphan Drug Designation in the
European Union for ALN-TTRsc02for the treatment of ATTR amyloidosis.
Advanced givosiran, an investigational RNAi therapeutic in development
for the treatment of acute hepatic porphyrias (AHPs).
new positive Phase 1 and Phase 1/2 open-label extension (OLE)
study results at the
European Association for the Study of the Liver(EASL) meeting.
The Company announces today that it has completed enrollment of
the first 30 patients in the ENVISION Phase 3 study, which
comprise the interim analysis cohort for a potential accelerated
approval by the
FDA. This positions the Company to report interim analysis results in the September timeframe and, pending FDAreview of the program at the time of interim analysis and assuming positive results, the Company expects to submit an NDA at or around year-end 2018.
The Company also announces today that notwithstanding productive
conversations with the EMA on a potential accelerated approval
pathway for givosiran, the Company has decided to file an MAA on
the full dataset from ENVISION, expected in 2019, to optimize
market access in
- Expanded the Alnylam Act™ program to include no-charge, third-party genetic testing and counseling requested by enrolled physicians for individuals who may carry a gene mutation known to be associated with AHPs. The Company announces that out of approximately 50 samples submitted to date by physicians with patients with symptoms consistent with AHPs, eight have tested positive for known mutations associated with AHPs.
- Presented new positive Phase 1 and Phase 1/2 open-label extension (OLE) study results at the
Advanced lumasiran, an investigational RNAi therapeutic in development
for the treatment of primary hyperoxaluria type 1 (PH1).
- Retained global rights to the program following the decision by Sanofi Genzyme to decline its opt-in for lumasiran’s development and commercialization.
As announced earlier today, the Company has reached alignment with
FDAon a pivotal study design for lumasiran with reduction at six months in urinary oxalate as the primary endpoint. In addition, the pivotal study will comprise approximately 25 patients with PH1. Alnylam is now guiding that it expects to initiate the lumasiran Phase 3 trial in mid-2018 with results expected in 2019. If positive, Alnylam expects to file an NDA in early 2020.
Lumasiran was recently granted Breakthrough Therapy Designation by
FDAas well as access to the EMA’s Priority Medicines (PRIME) scheme.
Sanofi, advanced fitusiran – an investigational RNAi therapeutic in development for the treatment of hemophilia A and B with or without inhibitors – with the initiation of dosing in the ATLAS Phase 3 program. The Company expects to fully transition development and commercialization leadership of the fitusiran program to Sanofiin mid-2018.
Alnylam’s partner, The
Medicines Company, completed enrollment in the ORION-9, -10, and -11 Phase 3 studies of inclisiran in 3,660 patients with atherosclerotic cardiovascular disease (ASCVD) or heterozygous familial hypercholesterolemia (HeFH).
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.
- Announced plans to collaborate with Regeneron to identify RNAi therapeutics targeting the genetically validated target HSD17B13 for nonalcoholic steatohepatitis (NASH) and potentially other related diseases.
- 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 settlement of all pending litigation between the Company
and Dicerna, in which Dicerna agreed to pay
$25 millionin cash and equity to Alnylam and also agreed to restrictions on its development and other activities relating to oligonucleotide-based therapeutics directed toward certain Alnylam targets, for periods ranging from eighteen months up to four years. The settlement does not include any licenses of Alnylam’s GalNAc technology platform patent rights or licenses of any other intellectual property.
Upcoming Events in Mid-2018
Alnylam expects to gain regulatory approval for patisiran from the
FDAin mid-2018. The FDAhas set an action date of August 11, 2018, under the Prescription Drug User Fee Act (PDUFA). The Company expects to launch patisiran in the U.S. shortly after FDAapproval.
Alnylam plans to file for regulatory approval for patisiran in
Japanand other global markets, including a Japanese NDA filing with the Pharmaceuticals and Medical Device Agency(PMDA) in mid-2018.
Alnylam intends to report topline interim results from the ENVISION
Phase 3 trial for givosiran in the September timeframe.
- The interim analysis is based on lowering of urinary aminolevulinic acid (ALA) levels at 3 months of treatment as a surrogate marker that is reasonably likely to predict clinical benefit.
- In addition, a blinded assessment of attack rate results will be conducted at the time of the interim analysis, which the Company expects will likely result in an increase in ENVISION study sample size to approximately 95 patients.
- Alnylam intends to initiate the lumasiran Phase 3 study in mid-2018.
Medicines Companyhas guided its intention to initiate enrollment in the ORION-4 cardiovascular outcomes (CVOT) study in mid-2018.
Sanofiexpect to enroll patients in the ATLAS Phase 3 program of fitusiran in patients with hemophilia A and B with or without inhibitors throughout the year.
Financial results for the quarter ended
“Alnylam’s strong balance sheet with approximately
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 first quarter of 2018 was
The non-GAAP net loss for the first quarter of 2018 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 Updated Financial Guidance
Alnylam remains on track to end 2018 with approximately
The Company now expects its 2018 annual non-GAAP R&D expenses to be in
the range of
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 first quarter 2018 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.
About LNP Technology
Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology.
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 800 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 and lumasiran, 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.
UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
(In thousands, except per share amounts)
|Three Months Ended
|Net revenues from collaborators||$||21,899||$||18,960|
|Research and development||96,857||86,984|
|General and administrative||72,447||38,487|
|Total operating expenses||169,304||125,471|
|Loss from operations||(147,405||)||(106,511||)|
|Other income (expense):|
|Other income (expense)||335||(2,907||)|
|Total other income (expense)||6,129||(779||)|
|Loss before income taxes||(141,276||)||(107,290||)|
|Benefit from income taxes||62||—|
|Net loss per common share - basic and diluted||$||(1.41||)||$||(1.25||)|
|Weighted-average common shares used to compute basic and diluted net loss per common share||99,979||86,027|
|Unrealized loss on marketable securities, net of tax||(420||)||(1,936||)|
|Reclassification adjustment for realized loss on marketable securities included in net loss||—||1,549|
ALNYLAM PHARMACEUTICALS, INC.
RECONCILIATION OF SELECTED GAAP MEASURES TO NON-GAAP MEASURES
(In thousands, except per share amounts)
|Three Months Ended
|Reconciliation of GAAP to Non-GAAP Research and development:|
|GAAP Research and development||$||96,857||$||86,984|
|Less: Stock-based compensation expenses||(10,137||)||(8,691||)|
|Non-GAAP Research and development||$||86,720||$||78,293|
|Reconciliation of GAAP to Non-GAAP General and administrative:|
|GAAP General and administrative||$||72,447||$||38,487|
|Less: Stock-based compensation expenses||(9,447||)||(7,026||)|
|Non-GAAP General and administrative||$||63,000||$||31,461|
|Reconciliation of GAAP to Non-GAAP Operating expenses:|
|GAAP Operating expenses||$||169,304||$||125,471|
|Less: Stock-based compensation expenses||(19,584||)||(15,717||)|
|Non-GAAP Operating expenses||$||149,720||$||109,754|
|Reconciliation of GAAP to Non-GAAP Net loss:|
|GAAP Net loss||$||(141,214||)||$||(107,290||)|
|Add: Stock-based compensation expenses||19,584||15,717|
|Non-GAAP Net loss||$||(121,630||)||$||(91,573||)|
|Reconciliation of GAAP to Non-GAAP Net loss per common share-basic and diluted:|
|GAAP Net loss per common share - basic and diluted||$||(1.41||)||$||(1.25||)|
|Add: Stock-based compensation expenses||0.19||0.19|
|Non-GAAP Net loss per common share - basic and diluted||$||(1.22||)||$||(1.06||)|
ALNYLAM PHARMACEUTICALS, INC.
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except share amounts)
|March 31,||December 31,|
|Cash, cash equivalents and fixed income marketable securities||$||1,568,512||$||1,704,537|
|Billed and unbilled collaboration receivables||50,768||34,002|
|Prepaid expenses and other assets||71,880||44,291|
|Property, plant and equipment, net||201,979||181,900|
|Accounts payable, accrued expenses and other liabilities||$||81,488||$||104,905|
|Total deferred revenue||43,531||84,780|
|Total deferred rent||13,315||8,614|
|Total stockholders’ equity (100.5 million and 99.7 million common shares issued and outstanding at March 31, 2018 and December 31, 2017, respectively)||1,754,805||1,766,431|
|Total liabilities and stockholders' equity||$||1,923,139||$||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
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom, 617-682-4340
(Investors and Media)
Josh Brodsky, 617-551-8276