Alnylam Pharmaceuticals Reports First Quarter 2017 Financial Results and Highlights Recent Period Activity
- Advanced Industry-Leading RNAi Therapeutics Pipeline with Eight
Clinical Programs, Including Three Programs in
- Maintained Strong Balance Sheet with $962 Million in Cash and Plans to End 2017 with Greater than $700 Million in Cash -
"2017 promises to be a pivotal year for Alnylam. With our patisiran
program, we look forward to the read-out of our APOLLO Phase 3 study
and, if the data are positive, our first NDA filing. In addition, we
expect to advance three additional programs into Phase 3 trials:
fitusiran, givosiran, and - with our partners at The Medicines Company -
First Quarter 2017 and Recent Significant Corporate Highlights
Advanced patisiran, an investigational RNAi therapeutic for the
treatment of polyneuropathy due to hereditary ATTR (hATTR)
amyloidosis, with final
24-month data from the Phase 2 open-label extension (OLE) study
presented at the
American Academy of Neurologymeeting and top-line results from the APOLLO Phase 3 study expected in mid-2017.
- Advanced fitusiran, an investigational RNAi therapeutic for the treatment of hemophilia and rare bleeding disorders, with positive new data presented at the 2017 European Association for Haemophilia and Allied Disorders (EAHAD) meeting.
- Advanced givosiran (ALN-AS1), an investigational RNAi therapeutic for the treatment of the acute hepatic porphyrias, with acceptance into the European Medicines Agency's PRIME program.
Alnylam's partner, The Medicines Company, announced positive final
results from the ORION-1 Phase 2 study of inclisiran, an
investigational RNAi therapeutic for the treatment of
hypercholesterolemia, at the
American Collegeof Cardiology's 66th Annual Scientific Session.
- The Medicines Company initiated the ORION-2 study of inclisiran in patients with Homozygous Familial Hypercholesterolemia (HoFH) as well as the ORION-3 study, a Phase 2 open-label cross-over extension study for patients completing the ORION-1 study.
In addition, The Medicines Company and Alnylam announced agreement
FDAon the Phase 3 clinical program for inclisiran.
Upcoming Events in Early and Mid-20171
- Alnylam announces today that it plans to host its 4th Annual RNAi Roundtable Series during the summer. This series will consist of webinars designed to inform attendees of the latest progress and upcoming milestones for many of the company's investigational RNAi therapeutic programs. More details for the series are forthcoming.
The Company also announces today that it plans to present additional
clinical data from the Phase 1 study of givosiran at the 2017
International Congress of Porphyrins and Porphyrias(ICPP), being held June 25- 28, 2017, in Bordeaux, France, in an oral presentation on Monday, June 26at 11:45amCentral European Time ( 5:45 am ET).
In addition, the Company announces that it plans to present additional
data from the Phase 2 OLE study of fitusiran at the
International Society on Thrombosis and Haemostasis(ISTH) 2017 Congress, being held July 8- 13, 2017, in Berlin, Germany, in an oral presentation on Monday, July 10at 2:45 pmCentral European Time ( 8:45 am ET).
- In early 2017, Alnylam plans to initiate the ATLAS Phase 3 program for fitusiran.
- In mid-2017, Alnylam plans to report top-line results from the APOLLO Phase 3 study of patisiran.
- Also in mid-2017, The Medicines Company plans to initiate a Phase 3 study of inclisiran in patients with atherosclerotic cardiovascular disease (ASCVD).
"Alnylam continues to maintain a strong balance sheet, ending the first
quarter of 2017 with approximately
Cash and Investments
GAAP Net Loss
The net loss according to accounting principles generally accepted in the
Research and Development Expenses
Research and development (R&D) expenses were
General and Administrative Expenses
General and administrative (G&A) expenses were
Conference Call Information
Management will provide an update on the company, discuss first quarter 2017 results, and discuss expectations for the future via conference call on Friday, May 5, 2017 at 8:30 a.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 13425320. A replay of the call will be available beginning at 11:30 a.m. ET on
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 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.
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 patients who have limited or inadequate treatment options. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of 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 three product candidates that are in late-stage development or will be in 2017. 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. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam.
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, fitusiran, givosiran, and inclisiran, its expectations regarding the timing of clinical studies and the presentation of clinical data, including for studies for patisiran, fitusiran, givosiran, and inclisiran, its expectations regarding the potential filing of an NDA for patisiran if the APOLLO Phase 3 study is positive, its expected cash position as of
The scientific information referenced in this news release relating to
Alnylam's investigational therapeutics is preliminary and investigative.
None of Alnylam's investigational therapeutics, including inclisiran
which is partnered with The Medicines Company, have been approved by the
|UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS|
|(In thousands, except per share amounts)|
Three Months Ended
|Net revenues from collaborators||$||18,960||$||7,345|
|Research and development||86,984||96,273|
|General and administrative||38,487||21,100|
|Total operating expenses||125,471||117,373|
|Loss from operations||(106,511)||(110,028)|
|Other income (expense):|
|Other (expense) income||(2,907)||5,241|
|Total other (expense) income||(779)||7,054|
|Net loss per common share - basic and diluted||$||(1.25)||$||(1.21)|
|Weighted-average common shares used to compute basic and diluted net loss per common share||86,027||85,277|
|Unrealized loss on marketable securities, net of tax||(1,936)||(8,224)|
Reclassification adjustment for realized loss (gain) on marketable
securities included in net loss
|UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS|
|(In thousands, except share amounts)|
|Cash, cash equivalents and fixed income marketable securities||$||812,227||$||942,601|
|Billed and unbilled collaboration receivables||19,457||23,334|
|Prepaid expenses and other assets||22,280||32,303|
|Property, plant and equipment, net||129,962||114,572|
|Accounts payable, accrued expenses and other liabilities||$||60,397||$||99,650|
|Total deferred revenue||82,804||82,932|
|Total deferred rent||9,824||10,007|
Total stockholders' equity (86.1 million and 85.9 million common
shares issued and outstanding and at
|Total liabilities and stockholders' equity||$||1,133,926||$||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
1 Early is Q1-Q2, Mid is Q2-Q3, and Late is Q3-Q4
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