Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2016 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 $1.1 Billion in Cash and Expects to End 2017 with Greater than $700 Million in Cash -
"We continue to advance a broad pipeline of investigational RNAi
therapeutics - including 8 programs in clinical development - across a
wide range of disease indications with high unmet need. In the fourth
quarter of 2016 and recent period, we reported positive clinical results
with fitusiran for hemophilia, givosiran for porphyria, and ALN-CC5 for
complement-mediated diseases. In addition, our partners at The Medicines
Company, reported positive interim clinical data with inclisiran for
hypercholesterolemia. On the other hand, we were disappointed to
announce the discontinuation of our revusiran program due to safety
findings, although our investigation to date indicates the findings are
unique to this program and the clinical setting," said
Fourth Quarter 2016 and Recent Significant Corporate Highlights
- Advanced patisiran for the treatment of polyneuropathy due to hereditary ATTR (hATTR) amyloidosis, with APOLLO Phase 3 data expected in mid-2017.
- Announced decision to discontinue development of revusiran, an investigational RNAi therapeutic that was being developed for the treatment of cardiomyopathy due to hATTR amyloidosis.
Advanced fitusiran for the treatment of hemophilia and rare bleeding
disorders (RBD), with positive new
data presented at the 2016
American Society of Hematology(ASH) meeting and additional data presented at the 2017 European Association for Haemophilia and Allied Disorders(EAHAD) meeting.
Alnylam and partner Sanofi Genzyme announced that Sanofi Genzyme
elected to exercise its right to co-develop and co-promote
fitusiran with Alnylam in
the United States, Canadaand Western Europe. Sanofi Genzyme retains commercial rights for rest of world.
- Alnylam and partner Sanofi Genzyme announced that Sanofi Genzyme elected to exercise its right to co-develop and co-promote fitusiran with Alnylam in
Advanced givosiran (ALN-AS1) for the treatment of acute hepatic
porphyrias with positive initial
clinical activity results reported at the 2016 ASH meeting from
Phase 1 study in acute intermittent porphyria (AIP) patients with
recurring porphyria attacks.
- Alnylam and partner Sanofi Genzyme announced that Sanofi Genzyme has elected not to opt in to develop and commercialize givosiran, providing Alnylam with full global control of the program for further development and potential commercialization.
Alnylam's partner, The Medicines Company, announced positive results
from the Day 90 interim analysis at the 2016
American Heart Associationmeeting and top-line Day 180 results for all 501 patients in the ongoing ORION-1 Phase 2 study of inclisiran (also known as ALN-PCSsc and PCSK9si), an investigational RNAi therapeutic for the treatment of hypercholesterolemia.
- 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.
clinical results at the 2016 ASH meeting from Phase 1/2 study of
ALN-CC5 in patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Alnylam and partner Sanofi Genzyme announced that Sanofi Genzyme has elected not to opt in to develop and commercialize ALN-CC5, providing Alnylam with full global control of the program for further development and potential commercialization.
- Reported positive initial clinical results from Phase 1 study of ALN-TTRsc02, an ESC-GalNAc conjugate targeting TTR for hATTR amyloidosis.
Upcoming Events in Early and Mid-2017
Alnylam announces today that it plans to present complete 24-month
data and additional analyses from the Phase 2 OLE study of patisiran
American Academy of Neurology(AAN) 69th Annual Meeting, being held April 22- 28, 2017 in Boston, Massachusetts, in an oral presentation on Wednesday, April 26at 8:24 am ET.
In addition, The Medicines Company plans to present full safety and
efficacy data from the ORION-1 Phase 2 study of inclisiran, with six-
to nine-month follow-up for all patients in the study, at the American
College of Cardiology's 66th Annual Scientific
Session, to be held March 17 - 19, 2017, in Washington, D.C., in a
late-breaker oral presentation on
Friday, March 17, at 1:30 pm ET.
- Also 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;
Present additional data from the Phase 2 OLE study of fitusiran,
likely at the
International Society on Thrombosis and Haemostasis(ISTH) 2017 Congress, being held July 8- 13, 2017, in Berlin, Germany;
Present additional data from Part C of the Phase 1 study of
givosiran, likely at the 2017
International Congress of Porphyrins and Porphyrias(ICPP), being held June 25- 28, 2017, in Bordeaux, France; and,
- The Medicines Company plans to initiate the Phase 3 program for inclisiran.
"Alnylam continues to maintain a strong balance sheet, ending 2016 with
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
2017 Financial Guidance
Alnylam expects that its cash, cash equivalents and marketable securities, and restricted investments balance will be greater than
Conference Call Information
Management will provide an update on the company, discuss fourth quarter 2016 results, and discuss expectations for the future via conference call on Wednesday, February 8, 2017 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 57758793. A replay of the call will be available beginning at 7:30 p.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 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,
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, inclisiran, and ALN-TTRsc02,
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
ongoing, comprehensive evaluation of the revusiran safety findings and
any indications or tentative conclusions based on the evaluation to
date, its expected cash position as of
The scientific information discussed in this news release relating to
Alnylam's investigational therapeutics is preliminary and investigative.
None of Alnylam's investigational therapeutics 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||$||17,454||$||7,551||$||47,159||$||41,097|
|Research and development||105,011||82,835||382,392||276,495|
|General and administrative||27,876||17,228||89,354||60,610|
|Total operating expenses||132,887||100,063||471,746||337,105|
|Loss from operations||(115,433||)||(92,512||)||(424,587||)||(296,008||)|
|Total other income||2,499||1,791||14,479||5,935|
|Net loss per common share - basic and diluted||$||(1.32||)||$||(1.07||)||$||(4.79||)||$||(3.45||)|
|Weighted-average common shares used to compute basic and diluted net loss per common share||85,843||84,871||85,596||83,992|
|Unrealized (loss) gain on marketable securities, net of tax||(5,502||)||11,588||(30,833||)||(44,394||)|
Reclassification adjustment for realized gain on marketable
included in net loss
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except share amounts)
|Cash, cash equivalents and marketable securities||$||942,601||$||1,280,951|
|Billed and unbilled collaboration receivables||23,334||8,298|
|Prepaid expenses and other assets||23,306||18,030|
|Property and equipment, net||114,572||27,812|
|Investment in equity securities of Regulus Therapeutics Inc.||8,997||51,419|
|Accounts payable, accrued expenses and other liabilities||$||99,650||$||46,886|
|Total deferred revenue||82,932||68,317|
|Total deferred rent||10,007||6,593|
Total stockholders' equity (85.9 million and 85.1 million common
shares issued and outstanding and at
|Total liabilities and stockholders' equity||$||1,262,810||$||1,386,510|
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
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