Alnylam Pharmaceuticals Reports Second Quarter 2017 Financial Results and Highlights Recent Period Activity
- Advanced Industry-Leading RNAi Therapeutics Pipeline, Including
Four Programs in
- On Track to Report APOLLO Phase 3 Top-Line Results for Patisiran in
Near Term, and Initiated ATLAS Phase 3
- Maintained Strong Balance Sheet with
"2017 is shaping up to be a pivotal year for Alnylam. With patisiran, we
expect to report top-line APOLLO Phase 3 study results in the coming
weeks. If positive, these study findings will support our first NDA
filing, planned by year-end, and our commercial transition in mid-2018
assuming regulatory approval. In parallel, we continue to advance our
late-stage pipeline of investigational RNAi therapeutics, with the
recently announced initiation of our ATLAS Phase 3 program for fitusiran
in hemophilia and expected Phase 3 starts by year-end for givosiran in
acute hepatic porphyrias and, with The Medicines Company, inclisiran in
Second Quarter 2017 and Recent Significant Corporate Highlights
Advanced patisiran, an investigational RNAi therapeutic for the
treatment of patients with hereditary ATTR (hATTR) amyloidosis with
polyneuropathy, with final
24-month data from the Phase 2 open-label extension (OLE) study
presented at the
American Academy of Neurologymeeting and top-line APOLLO Phase 3 data expected in mid-2017.
Advanced fitusiran, an investigational RNAi therapeutic for the
treatment of hemophilia and rare bleeding disorders, with positive
new data from the Phase 2 OLE study presented at the
International Society on Thrombosis and Haemostasis2017 Congress.
Results from the Phase 1 study were published in
The New England Journal of Medicinein a paper titled, "Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy."
- Alnylam and partner Sanofi Genzyme announced the initiation of the ATLAS Phase 3 program, a global, multicenter clinical program designed to evaluate the safety and efficacy of fitusiran in patients with hemophilia A and B with or without inhibitors.
- Results from the Phase 1 study were published in
Advanced givosiran, an investigational RNAi therapeutic for the
treatment of acute hepatic porphyrias (AHPs), with positive
new data presented at the 2017
International Congress on Porphyrins and Porphyrias(ICPP) from the ongoing randomized, double-blind, placebo-controlled Phase 1 study in recurrent attack porphyria patients, as well as positive initial results from the ongoing Phase 1 OLE study.
In addition, received Breakthrough Therapy designation from the
U.S. Food and Drug Administration(FDA) for givosiran for the prophylaxis of attacks in patients with acute hepatic porphyria.
- In addition, received Breakthrough Therapy designation from the
Alnylam and partner The Medicines Company announced agreement with the
FDAon a Phase 3 clinical program for inclisiran, an investigational RNAi therapeutic for the treatment of hypercholesterolemia, with LDL-C lowering as the primary endpoint for the initial pivotal trial program, which is expected to initiate in late 2017.
Completed successful public offering of common stock, with concurrent
private placement from Sanofi Genzyme, totaling
$376.5 millionin net proceeds.
- Alnylam will continue to host its 4th Annual RNAi Roundtable Series, which kicked off last week. This series consists 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 can be found here.
Alnylam plans to report top-line results from the patisiran APOLLO
Phase 3 study in mid-2017. Full results are expected to be presented
in late 2017 at the 1st European ATTR Amyloidosis Meeting
for Patients and Doctors, being held
November 2-3, 2017in Paris, France.
- If the APOLLO Phase 3 data are positive, Alnylam expects to file its first New Drug Application (NDA) in late 2017 and Marketing Authorisation Application (MAA) shortly thereafter.
- Alnylam plans to initiate a Phase 3 study of givosiran in late 2017, pending successful alignment on trial design with global regulatory authorities.
- The Medicines Company has announced its intention to initiate a Phase 3 study of inclisiran in patients with atherosclerotic cardiovascular disease (ASCVD) in late 2017.
Financial results for the quarter ended
"Alnylam continues to maintain a strong balance sheet," said
Cash and Investments
In addition, Sanofi Genzyme exercised its right to purchase, in a
concurrent private placement, 297,501 shares of common stock, at the
public offering price of
GAAP and Non-GAAP Net Loss
The net loss according to accounting principles generally accepted in the
The non-GAAP net loss for the second 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.
Research and Development Expenses
Research and development (R&D) expenses were
General and Administrative Expenses
General and administrative (G&A) expenses were
Alnylam remains on track to end 2017 with greater than
Conference Call Information
Management will provide an update on the company and discuss second quarter 2017 results as well as expectations for the future via conference call on
In the case of patisiran, Alnylam will advance the product in
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding protein synthesis 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, with the goal of preventing disease-causing proteins from being made.
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.
Use of Non-GAAP Financial Measures
This press release contains non-GAAP financial measures, including net loss 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 this 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 net loss 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, non-GAAP net loss is 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 net loss is provided later in this press release.
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 and MAA for patisiran if the APOLLO Phase 3 study is positive and its expected transition to commercial operations in mid-2018 if regulators approve patisiran, its expected cash, cash equivalents, fixed marketable securities and restricted investments 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
Six Months Ended
|Net revenues from collaborators||$||15,932||$||8,709||$||34,892||$||16,054|
|Research and development||90,627||83,172||177,611||179,445|
|General and administrative||45,779||17,987||84,266||39,087|
|Total operating expenses||136,406||101,159||261,877||218,532|
|Loss from operations||(120,474||)||(92,450||)||(226,985||)||(202,478||)|
|Other income (expense):|
|Other (expense) income||(523||)||229||(3,430||)||5,470|
|Total other income||2,054||2,321||1,275||9,375|
|Net loss per common share - basic and diluted||$||(1.34||)||$||(1.05||)||$||(2.59||)||$||(2.26||)|
|Weighted-average common shares used to compute basic and diluted net loss per common share||88,098||85,545||87,068||85,411|
|Unrealized loss on marketable securities, net of tax||(476||)||(18,331||)||(2,412||)||(26,555||)|
|Reclassification adjustment for realized loss (gain) on marketable securities included in net loss||345||(954||)||1,894||(6,110||)|
|RECONCILIATION OF GAAP NET LOSS TO NON-GAAP NET LOSS|
|(In thousands, except per share amounts)|
Three Months Ended
Six Months Ended
|GAAP net loss||$||(118,420||)||$||(90,129||)||$||(225,710||)||$||(193,103||)|
|Stock-based compensation expenses||24,030||15,816||39,747||39,296|
|Non-GAAP net loss||$||(94,390||)||$||(74,313||)||$||(185,963||)||$||(153,807||)|
|GAAP net loss per common share - basic and diluted||$||(1.34||)||$||(1.05||)||$||(2.59||)||$||(2.26||)|
|Adjustment (as detailed above)||0.27||0.18||0.45||0.46|
|Non-GAAP net loss per common share - basic and diluted||$||(1.07||)||$||(0.87||)||$||(2.14||)||$||(1.80||)|
|UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS|
|(In thousands, except share amounts)|
|Cash, cash equivalents and fixed income marketable securities||$||1,097,841||$||942,601|
|Billed and unbilled collaboration receivables||15,405||23,334|
|Prepaid expenses and other assets||23,504||32,303|
|Property, plant and equipment, net||147,533||114,572|
|Accounts payable, accrued expenses and other liabilities||$||69,805||$||99,650|
|Total deferred revenue||81,873||82,932|
|Total deferred rent||9,639||10,007|
Total stockholders' equity (91.7 million and 85.9 million common
shares issued and outstanding at
|Total liabilities and stockholders' equity||$||1,434,283||$||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
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