Alnylam Pharmaceuticals Reports Third Quarter 2017 Financial Results and Highlights Recent Period Activity
- Reported Positive APOLLO Phase 3 Results for Patisiran, with Plans for First New Drug Application (NDA) Filing by Year-End -
- Advanced Four RNAi Therapeutics in
- Maintained Strong Balance Sheet with $1.15 Billion in Cash and Plans to End 2017 with Greater than $1 Billion in Cash -
"In our view, 2017 has proven to be a remarkable year for RNAi
therapeutics, for Alnylam, and, most importantly, for the patients that
we serve. With patisiran, our recent APOLLO Phase 3 study results
demonstrate what we believe to be the transformative potential for RNAi
therapeutics as a new class of innovative medicines. With these data, we
expect to submit our first regulatory filings in the coming months, and
are planning for the possibility of having regulatory approval for
patisiran in mid-2018," said
Third Quarter 2017 and Recent Significant Corporate Highlights
Advanced patisiran, an investigational RNAi therapeutic in development
for the treatment of patients with hereditary ATTR (hATTR)
amyloidosis, with positive
results from the APOLLO Phase 3 study (N=225).
Patisiran met its primary endpoint (p = 9.26 x 10-24)
with a 34.0 point mean difference relative to placebo and a
negative 6.0 point mean change (improvement) relative to baseline
in the modified neuropathy impairment score (mNIS+7) at 18 months,
as well as all secondary endpoints (p less than 0.001), including
a 21.1 point mean difference relative to placebo and a negative
6.7 point mean change (improvement) relative to baseline in the
NORFOLKQuality of Life-Diabetic Neuropathy score ( NORFOLKQOL-DN) (p = 1.10 x 10-10), at 18 months.
- Patisiran also demonstrated a favorable safety and tolerability profile relative to placebo. The most commonly reported adverse events (AEs) for patisiran were generally mild to moderate and included peripheral edema (29.7 percent) and infusion-related reactions (IRRs) (18.9 percent), and the frequency of deaths and serious adverse events (SAEs) was similar in the patisiran and placebo groups. No deaths were considered drug-related.
- Specifically, the Company believes that these data support a potentially "best-in-class" product profile, with significant benefit relative to placebo, negative mean and median values (improvement) for mNIS+7 and QOL measures relative to baseline, and encouraging safety and tolerability.
- In addition, patisiran achieved significant effects in the study's cardiac subpopulation, including on disease biomarker, echocardiographic, and functional parameters.
- The Company believes the totality of the APOLLO data are consistent with a clinically meaningful impact for patisiran on hATTR amyloidosis, and plans to submit an NDA for patisiran by the end of 2017 and a Marketing Authorisation Application (MAA) shortly thereafter.
- Patisiran met its primary endpoint (p = 9.26 x 10-24) with a 34.0 point mean difference relative to placebo and a negative 6.0 point mean change (improvement) relative to baseline in the modified neuropathy impairment score (mNIS+7) at 18 months, as well as all secondary endpoints (p less than 0.001), including a 21.1 point mean difference relative to placebo and a negative 6.7 point mean change (improvement) relative to baseline in the
Advanced ALN-TTRsc02, a subcutaneously administered investigational
RNAi therapeutic in development for the treatment of ATTR amyloidosis.
- Presented updated Phase 1 data showing up to 95% transthyretin (TTR) knockdown with a single 50 mg dose, with durability supportive of a once quarterly and, possibly, bi-annual subcutaneous dose regimen.
- Reaffirmed guidance to initiate a Phase 3 program for ALN-TTRsc02 in 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 the
U.S. Food and Drug Administration(FDA) on the design of ENVISION, including an interim analysis based on reduction of urinary aminolevulinic acid (ALA), a biomarker that the FDAconsiders to be reasonably likely to predict clinical benefit.
The Company has also reached alignment on the ENVISION Phase 3
study design with the
European Medicines Agency(EMA).
The Company is guiding 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 the
Advanced fitusiran, an investigational RNAi therapeutic in development
for the treatment of hemophilia A and B with or without inhibitors,
positive data from the Phase 2 open-label extension (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."
- 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.
- The Company temporarily suspended dosing in all ongoing studies of fitusiran following the observation of a fatal thrombotic SAE that occurred in a patient with hemophilia A without inhibitors who was receiving fitusiran in the Phase 2 OLE study. Alnylam and fitusiran study investigators have aligned on a risk management plan for further advancement of fitusiran and are now conferring with global regulators with the goal of resuming dosing as soon as possible, potentially by the end of 2017.
- Results from the Phase 1 study were published in
Alnylamand The Medicines Company announced initiation of the ORION-11 Phase 3 study of inclisiran, an investigational RNAi therapeutic targeting PCSK9 in development for the treatment of hypercholesterolemia, in patients with atherosclerotic cardiovascular disease (ASCVD).
The companies announced new
positive data from the ORION-1 Phase 2 study of
inclisiran at the
European Society of Cardiology Congress2017.
- The companies announced new positive data from the ORION-1 Phase 2 study of inclisiran at the
- Advanced cemdisiran (formerly known as ALN-CC5), a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases, with the initiation of a Phase 2 clinical study in patients with atypical hemolytic-uremic syndrome (aHUS).
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.
- Alnylam announced a licensing agreement with Vir Biotechnology for the development and commercialization of RNAi therapeutics for infectious diseases, including hepatitis B.
Advanced RNAi platform technology with new
pre-clinical data on Alnylam's next generation "Enhanced
Stabilization Chemistry Plus" (ESC+) GalNAc-siRNA conjugate platform
presented at the 13th Annual Meeting of the
Oligonucleotide Therapeutics Society.
Alnylam announces today that Alnylam scientists and collaborators will
present new results from multiple pipeline programs at the 59th
American Society of Hematology(ASH) Annual Meeting, being held December 9- 12, 2017 in Atlanta, Georgia. Presentations include:
Explore: A Prospective, Multinational History Study of Patients
with Acute Hepatic Porphyrias (AHP) with Recurrent Attacks
Session: 102. Regulation of Iron Metabolism: Poster II
Sunday, December 10, 6:00 - 8:00 p.m. ET
Perioperative Management in Patients with Hemophilia Receiving
Fitusiran, an Investigational RNAi Therapeutic Targeting
Antithrombin for the Treatment of Hemophilia
Session: 322. Disorders of Coagulation or Fibrinolysis: Poster II
Sunday, December 10, 6:00 - 8:00 p.m. ET
In Silico Modeling of the Impact of Antithrombin Lowering on
Thrombin Generation in Rare Bleeding Disorders
Session: 321. Blood Coagulation and Fibrinolytic Factors: Poster III
Monday, December 11, 6:00 - 8:00 p.m. ET
- Explore: A Prospective, Multinational History Study of Patients with Acute Hepatic Porphyrias (AHP) with Recurrent Attacks
Alnylam plans to file its first NDA for patisiran with the
FDAby the end of 2017, followed by an MAA in the EU in early 2018.
- Alnylam plans to meet with global regulatory authorities with the goal of reaching agreement on a risk mitigation plan and resumption of dosing in fitusiran clinical studies.
Financial results for the quarter ended
"Alnylam's strong balance sheet and overall financial position allow us
to build our commercial capabilities in preparation for anticipated
product launches in the
Cash and Investments
GAAP and Non-GAAP Net Loss
The net loss according to accounting principles generally accepted in the
The non-GAAP net loss for the third 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
Alnylam remains on track to end 2017 with greater than
Conference Call Information
Management will provide an update on the Company and discuss third 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 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 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 600 people 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.
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, cemdisiran and lumasiran, its expectations regarding the timing of clinical studies and the presentation of clinical data, including for studies for fitusiran, givosiran, inclisiran, cemdisiran and lumasiran, its expectations regarding the potential of its ESC+ GalNAc-siRNA conjugate platform, its expectations regarding the potential filing of an NDA for patisiran by the end of 2017 and an MAA shortly thereafter, its expected transition to commercial operations in mid-2018 if patisiran is approved, its plans for the development and commercialization of RNAi therapeutics for infectious diseases with Vir Biotechnology, its expected cash, cash equivalents, fixed income 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
Three Months Ended
Nine Months Ended
|Net revenues from collaborators||$||17,096||$||13,651||$||51,988||$||29,705|
|Research and development||95,252||97,936||272,863||277,381|
|General and administrative||47,644||22,391||131,910||61,478|
|Total operating expenses||142,896||120,327||404,773||338,859|
|Loss from operations||(125,800||)||(106,676||)||(352,785||)||(309,154||)|
|Other income (expense):|
|Other (expense) income||(433||)||401||(3,863||)||5,871|
|Total other income||2,863||2,605||4,138||11,980|
|Net loss per common share - basic and diluted||$||(1.34||)||$||(1.21||)||$||(3.93||)||$||(3.48||)|
Weighted-average common shares used to compute basic and diluted net loss per common share
Unrealized gain (loss) on marketable securities, net of tax
Reclassification adjustment for realized loss (gain) on marketable securities included in net loss
Three Months Ended
Nine Months Ended
Reconciliation of GAAP to
|Less: Stock-based compensation expenses||(15,090||)||(9,341||)||(37,035||)||(32,974||)|
|Reconciliation of GAAP to Non-GAAP General and administrative:|
|GAAP General and administrative||$||47,644||$||22,391||$||131,910||$||61,478|
|Less: Stock-based compensation expenses||(10,865||)||(6,240||)||(28,667||)||(21,903||)|
|Non-GAAP General and administrative||$||36,779||$||16,151||$||103,243||$||39,575|
|Reconciliation of GAAP to Non-GAAP Operating expenses:|
|GAAP Operating expenses||$||142,896||$||120,327||$||404,773||$||338,859|
|Less: Stock-based compensation expenses||(25,955||)||(15,581||)||(65,702||)||(54,877||)|
|Non-GAAP Operating expenses||$||116,941||$||104,746||$||339,071||$||283,982|
|Reconciliation of GAAP to Non-GAAP Net loss:|
|GAAP Net loss||$||(122,937||)||$||(104,071||)||$||(348,647||)||$||(297,174||)|
|Add: Stock-based compensation expenses||25,955||15,581||65,702||54,877|
|Non-GAAP Net loss||$||(96,982||)||$||(88,490||)||$||(282,945||)||$||(242,297||)|
|Reconciliation of GAAP to Non-GAAP Net loss per common share-basic and diluted:|
|GAAP Net loss per common share - basic and diluted||$||(1.34||)||$||(1.21||)||$||(3.93||)||$||(3.48||)|
|Add: Stock-based compensation expenses||0.28||0.18||0.74||0.65|
|Non-GAAP Net loss per common share - basic and diluted||$||(1.06||)||$||(1.03||)||$||(3.19||)||$||(2.83||)|
|Cash, cash equivalents and fixed income marketable securities||$||999,837||$||942,601|
|Billed and unbilled collaboration receivables||14,644||23,334|
|Prepaid expenses and other assets||31,901||32,303|
|Property, plant and equipment, net||161,899||114,572|
|Accounts payable, accrued expenses and other liabilities||$||63,341||$||99,650|
|Total deferred revenue||76,888||82,932|
|Total deferred rent||9,217||10,007|
Total stockholders' equity (92.4 million and 85.9 million common
shares issued an
|Total liabilities and stockholders' equity||$||1,358,281||$||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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