Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2014 Financial Results and Highlights Recent Period Progress
- Introduced Pipeline Growth Strategy for RNAi Therapeutics in Three Strategic Therapeutic Areas, or "STArs," and Launched "Alnylam 2020" Guidance for Advancement and Commercialization of RNAi Therapeutics -
- Presented Positive Data from Multiple Clinical Programs, Including Initial Evidence of Potential Disease Modifying Effects with Patisiran and ALN-AT3 -
- Advanced Revusiran into ENDEAVOUR Phase 3 Trial and Added Three New Programs into Clinical Stages -
- Maintained Strong Balance Sheet with
"Alnylam made excellent progress in the fourth quarter of 2014 and the
recent period, as we continued advancing RNAi therapeutics in clinical
studies. Amongst other highlights, we reported clinical data from our
patisiran and ALN-AT3 programs showing what we believe to be early
evidence for the translation of RNAi-mediated knockdown into potential
clinical benefit for patients. We also executed on our pipeline goals,
with strong enrollment in our patisiran APOLLO Phase 3 study and
advancement of revusiran into our ENDEAVOUR Phase 3 trial. In addition,
we filed three Clinical Trial Applications for ALN-PCSsc, ALN-CC5, and
ALN-AS1, and have initiated Phase 1 studies for two of those programs,"
"Alnylam continues to maintain a very strong balance sheet, ending 2014 with approximately $882 million in cash," said Michael Mason, Vice President, Finance & Treasurer. "We also further strengthened our balance sheet earlier this year with a public offering and concurrent private placements from Genzyme that resulted in net proceeds of approximately $567 million. This financing results in a balance sheet that allows us to invest in a broad pipeline of RNAi therapeutics across all three STArs, which we believe should enable us to achieve our ‘Alnylam 2020' guidance. As for financial guidance this year, we expect to end 2015 with greater than $1.2 billion in cash."
Fourth Quarter 2014 and Recent Significant Corporate Highlights
- Introduced pipeline growth strategy for RNAi therapeutics in three 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 dyslipidemia, hypertension, non-alcoholic steatohepatitis (NASH), and type 2 diabetes; and Hepatic Infectious Disease, with a pipeline of RNAi therapeutics that address the major global health challenges of hepatic infectious diseases.
- Launched "Alnylam 2020" Guidance for advancement and intended commercialization of RNAi Therapeutics. 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.
Advanced pipeline programs in Genetic Medicine STAr.
Advanced investigational RNAi therapeutic programs for the
treatment of transthyretin (TTR)-mediated amyloidosis (ATTR).
- Continued enrollment in APOLLO Phase 3 study of patisiran in ATTR patients with Familial Amyloidotic Polyneuropathy (FAP)
- Reported positive six-month clinical data from patisiran Phase 2 open-label extension (OLE) study, showing tolerability, sustained TTR knockdown, and promising initial evidence for potential stabilization of neuropathy progression.
- Initiated ENDEAVOUR Phase 3 study with revusiran. ENDEAVOUR is a randomized, double-blind, placebo-controlled, global study designed to evaluate the efficacy and safety of revusiran in ATTR patients with Familial Amyloidotic Cardiomyopathy (FAC).
- Presented positive initial Phase 2 data with revusiran, with up to 98.2% knockdown of serum TTR; revusiran administration was found to be generally well tolerated in patients with advanced cardiac disease.
- Initiated Phase 2 OLE study with revusiran to evaluate tolerability and clinical activity with long-term dosing for up to two years.
- Reported positive initial results from Phase 1 trial of ALN-AT3, including initial evidence for the potential correction of the hemophilia phenotype with an up to 334% increase in thrombin generation and marked improvement in whole blood clotting.
- Initiated Phase 1/2 trial with ALN-CC5. The trial is being conducted initially in normal human volunteers, and then in patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Filed Clinical Trial Application (CTA) to initiate a Phase 1 trial with ALN-AS1 in acute intermittent porphyria (AIP) patients who are asymptomatic "high excreters" (ASHE), and then in AIP patients who experience recurrent porphyria attacks.
- Expanded Genetic Medicine pipeline with ALN-GO1, an investigational RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of Primary Hyperoxaluria Type 1 (PH1).
- Advanced investigational RNAi therapeutic programs for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR).
Advanced pipeline programs in Cardio-Metabolic Disease STAr
- Initiated Phase 1 trial with ALN-PCSsc in normal human volunteers with elevated LDL-C at baseline.
Advanced pipeline programs in Hepatic Infectious Disease STAr
- Selected Development Candidate (DC) for ALN-HBV, showing an up to 3.9 log10 knockdown of hepatitis surface antigen (HBsAg) after a single subcutaneous dose in a rodent model of HBV infection.
- Expanded Hepatic Infectious Disease pipeline with ALN-HDV for Hepatitis Delta Virus (HDV) infection and ALN-PDL for chronic liver infections.
- Formed new agreement with Isis Pharmaceuticals, extending the companies' decade-long alliance to lead the development and commercialization of RNA therapeutics.
Completed successful public offering of common stock, with concurrent
private placements from Genzyme, totaling
$567 millionin net proceeds.
Announced changes to Board of Directors and Management Team
Michael W. Bonneyto its Board of Directors.
Alnylam announces today certain promotions and appointments,
Akshay Vaishnaw, M.D., Ph.D. to the position of Executive Vice President of Research & Development and Chief Medical Officer from the position of Executive Vice President, Chief Medical Officer.
Rachel Meyers, Ph.D. to the position of Senior Vice President of Research from the position of Vice President of Research & RNAi Lead Development.
Kevin Fitzgerald, Ph.D. to the position of Vice President of Research from the position of Senior Director of Research.
Pushkal Garg, M.D. to the position of Senior Vice President, Clinical Development.
Eric Greento the position of Vice President, General Manager, TTR Program.
Marcie Ruddy, M.D. to the position of Vice President, Drug Safety & Pharmacovigilance.
In addition, Alnylam announced the planned departure of
Laurence Reid, Ph.D., who held the position of Senior Vice President, Chief Business Officer.
- Alnylam elected
Upcoming Events in Early and Mid-2015
Alnylam announces today that it plans to present 12-month OLE study
data with patisiran at the
American Academy of Neurology67th Annual Meeting, being held April 18- 25, 2015 in Washington, D.C., in an oral presentation on Tuesday, April 21at 1:30 p.m. ET.
The company also announces today that it plans to present additional
clinical results from the Phase 2 trial of revusiran at the
American College of Cardiology(ACC) 64th Annual Scientific Session & Expo, being held March 14- 16, 2015 in San Diego, California, in a poster presentation on Sunday, March 15at 1:30 p.m. PT( 4:30 p.m. ET).
Also at the ACC meeting, Alnylam plans to present results of a natural
history study of patients with TTR cardiac amyloidosis in a meeting
with clinicians from the
Association of Black Cardiologistson Sunday, March 15.
In addition, during early and mid-2015, Alnylam plans to:
- Present additional data from Phase 1 trial of ALN-AT3 in development for the treatment of hemophilia and rare bleeding disorders
- Present initial data from the Phase 1/2 trial of ALN-CC5 in development for the treatment of complement-mediated diseases
- Initiate Phase 1 trial of ALN-AS1 in development for the treatment of hepatic porphyrias
- Present initial data from Phase 1 trial of ALN-PCSsc in development for the treatment of hypercholesterolemia
- File CTA for ALN-AAT in development for the treatment of alpha-1 antitrypsin (AAT) deficiency-associated liver disease
- Select DC for ALN-GO1 in development for the treatment of PH1
Non-GAAP Net Loss
The non-GAAP net loss for the year ended
GAAP Net Loss
The net loss according to GAAP for the fourth quarter of 2014 was
Research and Development Expenses
Research and development (R&D) expenses were
In 2014, the company incurred a
General and Administrative Expenses
General and administrative (G&A) expenses were
Benefit from (Provision for) Income Taxes
The company had a benefit from income taxes of
Investment in Regulus Therapeutics
The company accounts for its investment in Regulus at fair value by
adjusting the value to reflect fluctuations in Regulus' stock price each
reporting period. At
2015 Financial Guidance
Alnylam expects that its cash, cash equivalents, and total marketable
securities balance will be greater than
Conference Call Information
Management will provide an update on the company, discuss fourth quarter and 2014 results, and discuss expectations for the future via conference call 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 GalNAc Conjugates and Enhanced Stabilization Chemistry (ESC)
GalNAc-siRNA conjugates are a proprietary Alnylam delivery platform and are designed to achieve targeted delivery of RNAi therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor. Alnylam's Enhanced Stabilization Chemistry (ESC) GalNAc-conjugate technology enables subcutaneous dosing with increased potency, durability, and a wide therapeutic index, and is being employed in several of Alnylam's genetic medicine programs, including programs in clinical development.
About LNP Technology
Alnylam has licenses to Tekmira 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 Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist, GlaxoSmithKline, Ascletis, Monsanto, The Medicines Company, and Genzyme, a Sanofi company. In addition, Alnylam holds an equity position in Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 200 peer-reviewed papers, including many in the world's top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, Cell,
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future expectations, plans and prospects, including without limitation, Alnylam's expectations regarding its "Alnylam 2020" guidance, Alnylam's views with respect to the potential for RNAi therapeutics, including patisiran, revusiran, ALN-AT3, ALN-CC5, ALN-PCSsc, ALN-AS1, ALN-AAT, ALN-HBV, and ALN-GO1, its expectations with respect to the timing, execution, and success of its clinical and pre-clinical trials, the expected timing of regulatory filings, including its plan to file IND or IND equivalent applications and/or initiate clinical trials for ALN-AS1 and ALN-AAT, , its expectations regarding reporting of data from its clinical and pre-clinical studies, including its studies for patisiran, revusiran, ALN-AT3, ALN-CC5, and ALN-PCSsc, as well as other research programs and technologies, its plans regarding commercialization of RNAi therapeutics, and Alnylam's expected cash position as of
|UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME (LOSS)|
|(In thousands, except per share amounts)|
Three Months Ended
|Net revenues from collaborators||$||24,019||$||10,847||$||50,561||$||47,167|
|Research and development (1)||55,546||32,106||190,249||112,957|
|In-process research and development||—||—||220,766||—|
|General and administrative (1)||14,185||8,333||44,526||27,152|
|Total operating expenses||69,731||40,439||455,541||140,109|
|Loss from operations||( 45,712)||(29,592)||(404,980)||(92,942)|
|Other income (expense):|
|Other income (expense)||1,452||(29)||1,817||(47)|
|Total other income||2,232||256||4,376||
|Loss before income taxes||(43,480)||(29,336)||(400,604)||(91,920)|
|Benefit from (provision for) income taxes||22,091||(3,021)||40,209||2,695|
|Net loss per common share - basic and diluted||$||(0.28)||$||(0.51)||$||(4.85)||$||(1.45)|
|Weighted average common shares used to compute basic and diluted net loss per common share||76,957||62,909||74,278||61,551|
|Comprehensive income (loss):|
|Unrealized gain (loss) on marketable securities, net of tax||35,091||(7,451)||31,127||4,055|
Reclassification adjustment for realized gain on marketable securities included in net loss
|Comprehensive income (loss)||$||12,188||$||(39,808)||$||(331,349)||$||(85,170)|
(1) Non-cash stock-based compensation expenses included in operating expenses are as follows:
Research and development
General and administrative
|UNAUDITED GAAP TO NON-GAAP RECONCILIATION: NET LOSS AND NET LOSS PER SHARE|
|(In thousands, except per share amounts)|
Three Months Ended
|GAAP net loss||$||(21,389)||$||(32,357)||$||(360,395)||$||(89,225)|
|In-process research and development||—||—||220,766||—|
|Non-GAAP net loss||$||(21,389)||$||(32,357)||$||(139,629)||$||(89,225)|
|GAAP net loss per common share - basic and diluted||$||(0.28)||$||(0.51)||$||(4.85)||$||(1.45)|
|Adjustment (as detailed above)||—||—||2.97||—|
|Non-GAAP net loss per common share - basic and diluted||$||(0.28)||$||(0.51)||$||(1.88)||$||(1.45)|
Use of Non-GAAP Financial Measures
The company supplements its condensed consolidated financial statements
presented on a GAAP basis by providing additional measures that are
considered "non-GAAP" financial measures under applicable
The company evaluates items on an individual basis, and considers both
the quantitative and qualitative aspects of the item, including (i) its
size and nature, (ii) whether or not it relates to the company's ongoing
business operations, and (iii) whether or not the company expects it to
occur as part of its normal business on a regular basis. In the year
|UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS|
|(In thousands, except share amounts)|
|Cash, cash equivalents and total marketable securities||
|Billed and unbilled collaboration receivables||39,937||4,248|
|Prepaid expenses and other current assets||9,739||3,910|
|Deferred tax assets||31,667||—|
|Property and equipment, net||21,740||16,448|
|Investment in equity securities of Regulus Therapeutics Inc.||94,583||45,452|
|Accounts payable and accrued expenses||
|Deferred tax liabilities||31,667||—|
|Total deferred revenue||66,854||126,090|
|Total deferred rent||6,016||4,037|
Total stockholders' equity (77.2 million and 63.7 million common
shares issued and outstanding and at
|Total liabilities and stockholders' equity||
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
Vice President, Investor Relations and Corporate Communications
Vice President, Finance and Treasurer
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