Nov 05, 2014 Press Release for Alnylam
Alnylam Pharmaceuticals Reports Third Quarter 2014 Financial Results and Highlights Recent Period Progress
Nov 05, 2014
- Reported Six-Month Clinical Data from Phase 2 Open-Label Extension (OLE) Study with Patisiran, Showing Tolerability, Sustained Transthyretin (TTR) Knockdown, and Promising Initial Evidence for Stabilization of Neuropathy Progression -
- Completed Phase 2 Enrollment and Initiated Phase 2 OLE Study with Revusiran (ALN-TTRsc), and Successfully Completed Non-Clinical Chronic Toxicology Studies to Enable Start of Phase 3 Trial -
- Continued Enrollment in Phase 1 Hemophilia Clinical Trial with ALN-AT3 -
- Filed Clinical Trial Application (CTA) for ALN-PCSsc, Now Approved -
- Advanced Additional Development Programs, Including ALN-CC5 for Complement-Mediated Diseases -
- Maintained Strong Balance Sheet with
"During the third quarter and recent period we continued advancing RNAi
therapeutics through the clinic and toward the market. Notably, we
reported six-month clinical data from our Phase 2 open-label extension
study with patisiran, showing what we believe to be promising initial
results. Indeed, with the important caveat that this is an open-label
study in a small number of patients, we are encouraged by what we
believe to be evidence for possible stabilization of neuropathy
progression after the first six months of treatment. We believe these
data will be increasingly meaningful as we monitor neuropathy
progression in patisiran-treated patients over time. In the meantime, we
are pleased with the pace of enrollment in our Phase 3 APOLLO trial of
patisiran in patients with polyneuropathy, where we aim to obtain
definitive evidence for patisiran safety and efficacy in a double-blind,
placebo-controlled randomized trial," said
"In addition to these advancements on the clinical front, we have made
strong progress with our pre-clinical programs. First, we presented new
data with ALN-CC5, an investigational RNAi therapeutic in development
for the treatment of complement-mediated diseases, showing potent,
clamped knockdown of serum C5 in non-human primate studies, as well as
promising results in pre-clinical disease models. We remain on track to
file a CTA for ALN-CC5 by the end of this year, with initial clinical
results expected in mid-2015. We also made excellent progress in other
development stage programs, including ALN-AS1 for hepatic porphyrias,
ALN-AAT for liver disease associated with alpha-1 antitrypsin
deficiency, and ALN-HBV for hepatitis B virus infection," said
Cash,
At
GAAP Net Loss
The net loss according to accounting principles generally accepted in
the U.S. (GAAP) for the third quarter of 2014 was
Revenues
Revenues were
Research and Development Expenses
Research and development (R&D) expenses were
General and Administrative Expenses
General and administrative (G&A) expenses were
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
2014 Financial Guidance
The company now expects that its cash, cash equivalents and total
marketable securities balance will be greater than
"Alnylam continues to maintain a very strong balance sheet, ending the
third quarter with
Third Quarter 2014 and Recent Significant Corporate Highlights
Key Pipeline Highlights
-
Reported Six-Month Clinical Data from Patisiran Phase 2 Open-Label
Extension (OLE) Study in Patients with Familial Amyloidotic
Polyneuropathy (FAP).
-
At the American Neurological Association's 2014 Annual Meeting
held
October 12 - 14, the company presented clinical results from the ongoing OLE study with patisiran. Results showed a mean 0.95 point decrease in modified Neuropathy Impairment Score (mNIS+7) at six months in 19 patients with mNIS+7 data available for the current analysis. This decrease in neuropathy progression compares favorably with the 7 to 10 point increase in mNIS+7 at six months that can be estimated from historical data sets in untreated FAP patients with similar baseline characteristics (Adams et al., International Symposium on Amyloidosis,April 2014 ; Berk et al., JAMA 310: 26588-67, 2013;Tafamidis European Medicines Agency Assessment Report, 2011). In addition, patisiran treatment achieved a sustained mean serum TTR knockdown at the 80% target level for over nine months, with an up to 89.6% knockdown achieved between doses. Patisiran was found to be generally well tolerated in this study out to one year of therapy, with no drug-related serious adverse events to date, and all 27 patients enrolled in the study continue to receive drug treatment. Alnylam plans to report results from the ongoing OLE study at least once a year. - Alnylam continues to enroll patients in its APOLLO Phase 3 trial, with over 26 sites in nine countries now open and active. The Phase 3 trial is intended to demonstrate the efficacy and safety of patisiran in support of marketing authorization in countries around the world.
-
At the American Neurological Association's 2014 Annual Meeting
held
-
Continued Advancement of Revusiran (re-VOO-si-ran; ALN-TTRsc), a
Subcutaneously Administered RNAi Therapeutic Targeting TTR in
Development for the Treatment of Patients with TTR-Mediated Cardiac
Amyloidosis.
-
The company announced earlier today that it has completed
enrollment in its pilot Phase 2 study of revusiran. This study was
aimed at evaluating the tolerability and preliminary clinical
activity of revusiran in patients with familial amyloidotic
cardiomyopathy (FAC) - which is caused by autosomal dominant
mutations in the TTR gene - or senile systemic amyloidosis (SSA) -
which is caused by idiopathic accumulation of wild-type TTR in the
heart. In the study, revusiran was administered for a period of 6
weeks. Alnylam expects to present initial results from the Phase 2
study at a meeting to be held during the
American Heart Association meeting in November. The company also announced today that its Phase 2 OLE study with revusiran is now open for enrollment. The OLE study will evaluate the safety and tolerability of long-term dosing with revusiran for up to two years, and will also measure effects of treatment toward a number of clinical endpoints, including mortality, hospitalization, and 6-minute walk distance (6MWD), in addition to cardiac biomarkers. The company intends to report clinical data from this study about once per year, with initial data in 2015. Alnylam also announced today that it has successfully completed discussions with both theFood and Drug Administration (FDA) andEuropean Medicines Agency (EMA) regarding design of a Phase 3 study with revusiran in TTR cardiac amyloidosis. Alnylam remains on track to initiate the Phase 3 study by the end of 2014. -
At the 10th Annual Meeting of the
Oligonucleotide Therapeutics Society (OTS) heldOctober 12 - 15, the company presented updated data from the Phase 1 trial of revusiran performed in healthy volunteers. The new results included TTR knockdown data for an additional cohort (N=6) receiving doses of 7.5 mg/kg, showing a mean max TTR knockdown of 87.9% and a maximum TTR knockdown of 96.2%. Revusiran was generally well tolerated with self-limiting mild or moderate injection site reactions as the most frequent adverse event, consistent with results from previous study cohorts. - In addition, the company presented results from non-clinical chronic Good Laboratory Practice (GLP) toxicology studies with revusiran. Data from 6-month toxicology studies in rats and 9-month toxicology studies in non-human primates (NHPs) showed that chronic dosing with revusiran was generally well tolerated, with a "no observed adverse effect level" (NOAEL) of greater than or equal to 30 mg/kg in the rat and, pending final histopathology results, greater than or equal to 200 mg/kg in NHPs. The completion of these toxicology studies confirms the wide therapeutic index for revusiran and provides encouraging results for the company's GalNAc-siRNA conjugate platform overall. It also enables the advancement of revusiran into a Phase 3 clinical trial, and supports the potential filing of the drug candidate's New Drug Application (NDA).
- Finally, Alnylam announced that it has initiated its DISCOVERY trial, a screening study examining the prevalence of TTR mutations in patients suspected of having cardiac amyloidosis. With this study, Alnylam aims to identify and facilitate the diagnosis of FAC. Results from DISCOVERY are expected to determine the frequency of TTR mutations in patients with clinical and/or radiological findings of cardiac amyloidosis and identify FAC patients who may be eligible for clinical trials of novel investigational therapeutics such as revusiran.
-
The company announced earlier today that it has completed
enrollment in its pilot Phase 2 study of revusiran. This study was
aimed at evaluating the tolerability and preliminary clinical
activity of revusiran in patients with familial amyloidotic
cardiomyopathy (FAC) - which is caused by autosomal dominant
mutations in the TTR gene - or senile systemic amyloidosis (SSA) -
which is caused by idiopathic accumulation of wild-type TTR in the
heart. In the study, revusiran was administered for a period of 6
weeks. Alnylam expects to present initial results from the Phase 2
study at a meeting to be held during the
-
Continued Advancement of ALN-AT3, an RNAi Therapeutic Targeting
Antithrombin (AT) in Development for the Treatment of Hemophilia and
Rare Bleeding Disorders (RBD). Alnylam continues to enroll people
with hemophilia in its Phase 1 trial with ALN-AT3. In
May 2014 , the company transitioned to the multiple ascending dose (MAD) "Part B" of the study, which is designed as an open-label, multi-dose, dose-escalation study enrolling up to 18 people with moderate-to-severe hemophilia A or B. The primary objective of this part of the study is to evaluate the safety and tolerability of multiple doses, specifically three doses, of subcutaneously administered ALN-AT3 in hemophilia subjects. Secondary objectives include assessment of clinical activity as determined by knockdown of circulating AT levels and increase in thrombin generation at pharmacologic doses of ALN-AT3. Alnylam intends to present initial clinical results from the Phase 1 study at the 56thAmerican Society of Hematology (ASH) Annual Meeting being heldDecember 6 - 9, 2014 inSan Francisco . Specifically, the company expects the presentation to include initial tolerability and clinical activity data from the first lowest-dose, dose-escalation cohort of subjects with hemophilia, in addition to data from the one cohort enrolled in the single ascending dose (SAD) phase performed in healthy volunteer subjects. In addition, the company expects to present initial tolerability data from the second dose-escalation cohort of subjects with hemophilia. Alnylam expects to provide a more complete presentation of the ALN-AT3 Phase 1 data in mid-2015.
-
Presented New Pre-Clinical Data with ALN-CC5, a Subcutaneously
Administered RNAi Therapeutic Targeting Complement Component C5 in
Development for the Treatment of Complement-Mediated Diseases.
-
At the 25th
International Complement Workshop heldSeptember 14 - 18, the company presented new data in NHPs showing that with either a monthly or twice-monthly subcutaneous dosing regimen, ALN-CC5 administration led to potent, clamped knockdown of serum C5 of up to 98.7% and inhibition of complement activity up to 96.8% for a period of up to 100 days from an ongoing study. - In addition, new pre-clinical results were reported for ALN-CC5 in a rat model of membranous nephropathy (MN). As compared with placebo, ALN-CC5 administration was associated with a statistically significant (p < 0.05) reduction by over 70% in the proteinuria associated with complement-mediated renal damage. These results demonstrate pre-clinical efficacy for ALN-CC5 in the setting of renal impairment, where clinical results with anti-C5 mAb therapy have shown limited effects.
- Finally, new data in a mouse anti-collagen antibody induced arthritis (CAIA) model showed that RNAi-mediated C5 knockdown preserves joint histology and prevented C3 deposition as effectively as an anti-C5 mAb. These results demonstrate that knockdown of liver-derived C5 is sufficient to achieve a therapeutic effect, and show the absence of a significant role for local complement production in this disease model.
- Alnylam remains on track to file its ALN-CC5 Clinical Trial Application (CTA) in late 2014 and expects to present initial clinical results in mid-2015.
-
At the 25th
-
Filed a CTA, Now Approved, to Initiate a Phase 1 Study for
ALN-PCSsc, a Subcutaneously Administered RNAi Therapeutic Targeting
PCSK9 in Development for the Treatment of Hypercholesterolemia.
- As per the filed CTA, the planned Phase 1 trial of ALN-PCSsc will be conducted as a randomized, single-blind, placebo-controlled, single- and multi-dose, dose-escalation study. The study is designed to enroll up to 76 healthy volunteer subjects with elevated baseline LDL-C (≥ 100 mg/dL), with subjects randomized 3:1, drug:placebo. The study will be performed in two phases: a single ascending dose (SAD) phase and a multiple dose (MD) phase. In the MD phase, subjects will receive two doses of either ALN-PCSsc or placebo administered four weeks apart. The MD phase will also include subjects both on and off statin co-medication. The primary objective of the Phase 1 study is to evaluate the safety and tolerability of ALN-PCSsc. Secondary objectives include assessment of clinical activity as determined by knockdown of plasma PCSK9 levels and serum LDL-C levels, as well as pharmacokinetics of ALN-PCSsc. Following approval of the CTA, Alnylam expects to initiate this study in late 2014 or early 2015, with data expected to be reported in mid-2015.
- ALN-PCSsc becomes the company's third GalNAc-siRNA conjugate to enter a clinical development stage, and the second program using the company's Enhanced Stabilization Chemistry (ESC)-GalNAc technology that provides enhanced potency and durability with a wide therapeutic window.
-
The company announces today that the CTA has been approved by the
Medicines and Healthcare Products Regulatory Agency (MHRA) in theUnited Kingdom . - Alnylam is collaborating with The Medicines Company on the advancement of ALN-PCSsc.
-
Advanced ALN-AS1, a Subcutaneously Administered RNAi Therapeutic
Targeting Aminolevulinic Acid Synthase-1 (ALAS-1) in Development for
the Treatment of Hepatic Porphyrias, Toward the Clinic.
- Alnylam remains on track to file an Investigational New Drug (IND) application, or IND equivalent, in late 2014 or early 2015.
-
In addition, Alnylam and collaborators from the
American Porphyria Consortium and The European Porphyria Network have initiated the EXPLORE trial, a prospective observational study of patients with hepatic porphyrias suffering from recurrent attacks. With this study, Alnylam and clinical investigators aim to learn more about the clinical course, management, and disease burden of patients. Data obtained from EXPLORE are also expected to assist the design of clinical trials with ALN-AS1.
-
Advanced Additional Development Stage Programs.
- Alnylam continued its development efforts on ALN-AAT, a subcutaneously administered RNAi therapeutic targeting alpha-1-antitrypsin (AAT) in development for the treatment of liver disease associated with AAT deficiency. The company remains on track to file an IND or IND equivalent for this program in mid-2015.
- Alnylam continued its development efforts on ALN-HBV, a subcutaneously administered RNAi therapeutic in development for the treatment of hepatitis B virus (HBV) infection. The company remains on track to select a Development Candidate (DC) by the end of 2014 and to file an IND or IND equivalent for this program around the end of 2015.
-
Advanced New Genetic Medicine Programs.
- Broadened Pipeline with ALN-AGT, a Subcutaneously Administered RNAi Therapeutic Targeting Angiotensinogen (AGT) in Development for the Treatment of Hypertensive Disorders of Pregnancy (HDP), Including Preeclampsia.
- Advanced ALN-GO1, an RNAi Therapeutic Targeting Glycolate Oxidase (GO) in Development for the Treatment of Primary Hyperoxaluria Type 1 (PH1), as New Program.
- Regulus Therapeutics Announced Positive Interim Clinical Data with RG-101, Providing Additional Clinical Proof-of-Concept for Alnylam's Proprietary GalNAc-Conjugate Delivery Technology. RG-101 is a GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment of hepatitis C virus (HCV) infection. GalNAc conjugates are a proprietary Alnylam delivery technology and are designed to achieve targeted delivery of RNA therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor. As reported by Regulus, interim results from the ongoing clinical study demonstrated that treatment with a single subcutaneous dose of 2 mg/kg of RG-101 as monotherapy resulted in significant and sustained reductions in HCV RNA, as well as a mean viral load reduction of 4.1 log10 at day 29. In addition, RG-101 was reported to be well tolerated in the study. Alnylam owns approximately 12% of Regulus' outstanding common stock, and is eligible to receive specified sublicense income for certain collaborative agreements entered into by Regulus, as well as royalties on net sales of certain products developed by Regulus or its collaborators.
Business and Organizational Highlights
-
Strengthened Intellectual Property (IP) Estate .-
Alnylam received a Notice of Allowance from
the United States Patent and Trademark Office (USPTO) for a new patent broadly covering conjugate-based delivery of RNA therapeutics. The Manoharan et al. patent application 13/693,478, which has now issued as US 8,828,956, includes claims directed to compositions including those comprising a modified RNA agent linked to a biantennary or triantennary ligand. Specifically, the now issued patent includes claims that broadly cover single-stranded or double-stranded, chemically modified RNA therapeutics conjugated with an N-acetylgalactosamine (GalNAc) ligand independent of length, sequence, or disease target. - In addition, the company received two new Notices of Allowance from the USPTO for patent applications from the Tuschl II family. The Tuschl et al. patent application 13/725,262 includes newly allowed claims directed to compositions that mediate RNAi, and comprising a double-stranded molecule with up to 25 base pairs and at least one nucleotide analogue. The Tuschl et al. patent application 12/591,829, which has now issued as US 8,853,384, includes claims directed to compositions comprising a double-stranded molecule of between 19 and 52 nucleotides in each strand with various end configurations, including a so-called "3' overhang" on the antisense strand.
-
Alnylam received a Notice of Allowance from
-
Expanded Board of Directors and Management Team.
-
Alnylam appointed
Amy W. Schulman to its Board of Directors. Ms. Schulman is the former Executive Vice President and General Counsel of Pfizer Inc., and served as the Business Unit Lead for Pfizer's Consumer Healthcare business. She currently serves on the faculty of Harvard Business School as a Senior Lecturer. In addition, she is a Venture Partner atPolaris Partners and the CEO of Arsia Therapeutics, one of Polaris' early stage portfolio companies. -
In addition, Alnylam expanded its Management Team with the
appointments of
Karen Anderson as Senior Vice President, Chief Human Resources Officer;Pushkal Garg , M.D., as Senior Vice President, Clinical Development;Laurie Keating as Senior Vice President, General Counsel, and Secretary; andMarko Kozul , M.D., as Vice President, Strategy.
-
Alnylam appointed
Conference Call Information
Management will provide an update on the company, discuss third quarter
2014 results, and discuss expectations for the future via conference
call on
About RNAi
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 Conjugates
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.
About
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 with a core
focus on RNAi therapeutics as genetic medicines, including programs as
part of the company's "Alnylam 5x15™" product strategy. Alnylam's
genetic medicine programs are RNAi therapeutics directed toward
genetically defined targets for the treatment of serious,
life-threatening diseases with limited treatment options for patients
and their caregivers. These include: patisiran (ALN-TTR02), an
intravenously delivered RNAi therapeutic targeting transthyretin (TTR)
for the treatment of TTR-mediated amyloidosis (ATTR) in patients with
familial amyloidotic polyneuropathy (FAP); revusiran (ALN-TTRsc), a
subcutaneously delivered RNAi therapeutic targeting TTR for the
treatment of ATTR in patients with TTR cardiac amyloidosis, including
familial amyloidotic cardiomyopathy (FAC) and senile systemic
amyloidosis (SSA); ALN-AT3, an RNAi therapeutic targeting antithrombin
(AT) for the treatment of hemophilia and rare bleeding disorders (RBD);
ALN-CC5, an RNAi therapeutic targeting complement component C5 for the
treatment of complement-mediated diseases; ALN-AS1, an RNAi therapeutic
targeting aminolevulinic acid synthase-1 (ALAS-1) for the treatment of
hepatic porphyrias including acute intermittent porphyria (AIP);
ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of
hypercholesterolemia; ALN-AAT, an RNAi therapeutic targeting alpha-1
antitrypsin (AAT) for the treatment of AAT deficiency-associated liver
disease; ALN-HBV, an RNAi therapeutic targeting the hepatitis B virus
(HBV) genome for the treatment of HBV infection; ALN-TMP, an RNAi
therapeutic targeting TMPRSS6 for the treatment of beta-thalassemia and
iron-overload disorders; ALN-ANG, an RNAi therapeutic targeting
angiopoietin-like 3 (ANGPTL3) for the treatment of genetic forms of
mixed hyperlipidemia and severe hypertriglyceridemia; ALN-AC3, an RNAi
therapeutic targeting apolipoprotein C-III (apoCIII) for the treatment
of hypertriglyceridemia; ALN-AGT, an RNAi therapeutic targeting
angiotensinogen (AGT) for the treatment of hypertensive disorders of
pregnancy (HDP), including preeclampsia; ALN-GO1, an RNAi therapeutic
targeting glycolate oxidase (GO) for the treatment of primary
hyperoxaluria type 1 (PH1); and other programs yet to be disclosed. As
part of its "Alnylam 5x15" strategy, as updated in early 2014, the
company expects to have six to seven genetic medicine product candidates
in clinical development - including at least two programs in Phase 3 and
five to six programs with human proof of concept - by the end of 2015.
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, and The Medicines Company.
In early 2014, Alnylam and Genzyme, a Sanofi company, formed a
multi-product geographic alliance on Alnylam's genetic medicine programs
in the rare disease field. Specifically, Alnylam will lead development
and commercialization of programs in
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 5x15" product strategy,
Alnylam's views with respect to the potential for RNAi therapeutics,
including patisiran (ALN-TTR02), revusiran (ALN-TTRsc), ALN-AT3,
ALN-CC5, ALN-PCSsc, ALN-AS1, ALN-AAT, ALN-HBV, ALN-AGT, 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 revusiran, ALN-CC5, ALN-AS1,
ALN-AAT, ALN-HBV, and ALN-GO1, 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, Genzyme's and The Medicines
Company's participation in the development and commercialization of RNAi
therapeutics with Alnylam, Alnylam's expected cash position as of
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UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS |
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|
(In thousands, except per share amounts) |
||||||||||||
|
Three Months Ended |
Nine Months Ended |
|||||||||||
| 2014 | 2013 | 2014 | 2013 | |||||||||
| Net revenues from collaborators | $ | 10,972 | $ | 8,991 | $ | 26,542 | $ | 36,320 | ||||
| Operating expenses: | ||||||||||||
|
Research and development (1) |
46,273 | 34,457 | 134,703 | 80,851 | ||||||||
| In-process research and development | — | — | 220,766 | — | ||||||||
| General and administrative (1) | 9,898 | 6,768 | 30,341 | 18,819 | ||||||||
| Total operating expenses | 56,171 | 41,225 | 385,810 | 99,670 | ||||||||
| Loss from operations |
(45,199) |
(32,234) | (359,268) | (63,350) | ||||||||
| Other income (expense): | ||||||||||||
| Interest income | 753 | 290 | 1,779 | 784 | ||||||||
| Other income (expense) | 524 | (12) | 365 | (18) | ||||||||
| Total other income | 1,277 | 278 | 2,144 | 766 | ||||||||
| Loss before income taxes | (43,922) | (31,956) | (357,124) | (62,584) | ||||||||
| (Provision for) benefit from income taxes | (67) | 2,270 | 18,118 | 5,716 | ||||||||
| Net loss | $ | (43,989) | $ | (29,686) | $ | (339,006) | $ | (56,868) | ||||
| Net loss per common share - basic and diluted | $ | (0.58) | $ | (0.48) | $ | (4.62) | $ | (0.93) | ||||
| Weighted average common shares used to compute basic and diluted net loss per common share | 76,408 | 62,416 | 73,375 | 61,103 | ||||||||
| Comprehensive loss: | ||||||||||||
| Net loss | $ | (43,989) | $ | (29,686) | $ | (339,006) | $ | (56,868) | ||||
| Unrealized (loss) gain on marketable securities, net of tax | (6,230) | (962) | (3,964) | 11,506 | ||||||||
|
Reclassification adjustment for realized gain on marketable securities included in net loss |
(567) | — | (567) | — | ||||||||
| Comprehensive loss | $ | (50,786) | $ | (30,648) | $ | (343,537) | $ | (45,362) | ||||
| (1) Non-cash stock-based compensation expenses included in operating expenses are as follows: | ||||||||||||
| Research and development | $ | 3,781 | $ | 6,805 | $ | 10,019 | $ | 11,092 | ||||
| General and administrative | 2,571 | 2,040 | 9,604 | 4,205 | ||||||||
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UNAUDITED GAAP TO NON-GAAP RECONCILIATION: NET LOSS AND NET LOSS PER SHARE |
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(In thousands, except per share amounts) |
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Three Months Ended |
Nine Months Ended |
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| 2014 | 2013 | 2014 | 2013 | |||||||||
| GAAP net loss | $ | (43,989) | $ | (29,686) | $ | (339,006) | $ | (56,868) | ||||
| Adjustment: | ||||||||||||
| In-process research and development | — | — | 220,766 | — | ||||||||
| Non-GAAP net loss | $ | (43,989) | $ | (29,686) | $ | (118,240) | $ | (56,868) | ||||
| GAAP net loss per common share - basic and diluted | $ | (0.58) | $ | (0.48) | $ | (4.62) | $ | (0.93) | ||||
| Adjustment (as detailed above) | — | — | 3.01 | — | ||||||||
| Non-GAAP net loss per common share - basic and diluted | $ | (0.58) | $ | (0.48) | $ | (1.61) | $ | (0.93) | ||||
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 nine
months ended
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UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS |
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(In thousands, except share amounts) |
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| 2014 | 2013 | |||||
| Cash, cash equivalents and total marketable securities | $ | 915,182 | $ | 350,472 | ||
| Billed and unbilled collaboration receivables | 384 | 4,248 | ||||
| Prepaid expenses and other current assets | 10,699 | 3,910 | ||||
| Property and equipment, net | 18,806 | 16,448 | ||||
| Investment in equity securities of Regulus Therapeutics Inc. | 41,286 | 45,452 | ||||
| Total assets | $ | 986,357 | $ | 420,530 | ||
| Accounts payable, accrued expenses and other liabilities | $ | 26,356 | $ | 20,056 | ||
| Total deferred revenue | 50,298 | 126,090 | ||||
| Total deferred rent | 5,225 | 4,037 | ||||
|
Total stockholders' equity (76.7 million and 63.7 million common
shares issued and outstanding and at |
904,478 | 270,347 | ||||
| Total liabilities and stockholders' equity | $ | 986,357 | $ | 420,530 | ||
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
or
Vice President, Finance and Treasurer
Source:
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Chief Corporate Communications Officer media@alnylam.com 617-682-4340
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