May 08, 2014 Press Release for Alnylam
Alnylam Pharmaceuticals Reports First Quarter 2014 Financial Results and Highlights Recent Period Activities
May 08, 2014
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- Achieved Positive Initial Results from Phase 2 Open-Label Extension (OLE) Study with Patisiran, Showing Sustained Clinical Activity with Transthyretin (TTR) Knockdown at an 80% Target Level and Tolerability with Extended Dosing -
- Initiated Phase 1 Study of ALN-AT3, an RNAi Therapeutic for Treatment of Hemophilia -
- Presented Pre-clinical Data with ALN-PCSsc Supporting Once-Monthly and Possibly Once-Quarterly Subcutaneous Dosing Regimen with an RNAi Therapeutic Targeting PCSK9 for the Treatment of Hypercholesterolemia -
- Selected Development Candidate (DC) for ALN-AAT for Alpha-1 Antitrypsin Deficiency-Associated Liver Disease; Announces Today Selection of DC for ALN-CC5, an RNAi Therapeutic Targeting Complement Component C5 -
- Strengthened Balance Sheet to
"The first quarter and recent period have been game changing for
Alnylam. Specifically, we believe our alliance with Genzyme strengthens
our efforts to bring RNAi therapeutics to patients with rare diseases as
potential breakthrough genetic medicines. The new collaboration
crystallizes Alnylam's strategy to develop and commercialize our
products in
"In addition to highlights noted, we also made advancements with other
pipeline programs and in other business development efforts. First, we
are pleased to announce today that we have selected our DC for ALN-CC5,
an RNAi therapeutic targeting complement C5 for the treatment of
complement-mediated diseases, and are on track to file our IND for this
program in late 2014. In addition, we have named a DC for our ALN-AAT
program, an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the
treatment of AAT deficiency-associated liver disease, with the goal of
filing an IND for this program in mid-2015. As with ALN-AT3 and
ALN-PCSsc, these two programs employ our Enhanced Stabilization
Chemistry (ESC) GalNAc-conjugate technology, which enables subcutaneous
dosing with increased potency, durability, and a wide therapeutic index.
We are very encouraged by the potent knockdown and durability results
emerging from our programs utilizing this improved technology," said
Cash,
At
Non-GAAP Net Loss
The non-GAAP net loss for the first quarter of 2014 was
GAAP Net Loss
The net loss according to accounting principles generally accepted in
the U.S. (GAAP) for the first quarter of 2014 was
Revenues
Revenues were
Research and Development Expenses
Research and development (R&D) expenses were
In the first quarter of 2014, the company incurred a
General and Administrative Expenses
General and administrative (G&A) expenses were
Benefit from Income Taxes
The company had a benefit from income taxes of
"Alnylam's balance sheet has never been stronger, and we have ended the
first quarter with just over
First Quarter 2014 and Recent Significant Corporate Highlights
Key "Alnylam 5x15" and Genetic Medicine Pipeline Highlights
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Continued to Advance Patisiran (ALN-TTR02) in Clinical Development
for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis (ATTR)
in Patients with Familial Amyloidotic Polyneuropathy (FAP).
- Alnylam continues to enroll patients in its APOLLO Phase 3 trial, with over ten sites in four countries currently enrolling. The Phase 3 trial is intended to demonstrate the efficacy and safety of patisiran in support of a New Drug Application (NDA) filing.
-
At the International Symposium on Amyloidosis (ISA) held
April 27 -May 1, 2014 , the company presented multiple updates from its patisiran program. Alnylam presented updated results from its Phase 2 study of patisiran, which was conducted in 29 patients with FAP where patients received two doses of drug administered as an intravenous infusion. Patisiran demonstrated a rapid, dose-dependent, and durable knockdown of serum TTR of up to 96% and an average TTR knockdown of approximately 80%. ALN-TTRsc was generally well tolerated in the Phase 2 study. - The company also presented initial positive results from its Phase 2 open-label extension (OLE) study with patisiran. The OLE study is enrolling patients that were treated in the Phase 2 study and is designed to evaluate the long-term safety and tolerability of patisiran administration; the company expects 27 patients to enroll in the study. The OLE study is measuring a number of clinical endpoints every six months, including the modified composite Neuropathy Impairment Score, termed "mNIS+7;" this score is also the primary endpoint of the Phase 3 APOLLO trial of patisiran in FAP. Preliminary results from 23 patients showed that the TTR knockdown observed following the first dose in the OLE study closely matched TTR knockdown shown in the Phase 2 study, with essentially superimposable pharmacodynamic effects. Further, repeat dosing of patisiran led to a sustained TTR knockdown of approximately 80% through up to day 168, equivalent to up to eight doses of drug, as measured in pre-dose blood samples. These data provide the first clinical evidence of sustained, RNAi-mediated TTR knockdown in FAP patients beyond two doses of patisiran. Repeat dosing of patisiran was found to be well tolerated, with minimal adverse events. In addition, the use of a proprietary, micro-dosing infusion regimen was found to significantly reduce the incidence of infusion-related reactions. Alnylam continues to plan on reporting initial clinical endpoint data from the OLE study in late 2014, where the initial dataset is expected to include the first readout of 6 month mNIS+7 results from approximately 20 patients.
- In addition, Alnylam and collaborators presented results at the ISA meeting of a natural history, cross-sectional analysis study of 283 FAP patients. These findings showed rapid progression of the Neuropathy Impairment Score and correlation with disease severity, providing support for Alnylam's Phase 3 APOLLO trial.
- Also at ISA, the company presented a pre-clinical update from its ATTR program. Alnylam scientists presented data in a mouse disease model confirming that the degree of TTR knockdown was highly correlated with regression of TTR tissue deposits. Further, comparative studies were performed with the TTR stabilizer tafamidis and a TTR-specific antisense oligonucleotide (ASO). In these pre-clinical studies, RNAi therapeutics targeting TTR were shown to have superior pharmacologic profiles, supporting the potential for RNAi therapeutics to emerge as the optimal therapeutic approach for the treatment of ATTR.
-
Finally, Alnylam is announcing today that it has completed
enrollment of healthy volunteers of Japanese descent in a Phase 1
study with patisiran. This study was performed to evaluate the
tolerability, pharmacokinetics, and pharmacodynamics of patisiran
in this population and is intended to support the expansion of the
APOLLO trial to include sites in
Japan .
-
Continued Advancement of ALN-TTRsc, a Subcutaneously Administered
RNAi Therapeutic Targeting TTR for the Treatment of ATTR Patients with
Cardiac Amyloidosis. The company continued enrollment in its Phase
2 study of ALN-TTRsc. This Phase 2 trial is aimed at evaluating the
tolerability and preliminary clinical activity of ALN-TTRsc 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. Based on encouraging enrollment, the
protocol for this study has recently been amended to enroll up to 25
FAC or SSA patients; the previous enrollment target had been 15. The
company still expects to present data from the Phase 2 trial in late
2014. Patients completing the Phase 2 trial will be eligible to
participate in an OLE study for further assessment of general
tolerability and clinical activity with long-term dosing; the
ALN-TTRsc Phase 2 OLE study is on track to be initiated in mid-2014.
Alnylam expects to begin a Phase 3 trial in TTR cardiac amyloidosis
patients by the end of 2014. In addition, the protocol for the
company's Phase 1 trial with ALN-TTRsc has been amended to enroll up
to 76 normal human volunteers; previous target enrollment had been 40
subjects and results
from that study were presented at the
Heart Failure Society of America 17th Annual Scientific Meeting inSeptember 2013 . The amendment was performed to enable determination of a fixed (i.e., non-weight-adjusted) dose regimen in support of the planned Phase 3 trial. Finally, ALN-TTRsc received a positive opinion for Orphan Drug Designation inEurope . -
Initiated Phase 1 Clinical Trial with ALN-AT3, a Subcutaneously
Administered RNAi Therapeutic Targeting Antithrombin (AT) for the
Treatment of Hemophilia and Rare Bleeding Disorders (RBD). ALN-AT3
is the company's first program to enter clinical development employing
Alnylam's Enhanced Stabilization Chemistry (ESC) GalNAc-conjugate
technology, which enables subcutaneous dosing with increased potency,
durability, and a wide therapeutic index. The Phase 1 study is being
conducted in the
U.K. as a single- and multi-dose, dose-escalation study comprised of two parts. Part A is a randomized, single-blind, placebo-controlled, single-dose, dose-escalation study, enrolling up to 24 healthy volunteer subjects. The primary objective of this part of the study is to evaluate the safety and tolerability of a single dose of ALN-AT3, with the potential secondarily to show changes in AT plasma levels at sub-pharmacologic doses. This part of the study is evaluating only low doses of ALN-AT3, with a dose-escalation stopping rule at no more than a 40% knockdown of AT. Part B of the study will be 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 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; thrombin generation is known to be a biomarker for bleeding frequency and severity in people with hemophilia (Dargaud, et al., Thromb Haemost; 93, 475-480 (2005)). The company expects to initiate Part B in mid-2014. Alnylam plans to present initial clinical results from the Phase 1 study by the end of this year, and will also have an update from the ALN-AT3 program at theWorld Federation of Hemophilia meeting taking placeMay 11 - 15, 2014 inMelbourne, Australia . -
Announces Selection of Development Candidate (DC) for ALN-CC5, a
Subcutaneously Administered RNAi Therapeutic Targeting Complement
Component C5. Alnylam announced today that it has selected a DC to
move forward into clinical trials from its ALN-CC5 program. ALN-CC5 is
in development for the treatment of complement-mediated diseases, such
as paroxysmal nocturnal hemoglobinuria (PNH), atypical
hemolytic-uremic syndrome (aHUS), myasthenia gravis, neuromyelitis
optica, amongst many others. The ALN-CC5 DC employs Alnylam's
ESC-GalNAc-conjugate technology. Previously, the company presented pre-clinical
data demonstrating that subcutaneous administration of ALN-CC5 in
non-human primates (NHPs) led to an up to 98% knockdown of serum C5
and an up to 94% inhibition of serum hemolytic activity. This level of
complement activity inhibition exceeds the 80% inhibition threshold
that has been validated as being associated with clinical benefit in
patients with PNH (Hillmen et al.,
N. Engl . J. Med. (2004) 350:552-559). Alnylam intends to present new pre-clinical results from its ALN-CC5 program at theInternational Conference on Complement Therapeutics taking placeJune 6 - 11 inOlympia ,Greece . The company plans to file an Investigational New Drug (IND) application or IND equivalent in late 2014; Alnylam's previous guidance for this program was to file an IND in late 2014 or early 2015. -
Presented New Pre-clinical Data on Subcutaneously Delivered RNAi
Therapeutics for Cardio-Metabolic Disease, including ALN-PCSsc - an
RNAi therapeutic Targeting PCSK9 for the Treatment of
Hypercholesterolemia - and Adds New Program.
- At the Arteriosclerosis, Thrombosis and Vascular Biology (ATVB) 2014 Scientific Sessions, the company presented an update on its cardio-metabolic disease programs. First, Alnylam presented new single-dose data in NHPs for ALN-PCSsc showing robust knockdown of PCSK9 of up to 96% and reduction in LDL-C of up to 77%, in the absence of statin co-administration. A single dose of ALN-PCSsc maintained greater than 50% reduction in LDL-C for over three months, a level of durability that the company believes supports a once-monthly, and possibly once-quarterly, subcutaneous dosing regimen. ALN-PCSsc employs the company's ESC-GalNAc-conjugate technology. The company plans to file an IND or IND equivalent for ALN-PCSsc by late 2014 or early 2015. The ALN-PCS program is partnered with The Medicines Company.
- In addition, the company presented pre-clinical data from a new program: ALN-AC3, an RNAi therapeutic targeting apolipoprotein C-III (apoCIII) for the treatment of hypertriglyceridemia. Alnylam plans to continue to conduct additional pre-clinical work in this program to finalize its DC.
-
Selected DC for ALN-AAT, an RNAi Therapeutic Targeting Alpha-1
Antitrypsin (AAT) for the Treatment of AAT Deficiency-Associated Liver
Disease. New pre-clinical
data were presented in a Late-Breaking Abstract Session at
Digestive Disease Week (DDW), held
May 3 - 6, 2014 inChicago, Illinois . AAT deficiency liver disease is caused by accumulation of mutant AAT protein ("Z-allele" or "Z-AAT") in liver tissue with subsequent liver injury, fibrosis, cirrhosis, and, in some cases, hepatocellular carcinoma. The ALN-AAT DC employs Alnylam's ESC-GalNAc-conjugate technology. In rodent studies, ALN-AAT showed potent and dose-dependent knockdown of serum AAT with a single-dose ED50 of 0.5 mg/kg. In multi-dose rodent experiments, subcutaneous administration at 0.5 mg/kg resulted in approximately 90% knockdown of serum AAT. Finally, initial single-dose NHP results were performed showing dose-dependent knockdown of serum AAT (a surrogate for AAT knockdown in the liver) with an ED50 of less than 3 mg/kg; these results are expected to support a multi-dose ED50 of less than 1 mg/kg, consistent with other ESC-GalNAc-siRNA conjugates. Alnylam plans to initiate IND-enabling studies with the goal of filing an IND or IND equivalent for ALN-AAT in mid-2015, positioning ALN-AAT as the seventh clinical program as part of the company's expanded "Alnylam 5x15" guidance.
Business and Organizational Highlights
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Formed Transformational Alliance with Genzyme for RNAi Therapeutics as Genetic Medicines. Genzyme and Alnylam formed an alliance to accelerate and expand the development and commercialization of RNAi therapeutics across the world. The alliance is structured as a multi-product geographic alliance in the field of rare diseases. Alnylam retains product rights inNorth America andWestern Europe , while Genzyme obtains the right to access Alnylam's current "5x15" and future genetic medicines pipeline in the rest of the world (ROW), including co-development/co-commercialization and/or global product rights for certain programs, including ALN-TTRsc. Genzyme became a major Alnylam shareholder through an upfront purchase of$700 million of newly issued stock at approximately$80 /share, representing an approximately 12% ownership position. This alliance significantly bolstered Alnylam's balance sheet to just over$1 billion in cash, enabling an increased investment in the company's RNAi therapeutics pipeline, and is expected to secure Alnylam's financial independence through to multiple product launches. -
Acquired Investigational RNAi Therapeutic Assets from Merck,
Including Sirna Therapeutics. The acquisition, which includes
Merck's wholly owned subsidiary
Sirna Therapeutics, Inc. , provides Alnylam with intellectual property and RNAi assets including pre-clinical therapeutic candidates, chemistry, and siRNA-conjugate and other delivery technologies. Under the terms of the agreement, in exchange for acquiring the stock ofSirna Therapeutics , Alnylam paid Merck an upfront payment of$175 million in cash and equity ($25 million cash/$150 million in Alnylam common stock). In addition, Merck is eligible to receive milestones and royalties based on advancement of certain pre-clinical candidates discovered by Merck and on Alnylam products covered by theSirna Therapeutics patent estate. -
Genzyme Exercised its Right to Purchase Additional Shares of
Alnylam Common Stock. Genzyme purchased 344,448 unregistered
shares of Alnylam's common stock in accordance with the terms of the
investor agreement between Alnylam and Genzyme, resulting in an
additional payment to Alnylam of approximately
$23.0 million . These shares became available for purchase by Genzyme as a result of Alnylam's issuance of shares of common stock to Merck in connection with Alnylam's acquisition ofSirna Therapeutics . The exercise of this right to purchase the maximum number of additional shares under the terms of the investor agreement allowed Genzyme to maintain its current ownership level of Alnylam common stock of approximately 12%. -
European Patent Office (EPO) Upheld the McSwiggen Patent in Oral
Opposition Proceedings. The EPO upheld the McSwiggen EP 1423406
('406) patent in oral opposition proceedings in
Munich, Germany . The McSwiggen patent estate broadly describes chemical modifications of RNAi therapeutics that the company believes are critical for achieving "drug-like" properties in siRNA, the molecules that mediate RNAi. Specifically, the McSwiggen patent covers chemical modifications in siRNA that are required to achieve potent GalNAc-siRNA conjugates, such as those derived from Alnylam's ESC-GalNAc-conjugate platform. The patent is owned by Alnylam, and was recently obtained through the company's acquisition ofSirna Therapeutics from Merck. The McSwiggen patent family comprises a core component of Alnylam's overall intellectual property (IP) estate for the advancement of RNAi therapeutics as a new class of medicines. -
Expanded Organization , Including Key Appointments to Management Team.-
Alnylam appointed
Pritesh Gandhi , PharmD, R.Ph. to the position of Vice President, Medical Affairs inApril 2014 . Before joining Alnylam, Dr. Gandhi served as Associate Vice President of Global Medical Affairs at Sanofi Oncology, where he spearheaded global publication, medical education, and medical communication strategy development and execution for products across oncology, hematology, and bone marrow and solid organ transplantation. Prior to Sanofi, he worked as Regional Director of Medical Scientific Relations at Alexion Pharmaceuticals, where he managed efforts to bridge the gap between the science of eculizumab and the practice of medicine in the area of paroxysmal nocturnal hemoglobinuria (PNH). Before joining the pharmaceutical industry, Dr. Gandhi held an academic appointment atMassachusetts College of Pharmacy as well as an adjunct position at TheUniversity of Massachusetts Medical School . At that time, he also practiced as Cardiovascular Pharmacotherapy Specialist atUMass Memorial Health Care . -
Alnylam also appointed
Laura Sepp-Lorenzino , Ph.D. to the position of Vice President, Entrepreneur-in-Residence inApril 2014 . Before joining Alnylam, Dr. Sepp-Lorenzino spent 14 years at Merck & Co., having most recently served asExecutive Director and Department Head, RNA Therapeutics Discovery Biology. In this role, she was responsible for identification and optimization of siRNAs and delivery vehicles, advancement of pre-clinical candidates, and development of an siRNA-conjugate platform to expand the repertoire of tissues accessible to in vivo siRNA delivery. Prior to RNAi, Dr. Sepp-Lorenzino worked in oncology drug discovery and development, having led theCancer Research Department atMerck West Point , and having been anAssistant Lab Member atMemorial Sloan-Kettering Cancer Center . -
In addition,
Victor Dzau , M.D. informed the company that he intends to resign from the Board of Directors effectiveJune 30, 2014 , as a result of his pending appointment as President of theInstitute of Medicine effectiveJuly 1, 2014 .
-
Alnylam appointed
"I want to thank Victor for his seven years of service on our Board of
Directors. Victor joined our Board as Alnylam was making a transition
from a platform technology company to a product-focused company,
advancing RNAi therapeutics as a new class of genetic medicines. As
such, he was an instrumental advisor in this evolution of Alnylam as
best exemplified by our ‘5x15' strategy," said Dr. Maraganore. "We wish
Victor the best in his new leadership of our nation's
Conference Call Information
Management will provide an update on the company, discuss first 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); 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
aminolevulinate 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 liver
disease; 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; 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, The Medicines Company, and
Genzyme, a Sanofi company. In
About "Alnylam 5x15™" and Genetic Medicines
The "Alnylam 5x15" strategy, launched in
Use of Non-GAAP Financial Measures
Alnylam has presented in this press release certain non-GAAP financial measures, including non-GAAP net loss and non-GAAP net loss per common share - basic and diluted. A reconciliation between these non-GAAP financial measures and the most comparable GAAP financial measures are included in the tables accompanying 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 expectations regarding its "Alnylam 5x15" product strategy,
Alnylam's views with respect to the potential for RNAi therapeutics,
including patisiran (ALN-TTR02) and ALN-TTRsc, ALN-AT3, ALN-CC5,
ALN-AS1, ALN-PCSsc, ALN-AAT, ALN-TMP, ALN-ANG, and ALN-AC3, 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-TTRsc, ALN-CC5, ALN-PCSsc, and
ALN-AAT, its expectations regarding reporting of data from its clinical
studies, including its studies for patisiran, ALN-TTRsc, and ALN-AT3, as
well as other research programs and technologies, its plans regarding
commercialization of RNAi therapeutics, Genzyme's participation in the
development and commercialization of RNAi therapeutics, Alnylam's views
with respect to the potential value of the assets acquired from Merck
and its ability to further its efforts to build a new class of
medicines, its expected cash position as of
|
Unaudited Condensed Consolidated Statements of Comprehensive Loss (In thousands, except per share amounts) |
|||||||||||
| Three Months Ended | |||||||||||
|
|
|||||||||||
| 2014 | 2013 | ||||||||||
| Net revenues from collaborators | $ | 8,275 | $ | 18,642 | |||||||
| Operating expenses: | |||||||||||
| Research and development (1) | 43,758 | 22,179 | |||||||||
| In-process research and development | 224,656 | - | |||||||||
| General and administrative (1) | 8,925 | 6,267 | |||||||||
| Total operating expenses | 277,339 | 28,446 | |||||||||
| Loss from operations | (269,064 | ) | (9,804 | ) | |||||||
| Other income (expense): | |||||||||||
| Interest income | 333 | 224 | |||||||||
| Other (expense) income | (82 | ) | 5 | ||||||||
| Total other income | 251 | 229 | |||||||||
| Loss before income taxes | (268,813 | ) | (9,575 | ) | |||||||
| Benefit from income taxes | 17,870 | 562 | |||||||||
| Net loss | $ | (250,943 | ) | $ | (9,013 | ) | |||||
| Net loss per common share - basic and diluted | $ | (3.70 | ) | $ | (0.15 | ) | |||||
| Weighted average common shares used to compute basic and diluted net loss per common share | 67,786 | 59,173 | |||||||||
| Comprehensive loss | |||||||||||
| Net loss | $ | (250,943 | ) | $ | (9,013 | ) | |||||
| Unrealized gain on marketable securities, net of tax | 5,313 | 5,159 | |||||||||
| Comprehensive loss | $ | (245,630 | ) | $ | (3,854 | ) | |||||
| (1) Non-cash stock-based compensation expenses included in operating expenses are as follows: | |||||||||||
| Research and development | $ | 3,681 | $ | 2,087 | |||||||
| General and administrative | 1,910 | 988 | |||||||||
|
Unaudited GAAP to Non-GAAP Reconciliation: Net Loss and Net Loss Per Share (In thousands, except per share amounts) |
||||||
| For the Three Months | ||||||
|
Ended |
||||||
| 2014 | 2013 | |||||
| GAAP net loss | $ | (250,943) | $ | (9,013) | ||
| Adjustment: | ||||||
| In-process research and development expense | 224,656 |
— |
||||
| Non-GAAP net loss | $ | (26,287) | $ | (9,013) | ||
| GAAP net loss per common share - basic and diluted | $ | (3.70) | $ | (0.15) | ||
| Adjustment (as detailed above) | 3.31 |
— |
||||
| Non-GAAP net loss per common share - basic and diluted | $ | (0.39) | $ | (0.15) | ||
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 first
quarter of 2014, the company's Non-GAAP net loss and Non-GAAP loss per
common share - basic and diluted financial measures excludes the
in-process research and development expense of
|
Unaudited Condensed Consolidated Balance Sheets (In thousands, except share amounts) |
|||||||||
|
2014 |
2013 |
||||||||
| Cash, cash equivalents and total marketable securities | $ | 1,000,515 | $ | 350,472 | |||||
| Billed and unbilled collaboration receivables | 164 | 4,248 | |||||||
| Prepaid expenses and other current assets | 8,409 | 3,910 | |||||||
| Property and equipment, net | 15,731 | 16,448 | |||||||
| Investment in equity securities of Regulus Therapeutics Inc. | 55,478 | 45,452 | |||||||
| Total assets | $ | 1,080,297 | $ | 420,530 | |||||
| Accounts payable, accrued expenses and other liabilities | $ | 54,841 | $ | 20,056 | |||||
| Total deferred revenue | 66,795 | 126,090 | |||||||
| Total deferred rent | 3,688 | 4,037 | |||||||
|
Total stockholders' equity (75.5 million and 63.7 million common
shares issued and outstanding and at |
954,973 | 270,347 | |||||||
| Total liabilities and stockholders' equity | $ | 1,080,297 | $ | 420,530 | |||||
|
|
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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
or
Vice President, Finance and Treasurer
Source:
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For Investor Inquiries, please contact:
Josh Brodsky
VP, Investor Relations & Corporate Communications investors@alnylam.com 617-551-8276
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