Aug 07, 2014 Press Release for Alnylam
Alnylam Pharmaceuticals Reports Second Quarter 2014 Financial Results and Highlights Recent Period Activities
Aug 07, 2014
- Achieved Positive Initial Results in Ongoing 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 -
- Reported Initial Positive Top-Line Data from Phase 1 Clinical Trial with ALN-AT3, Demonstrating Statistically Significant Knockdown of Antithrombin (AT) and Increase in Thrombin Generation -
- Broadened Pre-Clinical Pipeline with Development Candidates for ALN-CC5 for the Treatment of Complement-Mediated Diseases and ALN-AAT for the Treatment of Alpha-1 Antitrypsin-Associated Liver Disease; Added ALN-HBV for the Treatment of Hepatitis B Virus (HBV) Infection as New Program -
- 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 -
- Maintained Strong Balance Sheet with
"During the second quarter and recent period, we continued to execute on
our expanded ‘Alnylam 5x15' product development strategy and reported on
multiple significant clinical and pre-clinical data sets. As we continue
enrolling patients in our APOLLO Phase 3 trial with patisiran, we were
pleased to report positive initial data from our ongoing Phase 2
open-label extension (OLE) study, showing sustained knockdown of serum
TTR protein levels in addition to a favorable tolerability profile.
Patients in the OLE study have been treated for up to nine months, and
there have been no discontinuations. Enrollment also continues in our
ALN-TTRsc Phase 2 study in TTR cardiomyopathy and we remain on track to
initiate our Phase 3 trial later this year," said
"In addition to advancements in our clinical pipeline, we also made
strong progress with our pre-clinical programs. First, we were pleased
to select our Development Candidate for ALN-CC5 - an RNAi therapeutic
targeting complement C5 for the treatment of complement-mediated
diseases - and we remain on track to file our IND for this program in
late 2014. Our new pre-clinical results with ALN-PCSsc - an RNAi
therapeutic targeting PCSK9 for the treatment of hypercholesterolemia -
highlight the potential for once-monthly or possibly once-quarterly
subcutaneous dosing which we believe has the potential to be disruptive
in a significant emerging market; we expect to file a clinical trial
application (CTA) for ALN-PCSsc later this year. In addition, we
selected a Development Candidate 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, and we added ALN-HBV - an RNAi therapeutic
targeting the hepatitis B viral (HBV) genome for the treatment HBV
infection - as a new program with the goal of filing an IND around
year-end 2015. All of these programs employ our Enhanced Stabilization
Chemistry (ESC) GalNAc-conjugate technology, that enables subcutaneous
dosing with increased potency, durability, and a wide therapeutic index,
and 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 second
quarter of 2014 was
GAAP Net Loss
The net loss according to accounting
principles generally accepted in the U.S. (GAAP) for the second quarter
of 2014 was
Revenues
Revenues were
Research and Development Expenses
Research and development
(R&D) expenses were
In the second
quarter of 2014, the company recorded a reduction of
General and Administrative Expenses
General and
administrative (G&A) expenses were
"Alnylam continues to maintain a very strong balance sheet, ending the
second quarter with
Second Quarter 2014 and Recent Significant Corporate Highlights
Key "Alnylam 5x15" and Genetic Medicine Pipeline Highlights
-
Continued to Advance Patisiran (ALN-TTR02) in 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 20 sites in 9 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 International Symposium on Amyloidosis (ISA) held
April 27 - May 1, 2014 the company presented multiple updates from its patisiran program. Amongst other data, the company presented initial positive results from its ongoing Phase 2 open-label extension (OLE) study. 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 has now completed enrollment in the study with 27 patients. After up to nine months of therapy, there have been no study drug discontinuations. 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 plans on reporting initial clinical endpoint data from the OLE study at the 2014American Neurological Association Annual Meeting, being heldOctober 12 - 14, 2014 inBaltimore , where the initial dataset is expected to include 6 month mNIS+7 results from approximately 20 patients. - In addition, Alnylam and collaborators presented results 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 design.
- Finally, the company presented a pre-clinical update with its ATTR program. Alnylam scientists presented data confirming that the degree of TTR knockdown in a mouse disease model 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 profiles.
-
Alnylam also completed enrollment of 12 healthy volunteers of
Japanese descent in a Phase 1 study with patisiran. This study 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 in Development for the Treatment of
ATTR Patients with Cardiac Amyloidosis. The company continued
enrollment in its pilot 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 to date, the protocol for this study was
amended to enroll up to 25 FAC or SSA patients; the previous
enrollment target had been 15. Pending abstract acceptance, the
company expects to present data from the Phase 2 trial at the American
Heart Association Scientific Sessions 2014, being held
November 15 - 19, 2014 inChicago . 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. Assuming positive results, 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 was amended to enroll up to 76 normal human volunteers; previous target enrollment had been 40 subjects and results from that study were presented at theHeart Failure Society of America 17th Annual Scientific Meeting inSeptember 2013 . The increase in target enrollment was performed to determine 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 . -
Reported Initial Positive Top-Line Data from Phase 1 Clinical Trial
with ALN-AT3, an RNAi Therapeutic Targeting Antithrombin (AT) in
Development for the Treatment of Hemophilia and Rare Bleeding
Disorders (RBD). Results were presented at the
World Federation of Hemophilia (WFH) 2014World Congress , heldMay 11 - 15, 2014 inMelbourne, Australia . In Part A of the Phase 1 study, human volunteer subjects received a single subcutaneous dose of ALN-AT3 and, per protocol, the maximum allowable level of AT knockdown was set at 40%. Initial results from the sole dose cohort enrolled (n=4, 3:1, drug:placebo) showed that a single, low subcutaneous ALN-AT3 dose at 0.03 mg/kg resulted in an up to 28-32% knockdown of AT at nadir that was statistically significant relative to placebo (p < 0.01 by ANOVA). This resulted in a statistically significant (p < 0.01) increase in peak thrombin generation, that was temporally associated and consistent with the degree of AT knockdown. ALN-AT3 was found to be well tolerated with no significant adverse events reported. ALN-AT3 is the company's first program to enter clinical development using Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency, durability, and a wide therapeutic index. The company has now 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. Assuming abstract acceptance, Alnylam intends to present initial clinical results from the Phase 1 study, including all available results in hemophilia subjects, at the 56thAmerican Society of Hematology (ASH) Annual Meeting being heldDecember 6 - 9, 2014 inSan Francisco . Finally, Alnylam announced earlier today that ALN-AT3 has received Orphan Drug Designation in theEuropean Union . -
Advanced Development Candidate for ALN-CC5, a Subcutaneously
Administered RNAi Therapeutic Targeting Complement Component C5 in
Development for the Treatment of Complement-Mediated Diseases. New
results were presented at the 7th International
Conference on Complement Therapeutics, held June 6 - 11, 2014,
in Olympia, Greece, demonstrating that ALN-CC5 led to an up to 98.7%
knockdown of serum C5 and an up to 96.8% inhibition of serum
complement activity in non-human primates (NHPs) with weekly
subcutaneous dose administration. ALN-CC5 utilizes Alnylam's
ESC-GalNAc-conjugate technology. Assuming abstract acceptance, Alnylam
intends to present additional pre-clinical results from its ALN-CC5
program at the 56th
American Society of Hematology (ASH) Annual Meeting being heldDecember 6 - 9, 2014 inSan Francisco . The company is on track to file its ALN-CC5 IND or IND equivalent in late 2014, and expects to present initial clinical results in mid-2015. -
Presented New Pre-clinical Data on Subcutaneously Delivered RNAi
Therapeutics for Cardio-Metabolic Diseases, including ALN-PCSsc - an
RNAi Therapeutic Targeting PCSK9 in Development for the Treatment of
Hypercholesterolemia - and Added 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 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 had previously guided
that it plans to file an IND or IND equivalent, for ALN-PCSsc by
late 2014 or early 2015; Alnylam is announcing today that it plans
to file a Clinical Trial Application (CTA) for ALN-PCSsc in late
2014 and expects to present initial clinical results in mid-2015.
The ALN-PCS program is partnered with The Medicines Company,
where, under the terms of the agreement, Alnylam will complete
certain pre-clinical studies and a Phase 1 clinical study of
ALN-PCSsc and The Medicines Company is responsible for leading and funding development from Phase 2 forward and commercializing the ALN-PCS program, if successful. - 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 conduct pre-clinical work in this program to finalize its Development Candidate.
-
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 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 had previously guided
that it plans to file an IND or IND equivalent, for ALN-PCSsc by
late 2014 or early 2015; Alnylam is announcing today that it plans
to file a Clinical Trial Application (CTA) for ALN-PCSsc in late
2014 and expects to present initial clinical results in mid-2015.
The ALN-PCS program is partnered with The Medicines Company,
where, under the terms of the agreement, Alnylam will complete
certain pre-clinical studies and a Phase 1 clinical study of
-
Announced Development Candidate for ALN-AAT, an RNAi Therapeutic
Targeting Alpha-1-Antitrypsin (AAT) in Development 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, and, in some cases, hepatocellular carcinoma. The ALN-AAT Development Candidate employs Alnylam's ESC-GalNAc-conjugate technology. Alnylam plans to initiate IND-enabling studies with the goal of filing an IND or IND equivalent for ALN-AAT in mid-2015. In addition,Alnylam and The Alpha-1 Project (TAP), the venture philanthropy subsidiary of the Alpha-1 Foundation, announced that they have entered into a collaboration agreement for the continued advancement of ALN-AAT. -
Published Pre-Clinical Results with ALN-AS1, an RNAi Therapeutic
Targeting Aminolevulinic Acid Synthase-1 (ALAS-1) for the Treatment of
Hepatic Porphyrias, in the Proceedings of the
National Academy of Sciences . In the paper, titled "RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice," Alnylam scientists and collaborators at theIcahn School of Medicine at Mount Sinai in New York City documented results from pre-clinical models of the human disease showing that RNAi therapeutics targeting ALAS-1 can completely block the abnormal production of toxic intermediates of the heme biosynthesis pathway that cause the symptoms and disease pathology of AIP. Alnylam is currently advancing ALN-AS1, a subcutaneously administered RNAi therapeutic targeting ALAS-1 for the treatment of hepatic porphyrias, including acute intermittent porphyria (AIP). ALN-AS1 utilizes the company's ESC-GalNAc-conjugate technology. Alnylam expects to file an IND or IND equivalent for ALN-AS1 in late 2014 or early 2015. -
Advanced ALN-HBV for the Treatment of Hepatitis B Virus (HBV)
Infection. The new ALN-HBV program derives from the
company's January 2014 acquisition of Merck's RNAi assets, including
their Sirna Therapeutics subsidiary. In the most comprehensive
pre-clinical study results presented to date with an RNAi therapeutic
for the treatment of HBV, Alnylam reported significant, multi-log
reductions in HBV surface antigen (HBsAg) and HBV viral titers, and
showed evidence for an immune-mediated therapeutic effect in
chronically infected chimpanzees. Specifically, the new
data, presented at TIDES 2014, held
May 12 - 15 inProvidence, Rhode Island , demonstrated an up to 2.3 log10 reduction HBsAg. Alnylam plans to advance an ESC-GalNAc-siRNA conjugate targeting the HBV genome for its ALN-HBV program, which should enable once monthly subcutaneous dose administration with potent and durable effects, and a wide therapeutic index. The company expects to select a Development Candidate in late 2014 and plans to file an IND or IND equivalent around year end 2015. - Presented Key Scientific Data on Enhanced ESC-GalNAc-siRNA Conjugate Technology. Data presented at TIDES 2014 showed that chemical modifications of siRNA that enhance in vitro stability result in higher liver exposure in vivo and lead to a significantly increased potency and durability of effect. As compared with the "standard template chemistry" (STC)-GalNAc-conjugate approach used in ALN-TTRsc, ESC-GalNAc-siRNA conjugates demonstrated a 10-fold increased potency in NHP studies and, based on results with ALN-AT3, an over 50-fold increased potency in humans. In addition, ESC-GalNAc-siRNA conjugates demonstrate a durability of knockdown effect that supports once-monthly or potentially even less frequent subcutaneous dosing regimens. Finally, ESC-GalNAc-conjugates demonstrate a wide therapeutic index based on multi-dose toxicology studies performed in rodents and NHP. This subcutaneous delivery technology is being used in all Alnylam clinical and pre-clinical programs with the exception of patisiran and ALN-TTRsc.
Business and Organizational Highlights
-
Strengthened Intellectual Property (IP) Estate .-
Alnylam announced that the McSwiggen EP 1423406 ('406) patent was
upheld by the European Patent Office (EPO) in opposition
proceedings. The McSwiggen patent estate comprises a core
component of Alnylam's overall IP estate for the advancement of
RNAi therapeutics, and was recently obtained through the company's
acquisition of
Sirna Therapeutics from Merck. The upheld McSwiggen patent broadly describes chemical modifications of RNAi therapeutics needed to achieve "drug-like" properties in siRNA, the molecules that mediate RNAi. - Alnylam announced that the United States Patent and Trademark Office (USPTO) issued a patent covering RNAi Therapeutics for the treatment of HBV infection. The new patent (U.S. patent no. 8,618,277, or "'277 patent") is part of the company's McSwiggen patent estate. Specifically, the '277 patent includes claims that the company believes are critical for the development of RNAi therapeutics for the treatment of HBV infection. This patent is held exclusively by Alnylam and is not licensed to any third parties.
- Finally, Alnylam received a Notice of Allowance from the USPTO for a new patent broadly covering conjugate-based delivery of RNA therapeutics. The Manoharan et al. patent application 13/693,478, (the "'478 Application") includes newly allowed claims directed to compositions including those comprising a modified RNA agent linked to a biantennary or triantennary ligand. Specifically, the allowed application 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.
-
Alnylam announced that the McSwiggen EP 1423406 ('406) patent was
upheld by the European Patent Office (EPO) in opposition
proceedings. The McSwiggen patent estate comprises a core
component of Alnylam's overall IP estate for the advancement of
RNAi therapeutics, and was recently obtained through the company's
acquisition of
-
Alnylam Added to
Russell 1000 Index. The Russell 1000 Index measures the performance of the large-cap segment of the U.S. equity universe. It is a subset of the Russell 3000 Index and includes approximately 1000 of the largest securities based on a combination of their market cap and current index membership. -
Expanded Board of Directors. 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's early stage portfolio companies.
Conference Call Information
Management will provide an
update on the company, discuss second 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 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-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.
Alnylam is also developing ALN-HBV, an RNAi therapeutic targeting the
hepatitis B virus (HBV) genome for the treatment of HBV infection. 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, ALN-AC3, and ALN-HBV, 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, ALN-AS1,
ALN-AAT, and ALN-HBV, its expectations regarding reporting of data from
its clinical and pre-clinical studies, including its studies for
patisiran, ALN-TTRsc, ALN-AT3, and ALN-CC5, 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, 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
|
Six Months Ended
|
|||||||||||||||||
| 2014 | 2013 | 2014 | 2013 | |||||||||||||||
| Net revenues from collaborators | $ | 7,295 | $ | 8,687 | $ | 15,570 | $ | 27,329 | ||||||||||
| Operating expenses: | ||||||||||||||||||
| Research and development (1) | 44,672 | 24,215 | 88,430 | 46,394 | ||||||||||||||
| In-process research and development | (3,890) | — | 220,766 | — | ||||||||||||||
| General and administrative (1) | 11,518 | 5,784 | 20,443 | 12,051 | ||||||||||||||
| Total operating expenses | 52,300 | 29,999 | 329,639 | 58,445 | ||||||||||||||
| Loss from operations | (45,005) | (21,312) | (314,069) | (31,116) | ||||||||||||||
| Other income (expense): | ||||||||||||||||||
| Interest income | 693 | 270 | 1,026 | 494 | ||||||||||||||
| Other expense | (77) | (11) | (159) | (6) | ||||||||||||||
| Total other income | 616 | 259 | 867 | 488 | ||||||||||||||
| Loss before income taxes | (44,389) | (21,053) | (313,202) | (30,628) | ||||||||||||||
| Benefit from income taxes | 315 | 2,884 | 18,185 | 3,446 | ||||||||||||||
| Net loss | $ | (44,074) | $ | (18,169) | $ | (295,017) | $ | (27,182) | ||||||||||
| Net loss per common share - basic and diluted | $ | (0.58) | $ | (0.29) | $ | (4.11) | $ | (0.45) | ||||||||||
| Weighted average common shares used to compute basic and diluted net loss per common share | 75,835 | 61,661 | 71,833 | 60,424 | ||||||||||||||
| Comprehensive loss: | ||||||||||||||||||
| Net loss | $ | (44,074) | $ | (18,169) | $ | (295,017) | $ | (27,182) | ||||||||||
| Unrealized (loss) gain on marketable securities, net of tax | (3,047) | 7,309 | 2,266 | 12,468 | ||||||||||||||
| Comprehensive loss | $ | (47,121) | $ | (10,860) | $ | (292,751) | $ | (14,714) | ||||||||||
|
(1) Non-cash stock-based compensation expenses included in operating expenses are as follows: |
||||||||||||||||||
|
Research and development |
$ |
2,557 |
$ |
2,200 |
$ |
6,238 |
$ |
4,287 |
||||||||||
|
General and administrative |
5,123 |
1,177 |
7,033 |
2,165 |
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|
|
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| UNAUDITED GAAP TO NON-GAAP RECONCILIATION: NET LOSS AND NET LOSS PER SHARE | ||||||||||||||||||
| (In thousands, except per share amounts) | ||||||||||||||||||
|
Three Months Ended
|
Six Months Ended
|
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| 2014 | 2013 | 2014 | 2013 | |||||||||||||||
| GAAP net loss | $ | (44,074) | $ | (18,169) | $ | (295,017) | $ | (27,182) | ||||||||||
| Adjustment: | ||||||||||||||||||
| In-process research and development | (3,890) | — | 220,766 | — | ||||||||||||||
| Non-GAAP net loss | $ | (47,964) | $ | (18,169) | $ | (74,251) | $ | (27,182) | ||||||||||
| GAAP net loss per common share - basic and diluted | $ | (0.58) | $ | (0.29) | $ | (4.11) | $ | (0.45) | ||||||||||
| Adjustment (as detailed above) | (0.05) | — | 3.08 | — | ||||||||||||||
| Non-GAAP net loss per common share - basic and diluted | $ | (0.63) | $ | (0.29) | $ | (1.03) | $ | (0.45) | ||||||||||
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 second
quarter of 2014 and first half of 2014, the company's Non-GAAP net loss
and Non-GAAP net loss per common share - basic and diluted financial
measures excludes the in-process research and development reduction to
expense of
|
|
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| UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS | |||||||
| (In thousands, except share amounts) | |||||||
|
|
|
||||||
| 2014 | 2013 | ||||||
| Cash, cash equivalents and total marketable securities |
|
|
|||||
| Billed and unbilled collaboration receivables | 89 | 4,248 | |||||
| Prepaid expenses and other current assets | 10,270 | 3,910 | |||||
| Property and equipment, net | 16,953 | 16,448 | |||||
| Investment in equity securities of Regulus Therapeutics Inc. | 49,450 | 45,452 | |||||
| Total assets |
|
|
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| Accounts payable, accrued expenses and other liabilities |
|
|
|||||
| Total deferred revenue | 59,550 | 126,090 | |||||
| Total deferred rent | 3,808 | 4,037 | |||||
|
Total stockholders' equity (76.3 million and 63.7 million common
shares issued and outstanding and at |
942,269 | 270,347 | |||||
| Total liabilities and stockholders' equity |
|
|
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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 Corporate Communications
or
Vice President, Finance and Treasurer
Source:
News Provided by Acquire Media
For Media Inquiries, please contact:
Christine Akinc
Chief Corporate Communications Officer media@alnylam.com 617-682-4340
For Investor Inquiries, please contact:
Josh Brodsky
VP, Investor Relations & Corporate Communications investors@alnylam.com 617-551-8276
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