May 06, 2013 Press Release for Alnylam
Alnylam Pharmaceuticals Reports First Quarter 2013 Financial Results
May 06, 2013
— Continued to Execute on "Alnylam 5x15" Product Strategy,
—
— Completed Successful Public Offering of Stock; Maintained Strong
Balance Sheet with
"This quarter we continued to execute on our ‘Alnylam 5x15' product
strategy, where we are advancing RNAi therapeutics toward genetically
defined targets for diseases with limited treatment options for patients
and their caregivers. With ALN-TTR02, we continued enrollment in our
ongoing Phase II study and now expect to report initial results in late
June. In addition, we remain on track to start our Phase III trial in
familial amyloidotic polyneuropathy patients later this year. Further,
we were very pleased this quarter to have initiated a Phase I clinical
trial for ALN-TTRsc, a subcutaneously administered RNAi therapeutic for
the treatment of transthyretin-mediated amyloidosis. Importantly,
ALN-TTRsc represents Alnylam's first RNAi therapeutic utilizing our
proprietary GalNAc conjugate delivery platform to enter the clinic, as
well as the industry's first systemic RNAi therapeutic to be delivered
subcutaneously," said
"In addition to the exciting clinical advancements we made in the past
quarter, we also made excellent progress on the business and operational
front. Specifically, we completed a very successful public offering of
stock that strengthens our balance sheet for execution on our ‘Alnylam
5x15' product strategy and continued retention of product rights in core
programs that we aim to directly commercialize. Indeed, we continue to
believe that Alnylam's direct advancement of certain programs through
clinical trials and to the market represents the best path for the
patients we serve and the optimal value creation strategy for our
shareholders," said
Cash,
At
Net Loss
The net loss according to accounting principles generally accepted in
the U.S. (GAAP) for the first quarter of 2013 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
Equity in loss of joint venture was zero for the first quarter of 2013
and
Interest Income
Interest income was
Benefit from Income Taxes
The company had a benefit from income taxes of
2013 Financial Guidance
The company expects that its cash, cash equivalents and total marketable
securities balance will be greater than
"Alnylam continues to maintain a very solid balance sheet, ending this
first quarter with
First Quarter 2013 and Recent Significant Corporate Highlights
Key "Alnylam 5x15" Program Highlights
-
Continued Advancement of ALN-TTR02, an RNAi Therapeutic Targeting
Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis
(ATTR). Alnylam continues to enroll patients in its Phase II trial
with ALN-TTR02. This is an open-label, multi-center, multi-dose,
dose-escalation trial to evaluate the safety and tolerability of two
doses of ALN-TTR02 and to demonstrate clinical activity based on
serial measurement of circulating serum levels of wild-type and mutant
TTR. Alnylam announced today that it expects to present initial data
from this Phase II trial at the 2013 Biennial Meeting of the
Peripheral Nerve Society , being heldJune 29 —July 3 in St. Malo,France , pending acceptance of submitted abstract. Further, the company plans on expanding the study to a total of about 30 patients with additional study sites to explore additional cohorts treated with a once-every-three week dosing regimen and a reduced steroid pre-medication regimen. Alnylam remains on track to initiate in mid-2013 an open-label extension (OLE) study of ALN-TTR02 for patients treated in the Phase II study. The ALN-TTR02 OLE study will include a number of clinical endpoint measurements with initial data expected to be presented in 2014. Assuming positive results from the Phase II study, the company remains on track to start a Phase III pivotal trial for ALN-TTR02 in familial amyloidotic polyneuropathy (FAP) patients by the end of 2013. Alnylam announced today that it has successfully completed certain long-term non-clinical studies in line with the development plan supporting the start of the Phase III trial and chronic dosing in humans. -
Initiated Dosing in a Phase I Clinical Trial for ALN-TTRsc, a
Subcutaneously Administered RNAi Therapeutic Targeting TTR for the
Treatment of ATTR. ALN-TTRsc comprises an siRNA conjugated to a
triantennary N-acetylgalactosamine (GalNAc) ligand, and is being
developed for the treatment of ATTR patients with familial amyloidotic
cardiomyopathy (FAC). The Phase I trial is being conducted in the
U.K. as a randomized, double-blind, placebo-controlled, single- and multi-dose, dose-escalation study, enrolling up to 40 healthy volunteer subjects. The primary objective of the study is to evaluate the safety and tolerability of single and multiple doses of subcutaneously administered ALN-TTRsc. Secondary objectives include assessment of clinical activity of the drug as measured by serum TTR levels. Alnylam expects to report data from this trial in mid-2013. In addition, the company plans to start a Phase II clinical study of ALN-TTRsc in FAC patients by the end of 2013 and, assuming positive results, expects to start a pivotal trial for ALN-TTRsc in 2014. -
Advanced ALN-AT3, an RNAi Therapeutic Targeting Antithrombin (AT)
for the Treatment of Hemophilia and Other Rare Bleeding Disorders
(RBD), Toward Investigational New Drug (IND) Filing. ALN-AT3 is a
GalNAc-conjugate siRNA targeting AT, an endogenous anticoagulant
protein expressed in the liver. The company remains on track to file
an IND or IND equivalent for ALN-AT3 in mid-2013 and expects to start
a Phase I study by year's end. Alnylam announced today that its
scientists will present new pre-clinical data from its ALN-AT3 program
at the
XXIV Congress of theInternational Society on Thrombosis and Haemostasis being heldJune 29 —July 4 inAmsterdam, The Netherlands . -
Designated ALN-AS1, an RNAi Therapeutic Targeting Aminolevulinate
Synthase 1 (ALAS-1) for the Treatment of Acute Intermittent Porphyria
(AIP), as a New "Alnylam 5x15" Program. AIP is an ultra-rare
genetic disease caused by loss of function mutations in
porphobilinogen deaminase (PBGD), an enzyme in the heme biosynthesis
pathway, that can result in accumulation of toxic heme precursors.
Patients with AIP suffer from acute and/or recurrent life-threatening
attacks with severe abdominal pain, peripheral and autonomic
neuropathy, and neuropsychiatric manifestations. Approximately 5,000
patients in the U.S. and
Europe suffer AIP attacks annually, and approximately 500 patients are afflicted with recurrent debilitating attacks. ALN-AS1 is a GalNAc-conjugate siRNA targeting ALAS-1, a liver-expressed, rate-limiting enzyme upstream of PBGD. Inhibition of ALAS-1 is known to reduce the accumulation of heme precursors that cause the clinical manifestations of AIP. Alnylam intends to present key pre-clinical data for this program at theInternational Congress of Porphyrins and Porphyrias being heldMay 16 — 18 in Lucerne,Switzerland . The company expects to identify a final development candidate by late 2013 and advance ALN-AS1 into the clinic in 2014. - Continued to Advance Additional "Alnylam 5x15" Pipeline Programs. Alnylam continued to advance additional programs including ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia; ALN-TMP, an RNAi therapeutic targeting TMPRSS6 for the treatment of beta-thalassemia and iron-overload disorders; ALN-AAT, an RNAi therapeutic targeting alpha-1 antitrypsin deficiency-mediated liver disease; and several other undisclosed programs.
-
Plans to Host R&D Day,
July 11 . Alnylam is planning to host and webcast an R&D Day inNew York City onThursday, July 11 . At this event, Alnylam scientists, management, and key clinical collaborators will review progress and provide updates on the "Alnylam 5x15" programs. Additional details will be provided in the coming weeks.
Key Partnered Program Highlights
-
Published Results from Phase I Clinical Trial and Extension Study
with ALN-VSP, an RNAi Therapeutic for the Treatment of Liver Cancer. The
new paper, titled "First-in-Man
Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in
Cancer Patients with Liver Involvement" appeared as an OnlineFirst
publication in the journal Cancer Discovery. In this study —
the most comprehensive study of a systemically administered RNAi
therapeutic to date — chronic dosing of up to 26 months with ALN-VSP
was found to be generally safe and well tolerated. Further, ALN-VSP
showed evidence of RNAi activity in biopsy samples and anti-tumor
activity, including disease control in 42% (13/31) of patients treated
at higher doses and a complete response in an endometrial cancer
patient with multiple liver metastases. ALN-VSP is partnered with
Ascletis Pharmaceuticals (Hangzhou) Co., Ltd. , a privately held U.S.-China joint venture pharmaceutical company, for further advancement in the Chinese market; Alnylam has retained rights in the rest of the world.
Business and Organizational Highlights
-
Formed Strategic Worldwide Alliance with The Medicines Company to Advance ALN-PCS for the Treatment of Hypercholesterolemia.Alnylam and The Medicines Company are collaborating to develop and commercialize RNAi therapeutics targeting PCSK9 for the treatment of hypercholesterolemia. This alliance includes ALN-PCS02 and ALN-PCSsc, which are RNAi therapeutic product candidates administered by intravenous infusion and subcutaneous injection, respectively. Alnylam has completed a Phase I clinical study of ALN-PCS02 in human volunteers with elevated baseline low-density lipoprotein cholesterol (LDL-C); the study was performed in the absence of statin co-administration. A single dose of ALN-PCS02 was associated with rapid, dose-dependent, and durable knockdown of plasma PCSK9 by up to 84% and reduction in LDL-C of up to 50%. In addition, Alnylam has presented pre-clinical data for ALN-PCSsc showing potent knockdown of PCSK9 following subcutaneous administration. Alnylam will lead completion of certain pre-clinical and Phase I clinical studies while The Medicines Company will lead and fund development from Phase II forward and commercialization of the ALN-PCS program if successful. Under the terms of the agreement, The Medicines Company has made an upfront cash payment of$25 million to Alnylam. In addition, Alnylam is eligible to receive development and commercial milestone payments totaling up to$180 million , as well as double-digit royalties on ALN-PCS product sales. -
Completed Successful Public Offering of Common Stock. Alnylam
completed a public offering of common stock resulting in the issuance
of a total of 9,200,000 shares and receipt of aggregate net proceeds,
after deducting underwriting discounts and commissions and other
estimated underwriting expenses, of approximately
$174 million . -
Continued to Strengthen Intellectual Property Estate Covering RNAi
Therapeutics. Alnylam received Notices of Allowance from the
United States Patent and Trademark Office (USPTO) for two new patents
from the Tuschl I patent family. This represents the first allowances
by the USPTO from the Tuschl I patent estate, which is co-exclusively
licensed to Alnylam for RNAi therapeutics on a worldwide basis through
an agreement with
Max Planck Innovation GmbH , the licensing agent for theMax Planck Society . -
Expanded Management Team. Alnylam appointed
Aria Tavana , Ph.D., to the newly created position of Vice President of Quality Assurance. Before joining Alnylam, Dr. Tavana was the head of Clinical and Commercial Manufacturing andProcess Development at Momenta Pharmaceuticals, where he was involved in the launch and commercial production of generic enoxaparin, as well as the development of candidate drugs for oncology and immunology indications. Prior to Momenta, Aria was at Biogen Idec, where he served as Director of Quality Assurance & Quality Control. In that role he oversaw quality assurance of commercial and clinical product lines, including final drug disposition for human use.
Conference Call Information
Management will provide an update on the company, discuss first quarter
2013 results, and discuss expectations for the future via conference
call on
About RNA Interference (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
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 for the treatment of genetically defined
diseases, including ALN-TTR for the treatment of transthyretin-mediated
amyloidosis (ATTR), ALN-AT3 for the treatment of hemophilia and rare
bleeding disorders (RBD), ALN-AS1 for the treatment of acute
intermittent porphyria, ALN-PCS for the treatment of
hypercholesterolemia, and ALN-TMP for the treatment of beta-thalassemia
and iron-overload disorders. As part of its "Alnylam 5x15TM"
strategy, the company expects to have five RNAi therapeutic products for
genetically defined diseases in clinical development, including programs
in advanced stages, on its own or with a partner by the end of 2015.
Alnylam has additional partnered programs in clinical or development
stages, including ALN-RSV01 for the treatment of respiratory syncytial
virus (RSV) infection and ALN-VSP for the treatment of liver cancers.
The company's leadership position on RNAi therapeutics and intellectual
property have enabled it to form major alliances with leading companies
including Merck, Medtronic, Novartis, Biogen Idec, Roche,
About "Alnylam 5x15™"
The "Alnylam 5x15" strategy, launched 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 ALN-TTR02, ALN-TTRsc, ALN-AT3, ALN-AS1, ALN-TMP, ALN-AAT,
ALN-VSP, ALN-PCS02, and ALN-PCSsc, its expectations with respect to the
timing 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 initiate clinical trials for ALN-AT3 and
ALN-AS1, its expectations regarding reporting of data from its clinical
studies, including its ALN-TTR02 and ALN-TTRsc studies, and its
pre-clinical studies, including its ALN-AT3 and ALN-AS1 studies, its
plans to seek a partner for its ALN-TMP and ALN-AAT programs, and other
"Alnylam 5x15" programs, its expectations regarding the receipt of
upfront and potential milestone and royalty payments under its agreement
with The Medicines Company, its expectations regarding the potential
market opportunity for ALN-AS1, its views with regard to the strength,
enforceability, and validity of its intellectual property estate, and
its expected cash position as of
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| Unaudited Condensed Consolidated Statements of Comprehensive Loss | ||||||
| (In thousands, except per share amounts) | ||||||
| Three Months Ended | ||||||
|
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| 2013 | 2012 | |||||
| Net revenues from research collaborators | $ | 18,642 | $ | 20,587 | ||
| Operating expenses: | ||||||
|
Research and development (1) |
22,179 | 21,074 | ||||
| General and administrative (1) | 6,267 | 10,406 | ||||
| Total operating expenses | 28,446 | 31,480 | ||||
| Loss from operations | (9,804) | (10,893) | ||||
| Other income (expense): | ||||||
| Equity in loss of joint venture (Regulus Therapeutics Inc.) | — | (889) | ||||
| Interest income | 224 | 223 | ||||
| Other income | 5 | 191 | ||||
| Total other income (expense) | 229 | (475) | ||||
| Loss before income taxes | (9,575) | (11,368) | ||||
| Benefit from income taxes | 562 | — | ||||
| Net loss | $ | (9,013) | $ | (11,368) | ||
| Net loss per common share - basic and diluted | $ | (0.15) | $ | (0.25) | ||
| Weighted average common shares used to compute basic and diluted net loss per common share | 59,173 | 46,210 | ||||
| Comprehensive loss | ||||||
| Net loss | $ | (9,013) | $ | (11,368) | ||
| Unrealized gain on marketable securities, net of tax | 5,159 | 128 | ||||
| Comprehensive loss | $ | (3,854) | $ | (11,240) | ||
| (1) Non-cash stock-based compensation expenses included in operating expenses are as follows: | ||||||
| Research and development | $ | 2,087 | $ | 2,088 | ||
| General and administrative | 988 | 1,068 | ||||
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| Unaudited Condensed Consolidated Balance Sheets | ||||||
| (In thousands, except share amounts) | ||||||
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| 2013 | 2012 | |||||
| Cash, cash equivalents and total marketable securities | $ | 400,829 | $ | 226,228 | ||
| Billed and unbilled collaboration receivables | 558 | 104 | ||||
| Prepaid expenses and other current assets | 3,542 | 2,641 | ||||
| Property and equipment, net | 18,983 | 19,799 | ||||
| Investment in equity securities of Regulus Therapeutics Inc. | 47,666 | 38,748 | ||||
| Total assets | $ | 471,578 | $ | 287,520 | ||
| Accounts payable and accrued expenses | 15,214 | 15,978 | ||||
| Total deferred revenue | 139,528 | 132,291 | ||||
| Total deferred rent | 5,280 | 5,198 | ||||
|
Total stockholders' equity (62.1 million and 52.5 million common
shares issued and outstanding at |
311,556 | 134,053 | ||||
| Total liabilities and stockholders' equity | $ | 471,578 | $ | 287,520 | ||
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:
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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