Aug 06, 2012 Press Release for Alnylam
Alnylam Pharmaceuticals Reports Second Quarter 2012 Financial Results
Aug 06, 2012
“This was an exceptional quarter and recent period for Alnylam, where we
reported positive clinical data from several RNAi therapeutic programs.
Within our ‘Alnylam 5x15’ product efforts, we showed that ALN-TTR02
achieves rapid, dose-dependent, durable, and specific knockdown of serum
transthyretin - the disease causing protein in transthyretin-mediated
amyloidosis - after just a single dose. Specifically, we showed
transthyretin knockdown of up to 94% with a nearly 80% level of
suppression sustained at one month. We believe these data document an
unprecedented level of clinical activity for RNAi therapeutics and serve
as an important industry milestone in the advancement of this new class
of medicines to patients,” said
“In addition to the important advancements made with our ‘Alnylam 5x15’
programs, we also made notable progress with our partnered RNAi
therapeutic programs. We reported top-line results from our Phase IIb
trial with ALN-RSV01 - an inhaled RNAi therapeutic for the treatment of
respiratory syncytial virus infection in lung transplant patients -
which we believe provide important evidence for clinical efficacy toward
new or progressive bronchiolitis obliterans syndrome, an irreversible
and life-threatening complication of infection in these patients. We
look forward to presenting additional data from our ALN-RSV01 study and
discussing our results with U.S. and European regulatory authorities,”
said
Cash,
At
Net Loss
The net loss according to accounting principles generally accepted in
the U.S. (GAAP) for the second quarter of 2012 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
Regulus Therapeutics
Equity in loss of joint venture was
Interest Income
Interest income was
2012 Financial Guidance
Alnylam expects that its cash, cash equivalents and total marketable
securities balance will be greater than
“Alnylam continues to maintain a solid financial profile, ending the
second quarter with approximately
Second Quarter 2012 and Recent Significant Corporate Highlights
Key “Alnylam 5x15” Program Highlights
-
Reported Positive Clinical Data with ALN-TTR02, an RNAi Therapeutic
Targeting Transthyretin (TTR) for the Treatment of
Transthyretin-Mediated Amyloidosis (ATTR). Results
from a Phase I study of ALN-TTR02 were presented at
Boston University School of Medicine , showing that administration of ALN-TTR02 led to robust knockdown of serum TTR protein levels of up to 94%; the overall results were highly significant (p<0.00001 by ANOVA). Suppression of TTR, the disease-causing protein in ATTR, was found to be rapid, dose dependent, durable, and specific after just a single dose. ALN-TTR02 was found to be generally safe and well tolerated in this Phase I normal human volunteer study, consistent with Alnylam’s broader clinical experience with lipid nanoparticle (LNP)-formulated siRNA, which now includes more than 100 patients or subjects, over 325 total doses administered, and a length of treatment exceeding two years. -
Advanced Clinical Development of ALN-TTR02 into Phase II Clinical Trial. Alnylam initiated a Phase II clinical trial of ALN-TTR02 aimed at evaluating safety and tolerability of multiple once-monthly doses of ALN-TTR02 in ATTR patients. The study will also evaluate the clinical activity of ALN-TTR02 based on measurement of serum levels of TTR. In addition, theU.S. Food & Drug Administration (FDA ) has provided Orphan Drug Designation to ALN-TTR02 as a therapeutic for the treatment of familial amyloidotic polyneuropathy (FAP), one of the predominant clinical manifestations of ATTR. Alnylam plans to start a pivotal trial for ALN-TTR02 in 2013. -
Presented Pre-clinical Data with ALN-TTRsc, a Subcutaneously
Administered RNAi Therapeutic for the Treatment of ATTR. Data
presented today at the XX International Roundtable on Nucleosides,
Nucleotides and Nucleic Acids being held
August 5-9 inMontreal, Canada , showed potent, dose-dependent, and durable knockdown of serum TTR with Alnylam’s GalNAc-conjugate approach in its ALN-TTRsc pre-clinical program. In non-human primates, subcutaneous doses of ALN-TTRsc as low as 2.5 mg/kg resulted in an approximately 80% reduction of TTR. In addition, in single dose and multi-dose pre-clinical safety studies in rodents and non-human primates, ALN-TTRsc was found to be generally safe and well tolerated with a “no adverse effect level” (NOAEL) greater than or equal to 300 mg/kg, and, thus, a therapeutic index exceeding 100-fold. Alnylam expects to file an investigational new drug (IND) application, or IND equivalent, for ALN-TTRsc by the end of 2012. - Presented Final Results from a Phase I Clinical Trial of ALN-TTR01, a First-Generation RNAi Therapeutic for the Treatment of ATTR. Data were presented at the XIII International Symposium on Amyloidosis. Results from this study showed that administration of ALN-TTR01 resulted in statistically significant reductions in serum TTR protein levels, including both wild-type and mutant TTR protein, in ATTR patients. ALN-TTR01 was found to be generally safe and well tolerated in this study. Based on the significantly improved efficacy of ALN-TTR02, Alnylam has no further development plans for ALN-TTR01.
-
Designated ALN-AT3, an RNAi Therapeutic Targeting Antithrombin (AT)
for the Treatment of Hemophilia, as Development Candidate; Presented
New Pre-clinical Data. ALN-AT3 was selected as the company’s
development candidate in its hemophilia program, and employs Alnylam’s
GalNAc-siRNA conjugate technology enabling subcutaneous dose
administration. Data
presented at the
World Federation of Hemophilia World Congress demonstrated that subcutaneous administration of ALN-AT3 results in potent, dose-dependent, and durable silencing of AT in pre-clinical models and that AT reduction can normalize thrombin generation in an animal model of hemophilia, establishing proof of concept for this program. Specifically, ALN-AT3 demonstrated potent activity in both mice and non-human primates, with an ED50 for AT plasma protein knockdown of approximately 1 mg/kg after a single subcutaneous dose. Further, in studies performed in mouse models of hemophilia, ALN-AT3 was found to achieve dose-dependent knockdown of endogenous AT and to significantly increase thrombin generation. Alnylam also announced new data today for ALN-AT3 at the XX International Roundtable on Nucleosides, Nucleotides and Nucleic Acids showing that weekly subcutaneous injections at doses as low as 0.75 mg/kg provided sustained AT knockdown of approximately 80%. In addition, administration of ALN-AT3 in non-human primates showed a very durable response, where a single subcutaneous dose of ALN-AT3 achieved nadir knockdown of AT plasma levels at about day 15, with effects lasting over 22 days. ALN-AT3 was found to be generally safe and well tolerated in all of these pre-clinical studies. In aggregate, these new data support a once-a-week or twice-a-month subcutaneous dosing paradigm. Alnylam expects to file an IND application, or IND equivalent, for ALN-AT3 in 2013. - Presented Positive Clinical Results in ALN-PCS Severe Hypercholesterolemia Program. ALN-PCS is a PCSK9 synthesis inhibitor that reduces intracellular and extracellular levels of PCSK9 resulting in lowered plasma levels of low-density lipoprotein cholesterol (LDL-C, or “bad” cholesterol). Data from a Phase I clinical trial presented at the American Heart Association’s Arteriosclerosis, Thrombosis and Vascular Biology 2012 Scientific Sessions demonstrated that administration of a single dose of ALN-PCS, in the absence of statin co-administration, resulted in statistically significant and durable knockdown of plasma PCSK9 of up to 84% and lowering of LDL-C of up to 50%. ALN-PCS was shown to be safe and well tolerated in this study. Alnylam plans to partner this program prior to conducting Phase II clinical studies.
Key Partnered Program Highlights
-
Announced Top-Line Results of Phase IIb Clinical Trial of
ALN-RSV01, an Inhaled RNAi Therapeutic for the Treatment of
Respiratory Syncytial Virus (RSV) Infection in Lung Transplant
Patients. The Phase IIb study was a double-blind,
placebo-controlled, randomized global study of ALN-RSV01 with a
primary endpoint of the incidence of new or progressive bronchiolitis
obliterans syndrome (BOS) at 180 days after RSV infection and study
enrollment. Results
showed that ALN-RSV01 was associated with a clinically meaningful
treatment effect, with a reduction of over 50% in the incidence of day
180 BOS as compared with placebo. The study narrowly missed the
primary endpoint in an “intent-to-treat” (ITTc) analysis of confirmed
RSV infected patients (p=0.058), but achieved statistically
significant reductions in prospectively defined analyses of ITTc
patients with their “last observation carried forward” (p=0.028), and
of ITTc patients treated “per protocol” (p=0.025). Alnylam plans to
present complete results of the study at the European Respiratory
Society Annual Congress to be held
September 1-5, 2012 inVienna . The company plans to meet with U.S. and European regulatory authorities later this year to determine next steps for this program and will communicate future development plans, if any, in the second half of the year. -
Presented Data from its Phase I Extension Study with ALN-VSP, an
RNAi Therapeutic for the Treatment of Liver Cancers. Alnylam
presented data
from its ALN-VSP program at the
American Society of Clinical Oncology (ASCO ) annual meeting. Overall, the results demonstrated disease control lasting more than six months in the majority of patients treated on the extension study protocol, including a complete response (CR) in an endometrial cancer patient who had multiple liver metastases. In this study, chronic dosing with ALN-VSP was found to be generally safe and well tolerated. Alnylam reported today that the endometrial cancer patient has maintained her CR and has completed dosing after 50 doses and 26 months of therapy; one patient with stable disease remains on protocol with 17 months of therapy received to date.
Business and Organizational Highlights
-
Earned Milestone Payment from GlaxoSmithKline in VaxiRNA™
Collaboration on Flu Vaccine Production. Alnylam earned a
development milestone payment from GlaxoSmithKline (GSK), totaling
$3.2 million . The milestone payment is related to progress in the companies’ collaboration on the use of VaxiRNA technology for the production of GSK influenza vaccine. Alnylam’s VaxiRNA platform uses RNAi technology for the enhanced manufacturing of vaccine products. -
Formed Collaboration with Ascletis to Develop ALN-VSP.
Alnylam and Ascletis Pharmaceuticals Co., Ltd. ., a privately held U.S.-China joint venture pharmaceutical company, formed a strategic collaboration for the development of ALN-VSP, a systemically delivered RNAi therapeutic for the treatment of liver cancers including hepatocellular carcinoma (HCC), a significant area of unmet need inChina . This collaboration provides Ascletis with the exclusive rights to develop and commercialize ALN-VSP inChina includingHong Kong ,Macau , andTaiwan . Alnylam will retain all rights in the rest of the world, and is eligible to receive milestones and royalties based on product sales. -
Expanded Board of Directors,
Scientific Advisory Board , and Management Team. Alnylam electedDennis A. Ausiello , M.D. to its Board ofDirectors and Scientific Advisory Board . Dr. Ausiello is the Jackson Professor of Clinical Medicine atHarvard Medical School and Chief of Medicine atMassachusetts General Hospital (MGH). Alnylam has also appointedOved Amitay to the newly created position of Vice President, Head of Commercial. Before joining Alnylam, Mr. Amitay was Vice President for Strategic Development for thePersonalized Genetic Health business atGenzyme Corporation , now aSanofi company, and General Manager, Gaucher Disease Portfolio. Oved brings significant commercial, clinical and scientific experience in genetic diseases. In addition, Akshay Vaishnaw, M.D., Ph.D. has been named Executive Vice President and Chief Medical Officer; he previously held the title of Senior Vice President and Chief Medical Officer.Jared Gollob , M.D., has been promoted to Vice President, Clinical Research, from Senior Director, andLubomir (Lubo) Nechev , Ph.D. has been promoted to Vice President, Process Sciences, from Senior Director.
Conference Call Information
Management will provide an update on the company, discuss second quarter
2012 results, and discuss expectations for the future via conference
call on
A live audio webcast of the call will also be available on the News & Investors page of the company’s website, www.alnylam.com. An archived webcast will be available on the Alnylam website approximately two hours after the event.
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, ALN-PCS for
the treatment of severe hypercholesterolemia, ALN-HPN for the treatment
of refractory anemia, and ALN-TMP for the treatment of
hemoglobinopathies. 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, ALN-VSP for the treatment of liver cancers, and
ALN-HTT for the treatment of Huntington’s disease. The company’s
leadership position on RNAi therapeutics and intellectual property have
enabled it to form major alliances with leading companies including
Merck,
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 and ALN-TTRsc, ALN-AT3, ALN-PCS, ALN-VSP and
ALN-RSV01, 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-TTR02, ALN-TTRsc and ALN-AT3, its
expectations regarding reporting data from its clinical studies,
including its ALN-RSV01 study, its plans to seek collaborations for its
ALN-PCS program, its expectations regarding the continued development of
ALN-VSP in
| Alnylam Pharmaceuticals, Inc. | ||||||||||||||||
| Unaudited Condensed Consolidated Statements of Comprehensive Loss | ||||||||||||||||
| (In thousands, except per share amounts) | ||||||||||||||||
|
Three Months Ended June 30, |
Six Months Ended June 30, |
|||||||||||||||
| 2012 | 2011 | 2012 | 2011 | |||||||||||||
| Net revenues from research collaborators | $20,884 | $20,614 | $41,471 | $41,511 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development (1) | 21,723 | 25,303 | 42,797 | 51,652 | ||||||||||||
| General and administrative (1) | 11,228 | 8,429 | 21,634 | 18,653 | ||||||||||||
| Total operating expenses | 32,951 | 33,732 | 64,431 | 70,305 | ||||||||||||
| Loss from operations | (12,067 | ) | (13,118 | ) | (22,960 | ) | (28,794 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Equity in loss of joint venture (Regulus Therapeutics Inc.) | (1,139 | ) | (1,012 | ) | (2,028 | ) | (2,084 | ) | ||||||||
| Interest income | 271 | 322 | 494 | 704 | ||||||||||||
| Other (expense) income | (21 | ) | (16 | ) | 170 | 65 | ||||||||||
| Total other income (expense) | (889 | ) | (706 | ) | (1,364 | ) | (1,315 | ) | ||||||||
| Net loss |
$(12,956 |
) |
$(13,824 |
) |
$(24,324 |
) |
$(30,109 |
) | ||||||||
| Net loss per common share - basic and diluted |
$(0.25 |
) |
$(0.33 |
) |
$(0.50 |
) |
$(0.71 |
) | ||||||||
|
Weighted average common shares used to compute basic |
51,280 | 42,379 | 48,877 | 42,369 | ||||||||||||
| Comprehensive loss: | ||||||||||||||||
| Net loss |
$(12,956 |
) |
$(13,824 |
) |
$(24,324 |
) |
$(30,109 |
) | ||||||||
| Unrealized gain (loss) on marketable securities | (25 | ) | (228 | ) | 103 | (960 | ) | |||||||||
| Comprehensive loss |
$(12,981 |
) |
$(14,052 |
) |
$(24,221 |
) |
$(31,069 |
) | ||||||||
|
(1) Non-cash stock-based compensation expenses |
||||||||||||||||
| Research and development | $1,998 | $2,830 | $4,086 | $5,495 | ||||||||||||
| General and administrative | 1,098 | 1,384 | 2,166 | 2,841 | ||||||||||||
|
Alnylam Pharmaceuticals, Inc. |
||||||
| Unaudited Condensed Consolidated Balance Sheets | ||||||
| (In thousands, except share amounts) | ||||||
|
June 30, 2012 |
December 31, 2011 |
|||||
| Cash, cash equivalents and total marketable securities | $292,765 | $260,809 | ||||
| Billed and unbilled collaboration receivables | 4,385 | 1,468 | ||||
| Prepaid expenses and other current assets | 6,933 | 4,158 | ||||
| Property and equipment, net | 14,206 | 14,643 | ||||
| Investment in joint venture (Regulus Therapeutics Inc.) | — | 564 | ||||
| Intangible assets, net | 188 | 275 | ||||
| Total assets | $318,477 | $281,917 | ||||
| Accounts payable and accrued expenses | $19,747 | $18,140 | ||||
| Total deferred revenue | 103,453 | 140,853 | ||||
| Total deferred rent | 4,936 | 4,211 | ||||
| Investment in joint venture (Regulus Therapeutics Inc.) | 1,364 | — | ||||
| Other liabilities | 690 | 716 | ||||
|
Total stockholders’ equity (52.0 million and 42.7 |
188,287 | 117,997 | ||||
| Total liabilities and stockholders' equity | $318,477 | $281,917 | ||||
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
Source:
Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Vice
President, Investor Relations and
Corporate Communications
or
Michael
Mason, 617-551-8327
Vice President, Finance and Treasurer
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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