Alnylam Pharmaceuticals Reports First Quarter 2012 Financial Results
Continued Progress with "Alnylam 5x15" including Initiation of Phase I Trial with ALN-TTR02
Reported Positive Clinical Results for ALN-PCS with up to 84% Knockdown of PCSK9 and 50% Lowering of LDL Cholesterol in Single Dose, Statin-Free Phase I Trial
Reports Approximately $317 Million in Cash and Expects to End 2012 with Greater than $250 Million in Cash
"We made significant clinical and business progress this quarter, including important achievements with our 'Alnylam 5x15' product development and commercialization strategy. First, we reported very encouraging clinical data from our Phase I ALN-PCS trial showing statistically significant and durable reductions of PCSK9 plasma levels of up to 84% and lowering of LDL cholesterol of up to 50%, in addition to demonstrating continued improved efficacy and tolerability for Alnylam's second-generation lipid nanoparticle delivery technology. Further, we initiated dosing in our Phase I clinical trial with ALN-TTR02, an RNAi therapeutic targeting the transthyretin gene for the treatment of transthyretin-mediated amyloidosis, which also utilizes our second-generation lipid nanoparticle delivery technology. We are on track to report clinical data from this study in the third quarter of this year," said
"With clear evidence from our ALN-TTR and ALN-PCS Phase I clinical trials that the RNAi pathway can be harnessed as a therapeutic strategy, we continue to focus on our 'Alnylam 5x15' efforts with what we believe to be our highest value opportunities, driving key programs toward pivotal trials," said
The net loss according to accounting principles generally accepted in the U.S. (GAAP) for the first quarter of 2012 was
Research and Development Expenses
Research and development (R&D) expenses were
General and Administrative Expenses
General and administrative (G&A) expenses were
Equity in loss of joint venture was
Interest income was
2012 Financial Guidance
Alnylam expects that its cash, cash equivalents and total marketable securities balance will be greater than
"Alnylam further strengthened its balance sheet this quarter with a public offering of 8.6 million shares of common stock that resulted in net proceeds of approximately
First Quarter 2012 and Recent Significant Corporate Highlights
Key "Alnylam 5x15" Program Highlights
Advanced Clinical Developmentof ALN-TTR Program for Transthyretin(TTR)-Mediated Amyloidosis (ATTR). Alnylam completed its ALN-TTR01 Phase I study and expects to report final data at the International Symposium on Amyloidosis meeting in May 2012. Preliminary data reported last year showed that ALN-TTR01 was generally safe and well tolerated and resulted in a statistically significant lowering of TTR serum levels in ATTR patients, thus demonstrating important human proof of concept for this innovative medicine.
Alnylam is advancing ALN-TTR02, which uses the same siRNA as ALN-TTR01, but is formulated in a more potent, proprietary second-generation lipid nanoparticle (LNP) technology using the "MC3" lipid. Alnylam has initiated a Phase I trial with ALN-TTR02 aimed at evaluating safety, tolerability, and clinical activity of ALN-TTR02 in healthy volunteers. Specifically, the study is evaluating the clinical activity of ALN-TTR02 toward knockdown of serum TTR, the disease-causing protein in patients with ATTR. Alnylam expects to present data from this study in the third quarter of 2012. In addition, Alnylam plans to start a Phase II multi-dose study of ALN-TTR02 in ATTR patients in the second half of 2012 and, assuming positive results, expects to start a pivotal trial for ALN-TTR02 in 2013. Alnylam also plans to advance ALN-TTRsc, which utilizes a GalNAc-conjugate delivery approach and subcutaneous dose administration.
Alnylam announced today that it has received a U.S. patent (U.S. Patent No. 8,168,775) for RNAi therapeutics targeting the transthyretin gene.
Continued Developmentof ALN-APC Program for Hemophilia. Alnylam is developing an RNAi therapeutic targeting Protein C, an endogenous anticoagulant pathway, for the treatment of hemophilia. Alnylam plans to advance its hemophilia program toward the clinic with a goal of initiating a Phase I clinical trial in the first half of 2013, with data expected in the second half of 2013. The company will present updated pre-clinical data from this program at the World Federation of Hemophiliain July 2012.
- Reported 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). New 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 reductions of PCSK9 plasma levels 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. These data also highlight continued improved efficacy and tolerability for Alnylam's second-generation LNP delivery technology. Alnylam plans to partner this program prior to conducting Phase II clinical studies.
Progresswith Additional "Alnylam 5x15" Programs. Alnylam designated ALN-TMP as its fifth "Alnylam 5x15" program. ALN-TMP comprises a systemically delivered RNAi therapeutic targeting transmembrane protease, serine 6 (Tmprss6) for the treatment of hemoglobinopathies, including beta-thalassemia and sickle cell anemia. Pre-clinical animal model studies with ALN-TMP have demonstrated corrective effects on iron overload in addition to broader disease modifying effects including improvements in hemoglobin levels and spleen histopathology. Alnylam plans to partner this program prior to conducting a Phase I clinical study. Alnylam also plans to partner its ALN-HPN program which targets the hepcidin pathway for the treatment of refractory anemia prior to conducting a Phase I clinical study.
Key Partner-Based Program Highlights
- Completed Enrollment in Phase IIb Clinical Study of ALN-RSV01 for the Treatment of Respiratory Syncytial Virus (RSV) Infection. The company has completed enrollment in its Phase IIb trial with ALN-RSV and expects to report results in mid-2012. The Phase IIb trial was designed as a multi-center, randomized, double-blind, placebo-controlled study in RSV-infected lung transplant patients. The ALN-RSV program is partnered with
Kyowa Hakko Kirin Co., Ltd.in Asia and Cubist Pharmaceuticals, Inc.worldwide except Asia.
- Completed Phase I Clinical Study of ALN-VSP for the Treatment of Liver Cancers. Alnylam has completed its Phase I study with ALN-VSP, a systemically delivered RNAi therapeutic targeting both vascular endothelial growth factor (VEGF) and kinesin spindle protein (KSP) for the treatment of liver cancers. Data reported to date from the Phase I study showed that ALN-VSP was generally well tolerated, demonstrated evidence for anti-tumor activity, and was found to mediate RNAi activity in both hepatic and extra-hepatic tumors. Complete data from this Phase I trial will be presented at the
American Society of Clinical Oncologymeeting in June 2012. The company intends to partner ALN-VSP prior to initiating a Phase II study.
- Provides Update on Huntington's Disease Program. ALN-HTT is the drug under development as part of a drug-device combination therapy for the treatment of Huntington's disease being advanced in collaboration with
Medtronic, Inc.and CHDI Foundation, Inc.As part of its alignment of resources on its ATTR and hemophilia programs, Alnylam has elected to exercise its option under its agreement with Medtronicto opt-out of the 50-50 expense/profit share arrangement of the ALN-HTT program. If Medtronicdecides to continue the ALN-HTT program, Alnylam would continue to supply ALN-HTT drug product for the program and would be entitled to receive milestones and royalty payments on future annual net sales.
Business and Organizational Highlights
- Completed Successful Public Offering of Common Stock. Alnylam completed a public offering of common stock resulting in the issuance of a total of 8,625,000 shares and receipt of aggregate net proceeds, after deducting underwriting discounts and commissions and other estimated offering expenses, of approximately
- Formed Delivery Collaboration with
Arrowhead Research Corporation. Alnylam granted Arrowhead a license under its intellectual property that enables the discovery, development, and commercialization of an RNAi therapeutic targeting the hepatitis B virus (HBV). In addition, Alnylam has received a license from Arrowhead to utilize their Dynamic Polyconjugate (DPC) delivery technology for an RNAi therapeutic product.
- Implemented Strategic Corporate Restructuring. Alnylam announced a strategic corporate restructuring, including an approximate 33% reduction in its workforce. The workforce reduction was implemented in order to align the company's resources to a more focused execution on its ATTR and hemophilia programs with accelerated clinical development plans. Alnylam expects the reduction in personnel costs, along with other external costs, to result in a savings of approximately
$20 millionin 2012 cash operating expenses.
- Expanded Board of
Directors and Scientific Advisory Board. Alnylam has elected Dennis A. Ausiello, M.D. to its Board of Directors and Scientific Advisory Board. Dr. Ausiello is the Jackson Professor of Clinical Medicine at Harvard Medical Schooland Chief of Medicine at Massachusetts General Hospital(MGH).
Conference Call Information
Management will provide an update on the company, discuss first 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, http://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 RNAs (siRNAs), 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 5x15™"
The "Alnylam 5x15" strategy, launched in
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-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, ALN-APC for the treatment of hemophilia, 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 partner-based 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,
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-APC, ALN-PCS, ALN-TMP, ALN-VSP, ALN-RSV01 and ALN-HTT, 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 and ALN-APC, its expectations regarding reporting data from its ongoing and planned clinical studies, including its studies for ALN-TTR01, ALN-TTR02, ALN-APC and ALN-RSV, its plans to seek collaborations for its ALN-PCS, ALN-HPN, ALN-TMP and ALN-VSP programs, its expectations regarding the continued development of ALN-HTT, its expected cash position as of
Alnylam Pharmaceuticals, Inc.
Unaudited Condensed Consolidated Statements of Comprehensive Loss
(In thousands, except per share amounts)
Three Months Ended
|Net revenues from research collaborators||$||20,587||$||20,897|
Research and development (1)
|General and administrative (1)||10,406||10,224|
|Total operating expenses||31,480||36,573|
|Loss from operations||(10,893)||(15,676)|
|Other income (expense):|
|Equity in loss of joint venture (Regulus Therapeutics Inc.)||(889)||(1,072)|
|Total other income (expense)||(475)||(609)|
|Net loss per common share - basic and diluted||$||(0.25)||$||(0.38)|
Weighted average common shares used to compute basic and diluted net loss per common share
|Unrealized gain (loss) on marketable securities||128||(732)|
(1) Non-cash stock-based compensation expenses included in operating expenses are as follows:
|Research and development||$||2,088||$||2,665|
|General and administrative||1,068||1,457|
Alnylam Pharmaceuticals, Inc.
|Unaudited Condensed Consolidated Balance Sheets|
|(In thousands, except share amounts)|
|March 31,||December 31,|
|Cash, cash equivalents and total marketable securities||$316,917||$260,809|
|Billed and unbilled collaboration receivables||2,192||1,468|
|Prepaid expenses and other current assets||7,252||4,158|
|Property and equipment, net||13,703||14,643|
|Investment in joint venture (Regulus Therapeutics Inc.)||-||564|
|Intangible assets, net||231||275|
|Accounts payable and accrued expenses||$17,345||$18,140|
|Total deferred revenue||120,784||140,853|
|Total deferred rent||3,810||4,211|
Total stockholders' equity (51.9 million and 42.7 million common shares issued and outstanding at March 31, 2012 and December 31, 2011, respectively)
|Total liabilities and stockholders' equity||$340,295||$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
Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Vice President, Investor Relations and
Michael Mason, 617-551-8327
Vice President, Finance and Treasurer