Alnylam Pharmaceuticals Reports First Quarter 2015 Financial Results and Highlights Recent Period Progress
- Launched "Alnylam 2020" Guidance for Advancement and Commercialization of RNAi Therapeutics -
- Advanced Multiple Clinical Programs and Presented Evidence for Potential Disease-Modifying Activity for Patisiran in Familial Amyloidotic Polyneuropathy (FAP) and ALN-AT3 in Hemophilia -
- Plans to Present New Clinical Data for ALN-AT3 and Initial Clinical Data for ALN-CC5 in Oral Presentations at Medical Meetings in June -
- Maintained Strong Balance Sheet with
"These are exciting times for RNAi therapeutics and Alnylam's efforts to
bring innovative medicines to patients. With our ‘Alnylam 2020' strategy
we aim to transition from a late-stage clinical development company to a
multi-product commercial-stage company with a sustainable development
pipeline - a profile that we believe has rarely been achieved in the
biopharmaceutical industry. Amongst many achievements in the first
quarter and recent period, we reported promising data on potential
disease modifying effects for patisiran in Familial Amyloidotic
Polyneuropathy (FAP) and ALN-AT3 in hemophilia. Specifically, in our
patisiran Phase 2 open-label extension - or ‘OLE' - study, we generated
what we believe to be continued evidence for a possible halting of
neuropathy progression after the first 12 months of treatment, with drug
administration generally well tolerated out to 17 months. Furthermore,
recent results from our Phase 1 trial with ALN-AT3 provide initial
evidence for potential correction of the hemophilia phenotype with an
encouraging tolerability profile. In aggregate, we believe these results
strengthen the bridge that we are building between RNAi-mediated
knockdown and potential clinical benefit for patients," said
First Quarter 2015 and Recent Significant Corporate Highlights
- Launched "Alnylam 2020" Guidance for advancement and commercialization of RNAi Therapeutics. Specifically, by the end of 2020, Alnylam expects to achieve a company profile with 3 marketed products, as well as 10 RNAi therapeutic clinical programs - including 4 in late stages of development - across its 3 Strategic Therapeutic Areas (STArs).
Advanced pipeline programs in Genetic Medicine STAr.
Advanced investigational RNAi therapeutic programs for the
treatment of transthyretin (TTR)-mediated amyloidosis (ATTR
- Continued enrollment in APOLLO Phase 3 study of patisiran in ATTR amyloidosis patients with Familial Amyloidotic Polyneuropathy (FAP).
- Reported positive 12-month clinical data from patisiran Phase 2 open-label extension (OLE) study, showing sustained TTR knockdown of up to a mean 88% and continued evidence for potential halting of neuropathy progression. Specifically, a mean 2.5 point decrease in neuropathy impairment score (mNIS+7) was observed after 12 months of patisiran administration, comparing favorably to a 13-18 point increase in untreated patients with similar baseline characteristics, as estimated from published historical data sets. Patisiran was also found to be generally well tolerated out to 17 months of drug administration.
Continued enrollment in
ENDEAVOURPhase 3 study of revusiran in ATTR amyloidosis patients with Familial Amyloidotic Cardiomyopathy (FAC).
- Presented complete Phase 2 data with revusiran in patients with TTR cardiomyopathy, showing tolerability and an up to 98.2% knockdown of serum TTR.
- Continued dosing FAC patients in revusiran Phase 2 OLE study designed to evaluate the tolerability and clinical activity of revusiran with long-term dosing for up to two years.
from a retrospective natural history study evaluating
disease progression in ATTR amyloidosis patients with FAC,
showing a 140 meter decline in 6 minute walk distance (6MWD)
at 18 months; the change in 6MWD at 18 months is a co-primary
endpoint measure in
Advanced ALN-AT3 for the treatment of hemophilia and rare bleeding
- Reported positive initial results from a small number of subjects in a Phase 1 trial of ALN-AT3, including an up to 70% knockdown of antithrombin (AT) and initial evidence for the potential correction of the hemophilia phenotype with an up to 334% increase in thrombin generation and marked improvement in whole blood clotting.
- The company announces today that it is transitioning to once-monthly subcutaneous dose cohorts in its ongoing Phase 1 study.
- The company also announces today that it has completed its chronic GLP toxicology studies of ALN-AT3, including a 9-month study in non-human primates and 6-month studies in rat and hemophilia A mice, with No Adverse Effect Level (NOAEL) doses that support further advancement of the program.
- Published pre-clinical study results in Nature Medicine documenting safety, efficacy, and durability of ALN-AT3 in rodent and non-human primate (NHP) models of hemophilia.
- Initiated Phase 1/2 trial with ALN-CC5. The trial is being conducted initially in normal human volunteers, and then is expected to move to patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Advanced investigational RNAi therapeutic programs for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis).
Advanced pipeline programs in Cardio-Metabolic Disease STAr
Continued dosing in Phase 1 trial with ALN-PCSsc in normal human
volunteers with elevated LDL-C at baseline.
- The company announces today that it has completed the single ascending dose (SAD) phase of the study and has initiated the multi-dose (MD) phase with or without statin co-administration.
- Formed new agreement with Isis Pharmaceuticals, extending the companies' decade-long alliance to lead the development and commercialization of RNA therapeutics.
Completed successful public offering of common stock, with
concurrent private placements from Genzyme, totaling
$567 millionin net proceeds.
Signed a 295,000 square foot lease with an affiliate of BioMed
Realty for Class A laboratory and office space in
Cambridgefor the company's future corporate headquarters.
The company announces today that
Cynthia Clayton, Vice President of Investor Relations and Corporate Communications, will be transitioning from Alnylam to pursue personal interests. Michael Mason, Alnylam's Vice President of Finance, will lead the company's investor relations area on an interim basis. The company has initiated an external search to identify a new person for this role. Joshua Brodsky, Senior Manager, Investor Relations and Corporate Communications, will report to Mr. Masonduring the transition period.
- Continued dosing in Phase 1 trial with ALN-PCSsc in normal human volunteers with elevated LDL-C at baseline.
"We want to thank Cynthia for her decade-long leadership of Alnylam's
corporate communications team. We are very sad to see her go, but are
happy that she'll have a chance to pursue her personal interests," said
Upcoming Events in Mid-2015
Alnylam announces today that it plans to present additional data from
its ALN-AT3 Phase 1 trial in subjects with hemophilia at the
International Society on Thrombosis and Haemostasis(ISTH) 2015 Congress, being held June 20- 25, 2015 in Toronto, in an oral presentation on Tuesday, June 23at 2:30 p.m. ET.
The company also announces today that it plans to present initial
single ascending dose (SAD) cohort data from its ALN-CC5 Phase 1/2
trial in normal human volunteers at the 20th
Congressof the European Hematology Association(EHA), being held June 11- 14, 2015 in Vienna, Austria, in an oral presentation on Sunday, June 14at 8:45 a.m.Central European Summer Time ( 2:45 a.m. ET).
In addition, during mid-2015, Alnylam plans to:
- Initiate Phase 1 trial with ALN-AS1 in development for the treatment of hepatic porphyrias
- Present initial data from Phase 1 trial of ALN-PCSsc in development for the treatment of hypercholesterolemia
- File Clinical Trial Application (CTA) for ALN-AAT in development for the treatment of alpha-1 antitrypsin (AAT) deficiency-associated liver disease
- Select Development Candidate (DC) for ALN-GO1 in development for the treatment of Primary Hyperoxaluria Type 1 (PH1)
"Alnylam continues to maintain a very strong balance sheet, with approximately $1.45 billion in cash as of the end of the first quarter of 2015," said Michael Mason, Vice President, Finance & Treasurer. "Our cash balance was bolstered in the quarter with net proceeds of approximately $567 million that resulted from a public offering and concurrent private placements from Genzyme. This financing resulted in a balance sheet that allows us to invest in a broad pipeline of RNAi therapeutics across all three Alnylam STArs, which we believe will enable us to realize our ‘Alnylam 2020' goals. As for financial guidance this year, we remain on track to end 2015 with greater than $1.2 billion in cash."
Under the investor agreement, Genzyme also has the right each January to
purchase a number of shares of Alnylam's common stock based on the
number of shares issued during the previous year for
compensation-related purposes. Genzyme exercised this right to purchase
196,251 shares of our common stock on
The exercise of these rights allowed Genzyme to maintain its current ownership level of Alnylam common stock of approximately 12%.
Non-GAAP Net Loss
The non-GAAP net loss for the first quarter of 2015 was
GAAP Net Loss
The net loss according to accounting principles generally accepted in
the U.S. (GAAP) for the first quarter of 2015 was
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
Conference Call Information
Management will provide an update on the company, discuss first quarter 2015 results, and discuss expectations for the future via conference call on
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-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.
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. Alnylam's pipeline of investigational RNAi therapeutics is focused in 3 Strategic Therapeutic Areas (STArs): Genetic Medicines, with a broad pipeline of RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics toward genetically validated, liver-expressed disease targets for unmet needs in cardiovascular and metabolic diseases; and Hepatic Infectious Disease, with a pipeline of RNAi therapeutics that address the major global health challenges of hepatic infectious diseases. In early 2015, Alnylam launched its "Alnylam 2020" guidance for the advancement and commercialization of RNAi therapeutics as a whole new class of innovative medicines. Specifically, by the end of 2020, Alnylam expects to achieve a company profile with 3 marketed products, 10 RNAi therapeutic clinical programs - including 4 in late stages of development - across its 3 STArs. The company's demonstrated commitment to RNAi therapeutics has enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen, Roche,
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 2020" guidance, Alnylam's views with respect to the potential for RNAi therapeutics, including patisiran, revusiran, ALN-AT3, ALN-CC5, ALN-PCSsc, ALN-AS1, ALN-AAT, and ALN-GO1, 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-AAT and ALN-AS1, its expectations regarding reporting of data from its clinical and pre-clinical studies, including its studies for patisiran, revusiran, ALN-AT3, ALN-CC5, and ALN-PCSsc, as well as other research programs and technologies, its plans regarding commercialization of RNAi therapeutics, and Alnylam's expected cash position as of
|Unaudited Condensed Consolidated Statements of Comprehensive Loss|
|(In thousands, except per share amounts)|
|Three Months Ended|
|Net revenues from collaborators||$||18,537||$||8,275|
|Research and development (1)||58,035||43,758|
|In-process research and development||—||224,656|
|General and administrative (1)||12,724||8,925|
|Total operating expenses||70,759||277,339|
|Loss from operations||(52,222||)||(269,064||)|
|Other income (expense):|
|Other income (expense)||—||(82||)|
|Total other income||1,014||251|
|Loss before income taxes||(51,208||)||(268,813||)|
|Benefit from income taxes||431||17,870|
|Net loss per common share - basic and diluted||$||(0.62||)||$||(3.70||)|
|Weighted average common shares used to compute basic and diluted net loss per common share||82,074||67,786|
|Unrealized gain on marketable securities, net of tax||3,622||5,313|
|(1) Non-cash stock-based compensation expenses included in operating expenses are as follows:|
|Research and development||$||5,346||$||3,681|
|General and administrative||2,890||1,910|
Unaudited GAAP to Non-GAAP Reconciliation: Net Loss and Net Loss Per Share
(In thousands, except per share amounts)
|For the Three Months|
|GAAP net loss||$||(50,777)||
|In-process research and development expense||
|Non-GAAP net loss||$||(50,777)||
|GAAP net loss per common share - basic and diluted||$||(0.62)||
|Adjustment (as detailed above)||
|Non-GAAP net loss per common share - basic and diluted||$||(0.62)||
Use of Non-GAAP Financial Measures
The company supplements its condensed consolidated financial statements presented on a GAAP basis by providing additional measures that are considered "non-GAAP" financial measures under applicable
The company evaluates items on an individual basis, and considers both
the quantitative and qualitative aspects of the item, including (i) its
size and nature, (ii) whether or not it relates to the company's ongoing
business operations, and (iii) whether or not the company expects it to
occur as part of its normal business on a regular basis. In the first
quarter of 2014, the company's Non-GAAP net loss and Non-GAAP loss per
common share - basic and diluted financial measures excludes the
in-process research and development expense of
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except share amounts)
|Cash, cash equivalents and total marketable securities||$||1,450,754||$||881,929|
|Billed and unbilled collaboration receivables||10,107||39,937|
|Prepaid expenses and other current assets||13,206||9,739|
|Deferred tax assets||33,667||31,667|
|Property and equipment, net||21,166||21,740|
|Investment in equity securities of Regulus Therapeutics Inc.||99,890||94,583|
|Accounts payable, accrued expenses and other liabilities||$||31,917||$||38,791|
|Deferred tax liabilities||33,667||31,667|
|Total deferred revenue||60,665||66,854|
|Total deferred rent||6,031||6,016|
Total stockholders' equity (84.2 million and 77.2 million common
|Total liabilities and stockholders' equity||$||1,628,790||$||1,079,595|
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, Finance and Treasurer
Senior Manager, Investor Relations and
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