Feb 11, 2016 Press Release for Alnylam
Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2015 Financial Results and Highlights Recent Period Progress
Feb 11, 2016
- Advanced Pipeline of Eight Clinical Stage Programs in 2015, with Ten or More Major Clinical Data Readouts, Start of Fitusiran Phase 3 Trials, and Three New IND Filings Planned in 2016 -
- Completed Enrollment in Patisiran Phase 3 APOLLO Trial, Positioning the Company for First Filing for Regulatory Approval in 2017 -
- Maintained Strong Balance Sheet with
"In 2015, including the fourth quarter, we made strong progress
advancing our pipeline and executing on our broader business objectives.
Through our focused efforts, we grew our pipeline to eight clinical
stage programs, achieved human proof of concept in multiple programs,
and accrued patients in our two pivotal Phase 3 trials, including the
APOLLO study, where we have now completed enrollment," said
Fourth Quarter 2015 and Recent Significant Corporate Highlights
-
Advanced investigational pipeline programs in Genetic Medicine STAr.
-
Advanced RNAi therapeutics programs for the treatment of
transthyrethin (TTR)-mediated amyloidosis (ATTR amyloidosis).
-
Completed APOLLO Phase 3 enrollment with 225 patients for
patisiran, in development for patients with Transthyretin
(TTR)-Mediated Amyloidosis (ATTR Amyloidosis).
- If the study is positive, the Company expects to submit a New Drug Application (NDA) and Marketing Authorisation Application (MAA) for patisiran, based on an analysis of the full APOLLO data set, in late 2017.
-
Reported positive initial 18-month clinical data from patisiran
Phase 2 open-label extension (OLE) study, showing
continued evidence for potential halting of neuropathy
progression. Patisiran was also found to be generally well
tolerated out to nearly two years of drug administration
through the data cutoff date.
-
Complete 18-month clinical data from the patisiran Phase 2
OLE is expected to be reported in an oral session at the
upcoming
American Academy of Neurology (AAN) annual meeting onApril 20, 2016 , inVancouver, Canada .
-
Complete 18-month clinical data from the patisiran Phase 2
OLE is expected to be reported in an oral session at the
upcoming
-
Continued enrollment in
ENDEAVOUR Phase 3 study of revusiran in ATTR amyloidosis patients with Familial Amyloidotic Cardiomyopathy (FAC), with data expected in 2018. - Reported initial 6-month clinical data from revusiran Phase 2 OLE study, showing sustained TTR knockdown representing the longest dosing experience reported to date for target gene knockdown with a GalNAc-siRNA conjugate. In majority of patients, revusiran was generally well tolerated out to 10 months of administration through the data cutoff date.
- Advanced Development Candidate (DC) for ALN-TTRsc02, an ESC-GalNAc-siRNA conjugate targeting TTR for the treatment of ATTR amyloidosis, with goal of filing a Clinical Trial Application (CTA) in early 2016, starting a Phase 1 study in mid-2016 with initial data in late 2016, and initiating a Phase 3 trial in 2017.
-
Completed APOLLO Phase 3 enrollment with 225 patients for
patisiran, in development for patients with Transthyretin
(TTR)-Mediated Amyloidosis (ATTR Amyloidosis).
-
Advanced fitusiran (ALN-AT3) for the treatment of hemophilia and
rare bleeding disorders (RBD).
-
Presented positive interim data from ongoing Phase
1 trial of fitusiran.
- Interim results showed that monthly subcutaneous doses achieved antithrombin (AT) lowering associated with statistically significant and clinically meaningful increases in thrombin generation and decreases in bleeding frequency in patients with hemophilia A and B.
- Fitusiran was also found to be generally well tolerated through the data cutoff date, including no clinically significant increases in D-dimer, a biomarker of excessive clot formation.
-
Announced that Sanofi Genzyme elected to opt into the
fitusiran program for development and commercialization
outside of
North America andWestern Europe , while retaining its future opt-in right to co-develop and co-promote fitusiran with Alnylam in North America and Western Europe, subject to certain restrictions.
-
Presented positive interim data from ongoing Phase
1 trial of fitusiran.
-
Advanced ALN-CC5 for the treatment of complement-mediated diseases.
-
Presented positive initial data from an ongoing Phase
1/2 clinical trial.
- Results showed that ALN-CC5 achieved clinically meaningful reductions in serum C5 and inhibition of complement activity.
- ALN-CC5 was also shown to be generally well tolerated, with no clinically significant, drug-related adverse events through the data cutoff date.
- Initiated Part C of ongoing Phase 1/2 trial in patients with paroxysmal nocturnal hemoglobinuria (PNH).
-
Presented positive initial data from an ongoing Phase
1/2 clinical trial.
-
Advanced ALN-AS1 for the treatment of acute hepatic porphyrias.
-
Alnylam announces today that it has enrolled its first patient
in Part C of the ongoing Phase 1 clinical trial. Part C is
being conducted in Acute Intermittent Porphyria (AIP) patients
experiencing multiple recurrent porphyria attacks, and will
evaluate the safety and tolerability of multiple doses of
ALN-AS1 as well as measures of clinical activity, including
reduction in frequency and severity of attack symptoms,
hospitalizations, quality of life, and reduction in the use of
heme and pain medications.
- The Company expects to report initial data from recurrent attack patients in late 2016 and, if these data are positive, to initiate a Phase 3 study in 2017.
-
Alnylam announces today that it has enrolled its first patient
in Part C of the ongoing Phase 1 clinical trial. Part C is
being conducted in Acute Intermittent Porphyria (AIP) patients
experiencing multiple recurrent porphyria attacks, and will
evaluate the safety and tolerability of multiple doses of
ALN-AS1 as well as measures of clinical activity, including
reduction in frequency and severity of attack symptoms,
hospitalizations, quality of life, and reduction in the use of
heme and pain medications.
-
Advanced ALN-AAT for the treatment of alpha-1 antitrypsin (AAT)
deficiency-associated liver disease.
- Continued dosing human volunteers in ongoing Phase 1 study.
-
Advanced ALN-GO1 for the treatment of primary hyperoxaluria type 1
(PH1).
-
Filed CTA to initiate Phase 1 study and obtained approval of
the CTA from
U.K. regulatory authorities. -
Alnylam announces today that the
United States Food & Drug Administration (FDA) has granted Orphan Drug Designation to ALN-GO1 for the treatment of PH1.
-
Filed CTA to initiate Phase 1 study and obtained approval of
the CTA from
-
Advanced RNAi therapeutics programs for the treatment of
transthyrethin (TTR)-mediated amyloidosis (ATTR amyloidosis).
-
Advanced investigational pipeline programs in Cardio-Metabolic Disease
STAr.
-
Advanced ALN-PCSsc for the treatment of hypercholesterolemia.
- Presented positive interim Phase 1 clinical data demonstrating PCSK9 knockdown and LDL-cholesterol lowering supportive of a potential bi-annual subcutaneous dose regimen.
- The Medicines Company initiated the Phase 2 ORION-1 trial for ALN-PCSsc. The trial is expected to recruit 480 patients with atherosclerotic cardiovascular disease (ASCVD) and elevated LDL-C.
-
Advanced ALN-PCSsc for the treatment of hypercholesterolemia.
-
Advanced investigational pipeline programs in Hepatic Infectious
Disease STAr.
- Completed pre-clinical, IND-enabling studies with ALN-HBV supporting a CTA filing expected in early 2016.
-
Alnylam announces today that in
February 2016 , Sanofi Genzyme exercised its right under the investor agreement between Alnylam and Sanofi Genzyme to purchase 205,030 shares of Alnylam's common stock based on the number of shares Alnylam issued for 2015 compensation-related purposes, at$69.75 per share, resulting in proceeds to Alnylam of$14.3 million .- The exercise of this right allowed Sanofi Genzyme to maintain its ownership level of Alnylam common stock of approximately 12 percent.
-
Expanded Alnylam's Management Team and Board of Directors.
-
Announced new leadership positions with
Alex Leather , M.D., Ph.D., General Manager, EU andCanada ;Alfred Boyle , Ph.D., Senior Vice President, Technical Operations; andAkin Akinc , Ph.D. as General Manager, Fitusiran. -
Announced changes to our Board of Directors including the
appointment of
Michael Bonney as Chairman and election ofDavid Pyott as a Director.
-
Announced new leadership positions with
-
Presented corporate updates at our R&D Day and the J.P. Morgan Annual
Healthcare Conference, in December and January, respectively, and
provided guidance on notable advances we expect to complete over the
next two years including:
- Ten or more major clinical data readouts in 2016;
- Five or more ongoing Phase 3 trials in 2017; and
- Our first Phase 3 data readout and NDA/MAA submissions in 2017.
(To view presentations of data described above, please visit www.alnylam.com/capella)
Upcoming Events in Early and Mid-2016
-
Alnylam announced today that it plans to present the complete 18-month
patisiran OLE data in an oral presentation at the
American Academy of Neurology (AAN) annual meeting onApril 20, 2016 inVancouver, Canada . -
Additional upcoming milestones for Alnylam pipeline programs include:
-
In early 2016, Alnylam plans to:
- File a CTA for ALN-TTRsc02;
- Initiate a Phase 1 study for ALN-GO1; and
- File a CTA for ALN-HBV.
-
In mid-2016, Alnylam plans to:
- Present 24-month patisiran Phase 2 OLE data;
- Present 12-month revusiran Phase 2 OLE data;
- Start ALN-TTRsc02 Phase 1 study;
- Present additional fitusiran Phase 1 data;
- Start first fitusiran Phase 3 study;
- Present ALN-CC5 Phase 1/2 data in PNH patients;
- Present initial Phase 1 data with ALN-AAT; and
- Start ALN-HBV Phase 1 study.
-
In early 2016, Alnylam plans to:
Financials
"Alnylam continues to maintain a strong balance sheet, ending 2015 with
approximately
Cash,
At
Non-GAAP Net Loss
The non-GAAP net loss for the year ended
GAAP Net Loss
The net loss according to accounting principles generally accepted in
the
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
The company accounts for its investment in Regulus at fair value by
adjusting the value to reflect fluctuations in Regulus' stock price each
reporting period. At
2016 Financial Guidance
Alnylam expects that its cash, cash equivalents, and total marketable
securities balance will be greater than
Conference Call Information
Management will provide an update on the company, discuss fourth quarter and 2015 results, and discuss expectations for the future via conference call on Thursday, February 11, 2016 at 4:30 p.m. ET. To access the call, please dial 877-312-7507 (domestic) or 631-813-4828 (international) five minutes prior to the start time and refer to conference ID 45176516. A replay of the call will be available beginning at 7:30 p.m. ET on February 11, 2016. To access the replay, please dial 855-859-2056 (domestic) or 404-537-3406 (international), and refer to conference ID 45176516.
In January 2014, Alnylam and Sanofi Genzyme, the specialty care global
business unit of Sanofi, formed an alliance to accelerate and expand the
development and commercialization of RNAi therapeutics across the world.
The alliance is structured as a multi-product geographic alliance in the
field of rare diseases. Alnylam retains product rights in North
America and Western Europe, while Sanofi Genzyme obtained the right to
access certain programs in Alnylam's current and future Genetic
Medicines pipeline in the rest of the world (ROW) through the end of
2019, together with certain broader co-development/co-commercialization
rights and global rights for certain products. In the case of patisiran,
Alnylam will advance the product in North America and Western Europe,
while Sanofi Genzyme will advance the product in the ROW. In the case of
revusiran, Alnylam and Sanofi Genzyme will co-develop/co-commercialize
the product in
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 LNP Technology
Alnylam has licenses to Arbutus 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. 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, Takeda,
Kyowa Hakko Kirin, Cubist, GlaxoSmithKline, Ascletis, Monsanto, The
Medicines Company, and Sanofi Genzyme. In addition, Alnylam holds an
equity position in Regulus Therapeutics Inc., a company focused on
discovery, development, and commercialization of microRNA therapeutics.
Alnylam scientists and collaborators have published their research on
RNAi therapeutics in over 200 peer-reviewed papers, including many in
the world's top scientific journals such as Nature, Nature Medicine,
Nature Biotechnology, Cell,
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future
expectations, plans and prospects, including without limitation,
Alnylam's views with respect to the potential for RNAi therapeutics,
including patisiran, revusiran, fitusiran, ALN-CC5, ALN-AS1, ALN-AAT,
ALN-GO1, ALN-PCSsc and ALN-HBV, its expectations regarding its STAr
pipeline growth strategy, its "Alnylam 2020" guidance for the
advancement and commercialization of RNAi therapeutics, its expectations
for the timing of filing of regulatory documents, including but not
limited to IND or CTA submissions for ALN-TTRsc02 and ALN-HBV, and
submission of an MAA and NDA for patisiran, its expectations regarding
the timing of the start of clinical studies and presentation of clinical
data, including its studies for patisiran, revusiran, fitusiran,
ALN-CC5, ALN-AS1, and ALN-AAT, 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 |
Year Ended |
||||||||||||
| 2015 | 2014 | 2015 | 2014 | ||||||||||
| Net revenues from collaborators | $ | 7,551 | $ | 24,019 | $ | 41,097 | $ | 50,561 | |||||
| Operating expenses: | |||||||||||||
|
Research and development (1) |
82,835 | 55,546 | 276,495 | 190,249 | |||||||||
| In-process research and development | — | — | — | 220,766 | |||||||||
| General and administrative (1) | 17,228 | 14,185 | 60,610 | 44,526 | |||||||||
| Total operating expenses | 100,063 | 69,731 | 337,105 | 455,541 | |||||||||
| Loss from operations | (92,512) | (45,712) | (296,008) | (404,980) | |||||||||
| Other income (expense): | |||||||||||||
| Interest income | 1,616 | 780 | 5,859 | 2,559 | |||||||||
| Other income | 175 | 1,452 | 76 | 1,817 | |||||||||
| Total other income | 1,791 | 2,232 | 5,935 | 4,376 | |||||||||
| Loss before income taxes | (90,721) | (43,480) | (290,073) | (400,604) | |||||||||
| Benefit from income taxes | — | 22,091 | — | 40,209 | |||||||||
| Net loss | $ | (90,721) | $ | (21,389) | $ | (290,073) | $ | (360,395) | |||||
| Net loss per common share - basic and diluted | $ | (1.07) | $ | (0.28) | $ | (3.45) | $ | (4.85) | |||||
| Weighted-average common shares used to compute basic and diluted net loss per common share | 84,871 | 76,957 | 83,992 | 74,278 | |||||||||
| Comprehensive income (loss): | |||||||||||||
| Net loss | $ | (90,721) | $ | (21,389) | $ | (290,073) | $ | (360,395) | |||||
| Unrealized gain (loss) on marketable securities, net of tax | 11,588 | 35,091 | (44,394) | 31,127 | |||||||||
|
Reclassification adjustment for realized gain on marketable securities included in net loss |
— | (1,514) | — | (2,081) | |||||||||
| Comprehensive (loss) income | $ | (79,133) | $ | 12,188 | $ | (334,467) | $ | (331,349) | |||||
| (1) Non-cash stock-based compensation expenses included in operating expenses are as follows: | |||||||||||||
| Research and development | $ | 9,257 | $ | 8,214 | $ | 27,086 | $ | 18,233 | |||||
| General and administrative | 6,263 | 5,224 | 18,697 | 14,828 | |||||||||
|
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UNAUDITED GAAP TO NON-GAAP RECONCILIATION: NET LOSS AND NET LOSS PER SHARE |
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|
(In thousands, except per share amounts) |
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|
Three Months Ended |
Year Ended |
||||||||||||
| 2015 | 2014 | 2015 | 2014 | ||||||||||
| GAAP net loss | $ | (90,721) | $ | (21,389) | $ | (290,073) | $ | (360,395) | |||||
| Adjustment: | |||||||||||||
| In-process research and development | — | — | — | 220,766 | |||||||||
| Non-GAAP net loss | $ | (90,721 ) | $ | (21,389) | $ | (290,073) | $ | (139,629) | |||||
| GAAP net loss per common share - basic and diluted | $ | (1.07) | $ | (0.28) | $ | (3.45) | $ | (4.85) | |||||
| Adjustment (as detailed above) | — | — | — | 2.97 | |||||||||
| Non-GAAP net loss per common share - basic and diluted | $ | (1.07) | $ | (0.28) | $ | (3.45) | $ | (1.88) | |||||
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 year
ended
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UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS |
|||||||
|
(In thousands, except share amounts) |
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|
|
||||||
| 2015 | 2014 | ||||||
| Cash, cash equivalents and total marketable securities | $ | 1,280,951 | $ | 881,929 | |||
| Billed and unbilled collaboration receivables | 8,298 | 39,937 | |||||
| Prepaid expenses and other assets | 18,030 | 9,739 | |||||
| Deferred tax assets | — | 31,667 | |||||
| Property and equipment, net | 27,812 | 21,740 | |||||
| Investment in equity securities of Regulus Therapeutics Inc. | 51,419 | 94,583 | |||||
| Total assets | $ | 1,386,510 | $ | 1,079,595 | |||
| Accounts payable, accrued expenses and other liabilities | $ | 46,886 | $ | 38,791 | |||
| Deferred tax liabilities | — | 31,667 | |||||
| Total deferred revenue | 68,317 | 66,854 | |||||
| Total deferred rent | 6,593 | 6,016 | |||||
|
Total stockholders' equity (85.1 million and 77.2 million common
shares issued and outstanding and at |
1,264,714 | 936,267 | |||||
| Total liabilities and stockholders' equity | $ | 1,386,510 | $ | 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
View source version on businesswire.com: http://www.businesswire.com/news/home/20160211006307/en/
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Investors
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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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