Nov 09, 2015 Press Release for Alnylam
Alnylam Pharmaceuticals Reports Third Quarter 2015 Financial Results and Highlights from Recent Period
Nov 09, 2015
- Advanced Late-Stage Clinical Pipeline: Patisiran APOLLO Phase 3 Trial on Track to Complete Enrollment in Next 3-4 Months; if Positive, Expect NDA Filing in 2017 -
- Presented Positive Clinical Data for ALN-PCSsc for Hypercholesterolemia and ALN-AS1 for Acute Hepatic Porphyrias -
- Maintained Strong Balance Sheet with
- Hosting R&D Day on
"The past quarter and recent period were marked by tremendous progress
in our efforts to advance innovative medicines to patients. Notably, we
made significant progress with our late-stage clinical programs,
including new data on patisiran and revusiran. Importantly, we're on
track to complete enrollment in our APOLLO Phase 3 trial of patisiran
within the next three to four months; if the study is positive, APOLLO
data are expected to support an NDA submission in 2017," said
Third Quarter 2015 and Recent Significant Corporate Highlights
-
Advanced pipeline programs in Genetic Medicine Strategic Therapeutic
Area (STAr).
-
Advanced investigational RNAi therapeutic programs for the
treatment of transthyretin (TTR)-mediated amyloidosis (ATTR
amyloidosis).
-
Continued enrollment in APOLLO Phase 3 study of patisiran in
ATTR amyloidosis patients with Familial Amyloidotic
Polyneuropathy (FAP).
- The company announced last week that it is on track to complete enrollment in APOLLO within the next three to four months. If positive, the company expects that the APOLLO study results will support an NDA submission for patisiran in 2017.
- Reported positive 12- and 18-month clinical data from patisiran Phase 2 open-label extension (OLE) study, showing sustained mean maximal TTR knockdown of 93% and continued evidence for potential halting of neuropathy progression. Importantly, we also documented first-ever objective evidence for an effect of patisiran toward improvement of nerve fiber density. Patisiran was also found to be generally well tolerated out to nearly two years of drug administration.
- Initiated Phase 3 OLE study ("APOLLO-OLE") with patisiran.
- Continued enrollment in ENDEAVOUR Phase 3 study of revusiran in ATTR amyloidosis patients with Familial Amyloidotic Cardiomyopathy (FAC).
- 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, 6 minute walk distance (6MWD) results at 6 months were stable compared with baseline, and revusiran was generally well tolerated out to 10 months of administration.
- 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.
-
Continued enrollment in APOLLO Phase 3 study of patisiran in
ATTR amyloidosis patients with Familial Amyloidotic
Polyneuropathy (FAP).
-
Advanced ALN-AT3 for the treatment of hemophilia and rare bleeding
disorders (RBD)
- Continued dosing hemophilia patients with once-monthly subcutaneous dose regimen in ongoing Phase 1 study.
- Initiated Phase 1 OLE study with ALN-AT3.
- Announced Genzyme's election to opt into ALN-AT3 program for development and potential future commercialization in territories outside of North America and Western Europe.
-
Advanced ALN-CC5 for the treatment of complement-mediated diseases.
- Continued dosing healthy volunteers in multiple ascending dose part of ongoing Phase 1 trial.
-
Advanced ALN-AS1 for the treatment of acute hepatic porphyrias
- Presented initial positive results from ongoing Phase 1 trial, showing that single subcutaneous doses of ALN-AS1 resulted in lowering of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic heme synthesis intermediates that mediate porphyria attacks. In addition, ALN-AS1 treatment resulted in potent, dose-dependent, and durable silencing of aminolevulinic acid synthase-1 (ALAS1) mRNA in liver. Further, ALN-AS1 was found to be generally well tolerated with no clinically significant drug-related adverse events through the data cut-off date.
- Presented initial data from EXPLORE trial, a multinational, prospective observational study of patients with hepatic porphyrias suffering from recurrent attacks.
- Initiated Phase 1/2 trial with ALN-AAT for the treatment of alpha-1 antitrypsin (AAT) deficiency-associated liver disease (alpha-1 liver disease).
-
Advanced ALN-GO1 for the treatment of primary hyperoxaluria type 1
(PH1)
- Selected ALN-GO1 DC, with plans to file CTA in late 2015 and initiate Phase 1 study in early 2016.
-
Advanced investigational RNAi therapeutic programs for the
treatment of transthyretin (TTR)-mediated amyloidosis (ATTR
amyloidosis).
-
Advanced investigational pipeline programs in Cardio-Metabolic Disease
and Hepatic Infectious Disease STArs.
-
Alnylam and The Medicines Company presented positive initial data from the ongoing Phase 1 trial of ALN-PCSsc for the treatment of hypercholesterolemia. The effects of ALN-PCSsc toward LDL-C were found to be comparable to reported results with anti-PCSK9 monoclonal antibodies, but showed a durability profile supportive of a once-quarterly and possibly bi-annual subcutaneous dose regimen. Further, ALN-PSCsc was found to be generally well tolerated. - Continued pre-clinical studies to support a CTA filing for ALN-HBV in late 2015.
-
- Advanced new innovations for investigational RNAi therapeutics with Bis-RNAiTM and ReversirTM platforms.
- Announced that the United States District Court for the District of Massachusetts granted Alnylam's summary judgment motions in Tuschl II patent inventorship dispute.
-
Expanded Management Team and Scientific Advisory Board -
Expanded Management Team with appointments of
Jim Bilotta as Vice President, Information Technology,Christine Regan Lindenboom as Vice President, Investor Relations & Corporate Communications, andGabriel Robbie , Ph.D., Vice President, Drug Metabolism and Pharmacokinetics. In addition, the Company promotedSusanna High to Senior Vice President, Strategy & Business Integration, andMartin Maier , Ph.D., to Vice President, Chemistry. -
Appointed
Nancy J. Brown , M.D., Chair,Department of Medicine and Physician-in-Chief ,Vanderbilt University School of Medicine , and Muthiah "Mano" Manoharan, Ph.D., Senior Vice President, Innovation Chemistry & Alnylam Distinguished Scientist, toAlnylam Scientific Advisory Board .
-
Expanded Management Team with appointments of
Upcoming Events in Late 2015
-
Alnylam announces today that it plans to host an R&D Day on
Thursday, December 10, 2015 from8:00 am to 11:30 am ET at the Sofitel New York inNew York City . -
In addition, during late 2015, Alnylam plans to:
-
Present updated data from ongoing Phase 1 clinical trial of
ALN-PCSsc at the
American Heart Association (AHA) Scientific Sessions 2015, being heldNovember 7 - 11, 2015, inOrlando, Florida , in late-breaking oral presentation onWednesday, November 11 , at10:45 am ET . -
Present multi-dose data from ongoing Phase 1 clinical trial of
ALN-CC5 at the 57th
American Society of Hematology (ASH) Annual Meeting and Exposition being heldDecember 5 - 8, 2015 inOrlando , in a poster presentation onSunday, December 6 at6:00 pm ET . This will include the first-ever report on results with weekly dose administration of ALN-CC5, as all previously reported data - including those in the now-published ASH abstract - pertain to single doses of study drug. -
Present monthly dosing data from ongoing Phase 1 clinical trial of
ALN-AT3 in an oral presentation at ASH, on
Monday, December 7 at11:30 am ET . - Initiate Part C of ongoing Phase 1 trial of ALN-CC5 in patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Initiate Phase 2 clinical trial with ALN-PCSsc, in collaboration with The Medicines Company, in late 2015.
- File CTA in late 2015 to initiate a clinical trial for ALN-GO1.
- File CTA in late 2015 to initiate a clinical trial for ALN-HBV.
-
Present updated data from ongoing Phase 1 clinical trial of
ALN-PCSsc at the
Financials
Cash,
At
GAAP Net Loss
The net loss according to accounting principles generally accepted in
the U.S. (GAAP) for the third quarter of 2015 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
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
Conference Call Information
Management will provide an
update on the company, discuss third quarter 2015 results, and discuss
expectations for the future via conference call on
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.
In January 2014, Alnylam and Genzyme, a
Sanofi company, 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 Genzyme obtained the right to access
certain programs in Alnylam's current and future Genetic Medicines
pipeline in the rest of the world (ROW,) including
co-development/co-commercialization and/or global product rights for
certain programs. In the case of patisiran and ALN-AT3, Alnylam will
advance the product in North America and Western Europe, while Genzyme
will advance the product in the ROW. In the case of revusiran, Alnylam
and Genzyme will co-develop/co-commercialize the product in
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
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, ALN-GO1 and ALN-HBV, 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
for ALN-GO1 and ALN-HBV, its expectations regarding reporting of data
from its clinical and pre-clinical studies, including its studies for
ALN-PCSsc, ALN-CC5 and ALN-AT3, its plans regarding commercialization of
RNAi therapeutics, its collaboration with Genzyme and Alnylam's 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
|
Nine Months Ended
|
|||||||||||||||
| 2015 | 2014 | 2015 | 2014 | |||||||||||||
| Net revenues from collaborators | $ | 6,324 | $ | 10,972 | $ | 33,546 | $ | 26,542 | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development (1) | 68,618 | 46,273 | 193,660 | 134,703 | ||||||||||||
| In-process research and development | — | — | — | 220,766 | ||||||||||||
| General and administrative (1) | 16,036 | 9,898 | 43,382 | 30,341 | ||||||||||||
| Total operating expenses | 84,654 | 56,171 | 237,042 | 385,810 | ||||||||||||
| Loss from operations | (78,330 | ) | ( 45,199 | ) | (203,496 | ) | (359,268 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Interest income | 1,610 | 753 | 4,243 | 1,779 | ||||||||||||
|
Other (expense) income |
(72 | ) | 524 | (99 | ) | 365 | ||||||||||
| Total other income | 1,538 | 1,277 | 4,144 |
2,144 |
||||||||||||
| Loss before income taxes | (76,792 | ) | (43,922 | ) | (199,352 | ) | (357,124 | ) | ||||||||
| (Provision for) Benefit from income taxes | — | (67 | ) | — | 18,118 | |||||||||||
| Net loss | $ | (76,792 | ) | $ | (43,989 | ) | $ | (199,352 | ) | $ | (339,006 | ) | ||||
| Net loss per common share - basic and diluted | $ | (0.91 | ) | $ | (0.58 | ) | $ | (2.38 | ) | $ | (4.62 | ) | ||||
| Weighted average common shares used to compute basic and diluted net loss per common share | 84,633 | 76,408 | 83,696 | 73,375 | ||||||||||||
| Comprehensive loss: | ||||||||||||||||
| Net loss | $ | (76,792 | ) | $ | (43,989 | ) | $ | (199,352 | ) | $ | (339,006 | ) | ||||
| Unrealized loss on marketable securities, net of tax | (25,981 | ) | (6,230 | ) | (55,982 | ) | (3,964 | ) | ||||||||
|
Reclassification adjustment for realized gain on marketable
securities
included in net loss |
— | (567 | ) | — | (567 | ) | ||||||||||
| Comprehensive loss | $ | (102,773 | ) | $ | (50,786 | ) | $ | (255,334 | ) | $ | (343,537 | ) | ||||
| (1) Non-cash stock-based compensation expenses included in operating expenses are as follows: | ||||||||||||||||
| Research and development | $ | 6,334 | $ | 3,781 | $ | 17,829 | $ | 10,019 | ||||||||
| General and administrative | 5,514 | 2,571 | 12,434 | 9,604 | ||||||||||||
|
Unaudited GAAP to Non-GAAP Reconciliation: Net Loss and Net Loss Per Share (In thousands, except per share amounts) |
||||||||||||||||
|
Three Months Ended
|
Nine Months Ended
|
|||||||||||||||
| 2015 | 2014 | 2015 | 2014 | |||||||||||||
| GAAP net loss | $ | (76,792 | ) | $ | (43,989 | ) | $ | (199,352 | ) | $ | (339,006 | ) | ||||
| Adjustment: | ||||||||||||||||
| In-process research and development | — | — | — | 220,766 | ||||||||||||
| Non-GAAP net loss | $ | (76,792 | ) | $ | (43,989 | ) | $ | (199,352 | ) | $ | (118,240 | ) | ||||
| GAAP net loss per common share - basic and diluted | $ | (0.91 | ) | $ | (0.58 | ) | $ | (2.38 | ) | $ | (4.62 | ) | ||||
| Adjustment (as detailed above) | — | — | — | 3.01 | ||||||||||||
| Non-GAAP net loss per common share - basic and diluted | $ | (0.91 | ) | $ | (0.58 | ) | $ | (2.38 | ) | $ | (1.61 | ) | ||||
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 nine
months ended
|
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands, except share amounts) |
||||||
|
|
|
|||||
| 2015 | 2014 | |||||
| Cash, cash equivalents and total marketable securities | $ | 1,336,274 | $ | 881,929 | ||
| Billed and unbilled collaboration receivables | 8,130 | 39,937 | ||||
| Prepaid expenses and other current assets | 17,453 | 9,739 | ||||
| Deferred tax assets | 9,406 | 31,667 | ||||
| Property and equipment, net | 26,316 | 21,740 | ||||
| Investment in equity securities of Regulus Therapeutics Inc. | 38,565 | 94,583 | ||||
| Total assets | $ | 1,436,144 | $ | 1,079,595 | ||
| Accounts payable and accrued expenses | $ | 32,716 | $ | 38,791 | ||
| Deferred tax liabilities | 9,406 | 31,667 | ||||
| Total deferred revenue | 66,090 | 66,854 | ||||
| Total deferred rent | 6,328 | 6,016 | ||||
|
Total stockholders' equity (84.7 million and 77.2 million common
shares issued and outstanding and at |
1,321,604 | 936,267 | ||||
| Total liabilities and stockholders' equity | $ | 1,436,144 | $ | 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
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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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