May 02, 2016 Press Release for Alnylam
Alnylam Pharmaceuticals Reports First Quarter 2016 Financial Results and Highlights Recent Period Progress
May 02, 2016
- Advances Pipeline of Ten Clinical Stage Programs, with Ten Additional Clinical Data Readouts Anticipated This Year -
- Expects to Complete Enrollment of Revusiran Phase 3 ENDEAVOUR Trial in Late 2016 with Data Readout in Mid-2018 -
- On Track to Start Fitusiran Phase 3 Program in Mid-2016; Plans to Report Updated Hemophilia Patient Data in July -
- Provides Update on ALN-CC5 Program and Development Path Forward; Plans to Present Initial Data in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients in June -
-
"At Alnylam, we continue to advance a broad pipeline of investigational
RNAi therapeutics - including 10 programs in clinical development and 2
programs in Phase 3 - across a broad range of disease indications. A
major milestone in the first quarter was completion of enrollment in our
APOLLO Phase 3 trial for patisiran, and we're on track to view results
in mid-2017. If positive, we expect to file our first regulatory
applications for approval later that same year. We're also making strong
progress in our ENDEAVOUR Phase 3 trial of revusiran, where we now
expect completion of enrollment in late 2016 and data readout in
mid-2018. In our fitusiran program in hemophilia, we look forward to
presenting important new results in July, including initial results in
patients with inhibitors, and are on track to start our two Phase 3
studies shortly thereafter," said
First Quarter 2016 and Recent Significant Corporate Highlights
-
Advanced investigational pipeline programs in Genetic Medicine
Strategic Therapeutic Area (STAr).
-
Advanced investigational RNAi therapeutics programs for the
treatment of transthyrethin (TTR)-mediated amyloidosis (ATTR
amyloidosis).
-
Completed enrollment in the APOLLO Phase 3 trial with
patisiran for the treatment of hereditary TTR-mediated
amyloidosis with polyneuropathy (hATTR-PN), also known as
familial amyloidotic polyneuropathy (FAP).
- Based on strong investigator and patient interest, the study was substantially over enrolled with 225 patients.
- Data from APOLLO are expected in mid-2017, and assuming positive results, the Company expects to submit an NDA and MAA for patisiran by the end of 2017 and launch in 2018.
-
Announced complete 18-month data from ongoing Phase 2
open-label extension (OLE) study with patisiran.
-
Data presented at the
American Academy of Neurology (AAN) Meeting provided continued evidence that patisiran has the potential to halt neuropathy progression in patients with hATTR-PN. In the first reported exploratory analysis of its kind, the degree of TTR knockdown observed in patients was shown to correlate with improvement in neuropathy impairment scores. Further, patisiran was found to be generally well tolerated with no drug related serious adverse events up to 25 months of treatment. The majority of adverse events were mild to moderate.
-
Data presented at the
-
Continued enrollment in ENDEAVOUR Phase 3 study with revusiran
for the treatment of hereditary TTR-mediated amyloidosis with
cardiomyopathy (hATTR-CM), also known as familial amyloidotic
cardiomyopathy (FAC).
- The Company announced today that it expects to complete ENDEAVOUR enrollment in late 2016 and report results in mid-2018.
-
Filed Clinical Trial Application (CTA) for ALN-TTRsc02, an
ESC-GalNAc-siRNA conjugate targeting TTR for the treatment of
ATTR amyloidosis, which is expected to enable a once-
quarterly subcutaneous dosing regimen.
- Assuming a positive Phase 1 study, the Company plans to initiate a Phase 3 trial in 2017.
-
Completed enrollment in the APOLLO Phase 3 trial with
patisiran for the treatment of hereditary TTR-mediated
amyloidosis with polyneuropathy (hATTR-PN), also known as
familial amyloidotic polyneuropathy (FAP).
-
Advanced fitusiran (ALN-AT3) for the treatment of hemophilia and
rare bleeding disorders (RBD).
- Initiated dosing of hemophilia patients with inhibitors in Part D of an ongoing Phase 1 clinical trial evaluating a once-monthly subcutaneous dose regimen of fitusiran. Both patients with hemophilia A with inhibitors and hemophilia B with inhibitors have now been dosed with fitusiran.
- Continued dosing patients in an ongoing Phase 1 OLE, where once-monthly doses of fitusiran are administered to patients with moderate or severe hemophilia A or B with or without inhibitors.
-
Alnylam is on track to initiate two Phase 3 trials: the first
in mid-2016 in hemophilia A and B patients with inhibitors;
and, the second in late 2016 in moderate or severe hemophilia
A and B patients without inhibitors.
- The Company has initiated discussions with global regulatory authorities to confirm specific trial designs.
-
Advanced ALN-CC5 for the treatment of complement-mediated diseases.
-
The Company announced today that it has achieved preliminary
evidence for clinical activity in a small number of paroxysmal
nocturnal hemoglobinuria (PNH) patients enrolled in Part C of
its ongoing Phase 1/2 trial, and it believes that based on LDH
data, the optimal development path for ALN-CC5 in PNH is for
eculizumab poor responders and for eculizumab sparing.
- The Company now plans to transition toward a new Phase 2 study in PNH patients focused on that development plan, which is expected to start by end of the year.
-
The Company plans to present initial data from a small
cohort of PNH patients in the ongoing Phase 1/2 study at
the
European Hematology Association (EHA) Meeting in June, as listed below.
- The Company also expects to initiate studies of ALN-CC5 as monotherapy and/or in combination with anti-C5 monoclonal antibodies in additional complement-mediated disease indications, such as atypical hemolytic uremic syndrome (aHUS) and myasthenia gravis, starting in early 2017.
-
The Company announced today that it has achieved preliminary
evidence for clinical activity in a small number of paroxysmal
nocturnal hemoglobinuria (PNH) patients enrolled in Part C of
its ongoing Phase 1/2 trial, and it believes that based on LDH
data, the optimal development path for ALN-CC5 in PNH is for
eculizumab poor responders and for eculizumab sparing.
-
Advanced ALN-AS1 for the treatment of acute hepatic porphyrias.
- Transitioned to Part C in ongoing Phase 1 study where ALN-AS1 is being evaluated in porphyria patients with recurrent attacks.
- The Company plans to initiate a Phase 3 trial in 2017, if the Phase 1 study results are positive.
- Advanced ALN-AAT for the treatment of alpha-1 antitrypsin (AAT) deficiency-associated liver disease.
-
Advanced ALN-GO1 for the treatment of primary hyperoxaluria type 1
(PH1).
- Initiated a Phase 1/2 clinical trial that is being conducted initially in normal healthy volunteers, and then will be conducted in patients with PH1.
- Received Orphan Drug Designations for ALN-GO1 from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA).
-
Added ALN-F12 as a new program in development pipeline.
- ALN-F12 is an RNAi therapeutic targeting factor XII in development for the treatment of hereditary angioedema and for thromboprophylaxis.
-
Advanced investigational pipeline programs in Cardio-Metabolic
Disease STAr.
- The Medicines Company continued enrollment in the Phase 2 ORION-1 trial for ALN-PCSsc.
-
Advanced investigational pipeline programs in Hepatic Infectious
Disease STAr.
- Filed and obtained approval for CTA for ALN-HBV, an RNAi therapeutic targeting the Hepatitis B Virus (HBV) genome for the treatment of HBV infection. The Company is on track to start a Phase 1 study in mid-2016.
-
Expanded Management Team
-
Expanded Management Team with appointments of
Patrick Berreby , Vice President of Supply Chain;Jae Kim , Vice President, Clinical Development; andAndy Orth , Vice President of Commercial Practice.
-
Expanded Management Team with appointments of
-
Advanced investigational RNAi therapeutics programs for the
treatment of transthyrethin (TTR)-mediated amyloidosis (ATTR
amyloidosis).
Upcoming Events in Mid- and Late 2016
-
Alnylam announced today that it will:
-
Present updated human volunteer data from the ongoing ALN-CC5
Phase 1/2 study in a poster presentation on
May 22, 2016 at the 53rdCongress of theEuropean Renal Association -European Dialysis and Transplant Association (ERA-EDTA) inVienna, Austria . -
Present initial ALN-CC5 results in PNH patients during an oral
presentation on
June 11, 2016 at the 21stCongress of theEuropean Hematology Association (EHA) Meeting inCopenhagen, Denmark . -
Present updated fitusiran Phase 1 data during an oral presentation
on
July 27, 2016 at theWorld Federation of Hemophilia (WFH) 2016World Congress inOrlando, Florida .
-
Present updated human volunteer data from the ongoing ALN-CC5
Phase 1/2 study in a poster presentation on
-
Additional upcoming milestones for Alnylam pipeline programs include:
-
In mid-2016, Alnylam plans to:
-
Present 24-month Phase 2 OLE data with patisiran, likely at
the International Symposium on Amyloidosis (ISA) Meeting to be
held
July 3 - 7, 2016 in Uppsala,Sweden , pending abstract acceptance; - Present 12-month Phase 2 OLE data with revusiran, also likely at the ISA Meeting, pending abstract acceptance;
- Start first fitusiran Phase 3 study in hemophilia A and B patients with inhibitors;
- Present initial Phase 1 data for ALN-AAT;
- Start ALN-HBV Phase 1 study; and
- Start ALN-TTRsc02 Phase 1 study.
-
Present 24-month Phase 2 OLE data with patisiran, likely at
the International Symposium on Amyloidosis (ISA) Meeting to be
held
-
In late 2016, Alnylam plans to:
- Present additional Phase 1 and initial Phase 1 OLE data with fitusiran;
- Start second fitusiran Phase 3 study in moderate or severe hemophilia A and B patients without inhibitors;
- Present initial ALN-AS1 data in recurrent attack porphyria patients;
- Present initial ALN-GO1 data in PH1 patients;
- Present initial ALN-TTRsc02 Phase 1 data;
- File a CTA for a new Genetic Medicine program; and
- Consistent with guidance from The Medicines Company, present initial Phase 2 data for ALN-PCSsc.
-
In mid-2016, Alnylam plans to:
Financials
"Alnylam continues to maintain a strong balance sheet, ending the first
quarter of 2016 with approximately
Cash,
At
Credit Agreements
In
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
Conference Call Information
Management will provide an update on the company, discuss first quarter
2016 results, and discuss expectations for the future via conference
call on Monday, May 2, 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 99665704. A
replay of the call will be available beginning at 7:30 p.m. ET on
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 Ionis, Novartis, Roche, Takeda, Merck, 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, ALN-HBV, ALN-TTRsc02, and ALN-F12, 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 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 for studies with patisiran,
revusiran, fitusiran, ALN-CC5, ALN-AS1, ALN-AAT, ALN-GO1, ALN-PCSsc,
ALN-HBV, and ALN-TTRsc02, its expected cash position as of
|
UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (In thousands, except per share amounts) |
||||||||||||
|
Three Months Ended |
||||||||||||
| 2016 | 2015 | |||||||||||
| Net revenues from collaborators | $ | 7,345 | $ | 18,537 | ||||||||
| Operating expenses: | ||||||||||||
| Research and development | 96,273 | 58,035 | ||||||||||
| General and administrative | 21,100 | 12,724 | ||||||||||
| Total operating expenses | 117,373 | 70,759 | ||||||||||
| Loss from operations | (110,028 | ) | (52,222 | ) | ||||||||
| Other income: | ||||||||||||
| Interest income | 1,813 | 1,014 | ||||||||||
| Other income | 5,241 | — | ||||||||||
| Total other income | 7,054 | 1,014 | ||||||||||
| Loss before income taxes | (102,974 | ) | (51,208 | ) | ||||||||
| Benefit from income taxes | — | 431 | ||||||||||
| Net loss | $ | (102,974 | ) | $ | (50,777 | ) | ||||||
| Net loss per common share - basic and diluted | $ | (1.21 | ) | $ | (0.62 | ) | ||||||
| Weighted-average common shares used to compute basic and diluted net loss per common share | 85,277 | 82,074 | ||||||||||
| Comprehensive loss: | ||||||||||||
| Net loss | $ | (102,974 | ) | $ | (50,777 | ) | ||||||
| Unrealized (loss) gain on marketable securities, net of tax | (8,224 | ) | 3,622 | |||||||||
|
Reclassification adjustment for realized gain on marketable
securities
included in net loss |
(5,156 | ) | — | |||||||||
| Comprehensive loss | $ | (116,354 | ) | $ | (47,155 | ) | ||||||
|
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands, except share amounts) |
||||||||||
|
|
|
|||||||||
| 2016 | 2015 | |||||||||
| Cash, cash equivalents and total marketable securities | $ | 1,208,290 | $ | 1,280,951 | ||||||
| Billed and unbilled collaboration receivables | 8,814 | 8,298 | ||||||||
| Prepaid expenses and other assets | 29,097 | 18,030 | ||||||||
| Property and equipment, net | 36,814 | 27,812 | ||||||||
| Investment in equity securities of Regulus Therapeutics Inc. | 33,934 | 51,419 | ||||||||
| Total assets | $ | 1,316,949 | $ | 1,386,510 | ||||||
| Accounts payable, accrued expenses and other liabilities | $ | 49,620 | $ | 46,886 | ||||||
| Total deferred revenue | 70,896 | 68,317 | ||||||||
| Total deferred rent | 7,382 | 6,593 | ||||||||
|
Total stockholders' equity (85.5 million and 85.1 million common
shares issued and outstanding and at |
1,189,051 | 1,264,714 | ||||||||
| Total liabilities and stockholders' equity | $ | 1,316,949 | $ | 1,386,510 | ||||||
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/20160502005867/en/
Investors and Media
or
Investors
Source:
News Provided by Acquire Media
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
MEDIA KIT
Essential assets and documents related to Alnylam
