Aug 06, 2015 Press Release for Alnylam
Alnylam Pharmaceuticals Reports Second Quarter 2015 Financial Results and Highlights Recent Period Activities
Aug 06, 2015
- Advanced Pipeline of Seven Investigational Clinical Programs and Presented Positive Clinical Results for Three: Patisiran for Familial Amyloidotic Polyneuropathy (FAP), ALN-AT3 for Hemophilia, and ALN-CC5 for Complement-Mediated Diseases -
- In Addition to Upcoming Presentation of ALN-PCSsc Clinical Results, Announces New Plan to Present Initial Clinical Data for ALN-AS1 Porphyria Program at Medical Meeting in September and Provides Update on Revusiran Phase 2 Open-Label Extension Study -
- Maintained Strong Balance Sheet with
"The second quarter of 2015 and recent period were marked by tremendous
progress in our efforts to bring innovative medicines to patients, as we
execute on our ‘Alnylam 2020' strategy. In our patisiran Phase 2
open-label extension - or ‘OLE' - study, we believe to have generated
increased evidence for a possible halting of neuropathy progression
after the first 12 months of treatment, with study drug administration
generally well tolerated out to 17 months. In our hemophilia program, we
believe that recent results from our ongoing Phase 1 trial provide
confirmatory evidence that ALN-AT3 has the potential to re-balance
hemostasis in people with severe hemophilia through normalization of
thrombin generation. We also reported initial positive data from our
ongoing Phase 1/2 trial with ALN-CC5 - an investigational RNAi
therapeutic for complement-mediated diseases - where we demonstrated
what we believe to be promising preliminary clinical activity for single
doses of study drug with potent and highly durable knockdown of serum C5
and inhibition of serum complement activity," said
Second Quarter 2015 and Recent Significant Corporate Highlights
-
Advanced pipeline of investigational programs in Genetic Medicine
Strategic Therapeutic Area (STAr).
-
Advanced RNAi therapeutic programs for the treatment of
transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis).
- Continued enrollment in APOLLO Phase 3 study of patisiran in ATTR patients with Familial Amyloidotic Polyneuropathy (FAP).
- Initiated Phase 3 open-label extension study ("APOLLO-OLE") with patisiran.
- Reported positive 12-month clinical data from patisiran Phase 2 OLE study, showing sustained TTR knockdown of up to a mean 88% and continued evidence for potential halting of neuropathy progression with a mean 2.5 point decrease in neuropathy impairment score (mNIS+7) at 12 months, comparing favorably to a 13-18 point increase estimated from the literature in untreated FAP patients with similar baseline characteristics; patisiran was also found to be generally well tolerated out to 17 months of study drug administration.
- Continued enrollment in ENDEAVOUR Phase 3 study of revusiran in ATTR patients with Familial Amyloidotic Cardiomyopathy (FAC).
-
Continued dosing FAC and senile systemic amyloidosis (SSA)
patients in revusiran Phase 2 OLE study, which was initiated
in
November 2014 and is designed to evaluate the tolerability and clinical activity of revusiran with long-term dosing for up to two years.-
The company reports today that three patients have
discontinued from the revusiran Phase 2 OLE study as of
August 5 due to injection site reactions (ISRs), including some with associated diffuse rash; the study drug remains otherwise generally well tolerated in the broader revusiran Phase 2 OLE study population. The company plans to present initial clinical results from the revusiran Phase 2 OLE study in late 2015.
-
The company reports today that three patients have
discontinued from the revusiran Phase 2 OLE study as of
-
Received Orphan Drug Designation (ODD) from the
United States Food & Drug Administration (FDA) for revusiran. -
The company announces today that it is advancing a Development
Candidate (DC) for an ESC-GalNAc-siRNA targeting TTR that is
expected to support a once-monthly and possibly once-quarterly
subcutaneous dose regimen. Further details and guidance on
this program will be presented at the
Oligonucleotide Therapeutics Society meeting to be heldOctober 11 - 14, 2015 in Leiden,The Netherlands .
-
Advanced ALN-AT3 for the treatment of hemophilia and rare bleeding
disorders (RBD).
- Reported positive interim results from the ongoing Phase 1 study, including an up to 86% knockdown of antithrombin (AT), and evidence for a potential re-balancing of hemostasis resulting in normalization of thrombin generation up to a mean increase of 350% and marked improvements in whole blood clotting. In addition, in a post hoc exploratory analysis, AT knockdown was found to be associated with reduced bleeding frequency, with the maximum bleed-free interval of 114 days in a patient with severe hemophilia A. ALN-AT3 was also shown to be generally well tolerated, without any clinically significant increases in D-dimer, a marker of pathologic clot formation.
-
Presented new
pre-clinical results with ALN-AT3 and published pre-clinical
study results in Nature Medicine.
-
These published pre-clinical data were the centerpiece of
a recent "Clinical
Implications" article in the
New England Journal of Medicine .
-
These published pre-clinical data were the centerpiece of
a recent "Clinical
Implications" article in the
-
Presented initial positive
Phase 1/2 study results with ALN-CC5 for the treatment of
complement-mediated diseases.
- Single subcutaneous doses of study drug achieved potent, statistically significant, and highly durable C5 knockdown of up to 96% and an up to 92% inhibition of serum complement activity, including an up to 61% inhibition of serum hemolytic activity; single doses of ALN-CC5 were generally well tolerated.
-
Initiated Phase 1 study with ALN-AS1 for the treatment of acute
hepatic porphyrias.
- The trial is being conducted initially in asymptomatic high excreter (ASHE) patients with acute intermittent porphyria (AIP), and then in AIP patients with recurrent attacks.
-
As detailed below, the company now plans to present initial
clinical data at a medical meeting in
mid-September 2015 .
-
Advanced ALN-AAT for the treatment of alpha-1 antitrypsin (AAT)
deficiency-associated liver disease (alpha-1 liver disease).
- Initiated Phase 1/2 study with ALN-AAT. The Phase 1/2 trial is being conducted initially in normal healthy volunteers, and, then in patients with alpha-1 liver disease.
-
Presented new
pre-clinical data at the Digestive Disease Week (DDW)
meeting held in
May 2015 .
-
Advanced ALN-GO1 for the treatment of primary hyperoxaluria.
- Continued pre-clinical studies to support selection of a DC for ALN-GO1 in mid-2015.
-
Advanced 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.
- The company announces today that it has completed enrollment in the ALN-PCSsc Phase 1 study in normal human volunteers with elevated LDL-C at baseline.
- Continued pre-clinical studies to support a Clinical Trial Application (CTA) for ALN-HBV in late 2015.
-
Signed 295,000 square foot lease with an affiliate of BioMed Realty
for Class A laboratory and office space in
Cambridge for the company's future corporate headquarters. - Appointed David-Alexandre "DA" Gros, M.D. to the position of Senior Vice President, Chief Business Officer.
Upcoming 3Q2015 Events
-
Alnylam announces today that it now plans to present initial single
ascending dose (SAD) cohort data from its ALN-AS1 Phase 1 study in
ASHE patients at the
International Congress of Porphyrins and Porphyrias (ICPP) 2015, being heldSeptember 14 - 16, 2015 in Düsseldorf,Germany , in an oral presentation onTuesday, September 15 .- In addition, during the ICPP meeting, Alnylam plans to present initial data from the EXPLORE trial, a prospective observational study to characterize the disease burden in patients with hepatic porphyrias.
-
The company also announces today that it plans to present complete
12-month data from the Phase 2 OLE study with patisiran at the
American Neurological Association (ANA) 2015 Annual Meeting, being heldSeptember 27 - 29, 2015 inChicago , in a poster presentation onMonday, September 28 at5:30 pm CT (6:30 pm ET ). -
In addition, Alnylam plans to:
-
Present data from the Phase 1 study of ALN-PCSsc in development
for the treatment of hypercholesterolemia at the
European Society of Cardiology (ESC) Congress 2015, being heldAugust 29 -September 2, 2015 inLondon , in a poster presentation onSunday, August 30 at8:30 am British Summer Time (3:30 am ET ). -
Present pre-clinical data for ALN-GO1 in development for the
treatment of Primary Hyperoxaluria Type 1 (PH1) at the 48th
Annual Scientific Meeting of the
European Society of Paediatric Nephrology (ESPN), being heldSeptember 3 - 5, 2015 inBrussels, Belgium , in an oral presentation onSeptember 5 .
-
Present data from the Phase 1 study of ALN-PCSsc in development
for the treatment of hypercholesterolemia at the
Upcoming RNAi Roundtables
Alnylam plans to continue hosting its series of online "RNAi Roundtables" in August and September, including events focused on the following RNAi therapeutics:
ALN-AAT for the treatment of AAT Deficiency-associated liver disease
- Alfica Sehgal, Ph.D., Principal Scientist, Research
-
Moderator:
Akshay Vaishnaw , M.D., Ph.D., Executive Vice President of R&D, Chief Medical Officer -
Guest Speaker:
Jeffrey Teckman , M.D., Professor,Department of Pediatrics ,St. Louis University School of Medicine
Patisiran and Revusiran for the treatment of Transthyretin
(TTR)-Mediated Amyloidosis
-
Eric Green , Vice President, General Manager, TTR Program -
Jared Gollob , M.D., Vice President, Clinical Research -
Moderator:
Barry Greene , President and Chief Operating Officer -
Guest Speaker:
Philip Hawkins , Ph.D., FRCP, FRCPath, FMedSci, Head, National Amyloidosis Centre, and Head, Periodic Fever Syndrome Service/Honorary consultant physician -
Guest Speaker:
Isabelle Lousada , President & CEO,Amyloidosis Research Consortium , and Chairman,Amyloidosis Foundation
The company announces today that it has scheduled additional RNAi Roundtables:
ALN-GO1 for the treatment of Primary Hyperoxaluria Type 1 (PH1)
-
David Erbe , Ph.D., Director, Research -
Moderator:
Barry Greene , President and Chief Operating Officer -
Guest Speaker:
Sally-Anne Hulton , M.D., FRCPCH, MRCP, FCP, MBBCh, Consultant Paediatric Nephrologist and Clinical Lead,Birmingham Children'sHospital NHS Trust
ALN-PCSsc for the treatment of Hypercholesterolemia
-
Kevin Fitzgerald , Ph.D., Vice President, Research -
David Kallend ,MBBS , Vice President and Global Medical Director, The Medicines Company -
Moderator:
Barry Greene , President and Chief Operating Officer -
Guest Speaker:
Marc S. Sabatine , M.D., M.P.H., Chairman, Thrombolysis inMyocardial Infarction (TIMI) Study Group at Brigham and Women's Hospital,Lewis Dexter , MD Distinguished Chair in Cardiovascular Medicine, and Professor of Medicine,Harvard Medical School
ALN-AS1 for the treatment of Acute Hepatic Porphyrias
-
Bill Querbes , Ph.D., Associate Director, Research -
Moderator:
John Maraganore , Ph.D., Chief Executive Officer -
Guest Speaker:
Robert J. Desnick , M.D., Ph.D., Dean for Genetics and Genomic Medicine, Professor and Chair Emeritus,Department of Genetics and Genomic Sciences ,Icahn School of Medicine atMount Sinai Hospital
Financials
"Alnylam continues to maintain a very strong balance sheet, with approximately $1.4 billion in cash as of the end of the second quarter of 2015," said Michael Mason, Vice President, Finance & Treasurer. "As for financial guidance this year, we remain on track to end 2015 with greater than $1.2 billion in cash."
Cash,
At
Non-GAAP Net Loss
The non-GAAP net loss for the second quarter of 2015 was
GAAP Net Loss
The net loss according to accounting principles generally accepted in
the U.S. (GAAP) for the second quarter of 2015 was
Revenues
Revenues were
Research and Development Expenses
Research and development (R&D) expenses were
In the second quarter of 2014, the company recorded a reduction of
General and Administrative Expenses
General and administrative (G&A) expenses were
Conference Call Information
Management will provide an update on the company, discuss second 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.
About GalNAc Conjugates and Enhanced Stabilization Chemistry (ESC) GalNAc Conjugates
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.
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,
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 its ESC-GalNAc-siRNA targeting TTR, 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, its
expectations regarding reporting of data from its clinical and
pre-clinical studies, including its studies for patisiran, revusiran,
ALN-PCSsc, ALN-AS1, ALN-HBV, ALN-GO1 and its ESC-GalNAc-siRNA targeting
TTR, 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 |
Six Months Ended |
|||||||||||
| 2015 | 2014 | 2015 | 2014 | |||||||||
| Net revenues from collaborators | $ | 8,685 | $ | 7,295 | $ | 27,222 | $ | 15,570 | ||||
| Operating expenses: | ||||||||||||
|
Research and development (1) |
67,007 | 44,672 | 125,042 | 88,430 | ||||||||
| In-process research and development | — | (3,890) | — | 220,766 | ||||||||
| General and administrative (1) | 14,622 | 11,518 | 27,346 | 20,443 | ||||||||
| Total operating expenses | 81,629 | 52,300 | 152,388 | 329,639 | ||||||||
| Loss from operations | (72,944) | (45,005) | (125,166) | (314,069) | ||||||||
| Other income (expense): | ||||||||||||
| Interest income | 1,619 | 693 | 2,633 | 1,026 | ||||||||
| Other expense | (27) | (77) | (27) | (159) | ||||||||
| Total other income | 1,592 | 616 | 2,606 | 867 | ||||||||
| Loss before income taxes | (71,352) | (44,389) | (122,560) | (313,202) | ||||||||
| (Provision for) Benefit from income taxes | (431) | 315 | — | 18,185 | ||||||||
| Net loss | $ | (71,783) | $ | (44,074) | $ | (122,560) | $ | (295,017) | ||||
| Net loss per common share - basic and diluted | $ | (0.85) | $ | (0.58) | $ | (1.47) | $ | (4.11) | ||||
| Weighted average common shares used to compute basic and diluted net loss per common share | 84,353 | 75,835 | 83,219 | 71,833 | ||||||||
| Comprehensive loss: | ||||||||||||
| Net loss | $ | (71,783) | $ | (44,074) | $ | (122,560) | $ | (295,017) | ||||
| Unrealized (loss) gain on marketable securities, net of tax | (33,623) | (3,047) | (30,001) | 2,266 | ||||||||
| Comprehensive loss | $ | (105,406) | $ | (47,121) | $ | (152,561) | $ | (292,751) | ||||
| (1) Non-cash stock-based compensation expenses included in operating expenses are as follows: | ||||||||||||
| Research and development | $ | 6,149 | $ | 2,557 | $ | 11,495 | $ | 6,238 | ||||
| General and administrative | 4,030 | 5,123 | 6,920 | 7,033 | ||||||||
|
|
||||||||||||
|
Unaudited GAAP to Non-GAAP Reconciliation: Net Loss and Net Loss Per Share |
||||||||||||
|
(In thousands, except per share amounts) |
||||||||||||
|
Three Months Ended |
Six Months Ended |
|||||||||||
| 2015 | 2014 | 2015 | 2014 | |||||||||
| GAAP net loss | $ | (71,783) | $ | (44,074) | $ | (122,560) | $ | (295,017) | ||||
| Adjustment: | ||||||||||||
| In-process research and development | — | (3,890) | — | 220,766 | ||||||||
| Non-GAAP net loss | $ | (71,783) | $ | (47,964) | $ | (122,560) | $ | (74,251) | ||||
| GAAP net loss per common share - basic and diluted | $ | (0.85) | $ | (0.58) | $ | (1.47) | $ | (4.11) | ||||
| Adjustment (as detailed above) | — | (0.05) | — | 3.08 | ||||||||
| Non-GAAP net loss per common share - basic and diluted | $ | (0.85) | $ | (0.63) | $ | (1.47) | $ | (1.03) | ||||
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 second
quarter of 2014 and first half 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 reduction to
expense of
|
|
||||||
|
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS |
||||||
|
(In thousands, except share amounts) |
||||||
|
|
|
|||||
| 2015 | 2014 | |||||
| Cash, cash equivalents and total marketable securities | $ | 1,396,800 | $ | 881,929 | ||
| Billed and unbilled collaboration receivables | 9,403 | 39,937 | ||||
| Prepaid expenses and other current assets | 15,117 | 9,739 | ||||
| Deferred tax assets | 19,654 | 31,667 | ||||
| Property and equipment, net | 25,169 | 21,740 | ||||
| Investment in equity securities of Regulus Therapeutics Inc. | 64,628 | 94,583 | ||||
| Total assets | $ | 1,530,771 | $ | 1,079,595 | ||
| Accounts payable and accrued expenses | $ | 33,473 | $ | 38,791 | ||
| Deferred tax liabilities | 19,654 | 31,667 | ||||
| Total deferred revenue | 62,716 | 66,854 | ||||
| Total deferred rent | 6,324 | 6,016 | ||||
|
Total stockholders' equity (84.5 million and 77.2 million common
shares issued and outstanding and at |
1,408,604 | 936,267 | ||||
| Total liabilities and stockholders' equity | $ | 1,530,771 | $ | 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/20150806006291/en/
Vice
President, Finance and Treasurer
or
Senior Manager, Investor Relations and
Corporate
Communications
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
