Alnylam Pharmaceuticals Reports Second Quarter 2016 Financial Results and Highlights Recent Period Progress
- Advanced Two Programs into Phase 1 Clinical Studies: ALN-TTRsc02 for Transthyretin-Mediated Amyloidosis and ALN-HBV for Chronic Hepatitis B Virus Infection -
- Presented Clinical Data from Patisiran, Revusiran, Fitusiran, and ALN-CC5 Programs -
- Maintained Strong Balance Sheet with $1.28 Billion in Cash and Remains On Track to End 2016 with Greater than $1.0 Billion in Cash -
"We continue to advance our pipeline of now ten investigational RNAi
therapeutics, an entirely new and innovative class of medicines, across
a broad range of diseases. We believe our two latest stage programs,
patisiran and revusiran, have demonstrated encouraging progress for
patients with hATTR amyloidosis," said
Second Quarter 2016 and Recent Significant Corporate Highlights
Advanced investigational pipeline programs in Genetic Medicine
Strategic Therapeutic Area (STAr).
Advanced investigational RNAi therapeutic programs for the
treatment of transthyrethin (TTR)-mediated amyloidosis (ATTR
- Reported positive initial 24-month data from ongoing Phase 2 open-label extension (OLE) study with patisiran for the treatment of hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN). Results showed that patisiran can potentially halt or improve neuropathy progression. Patisiran administration was also found to be generally well tolerated in hATTR-PN patients out to 25 months, with no drug-related serious adverse events (SAEs) reported through the data transfer date.
- Presented baseline demographic data from APOLLO Phase 3 study of patisiran, revealing enrollment of a globally representative patient population with a wide range of disease severity and TTR mutations.
- Reported initial 12-month results from ongoing Phase 2 OLE study with revusiran for the treatment of hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM).
- Initiated Phase 1 clinical trial for ALN-TTRsc02, an Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA conjugate targeting TTR for the treatment of ATTR amyloidosis, which is expected to enable a low volume, once quarterly, subcutaneous dosing regimen.
positive interim clinical results from Phase 1 study of fitusiran
for the treatment of hemophilia and rare bleeding disorders (RBD).
- Fitusiran achieved a median estimated annualized bleeding rate (ABR) of zero in hemophilia patients without inhibitors. In the initial low-dose cohort of patients with inhibitors, fitusiran achieved antithrombin lowering, increased thrombin generation, and preliminary evidence for reduced bleeding. Fitusiran administration was generally well tolerated in patients with and without inhibitors, with no SAEs related to study drug, and no thromboembolic events or laboratory evidence (based on D-dimer, platelet count, fibrinogen, and/or PT/INR) of pathologic clot formation through the data transfer date.
- Continued dosing hemophilia patients in ongoing Phase 1/2 OLE study, with patients currently having received up to 13 months of dosing.
- The Company also updated its guidance for Phase 3 initiation, and now plans to start studies in early 2017.
initial clinical results in patients with paroxysmal nocturnal
hemoglobinuria (PNH) from ongoing Phase 1/2 study of ALN-CC5
for the treatment of complement-mediated diseases.
- Initial data support the potential for ALN-CC5 to reduce the dose and frequency of eculizumab, as well as to improve disease control in eculizumab inadequate responders.
- ALN-CC5 was generally well tolerated in patients with PNH after multiple doses, with the majority of adverse events (AEs) being mild or moderate in severity. There were no drug related SAEs or discontinuations due to AEs in the study through the data transfer date.
The Company announces today that the
European Medicines Agency(EMA) has granted Orphan Drug Designation to ALN-AS1 for the treatment of acute hepatic porphyrias.
The Company also announces today the publication of pre-clinical
data with ALN-GO1 for the treatment of primary hyperoxaluria type
1 (PH1) in the
Journal of the American Society of Nephrology(Liebow et al., J Am Soc Nephrol, 2016; doi:10.1681/ASN.2016030338).
- Advanced investigational RNAi therapeutic programs for the treatment of transthyrethin (TTR)-mediated amyloidosis (ATTR amyloidosis).
Advanced investigational pipeline programs in Cardio-Metabolic Disease
- The Medicines Company announced completion of enrollment in its Phase 2 ORION-1 study for ALN-PCSsc (also known as PCSK9si). The trial enrolled 501 patients with atherosclerotic cardiovascular disease (ASCVD), exceeding the original enrollment target of 480 patients.
Advanced investigational pipeline programs in Hepatic Infectious
- Initiated Phase 1/2 clinical trial with ALN-HBV for the treatment of hepatitis B virus (HBV) infection.
Broke ground on new manufacturing facility in
Norton, Massachusetts. The 200,000 square foot, state-of-the-art facility is being built to support the Company's expanding development pipeline and transition toward commercial stage.
Expanded Management Team with appointments of
Adrian Dana, M.D., Vice President of Drug Safety and Pharmacovigilance; Brendan Martin, General Manager, UKand Ireland; Jeffrey Miller, Vice President, General Manager, CC5 Program; and Andrew Slugg, Vice President of Regulatory Affairs.
Upcoming Events in 2H 2016
Alnylam announces today that it plans to present clinical data at
these upcoming conferences:
Complete data from Parts A and B (single and multiple ascending
dose, respectively) of the ongoing Phase 1 clinical trial of
ALN-AS1 in patients who are asymptomatic "high excreters" (ASHE)
at the 2016
Society for the Study of Inborn Errorsof Metabolism (SSIEM) Annual Symposium, being held September 6- 9, 2016 in Rome, Italy, in an oral presentation on Wednesday, September 7, at 2:15 pmCentral European Summer Time( 8:15 am ET).
Initial clinical results from the ongoing Phase 1/2 study of
ALN-GO1 at the 17th
Congressof the International Pediatric Nephrology Association(IPNA), being held September 20- 24, 2016 in Iguaçu, Brazil, in an oral presentation on Saturday, September 24, at 3:25 pmBrasília Time ( 2:25 pm ET).
Initial Phase 1/2 data for ALN-AAT at the 12th Annual Meeting of
Oligonucleotide Therapeutics Society(OTS), being held September 25- 28, 2016 in Montreal, Canada;
- Complete data from Parts A and B (single and multiple ascending dose, respectively) of the ongoing Phase 1 clinical trial of ALN-AS1 in patients who are asymptomatic "high excreters" (ASHE) at the 2016
Alnylam also announces today that it plans to host an R&D Day on the
Friday, December 16, 2016at the Westin New Yorkat Times Squarein New York City.
In addition, in the second half of 2016, Alnylam plans to:
Complete enrollment in
ENDEAVOURPhase 3 study of revusiran;
- Present additional clinical data from the fitusiran Phase 1 study and initial data from the Phase 1/2 OLE study;
- Start a new Phase 2 trial with ALN-CC5 in inadequate eculizumab responder PNH patients;
- Present additional clinical data from the ongoing Phase 1/2 trial of ALN-CC5 in PNH patients;
- Present initial ALN-AS1 data in recurrent attack porphyria patients;
- Present initial ALN-TTRsc02 Phase 1 data;
- File a Clinical Trial Application for a new Genetic Medicine program; and
- Consistent with guidance from The Medicines Company, present initial Phase 2 data for ALN-PCSsc.
- Complete enrollment in
Upcoming RNAi Roundtables
Alnylam plans to continue hosting its series of online "RNAi
Roundtables" in August, September, and October. Upcoming events
Fitusiran for the treatment of hemophilia and rare bleeding
Monday, August 22, 10:30 - 11:45 am ET
Akin Akinc, Ph.D., Vice President, General Manager, Fitusiran
Benny Sorensen, M.D., Ph.D., Senior Director, Clinical Research
Brian O'Mahony, Chief Executive, Irish Haemophilia Society Ltd.and person living with severe hemophilia B
ALN-CC5 for the treatment of complement-mediated diseases
Wednesday, August 31, 11:00 am- 12:00 pm ET
Jeff Miller, Vice President, General Manager, CC5 Program
Pushkal Garg, M.D., Senior Vice President, Clinical Development
Anita Hill, M.D., Ph.D., MRCP, FRCPath, Consultant Haematologist for Leeds Teaching Hospitals NHS Trust, UK, and Lead for the National PNH Service in England
ALN-AS1 for the treatment of acute hepatic porphyrias
Tuesday, September 13, 11:30 am- 12:45 pm ET
John Maraganore, Ph.D., Chief Executive Officer
William Querbes, Ph.D., Associate Director, Research
Ariel Lager, living with Acute Intermittent Porphyria
ALN-GO1 for the treatment of primary hyperoxaluria type 1 (PH1)
Tuesday, September 27, 10:00 - 11:00 am ET
Barry Greene, President and Chief Operating Officer
David Erbe, Ph.D., Director, Research
Sally-Anne Hulton, M.D., FRCPCH, MRCP, FCP, MBBCh, Consultant Paediatric Nephrologist and Clinical Lead, Birmingham Children's Hospital NHS Trust
Jennifer Lawrence, M.D. (mother of George Tidmore, a PH1 patient)
ALN-HBV for the treatment of hepatitis B virus (HBV) infection
Tuesday, October 11, 9:00 am- 10:00 am ET
Barry Greene, President and Chief Operating Officer
Laura Sepp-Lorenzino, Ph.D., Vice President, Entrepreneur-in-Residence
Heiner Wedemeyer, M.D., Managing Senior Physician and Assistant Professor in the Department of Gastroenterology, Hepatology and Endocrinologyat Hannover Medical School
- Fitusiran for the treatment of hemophilia and rare bleeding disorders
Additional details for the RNAi Roundtables will be provided at www.alnylam.com/roundtables.
"Alnylam continues to maintain a strong balance sheet, ending the second
quarter of 2016 with approximately
Cash and Investments
GAAP Net Loss
The net loss according to accounting principles generally accepted in
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 second quarter
2016 results, and discuss expectations for the future via conference
call on Thursday, August 4, 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 56306704. 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
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.
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, and ALN-HBV, its expectations for the timing of
filing of regulatory documents, its expectations regarding the timing of
clinical studies and the presentation of clinical data, including for
The scientific information discussed in this news release relating to
Alnylam's investigational therapeutics is preliminary and investigative.
None of Alnylam's investigational therapeutics have been approved by the
UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
(In thousands, except per share amounts)
Three Months Ended
Six Months Ended
|Net revenues from collaborators||$||8,709||$||8,685||$||16,054||$||27,222|
|Research and development||83,172||67,007||179,445||125,042|
|General and administrative||17,987||14,622||39,087||27,346|
|Total operating expenses||101,159||81,629||218,532||152,388|
|Loss from operations||(92,450)||(72,944)||(202,478)||(125,166)|
|Other income (expense)||229||(27)||5,470||(27)|
|Total other income||2,321||1,592||9,375||2,606|
|Loss before income taxes||(90,129)||(71,352)||(193,103)||(122,560)|
|Provision for income taxes||—||(431)||—||—|
|Net loss per common share - basic and diluted||$||(1.05)||$||(0.85)||$||(2.26)||$||(1.47)|
|Weighted-average common shares used to compute basic and diluted net loss per common share||85,545||84,353||85,411||83,219|
|Unrealized loss on marketable securities, net of tax||(18,331)||(33,623)||(26,555)||(30,001)|
Reclassification adjustment for realized gain on marketable securities included in net loss
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except share amounts)
|Cash, cash equivalents and marketable securities||$||1,130,299||$||1,280,951|
|Billed and unbilled collaboration receivables||9,514||8,298|
|Prepaid expenses and other assets||23,623||18,030|
|Property and equipment, net||55,394||27,812|
|Investment in equity securities of Regulus Therapeutics Inc.||13,332||51,419|
|Accounts payable, accrued expenses and other liabilities||$||52,152||$||46,886|
|Total deferred revenue||73,695||68,317|
|Total deferred rent||7,833||6,593|
|Long term debt||150,000||—|
Total stockholders' equity (85.6 million and 85.1 million common
shares issued and outstanding and at
|Total liabilities and stockholders' equity||$||1,382,162||$||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
(Investors and Media)
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