May 01, 2019 Press Release for Alnylam
Alnylam Pharmaceuticals Reports First Quarter 2019 Financial Results and Highlights Recent Period Activity
May 01, 2019
− Achieved First Quarter 2019 ONPATTRO® Global Net Product Revenues of
− Achieved Positive Results in ENVISION Phase 3 Study of Givosiran –
− Formed Broad Collaboration with Regeneron to Discover, Develop and Commercialize RNAi Therapeutics Focused on Ocular and Central Nervous System (CNS) Diseases –
− Strengthened Balance Sheet with
“The first quarter of 2019 and recent weeks were a remarkable period of advancement for Alnylam toward our goal of building a leading biopharmaceutical company. Specifically, we demonstrated good progress in global commercialization of ONPATTRO, continued success in our R&D efforts with positive Phase 3 results for givosiran, and a strong commitment to future pipeline growth through our landmark ocular and CNS disease alliance with Regeneron. In addition, we significantly strengthened our balance sheet through both an equity offering in January and partnership-based equity funding and cash that we’ll receive upon closing of the Regeneron transaction,” said
“In the first quarter, we made significant progress in our global commercialization of ONPATTRO, as we advanced our efforts to help patients secure access to this important new medicine. With over 400 patients on commercial product, we’re very pleased with our team’s launch execution in over 10 countries where ONPATTRO is now available, and we look forward to continued growth in all existing and many new countries in the months to come. Of note, we saw strong new patient demand in the U.S. and EU in the first quarter, beyond the initial stages of our launch that had been largely driven by conversion of patients from our expanded access program as well as patients known to clinical study sites,” said
First Quarter 2019 and Recent Significant Corporate Highlights
Commercial Performance in First Quarter 2019
- Achieved global net product revenues for the first quarter of 2019 of
$26.3 million for ONPATTRO. - Attained over 400 patients worldwide on commercial ONPATTRO treatment since launch as of
March 31, 2019 . - Received 77 Start Forms in the U.S. in the first quarter, with over 90 percent from newly identified patients not previously treated in the ONPATTRO Expanded Access Program.
- Start Forms in the first quarter came from a diverse range of prescribing physician specialties, including 55 percent from cardiologists and 35 percent from neurologists, with 65 percent of patients covered by
Medicare . - In addition, the Company reports continued strength in the number of U.S. patients receiving ONPATTRO from channels outside of its Alnylam Assist patient hub where the Company does not receive Start Forms.
- Start Forms in the first quarter came from a diverse range of prescribing physician specialties, including 55 percent from cardiologists and 35 percent from neurologists, with 65 percent of patients covered by
- Reported continued progress with value-based agreements (VBAs) with commercial payers in the U.S. and with market access efforts globally.
- Since launch, Alnylam has achieved access to ONPATTRO, if prescribed, for greater than 90 percent of U.S. lives across commercial,
Medicare ,Medicaid , and other government payer categories. In addition, Alnylam has completed definitive VBAs with 10 commercial U.S. payers to date. - In the EU, Alnylam reported favorable Health Technology Assessment (HTA) ratings from health authorities in several countries, including in France (“ASMR III”), Germany (“considerable benefit”), and the Netherlands where a first-of-its-kind VBA was reached with all health insurers to reimburse ONPATTRO. The Company remains on track with pricing and reimbursement procedures in nearly all EU markets, and encouraging discussions with authorities have progressed significantly over the quarter.
- Since launch, Alnylam has achieved access to ONPATTRO, if prescribed, for greater than 90 percent of U.S. lives across commercial,
R&D Highlights
- Advanced patisiran (the non-branded name for ONPATTRO), an intravenously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis.
- Obtained alignment with the
U.S. Food and Drug Administration (FDA ) on the design of APOLLO-B, a randomized, double-blind, placebo-controlled Phase 3 study of patisiran in hereditary and wild-type ATTR amyloidosis patients with cardiomyopathy, with the goal of starting the trial in mid-2019.
- Obtained alignment with the
- Advanced vutrisiran (ALN-TTRsc02), a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis.
- Continued enrollment in the HELIOS-A Phase 3 study of vutrisiran in hereditary ATTR amyloidosis patients with plans to initiate additional Phase 3 studies, including in hereditary and wild-type ATTR amyloidosis cardiomyopathy, in late 2019.
- Advanced givosiran, an investigational RNAi therapeutic in development for the treatment of acute hepatic porphyria (AHP).
- Achieved positive results in the ENVISION Phase 3 study and presented data at the
European Association for the Study of the Liver (EASL) 54th AnnualInternational Liver Congress . - On track to complete submission of a New Drug Application (NDA) and submit a Marketing Authorisation Application (MAA) for givosiran in mid-2019.
- Achieved positive results in the ENVISION Phase 3 study and presented data at the
- Advanced lumasiran, an investigational RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1).
- Continued enrollment in the ILLUMINATE-A Phase 3 study of lumasiran in PH1 patients aged six or older with mild-to-moderate renal impairment, and remain on track to report results by year-end 2019.
- Presented new positive results from the ongoing Phase 2 open-label extension (OLE) study of lumasiran at the
International Society of Nephrology (ISN) 2019 Annual Meeting. - Initiated ILLUMINATE-B, a global Phase 3 pediatric study of lumasiran in PH1 patients under six years of age.
- Alnylam’s partner, The
Medicines Company , announced in April that the Independent Data Monitoring Committee for ongoing inclisiran Phase 3 clinical trials (ORION 9, 10, and 11) conducted its sixth planned review of safety and efficacy data from the ORION trials and recommended that the trials continue without modification.- The safety database for inclisiran now provides more than 3,000 patient-years of exposure to an RNAi therapeutic, representing the industry’s most comprehensive body of safety data for an RNA therapeutic.
- Alnylam’s partner,
Sanofi , continues enrollment in the ATLAS Phase 3 program with fitusiran in patients with hemophilia A or B with and without inhibitors. - Advanced early- and mid-stage RNAi clinical pipeline.
- Initiated a Phase 2 study of cemdisiran, an investigational RNAi therapeutic targeting complement C5 for the treatment of complement-mediated diseases, in IgA nephropathy.
- Filed a Clinical Trial Authorisation (CTA) application and received approval to initiate a Phase 1 study of ALN-AGT, an investigational RNAi therapeutic targeting angiotensinogen (AGT) for the treatment of hypertension in high unmet need populations, including patients with resistant or refractory hypertension, chronic kidney disease or heart failure.
Additional Business Highlights
- Announced a broad collaboration with
Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize RNAi therapeutics focused on ocular and CNS diseases. Subject to Hart-Scott-Rodino (HSR) clearance, the Company anticipates closing this transaction during the second quarter of 2019. - Announced the conclusion of the research and option phase of the Company’s 2014 collaboration with
Sanofi focused on advancing RNAi therapeutics for rare genetic diseases. - Entered into a collaboration with 23andMe to support the addition of a new Genetic Health Risk report for Hereditary Amyloidosis (TTR-related), which will help 23andMe customers who have opted in to receive such reports learn more about their genetic risk for the three most common TTR variants in
the United States (V122I, V30M, and T60A). Read more about the report here.
Upcoming Events in Mid-2019
- Alnylam expects to receive regulatory approvals for ONPATTRO in
Japan andCanada . - Alnylam plans to complete its rolling NDA submission and file an MAA for givosiran.
- Alnylam expects to complete enrollment in the ILLUMINATE-A Phase 3 study of lumasiran.
- The Company also expects to initiate the ILLUMINATE-C Phase 3 study of lumasiran in patients with impaired renal function.
- Alnylam expects to initiate the APOLLO-B Phase 3 study of patisiran in hereditary and wild-type ATTR amyloidosis patients with cardiomyopathy.
- Alnylam’s partner, The
Medicines Company , expects to report initial topline results from the inclisiran Phase 3 clinical trials in the third quarter.
Financial Results for the Quarter Ended
“2019 is off to a strong start for Alnylam, as we ended the first quarter with a balance sheet of
Cash and Investments
At
In
Cash at
GAAP and Non-GAAP Net Loss
The net loss according to accounting principles generally accepted in the U.S. (GAAP) for the first quarter of 2019 was
The non-GAAP net loss for the first quarter of 2019 was
See “Use of Non-GAAP Financial Measures” below for a description of non-GAAP financial measures and a reconciliation between GAAP and non-GAAP net loss appearing later in this press release.
ONPATTRO Revenues, Net
Net product revenues from sales of ONPATTRO were
Net Revenues from Collaborators
Net revenues from collaborators were
GAAP research and development (R&D) expenses were
Non-GAAP R&D expenses were
GAAP and Non-GAAP Selling, General and Administrative Expenses
GAAP selling, general and administrative (SG&A) expenses were
Non-GAAP SG&A expenses were
2019 Updated Financial Guidance
Alnylam is updating its 2019 annual non-GAAP R&D expenses to be in the range of
The Company expects its current cash, cash equivalents, and marketable debt securities will support company operations for multiple years based upon its current operating plan.
Use of Non-GAAP Financial Measures
This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses and non-recurring gains outside the ordinary course of the Company’s business. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies.
The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in the press release are stock-based compensation expense and the gain on litigation settlement. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards. The Company has excluded the impact of the gain on litigation settlement because the Company believes this item is a one-time event occurring outside the ordinary course of the Company’s business.
The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between GAAP and non-GAAP measures is provided later in this press release.
The Company does not provide in this press release a reconciliation of its estimated 2019 non-GAAP R&D and non-GAAP SG&A expense guidance to the comparable GAAP measures because it is not able to estimate 2019 stock-based compensation expense without unreasonable efforts. The Company’s stock-based compensation expense is subject to significant fluctuations from period to period due to variability in the probability of performance-based vesting events for stock options and restricted stock units and changes in the Company’s stock price which materially impacts the recognition, timing of expense and fair value of these awards. In addition, the Company believes such reconciliations for its 2019 financial guidance would imply a degree of precision that would be confusing or misleading to investors.
Conference Call Information
Management will provide an update on the Company and discuss first quarter 2019 results as well as expectations for the future via conference call on
About ONPATTRO® (patisiran)
Patisiran, based on Nobel Prize-winning science, is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis. It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. Patisiran blocks the production of TTR in the liver, reducing its accumulation in the body’s tissues in order to halt or slow down the progression of the disease. In
Important Safety Information
Infusion-Related Reactions
Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. In a controlled clinical study, 19 percent of ONPATTRO-treated patients experienced IRRs, compared to 9 percent of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.
To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, paracetamol, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.
Reduced Serum Vitamin A Levels and Recommended Supplementation
ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.
Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).
Adverse Reactions
The most common adverse reactions that occurred in patients treated with ONPATTRO were respiratory-tract infection (29 percent) and infusion-related reactions (19 percent).
About LNP Technology
Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology.
About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
About
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of diseases with high unmet need. ONPATTRO® (patisiran) is the first-ever RNAi therapeutic approved by the U.S.
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, vutrisiran, givosiran, lumasiran, inclisiran, fitusiran, cemdisiran, and ALN-AGT, its plans for additional global regulatory filings and product launches for ONPATTRO, its expectations regarding the status of pricing and reimbursement procedures outside the U.S., its expectations regarding the timing for the initiation of its APOLLO-B Phase 3 study of patisiran, its plans to initiate additional Phase 3 studies for vutrisiran in late 2019, its expectations regarding the complete submission of an NDA for givosiran with the
With the exception of ONPATTRO (patisiran), none of Alnylam’s investigational therapeutics have been approved by the
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ALNYLAM PHARMACEUTICALS, INC. |
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Three Months Ended |
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| 2019 | 2018 | |||||||||
| Revenues: | ||||||||||
| Product revenues, net | $ | 26,291 | $ | — | ||||||
| Net revenues from collaborators | 7,003 | 21,899 | ||||||||
| Total revenues | 33,294 | 21,899 | ||||||||
| Cost and expenses: | ||||||||||
| Cost of goods sold | 3,347 | — | ||||||||
| Research and development | 129,127 | 96,857 | ||||||||
| Selling, general and administrative | 89,608 | 72,447 | ||||||||
| Total costs and expenses | 222,082 | 169,304 | ||||||||
| Loss from operations | (188,788 | ) | (147,405 | ) | ||||||
| Other income (expense): | ||||||||||
| Interest income | 7,525 | 5,794 | ||||||||
| Other income | 43 | 335 | ||||||||
| Total other income | 7,568 | 6,129 | ||||||||
| Loss before income taxes | (181,220 | ) | (141,276 | ) | ||||||
| (Provision) benefit for income taxes | (695 | ) | 62 | |||||||
| Net loss | $ | (181,915 | ) | $ | (141,214 | ) | ||||
| Net loss per common share - basic and diluted | $ | (1.73 | ) | $ | (1.41 | ) | ||||
| Weighted-average common shares used to compute basic and diluted net loss per common share | 105,400 | 99,979 | ||||||||
| Comprehensive loss: | ||||||||||
| Net loss | $ | (181,915 | ) | $ | (141,214 | ) | ||||
| Unrealized gain (loss) on marketable securities, net of tax | 360 | (420 | ) | |||||||
| Comprehensive loss | $ | (181,555 | ) | $ | (141,634 | ) | ||||
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ALNYLAM PHARMACEUTICALS, INC. |
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Three Months Ended |
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| 2019 | 2018 | |||||||||
| Reconciliation of GAAP to Non-GAAP Research and development: | ||||||||||
| GAAP Research and development | $ | 129,127 | $ | 96,857 | ||||||
| Less: Stock-based compensation expenses | (16,125 | ) | (10,137 | ) | ||||||
| Non-GAAP Research and development | $ | 113,002 | $ | 86,720 | ||||||
| Reconciliation of GAAP to Non-GAAP Selling, general and administrative: | ||||||||||
| GAAP Selling, general and administrative | $ | 89,608 | $ | 72,447 | ||||||
| Less: Stock-based compensation expenses | (15,907 | ) | (9,447 | ) | ||||||
| Non-GAAP Selling, general and administrative | $ | 73,701 | $ | 63,000 | ||||||
| Reconciliation of GAAP to Non-GAAP Operating costs and expenses: | ||||||||||
| GAAP Operating costs and expenses | $ | 222,082 | $ | 169,304 | ||||||
| Less: Stock-based compensation expenses | (32,032 | ) | (19,584 | ) | ||||||
| Non-GAAP Operating costs and expenses | $ | 190,050 | $ | 149,720 | ||||||
| Reconciliation of GAAP to Non-GAAP Net loss: | ||||||||||
| GAAP Net loss | $ | (181,915 | ) | $ | (141,214 | ) | ||||
| Add: Stock-based compensation expenses | 32,032 | 19,584 | ||||||||
| Non-GAAP Net loss | $ | (149,883 | ) | $ | (121,630 | ) | ||||
| Reconciliation of GAAP to Non-GAAP Net loss per common share-basic and diluted: | ||||||||||
| GAAP Net loss per common share - basic and diluted | $ | (1.73 | ) | $ | (1.41 | ) | ||||
| Add: Stock-based compensation expenses | 0.31 | 0.19 | ||||||||
| Non-GAAP Net loss per common share - basic and diluted | $ | (1.42 | ) | $ | (1.22 | ) | ||||
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ALNYLAM PHARMACEUTICALS, INC. |
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| March 31, | December 31, | |||||||
| 2019 | 2018 | |||||||
| Cash, cash equivalents and marketable debt securities | $ | 1,244,537 | $ | 1,082,949 | ||||
| Restricted investments | 44,825 | 44,825 | ||||||
| Accounts receivable, net | 33,801 | 18,760 | ||||||
| Inventory | 32,001 | 24,068 | ||||||
| Prepaid expenses and other assets | 90,711 | 83,542 | ||||||
| Property, plant and equipment, net | 341,712 | 320,658 | ||||||
| Operating lease right-of-use lease assets | 226,412 | — | ||||||
| Total assets | $ | 2,013,999 | $ | 1,574,802 | ||||
| Accounts payable, accrued expenses and other liabilities | $ | 134,616 | $ | 177,392 | ||||
| Total deferred revenue | 3,120 | 3,954 | ||||||
| Total deferred rent | — | 61,491 | ||||||
| Long-term debt | 30,000 | 30,000 | ||||||
| Total operating lease liability | 299,277 | — | ||||||
| Total stockholders’ equity (106.4 million shares issued and outstanding at March 31, 2019; 101.2 million shares issued and outstanding at December 31, 2018 ) | 1,546,986 | 1,301,965 | ||||||
| Total liabilities and stockholders' equity | $ | 2,013,999 | $ | 1,574,802 | ||||
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: https://www.businesswire.com/news/home/20190501005508/en/
Source:
Christine Regan Lindenboom
(Investors and Media)
617-682-4340
Josh Brodsky
(Investors)
617-551-8276
For Media Inquiries, please contact:
Christine Lindenboom
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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