Full Press Release Details
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom
(Investors and Media)
Alnylam Pharmaceuticals Reports Third Quarter 2021 Financial Results and Highlights Recent
- Achieved Third Quarter 2021 Combined Net Product Revenues of $167 Million for ONPATTRO , GIVLAARI , and OXLUMO
- Reported Positive Topline 18-Month Results from HELIOS-A Phase 3 Study of Vutrisiran in hATTR Amyloidosis Patients with Polyneuropathy
Completed Enrollment Ahead of Schedule in HELIOS-B Phase 3 Study of Vutrisiran in ATTR
Amyloidosis Patients with Cardiomyopathy
Reiterated 2021 Financial Guidance, Including Combined Net Product Revenues
of $640-$665 Million
Founding Alnylam CEO John Maraganore to Transition CEO
Leadership to Alnylam President Yvonne Greenstreet at Year End
CAMBRIDGE, Mass., October 28, 2021 Alnylam Pharmaceuticals,
Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter ended September 30, 2021 and reviewed recent business highlights.
Our third quarter commercial performance was highlighted by strength from ONPATTRO, and continued execution on the
on-going launches of GIVLAARI and OXLUMO. Another notable achievement was our announcement of positive topline 18-month results from the
HELIOS-A Phase 3 study of vutrisiran in patients with hATTR amyloidosis with polyneuropathy, including improvements in exploratory and cardiac amyloid endpoints, which we believe are encouraging
hypothesis-generating data ahead of upcoming readouts from the APOLLO-B and HELIOS-B Phase 3 studies of patisiran and vutrisiran, respectively, said John
Maraganore, Ph.D., Chief Executive Officer of Alnylam. We also made significant progress with many of our other programs, and we remain focused on executing on our Alnylam
P5x25 vision, through which we intend to deliver transformative medicines to patients around the world for rare and prevalent diseases, advancing a robust and high-yielding
pipeline of first and/or best-in-class product candidates from our organic product engine, while delivering exceptional financial performance. Lastly, with the
announcement this morning of my planned transition from Alnylam at year end, I d like to emphasize how thrilled I am about the future of Alnylam under Yvonne s upcoming stewardship. I have immense confidence in her ability to continue to
deliver on the promise of RNAi therapeutics for patients around the world.
Third Quarter 2021 and Recent Significant Corporate Highlights
Commercial Performance
ONPATTRO (patisiran)
GIVLAARI (givosiran)
Vutrisiran, a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis
(the non-proprietary name for OXLUMO), for the treatment of primary hyperoxaluria type 1 (PH1)
Cemdisiran, an investigational RNAi therapeutic in development for the treatment of
complement-mediated diseases
Fitusiran, an investigational RNAi therapeutic in development for the treatment of hemophilia A or B
with and without inhibitors, in collaboration with Sanofi
Early- and mid-stage investigational RNAi
therapeutic pipeline programs and RNAi platform
Additional Business Updates
Alnylam announces today the upcoming presentations of clinical data at medical congresses:
In addition, in late 2021, Alnylam intends to:
Financial Results for the Quarter Ended
We are pleased with the increase in patients across our commercial product portfolio in the third quarter of 2021, as
our three wholly owned products continue to serve the unmet needs of patients globally. We also further strengthened our balance sheet with receipt of the second $500 million payment from Blackstone related to the partial monetization of the
inclisiran royalty, said Jeff Poulton, Chief Financial Officer of Alnylam. We are reiterating our 2021 financial guidance, which includes our expectation to achieve between $640 million and $665 million in combined net product
revenues across our three wholly owned commercial brands for the full year 2021. We look forward to continued strong topline growth balanced with disciplined investment in our operations, which we believe will transition us toward a self-sustainable
financial profile, aligned with our Alnylam P5x25 strategy.
(in thousands, except per share amounts)
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| ONPATTRO net product revenues | $ | 120,317 | $ | 82,516 | $ | 336,107 | $ | 215,715 | ||||||||
| GIVLAARI net product revenues | 31,833 | 16,690 | 87,136 | 32,962 | ||||||||||||
| OXLUMO net product revenues | 14,894 | 40,381 | ||||||||||||||
| Total net product revenues | $ | 167,044 | $ | 99,206 | $ | 463,624 | $ | 248,677 | ||||||||
| Net revenue from collaborations | $ | 20,136 | $ | 26,647 | $ | 121,328 | $ | 80,614 | ||||||||
| Royalty revenue | $ | 453 | $ | $ | 800 | $ | ||||||||||
| GAAP operating loss | $ | (181,677 | ) | $ | (225,199 | ) | $ | (514,091 | ) | $ | (634,216 | ) | ||||
| Non-GAAP operating loss | $ | (138,310 | ) | $ | (158,522 | ) | $ | (382,956 | ) | $ | (498,886 | ) | ||||
| GAAP net loss | $ | (204,514 | ) | $ | (253,291 | ) | $ | (594,364 | ) | $ | (614,741 | ) | ||||
| Non-GAAP net loss | $ | (179,838 | ) | $ | (183,597 | ) | $ | (524,502 | ) | $ | (546,679 | ) | ||||
| GAAP net loss per common share - basic and diluted | $ | (1.72 | ) | $ | (2.18 | ) | $ | (5.04 | ) | $ | (5.37 | ) | ||||
| Non-GAAP net loss per common share - basic and diluted | $ | (1.51 | ) | $ | (1.58 | ) | $ | (4.44 | ) | $ | (4.77 | ) |
Net Product Revenues
Net Revenues from Collaborations
Third Quarter and Year-to-Date 2021 Expenses
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| GAAP research and development expenses | $ | 194,572 | $ | 161,783 | $ | 563,106 | $ | 486,350 | ||||||||
| Non-GAAP research and development expenses | $ | 172,155 | $ | 148,080 | $ | 503,228 | $ | 440,808 | ||||||||
| GAAP selling, general and administrative expenses | $ | 142,075 | $ | 167,472 | $ | 434,257 | $ | 422,129 | ||||||||
| Non-GAAP selling, general and administrative expenses | $ | 121,125 | $ | 114,498 | $ | 363,000 | $ | 332,341 |
Research & Development (R&D) Expenses
Selling, General & Administrative (SG&A) Expenses
Other Financial Highlights
Other (Expense) Income
Cash and Investments
A reconciliation of our GAAP to non-GAAP results for the current
quarter is included in the tables of this press release.
2021 Financial Guidance
Full year 2021 financial guidance is reiterated and consists of the following:
| Combined net product revenues for ONPATTRO, GIVLAARI and OXLUMO | $ | 640 million - $665 million | ||
| Net revenues from collaborations and royalties | $ | 150 million - $200 million | ||
| GAAP R&D and SG&A expenses | $ | 1,335 million - $1,455 million | ||
| Non-GAAP R&D and SG&A expenses* | $ | 1,175 million - $1,275 million |
Use of Non-GAAP Financial
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.
included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in this press release are stock-based compensation expenses, unrealized (gains)
losses on marketable equity securities, costs associated with our strategic financing collaboration, upfront payment on license and collaboration agreement, change in estimate of contingent liabilities and loss on contractual 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 unrealized (gains) losses on marketable equity securities because the Company does not believe these adjustments accurately
reflect the performance of the Company s ongoing operations for the period in which such gains or losses are reported, as their sole purpose is to adjust amounts on the balance sheet. The Company has excluded the impact of the costs associated
with our strategic financing collaboration, upfront payment on license and collaboration agreement, change in estimate of contingent liabilities and loss on contractual settlement because the Company believes these items are non-recurring transactions 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.
Conference Call Information
Management will provide an update on the Company and discuss third quarter 2021 results as well as expectations for the future via conference call on Thursday,
October 28, 2021 at 8:30 am ET. To access the call, please dial 877-312-7507 (domestic) or +1-631-813-4828 (international) five minutes prior to the start time and refer to conference ID 1428538. A replay of the call will be available beginning at 11:30 am ET on the day of the call. To
access the replay, please dial 855-859-2056 (domestic) or
+1-404-537-3406 (international) and refer to conference ID 1428538.
A live audio webcast of the call will be available on the Investors section of the Company s website at www.alnylam.com/events. An archived
webcast will be available on the Alnylam website approximately two hours after the event.
ONPATTRO (patisiran)
ONPATTRO is an RNAi therapeutic that was approved in the United States
and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and
in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) and should be administered via a healthcare professional. It is designed to target and
silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. ONPATTRO 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 polyneuropathy associated with the disease. For more information about ONPATTRO, including please see the full US Prescribing Information, visit ONPATTRO.com.
About GIVLAARI (givosiran)
GIVLAARI is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) approved in the United States and Brazil for the treatment of adults with
acute hepatic porphyria (AHP). GIVLAARI is also approved in the European Union for the treatment of AHP in adults and adolescents aged 12 years and older. In the pivotal study, givosiran was shown to significantly reduce the rate of porphyria
attacks that required hospitalizations, urgent healthcare visits or intravenous hemin administration at home compared to placebo. GIVLAARI is Alnylam s first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. GIVLAARI is administered via subcutaneous injection once monthly at a dose based on actual body weight and should be administered by a healthcare
professional. GIVLAARI works by specifically reducing elevated levels of aminolevulinic acid synthase 1 (ALAS1) messenger RNA (mRNA), leading to reduction of toxins associated with attacks and other disease manifestations of AHP. For more
information about GIVLAARI, including the full U.S. Prescribing Information. visit GIVLAARI.com.
About OXLUMO (lumasiran)
OXLUMO is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in
pediatric and adult patients. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1. OXLUMO works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate
the toxic metabolite responsible for the clinical manifestations of PH1. In the pivotal ILLUMINATE-A study, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with
the majority of patients reaching normal or near-normal levels. Injection site reactions (ISRs) were the most common drug-related adverse reaction. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO
demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. OXLUMO utilizes Alnylam s Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate technology designed to increase