Full Press Release Details
Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Period Activity
Achieved Fourth Quarter and Full Year 2023 Global Net Product Revenues of $346 Million and $1,241 Million, Respectively, Representing 39% Annual Growth Compared to 2022 -
Company Announces Updated Statistical Analysis Plan and Timing for HELIOS-B Phase 3 Study of Vutrisiran -
Announces U.S. FDA Clearance to Initiate Multiple-Dosing in ALN-APP Phase 1 Study -
Provides 2024 Combined Net Product Revenue Guidance of $1,400 Million to $1,500 Million, in Addition to Collaboration and Royalty Revenue and Operating Expense Guidance -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--February 15, 2024--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the fourth quarter and full year ended December
31, 2023 and reviewed recent business highlights.
"2023 was a year of strong execution at Alnylam. We delivered robust product revenue growth across our four wholly-owned commercial medicines, with $1.24 billion in global net product revenues, and achieved over 5,000 patients now being
treated with an Alnylam commercial medicine. We also extended our leadership in RNAi with the first clinical demonstration of gene silencing in the human brain using an RNAi therapeutic, strengthened our business for the future through a
landmark partnership with Roche and delivered solid financial performance," said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. "Looking ahead to 2024, we are excited for a number of important milestones across the pipeline,
including results from the HELIOS-B Phase 3 study of vutrisiran as well as the KARDIA-2 Phase 2 study of zilebesiran, and six clinical study starts. This progress sets us up for delivering on our Alnylam P5x25 goals of
becoming a top-tier biotech company delivering sustained innovation and exceptional financial results."
Fourth Quarter 2023 and Recent Significant Corporate Highlights
Commercial Performance
Total TTR: ONPATTRO (patisiran) & AMVUTTRA (vutrisiran)
Achieved global net product revenues for ONPATTRO and AMVUTTRA for the fourth quarter of $79 million and $175 million, respectively, representing 10% total TTR quarterly growth compared to Q3 2023, and full year 2023 revenues of $355
million and $558 million, respectively, representing 40% total TTR annual growth compared to full year 2022.
Attained over 4,060 hATTR amyloidosis patients with polyneuropathy worldwide on commercial treatment with ONPATTRO or AMVUTTRA as of December 31, 2023.
Total Ultra-Rare: GIVLAARI (givosiran) & OXLUMO (lumasiran)
Achieved global net product revenues for GIVLAARI and OXLUMO for the fourth quarter of $59 million and $33 million, respectively, representing 11% total Ultra-Rare quarterly growth compared to Q3 2023, and full year 2023 revenues of $219
million and $110 million, respectively, representing 35% total Ultra-Rare annual growth compared to full year 2022.
Attained over 650 patients on commercial GIVLAARI and over 430 patients on commercial OXLUMO worldwide as of December 31, 2023.
Alnylam announces today updates to the statistical analysis plan for the HELIOS-B Phase 3 study of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy. These will include updates to the primary and secondary endpoint
structure, as well as study exposure. Topline results are expected to be available in late June or early July. Details will be discussed on the conference call this morning.
Published results from Phase 3 APOLLO-B study of patisiran in the New England Journal of Medicine.
Presented positive initial Phase 1 results with ALN-TTRsc04 demonstrating rapid knockdown achieved by a single dose with mean serum TTR reduction up to 97%, with durability supporting potential for annual dosing and an encouraging
Presented positive results from the KARDIA-1 Phase 2 dose-ranging study of zilebesiran, an investigational RNAi therapeutic in development to treat hypertension patients at high cardiovascular risk, during the American Heart
Association (AHA) Scientific Sessions.
Announces today that the U.S. Food and Drug Administration (FDA) has provided clearance to initiate the multiple-dose part (Part B) of the ongoing Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid
precursor protein (APP) in development for the treatment of Alzheimer's disease and cerebral amyloid angiopathy. The FDA has confirmed that multiple-dosing in the Phase 1 study may proceed at doses up to 180 mg given every six months, which
covers all dose regimens planned to be explored in Part B. A partial clinical hold remains for higher or more frequent dosing regimens.
Reported updated positive interim results for the ongoing single ascending dose portion of the Phase 1 study of ALN-APP in patients with early-onset Alzheimer's disease at the 2023 Alzheimer's Association International Conference and at
the 16th Clinical Trials in Alzheimer's Disease conference.
Presented positive initial Phase 1 results with ALN-KHK demonstrating robust target engagement and an encouraging safety profile, supporting continued development as a novel treatment for type 2 diabetes mellitus.
Filed an Investigational New Drug (IND) application for ALN-BCAT, an investigational RNAi therapeutic targeting -catenin in development for the treatment of hepatocellular carcinoma.
Sanofi presented positive results from the ATLAS-OLE Phase 3 extension study of fitusiran, demonstrating a substantially improved safety profile and consistent bleed protection in people with hemophilia A or B, with or without
inhibitors. Specifically, the risk of thrombosis was reduced, with rates comparable to those reported in the general hemophilia population.
Sanofi expects to submit a New Drug Application (NDA) to the FDA in 2024.
Additional Business Updates
Ranked #1 on Boston Globe's Top Places to Work list for 2023 in the "Largest Employer" category.
Recognized by Science magazine as a Top Employer for the fifth consecutive year.
Alnylam announces today that results from the KARDIA-2 Phase 2 study of zilebesiran will be presented in a Late-Breaker presentation at the American College of Cardiology Scientific Sessions 2024 on April 7, 2024 in Atlanta, Georgia.
In early 2024, Alnylam intends to:
Report topline results from the HELIOS-B Phase 3 study of vutrisiran in late June or early July.
Initiate the KARDIA-3 Phase 2 study of zilebesiran.
Initiate a Phase 2 study of ALN-APP in patients with cerebral amyloid angiopathy.
Initiate Part B of the Phase 1 study of ALN-KHK.
Initiate a Phase 1 study of ALN-BCAT.
Financial Highlights for the Fourth Quarter and Year End 2023
| Three Months Ended December 31, | Twelve Months Ended December 31, | ||||||||||||||
| (In thousands, except per share amounts) | 2023 | 2022 | 2023 | 2022 | |||||||||||
| Net product revenues | $ | 346,288 | $ | 261,675 | $ | 1,241,474 | $ | 894,329 | |||||||
| Net revenue from collaborations | $ | 76,407 | $ | 70,645 | $ | 546,185 | $ | 134,912 | |||||||
| Royalty revenue | $ | 17,023 | $ | 2,715 | $ | 40,633 | $ | 8,177 | |||||||
| GAAP Operating loss | $ | (116,404 | ) | $ | (188,614 | ) | $ | (282,175 | ) | $ | (785,072 | ) | |||
| Non-GAAP Operating loss | $ | (74,410 | ) | $ | (145,847 | ) | $ | (60,495 | ) | $ | (554,423 | ) | |||
| GAAP Net loss | $ | (137,870 | ) | $ | (207,493 | ) | $ | (440,242 | ) | $ | (1,131,156 | ) | |||
| Non-GAAP Net loss | $ | (96,643 | ) | $ | (171,522 | ) | $ | (201,618 | ) | $ | (790,609 | ) | |||
| GAAP Net loss per common share - basic and diluted | $ | (1.10 | ) | $ | (1.68 | ) | $ | (3.52 | ) | $ | (9.30 | ) | |||
| Non-GAAP Net loss per common share - basic and diluted | $ | (0.77 | ) | $ | (1.39 | ) | $ | (1.61 | ) | $ | (6.50 | ) |
For an explanation of our use of non-GAAP financial measures refer to the "Use of Non-GAAP Financial Measures" section later in this press release and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP
measures, see the table at the end of this press release.
Net Product Revenues
| Three Months Ended December 31, | Twelve Months Ended December 31, | ||||||||||
| (In thousands) | 2023 | 2022 | 2023 | 2022 | |||||||
| ONPATTRO net product revenues | $ | 79,006 | $ | 122,221 | $ | 354,546 | $ | 557,608 | |||
| AMVUTTRA net product revenues | 175,254 | 68,566 | 557,838 | 93,795 | |||||||
| Total TTR net product revenues | 254,260 | 190,787 | 912,384 | 651,403 | |||||||
| GIVLAARI net product revenues | 59,298 | 47,058 | 219,251 | 173,144 | |||||||
| OXLUMO net product revenues | 32,730 | 23,830 | 109,839 | 69,782 | |||||||
| Total net product revenues | $ | 346,288 | $ | 261,675 | $ | 1,241,474 | $ | 894,329 |
| Year over Year % Growth | |||||||||||
| Three Months Ended December 31, 2023 | Twelve Months Ended December 31, 2023 | ||||||||||
| As Reported | At CER* | As Reported | At CER* | ||||||||
| Total TTR net product revenues | 33 | % | 31 | % | 40 | % | 40 | % | |||
| GIVLAARI net product revenues | 26 | % | 24 | % | 27 | % | 26 | % | |||
| OXLUMO net product revenues | 37 | % | 32 | % | 57 | % | 55 | % | |||
| Total net product revenues | 32 | % | 30 | % | 39 | % | 39 | % | |||
| * CER = Constant Exchange Rate, representing growth calculated as if the exchange rates had remained unchanged from those used in 2022. CER is a non-GAAP measure. |
Net product revenues increased 32% and 39% at actual currency during the three and twelve months ended December 31, 2023, respectively, compared to the same periods in 2022, and 30% and 39% at CER, respectively. The increases are primarily
due to increased patients on our commercial TTR therapies driven by the launch of AMVUTTRA in the third quarter of 2022 as well as increased patients on GIVLAARI and OXLUMO therapies.
Net Revenues from Collaborations
Net revenues from collaborations increased 8% and 305% during the three and twelve months ended December 31, 2023, respectively, as compared to the same periods in 2022, primarily due to revenue recognized under our Collaboration and
License Agreement with Roche, as executed in July 2023, and revenue recognized under our Novartis Collaboration Agreement associated with the achievement of specified commercialization and regulatory milestones.
| Three Months Ended December 31, | Twelve Months Ended December 31, | ||||||||||||||
| (In thousands) | 2023 | 2022 | 2023 | 2022 | |||||||||||
| Cost of goods sold | $ | 71,975 | $ | 46,172 | $ | 268,216 | $ | 140,174 | |||||||
| Cost of goods sold as a percentage of net product revenues | 20.8 | % | 17.6 | % | 21.6 | % | 15.7 | % | |||||||
| Cost of collaborations and royalties | $ | 13,883 | $ | 5,094 | $ | 42,190 | $ | 28,643 | |||||||
| GAAP research and development expenses | $ | 272,141 | $ | 262,039 | $ | 1,004,415 | $ | 883,015 | |||||||
| Non-GAAP research and development expenses | $ | 253,056 | $ | 245,095 | $ | 907,142 | $ | 790,854 | |||||||
| GAAP selling, general and administrative expenses | $ | 198,123 | $ | 210,344 | $ | 795,646 | $ | 770,658 | |||||||
| Non-GAAP selling, general and administrative expenses | $ | 175,214 | $ | 184,521 | $ | 671,239 | $ | 632,170 |
Cost of goods sold as a percentage of net product revenues increased during the three and twelve months ended December 31, 2023, respectively, as compared to the same periods in 2022, primarily due to increased volume and rate of royalties
payable on net sales of AMVUTTRA associated with tiered royalty percentages, in addition to increased excess and obsolete charges primarily due to canceling manufacturing commitments and the impairment of ONPATTRO inventory that had been
manufactured for future demand associated with the ATTR amyloidosis with cardiomyopathy indication for patisiran for which we did not receive regulatory approval.
Research & Development (R&D) Expenses
GAAP and non-GAAP R&D expenses increased during the three and twelve months ended December 31, 2023, compared to the same periods in 2022, primarily due to increased headcount and infrastructure expenses to support our R&D
pipeline, development expenses associated with the KARDIA-1 and KARDIA-2 zilebesiran Phase 2 studies, and manufacturing and research related expenses associated with our pre-clinical and developmental activities. GAAP R&D expenses further
increased during the twelve month period due to increased stock-based compensation expense related to the accounting for certain performance-based awards during the period.
Selling, General & Administrative (SG&A) Expenses
GAAP and non-GAAP SG&A expenses decreased during the three months ended December 31, 2023, compared to the same period in 2022, primarily due to increased legal expenses in 2022 associated with the Patent Infringement Lawsuits we filed
in March 2022 and the Department of Justice investigation, which closed in August 2023, and increased expenses in support of the global launch of AMVUTTRA in the third quarter of 2022.
GAAP and non-GAAP SG&A expenses increased during the twelve months ended December 31, 2023, compared to the same period in 2022, primarily due to increased headcount and other strategic investments in support of the global launch of
AMVUTTRA and other expenses to support our strategic growth.
Other Financial Highlights
Cash, cash equivalents and marketable securities were $2.44 billion as of December 31, 2023, compared to $2.19 billion as of December 31, 2022, with the increase primarily due to the receipts of a $310 million upfront payment from Roche in
connection with our partnership to co-develop and co-commercialize zilebesiran, a $100 million payment from Regeneron in connection with the achievement of certain criteria during early clinical development for our CNS program, ALN-APP, and
nearly $150 million from employee option award exercises, offset by our operating loss for the year.
A reconciliation of our GAAP to non-GAAP results for the current quarter is included in the tables at the end of this press release.
2024 Financial Guidance
Full year December 31, 2024 financial guidance consists of the following:
| Combined net product revenues for AMVUTTRA, ONPATTRO, GIVLAARI and OXLUMO 1 | $1,400 million - $1,500 million |
| Net Product Revenue Growth vs. 2023 at reported Fx rates 1 | 13% - 21% |
| Net Product Revenue Growth vs. 2023 at constant exchange rates* | 13% - 21% |
| Net revenues from collaborations and royalties | $325 million - $425 million |
| GAAP R&D and SG&A expenses | $1,900 million - $2,050 million |
| Non-GAAP R&D and SG&A expenses 2 | $1,675 million - $1,775 million |
| 1 Uses January 31, 2024 FX rates including: 1 EUR = 1.08 USD and 1 USD = 147 JPY | |
| 2 Primarily excludes $225-$275 million of stock-based compensation expense from estimated GAAP R&D and SG&A expenses. | |
| * CER = Constant Exchange Rate, representing growth calculated as if the exchange rates had remained unchanged from those used in the twelve months ended December 31, 2023. CER is a non-GAAP measure. |
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 this press release are, as applicable, stock-based compensation expenses, realized and unrealized
(gains) losses on marketable equity securities and loss on the extinguishment of debt. 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 realized and 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 loss on the extinguishment of debt because the Company believes the item is a non-recurring transaction outside the ordinary course of the Company's business.
Percentage changes in revenue growth at CER are presented excluding the impact of changes in foreign currency exchange rates for investors to understand the underlying business performance. The current period's foreign currency revenue values
are converted into U.S. dollars using the average exchange rates from the prior period.
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 fourth quarter and full year 2023 results as well as expectations for the future via conference call on Thursday, February 15, 2024 at 8:30 am ET. To access the call, please
register online at https://register.vevent.com/register/BI94c4772acd31430480d414497e322a2f. Participants are requested to register at least 15 minutes before the start of the call. A replay of the call will be available two hours after the call
and archived on the same web page for six months.
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.