Full Press Release Details
Alnylam Pharmaceuticals Reports Third Quarter 2023 Financial Results and Highlights Recent Period Activity
Achieved Third Quarter 2023 Global Net Product Revenues of $313 Million, Representing 35% Year-Over-Year Growth Compared to Q3 2022 -
Received Complete Response Letter from U.S. FDA for Supplemental New Drug Application for Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis -
Company Remains On Track to Report Topline Results from HELIOS-B Phase 3 Study in Early 2024 -
- Reported Positive Topline Results from KARDIA-1 Phase 2 Study of Zilebesiran -
Updated 2023 Guidance for Revenues from Collaborations and Royalties to $575 Million to $625 Million; Reiterated 2023 Guidance for Combined Net Product Revenues of $1,200 Million
CAMBRIDGE, Mass.--(BUSINESS WIRE)--November 2, 2023--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter ended
September 30, 2023 and reviewed recent business highlights.
"We continued to deliver strong commercial results in the third quarter, with the successful launch of AMVUTTRA contributing to 35% year-over-year growth in net product revenues," said Yvonne Greenstreet, MBChB, Chief Executive Officer of
Alnylam. "While we were disappointed by the recent Complete Response Letter for ONPATTRO for the treatment of the cardiomyopathy of ATTR amyloidosis, we remain confident in the HELIOS-B Phase 3 study of vutrisiran, which is on track to deliver
topline results in early 2024. We're also excited about advancements across our broad pipeline of RNAi therapeutics, including recent positive topline results from the KARDIA-1 Phase 2 study of zilebesiran, as well as upcoming Phase 1 results
for ALN-TTRsc04 and ALN-KHK by year-end. With this continued execution we remain on track to meet our Alnylam P5x25 goals of becoming a top-tier biotech company delivering sustained innovation and exceptional financial
Third 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 third quarter of $82 million and $149 million, respectively, representing 35% total TTR reported year-over-year growth compared to Q3 2022.
Attained over 3,790 hATTR amyloidosis patients with polyneuropathy worldwide on commercial treatment with ONPATTRO or AMVUTTRA as of September 30, 2023.
Total Ultra-Rare: GIVLAARI (givosiran) & OXLUMO (lumasiran)
Achieved global net product revenues for GIVLAARI and OXLUMO for the third quarter of $54 million and $29 million, respectively, representing 33% total Ultra-Rare reported year-over-year growth compared to Q3 2022.
Attained over 625 patients worldwide on commercial GIVLAARI treatment as of September 30, 2023.
Attained over 375 patients worldwide on commercial OXLUMO treatment as of September 30, 2023.
Leqvio launch in the U.S. and other markets is ongoing, with focus on patient on-boarding, removing access hurdles and enhancing medical education. In the U.S., Leqvio is covered at or near label for 76% of patients. More than 55% of
Leqvio source of business in the U.S. is now through "Buy and Bill" acquisition mode. FDA expanded the label to include primary hyperlipidemia (patients at increased risk of ASCVD) and the removal of four adverse reactions from the safety
section as well as Limitations of Use. In Q3 2023, Leqvio was approved in China and in Japan and is now approved in 93 countries.
Reported positive topline results from the KARDIA-1 Phase 2 dose-ranging study of zilebesiran, an investigational RNAi therapeutic in development to treat hypertension in patients at high cardiovascular risk.
Published results from Phase 1 study of zilebesiran in the New England Journal of Medicine.
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 on Alzheimer's Disease conference.
Based on the achievement of specified clinical proof-of-principle criteria for the Phase 1 ALN-APP program, Alnylam received a $100 million milestone payment from Regeneron.
Presented new 24-month results from an interim analysis of the open-label extension period of the APOLLO-B Phase 3 study of patisiran in patients with the cardiomyopathy of transthyretin-mediated amyloidosis at the Heart Failure
Society of America Annual Scientific Meeting 2023.
Published results from Phase 3 APOLLO-B study of patisiran in the New England Journal of Medicine.
Sanofi reported positive data for fitusiran, in development for the treatment of hemophilia A or B, with or without inhibitors, from the Phase 3 open-label extension study (ATLAS-OLE).
Sanofi is currently in discussions with the FDA regarding filing in 2024.
Additional Business Updates
Entered into a global strategic collaboration with Roche for the co-development and co-commercialization of zilebesiran.
Recognized by Science magazine as a Top Employer for the fifth consecutive year.
Alnylam announces today that it will present a review of its RNAi platform and pipeline progress at a virtual R&D Day on December 13, 2023.
Alnylam announces today that results from the KARDIA-1 Phase 2 dose-ranging study of zilebesiran will be presented as a Late-Breaker at the American Heart Association Scientific Sessions 2023 on November 11, 2023, in Philadelphia,
In addition, in late 2023:
Alnylam intends to report topline results from the Phase 1 study of ALN-TTRsc04.
Alnylam intends to report topline results from the Phase 1 study of ALN-KHK.
Vir is conducting multiple trials evaluating the potential for ALN-HBV02 (VIR-2218) and VIR-3434 to achieve a functional cure for chronic hepatitis B. Phase 2 data readouts are on track for Q4 2023.
Vir also announced that initial Phase 2 data readouts for the SOLSTICE trial evaluating ALN-HBV02 (VIR-2218) and VIR-3434 as monotherapy and in combination for the treatment of people living with chronic hepatitis delta, the
most aggressive form of viral hepatitis, are expected in Q4 2023.
Financial Results for the Quarter Ended September 30, 2023
| Three Months Ended September 30, | ||||||
| (in thousands, except per share amounts) | 2023 | 2022 | ||||
| Net product revenues | $ | 313,153 | $ | 232,267 | ||
| Net revenue from collaborations | $ | 427,472 | $ | 29,297 | ||
| Royalty revenue | $ | 9,905 | $ | 2,742 | ||
| GAAP Operating income (loss) | $ | 213,867 | $ | (258,040 | ) | |
| Non-GAAP Operating income (loss) | $ | 277,804 | $ | (129,922 | ) | |
| GAAP Net income (loss) | $ | 147,753 | $ | (405,920 | ) | |
| Non-GAAP Net income (loss) | $ | 228,534 | $ | (193,366 | ) | |
| GAAP net income (loss) per common share - basic | $ | 1.18 | $ | (3.32 | ) | |
| Non-GAAP net income (loss) per common share - basic | $ | 1.83 | $ | (1.58 | ) | |
| GAAP net income (loss) per common share - diluted | $ | 1.15 | $ | (3.32 | ) | |
| Non-GAAP net income (loss) per common share - diluted | $ | 1.74 | $ | (1.58 | ) | |
| * 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 September 30, | Year over Year % Growth | ||||||||||
| (in thousands, except percentages) | 2023 | 2022 | As Reported | At CER* | |||||||
| ONPATTRO net product revenues | $ | 81,589 | $ | 144,950 | (44 | )% | (46 | )% | |||
| AMVUTTRA net product revenues | 148,680 | 25,229 | 489 | % | 488 | % | |||||
| Total TTR net product revenues | $ | 230,269 | $ | 170,179 | 35 | % | 34 | % | |||
| GIVLAARI net product revenues | 54,148 | 45,659 | 19 | % | 17 | % | |||||
| OXLUMO net product revenues | 28,736 | 16,429 | 75 | % | 69 | % | |||||
| Total net product revenues | $ | 313,153 | $ | 232,267 | 35 | % | 33 | % | |||
| * CER = Constant Exchange Rate, representing growth calculated as if the exchange rates had remained unchanged from those used in the third quarter 2022. CER is a Non-GAAP measure. 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 increased 35% during the third quarter 2023, as compared to the prior year, and 33% at CER. The increase is primarily related to growth in our TTR product revenues driven by the launch of AMVUTTRA in the third quarter
of 2022 as well as increased patients on GIVLAARI and OXLUMO therapies. The increase was offset by a decrease of demand for ONPATTRO due to patient switched to AMVUTTRA.
Net Revenues from Collaborations
The increase in net revenues from collaborations, as compared to the prior year, is due to revenue related to our collaborations with Roche and Regeneron. During the quarter, we recognized $310 million of revenue from the upfront payment
received from Roche and $65 million in connection with our Regeneron Collaboration. The $65 million of revenue is a cumulative adjustment from the $100 million milestone received for achieving certain criteria during early clinical
development for our CNS program, ALN-APP.
| Three Months Ended September 30, | 2023 vs 2022 | |||||||||
| (in thousands, except percentages) | 2023 | 2022 | % Change* | |||||||
| Cost of goods sold | $ | 79,473 | $ | 36,507 | 118 | % | ||||
| Cost of goods sold as a percentage of net product revenues | 25.4 | % | 15.7 | % | 9.7 | % | ||||
| Cost of collaborations | $ | 4,836 | $ | 4,609 | 5 | % | ||||
| GAAP research and development expenses | $ | 253,179 | $ | 245,371 | 3 | % | ||||
| Non-GAAP research and development expenses | $ | 224,024 | $ | 192,409 | 16 | % | ||||
| GAAP selling, general and administrative expenses | $ | 199,175 | $ | 235,859 | (16 | )% | ||||
| Non-GAAP selling, general and administrative expenses | $ | 164,393 | $ | 160,703 | 2 | % | ||||
| *For dollar values, we calculate the percentage of change during Q3 2023 compared to Q3 2022. For cost of goods sold as a percentage of net product revenues, we calculate the basis point change during Q3 2023 compared to Q3 2022. |
Cost of goods sold as a percent of net product revenues increased during the third quarter 2023, as compared to the prior year, primarily due to recording an impairment of ONPATTRO inventory that had been manufactured for future demand
associated with the ATTR 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 third quarter 2023, as compared to the prior year, primarily due to increased costs related to clinical activities and increased headcount to support our R&D pipeline and an
expense for the achievement of a certain clinical milestone payable to a partner. Increased GAAP R&D expenses were offset by decreased stock-based compensation expense related to the accounting for certain performance-based awards that
Selling, General & Administrative (SG&A) Expenses
GAAP SG&A expenses decreased during the third quarter 2023, as compared to the prior year, primarily due to decreased stock-based compensation expense related to the accounting for certain performance-based awards that vested in 2022.
Non-GAAP SG&A expenses increased in the third quarter of 2023, compared to 2022, primarily due to increased headcount and other investments supporting our strategic growth including the global launch of AMVUTTRA.
Other Financial Highlights
Cash, cash equivalents and marketable securities were $2.41 billion as of September 30, 2023 compared to $2.19 billion as of December 31, 2022 with the increase primarily related to the $310 million upfront payment received from Roche in
connection with our partnership to co-develop and co-commercialize zilebesiran, offset by our operating loss year-to-date.
The adjustments to the non-GAAP measures provided in the financial results above and in the financial guidance below are described under "Use of Non-GAAP Financial Measures" later in this press release. A reconciliation of our GAAP to
non-GAAP results presented in this release is included in the tables at the end of this press release.
2023 Financial Guidance
Full year 2023 financial guidance has been updated as follows:
| Provided 2/23/2023 1 | Updated 11/2/2023 | ||
| Combined net product revenues for ONPATTRO, AMVUTTRA, GIVLAARI and OXLUMO 1 | $1,200 million - $1,285 million | Unchanged | |
| Net Product Revenue Growth vs. 2022 at reported FX rates 1 | 34% to 44% | Unchanged | |
| Net Product Revenue Growth vs. 2022 at CER* | 34% to 44% | Unchanged | |
| Net revenues from collaborations and royalties | $100 million - $175 million | $575 million - $625 million | |
| GAAP R&D and SG&A expenses | $1,790 million - $1,885 million | Unchanged | |
| Non-GAAP R&D and SG&A expenses 2 | $1,575 million - $1,650 million | Unchanged | |
| 1 Uses December 31, 2022 FX rates including: 1 EUR = 1.07 USD and 1 USD = 131 JPY | |||
| 2 Excludes $215 - $235 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, 2022. 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 certain losses 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 non-GAAP financial measures we present include Non-GAAP Operating income (loss), Non-GAAP Net income (loss), Non-GAAP Net income (loss) per common share - basic, Non-GAAP Net income (loss) per common share - diluted and Non-GAAP R&D
and SG&A expenses. 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 stock-based compensation expenses and realized and
unrealized losses on marketable equity securities. 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 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
Percentage changes in revenue growth at CER, also a non-GAAP financial measure, 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 historical GAAP and non-GAAP measures presented in this release is provided later in this press release.
Conference Call Information
Management will provide an update on the Company and discuss third quarter 2023 results as well as expectations for the future via conference call on Thursday, November 2, 2023 at 8:30 am ET. To access the call, please register online at
https://register.vevent.com/register/BI29582eb5fd274b758554baecef8f03fa. Participants are requested to register at a minimum 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.
About ONPATTRO (patisiran)
ONPATTRO is an RNAi therapeutic that is 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).
It is designed to target and silence TTR messenger RNA, thereby reducing the production of TTR protein before it is made. Reducing the pathogenic protein leads to a reduction in amyloid deposits in tissues. For more information about ONPATTRO,
including full Prescribing Information, visit ONPATTRO.com.
About AMVUTTRA (vutrisiran)
AMVUTTRA (vutrisiran) is an RNAi therapeutic approved in the United States for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It is a double-stranded small interfering RNA
(siRNA) that targets mutant and wild-type transthyretin (TTR) messenger RNA (mRNA). Using Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, AMVUTTRA is designed for increased potency and high metabolic
stability to allow for subcutaneous injection once every three months (quarterly). Results from the pivotal HELIOS-A Phase 3 study demonstrate AMVUTTRA rapidly reduces serum TTR levels, has the potential to reverse neuropathy impairment