Full Press Release Details
| Contacts: Alnylam Pharmaceuticals, Inc. | ||
| Christine Regan Lindenboom (Investors and Media) 617-682-4340 Josh Brodsky (Investors) 617-551-8276 |
Alnylam Pharmaceuticals Reports Second Quarter 2023 Financial Results and Highlights Recent Period Activity
Achieved Second Quarter 2023 Global Net Product Revenues of $306 Million, Representing 43% Year-Over-Year Growth Compared to
Submitted 18-Month APOLLO-B
Data to the U.S. Food and Drug Administration as Amendment to Supplemental New Drug Application for Patisiran
Presented Updated Positive Interim Results from Phase 1 Study of ALN-APP in Patients with
Early-Onset Alzheimer s Disease
Entered into Global Strategic Collaboration with Roche for Co-Development and Co-Commercialization of Zilebesiran
U.S. Attorney s Office for District of Massachusetts Concluded and Closed Investigation Regarding Marketing and Promotion of
ONPATTRO, with no Action Taken
Akshay Vaishnaw, M.D., Ph.D., Named Alnylam s First Chief Innovation Officer
Reiterated 2023 Financial Guidance, Including Combined Net Product Revenues of $1,200 Million to $1,285 Million
CAMBRIDGE, Mass., August 3, 2023 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company,
today reported its consolidated financial results for the second quarter ended June 30, 2023 and reviewed recent business highlights.
second quarter of 2023 was a very productive one at Alnylam, with commercial execution delivering 43% year-over-year growth in net product revenues, supported by the strong ongoing launch of AMVUTTRA, said Yvonne Greenstreet, MBChB, Chief
Executive Officer of Alnylam. We also had some very important data readouts in the quarter and recent period, including updated interim results from the Phase 1 trial of ALN-APP in patients with early
onset Alzheimer s disease, as well as positive 18-month results from the APOLLO-B Phase 3 study of patisiran, that we believe reaffirm the potential of patisiran in
ATTR amyloidosis with cardiomyopathy. Furthermore, we entered into an exciting new partnership with Roche for the development and commercialization of zilebesiran. This progress underscores our commitment to executing across all areas of the
business in order to meet our Alnylam P5x25 goals of becoming a top-tier biotech company delivering sustained innovation and
exceptional financial results.
Second Quarter 2023 and Recent Significant Corporate Highlights
Commercial Performance
Total TTR: ONPATTRO (patisiran) & AMVUTTRA (vutrisiran)
Total Ultra-Rare: GIVLAARI (givosiran) & OXLUMO (lumasiran)
Presented new results from an
interim analysis of data from the open-label extension (OLE) period of the APOLLO-B Phase 3 study of patisiran, demonstrating continued evidence of sustained benefit across measures of functional
capacity and health status and quality of life, as well as cardiac stress and injury. Patisiran demonstrated a safety profile consistent with that observed in the 12-month double-blind period, with no new
The Company announces today that a U.S. Expanded Access Program (EAP) for patisiran that was initiated in
August 2022 has fully enrolled, with 200 patients across 20 centers in less than a year. The patisiran EAP aims to provide access to patisiran for patients with ATTR amyloidosis with cardiomyopathy who have had an inadequate response to or
cannot tolerate currently available treatment.
Published results from Phase 1 study of zilebesiran in the New England Journal of Medicine,
showing that, compared to placebo, zilebesiran was associated with dose-dependent reductions in serum AGT, achieving tonic blood pressure control with consistent and durable blood pressure reduction throughout a
24-hour period, sustained up to six months after single doses of 200 mg of zilebesiran. Zilebesiran also demonstrated an acceptable safety profile supporting continued clinical development.
Completed enrollment in the KARDIA-2 Phase 2 study, evaluating the safety and efficacy of zilebesiran in patients with
uncontrolled hypertension when added on top of another antihypertensive medication.
Reported updated positive interim results for the ongoing single ascending dose (SAD) portion of the Phase 1 study of
ALN-APP in patients with early-onset Alzheimer s disease (EOAD) at the 2023 Alzheimer s Association International Conference (AAIC).
Sanofi announced that results from the ATLAS-INH and ATLAS-A/B studies
evaluating the efficacy and safety of fitusiran were published respectively in The Lancet and The Lancet Haematology, reinforcing the potential of investigational fitusiran to transform the current standard of care and address
unmet needs for all types of hemophilia, regardless of inhibitor status.
Additional Key Pipeline Progress:
Additional Business Updates
Financial Results for the Quarter Ended June 30,
| Three Months Ended June 30, | ||||||||
| (in thousands, except per share amounts) | 2023 | 2022 | ||||||
| Net product revenues | $ | 305,705 | $ | 213,515 | ||||
| Net revenue from collaborations | $ | 5,844 | $ | 9,025 | ||||
| Royalty revenue | $ | 7,205 | $ | 2,278 | ||||
| GAAP Operating loss | $ | (229,831 | ) | $ | (191,686 | ) | ||
| Non-GAAP Operating loss* | $ | (154,029 | ) | $ | (161,215 | ) | ||
| GAAP Net loss | $ | (276,024 | ) | $ | (277,402 | ) | ||
| Non-GAAP Net loss* | $ | (201,622 | ) | $ | (245,834 | ) | ||
| GAAP Net loss per common share - basic and diluted | $ | (2.21 | ) | $ | (2.29 | ) | ||
| Non-GAAP Net loss per common share - basic and diluted* | $ | (1.62 | ) | $ | (2.03 | ) |
Net Product Revenues
| Three Months Ended June 30, | Year over Year % Growth | |||||||||||||||
| (in thousands, except percentages) | 2023 | 2022 | As Reported | At CER* | ||||||||||||
| ONPATTRO net product revenues | $ | 91,458 | $ | 153,428 | (40 | )% | (40 | )% | ||||||||
| AMVUTTRA net product revenues | 132,136 | N/A | N/A | |||||||||||||
| Total TTR net product revenues | $ | 223,594 | $ | 153,428 | 46 | % | 47 | % | ||||||||
| GIVLAARI net product revenues | 57,899 | 45,150 | 28 | % | 28 | % | ||||||||||
| OXLUMO net product revenues | 24,212 | 14,937 | 62 | % | 62 | % | ||||||||||
| Total net product revenues | $ | 305,705 | $ | 213,515 | 43 | % | 44 | % |
Net Revenues from Collaborations
| Three Months Ended June 30, | 2023 vs 2022 | |||||||||||
| (in thousands, except percentages) | 2023 | 2022 | % Change* | |||||||||
| Cost of goods sold | $ | 75,336 | $ | 34,038 | 121 | % | ||||||
| Cost of goods sold as a percentage of net product revenues | 24.6 | % | 15.9 | % | 8.7 | % | ||||||
| Cost of collaborations | $ | 10,034 | $ | 6,770 | 48 | % | ||||||
| GAAP research and development expenses | $ | 248,526 | $ | 205,712 | 21 | % | ||||||
| Non-GAAP research and development expenses | $ | 215,725 | $ | 195,074 | 11 | % | ||||||
| GAAP selling, general and administrative expenses | $ | 214,689 | $ | 169,984 | 26 | % | ||||||
| Non-GAAP selling, general and administrative expenses | $ | 171,688 | $ | 150,151 | 14 | % |
Research & Development (R&D) Expenses
Selling, General & Administrative (SG&A) Expenses
Other Financial Highlights
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.
Full year 2023 financial guidance is reiterated and consists of the following:
| Combined net product revenues for ONPATTRO, AMVUTTRA, GIVLAARI and OXLUMO 1,2 | $1,200 million $1,285 million | |
| Net Product Revenue Growth vs. 2022 at reported Fx rates 1 | 34% to 44% | |
| Net Product Revenue Growth vs. 2022 at constant exchange rates* | 34% to 44% | |
| Net revenues from collaborations and royalties | $100 million $175 million | |
| GAAP R&D and SG&A expenses | $1,790 million $1,885 million | |
| Non-GAAP R&D and SG&A expenses 3 | $1,575 million $1,650 million |
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 loss, Non-GAAP Net loss, Non-GAAP Net loss per common share basic and
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 the balance sheet.
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.
Management will provide an update on the Company and discuss second quarter 2023 results as well as expectations for the future via
conference call on Thursday, August 3, 2023 at 8:30 am ET. To access the call, please register online at https://register.vevent.com/register/BI8e6d8bfa374f41278e284759aa762e9e. 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.
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.
AMVUTTRA (vutrisiran)
(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 relative to baseline and improves other key measures of disease burden relative to external placebo in patients with the polyneuropathy of hATTR amyloidosis. For more
information about AMVUTTRA, including the full U.S. Prescribing Information, visit AMVUTTRA.com.
About GIVLAARI (givosiran)
GIVLAARI (givosiran) 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 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.
OXLUMO (lumasiran) is an RNAi therapeutic targeting hydroxyacid oxidase 1
(HAO1). HAO1 encodes glycolate oxidase (GO). Thus, by silencing HAO1 and depleting the GO enzyme, OXLUMO inhibits production of oxalate the metabolite that directly contributes to the pathophysiology of PH1. OXLUMO utilizes Alnylam s
Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. OXLUMO has received regulatory approvals from the U.S. Food and Drug
Administration (FDA) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients and from the European Medicines Agency (EMA) for the treatment of PH1 in all age groups. 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. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. In the