Full Press Release Details
Alnylam Pharmaceuticals Reports First Quarter 2022 Financial Results and Highlights Recent Period Activity
Achieved First Quarter 2022 Global Net Product Revenues of $187 Million for ONPATTRO , GIVLAARI , and OXLUMO -
Reported Positive 18-Month Results from HELIOS-A Phase 3 Study of Vutrisiran in hATTR Amyloidosis Patients with Polyneuropathy -
Announced 3-Month Extension of Review Period for New Drug Application for Vutrisiran due to Amendment to Address Pending Inspection Classification at Third-Party Secondary
Packaging and Labeling Facility -
- Decreased 2022 Guidance Range for Combined Net Product Revenues from $900 Million - $1 Billion to $870 - $930 Million -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--April 28, 2022--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter ended
March 31, 2022 and reviewed recent business highlights.
"The first quarter of 2022 saw a steady increase in patients on therapy across all products in our commercial portfolio, despite combined product revenues that were down compared to Q4 2021, as anticipated, given several non-recurring revenue
benefits in the fourth quarter of 2021, as well as some pandemic-related impact experienced early in the quarter. We continue to make progress with our pipeline programs, including our TTR franchise, for which we reported positive 18-month
results from the HELIOS-A study of vutrisiran, and we remain on track for topline results from the APOLLO-B study of patisiran in the middle of this year," said Yvonne Greenstreet, MBChB, MBA, Chief Executive Officer of Alnylam. "With our
earlier stage programs, we're excited to have initiated our first clinical study of an Alnylam investigational RNAi therapeutic targeting a CNS disorder with the start of our Phase 1 study for ALN-APP in patients with early-onset Alzheimer's
disease, and are announcing today that the Phase 1 study of ALN-XDH in patients with gout also has been initiated. These programs underscore our commitment to advancing innovative new medicines into the clinic, and we anticipate data from both
programs later this year. We look forward to continuing our steady execution across our commercial products and advancement of our clinical development pipeline, with many key upcoming milestones to mark our progress toward achieving our Alnylam
First Quarter 2022 and Recent Significant Corporate Highlights
Commercial Performance
ONPATTRO (patisiran)
Achieved global net product revenues for the first quarter of 2022 of $137 million, representing a 1% decrease compared to Q4 2021.
Attained over 2,200 hATTR amyloidosis patients with polyneuropathy worldwide on commercial ONPATTRO treatment as of March 31, 2022, up from over 2,050 commercial patients at year end 2021, representing 7% quarterly growth.
GIVLAARI (givosiran)
Achieved global net product revenues for the first quarter of 2022 of $35 million, representing a 13% decrease compared to Q4 2021.
Attained over 400 patients worldwide on commercial GIVLAARI treatment as of March 31, 2022, up from over 350 commercial patients at year end 2021, representing 14% quarterly growth.
Achieved global net product revenues for the first quarter of 2022 of $15 million, representing a 24% decrease compared to Q4 2021.
Attained over 160 patients worldwide on commercial OXLUMO treatment as of March 31, 2022, up from over 140 commercial patients at year end 2021, representing 14% quarterly growth.
Launch in the U.S. and in other markets is ongoing, with focus on patient on-boarding, removing access hurdles and driving urgency to treat. Leqvio is now approved in more than 55 countries, with most awaiting reimbursement.
Vutrisiran, a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis and Stargardt disease
Reported positive results for 18-month endpoints and safety from the HELIOS-A Phase 3 study in hATTR amyloidosis patients with polyneuropathy.
Vutrisiran met all 18-month secondary endpoints, including statistically significant improvements in neuropathy impairment, quality of life (QoL), gait speed, nutritional status and overall disability, relative to external placebo.
Vutrisiran continued to demonstrate halting or reversal of polyneuropathy, with improvements in neuropathy impairment and QoL, relative to baseline.
Additionally, vutrisiran continued to demonstrate an encouraging safety and tolerability profile.
At month 18, vutrisiran also showed improvements in exploratory cardiac endpoints, including NT-proBNP, a measure of cardiac stress; certain echocardiographic parameters, relative to placebo; and technetium uptake in the heart,
providing potential evidence for reduced cardiac amyloid burden.
Announced that the U.S. Food and Drug Administration (FDA) has extended the review timeline of the New Drug Application (NDA) for vutrisiran to allow for review of newly added information related to the new secondary packaging and labeling
The updated Prescription Drug User Fee Act (PDUFA) goal date to allow for this review is July 14, 2022. No additional clinical data have been requested by the FDA.
Vutrisiran is also under review by the European Medicines Agency (EMA), the Brazilian Health Regulatory Agency (ANVISA) and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA).
Lumasiran (the non-proprietary name for OXLUMO), for the treatment of primary hyperoxaluria type 1 (PH1), and in development for the treatment of recurrent kidney stone disease
Announced U.S. FDA acceptance of the Company's supplemental NDA for OXLUMO for the treatment of advanced PH1.
The FDA has set a PDUFA goal date of October 6, 2022.
Additionally, a Type II variation for lumasiran to amend the label to further inform on the use of lumasiran in patients with advanced PH1 was submitted in December 2021 and is undergoing review by the Committee for Medicinal Products for
Human Use (CHMP) with a decision expected by year-end.
Early- and mid-stage investigational RNAi therapeutic pipeline programs and RNAi platform
Initiated a Phase 1 study of ALN-APP in patients with early-onset Alzheimer's disease (EOAD).
The Company announces today that it has initiated a Phase 1 study of ALN-XDH in patients with gout.
The Company announces today that, due in part to impacts related to COVID-19 as well as the ongoing situation in Ukraine, it now expects to complete enrollment in the KARDIA-1 Phase 2 monotherapy study of zilebesiran in patients
with mild-to-moderate hypertension in early 2023, with topline results now expected in mid-2023.
Vir Biotechnology reported encouraging results from its Phase 2 trial evaluating ALN-HBV02 (VIR-2218), an investigational RNAi therapeutic in development for the treatment of chronic hepatitis B virus (HBV) infection, in
combination with its investigational monoclonal antibody VIR-3434.
Additional Business Updates
Appointed Akshay Vaishnaw, M.D., Ph.D., formerly President, Research and Development, as President effective January 1, 2022.
Appointed Indrani Franchini as Chief Legal Officer effective January 31, 2022.
Entered into a collaboration with Novartis to explore a targeted therapy designed to promote the regrowth of functional liver cells and to provide an alternative to transplantation for patients with liver failure.
Launched a partnership with Our Future Health, the UK's largest ever health research program that aims to genotype samples from up to 5 million participants.
Published 2021 Corporate Responsibility Report.
Filed separate patent infringement suits against Pfizer, Inc. and Moderna, Inc., seeking damages for infringement of U.S. Patent No. 11,246,933 in the parties' manufacture and sale of their messenger RNA (mRNA) COVID-19 vaccines. The
patent relates to Alnylam's biodegradable cationic lipids that are foundational to the success of the mRNA COVID-19 vaccines.
In early and mid-2022, Alnylam intends to:
Launch vutrisiran in the U.S., assuming successful review and approval from the FDA, for the treatment of hATTR amyloidosis patients with polyneuropathy.
Report topline results from the APOLLO-B Phase 3 study of patisiran in ATTR amyloidosis patients with cardiomyopathy.
Report results from the Phase 2 monotherapy study of cemdisiran in patients with IgA nephropathy.
Novartis plans to report results from its ORION-3 Phase 2 study of inclisiran in patients with heterozygous familial hypercholesterolemia or pre-existing atherosclerotic cardiovascular disease on background statin +/- ezetimibe
Report topline results from Part B of the Phase 1 study of ALN-HSD in patients with NASH.
Financial Results for the Quarter Ended March 31, 2022
Financial Highlights
(in thousands, except per share amounts)
| Three Months Ended March 31, | |||||||
| 2022 | 2021 | ||||||
| ONPATTRO net product revenues | $ | 137,009 | $ | 101,951 | |||
| GIVLAARI net product revenues | 35,277 | 24,673 | |||||
| OXLUMO net product revenues | 14,586 | 9,145 | |||||
| Total net product revenues | $ | 186,872 | $ | 135,769 | |||
| Net revenue from collaborations | $ | 25,945 | $ | 41,797 | |||
| Royalty revenue | $ | 442 | $ | - | |||
| GAAP operating loss | $ | (146,732 | ) | $ | (186,254 | ) | |
| Non-GAAP operating loss | $ | (117,439 | ) | $ | (130,564 | ) | |
| GAAP other expense | $ | (92,624 | ) | $ | (13,021 | ) | |
| Non-GAAP other expense | $ | (61,463 | ) | $ | (60,037 | ) | |
| GAAP net loss | $ | (240,341 | ) | $ | (200,291 | ) | |
| Non-GAAP net loss | $ | (179,887 | ) | $ | (191,617 | ) | |
| GAAP net loss per common share - basic and diluted | $ | (2.00 | ) | $ | (1.71 | ) | |
| Non-GAAP net loss per common share - basic and diluted | $ | (1.49 | ) | $ | (1.64 | ) |
Net Product Revenues
Net product revenues increased 38% compared to the first quarter of 2021, primarily due to increased patients on ONPATTRO, GIVLAARI, and OXLUMO therapies, offset by an unfavorable impact from foreign exchange rates on our international
Net Revenues from Collaborations
Net revenues from collaborations decreased 38% compared to the first quarter of 2021, primarily due to a decrease in revenue from our collaboration with Regeneron.
First Quarter 2022 Expenses
| Three Months Ended March 31, | |||||
| 2022 | 2021 | ||||
| GAAP research and development expenses | $ | 169,893 | $ | 185,899 | |
| Non-GAAP research and development expenses | $ | 158,276 | $ | 161,524 | |
| GAAP selling, general and administrative expenses | $ | 154,471 | $ | 146,859 | |
| Non-GAAP selling, general and administrative expenses | $ | 136,795 | $ | 115,544 |
Research & Development (R&D) Expenses
GAAP R&D expenses decreased compared to the first quarter of 2021, primarily due to decreased stock-based compensation expense primarily due to the accounting for certain performance-based awards.
Selling, General & Administrative (SG&A) Expenses
GAAP and Non-GAAP SG&A expenses increased compared to the first quarter of 2021, primarily due to increased legal expenses, charitable contributions, and other expenses to support our strategic growth. On a GAAP basis, these increases
were offset by decreased stock-based compensation expense primarily due to the accounting for certain performance-based awards.
Other Financial Highlights
GAAP other expense increased compared to the first quarter of 2021, primarily due to approximately $80 million of realized and unrealized losses on our marketable equity securities holdings and an increase in interest expenses of
approximately $10 million due to additional draws on our term loan facility.
Cash, cash equivalents and marketable securities were $2.24 billion as of March 31, 2022 compared to $2.44 billion as of December 31, 2021 with the decrease primarily due to our operating loss in the first quarter of 2022.
A reconciliation of our GAAP to non-GAAP results for the current quarter is included in the tables of this press release.
2022 Updated Financial Guidance
Full year 2022 financial guidance has been updated as follows:
| Provided 2/10/2022 ($ millions) 1 | Updated 4/28/2022 ($ millions) 2 | |
| Combined net product revenues for ONPATTRO, GIVLAARI, OXLUMO and vutrisiran 3 | $900 - $1,000 | $870 - $930 |
| Net revenues from collaborations and royalties | $175 - $225 | Unchanged |
| GAAP R&D and SG&A expenses | $1,630 - $1,750 | $1,620 - $1,700 |
| Non-GAAP R&D and SG&A expenses 4 | $1,400 - $1,500 | $1,390 - $1,450 |
| 1 Prior FY 2022 Guidance utilized January 31, 2022 FX rates of: 1 EUR = 1.12 USD; 1 GBP = 1.34 USD; 1 CHF = 1.08 USD; 1 CAD = 0.79 USD, 1 USD = 115 JPY 2 Updated FY 2022 Guidance utilizes April 18, 2022 FX rates of: 1 EUR = 1.08 USD; 1 GBP = 1.31 USD; 1 CHF = 1.06 USD; 1 CAD = 0.79 USD, 1 USD = 126 JPY 3 Assuming FDA approval of vutrisiran by the July 14, 2022 PDUFA goal date 4 Primarily excludes $230-$250 million of stock-based compensation expense from estimated GAAP R&D and SG&A expenses. |
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 stock-based compensation expenses and realized and unrealized (gains) 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 (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 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 first quarter 2022 results as well as expectations for the future via conference call on Thursday, April 28, 2022 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 2758447. 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 2758447.
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.