Full Press Release Details
Alnylam Pharmaceuticals Reports Third Quarter 2022 Financial Results and Highlights Recent Period Activity
Achieved Third Quarter 2022 Global Net Product Revenues of $232 Million for ONPATTRO , AMVUTTRA , GIVLAARI , and OXLUMO -
Strong First Full Quarter of AMVUTTRA in U.S.: Achieving $25 Million in Net Product Revenues and Driving 30% U.S. Total TTR Growth Compared with Q2 2022 -
Reported Positive Results from the APOLLO-B Phase 3 Study of Patisiran, and Remains on Track for a Supplemental New Drug Application (sNDA) Submission in Late 2022 -
- Reiterated 2022 Combined Net Product Revenue Guidance of $870-$930 Million -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--October 27, 2022--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter ended September 30, 2022 and
reviewed recent business highlights.
"The third quarter of 2022 was one of strong execution across our commercial and clinical development operations. AMVUTTRA has completed its first full quarter on the market since its U.S. approval and launch in June and is off to a great
start, demonstrating the potential of our RNAi therapeutics portfolio in patients with hATTR amyloidosis with polyneuropathy which achieved 30% U.S. growth compared to Q2. We're also looking forward to potentially expanding the opportunity
for this franchise and are encouraged by the positive APOLLO-B Phase 3 results for patisiran in patients with ATTR amyloidosis with cardiomyopathy presented recently. We are on track to submit an sNDA for ONPATTRO by year-end and hope to
bring this treatment option to patients in 2023 assuming successful regulatory review and approval," said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. "The rest of our pipeline continues to progress well, and we are excited
for several important upcoming milestones, including results for a potential biannual dose regimen for vutrisiran, preliminary Phase 1 results for ALN-XDH in patients with gout, completion of enrollment in the KARDIA-2 Phase 2 study of
zilebesiran, and initiation of a Phase 1 study of ALN-TTRsc04 in healthy volunteers. We are encouraged by our steady and continuous execution on all fronts, and believe we are on track to achieve our Alnylam P5x25 goals and
become a top-tier biotech company."
Third Quarter 2022 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 2022 of $145 million and $25 million, respectively, representing a total TTR increase of 11% compared to Q2 2022, with the U.S. market contributing
30% total TTR growth underpinned by the strength of the AMVUTTRA launch.
Attained over 2,580 hATTR amyloidosis patients with polyneuropathy worldwide on commercial treatment with ONPATTRO or AMVUTTRA as of September 30, 2022, up from over 2,400 on commercial ONPATTRO as of June 30, 2022, representing 8% total
TTR quarterly growth.
Received 475 Start Forms in the U.S. for AMVUTTRA from launch through September 30, 2022, with 46% representing new patients and 54% representing patients switching from ONPATTRO.
GIVLAARI (givosiran)
Achieved global net product revenues for the third quarter of 2022 of $46 million, representing a 1% increase compared to Q2 2022.
Attained over 460 patients worldwide on commercial GIVLAARI treatment as of September 30, 2022, up from over 420 commercial patients as of June 30, 2022, representing 10% quarterly growth.
Achieved global net product revenues for the third quarter of 2022 of $16 million, representing a 10% increase compared to Q2 2022.
Attained over 230 patients worldwide on commercial OXLUMO treatment as of September 30, 2022, up from over 200 commercial patients as of June 30, 2022, representing 15% quarterly growth.
Launch in the U.S. and other markets is ongoing, with focus on patient onboarding, removing access hurdles and enhancing medical education.
Patisiran (the non-proprietary name for ONPATTRO), in development for the treatment of ATTR amyloidosis.
Reported positive results from the APOLLO-B Phase 3 study in patients with ATTR amyloidosis with cardiomyopathy. The Company remains on track to submit an sNDA for review by the United States Food and Drug Administration (FDA) by
Vutrisiran (the non-proprietary name for AMVUTTRA), in development for the treatment of ATTR amyloidosis.
Received marketing authorization for AMVUTTRA for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy in Europe and the UK, as well as approval for transthyretin (TTR)
type familial amyloidosis with polyneuropathy in Japan.
Alnylam announces today that it does not plan to conduct the optional interim analysis for the HELIOS-B Phase 3 study in patients with ATTR amyloidosis with cardiomyopathy. The study remains on track for topline results in early 2024.
The Company also announces today that it is considering options for the best path toward advancing an RNAi therapeutic for the treatment of Stargardt Disease. At this time, it will not initiate a Phase 3 study of vutrisiran in Stargardt
Disease in late 2022, as previously guided, as it continues to evaluate the impact of the Inflation Reduction Act.
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.
Based on the successful outcome of the ILLUMINATE-C study in children and adults with advanced PH1, received approval from the U.S. FDA of an sNDA for OXLUMO, expanding the indication for the treatment of PH1 to lower urinary oxalate and
plasma oxalate levels in pediatric and adult patients, and received approval from the European Medicines Agency (EMA) of a Type II variation to include the ILLUMINATE-C data in the label.
Cemdisiran, in development for the treatment of complement-mediated diseases, in collaboration with Regeneron.
Reported positive results from the Phase 2 study in patients with immunoglobulin A nephropathy (IgAN).
Alnylam is working with Regeneron to finalize plans for the Phase 3 clinical development of cemdisiran in IgAN.
Early- and mid-stage investigational RNAi therapeutic pipeline programs and RNAi platform
Published preclinical results in Nature Biotechnology based on novel conjugate technology facilitating delivery of siRNA to the CNS and other extrahepatic tissues.
Vir Biotechnology announced that the first patient has been dosed in the Phase 2 SOLSTICE clinical trial evaluating ALN-HBV02 (VIR-2218) and VIR-3434 as monotherapy and in combination for the treatment of people living with
chronic hepatitis D virus (HDV), which occurs as a simultaneous co-infection or super-infection alongside hepatitis B virus (HBV).
Published research findings in Nature Communications identifying mutations in the INHBE gene associated with protection against abdominal obesity and metabolic syndrome.
Reported preliminary data from the ongoing Phase 1 study of ALN-HSD, in development for the treatment of nonalcoholic steatohepatitis (NASH).
Alnylam announces today that enrollment and dose escalation continue in the Phase 1 study of ALN-APP in patients with early onset Alzheimer's Disease, in collaboration with Regeneron, and initial results are now expected in early
Filed a Clinical Trial Application (CTA) for ALN-TTRsc04, the first clinical program from the Company's IKARIA platform, aimed at achieving an annual dosing regimen with highly potent and reversible effects of TTR lowering.
Additional Business Updates
Appointed Piyush Sharma as Chief Ethics and Compliance Officer and Evan Lippman as Chief Corporate Development and Strategy Officer.
Issued $1,035 million convertible senior notes with proceeds primarily used to pay down Blackstone $700 million credit facility and approximately $200 million in prepayment premiums under the credit facility, the purchase of capped call
transactions, and underwriter fees.
Ranked #1 on Fast Company's fourth annual list of Best Workplaces for Innovators.
Named a Top Place to Work for Women by Fortune magazine.
In late 2022, Alnylam intends to:
Present a review of its pipeline and platform activities at its upcoming R&D Day being held virtually on Thursday, December 15, 2022.
Submit an sNDA to the FDA for review for ONPATTRO for the treatment of patients with ATTR amyloidosis with cardiomyopathy.
Report results on a biannual dose regimen for vutrisiran.
Initiate a Phase 1 study for ALN-TTRsc04 in healthy volunteers.
Complete enrollment in the Phase 2 study of lumasiran in patients with recurrent renal stones.
Complete enrollment in the KARDIA-2 Phase 2 study of zilebesiran (at or around year-end).
Report preliminary results from the Phase 1 study of ALN-XDH in patients with gout.
Submit CTA filings for ALN-KHK for the treatment of metabolic liver disease, including diabetes, and in collaboration with its partner Regeneron, submit an IND for ALN-PNP for the treatment of NASH.
Financial Results for the Quarter Ended September 30, 2022
Financial Highlights
| Three Months Ended September 30, | |||||||
| (in thousands, except per share amounts) | 2022 | 2021 | |||||
| Net product revenues | $ | 232,267 | $ | 167,044 | |||
| Net revenue from collaborations | $ | 29,297 | $ | 20,136 | |||
| Royalty revenue | $ | 2,742 | $ | 453 | |||
| GAAP Operating loss | $ | (258,040 | ) | $ | (181,677 | ) | |
| Non-GAAP Operating loss | $ | (129,922 | ) | $ | (148,310 | ) | |
| GAAP Other expense, net | $ | (147,903 | ) | $ | (22,559 | ) | |
| Non-GAAP Other expense, net | $ | (63,467 | ) | $ | (41,250 | ) | |
| GAAP Net loss | $ | (405,920 | ) | $ | (204,514 | ) | |
| Non-GAAP Net loss | $ | (193,366 | ) | $ | (189,838 | ) | |
| GAAP Net loss per common share - basic and diluted | $ | (3.32 | ) | $ | (1.72 | ) | |
| Non-GAAP Net loss per common share - basic and diluted | $ | (1.58 | ) | $ | (1.59 | ) |
Net Product Revenues
| Three Months Ended September 30, | Year over Year % Growth | ||||||||||
| (in thousands, except percentages) | 2022 | 2021 | As Reported | At CER* | |||||||
| ONPATTRO net product revenues | $ | 144,950 | $ | 120,317 | 20 | % | 31 | % | |||
| AMVUTTRA net product revenues | 25,229 | ---- | N/A | N/A | |||||||
| Total TTR net product revenues | 170,179 | 120,317 | 41 | % | 52 | % | |||||
| GIVLAARI net product revenues | 45,659 | 31,833 | 43 | % | 50 | % | |||||
| OXLUMO net product revenues | 16,429 | 14,894 | 10 | % | 20 | % | |||||
| Total net product revenues | $ | 232,267 | $ | 167,044 | 39 | % | 49 | % |
* CER = Constant Exchange Rate, representing growth calculated as if the exchange rates had remained unchanged from those used in the third quarter 2021. CER is a Non-GAAP measure.
Net product revenues increased 39% at actual currency during the third quarter of 2022, as compared to the prior year, and 49% at CER. The increase is primarily due to increased patients on ONPATTRO, AMVUTTRA, GIVLAARI, and OXLUMO.
Net Revenues from Collaborations
Net revenues from collaborations increased 45% during the third quarter 2022, as compared to the prior year, primarily due to an increase in revenue from our collaboration with Regeneron resulting from the timing of reimbursable
| Three Months Ended September 30, | |||||
| (in thousands) | 2022 | 2021 | |||
| GAAP research and development expenses | $ | 245,371 | $ | 194,572 | |
| Non-GAAP research and development expenses | $ | 192,409 | $ | 182,155 | |
| GAAP selling, general and administrative expenses | $ | 235,859 | $ | 142,075 | |
| Non-GAAP selling, general and administrative expenses | $ | 160,703 | $ | 121,125 |
Research & Development (R&D) Expenses
GAAP and non-GAAP R&D expenses increased during the third quarter 2022, as compared to the prior year, primarily due to increases in headcount to support our R&D pipeline and development expenses associated with the KARDIA-1 and
KARDIA-2 zilebesiran Phase 2 studies, offset by a decrease in the cost of clinical batches manufactured during the quarter. GAAP R&D expenses further increased due to increased stock-based compensation expense related to the accounting
for certain performance-based awards that vested during the period.
Selling, General & Administrative (SG&A) Expenses
GAAP and non-GAAP SG&A expenses increased during the third quarter 2022, as compared to the prior year, primarily due to increased headcount and other strategic investments in support of the U.S. AMVUTTRA launch and other corporate
purposes. GAAP SG&A expenses further increased due to stock-based compensation expense related to the accounting for certain performance-based awards that vested during the period.
Other Financial Highlights
GAAP Other expense, net, increased during the third quarter 2022 as compared to the prior year, primarily due to a $77 million loss on the extinguishment of the Blackstone credit agreement and a $25 million loss from the fair value
adjustment on the development derivative liability.
Cash, cash equivalents and marketable securities were $2.27 billion as of September 30, 2022 compared to $2.44 billion as of December 31, 2021 with the decrease primarily due to our year-to-date operating loss in 2022. This decrease was
largely offset by approximately $200 million received from employee option award exercises and approximately $135 million received from the issuance of convertible debt, net of repayment of borrowings, inclusive of prepayment premiums under
the credit facility, the purchase of capped call transactions, and underwriter fees.
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 4/28/2022 1 | Updated 10/27/2022 2 | ||
| Combined net product revenues for ONPATTRO, GIVLAARI, OXLUMO and AMVUTTRA | $870 million - $930 million | Unchanged | |
| Net revenues from collaborations and royalties | $175 million - $225 million | $100 million - $150 million | |
| GAAP R&D and SG&A expenses | $1,620 million - $1,700 million | Unchanged | |
| Non-GAAP R&D and SG&A expenses 3 | $1,390 million - $1,450 million | Unchanged | |
| 1 Prior FY 2022 guidance utilized 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 2 Updated FY 2022 guidance utilizes September 27, 2022 FX rates of: 1 EUR = 0.96 USD; 1 GBP = 1.08 USD; 1 CHF = 1.01 USD; 1 CAD = 0.73 USD, 1 USD = 145 JPY 3 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, 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.