ALNYLAM PHARMACEUTICALS, INC.
For the period ended Jun 30, 2025. Filed Jul 31, 2025.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Jun 30, 2025 · Filed Jul 31, 2025
ALNY$155.94-0.5%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-03-31; filed 2025-05-01.
0001178670-25-000052
ITEM 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONSCurrent report: 10-Q
Period ended 2025-06-30; filed 2025-07-31.
0001178670-25-000090
ITEM 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONSThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 105 previous paragraphs and 107 current paragraphs.
68 unchanged; 31 reworded; 5 added; 4 removed; 2 previous and 3 current paragraphs rearranged.
Tables are not compared: 10 previous and 10 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (5)
Current report
•Achieved approvals of AMVUTTRA for the treatment of ATTR amyloidosis with cardiomyopathy in numerous global markets.
Current report
◦Received regulatory approvals from ANVISA, the EC, the MHRA, and the PMDA.
Current report
•recognition of $185.0 million of revenue under our Regeneron Collaboration in the six months ended June 30, 2024; and
Current report
Selling, general and administrative expenses for the three and six months ended June 30, 2025 increased as compared to the same periods in 2024, primarily due to higher employee compensation costs, including stock-based compensation, and increased marketing investment associated with AMVUTTRA launch in ATTR-CM.
Current report
We calculate our provision for income taxes during interim reporting periods by applying an estimate of the annual effective tax rate for the full year to pretax income or loss excluding unusual or infrequent occurring discrete items. As a result, we may experience significant fluctuations in the effective book tax rate (that is, our tax expense divided by pre-tax book income) from period to period.
Removed paragraphs (4)
Previous report
•Marketing authorization applications based on data from the HELIOS-B Phase 3 clinical trial are currently under review by several global health agencies including the EMA and the Japanese PMDA.
Previous report
•increased revenue recognized in connection with our Regeneron Collaboration as a result of an increase in activities under our licensed programs; and
Previous report
•increased stock-based compensation expense;
Previous report
For the three months ended March 31, 2025, the increase in selling, general and administrative expenses, as compared to the same period in 2024, was primarily due to increased marketing investment associated with the promotion of our TTR therapies and increased employee compensation expenses.
Reworded paragraphs (31)
Previous wording
AMVUTTRA is approved in the U.S. for the treatment of hereditary transthyretin-mediated amyloidosis, or hATTR amyloidosis, with polyneuropathy in adults, in the European Union, or EU, and the United Kingdom, or UK, for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, in Japan for the treatment of transthyretin, or TTR, type familial amyloidosis with polyneuropathy, and in multiple additional countries. Regulatory reviews continue in other territories. In March 2025, we announced that the United States Food and Drug Administration, or the FDA, approved our supplemental New Drug Application, or sNDA, for AMVUTTRA for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. AMVUTTRA was also approved by the Brazilian Health Regulatory Agency, or ANVISA, for the treatment of ATTR amyloidosis with cardiomyopathy. In addition, vutrisiran (the generic name for AMVUTTRA) is also under regulatory review by the European Medicines Agency, or the EMA, the Japanese Health Authority, or PMDA, and the Colombian Health Authority for the treatment of ATTR amyloidosis with cardiomyopathy.
Current wording
AMVUTTRA is approved in the U.S. for the treatment of hereditary transthyretin-mediated amyloidosis, or hATTR amyloidosis, with polyneuropathy in adults, in the European Union, or EU, and the United Kingdom, or UK, for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, in Japan for the treatment of transthyretin, or TTR, type familial amyloidosis with polyneuropathy, and in multiple additional countries. Regulatory reviews continue in other territories. In March 2025, the United States Food and Drug Administration, or the FDA, approved our supplemental New Drug Application, or sNDA, for AMVUTTRA for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. In June 2025, the European Commission, or EC, granted approval of AMVUTTRA for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy, following a positive opinion from the Committee for Medicinal Products for Human Use of the European Medicines Agency. AMVUTTRA has also been approved by each of the Brazilian Health Regulatory Agency, or ANVISA, the Japanese Health Authority, or PMDA, the UK’s Medicines and Healthcare Products Regulatory Agency, or MHRA, and the Colombian Health Authority for the treatment of ATTR amyloidosis with cardiomyopathy.
Previous wording
ONPATTRO is approved in the U.S. for the treatment of the polyneuropathy of hATTR amyloidosis in adults and has also been approved in the EU for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, in Japan for the treatment of TTR-type familial amyloidosis with polyneuropathy, and in multiple additional countries. In February 2025, ONPATTRO received regulatory approval from ANVISA for the treatment of ATTR amyloidosis with cardiomyopathy.
Current wording
ONPATTRO is approved in the United States, or U.S., for the treatment of the polyneuropathy of hATTR amyloidosis in adults and has also been approved in the EU for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, in Japan for the treatment of TTR-type familial amyloidosis with polyneuropathy, and in multiple additional countries. In February 2025, ONPATTRO received regulatory approval from ANVISA for the treatment of ATTR amyloidosis with cardiomyopathy.
Previous wording
Leqvio (inclisiran), our fifth product, is being developed and commercialized by our collaborator Novartis AG, or Novartis, and has received marketing authorization from the European Commission, or EC, for the treatment of adults with hypercholesterolemia or mixed dyslipidemia and from the FDA as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia, or HeFH, or clinical atherosclerotic cardiovascular disease, or ASCVD, who require additional lowering of low-density lipoprotein cholesterol, or LDL-C. In July 2023, the FDA approved an expanded indication for Leqvio to include treatment of adults with high LDL-C and who are at increased risk of heart disease. Leqvio has also been approved in China and Japan, and as of the end of April 2025, Leqvio had been registered in more than 100 countries.
Current wording
Leqvio (inclisiran), our fifth product, is being developed and commercialized by our collaborator Novartis AG, or Novartis, and has received marketing authorization from the EC for the treatment of adults with hypercholesterolemia or mixed dyslipidemia and from the FDA as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia, or HeFH, or clinical atherosclerotic cardiovascular disease, or ASCVD, who require additional lowering of low-density lipoprotein cholesterol, or LDL-C. In July 2023, the FDA approved an expanded indication for Leqvio to include treatment of adults with high LDL-C and who are at increased risk of heart disease. Leqvio has also been approved in China and Japan, and as of the end of June 2025, Leqvio had been registered in more than 106 countries worldwide and was commercially available in 86 countries.
Previous wording
In addition to our marketed products, as part of our Alnylam P 5 x25 strategy, we have multiple drivers of future growth, including the development of transformative medicines to treat prevalent disease. In addition to Leqvio, we are advancing zilebesiran, an investigational, subcutaneously administered RNAi therapeutic targeting angiotensinogen in development for the treatment of hypertension. In 2023, we entered into a Collaboration and License Agreement, or the Roche Collaboration and License Agreement, with F. Hoffmann-La Roche Ltd. and Genentech, Inc. or, collectively, Roche, pursuant to which we established a worldwide, strategic collaboration for the joint development and commercialization of zilebesiran. In March 2024, we reported positive topline results from our KARDIA-2 clinical trial, which is designed to evaluate the safety and efficacy of zilebesiran administered biannually as a concomitant therapy in patients whose blood pressure is not adequately controlled by a standard of care antihypertensive medication. In April 2024, we dosed the first patient in our KARDIA-3 Phase 2 clinical trial, which is designed to evaluate the efficacy and safety of zilebesiran as an add-on therapy in adult patients with high cardiovascular risk and uncontrolled hypertension despite treatment with two to four standard of care antihypertensive medications, and we expect to report results from the KARDIA-3 Phase 2 clinical trial in the second half of 2025. In January 2025, we announced that we expect to initiate a Phase 3 cardiovascular outcomes clinical trial in the second half of 2025.
Current wording
In addition to our marketed products, as part of our Alnylam P 5 x25 strategy, we have multiple drivers of future growth, including the development of transformative medicines to treat prevalent disease. In addition to Leqvio, we are advancing zilebesiran, an investigational, subcutaneously administered RNAi therapeutic targeting angiotensinogen in development for the treatment of hypertension. In 2023, we entered into a Collaboration and License Agreement, or the Roche Collaboration and License Agreement, with F. Hoffmann-La Roche Ltd. and Genentech, Inc. or, collectively, Roche, pursuant to which we established a worldwide, strategic collaboration for the joint development and commercialization of zilebesiran. In March 2024, we reported positive topline results from our KARDIA-2 clinical trial, which is designed to evaluate the safety and efficacy of zilebesiran administered biannually as a concomitant therapy in patients whose blood pressure is not adequately controlled by a standard of care antihypertensive medication. We expect to report results from our KARDIA-3 Phase 2 clinical trial, which is designed to evaluate the efficacy and safety of zilebesiran as an add-on therapy in adult patients with high cardiovascular risk and uncontrolled hypertension despite treatment with two to four standard of care antihypertensive medications, in the second half of 2025. We expect to initiate a Phase 3 cardiovascular outcomes clinical trial in the second half of 2025 following our reporting of results from the KARDIA-3 Phase 2 clinical trial.
Previous wording
We are advancing nucresiran (formerly ALN-TTRsc04), an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis. In November 2024, we announced positive results from the ongoing Phase 1 clinical trial of nucresiran in healthy volunteers. These results demonstrated that a single dose of nucresiran at 300 mg or higher resulted in mean reductions of serum TTR of greater than 90% from baseline achieved at Day 15 and were sustained through at least Day 180. We expect to initiate the TRITON-CM Phase 3 clinical trial of nucresiran in patients with ATTR amyloidosis with cardiomyopathy in the first half of 2025, and expect to initiate the TRITON-PN Phase 3 clinical trial of nucresiran in patients with hATTR polyneuropathy in late 2025.
Current wording
We are advancing nucresiran (formerly ALN-TTRsc04), an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis. In November 2024, we announced positive results from the ongoing Phase 1 clinical trial of nucresiran in healthy volunteers. These results demonstrated that a single dose of nucresiran at 300 mg or higher resulted in mean reductions of serum TTR of greater than 90% from baseline achieved at Day 15 and were sustained through at least Day 180. We have initiated the TRITON-CM Phase 3 clinical trial of nucresiran in patients with ATTR amyloidosis with cardiomyopathy and expect to initiate the TRITON-PN Phase 3 clinical trial of nucresiran in patients with hATTR polyneuropathy in late 2025.
Previous wording
We are also advancing mivelsiran (formerly ALN-APP), an investigational RNAi therapeutic targeting amyloid precursor protein in development for the treatment of Alzheimer’s disease, or AD, and cerebral amyloid angiopathy, or CAA. In October 2024, we announced positive initial results from the multiple dose portion of the Phase 1 clinical trial of mivelsiran in patients with early-onset AD. We expect to initiate a Phase 2 clinical trial of mivelsiran in patients with AD in the second half of 2025. In July 2024, we initiated dosing in the cAPPricorn-1 Phase 2 clinical trial of mivelsiran in patients with CAA.
Current wording
We are also advancing mivelsiran (formerly ALN-APP), an investigational RNAi therapeutic targeting amyloid precursor protein in development for the treatment of Alzheimer’s disease, or AD, and cerebral amyloid angiopathy, or CAA. In July 2025, we presented multiple and single dose data from the Phase 1 clinical trial of mivelsiran in patients with early-onset AD. These data demonstrated that single and multiple doses of mivelsiran were generally well tolerated and demonstrated robust, durable, dose-dependent reductions of soluble amyloid precursor protein beta (sAPPβ) in cerebrospinal fluid. Further lowering of sAPPβ was observed after administration of a second 50 mg dose of mivelsiran. We expect to initiate a Phase 2 clinical trial of mivelsiran in patients with AD in the second half of 2025. In July 2024, we initiated dosing in the cAPPricorn-1 Phase 2 clinical trial of mivelsiran in patients with CAA.
Previous wording
We have incurred significant losses since we commenced operations in 2002 and as of March 31, 2025, we had an accumulated deficit of $7.35 billion. Historically, we have generated losses principally from costs associated with research and development activities, acquiring, filing and expanding intellectual property rights, and selling, general and administrative costs. As a result of planned expenditures for research and development activities relating to our research platform, our drug development programs, including clinical trial and manufacturing costs, the continued build-out of late-stage clinical and commercial capabilities, including global commercial operations, continued management and growth of our patent portfolio, collaborations and general corporate activities, we may incur additional operating losses. While we are targeting financial self-sustainability by the end of 2025, we will continue to require significant resources over the next several years as we expand our efforts to discover, develop and commercialize RNAi therapeutics. We anticipate that our operating results will continue to fluctuate for the foreseeable future, therefore, period-to-period comparisons should not be relied upon as predictive of the results in future periods.
Current wording
We have incurred significant losses since we commenced operations in 2002 and as of June 30, 2025, we had an accumulated deficit of $7.41 billion. Historically, we have generated losses principally from costs associated with research and development activities, acquiring, filing and expanding intellectual property rights, and selling, general and administrative costs. As a result of planned expenditures for research and development activities relating to our research platform, our drug development programs, including clinical trial and manufacturing costs, the continued build-out of late-stage clinical and commercial capabilities, including global commercial operations, continued management and growth of our patent portfolio, collaborations and general corporate activities, we may incur additional operating losses. While we are targeting financial self-sustainability by the end of 2025, we will continue to require significant resources over the next several years as we expand our efforts to discover, develop and commercialize RNAi therapeutics. We anticipate that our operating results will continue to fluctuate for the foreseeable future, and therefore, period-to-period comparisons should not be relied upon as predictive of the results in future periods.
Previous wording
Our broad pipeline includes six approved products and multiple late and early-stage investigational RNAi therapeutics across a broad range of disease areas and indications. We describe our commercial and clinical-stage pipeline in more detail below. The clinical-stage therapeutics described below are in various stages of clinical development and the scientific information included about these therapeutics is preliminary and investigative. These clinical-stage therapeutics have not been approved by the FDA, EMA, or any other health authority and no conclusions can or should be drawn regarding the safety or efficacy of these investigational therapeutics.
Current wording
Our broad pipeline includes six approved products and multiple late and early-stage investigational RNAi therapeutics across a broad range of disease areas and indications. We describe our commercial and clinical-stage pipeline in more detail below. The clinical-stage therapeutics described below are in various stages of clinical development and the scientific information included about these therapeutics is preliminary and investigative. These clinical-stage therapeutics have not been approved by the FDA, European Medicines Agency, or EMA, or any other health authority and no conclusions can or should be drawn regarding the safety or efficacy of these investigational therapeutics.
Previous wording
During the first quarter of 2025 and recent period, we reported the following updates from our commercially approved products and our late-stage clinical programs:
Current wording
During the second quarter of 2025 and recent period, we reported the following updates from our commercially approved products and our late-stage clinical programs:
Previous wording
•We achieved global net product revenues for AMVUTTRA and ONPATTRO for the first quarter of 2025 of $310.0 million and $49.5 million, respectively.
Current wording
•We achieved global net product revenues for AMVUTTRA and ONPATTRO for the second quarter of 2025 of $492.0 million and $52.5 million, respectively.
Previous wording
•We achieved global net product revenues for GIVLAARI and OXLUMO for the first quarter of 2025 of $67.0 million and $42.1 million, respectively.
Current wording
•We achieved global net product revenues for GIVLAARI and OXLUMO for the second quarter of 2025 of $80.8 million and $46.9 million, respectively.
Previous wording
•Received U.S. FDA approval of the sNDA for AMVUTTRA for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.
Current wording
◦Announced that nucresiran received Fast Track Designation from the FDA’s Division of Cardiology and Nephrology for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin mediated amyloidosis in adults to reduce mortality, cardiovascular hospitalizations, and urgent heart failure visits.
Previous wording
Regeneron. In April 2019, we entered into a global, strategic collaboration with Regeneron to discover, develop and commercialize RNAi therapeutics for a broad range of diseases by addressing therapeutic targets expressed in the eye and CNS, in addition to a select number of targets expressed in the liver, which we refer to as the Regeneron Collaboration. The Regeneron Collaboration is governed by a Master Agreement, referred to as the Regeneron Master Agreement, which became effective in May 2019.
Current wording
Regeneron. In April 2019, we entered into a global, strategic collaboration with Regeneron to discover, develop and commercialize RNAi therapeutics for a broad range of diseases by addressing therapeutic targets expressed in the eye and CNS, in addition to a select number of targets expressed in the liver, which we refer to as the Regeneron Collaboration. The
Previous wording
Net product revenues increased during the three months ended March 31, 2025, as compared to the same period in 2024, due to growth from AMVUTTRA driven by increased patient demand, partially offset by a decrease in ONPATTRO due to patient switches to AMVUTTRA, as well as an increased number patients on GIVLAARI.
Current wording
Net product revenues increased during the three and six months ended June 30, 2025, as compared to the same period in 2024, primarily due to growth from AMVUTTRA driven by increased patient demand, mainly in patients with ATTR-CM in the U.S., which was partially offset by a decrease in ONPATTRO due to patient switches to AMVUTTRA, and due to growth from an increased number of patients on GIVLAARI and OXLUMO.
Previous wording
Net revenues from collaborations decreased during the three months ended March 31, 2025, as compared to the same period in 2024, primarily driven by:
Current wording
Net revenues from collaborations decreased during the three months ended June 30, 2025, as compared to the same period in 2024, primarily driven by recognition of $185.0 million of revenue under our collaboration with Regeneron Pharmaceuticals in the three months ended June 30, 2024, as we modified the collaboration in June 2024 and provided Regeneron with an exclusive license to develop, manufacture and commercialize cemdisiran as a monotherapy. Net revenues from collaborations decreased during the six months ended June 30, 2025, as compared to the same period in 2024, primarily driven by:
Previous wording
•recognition of $65.0 million of milestone revenue under our Roche Collaboration associated with the dosing of the first patient in the zilebesiran KARDIA-3 clinical trial during the three months ended March 31, 2024.
Current wording
•recognition of $65.0 million of milestone revenue under our Roche Collaboration in March 2024 associated with the dosing of the first patient in the zilebesiran KARDIA-3 Phase 2 clinical trial.
Previous wording
Royalty revenue increased during the three months ended March 31, 2025, as compared to the same period in 2024, due to increased volume and rate of royalties earned from global net sales of Leqvio by our collaborator, Novartis.
Current wording
Royalty revenue increased during the three and six months ended June 30, 2025, as compared to the same period in 2024, due to increased volume and rate of royalties earned from global net sales of Leqvio by our collaborator, Novartis.
Previous wording
Recognition of our combined net revenues from collaborations and royalty revenue is dependent on a variety of factors, including the level of work reimbursed by collaborators, achievement of milestones under our collaboration agreements, and royalties associated with sales of Leqvio and Qfitlia. We expect net revenues from collaborations will increase in 2025, as compared to 2024, primarily driven by higher anticipated revenues under our Roche Collaboration and License Agreement. We expect our royalty revenue will increase in 2025, as compared to 2024, due to the continued growth of royalties earned from global net sales of Leqvio by our collaborator, Novartis, and Qfitlia by our collaborator, Sanofi.
Current wording
Recognition of our combined net revenues from collaborations and royalty revenue is dependent on a variety of factors, including the level of work reimbursed by collaborators, achievement of milestones under our collaboration agreements, and royalties associated with sales of Leqvio and Qfitlia. We expect net revenues from collaborations will increase in 2025, as compared to 2024, primarily driven by higher anticipated revenues under our Roche Collaboration and License Agreement. We expect our royalty revenue will increase in 2025, as compared to 2024, due to the continued growth of royalties earned from global net sales of Leqvio by our collaborator, Novartis, and royalties earned from global net sales of Qfitlia by our collaborator, Sanofi.
Previous wording
We expect our cost of goods sold, including cost of goods sold as a percentage of net product revenues, will increase during 2025, as compared to 2024, primarily as a result of an expected increase in net product revenues and increased royalties payable on net sales of AMVUTTRA.
Current wording
We expect our cost of goods sold, including cost of goods sold as a percentage of net product revenues, will increase during 2025, as compared to 2024, primarily as a result of an expected increase in sales of AMVUTTRA and an associated increase in royalties payable on net sales of AMVUTTRA.
Previous wording
Cost of collaborations and royalties decreased during the three months ended March 31, 2025, as compared to the same period in 2024, primarily due to decreased demand for GalNAc material supplied to our collaborators in support of certain product manufacturing as our collaborators transition to producing the material independently.
Current wording
Cost of collaborations and royalties decreased during the three and six months ended June 30, 2025, as compared to the same periods in 2024, primarily due to decreased demand for GalNAc material supplied to our collaborators in support of certain product manufacturing as our collaborators transition to producing the material independently.
Previous wording
We expect cost of collaborations and royalties will continue to decrease during 2025, as compared to 2024, as a result of our collaborators continuing to transition to produce GalNAc independently.
Current wording
We expect cost of collaborations and royalties will continue to decrease during 2025, as compared to 2024, as a result of our collaborators continuing to transition to produce GalNAc material independently.
Previous wording
Research and development expenses for the three months ended March 31, 2025 increased 2% as compared to the same period in 2024, which was primarily due to the following:
Current wording
Research and development expenses for the three and six months ended June 30, 2025 increased as compared to the same periods in 2024, which was primarily due to:
Previous wording
•increased clinical trial expenses primarily associated with zilebesiran in the KARDIA-3 clinical trial and mivelsiran in the cAPPRicorn-1 clinical trial due to increased Phase 2 activities, as well as startup activities and other clinical trial expenses associated with zilebesiran in the KARDIA-6 cardiovascular outcomes trial and nucresiran in the TRITON-CM Phase 3 clinical trial in patients with ATTR amyloidosis with cardiomyopathy.
Current wording
•increased clinical trial expenses associated with startup activities for zilebesiran in the Phase 3 cardiovascular outcomes trial and nucresiran in the TRITON-CM Phase 3 trial in patients with ATTR-CM.
Previous wording
•decreased expenses within other clinical programs, specifically the HELIOS-B Phase 3 clinical trial of vutrisiran due to the wind-down of clinical activities.
Current wording
•decreased expenses within other clinical programs, in particular for zilebesiran in the KARDIA-2 Phase 2 clinical trial due to the wind-down of clinical activities.
Previous wording
We expect that research and development expenses combined with selling, general and administrative expenses will continue to increase during 2025, as compared to 2024, as we continue to build out our global commercial and compliance infrastructure, launch our current commercial products into new markets, prepare for future commercial product launches, including the launch of AMVUTTRA for the treatment of ATTR amyloidosis with cardiomyopathy, advance our product candidates, including collaborated programs, into later-stage development, advance and develop our platform and preclinical pipeline, and prepare regulatory submissions. However, we expect that certain expenses will be variable depending on the timing of manufacturing batches, clinical trial enrollment and results, regulatory review of our product candidates and programs, and stock-based compensation expenses based on our determination regarding the probability of vesting for performance-based awards.
Current wording
We expect that research and development expenses combined with selling, general and administrative expenses will continue to increase during 2025, as compared to 2024, as we continue to build out our global commercial and compliance infrastructure, launch our current commercial products into new markets, prepare for future commercial product launches, including the continued launch of AMVUTTRA for the treatment of ATTR amyloidosis with cardiomyopathy, advance our product candidates, including collaborated programs, into later-stage development, advance and develop our platform and preclinical pipeline, and prepare regulatory submissions. However, we expect that certain expenses will be variable depending on the timing of manufacturing batches, clinical trial enrollment and results, regulatory review of our product candidates and programs, and stock-based compensation expenses based on our determination regarding the probability of vesting for performance-based awards.
Previous wording
Total other expense increased during the three months ended March 31, 2025, as compared to the same period in 2024, primarily due to increased loss associated with the change in fair value of the development derivative liability as a result of valuation updates primarily driven by the regulatory approval of AMVUTTRA for the treatment of ATTR amyloidosis with cardiomyopathy.
Current wording
Total other expense decreased during the three months ended June 30, 2025, as compared to the same period in 2024, primarily due to decreased loss associated with the change in fair value of the development derivative liability as a result of valuation updates during the three months ended June 30, 2024 driven by the regulatory approval of AMVUTTRA for the treatment of ATTR amyloidosis with cardiomyopathy. Total other expense decreased during the six months ended June 30, 2025, as compared to the same period in 2024, primarily due to increased net realized and unrealized foreign currency transaction gains, which was partially offset by increased interest expense associated with the liability related to the sale of future royalties and increased loss associated with the change in fair value of the development derivative liability.
Previous wording
The Company’s provision for income taxes relates to foreign and state income taxes. The provision for income taxes increased during the three months ended March 31, 2025, as compared to the same period in 2024, which primarily relates to foreign income taxes due to income generated in Switzerland. We expect to utilize net operating losses in Switzerland to offset taxable income and current taxes resulting in a deferred income tax provision. We continue to maintain a full valuation allowance against our net deferred tax assets in the U.S. and certain deferred tax assets in Switzerland.
Current wording
Our provision for income taxes relates to foreign and state income taxes. The provision for income taxes increased during the three and six months ended June 30, 2025, as compared to the same periods in 2024, which primarily relates to foreign income taxes due to income generated in Switzerland, as well as state income taxes in the U.S. We expect to utilize net operating losses in Switzerland to offset taxable income and current taxes resulting in a deferred income tax provision. We continue to maintain a full valuation allowance against our net deferred tax assets in the U.S. and certain deferred tax assets in Switzerland.
Previous wording
Net cash used in operating activities increased during the three months ended March 31, 2025, compared to the same period in 2024, primarily due to higher annual incentive bonus payouts, as well as interest payments associated with the liability related to the sale of future royalties, partially offset by stronger cash receipts from increased product sales.
Current wording
Net cash provided by operating activities decreased during the six months ended June 30, 2025, compared to the same period in 2024, primarily due to increased employee compensation and decreased cash received from our collaborators, partially offset by stronger cash receipts from increased product sales.
Previous wording
Net cash provided by investing activities increased during the three months ended March 31, 2025, compared to the same period in 2024, primarily due to the timing of sales, maturities, and purchases of our marketable securities.
Current wording
Net cash used in investing activities decreased during the six months ended June 30, 2025, compared to the same period in 2024, primarily due to the timing of sales, maturities, and purchases of our marketable securities.
Previous wording
Net cash provided by financing activities increased during the three months ended March 31, 2025, compared to the same period in 2024, primarily due to increased net proceeds from the issuance of common stock in connection with stock option exercises.
Current wording
Net cash provided by financing activities decreased during the six months ended June 30, 2025, compared to the same period in 2024, primarily due to lower net proceeds from the issuance of common stock in connection with stock option exercises, as well as higher net payments to BXLS V Bodyguard – PCP L.P. and BXLS Family Investment Partnership V – ESC L.P. for the development derivative liability.
Previous wording
Our expected working and other capital requirements are described in our 2024 Annual Report on Form 10-K in “Part II, Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations.” As of March 31, 2025, other than the changes disclosed in the “Notes to Condensed Consolidated Financial Statements” and “Liquidity and Capital Resources” section in this Quarterly Report on Form 10-Q, there have been no other material changes to our expected working and other capital requirements as described in our 2024 Annual Report on Form 10-K.
Current wording
Our expected working and other capital requirements are described in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 in “Part II, Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations.” As of June 30, 2025, other than the changes disclosed in the “Notes to Condensed Consolidated Financial Statements” and “Liquidity and Capital Resources” section in this Quarterly Report on Form 10-Q, there have been no other material changes to our expected working and other capital requirements as described in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024.
Rearranged current paragraphs (3)
Current report — wording carried from elsewhere
•Initiated the TRITON-CM Phase 3 trial of nucresiran in patients with ATTR amyloidosis with cardiomyopathy.
Current report — wording carried from elsewhere
Regeneron Collaboration is governed by a Master Agreement, referred to as the Regeneron Master Agreement, which became effective in May 2019.
Current report — wording carried from elsewhere
Cost of goods sold, including cost of goods sold as a percentage of net product revenues, increased during the three and six months ended June 30, 2025, as compared to the same periods in 2024, primarily as a result of increased sales of AMVUTTRA and an associated increase in royalties payable on net sales of AMVUTTRA.
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
•Received approval from ANVISA for AMVUTTRA for the treatment of ATTR amyloidosis with cardiomyopathy.
Previous report — wording carried elsewhere
Cost of goods sold as a percentage of net product revenues was consistent during the three months ended March 31, 2025, as compared to the same period in 2024.