Quarterly Report for Quarter Ending September 30, 2025 (Form 10-Q)

Management's Discussion and Analysisof Financial Condition and Results of Operations.

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our unaudited condensed financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and our audited financial statements and related notesfor the year ended December 31, 2024 included in our Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 28, 2025, which we refer to as the 2024 Annual Report on Form 10-K.

This Quarterly Report on Form 10-Q contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this Quarterly Report on Form 10-Q, including statements regarding our strategy, future operations, future financial position, future revenue, projected costs, prospects, plans and objectives of management and expected market growth are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make. Please also refer to those factors described in "Part I, Item 1A. Risk Factors" of our 2024 Annual Report on Form 10-K for important factors that we believe could cause actual results to differ materially from those in our forward-looking statements.

Overview

We are a commercial-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic compounds to treat diseases with high unmet needs through the inhibition of the complement system, which is an integral component of the immune system. We believe this approach has the potential to effectively control diseases with high unmet need that are driven by excessive complement activation. We currently have two marketed drugs that target C3, the central protein in the complement cascade: SYFOVRE (pegcetacoplan injection), approved by the U.S. Food and Drug Administration, or FDA, in February 2023 for the treatment of geographic atrophy secondary to age-related macular degeneration, or GA; and EMPAVELI (pegcetacoplan), approved by the FDA in May 2021 for the treatment of paroxysmal nocturnal hemoglobinuria, or PNH and in July 2025 for the treatment of C3 glomerulopathy, or C3G, and primary immune complex membranoproliferative glomerulonephritis, or IC-MPGN.

We believe SYFOVRE has the potential to be the standard of care for patients with GA, a disease that affects an estimated 1.5 million people in the United States. While we have exclusive, worldwide commercialization rights for intravitreal pegcetacoplan, we intend to focus our commercialization efforts in the U.S. and explore international expansion in select markets, including Australia, where we received marketing approval in January 2025. We launched SYFOVRE in the United States in March 2023. For the three months ended September 30, 2025 and 2024, we generated $150.9 million and $152.0 million, respectively, in U.S. net product revenue from sales of SYFOVRE. For the nine months ended September 30, 2025 and 2024, we generated $431.7 million and $444.0 million, respectively, in U.S. net product revenue from sales of SYFOVRE. We are also developing a next-generation therapy by combining SYFOVRE treatment with APL-3007, which is a small interfering RNA, or siRNA, aimed at comprehensively blocking complement activity in the retina and the choroid. We initiated a Phase 2 multi-dose trial with this therapy in patients with GA in June 2025.

We believe that EMPAVELI has the potential to be a best-in-class treatment for a range of indications with high unmet needs. We have exclusive U.S. commercialization rights for EMPAVELI. For the three months ended September 30, 2025 and 2024, we generated $26.8 million and $24.6 million, respectively, in U.S. net product revenue from sales of EMPAVELI. For the nine months ended September 30, 2025 and 2024, we generated $67.3 million and $74.7 million, respectively, in U.S. net product revenue from sales of EMPAVELI. In July 2025, the FDA approved EMPAVELI for the treatment of C3G and IC-MPGN, two nephrological conditions, which together affect an estimated 5,000 people in the United States. This approval was based on data from the VALIANT study, which demonstrated positive effects on the three key markers of disease at six months, including a 68% reduction in proteinuria in C3G and IC-MPGN patients compared to placebo (p < 0.0001), the primary endpoint; stabilization of kidney function (nominal p=0.03), as measured by estimated glomerular filtration rate, and a reduction in C3c staining intensity (nominal p<0.0001) in a substantial proportion of patients. Data also demonstrated favorable safety and tolerability results, consistent with pegcetacoplan's established profile. Results were consistent across all subgroups, including disease type, age, and transplant status.

Our collaboration partner, Swedish Orphan Biovitrum AB (Publ), or Sobi, has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan outside of the United States. For the three months ended September 30, 2025 and 2024, we received $5.2 million and $5.0 million, respectively, in royalties from our collaboration partner, Sobi, which has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan outside of the United States. For the nine months ended September 30, 2025 and 2024, we received $12.6 million and $13.9 million, respectively, in royalties from Sobi. On July 1, 2025, we entered into a Royalty Buy-Down Agreement (the "Royalty Agreement") with Sobi under which Sobi paid us an upfront payment of $275.0 million, and

agreed to pay up to an aggregate of $25.0 million upon the European Medicines Agency ("EMA") approval of Aspaveli for C3G and IC-MPGN (collectively, the "purchase price"), and we agreed to reduce Sobi's royalty payment obligations under the Collaboration Agreement by 90%, subject to defined caps tied to Aspaveli's performance, including an initial cap of 1.45x of the purchase price. If a cap is met, Sobi's royalty payment obligations under the Collaboration Agreement will revert to 100%.

We expect to initiate two pivotal clinical trials with EMPAVELI by year-end 2025, one for the treatment of primary focal segmental glomerulosclerosis, or FSGS, and one for delayed graft function, or DGF. FSGS and DGF are both rare, severe nephrology conditions with no approved therapies in which complement overactivation plays a significant role.

Finally, we are developing new product candidates to further advance our pipeline. Through our collaboration with Beam Therapeutics, Inc., or Beam, we have commenced preclinical studies for a treatment targeting the neonatal Fc receptor, or FcRn, which has the potential to be a first-in-class gene editing treatment for future target indications with one-time dosing. We are also developing other programs outside of gene editing leveraging our proprietary in-house capabilities.

To date, we have financed our operations primarily through $2.6 billion in net proceeds from public offerings of our common

stock and pre-funded warrants to purchase common stock, $414.1 million in payments and royalties from Sobi pursuant to our collaboration agreement, $275.0 million from the royalty monetization, $532.5 million under various credit arrangements, including with Sixth Street Lending Partners, or Sixth Street, and SFJ Pharmaceuticals Group, or SFJ, and $98.8 million relating to the unwinding of certain capped call transactions in March 2024, as well as from the proceeds of our operations. To date, we have exchanged $425.4 million and converted $0.7 million of aggregate principal amount of our Convertible Notes (further discussed below) for shares of our common stock.

Excluding the net income generated from the Sobi Royalty Agreement for the three and nine months ended September 30, 2025, we have incurred significant net operating losses since inception, and expect to incur net operating losses in the current year. Our net income was $215.7 million and $81.3 million for the three and nine months ended September 30, 2025, compared to net losses of $57.4 million and $161.5 million for the three and nine months ended September 30, 2024. As of September 30, 2025, we had an accumulated deficit of $3.0 billion.

Our operating results may fluctuate significantly from quarter to quarter and year to year. We anticipate that we will continue to incur significant commercialization expenses related to sales, marketing, medical affairs, manufacturing, distribution and other commercial infrastructure associated with the commercialization of EMPAVELI for the treatment of PNH and other indications and the commercialization of SYFOVRE for the treatment of GA. In addition, we expect to continue to incur these expenses if and as we continue to develop and conduct our ongoing and planned clinical trials of pegcetacoplan and our other product candidates; initiate and continue research and preclinical and clinical development efforts for any future product candidates; seek to identify and develop additional product candidates for complement-dependent diseases; seek regulatory and marketing approvals for our product candidates that successfully complete clinical trials, if any; establish sales, marketing, distribution and other commercial infrastructure to commercialize any additional products for which we may obtain marketing approval; require the manufacture of larger quantities of product candidates for clinical development and, potentially, commercialization; maintain, expand and protect our intellectual property portfolio; hire and retain additional personnel, such as clinical, quality control, regulatory and scientific personnel; add operational, financial and management information systems and personnel, including personnel to support our product development and add equipment and physical infrastructure to support our research and development programs and commercialization.

Financing Agreement and Credit Facility

On May 13, 2024, we entered into a financing agreement, or the Sixth Street Financing Agreement, with certain of our material subsidiaries as guarantors party thereto, the lenders party thereto, or the Lenders, and Sixth Street Lending Partners, as the administrative agent and collateral agent for the Lenders.

The Sixth Street Financing Agreement provides for a senior secured term loan facility of up to $475.0 million, or the Credit Facility, consisting of an initial draw of $375.0 million at closing and a potential additional $100.0 million draw at our option upon satisfaction of a $50.0 million minimum cash requirement and a requirement that our trailing three-month sales of SYFOVRE is at least $180.0 million prior to the $100.0 million draw. The Company did not draw down the additional $100.0 million and the option expired September 30, 2025.

The Credit Facility matures on May 13, 2030 (the "Maturity Date") and bears interest at an annual rate equal to 3-month Term SOFR (subject to 1.00% floor), plus 5.75%. Certain additional commitment and undrawn amount fees are also payable in connection with the Credit Facility.

The net proceeds from the initial draw of the Credit Facility were approximately $358.2 million, net of $16.8 million of issuance costs. We used the majority of the proceeds of the draw at closing to buy out our remaining obligations to SFJ, in the amount of approximately $326.5 million.

The Credit Facility does not provide for scheduled amortization payments during the term. All principal will be due on the Maturity Date. We have the right to prepay loans under the Credit Facility at any time. We are required to repay loans under the Credit Facility with proceeds from certain asset sales, condemnation events and extraordinary receipts, subject, in some cases, to reinvestment rights. Repayments are subject to a prepayment premium. Repayments may be made after the first year of the loan and are subject to a prepayment premium up to 3% depending on timing.

On July 1, 2025, the lenders and Sixth Street Financing consented to the Royalty Agreement with Sobi (the "Consent"), and, in connection with that consent, we agreed to extend by one year from the effective date of the Consent the periods in which certain prepayment premiums would be owed under the Sixth Street Financing Agreement. This effectively extended the period the prepayment premium would be owed from one year after the date of the initial draw, or May 13, 2024, to one year after the effective date of the Consent, or July 1, 2025.

All obligations under the Sixth Street Financing Agreement are secured on a first-priority basis, subject to certain exceptions, by security interests in substantially all of our assets and assets of our material subsidiaries, including our intellectual property, and are guaranteed by our material subsidiaries, including foreign subsidiaries, subject to certain exceptions.

The Sixth Street Financing Agreement contains customary covenants, including, without limitation, a financial covenant to maintain liquidity of at least $50.0 million if our market capitalization is below $3.0 billion, and negative covenants that, subject to certain exceptions, restrict indebtedness, liens, investments (including acquisitions), fundamental changes, asset sales and licensing transactions, dividends, modifications to material agreements, payment of subordinated indebtedness, and other matters customarily restricted in such agreements. Among other permissions, we are permitted, on terms and conditions set forth on the Sixth Street Financing Agreement, to enter into a separate asset-based financing arrangement with a third party in an amount of up to $100.0 million, which amount is increased to $200.0 million upon certain sales or market capitalization thresholds, and to have outstanding convertible unsecured notes in an amount equal to the greater of $400.0 million and 10% of our market capitalization, but not to exceed $600.0 million. We are subject to restrictions on sales and licensing transactions with respect to our core intellectual property, defined to include SYFOVRE, EMPAVELI, and other pegcetacoplan product assets, subject to certain exceptions, including certain transactions related to areas outside the United States and Europe.

The Sixth Street Financing Agreement also contains certain events of default after which loans under the Credit Facility may be due and payable immediately, including payment defaults, material inaccuracy of representations and warranties, covenant defaults, bankruptcy and insolvency proceedings, cross-defaults to certain other agreements, judgments against us and our subsidiaries, and change of control.

Convertible Notes

On September 16, 2019, we completed a private offering of convertible notes, or the 2019 Convertible Notes, with an aggregate principal amount of $220.0 million issued pursuant to an indenture, or the Indenture, with U.S. Bank National Association, as trustee.

The net proceeds from the sale of the 2019 Convertible Notes were approximately $212.9 million after deducting the initial purchasers' discounts and commissions of $6.6 million and offering expenses of $0.5 million. We used $28.4 million of the net proceeds from the sale of the 2019 Convertible Notes to pay the cost of the capped call transactions in September 2019 described below.

On May 12, 2020, we issued convertible notes, or the 2020 Convertible Notes, with an aggregate principal amount of $300.0 million. The net proceeds from the sale of the 2020 Convertible Notes were approximately $322.9 million after deducting the purchasers' discounts and commission of $5.7 million and offering expenses of $0.3 million. We used $43.1 million of the net proceeds from the sale to pay the cost of the additional capped call transactions in May 2020 described below.

The 2019 Convertible Notes and the 2020 Convertible Notes are referred to together as the Convertible Notes. The Convertible Notes are our senior unsecured obligations and bear interest at a rate of 3.5% per year payable semiannually in arrears on March 15 and September 15 of each year, beginning on March 15, 2020. The Convertible Notes will mature on September 15, 2026, unless converted earlier, redeemed or repurchased in accordance with their terms.

The Convertible Notes are convertible into shares of our common stock at an initial conversion rate of 25.3405 shares per $1,000 principal amount of notes (equivalent to an initial conversion price of approximately $39.4625 per share of common stock). The conversion rate is subject to customary anti-dilution adjustments. In addition, following certain events that occur prior to the maturity date or if we deliver a notice of redemption, we will increase the conversion rate for a holder who elects to convert its Convertible Notes in connection with such corporate event or a notice of redemption, as the case may be, in certain circumstances as provided in the Indenture.

Prior to March 15, 2026, the Convertible Notes are convertible only under the following circumstances:

On, or after, March 15, 2026 until the close of business on the second scheduled trading day immediately preceding the maturity date of the Convertible Notes, holders may convert the Convertible Notes at any time regardless of the foregoing circumstances. Upon conversion of the Convertible Notes, we will pay or deliver, as the case may be, cash, shares of our common stock or a combination of cash and shares of common stock, at our election.

As of September 20, 2023, we may redeem for cash all or a portion of the Convertible Notes, at our option, if the last reported sale price of our common stock has been at least 130% of the conversion price then in effect for at least 20 trading days (whether or not consecutive), including the trading day immediately preceding the date on which we provide a notice of redemption, during any 30 consecutive trading day period ending on, and including, the trading day immediately preceding the date on which we provide notice of redemption. The redemption price will be equal to 100% of the principal amount of the Convertible Notes to be redeemed, plus accrued and unpaid interest to, but excluding, the redemption date. If we call any Convertible Notes for redemption, it will constitute a "make-whole fundamental change" with respect to such Convertible Notes, in which case the conversion rate applicable to the conversion of such Notes, if converted in connection with the redemption, will be increased in certain circumstances. We have not called for redemption any of the Convertible Notes as of September 30, 2025.

If we undergo a "fundamental change," as defined in the Indenture, prior to maturity, subject to certain conditions, holders may require us to repurchase for cash all or any portion of their Convertible Notes at a fundamental change repurchase price equal to 100% of the principal amount of the notes to be repurchased, plus any accrued and unpaid interest to, but excluding, the fundamental change repurchase date.

In January 2021, July 2021 and July 2022, the Company entered into separate, privately negotiated exchange agreements to modify the conversion terms with certain holders of its 2019 Convertible Notes and 2020 Convertible Notes. Under the terms of these exchange agreements, in January 2021, July 2021 and July 2022, the holders exchanged approximately $126.1 million of 2019 Convertible Notes, $201.1 million of 2019 Convertible Notes and 2020 Convertible Notes, and $98.1 million of 2020 Convertible Notes, respectively, in aggregate principal amount held by them for an aggregate of 3,906,869 shares, 5,992,217 shares and 3,027,018 shares, respectively, of common stock issued by the Company. The Company accounted for the conversion of the debt as an inducement by expensing the fair value of the shares that were issued in excess of the original terms of the Convertible Notes.

The conditional conversion feature of the Convertible Notes was not triggered as of September 30, 2025 and as of December 31, 2024.

As of September 30, 2025, we held in treasury Convertible Notes in principal amount of $425.4 million which have not been cancelled.

Collaboration Agreement with Sobi

On October 27, 2020, we entered into the Sobi collaboration agreement, concerning the development and commercialization of pegcetacoplan and specified other structurally and functionally similar compstatin analogues or derivatives for use systemically or for local non-ophthalmological administration, collectively referred to as the licensed products. We granted Sobi an exclusive (subject to certain rights retained by us), sublicensable license of certain patent rights and know-how to develop and commercialize licensed products in all countries outside of the United States. Under the Sobi collaboration agreement, Sobi made an upfront payment of $250.0 million in November 2020, and agreed to pay up to an aggregate of $915.0 million upon the achievement of specified one-time regulatory and commercial milestone events, including a $50.0 million milestone payable following the first regulatory and reimbursement approval of systemic pegcetacoplan in any major European country, and to reimburse us for up to $80.0 million in development costs. Since contract inception, we have recognized $65.0 million in contra-research and development expenses and waived the remaining $15.0 million in connection with the decision to discontinue the CAD program.

The European Commission approved systemic Aspaveli (pegcetacoplan) for the treatment of adults with PNH in December 2021. In March 2022, we earned a $50.0 million payment from Sobi related to the first regulatory and reimbursement milestone in Europe, which we received in April 2022. Up until June 30, 2025, we were also entitled to receive tiered, double-digit royalties (ranging from high teens to high twenties) on sales of licensed products outside of the United States, subject to customary deductions and third-party payment obligations, until the latest to occur of: (i) expiration of the last-to-expire of specified licensed patent rights; (ii) expiration of regulatory exclusivity; and (iii) ten (10) years after the first commercial sale of the applicable licensed product, in each case on a licensed product-by-licensed product and country-by-country basis. On July 1, 2025, we and certain of our subsidiaries entered into the Royalty Agreement, in which we agreed to reduce Sobi's royalty payment obligations under the Collaboration Agreement by 90%, effective as of July 1, 2025, subject to defined caps tied to Aspaveli's performance, including an initial cap of 1.45x the amounts paid by Sobi to us under the Royalty Agreement. If a cap is met, Sobi's royalty payment obligations under the Collaboration Agreement will revert to 100%. We remain responsible for our license fee obligations (including royalty obligations) to the University of Pennsylvania.

Financial Operations Overview

Revenue

Our revenues consist of product sales of EMPAVELI and SYFOVRE, and revenues derived from our collaboration agreement with Sobi.

Revenue is recognized when, or as, we satisfy a performance obligation by transferring a promised good or service to a customer. An asset is transferred when, or as, the customer obtains control of that asset. For performance obligations that are satisfied over time, we recognize revenue using an input or output measure of progress that best depicts the satisfaction of the relevant performance obligation.

Product Revenues

Product revenue is derived from our sales of our commercial products, EMPAVELI and SYFOVRE, in the United States.

Licensing and Other Revenue

Licensing and other revenue is derived from our collaboration agreement with Sobi concerning the development and commercialization of pegcetacoplan and specified other compstatin analogues or derivatives for use systemically or for local non-ophthalmic administration.

Cost of Sales

Cost of sales consists primarily of costs associated with the manufacturing of EMPAVELI and SYFOVRE, product supplied to Sobi, and royalties owed to our licensor for such sales and certain period costs.

Research and Development Expenses

Research and development expenses consist primarily of costs incurred for our research activities, including our drug discovery efforts, and the development of our product candidates, which include:

Research and development costs are expensed as incurred. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized. The capitalized amounts are expensed as the

related goods are delivered or the services are performed. We have not provided program costs since inception because historically we have not tracked or recorded our research and development expenses on a program-by-program basis from inception.

The successful development of our product candidates in clinical development is highly uncertain. Accordingly, at this time, we cannot reasonably estimate the nature, timing and costs of the efforts that will be necessary to complete the remainder of the clinical development of these product candidates. We are also unable to predict when, if ever, material net cash inflows will commence from pegcetacoplan in other jurisdictions and indications or any other potential product candidates. This is due to the numerous risks and uncertainties associated with developing therapeutics, including the uncertainties of:

A change in the outcome of any of these variables with respect to the development of any of our product candidates would significantly change the costs and timing associated with the development of that product candidate.

Research and development activities are central to our business model. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect research and development costs to increase for the foreseeable future as our product candidate development programs progress. However, we do not believe that it is possible at this time to accurately project total program-specific expenses through commercialization. There are numerous factors associated with the successful commercialization of any of our product candidates, including future trial design and various regulatory requirements, many of which cannot be determined with accuracy at this time based on our stage of development. Additionally, future commercial and regulatory factors beyond our control will impact our clinical development programs and plans.

Selling, General and Administrative Expenses

Selling, general and administrative expenses consist primarily of costs associated with the commercialization of approved products and general and administrative costs to support operations, including salaries, bonuses, benefits and share-based compensation. Selling expenses include product marketing, sales operations costs, and other costs incurred to support our sales efforts. General and administrative expenses include corporate support functions such as executive management, finance and accounting, business development, legal, human resources, information technology, and associated external costs to support those functions. Other significant costs include costs associated with medical affairs, drug safety and pharmacovigilance, quality and regulatory, costs not otherwise included in research and development expenses, legal fees relating to patent and corporate matters, and fees for accounting and consulting services. Marketing and advertising costs include marketing literature, promotional activities, conferences and seminars, branding and sponsorships.

We anticipate that our selling, general and administrative expenses will increase in the future to support continued commercial activities for our approved products, potential commercialization of our product candidates and costs of operating as a public company.

Critical Accounting Estimates

This discussion and analysis of our financial condition and results of operations is based on our financial statements, which we have prepared in accordance with U.S. generally accepted accounting principles. The preparation of these financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported amounts of expenses during the reported periods. On an ongoing basis, we evaluate our estimates and judgments, including those related to product revenue, inventory and accrued research and development expenses, which we described in our 2024 Annual Report on Form 10-K. We base our estimates on historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.

Our significant accounting policies are described in Note 2 of Part I, Item 1 of this Quarterly Report on Form 10-Q and in Part I, Item 7, "Critical Accounting Estimates" in our 2024 Annual Report on Form 10-K. There have been no changes to our critical accounting policies and estimates since our 2024 Annual Report on Form 10-K.

Results of Operations

Three Months Ended September 30, 2025 and 2024 (in thousands, except percentages)

Product Revenue, Net

Our product revenue, net is derived from EMPAVELI and SYFOVRE sales in the United States. We recognized $177.8 million and $176.6 million of net product revenue for the three months ended September 30, 2025 and 2024, respectively. The net product revenue of $177.8 million for the three months ended September 30, 2025 consists of $26.8 million in net product revenue from sales of EMPAVELI and $150.9 million in net product revenue from sales of SYFOVRE. The net product revenue of $176.6 million for the three months ended September 30, 2024 consists of $24.6 million in net product revenue from sales of EMPAVELI and $152.0 million in net product revenue from sales of SYFOVRE. The decrease in net product revenue for SYFOVRE was primarily driven by increased rebates, partially offset by an increase in volume. The increase in net product revenue for EMPAVELI was primarily driven by an increase in volume.

Licensing and Other Revenue

Licensing and other revenue of $280.8 million for the three months ended September 30, 2025 consisted of $5.2 million in revenue from product supplied to Sobi, $0.6 million in royalty revenue from Sobi and $275.0 million from the Sobi Royalty Agreement. Licensing and other revenue of $20.3 million for the three months ended September 30, 2024 consisted of $15.3 million in revenue from product supplied to Sobi and $5.0 million in royalty revenue from Sobi. The increase in licensing and other revenue was primarily driven by a $275.0 million increase due to the upfront payment from Sobi under the Royalty Agreement, which was partially offset by a $10.1 million decrease in product supplied to Sobi and a decrease in royalty revenue of $4.3 million.

Cost of Sales

Cost of sales was $24.5 million for the three months ended September 30, 2025 and $33.6 million for the three months ended September 30, 2024. The decrease in cost of sales was primarily driven by a $5.9 million decrease due to lower volumes of product supplied to Sobi, a $0.8 million decrease in expenses incurred related to excess, obsolete or scrapped inventory, and a $6.1 million decrease due to costs incurred in connection with cancellable purchase commitments. The decreases were partially offset by a $3.5 million increase due to higher volume from commercial sales and product provided under our patient assistance programs.

In addition, prior to receiving FDA approval for EMPAVELI and SYFOVRE, the costs associated with the manufacturing of EMPAVELI and SYFOVRE inventory were expensed as incurred as research and development expense. This resulted in inventory being sold during the three months ended September 30, 2025 and 2024 for which a portion of the costs had previously been expensed prior to FDA approval. This did not materially impact cost of sales for the three months ended September 30, 2025 and 2024. We

expect this may continue to impact the cost of sales and research and development expense as we continue to sell to customers or use the remaining pre-FDA approved inventory in preclinical or clinical studies. As of September 30, 2025 and December 31, 2024, the remaining pre-FDA approved inventory was $18.1 million and $19.5 million, respectively, which primarily consisted of raw materials and semi-finished goods.

Research and Development Expenses

The following table summarizes our research and development expenses incurred during the three months ended September 30, 2025 and 2024 (in thousands, except percentages):

(1) Includes discontinued clinical activities related to the ALS and CAD programs, amounting to $1.9 million for the three months ended September 30, 2024.

Research and development expenses decreased by $20.4 million to $68.2 million for the three months ended September 30, 2025 from $88.6 million for the three months ended September 30, 2024, a decrease of 23%. The decrease in research and development expenses was primarily attributable to a $9.2 million decrease in program specific external costs, a $10.3 million decrease in non-program specific external costs and a $1.1 million decrease in compensation and related personnel costs, which was partially offset by an increase of $0.2 million in other expenses.

The decrease in our program-specific external costs of $9.2 million was driven by a $4.2 million decrease in C3G & IC-MPGN costs due to lower costs related to the VALIANT study, and a $5.6 million decrease in other development and discovery costs primarily due to a decrease of $1.7 million in our previously announced discontinuation of the CAD program and a decrease of $3.7 million other development and discovery costs. These decreases were partially offset by an increase of $0.6 million in GA costs associated with the product lifecycle development and an increase of $0.5 million in PNH costs due to higher activity related to post trial access programs.

The decrease in compensation and related personnel costs was driven by a decrease of $0.4 million in salaries and benefits and a decrease of $0.7 million in share-based compensation expense.

Selling, General and Administrative Expenses

Selling, general and administrative expenses increased by $20.7 million to $142.7 million for the three months ended September 30, 2025, from $122.0 million for the three months ended September 30, 2024, an increase of 17%. The increase was primarily attributable to an increase of $2.9 million in general commercial activities, an increase of $2.2 million in personnel costs and a $16.3 million increase in general and administrative expenses, including office expenses, travel expenses, insurance expenses, professional and consulting fees, and other general and administrative expenses which was partially offset by a decrease of $0.7 million in factoring fees. The increase in personnel related costs of $2.2 million consisted of a $1.1 million increase in salaries and benefits and an increase of $1.1 million in share-based compensation expense.

Interest Income

Interest income was $4.4 million for the three months ended September 30, 2025, an increase of $1.5 million compared to $2.9 million for the three months ended September 30, 2024. The increase in interest income was primarily attributable to increased investments during the three months ended September 30, 2025.

Interest Expense

Interest expense was $11.3 million for the three months ended September 30, 2025, a decrease of $1.3 million, compared to $12.5 million for the three months ended September 30, 2024. The decrease is primarily due to a decrease in interest incurred under the Credit Facility.

Nine Months Ended September 30, 2025 and 2024 (in thousands, except percentages)

Product Revenue, Net

Our product revenue, net is derived from EMPAVELI and SYFOVRE sales in the United States. We recognized $499.0 million and $518.8 million of net product revenue for the nine months ended September 30, 2025 and 2024, respectively. The net product revenue of $499.0 million for the nine months ended September 30, 2025, consisted of $67.3 million in net product revenue from sales of EMPAVELI and $431.7 million in net product revenue from sales of SYFOVRE. The net product revenue of $518.8 million for the nine months ended September 30, 2024, consisted of $74.7 million in net product revenue from sales of EMPAVELI and $444.0 million in net product revenue from sales of SYFOVRE. The decrease in net product revenue for SYFOVRE was primarily driven by increased rebates, partially offset by an increase in volume. The decrease in net product revenue for EMPAVELI was primarily driven by increased competitive pressure from the availability of oral products.

Licensing and Other Revenue

Licensing and other revenue of $304.8 million during the nine months ended September 30, 2025 consisted of $17.2 million in revenue for product supplied to Sobi and $12.6 million in royalty revenue from Sobi and $275.0 million from the Sobi Royalty Agreement. Licensing and other revenue of $50.1 million during the nine months ended September 30, 2024 consisted of $36.2 million in revenue for product supplied to Sobi and $13.9 million in royalty revenue from Sobi. The increase in licensing and other revenue was primarily driven by an increase of $275.0 million due to the upfront payment from Sobi under the Royalty Agreement, which was partially offset by a decrease in product supplied to Sobi of $19.0 million and a decrease in royalty revenue of $1.3 million.

Cost of Sales

Cost of sales was $72.5 million for the nine months ended September 30, 2025 and $76.9 million for the nine months ended September 30, 2024. The decrease in cost of sales was primarily driven by an $8.1 million decrease due to lower volume of product supplied to Sobi and a decrease of $3.4 million due to costs incurred in connection with cancellable purchase commitments. The decreases were partially offset by an increase of $6.7 million due to higher volumes from commercial sales and product provided under our patient assistance programs and an increase of $0.3 million in expenses incurred related to excess, obsolete or scrapped inventory.

In addition, prior to receiving FDA approval for EMPAVELI and SYFOVRE, the costs associated with the manufacturing of EMPAVELI and SYFOVRE inventory were expensed as incurred as research and development expense. This resulted in inventory being sold during the nine months ended September 30, 2025 and 2024 for which a portion of the costs had previously been expensed prior to FDA approval. This did not materially impact cost of sales for the nine months ended September 30, 2025 and 2024. We expect this may continue to impact the cost of sales and research and development expense as we continue to sell to customers or use the remaining pre-FDA approved inventory in preclinical or clinical studies. As of September 30, 2025 and December 31, 2024, the remaining pre-FDA approved inventory was $18.1 million and $19.5 million, respectively, which primarily consisted of raw materials and semi-finished goods.

Research and Development Expenses

The following table summarizes our research and development expenses incurred during the nine months ended September 30, 2025 and 2024 (in thousands, except percentages):

(1) Includes discontinued clinical activities related to the ALS and CAD programs, amounting to $1.1 million and $20.2 million for the nine months ended September 30, 2025 and 2024, respectively.

Research and development expenses decreased by $29.6 million to $221.6 million for the nine months ended September 30, 2025 from $251.2 million for the nine months ended September 30, 2024, a decrease of 12%. The decrease in research and development expenses was primarily attributable to an $18.7 million decrease in program-specific external costs, a $9.3 million decrease in non-program specific external costs and a decrease of $1.6 million in compensation and related personnel costs.

The decrease in our program-specific external costs of $18.7 million was driven by a $6.2 million decrease in PNH costs due to lower costs incurred relating to clinical activities, a $9.3 million decrease in C3G & IC-MPGN costs due to lower costs related to the VALIANT study, and a $11.8 million decrease in other development and discovery costs primarily due to a decrease of $19.1 million in previously announced discontinuation of the ALS and CAD programs, which were partially offset by an increase of $7.3 million in other development and discovery costs primarily attributable to our efforts on the Beam FcRn collaboration. These decreases were partially offset by an increase of $9.0 million in GA costs associated with the product lifecycle development.

The decrease in compensation and related personnel costs was driven by a decrease of $4.6 million in share-based compensation expense, which was partially offset by $3.0 million in salaries and benefits.

Selling, General and Administrative Expenses

Selling, general and administrative expenses increased by $23.6 million to $403.2 million for the nine months ended September 30, 2025, from $379.6 million for the nine months ended September 30, 2024, an increase of 6%. The increase was primarily attributable to an increase of $5.7 million in professional and consulting fees, an increase of $0.6 million in personnel cost, an increase of $1.8 million in factoring fees and a $15.5 million increase in general and administrative expenses, including office expenses, travel expenses, insurance expenses, and other general and administrative expenses. The increase in personnel related costs of $0.6 million consisted of a $1.4 million increase in salaries and benefits, an increase of $0.7 million in recruitment expenses, and partially offset by a $1.5 million decrease in share-based compensation expense.

Loss on extinguishment of development liability

We paid our remaining obligations under the SFJ agreement in May 2024. We concluded that the development liability was extinguished as of the payoff date. The difference of $1.9 million between the reacquisition price of $326.5 million and the net carrying value of the development liability of $324.6 million was recorded as a loss on the extinguishment of the development liability as of September 30, 2024.

Interest Income

Interest income was $9.6 million for the nine months ended September 30, 2025, an increase of $0.3 million compared to $9.4 million for the nine months ended September 30, 2024. The increase in interest income was primarily attributable to increased investments during the nine months ended September 30, 2025.

Interest Expense

Interest expense was $33.5 million for the nine months ended September 30, 2025, an increase of $4.6 million compared to $28.9 million for the nine months ended September 30, 2024. The increase is primarily due to the interest incurred under the Credit Facility given the Credit Facility commenced May 2024.

Income Tax Expense

Income tax expense was $1.4 million for the nine months ended September 30, 2025, an increase of $0.5 million, compared to $0.9 million for the nine months ended September 30, 2024. The increase primarily pertained to an increase in state taxes.

Liquidity and Capital Resources

Sources of Liquidity

To date, we have financed our operations primarily through approximately $2.6 billion in net proceeds from public and private offerings of our common stock and convertible securities, $414.1 million in payments and royalties from Sobi pursuant to our collaboration agreement, $275.0 million from the royalty monetization, $532.5 million under various credit arrangements, including with Sixth Street and SFJ, and $98.8 million relating to the unwinding of the capped call transactions in March 2024, as well as from the proceeds of our operations.

In May 2024, we entered into the Sixth Street Financing Agreement, which provides for the Credit Facility, consisting of an initial draw of $375.0 million at closing and a potential additional $100.0 million draw at our option upon satisfaction of a $50.0 million minimum cash requirement and a requirement that our trailing three-month sales of SYFOVRE is at least $180.0 million prior to the $100.0 million draw. The Credit Facility matures on May 13, 2030 and bears interest at an annual rate equal to the 3-month Secured Overnight Financing Rate (SOFR) + 5.75% (subject to 1.00% floor). Certain additional commitment and undrawn amount fees are also payable in connection with the Credit Facility. We used the majority of the proceeds of the $375.0 million draw at closing to buy out our remaining obligations owed to SFJ, in the amount of approximately $326.5 million.

We are permitted under the Sixth Street Financing Agreement to enter into a separate asset-based financing arrangement with a third party in an amount of up to $100.0 million, which amount is increased to $200.0 million upon certain sales or market capitalization thresholds, and to have outstanding convertible unsecured notes in an amount equal to the greater of $400.0 million and 10% of our market capitalization, but not to exceed $600.0 million.

In August 2024, the Company entered into an agreement (the "Factoring Agreement") to sell certain accounts receivable to a third-party financial institution at a discount to the face value of the accounts receivable. Under the Factoring Agreement, the maximum amount of outstanding accounts receivables sold at any time is $100.0 million. The accounts receivable sold that remained outstanding as of September 30, 2025 and December 31, 2024 was zero and $86.1 million, respectively.

In November 2023, we entered into a sales agreement, or the sales agreement, with Cowen and Company, LLC, or Cowen, as agent, pursuant to which we may offer and sell shares of our common stock having an aggregate offering from of up to $300.0 million from time to time. Any sales made under the sales agreement will be made at market prices by any method that is deemed to be an "at the market offering" as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933. Any sales under the sales agreement will be made pursuant to our registration statement on Form S-3, which became effective on February 22, 2023. We agreed to pay Cowen compensation of up to 3.0% of the gross proceeds of the sale of shares made under the sales agreement. We did not make any sales under the sales agreement during the nine months ended September 30, 2025.

In February 2023, we issued and sold 4,007,936 shares of our common stock and, in lieu of common stock to investors who so chose, pre-funded warrants to purchase 2,380,956 shares of our common stock in a follow-on offering, including 833,333 shares sold

pursuant to the underwriters' exercise in full of their option to purchase additional shares of common stock. The price to the public of the shares of common stock was $63.00 per share and the price to the public of the pre-funded warrants was $62.9999 per pre-funded warrant. The pre-funded warrants have an exercise price equal to $0.0001 per share and do not expire. The pre-funded warrants were accounted for as equity instruments. We received total net proceeds of $384.4 million, after deducting underwriting discounts and commissions of $18.8 million and offering cost of $0.3 million. As of September 30, 2025, pre-funded warrants to purchase 80,965 shares of our common stock were still outstanding.

In February 2024, we entered into agreements with the capped call counterparties to unwind a portion of the capped call transactions. The unwind agreements applied to the portion of the capped call transactions in a notional amount corresponding to the $426.1 million principal amount of Convertible Notes that we held in treasury as of December 31, 2024 or have been previously converted. The unwind transactions were settled at volume-weighted average price per share of $64.11, which resulted in cash proceeds to us of $98.8 million. As of September 30, 2025, the remaining capped call transactions had a notional amount corresponding to $93.9 million principal amount of Convertible Notes.

Cash Flows

The following table provides information regarding our cash flows for the nine months ended September 30, 2025 and 2024 (in thousands):

Net Cash Provided by/(Used in) Operating Activities

Net cash provided by operating activities was $59.5 million for the nine months ended September 30, 2025 and consisted primarily of a net income of $81.3 million, adjusted for $85.8 million of non-cash items, including share-based compensation expense of $82.8 million, depreciation expense of $1.2 million and amortization of discounts for credit facility of $1.6 million. Further, it included a net increase in operating assets and liabilities of $107.7 million, which was driven by an increase in accounts receivable of $80.6 million, an increase in inventory of $6.6 million, an increase in prepaid assets of $13.2 million, a decrease in other current assets of $1.6 million, an increase in other assets of $4.2 million, a decrease in accrued expenses of $0.2 million, and a decrease of $4.7 million in other liabilities. The change in accounts receivable was primarily driven by a reduction in the derecognition of certain accounts receivable under our Factoring Agreement.

Net cash used in operating activities was $107.2 million for nine months ended September 30, 2024 and consisted primarily of a net loss of $161.5 million, adjusted for $101.0 million of non-cash items, including share-based compensation expense of $87.8 million, depreciation expense of $1.4 million, a loss on the extinguishment of development liability of $1.9 million and accretion of discount to the development liability of $8.9 million. Further, it included a net increase in operating assets and liabilities of $46.7 million, which was driven by an increase in accounts receivable of $72.6 million, an increase in inventory of $20.8 million, a decrease in prepaid assets of $15.2 million, a decrease in other current assets of $10.7 million, a decrease in accounts payable of $0.4 million, an increase in accrued expenses of $12.1 million, an increase in other liabilities of $7.5 million, and an increase in deferred revenue of $1.9 million. The increase in accounts receivable was reduced by the transfer of $56.6 million of accounts receivable during the nine months ended September 30, 2024 to CITIBANK N.A. ("Citi") under the factoring agreement entered into on August 5, 2024.

Net Cash Provided by Financing Activities

Net cash provided by financing activities was $7.6 million during the nine months ended September 30, 2025 and consisted primarily of proceeds from the exercise of stock options and proceeds from the employee stock purchase plan.

Net cash provided by financing activities was $153.2 million during the nine months ended September 30, 2024 and consisted primarily of net proceeds from the initial draw of the Credit Facility of $363.9 million, the settlement of capped call unwind transactions of $98.8 million, $14.0 million of proceeds from the exercise of stock options and $3.2 million proceeds from the issuance of our common stock under the employee stock purchase plan partially offset by repayment of $326.5 million for the development liability.

Funding Requirements

We expect to continue to incur expenses to support our ongoing commercial activities related to product manufacturing, marketing, sales and distribution of EMPAVELI for PNH, C3G and IC-MPGN and SYFOVRE for GA. In addition, we expect to continue to incur expenses as we prioritize the ongoing development of systemic pegcetacoplan and focus our research initiatives on high potential opportunities.

Together with the cash that we anticipate will be generated from sales of EMPAVELI and SYFOVRE, we expect that our current cash and cash equivalents will be sufficient to fund our projected operating expenses and capital expenditure requirements for at least the next 12 months, as well as our anticipated longer-term cash requirements and obligations. Our expectations regarding our short-term and long-term funding requirements are based on assumptions that may prove to be wrong, and we may need additional capital resources to fund our operating plans and capital expenditure requirements.

We are devoting substantial resources to the commercial infrastructure for SYFOVRE for GA. We are also devoting substantial resources to the development of our product candidates. Because of the numerous risks and uncertainties associated with the commercialization of EMPAVELI and SYFOVRE and development of other product candidates, and because the extent to which we may enter into collaborations with third parties for any of these activities is unknown, we are unable to estimate the amounts of increased capital outlays and operating expenses associated with the research, development and commercialization. Our future funding requirements and long-term capital requirements will depend on many factors, including:

If our cash and cash equivalents, and cash generated from sales of EMPAVELI and SYFOVRE are not sufficient to fund our planned expenditures, we will need to finance our cash needs through external sources of funds, which may include equity offerings, debt financings, collaborations, strategic alliances or licensing arrangements. We currently do not have any committed external source of funds.

If we are unable to generate sufficient funds from sales of EMPAVELI and SYFOVRE, or raise additional funds when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.

Contractual Obligations

The disclosure of our contractual obligations and commitments is set forth under the heading "Management's Discussion and Analysis of Financial Condition and Results of Operations-Contractual Obligations" in our 2024 Annual Report on Form 10-K. See Note 11. Commitments and Contingencies in the Notes to Unaudited Condensed Consolidated Financial Statements in Part I, Item I of this Form 10-Q for a discussion of obligations and commitments.