Financial Data and Key Metrics Changes - The net loss for the second quarter of 2025 was $25.4 million or $0.43 per share, compared to a net loss of $33.5 million or $0.58 per share in the first quarter of the same year [9][24] - As of June 30, 2025, the company had $28.7 million in cash and investments, bolstered by a registered direct offering that raised $20.6 million in net proceeds [9][10] - Total operating expenses from continuing operations for the second quarter were $32.4 million, a decrease of $2.6 million from the first quarter [26] Business Line Data and Key Metrics Changes - Research and development expenses in the second quarter were primarily focused on Zaltanopart and narsoplimab [27] - Interest expense for the second quarter was near zero, primarily due to an $8.5 million non-cash remeasurement adjustment related to the DRI Amidrea royalty obligation [27] Market Data and Key Metrics Changes - The global market for paroxysmal nocturnal hemoglobinuria (PNH) is projected to grow at 11% annually, reaching over $10 billion by February 2032 [20] - The complement inhibitor segment is expected to more than double from $2.2 billion today to $4.7 billion over the next seven years [20] Company Strategy and Development Direction - The company is focused on the anticipated approval and launch of narsoplimab, targeting the initial indication of stem cell transplant-associated thrombotic microangiopathy (TATMA) [11][12] - The company aims to leverage its experienced field marketing team and skilled sales force to drive rapid uptake of narsoplimab upon approval [18] - Discussions regarding potential asset acquisition and licensing agreements involving clinical assets are ongoing, with one transaction expected to be a multibillion-dollar deal [10][11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the approval process for narsoplimab, with the FDA indicating that labeling discussions are planned to begin no later than October 2025 [13] - The company is optimistic about the market opportunity for narsoplimab, especially given the safety and efficacy data compared to existing treatments [14][15] Other Important Information - The company has adequate supply for narsoplimab for the first several years post-launch, ensuring no supply challenges [53] - The company is advancing its oncology platform, including IND enabling studies for its Oncotox biologics program targeting acute myeloid leukemia (AML) [23] Q&A Session Summary Question: Comparison of narsoplimab launch to Omidria - Management noted that the market for narsoplimab is significantly more focused than that for Omidria, with fewer salespeople needed due to the limited number of transplant centers [37][38] Question: Comfort level of clinicians regarding narsoplimab - Physicians are eagerly awaiting the approval of narsoplimab, viewing it as a much-needed solution for patients facing lethal complications post-transplant [44] Question: Anticipated launch timeline post-approval - If approved in December, the launch would occur in the first quarter of the following year, with substantial supply already in place [50][51] Question: FDA's request leading to PDUFA delay - The FDA requested additional analyses, which were extensive, leading to the three-month delay in the PDUFA date [58][60] Question: Potential partnerships - There is substantial interest in partnerships across the company's programs, but specific details are limited due to confidentiality requirements [61]

Part I — Financial Information This section presents the company's financial statements, MD&A, market risk disclosures, and controls and procedures [Item 1. Financial Statements](index=6&type=section&id=Item%201.%20Financial%20Statements) A **$58.9 million net loss** for H1 2025, with decreasing assets and shareholders' deficit, prompts a going concern warning [Condensed Consolidated Financial Statements](index=6&type=section&id=Condensed%20Consolidated%20Financial%20Statements) Unaudited financial statements show decreased cash, a **$58.9 million net loss**, and negative operational cash flow Condensed Consolidated Balance Sheets (in thousands) | | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Total current assets** | $69,647 | $134,120 | | **Total assets** | $200,569 | $277,079 | | **Total current liabilities** | $85,503 | $79,526 | | **Total liabilities** | $429,254 | $459,688 | | **Total shareholders' deficit** | ($228,685) | ($182,609) | Condensed Consolidated Statements of Operations (in thousands) | | Three Months Ended June 30, | Six Months Ended June 30, | | :--- | :--- | :--- | | | **2025** | **2024** | **2025** | **2024** | | Total costs and expenses | $32,354 | $59,157 | $67,323 | $98,191 | | Loss from operations | ($32,354) | ($59,157) | ($67,323) | ($98,191) | | Net loss from continuing operations | ($25,889) | ($65,125) | ($63,455) | ($108,975) | | Net income from discontinued operations | $465 | $9,084 | $4,571 | $15,750 | | **Net loss** | **($25,424)** | **($56,041)** | **($58,884)** | **($93,225)** | | Net loss per share | ($0.43) | ($0.97) | ($1.01) | ($1.60) | Condensed Consolidated Statements of Cash Flows (in thousands) | | Six Months Ended June 30, | | :--- | :--- | :--- | | | **2025** | **2024** | | Net cash used in operating activities | ($57,779) | ($87,765) | | Net cash provided by investing activities | $59,838 | $11,225 | | Net cash provided by (used in) financing activities | ($3,555) | $71,555 | | **Net decrease in cash and cash equivalents** | **($1,496)** | **($4,985)** | [Notes to Condensed Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Notes detail liquidity issues, going concern, narsoplimab BLA resubmission, debt restructuring, capital raising, and discontinued operations accounting - The company's need to raise additional capital raises substantial doubt about its ability to continue as a **going concern**; financial statements do not include adjustments related to this uncertainty[49](index=49&type=chunk)[50](index=50&type=chunk) - The Biologics License Application (BLA) for narsoplimab in TA-TMA was resubmitted to the FDA in March 2025, with the PDUFA target action date extended to **December 26, 2025**; a Marketing Authorization Application (MAA) was submitted to the EMA in June 2025, with an opinion expected in mid-2026[28](index=28&type=chunk)[29](index=29&type=chunk) - In May 2025, the company restructured its debt by exchanging **$70.8 million** of its 2026 Notes for new 2029 Notes and converting an additional **$10.0 million** of 2026 Notes into common stock, reducing the aggregate principal of the 2026 Notes from **$97.9 million to $17.1 million**[41](index=41&type=chunk)[43](index=43&type=chunk)[116](index=116&type=chunk) - The company raised **$6.4 million** through its at-the-market (ATM) facility in the first six months of 2025 and an additional **$20.6 million** through a registered direct offering in July 2025[45](index=45&type=chunk)[47](index=47&type=chunk)[149](index=149&type=chunk) - The sale of OMIDRIA is accounted for as a discontinued operation; as of June 30, 2025, the company reported an OMIDRIA contract royalty asset of **$143.0 million** and a related OMIDRIA royalty obligation of **$197.7 million**[60](index=60&type=chunk)[89](index=89&type=chunk)[136](index=136&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=32&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) MD&A details clinical programs, **reduced R&D expenses**, liquidity, debt restructuring, and capital raising, reiterating going concern doubts [Overview](index=32&type=section&id=Overview) Omeros, a clinical-stage biopharmaceutical company, focuses on immunologic diseases with narsoplimab (TA-TMA BLA) and zaltenibart (PNH, Phase 3 paused) - The BLA for narsoplimab in TA-TMA has a PDUFA target action date of **December 26, 2025**, following an extension by the FDA[160](index=160&type=chunk) - Phase 3 clinical development for zaltenibart in PNH has been temporarily paused to prioritize the use of available capital[167](index=167&type=chunk) - The PDE7 inhibitor program (OMS527) for cocaine use disorder is supported by a NIDA grant, with a clinical study targeted for the first part of 2026[168](index=168&type=chunk) [Results of Operations](index=36&type=section&id=Results%20of%20Operations) Financial results show a significant **36% reduction in R&D expenses** for the six-month period, with decreased SG&A and interest expenses Research and Development Expenses (in thousands) | | Six Months Ended June 30, | | :--- | :--- | :--- | | | **2025** | **2024** | | MASP-3 program - OMS906 (zaltenibart) | $12,355 | $13,426 | | MASP-2 program - OMS721 (narsoplimab) | $6,055 | $28,688 | | **Total research and development expenses** | **$45,855** | **$72,119** | - For the six months ended June 30, 2025, R&D expenses decreased by **$26.3 million** compared to the prior year, mainly due to a **$22.6 million reduction** in costs for the narsoplimab program and lower manufacturing expenses for zaltenibart[192](index=192&type=chunk)[193](index=193&type=chunk) - SG&A expenses decreased by **$4.6 million (18%)** for the six months ended June 30, 2025, compared to the prior year, primarily due to capitalizing debt issuance costs and reduced consulting fees[198](index=198&type=chunk) - Total interest expense decreased by **$13.8 million** for the six months ended June 30, 2025, primarily due to a **$13.7 million favorable non-cash remeasurement** on the OMIDRIA royalty obligation and lower interest on the reduced 2026 Notes balance[201](index=201&type=chunk)[204](index=204&type=chunk) [Financial Condition – Liquidity and Capital Resources](index=40&type=section&id=Financial%20Condition%20%E2%80%93%20Liquidity%20and%20Capital%20Resources) The company held **$28.7 million in cash and investments** as of June 30, 2025, facing going concern doubts, while pursuing a potential licensing deal - Cash, cash equivalents, and short-term investments were **$28.7 million** at June 30, 2025, with cash used in operations totaling **$57.8 million** for the first six months of the year[212](index=212&type=chunk) - The company is in discussions for a potential licensing agreement with a multi-billion total transaction value; if closed, the upfront payment is expected to repay its Term Loan and provide sufficient capital for more than 12 months of operations, though no assurance can be given that the transaction will be consummated[216](index=216&type=chunk) - The company's financial condition and need to raise additional capital raise substantial doubt about its ability to continue as a **going concern**[219](index=219&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=43&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) Market risk stems from investment securities and debt instruments, with investment risk managed and debt risk involving fixed/variable-rate loans - Investment risk is considered low as funds are held in high-credit quality, short-term securities like money market funds and U.S. treasury bills[233](index=233&type=chunk) - The company has exposure to interest rate changes through its Term Loan, which bears interest based on the adjusted SOFR[235](index=235&type=chunk) - The 2029 Notes contain an embedded derivative that is marked to fair value each period, exposing the company to gains or losses based on changes in stock price, volatility, and interest rates[234](index=234&type=chunk) [Item 4. Controls and Procedures](index=43&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective as of June 30, 2025, with no material changes to internal control over financial reporting - Disclosure controls and procedures were deemed effective as of June 30, 2025[236](index=236&type=chunk) - There were no material changes in internal control over financial reporting during the quarter[237](index=237&type=chunk) Part II — Other Information This section provides disclosures on legal proceedings, risk factors, equity sales, defaults, mine safety, insider trading, and exhibits [Item 1. Legal Proceedings](index=44&type=section&id=Item%201.%20Legal%20Proceedings) The company was not involved in any material legal proceedings as of the filing date of this Form 10-Q - As of the filing date, the company was not involved in any material legal proceedings[239](index=239&type=chunk) [Item 1A. Risk Factors](index=44&type=section&id=Item%201A.%20Risk%20Factors) Investors are directed to comprehensive risk factors in the 2024 Annual Report on Form 10-K, which could impact stock price - Investors are directed to the Risk Factors section of the company's 2024 Annual Report on Form 10-K for a comprehensive discussion of risks[240](index=240&type=chunk) [Other Items (Items 2, 3, 4, 5, 6)](index=44&type=section&id=Other%20Items%20%28Items%202%2C%203%2C%204%2C%205%2C%206%29) This section addresses standard disclosures, noting several items as not applicable, no new insider trading plans, and listing filed exhibits - Items 2 (Unregistered Sales of Equity Securities), 3 (Defaults Upon Senior Securities), and 4 (Mine Safety Disclosures) are not applicable[241](index=241&type=chunk)[242](index=242&type=chunk)[243](index=243&type=chunk) - No directors or Section 16 reporting officers adopted or terminated a Rule 10b5-1 trading plan during the three months ended June 30, 2025[244](index=244&type=chunk) - Item 6 lists exhibits filed with the 10-Q, including debt agreements and officer certifications[245](index=245&type=chunk)

[Key Highlights and Financial Summary](index=1&type=section&id=Key%20Highlights%20and%20Financial%20Summary) Omeros significantly reduced its net loss in Q2 2025, primarily due to lower manufacturing expenses, while strengthening its balance sheet through a registered direct offering and debt restructuring. Financial Summary | Metric | Q2 2025 | Q2 2024 | H1 2025 | H1 2024 | | :--- | :--- | :--- | :--- | :--- | | Net Loss | $25.4M | $56.0M | $58.9M | $93.2M | | Net Loss Per Share | $0.43 | $0.97 | $1.01 | $1.60 | - The FDA accepted the resubmitted Biologics License Application (BLA) for narsoplimab in TA-TMA, extending the PDUFA target action date to **December 26, 2025**, following an information request[2](index=2&type=chunk) - The company improved its balance sheet by exchanging **$70.8 million** of 2026 convertible notes for new 2029 notes and converting an additional **$10.0 million** into common stock, reducing the 2026 notes principal from **$97.9 million** to **$17.1 million**[2](index=2&type=chunk) - Omeros is in advanced discussions for a potential asset acquisition or licensing agreement with a total transaction value potentially in the **multi-billions**, exclusive of royalties, with an expected upfront payment to repay all term debt and fund over 12 months of operations[2](index=2&type=chunk)[3](index=3&type=chunk) [CEO Statement](index=2&type=section&id=CEO%20Statement) CEO Gregory A. Demopulos highlighted significant balance sheet improvements, including over $100 million in debt reduction and new capital, alongside pipeline advancements for narsoplimab, OMS527, and OncotoX-AML. - The company significantly improved its balance sheet, reducing near-term debt by over **$100 million** and adding new capital through an equity financing with Polar Asset Management[4](index=4&type=chunk) - Key pipeline programs are advancing: NIDA continues to fund the PDE7 inhibitor program (OMS527) for cocaine use disorder, and the OncotoX-AML program is on track to enter the clinic in 18-24 months with promising data[4](index=4&type=chunk) - Other pipeline assets, including the MASP-3 inhibitor zaltenibart and the long-acting MASP-2 inhibitor OMS1029, are paused but ready to initiate Phase 3 and Phase 2 trials, respectively, as the company prioritizes narsoplimab[4](index=4&type=chunk) [Pipeline and Recent Developments](index=3&type=section&id=Pipeline%20and%20Recent%20Developments) Omeros is actively preparing for the commercial launch of narsoplimab, advancing the OMS527 program for cocaine addiction towards a clinical study in H1 2026, and progressing the OncotoX-AML oncology program towards clinical trials within 18-24 months. [Narsoplimab (MASP-2 Inhibitor)](index=3&type=section&id=Narsoplimab%20%28MASP-2%20Inhibitor%29) The company is preparing for the commercial launch of narsoplimab for TA-TMA, focusing on high-priority transplant centers and engaging with payers, supported by recent publications on its safety and survival benefits. - Commercial launch preparations are underway, including a phased onboarding of experienced sales professionals to target the highest-volume transplant centers first[5](index=5&type=chunk) - Pre-approval discussions with hospital formulary decision-makers and payers have yielded encouraging feedback, with stakeholders recognizing narsoplimab's strong clinical profile[5](index=5&type=chunk) - A recent study highlighted that the off-label C5 inhibitor eculizumab was associated with an **8.5-fold higher rate of bacteremia infections** and a **sixfold higher one-year infection-related mortality** in pediatric TA-TMA patients[5](index=5&type=chunk) [OMS527 (PDE7 Inhibitor Program)](index=3&type=section&id=OMS527%20%28PDE7%20Inhibitor%20Program%29) The OMS527 program, targeting cocaine use disorder, is supported by a NIDA grant, has completed preclinical interaction studies without safety issues, and is preparing for a clinical in-patient study targeted for the first half of 2026. - The program is supported by a **$6.24 million** grant from the National Institute on Drug Abuse (NIDA) to develop OMS527 for cocaine use disorder[5](index=5&type=chunk) - Preclinical cocaine interaction and toxicology studies, designed by NIDA, were successfully completed with no safety findings, supporting the planned human study[5](index=5&type=chunk) - An in-patient clinical study in cocaine users is targeted for the first part of 2026, pending submission of additional preclinical information requested by the FDA[5](index=5&type=chunk) [Oncology Platform (OncotoX-AML)](index=3&type=section&id=Oncology%20Platform%20%28OncotoX-AML%29) The OncotoX-AML program is advancing through IND-enabling work, with a projected timeline to enter clinical trials in 18-24 months, supported by strong preclinical data and a new clinical steering committee. - The OncotoX-AML therapeutic is estimated to enter the clinic in 18-24 months, with IND-enabling work advancing, albeit at a limited scope to preserve capital[5](index=5&type=chunk) - In preclinical models, the OncotoX-AML therapeutic has consistently demonstrated superior efficacy to current AML standard of care treatments and shows broad application across various genetic mutations[5](index=5&type=chunk) - A newly established Oncology Clinical Steering Committee, composed of leaders from premier U.S. cancer centers, will help guide the program's development[5](index=5&type=chunk) [Q2 2025 Financial Results](index=4&type=section&id=Q2%202025%20Financial%20Results) Omeros reported a Q2 2025 net loss of $25.4 million, a significant improvement from Q2 2024, driven by a $26.8 million decrease in operating expenses, ending the quarter with $28.7 million in cash, subsequently bolstered by over $22 million in financing. [Consolidated Statements of Operations](index=4&type=section&id=Consolidated%20Statements%20of%20Operations) Omeros reported a Q2 2025 net loss of $25.4 million, a significant improvement from the $56.0 million loss in Q2 2024, driven by a $26.8 million decrease in total operating expenses, mainly due to lower narsoplimab manufacturing costs. Income Statement (in thousands) | Income Statement (in thousands) | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | Total costs and expenses | $32,354 | $59,157 | | Loss from operations | ($32,354) | ($59,157) | | Net loss | ($25,424) | ($56,041) | | Net loss per share | ($0.43) | ($0.97) | - The **$26.8 million** decrease in quarterly operating expenses was primarily due to **$17.6 million** in narsoplimab manufacturing costs incurred in Q2 2024 and a temporary pause of certain programs to preserve capital[10](index=10&type=chunk) - OMIDRIA royalties earned in Q2 2025 were **$8.6 million**, down from **$10.9 million** in Q2 2024[9](index=9&type=chunk) [Consolidated Balance Sheet](index=8&type=section&id=Consolidated%20Balance%20Sheet) As of June 30, 2025, Omeros had total assets of $200.6 million and a total shareholders' deficit of $228.7 million, with current assets decreasing due to reduced cash and short-term investments, and a restructured debt profile. Balance Sheet (in thousands) | Balance Sheet (in thousands) | June 30, 2025 | Dec 31, 2024 | | :--- | :--- | :--- | | Cash and short-term investments | $28,744 | $90,132 | | Total current assets | $69,647 | $134,120 | | Total assets | $200,569 | $277,079 | | Total liabilities | $429,254 | $459,688 | | Total shareholders' deficit | ($228,685) | ($182,609) | [Liquidity and Capital Resources](index=4&type=section&id=Liquidity%20and%20Capital%20Resources) The company held $28.7 million in cash and short-term investments at the end of Q2 2025, subsequently raising an additional $20.6 million net through a registered direct offering and $2.1 million gross via its ATM facility, while maintaining a minimum cash balance of $25.0 million. - As of June 30, 2025, the company had **$28.7 million** in cash and short-term investments[7](index=7&type=chunk) - Subsequent to quarter-end, the company raised **$20.6 million** in net cash proceeds from a registered direct offering and **$2.1 million** in gross proceeds from its ATM facility[8](index=8&type=chunk) - A covenant in the company's credit agreement requires maintaining a minimum of **$25.0 million** in unrestricted cash, cash equivalents, and short-term investments[7](index=7&type=chunk) [Corporate Information](index=4&type=section&id=Corporate%20Information) This section provides details about the company's conference call, a corporate overview of its focus on immunologic disorders and its drug pipeline, and the standard forward-looking statements disclaimer. - Omeros is a biopharmaceutical company focused on discovering and developing therapeutics for immunologic disorders, cancers, and addictive disorders[18](index=18&type=chunk)[19](index=19&type=chunk) - The company's lead product candidate, narsoplimab, targets the lectin pathway of complement and is under review by the FDA and EMA for TA-TMA[19](index=19&type=chunk) - The press release contains forward-looking statements subject to a 'safe harbor' provision, and actual results could differ materially from expectations due to various risks outlined in SEC filings[20](index=20&type=chunk)

Group 1 - Omeros (OMER) has experienced a significant decline of 54.8% over the past four weeks, but it is now in oversold territory, indicating a potential trend reversal [1] - The Relative Strength Index (RSI) for OMER is currently at 24.55, suggesting that the heavy selling pressure may be exhausting, which could lead to a price rebound [5] - There is strong consensus among Wall Street analysts that OMER will report better earnings than previously predicted, with a 6.9% increase in the consensus EPS estimate over the last 30 days [7] Group 2 - OMER holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate revisions and EPS surprises, indicating a strong potential for a turnaround [8]

Group 1 - The article discusses the author's first treatment of Omeros in 2025, following extensive coverage in the previous year [1] - The author emphasizes the learning process involved in investing, viewing failures as tuition and successes as lessons learned, particularly in the healthcare sector [2] - The author has a beneficial long position in Omeros shares, indicating a personal investment interest [3] Group 2 - The author may engage in buying or selling shares of any mentioned company within the next 72 hours, suggesting potential volatility or changes in position [4] - Seeking Alpha clarifies that past performance does not guarantee future results and that no specific investment advice is being provided [5]

Group 1 - Omeros Corporation held its Q1 2025 earnings conference call on May 15, 2025, at 4:30 PM ET [1] - The call included participants from the company such as the Chairman & CEO, Chief Accounting Officer, and Chief Commercial Officer [1][6] - The call was recorded and a replay will be available on the company's website for one week [1] Group 2 - Jennifer Williams, representing Investor Relations, opened the call and thanked participants for joining [2][3] - Forward-looking statements were mentioned, indicating that they are based on management's beliefs and expectations and are subject to change [4] - The call was turned over to Dr. Gregory Demopulos, the Chairman and CEO, who was joined by other key executives [5][6]

Financial Data and Key Metrics Changes - The company reported a net loss of $33.5 million or $0.58 per share for the first quarter of 2025, compared to a net loss of $31.4 million or $0.54 per share in the fourth quarter of the previous year [5][18] - As of March 31, 2025, the company had $52.4 million in cash and investments on hand [18] - The company entered into an exchange agreement to convert approximately $71 million of 2026 convertible notes into new 9.5% convertible senior notes due in 2029, significantly improving financial flexibility [5][19] Business Line Data and Key Metrics Changes - Research and development expenses in the first quarter were primarily focused on narsoplimab and zaltanibart [20] - OMIDRIA royalties for the first quarter totaled $6.7 million based on net sales of $22.3 million, a decrease from $10.1 million in royalties on net sales of $33.6 million in the previous quarter [22][23] Market Data and Key Metrics Changes - The market opportunity for narsoplimab is estimated at nearly $1 billion annually, with expectations for it to become a cornerstone asset for transplant experts [11] - The global market for paroxysmal nocturnal hemoglobinuria (PNH) is projected to grow to over $10 billion by 2032, with significant unmet needs in the complement inhibitor market [12] Company Strategy and Development Direction - The company is focused on the anticipated approval and launch of narsoplimab for the treatment of TATMA, with a target FDA action date of September 25 [8] - The company is also developing zaltanibart for PNH, with ongoing clinical trials and a temporary pause on the phase three program due to capital considerations [12][14] Management's Comments on Operating Environment and Future Outlook - Management emphasized the importance of managing costs and maintaining liquidity while advancing key initiatives [7] - The company is actively pursuing partnerships for non-dilutive funding to support operations and the launch of narsoplimab [6] Other Important Information - The FDA has accepted the resubmitted BLA for narsoplimab, and the company is preparing for a successful launch [8] - The company is suspending its expanded access program for narsoplimab to focus resources on the successful launch [9][10] Q&A Session Summary Question: Can you provide details on launch plans and patient access? - Management expressed confidence in the launch preparations, highlighting engagement with key transplant centers and payers [29][31][34] Question: Can you detail the patient demographics and associated costs? - Management explained that TATMA is a complication of stem cell transplants that can arise unpredictably, leading to significant healthcare costs [41][42] - The economic value of narsoplimab was discussed, emphasizing its potential to reduce costs associated with untreated patients [44][45]

Financial Data and Key Metrics Changes - The company reported a net loss of $33.5 million or $0.58 per share for the first quarter of 2025, compared to a net loss of $31.4 million or $0.54 per share in the fourth quarter of the previous year [5][18] - As of March 31, 2025, the company had $52.5 million in cash and investments on hand [5][18] - The company has reduced its total debt by $10 million and lowered near-term repayment obligations from approximately $118 million to $17 million [6][19] Business Line Data and Key Metrics Changes - Research and development expenses in the first quarter were heavily focused on narsoplimab and zaltanibart [20] - OMIDRIA royalties for the first quarter totaled $6.7 million based on net sales of $22.3 million, a decrease from $10.1 million in royalties on net sales of $33.6 million in the previous quarter [22][23] Market Data and Key Metrics Changes - The market opportunity for narsoplimab is estimated at nearly $1 billion annually, with expectations for it to become a cornerstone asset for transplant experts [11] - The global market for paroxysmal nocturnal hemoglobinuria (PNH) is projected to grow about 11% annually to over $10 billion by 2032 [12] Company Strategy and Development Direction - The company is focused on the anticipated approval and launch of narsoplimab for the treatment of TATMA, with a target FDA action date of September 25 [8] - The company is also working on a marketing authorization application for narsoplimab in Europe, targeting completion later this quarter [9] - Development spending on other programs, including the long-acting MASP-two inhibitor OMS1029, remains limited as the company prioritizes core development initiatives [12][16] Management's Comments on Operating Environment and Future Outlook - Management emphasized the importance of maintaining financial flexibility and managing costs while advancing key initiatives [7] - The company is actively pursuing partnerships for non-dilutive funding to support operations [6] - Management expressed confidence in the successful launch of narsoplimab, highlighting the compelling data and the unique market position of the product [36] Other Important Information - The company has suspended its expanded access program for narsoplimab to prioritize resources for the anticipated launch [9][10] - The company is committed to supporting patients currently treated under the EAP despite the program's discontinuation [10] Q&A Session Summary Question: Can you provide details on launch plans and patient access? - Management indicated they are well-prepared for the launch, focusing on key transplant centers and engaging with payers for product information exchanges [31][34] Question: Can you elaborate on the patients affected by TATMA and the associated costs? - Management explained that TATMA is a complication of stem cell transplants that can arise unpredictably, leading to significant healthcare costs due to the severity of the condition [42][44]

Financial Data and Key Metrics Changes - The company reported a net loss of $33.5 million or $0.58 per share for the first quarter of 2025, compared to a net loss of $31.4 million or $0.54 per share in the fourth quarter of the previous year [5][19] - As of March 31, 2025, the company had $52.5 million in cash and investments on hand [5][19] - The company has reduced its total outstanding debt by $10 million and lowered its near-term repayment obligations from approximately $118 million to $17 million [6][20] Business Line Data and Key Metrics Changes - Research and development expenses in the first quarter were heavily focused on narsoplimab and zaltanibart [21] - OMIDRIA royalties for the first quarter totaled $6.7 million based on net sales of $22.3 million, a decrease from $10.1 million in royalties on net sales of $33.6 million in the previous quarter [23][24] Market Data and Key Metrics Changes - The market opportunity for narsoplimab is estimated at nearly $1 billion annually, with expectations for it to become a cornerstone asset for transplant experts [12] - The global market for paroxysmal nocturnal hemoglobinuria (PNH) is projected to grow about 11% annually to over $10 billion by 2032 [13] Company Strategy and Development Direction - The company is focused on the successful launch of narsoplimab for the treatment of TATMA, following the anticipated FDA approval [8][9] - The company is also developing zaltanibart, targeting PNH, while managing costs and pausing certain programs to prioritize resources [14][16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming launch of narsoplimab, highlighting the preparation and engagement with key transplant centers and payers [35][36] - The company is committed to managing cash and liquidity effectively to support operations and capitalize on growth opportunities [7][49] Other Important Information - The FDA has accepted the resubmitted BLA for narsoplimab, with a target action date of September 25 [9] - The company is also working on a marketing authorization application for narsoplimab in Europe, targeting submission later this quarter [10] Q&A Session Summary Question: Can you provide details on launch plans and patient access? - Management indicated that the commercial team is well-prepared for the launch, focusing on key transplant centers and engaging with payers for product information exchanges [30][31][35] Question: Can you elaborate on the patients affected by TATMA and associated costs? - Management explained that TATMA is a complication of stem cell transplants that can arise unpredictably, leading to significant healthcare costs due to the severity of the condition [38][44] - The economic value of narsoplimab is highlighted by its potential to reduce costs associated with untreated patients, emphasizing outpatient treatment benefits [46][47]

[Part I — Financial Information](index=6&type=section&id=Part%20I%20%E2%80%94%20Financial%20Information) [Financial Statements (unaudited)](index=6&type=section&id=Item%201.%20Financial%20Statements%20(unaudited)) The company reported a reduced net loss for Q1 2025 but faces a going concern warning due to recurring losses and upcoming debt maturities Condensed Consolidated Statements of Operations (Q1 2025 vs Q1 2024) | Metric | Q1 2025 (In thousands) | Q1 2024 (In thousands) | | :--- | :--- | :--- | | **Total costs and expenses** | $34,969 | $39,034 | | Research and development | $23,846 | $26,770 | | Selling, general and administrative | $11,123 | $12,264 | | **Loss from operations** | $(34,969) | $(39,034) | | Interest expense | $(3,654) | $(8,231) | | Interest and other income | $1,058 | $3,415 | | **Net loss from continuing operations** | $(37,565) | $(43,850) | | Net income from discontinued operations | $4,105 | $6,666 | | **Net loss** | **$(33,460)** | **$(37,184)** | | Net loss per share (basic and diluted) | $(0.58) | $(0.63) | Condensed Consolidated Balance Sheet Highlights (As of March 31, 2025) | Account | March 31, 2025 (In thousands) | December 31, 2024 (In thousands) | | :--- | :--- | :--- | | Cash and cash equivalents | $4,261 | $3,400 | | Short-term investments | $48,150 | $86,732 | | **Total current assets** | $96,181 | $134,120 | | **Total assets** | $235,161 | $277,079 | | **Total current liabilities** | $83,222 | $79,526 | | **Total liabilities** | $448,714 | $459,688 | | **Total shareholders' deficit** | $(213,553) | $(182,609) | Condensed Consolidated Statements of Cash Flows (Q1 2025 vs Q1 2024) | Activity | Q1 2025 (In thousands) | Q1 2024 (In thousands) | | :--- | :--- | :--- | | Net cash used in operating activities | $(35,841) | $(41,780) | | Net cash provided by (used in) investing activities | $38,541 | $(61,925) | | Net cash (used in) provided by financing activities | $(1,839) | $98,431 | | **Net increase (decrease) in cash** | $861 | $(5,274) | - The company's recurring losses and cash balance, combined with the 2026 Notes maturity, raise **substantial doubt about its ability to continue as a going concern**[44](index=44&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=27&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses clinical pipeline updates, reduced operating expenses, and significant liquidity challenges including a going concern warning - The BLA for narsoplimab in TA-TMA was resubmitted to the FDA and has been assigned a **PDUFA target action date of September 25, 2025**[29](index=29&type=chunk)[132](index=132&type=chunk) - The company has **temporarily paused its Phase 3 clinical development program for zaltenibart** in PNH to prioritize available capital[32](index=32&type=chunk)[137](index=137&type=chunk) - In May 2025, the company restructured its debt, reducing the 2026 Notes principal from $97.9 million to **$27.1 million** and alleviating a potential $20.0 million prepayment on its Term Loan[157](index=157&type=chunk)[158](index=158&type=chunk) - The company's recurring losses and cash position raise **substantial doubt about its ability to continue as a going concern**, necessitating the need to raise additional capital[179](index=179&type=chunk)[180](index=180&type=chunk) [Results of Operations](index=32&type=section&id=Results%20of%20Operations) Total costs and expenses decreased year-over-year, driven by lower R&D, SG&A, and interest expenses Research and Development Expenses (Q1 2025 vs Q1 2024) | Category (In thousands) | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | MASP-3 program - OMS906 (zaltenibart) | $7,030 | $5,253 | | MASP-2 program - OMS721 (narsoplimab) | $2,631 | $6,685 | | MASP-2 program - OMS1029 and other | $364 | $1,126 | | Preclinical research and development | $1,383 | $1,580 | | Internal overhead and other expenses | $11,402 | $11,112 | | Stock-based compensation expenses | $1,036 | $1,014 | | **Total R&D Expenses** | **$23,846** | **$26,770** | - R&D expenses are expected to be lower in Q2 2025 compared to Q1 2025 due to the pause in two Phase 3 clinical trials for zaltenibart and other program reductions[163](index=163&type=chunk) Selling, General and Administrative Expenses (Q1 2025 vs Q1 2024) | Category (In thousands) | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | SG&A (excluding stock-based comp) | $9,706 | $10,620 | | Stock-based compensation | $1,417 | $1,644 | | **Total SG&A Expenses** | **$11,123** | **$12,264** | - Interest expense decreased by $4.6 million YoY, mainly due to a favorable non-cash remeasurement adjustment on the OMIDRIA royalty obligation and lower interest on repurchased 2026 Notes[171](index=171&type=chunk) [Financial Condition – Liquidity and Capital Resources](index=36&type=section&id=Financial%20Condition%20%E2%80%93%20Liquidity%20and%20Capital%20Resources) The company holds $52.4 million in cash and investments but faces substantial going concern doubt, exploring capital raising options - As of March 31, 2025, the company had **$52.4 million in cash, cash equivalents, and short-term investments**[178](index=178&type=chunk) - A credit agreement covenant requires the company to maintain **$25.0 million of unrestricted cash**, cash equivalents, and short-term investments at all times[178](index=178&type=chunk) - The company has an at-the-market (ATM) equity offering facility for up to $150.0 million and raised **$3.5 million in net proceeds** after quarter-end[182](index=182&type=chunk) - The company has taken steps to manage expenses by **delaying clinical trials and reducing selected R&D efforts**, with further reductions possible if necessary[183](index=183&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=38&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's market risk is primarily limited to its high-credit-quality, short-term investment securities portfolio - The company's primary market risk exposure is from its investment securities, which consist of **high-credit-quality securities** like money market funds[195](index=195&type=chunk) - Due to the short-term maturities of its investments, the company does not believe an increase in market rates would materially impact the realized value of its investment portfolio[195](index=195&type=chunk) [Controls and Procedures](index=38&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of the quarter-end with no material changes to internal controls - As of March 31, 2025, the principal executive and financial officers concluded that the company's **disclosure controls and procedures were effective** at the reasonable assurance level[196](index=196&type=chunk) - **No material changes** in internal control over financial reporting occurred during the quarter that are reasonably likely to materially affect these controls[197](index=197&type=chunk) [Part II — Other Information](index=39&type=section&id=Part%20II%20%E2%80%94%20Other%20Information) [Legal Proceedings](index=39&type=section&id=Item%201.%20Legal%20Proceedings) The company was not involved in any material legal proceedings as of the filing date - The company was **not involved in any material legal proceedings** as of the date of the 10-Q filing[199](index=199&type=chunk) [Risk Factors](index=39&type=section&id=Item%201A.Risk%20Factors) Investors are referred to the company's 2024 Form 10-K for a detailed discussion of significant business risks and uncertainties - The report refers to the risk factors described in the company's **Annual Report on Form 10-K for the year ended December 31, 2024**, for a comprehensive understanding of potential risks[200](index=200&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=39&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This item is not applicable for the reporting period - Not applicable[201](index=201&type=chunk) [Other Information](index=39&type=section&id=Item%205.%20Other%20Information) No directors or Section 16 officers adopted or terminated Rule 10b5-1 trading plans during the first quarter of 2025 - **No directors or Section 16 reporting officers adopted or terminated any Rule 10b5-1 trading plans** during the three months ended March 31, 2025[204](index=204&type=chunk) [Exhibits](index=40&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the report, including required officer certifications and Inline XBRL data - The report includes certifications from the Principal Executive Officer and Principal Financial Officer as required by the **Sarbanes-Oxley Act of 2002**[205](index=205&type=chunk)