Summary of Omeros Corporation Conference Call Company Overview - **Company**: Omeros Corporation - **Product**: Yarcomlia (narsoplimab) - **Industry**: Biotechnology, specifically focused on treatments for hematopoietic stem cell transplant-associated thrombotic microangiopathy (TATMA) Key Points FDA Approval and Product Launch - Yarcomlia received FDA approval on December 23, 2025, marking it as the first and only therapy approved for TATMA, a life-threatening complication of stem cell transplantation [3][4] - The approval is a significant milestone for Omeros, being their second FDA-approved product after Omidria [4] - The commercial launch of Yarcomlia began on January 2, 2026, with a fully trained commercial team reaching out to U.S. transplant centers [9][10] Clinical Data and Efficacy - Approval was based on a pivotal trial involving 28 patients and an expanded access program with 221 patients [5] - Yarcomlia demonstrated a 61% complete response rate in the pivotal study and a 68% rate in the expanded access program [6] - The 100-day survival rate post-TMA diagnosis was 73% in the pivotal study and 74% in the expanded access program [6] - In high-risk patients who had failed other treatments, Yarcomlia achieved a 50% one-year survival rate compared to historical rates of less than 20% [8] Safety Profile - Yarcomlia has a differentiated safety profile with no box warning, no risk evaluation and mitigation strategy (REMS), and no vaccination requirement, unlike other off-label therapies [5][8] - Historical use of C5 inhibitors, such as eculizumab, has been associated with significantly higher risks of infection and mortality [9][36] Market Opportunity and Commercial Strategy - Approximately 11,000 allogeneic transplants are performed annually in the U.S., with TATMA developing in up to 56% of these cases [10][48] - Omeros plans to prioritize the top 80 transplant centers that account for about 80% of procedures [10] - The initial price for Yarcomlia is set at approximately $36,000 per vial, with median utilization expected to be 8 to 10 vials per treatment course [12] - A national ICD-10 diagnostic code for TATMA and specific CPT codes for Yarcomlia have been established to facilitate reimbursement [12][38] Reimbursement and Financial Support - Omeros has applied for a new technology add-on payment (NTAP) to assist with costs for Medicare patients, expected to be effective in October 2026 [13][25] - The company has a robust reimbursement strategy in place, including personalized assistance programs for providers [14] Future Plans and Pipeline - Omeros is evaluating partnerships for the commercialization of Yarcomlia outside the U.S. and plans to expand its indications [15] - The company aims to advance its long-acting MASP2 inhibitor, OMS 1029, and its MASP2 small molecule program [15] - The focus is on making Yarcomlia the standard of care for TATMA and ensuring reliable supply and access [43][54] Expert Insights - Experts on the call emphasized the importance of early intervention with Yarcomlia, which could lead to better patient outcomes compared to historical treatment approaches [17][19] - The shift in treatment paradigms is expected to improve overall management of TATMA patients, allowing for continued use of immunosuppressive therapies [19][22] Conclusion - The approval and launch of Yarcomlia represent a significant advancement in the treatment of TATMA, with a clear objective to establish it as the standard of care [43][54] - Omeros is committed to executing its strategy effectively to ensure patient access and improve clinical outcomes for those affected by TATMA [54]

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]