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]