Swedish Orphan Biovitrum (OTCPK:SWOB.Y) 2026 Capital Markets Day February 18, 2026 07:00 AM ET Company ParticipantsDuane H. Barnes - Head of North AmericaEvangelos Giamarellos-Bourboulis - Chair of European Sepsis AllianceGerard Tobin - Head of Investor RelationsGuido Oelkers - CEOHenrik Stenqvist - CFOKirsty Ross-Stewart - VP of Pharmaceuticals Equity ResearchKlaus Parhofer - Professor of Metabolism and EndocrinologyLydia Abad-Franch - Head of R&D and Chief Medical OfficerMattias Häggblom - Co-Head of Equi ...

Okay, so let me bring you to a free clinic event. A 12-year-old boy lights up as he recalls his latest swimming practice. His mother, meanwhile, clutches a crumbled constant form.Worry edge on her face. Across the room, a 28-year-old injection specialist demonstrate his injection skill while the doctor sits next to him. Seems like he just bit into a lemon.Both have hemophilia, a rare disease that affects around 217,000 people worldwide, where blood doesn't clot properly, which leads to excessive bleeding. Y ...

Company Overview - Mereo is a rare disease company focused on developing treatments for conditions such as osteogenesis imperfecta (OI) [1][2] - The company has three clinical programs, with two of them being in late-stage development [2] Patient Impact - The presentation highlighted the personal stories of patients like Martin, who has Type 3 OI and has been on bisphosphonate treatment since he was six months old, experiencing hundreds of fractures [2] - The impact of OI on patients' lives is significant, but individuals like Martin are managing to pursue their education and careers despite the challenges [2]

Core Insights - ANI Pharmaceuticals is transforming into a leading rare disease company, focusing on high growth and profitability [2] - The company projects over $1 billion in revenue by 2026, representing a 26% year-on-year growth compared to 2025, with a significant 39% growth anticipated for the year prior [3] - The rare disease segment is expected to account for approximately 60% of total revenues, driven by the lead asset, purified Cortrophin Gel, which offers substantial multiyear growth opportunities [3] Financial Performance - The generics business is generating strong cash flows due to superior R&D capabilities and operational execution, alongside U.S. manufacturing [3] - The virtuous cycle of growth is created by EBITDA and cash flows from both generics and brands, enabling further investment in the rare disease sector [3]

Core Viewpoint - Travere Therapeutics emphasizes its commitment to rare diseases, driven by personal connections among its team members, including the CEO, who is a rare cancer survivor [3]. Group 1: Company Overview - Travere Therapeutics is represented by CEO Eric Dube at the 44th Annual JPMorgan Healthcare Conference, highlighting the company's focus on rare diseases [1][2]. - The company adopts a patient-inspired approach in its operations, reflecting the personal experiences of its employees with rare diseases [3]. Group 2: Performance and Priorities - Travere announced updates regarding its performance expectations for 2025 and outlined three key priorities for the company [4].

Overview of SKID Treatment - The report focuses on a clinical trial for Severe Combined Immunodeficiency (SKID), also known as "bubble boy disease," and its successful treatment [2][3] - Untreated SKID has a life expectancy of approximately 2 years [4] - A clinical trial at UCLA, led by Dr Donald Con, offered gene therapy and bone marrow transplant as potential treatments [5][6] Treatment and Success Rate - The experimental treatment involves extracting stem cells from the patient's bone marrow, adding a normal ADA gene, and reintroducing the cells after chemotherapy to rebuild the immune system [7] - The clinical trial achieved a 95% success rate, with 59 out of 62 patients experiencing completely restored immune function over a 5-year period [8] - Post-treatment, children are able to live normal lives, attending school and engaging in typical childhood activities without unusual infections [8] Impact and Future Availability - UCLA has licensed the gene therapy to a biotech company, aiming for FDA approval within the next 2 to 3 years [15] - Babies are now screened for SKID before leaving the hospital, providing parents with early awareness [13]

Core Insights - The company has transitioned from being primarily a diabetes-focused entity to diversifying its portfolio, particularly in the area of rare diseases and orphan lung conditions [1] - The partnership with UT has been fruitful and continues to develop positively, indicating a strong collaborative effort in research and development [1] - The company aims to create more catalysts for growth and positive developments to attract momentum and catalyst-driven investors [1]

Core Insights - The company is focused on the successful launch of FILSPARI in IgA nephropathy as its top priority for the year [1] - There have been positive performance results in the first half of the year, with plans for exciting updates in the latter half [1] - The company has modified the liver monitoring REMS and removed the pregnancy testing REMS to simplify patient access [1] - The second priority involves ongoing work with the FDA for potential approval in FSGS [2]

Financial Data and Key Metrics Changes - The company is preparing to file its New Drug Application (NDA) for bitopertin in October, with a potential PDUFA date between June and October of the following year, marking a significant milestone for the company [4][5] - The company reported a P-value of less than 0.001 for the reduction in protoporphyrin-9 (PP9), indicating a clinically meaningful reduction in patients with erythropoietic protoporphyria [9][10] Business Line Data and Key Metrics Changes - The lead program, bitopertin, is in the accelerated approval pathway with the FDA, focusing on rare diseases related to red blood cell biology [4][5] - The second program is expected to present data on anemia of myelofibrosis and anemia of chronic kidney disease at upcoming conferences [5][6] Market Data and Key Metrics Changes - The company identified approximately 14,000 diagnosed patients in the U.S. for the target disease, with enrollment in trials not being a problem due to strong patient advocacy [18][23] - The company plans to leverage claims data to identify and validate physicians treating these patients, enhancing their market access strategy [23][24] Company Strategy and Development Direction - The company is focused on increasing disease awareness and ensuring that patients and physicians are informed about available therapies, as historically there have been no options for these patients [22][23] - The company aims to build a self-sustaining entity driven by revenues from bitopertin, with plans to explore additional indications within their iron modulation programs [60][61] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory pathway for bitopertin, citing positive interactions with the FDA regarding the use of PP9 as a surrogate endpoint [10][11] - The management highlighted the importance of continuous engagement with healthcare providers to ensure that patients receive appropriate care and treatment options [26][30] Other Important Information - The company has a strong financial position with $650 million on the balance sheet, which is expected to fund operations into 2028 [60] - The company has orphan protection for bitopertin, providing seven years of exclusivity in the U.S. plus potential pediatric extensions [36] Q&A Session Summary Question: Can you recap the interactions with the FDA regarding the accelerated approval path for bitopertin? - Management detailed a series of meetings with the FDA that led to the decision to pursue an accelerated approval pathway, emphasizing the strong rationale for using PP9 as a surrogate endpoint [10][11] Question: What are the key differences between the Apollo and Aurora trials? - Management explained that the Apollo trial will focus on measuring the time patients can spend in light, which is a clinically meaningful endpoint, alongside the reduction in PP9 levels [18][19] Question: How does the company plan to address the patient population for bitopertin? - Management discussed the importance of identifying engaged patients who regularly seek healthcare, estimating about 6,000 such patients, while also planning to reach out to the remaining diagnosed but less engaged patients [26][27] Question: What is the expected market opportunity for the company's anemia treatments? - Management indicated that there is a significant unmet need for anemia treatments in myelofibrosis, with an estimated market of about 22,000 patients, and potential pricing similar to existing therapies [42][44] Question: How does the company view its capital position and future financing needs? - Management expressed confidence in their capital position, indicating that they are well-funded to support ongoing trials and commercial preparations without immediate need for additional equity financing [60][61]

Financial Data and Key Metrics Changes - Total revenues grew by 26% year-on-year in Q2 2025, reaching $80,400,000, with a significant operating profit of $12,900,000 compared to a loss in the previous year [3][29] - Gross profit increased by 27% to $84,200,000, primarily driven by revenue growth [29] - For the first half of 2025, total revenues increased by 33% and gross profit increased by 37% compared to the same period in 2024 [30] Business Line Data and Key Metrics Changes - Reconnect revenue grew by 28% year-on-year, contributing significantly to the overall revenue growth [4][8] - Joenja achieved a revenue of $12,800,000 in Q2 2025, with a 15% quarter-on-quarter growth and a notable increase in patient uptake [13][14] Market Data and Key Metrics Changes - The U.S. market saw a 27% increase in new patient enrollments for Reconnect, driven by the addition of new prescribers [10] - Joenja's patient base is expanding, with 185 patients identified globally, including a launch in the UK [14][18] Company Strategy and Development Direction - The company aims to build a leading global rare disease platform, leveraging its strong capabilities and pipeline [7] - The focus is on expanding the patient base for Joenja through reclassification of VUS patients and geographic expansion [15][34] - The company is raising its 2025 total revenue guidance to between $335 million and $350 million, reflecting confidence in continued growth [32] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the growth potential of both Reconnect and Joenja, highlighting the unique value propositions of their products [33][34] - The company is committed to optimizing capital allocation and reducing G&A expenses by 15% to support growth [32] Other Important Information - The company has a high-value late-stage pipeline with two assets, each with over $1 billion potential [4][6] - The acquisition of Abliva is expected to enhance the company's pipeline and market position [29] Q&A Session Summary Question: How will the VUS patient reclassification translate to new patients on paid therapy? - Management expects about 20% of the 1,400 identified VUS patients to be reclassified as APDS, which will take time as diagnostic labs process the data [39][41] Question: Can you comment on the revenue growth for Joenja? - The revenue growth for Joenja was impacted by increased stock inventory in Q2 last year, not a change in conversion rates [47] Question: Are there segments of the Reconnect patient population at risk from the launch of sevitralstat? - The majority of Reconnect patients are those who have failed other treatments, and the patient populations targeted by both drugs are different [48][52] Question: Does the OpEx forecast include milestones on leniolumab? - Yes, the forecast includes a $5 million milestone related to leniolumab [68]