Full Year 2025 Results Call Corporate Update & Financial Results February 26, 2026 CONFIDENTIAL & PROPRIETARY Forward-looking statements This presentation contains forward-looking statements, including statements regarding, among other things, future results, performance or achievements, expectations regarding pipeline development, anticipated approval and commercialization of navenibart, pharmaceutical research and development, such as drug discovery, preclinical and clinical development activities and rel ...

Mirum Pharmaceuticals (NasdaqGM:MIRM) Q4 2025 Earnings call February 25, 2026 04:30 PM ET Company ParticipantsAndrew McKibben - SVP of Strategic Finance and Investor RelationsCharles Wallace - Managing DirectorChris Peetz - CEOEric Bjerkholt - CFOJames Condulis - VP of Biotechnology Equity ResearchJoanne Quan - Chief Medical OfficerJoe Kim - Assistant VPJonathan Wolleben - Managing DirectorJoseph Thome - Managing DirectorRyan Deschner - Director of Equity ResearchConference Call ParticipantsBrian Skorney - ...

Soleno Therapeutics (NasdaqCM:SLNO) Q4 2025 Earnings call February 25, 2026 04:30 PM ET Speaker1Good afternoon, thank you for standing by. Welcome to Soleno Therapeutics' fourth quarter and full year 2025 financial and operating results conference call and webcast. Currently, all participants are in a listen-only mode. After the speaker presentation, there will be a question-and-answer session. As a reminder, today's webcast is being recorded. I would now like to introduce Brian Ritchie of LifeSci Advisors. ...

Mirum Pharmaceuticals (NasdaqGM:MIRM) Q4 2025 Earnings call February 25, 2026 04:30 PM ET Speaker5Good afternoon, and welcome to Mirum Pharmaceuticals' fourth quarter and full year 2025 conference call. My name is Elliot, and I'll be your operator today. All lines are currently in listen-only mode, and there'll be an opportunity for Q&A after management's prepared remarks. I'd now like to hand the conference over to Andrew McKibben, SVP of Strategic Finance and Investor Relations. Please go ahead.Speaker0Th ...

DESMODA is the first and only FDA-approved desmopressin oral solutionCommercial launch expected on March 9thDESMODA eliminates tablet splitting and crushing, enabling precise, individualized dosing for patients of all agesThe product is expected to be a significant long-term growth contributor, with potential peak sales of $30-50 million annually and patent protection extending through 2044 DEER PARK, Ill., Feb. 25, 2026 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), ...

BridgeBio (NasdaqGS:BBIO) Q4 2025 Earnings call February 24, 2026 04:30 PM ET Speaker9Good afternoon. I'll be your conference operator today. All lines have been placed on mute to prevent any background noise. After the company's remarks, there will be a question and answer session. If you would like to ask a question, press star followed by 1 on your telephone keypad. If you'd like to withdraw your question, press the pound key. Thank you. Before we begin, I'd like to remind everyone that today's call may ...

Core Viewpoint - Soleno Therapeutics is currently not providing full-year guidance as it seeks to better understand its business dynamics, but management indicates that guidance may be offered in the future when more comfortable with the data [1] Group 1: Company Overview - Soleno Therapeutics is a commercial-stage company focused on rare diseases, specifically with an approved product for Prader-Willi syndrome (PWS), which is characterized by hyperphagia [3][18] - The company has been marketing its product for approximately three quarters [3] Group 2: Launch and Market Dynamics - The company expects to capture about 10% of the total addressable market (TAM), translating to approximately 1,000 start forms over a 9 to 12-month period, with variability from quarter to quarter [2][6] - The first-quarter report will be the last to disclose start-form metrics, after which the focus will shift to revenue and active patient reporting [2][6] Group 3: Reimbursement and Coverage - Reimbursement has exceeded expectations, with coverage through nearly 48 Medicaid programs and Medicare being a strong payer [5][14] - The prescriber base has grown to over 600, although many prescribers are new to PWS and require education on the drug [15] Group 4: Patient Conversion and Revenue - Start forms typically convert to active patients within 30 to 45 days, with a single-digit cancellation rate and overall discontinuation around 15% to 20% [6][8] - Revenue is primarily driven by active paid patients, adherence, wholesale acquisition cost (WAC), and gross-to-net dynamics [8] Group 5: International Regulatory Progress - The company is progressing with its European regulatory review, expecting Day 180 questions by the end of February for a patient pool of approximately 9,500 in the EU4 plus the UK [5][16] - Soleno is building its European team and is considering direct market engagement while also exploring partnerships [16] Group 6: Future Plans and Capital Deployment - Capital deployment priorities include supporting U.S. and international launches, pursuing additional indications for its product, and evaluating inorganic growth opportunities, though significant deals are not anticipated in the short term [17]

Financial Data and Key Metrics Changes - In Q4 2025, Agios Pharmaceuticals reported net revenue of $20 million from PYRUKYND, an increase of 86% year-over-year and a sequential increase of 55% compared to $13 million in Q3 2025 [10][11] - Full-year 2025 revenue for PYRUKYND reached $54 million, reflecting robust year-on-year growth [7][10] - The company ended Q4 2025 with approximately $1.2 billion in cash, cash equivalents, and marketable securities, providing flexibility for future investments [8][11] Business Line Data and Key Metrics Changes - U.S. revenue for PYRUKYND in Q4 was $16 million, driven by commercial focus in PK deficiency and favorable growth adjustments [10][11] - Ex-U.S. revenue was $4 million in Q4, primarily reflecting inventory stocking ahead of demand from PK deficiency patients in Europe [11] - The company anticipates U.S. PK deficiency revenues to be in the range of $45-$50 million in 2026 [10] Market Data and Key Metrics Changes - The ACTIVASE launch for thalassemia in the U.S. is underway, with strong early demand and 44 prescriptions written by REMS-certified physicians as of January 30 [15][41] - The company has received approval for mitapivat for thalassemia in two regions, with plans for commercialization in Europe and the GCC [18][19] Company Strategy and Development Direction - Agios has outlined its 2026 strategic priorities, focusing on executing the ACTIVASE launch, expanding the PK activation franchise, advancing early-stage pipeline programs, and maintaining financial discipline [5][6] - The company aims to leverage its existing commercial presence in thalassemia and PK deficiency to achieve profitability [12][72] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the early market response to ACTIVASE, noting that physicians view its profile as addressing significant gaps in current treatment options for thalassemia [16][18] - The company anticipates a pivotal year ahead with multiple catalysts, including the pre-sNDA meeting for mitapivat and phase 2 data for tebapivat [25][26] Other Important Information - The company is committed to financial discipline, with operating expenses in 2026 expected to be roughly flat compared to 2025 [12] - Agios is focused on maximizing the launch of ACTIVASE while continuing to advance its pipeline programs [8][25] Q&A Session Summary Question: Can you provide insights on the ACTIVASE launch and prescription updates? - Management noted that early demand is encouraging, with 44 prescriptions written in the first five weeks of launch, and expects prescriptions to grow ahead of revenues due to a 10-12 week conversion time to treatment initiation [31][41] Question: What are the expectations for the phase 2 data in sickle cell disease? - Management indicated that the phase 2 tebapivat trial is a dose-finding study, and they are excited about the potential hemoglobin response based on previous data [35][36] Question: Can you discuss the payer dynamics for ACTIVASE? - The payer mix is similar to PK deficiency, with most patients under commercial payers, and initial access will occur through medical exceptions [93] Question: What is the rationale for higher dosing in lower-risk MDS? - Higher doses are being tested in MDS due to faster metabolism in patients compared to sickle cell disease, which is modeled after healthy volunteers [101] Question: How does the REMS program for thalassemia impact sickle cell disease? - Management believes that the experience gained from the thalassemia REMS program will provide a solid foundation for executing a similar program if needed for sickle cell disease [104]

Financial Data and Key Metrics Changes - In Q4 2025, Agios Pharmaceuticals reported worldwide PYRUKYND revenue of $20 million, an increase of 86% compared to Q4 2024, and a sequential increase of 55% from $13 million in Q3 2025 [9][10] - Full-year 2025 revenue for PYRUKYND reached $54 million, reflecting robust year-on-year growth [5][9] - The company ended Q4 2025 with approximately $1.2 billion in cash, cash equivalents, and marketable securities, providing flexibility for future investments [6][10] Business Line Data and Key Metrics Changes - U.S. fourth quarter revenues for PYRUKYND were $16 million, driven by commercial focus in PK deficiency and favorable growth adjustments [9] - Ex-U.S. revenue was $4 million in Q4, primarily reflecting inventory stocking ahead of demand [10] - Cost of sales for Q4 was $1.9 million, R&D expenses were $88.1 million, and SG&A expenses were $51.6 million, roughly flat year-on-year [10] Market Data and Key Metrics Changes - The ACTIVASE launch for thalassemia in the U.S. is underway, with expectations for U.S. PK deficiency revenues in 2026 to be in the range of $45-$50 million [9][10] - The company anticipates a sequential decline in ex-U.S. revenues into Q1 2026 due to inventory adjustments [10] Company Strategy and Development Direction - The company outlined its 2026 strategic priorities, focusing on executing the ACTIVASE launch, expanding the PK activation franchise, advancing early-stage pipeline programs, and maintaining financial discipline [4][5] - Agios aims to leverage its capital-efficient global commercial model to focus investments on the U.S. market, which presents the most significant revenue opportunity [16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the early demand for ACTIVASE, noting strong engagement from physicians and a healthy breadth of early prescribers [14][40] - The company sees a clear path to profitability through its existing commercial presence in thalassemia and PK deficiency, with operating expenses expected to remain flat in 2026 [11][68] Other Important Information - The company received FDA approval for ACTIVASE on December 23, 2025, and the U.S. launch is underway with positive early feedback from physicians [18][19] - Enrollment in the phase II sickle cell disease trial of tebapivat is complete, with top-line results expected in the second half of 2026 [6][20] Q&A Session Summary Question: How does the ACTIVASE launch translate to treatment initiation and revenue recognition? - Management indicated that prescriptions are expected to grow ahead of revenues due to a 10-12 week conversion time from prescription to treatment initiation [29][30] Question: What are the expectations for the phase II data in sickle cell disease? - Management expressed excitement about the trial, noting that it is a dose-finding study aimed at exploring hemoglobin response [34][35] Question: What is the payer mix for ACTIVASE and how does it affect revenue? - The payer mix is similar to PK deficiency, with most patients under commercial payers, and initial market access will occur through medical exceptions [90] Question: What is the rationale for higher dosing in lower-risk MDS compared to sickle cell disease? - Higher doses in MDS are due to faster metabolism in MDS patients, while sickle cell patients metabolize the drug similarly to healthy volunteers [98] Question: What is the expected timeline for profitability? - Management has not provided specific timing but indicated that thalassemia presents a meaningful opportunity for profitability [68][70]

Core Insights - Soligenix is a late-stage biopharmaceutical company focused on developing treatments for rare diseases with unmet medical needs, highlighting significant progress in its clinical pipeline and upcoming milestones for 2026 [1][2] Clinical Developments - Top-line results from the Phase 2a trial of SGX302 for mild-to-moderate psoriasis showed that the gel therapy was well tolerated, with one patient achieving "Almost Clear" status and over 50% improvement in PASI score [1] - The Phase 2a trial of SGX945 for Behçet's Disease demonstrated biological efficacy, with results comparable to apremilast, indicating potential for home-based treatment in future studies [1] - Ongoing studies for HyBryte™ in early-stage cutaneous T-cell lymphoma (CTCL) show promising results, with 75% of patients achieving "Treatment Success" after 18 weeks of treatment [1] Market Opportunities - Peak annual net sales for HyBryte™ in the U.S. are expected to exceed $90 million, with a total addressable worldwide CTCL market estimated at over $250 million annually [2] - The psoriasis market opportunity with SGX302 is projected to exceed $1 billion annually, while SGX945 for Behçet's Disease is estimated at approximately $200 million annually [2] - Overall, the potential for significant commercial returns across the rare disease pipeline is estimated at around $2 billion in global annual sales [2] Financial Position - As of September 30, 2025, the company reported approximately $10.5 million in cash, with additional non-dilutive funding of about $500 thousand from New Jersey's NOL sales program [2] - The company is focused on advancing its development programs and has sufficient capital to meet its goals through 2026 [2]