MBX Biosciences FY Conference Summary Company Overview - **Company**: MBX Biosciences (NasdaqGS:MBX) - **Focus**: Pioneering Precision Endocrine Peptides (PEPs) for treating endocrine and metabolic disorders [1][4] Key Updates and Expectations for 2026 - **Pivotal Year**: 2026 is expected to be crucial with significant milestones across three clinical stage programs and advancements in obesity discovery and preclinical development [1] - **Cash Runway**: The company has a cash runway extending into 2029, providing flexibility in advancing its pipeline [3] Clinical Programs Canvuparatide - **Phase II Meeting**: An end-of-phase II meeting with the FDA is scheduled, with updates on study design for phase III expected [2] - **Phase II Update**: One-year data will be released in Q2, with a global registrational phase III study initiation planned for Q3 [2] - **Response Rate**: A 63% response rate was observed in a 12-week placebo-controlled study, with 79% of patients responding during the open-label extension [9][10] Obesity Portfolio - **MBX-4291**: A once-monthly GLP-1/GIP co-agonist with anticipated 12-week data from a multiple ascending dose study [2] - **New Candidates**: Two additional candidate selections targeting amylin and glucagon are expected in Q2 and Q3 [2] Imapextide - **Phase IIa Study**: A pharmacodynamic study is underway for post-bariatric hypoglycemia, with results expected in Q2 2024 [24][25] Technology and Differentiation - **PEP Technology**: The proprietary PEP technology allows for slow release and steady exposure, improving tolerability and reducing side effects associated with traditional therapies [4][19] - **Best-in-Class Potential**: The company aims to develop best-in-class drugs, with positive feedback from the physician community regarding their clinical data [12][15] Market Opportunity - **Hypoparathyroidism**: The disease has a prevalence of over 250,000 in the US and EU, with current treatments being inadequate [7] - **Obesity Treatment**: The company recognizes obesity as a heterogeneous disease requiring diverse therapeutic options, with a focus on improving tolerability and adherence [18] Financial Position - **Cash Balance**: Year-end cash balance of $373 million, projected to increase to $460 million post recent stock sale, supporting operations through 2029 [34][35] - **Commercialization Plans**: Preparations for the commercialization of canagliflozin are underway, with a focus on recruiting a Chief Commercial Officer [36] Conclusion - **Growth Potential**: MBX Biosciences is positioned for a catalyst-rich year with multiple data releases and a strong pipeline aimed at addressing significant health issues in endocrine and metabolic disorders [33][36]

Phase 2 Trial and Open-Label Extension Top-Line Results: Once-Weekly Canvuparatide in Adults with Hypoparathyroidism September 22, 2025 Disclaimer This presentation includes forward looking statements. All statements other than statements of historical facts contained in this presentation, including statements regarding our product candidates, preclinical study and/or clinical trial timelines, including projected data announcements, future results of operations and financial position, strategy and plans, in ...

Core Insights - Ascendis Pharma announced positive long-term data from the Phase 3 PaTHway Trial for TransCon PTH, showing sustained efficacy in treating adults with hypoparathyroidism [1][4] - The trial demonstrated significant improvements in biochemical markers, kidney function, and quality of life for patients [1][3] Group 1: Trial Details - The PaTHway Trial was a Phase 3, randomized, double-blind, placebo-controlled study involving 82 adults with chronic hypoparathyroidism, followed by an open-label extension [2] - At Week 156, 88% of patients had normal serum calcium levels, and 96% were independent from conventional therapy [3] - The trial maintained high retention rates, with 89% of participants completing the 3.5-year study [2] Group 2: Efficacy and Safety - Mean estimated glomerular filtration rate (eGFR) increased by 8.76 mL/min/1.73 m² across all participants, with a notable increase of 13.98 mL/min/1.73 m² in those with baseline eGFR < 60 [3] - Patients reported continued improvements in hypoparathyroidism-related symptoms and health-related quality of life, with normalization of 24-hour urine calcium excretion [3] - TransCon PTH treatment was well-tolerated, with no new safety signals identified and most treatment-emergent adverse events being mild or moderate [3] Group 3: Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform [6] - The company aims to address unmet medical needs and improve patient outcomes through its product innovations [6]