Summary of Septerna's Presentation at the J.P. Morgan Healthcare Conference Company Overview - Company Name: Septerna - Focus: Development of drugs targeting G-Protein Coupled Receptors (GPCRs) using the Native Complex Platform for drug discovery [2][5] Key Programs 1. SCP479: - Target: Parathyroid hormone receptor agonist for hypoparathyroidism - Status: Wrapping up IND-enabling studies, expected to enter phase one trials in the first half of 2025 [3][25] - Market Opportunity: Significant unmet need; current standard of care involves high doses of calcium and vitamin D [46] - Clinical Data: Early studies show normalization of serum calcium levels in animal models [19][24] - Pharmaceutical Properties: Projected human half-life of 40-80 hours, supporting once-daily oral dosing [23] 2. SCP631: - Target: MRGPRX2 for mast cell-driven diseases, including chronic spontaneous urticaria - Status: Phase one trial wrapping up, with data expected to be shared at the Quad AI meeting in March [4][31] - Mechanism: Insurmountable negative allosteric modulator, effectively turning off the receptor [28][48] - Market Need: Significant unmet need for second-line treatment options for patients refractory to high-dose antihistamines [27] 3. Other Programs: - Thyroid-Stimulating Hormone Receptor (TSHR): Targeting Graves' disease and thyroid eye disease with a negative allosteric modulator [34][35] - Incretin Receptor Agonist Program: Collaboration with Novo Nordisk focusing on multiple receptors [39] Market Insights - Hypoparathyroidism Market: Approximately 70,000-80,000 patients in the U.S.; current treatments are not fully effective, creating demand for oral options [46][47] - Mast Cell Disorders: Growing evidence of mast cell involvement in various conditions, indicating a broad opportunity for SCP631 [50] Platform and Technology - Native Complex Platform: Allows for the isolation of GPCRs in a functional form, enabling high-resolution structure determination and screening of billions of compounds [7][9] - Drug Discovery Efficiency: Rapid optimization of compounds, with timelines from medicinal chemistry to active compounds in animals being less than a year [10] Financial Position - Capitalization: Well-capitalized with cash runway into at least 2029 following IPO and collaboration deals [3] - Collaboration with Novo Nordisk: Includes three receptors and undisclosed targets, with Novo covering all R&D costs going forward [39] Regulatory and Development Strategy - Phase One Trials: Focus on safety, tolerability, and pharmacokinetics, with plans to pivot quickly to patient studies based on promising results [45] - IP Strategy: Protecting novel binding pockets and chemical structures identified through the platform [52] Conclusion - Future Focus: Execution on the current pipeline while remaining opportunistic for business development and partnerships [53]
Septerna (NasdaqGM:SEPN) FY Conference Transcript