Egetis Therapeutics (EGTX) investor relations material

Strategic highlights and product development

• Emcitate (tiratricol) launched in Germany in May 2025 as the first and only approved treatment for MCT8 deficiency in the EU, with FDA PDUFA date set for September 28, 2026.

• Emcitate has received orphan drug designation, breakthrough therapy, and rare pediatric disease designations, securing market exclusivity and a potential priority review voucher in the US.

• The company is executing a phased EU launch, starting with Germany and expanding to other major European markets, while preparing for a US launch in Q4 2026.

• Partnerships established for Japan (Fujimoto), Türkiye, Central/Eastern/Southeastern Europe (Er-Kim), and the Gulf region (taiba rare) to optimize global reach.

• Expansion into Resistance to Thyroid Hormone Type Beta (RTH-β) is being considered, with orphan drug designation granted for this indication.

Clinical and regulatory progress

• Emcitate demonstrated significant and durable reduction of T3 levels, normalization of thyrotoxicosis, and improvements in cardiovascular health and bodyweight in MCT8 deficiency patients.

• Real-world and clinical trial data show a threefold reduction in mortality risk for patients treated with tiratricol.

• European Thyroid Association recommends Emcitate as long-term therapy for all MCT8 deficiency patients, with potential for US guideline adoption post-approval.

• Regulatory milestones include EU approval in February 2025, US NDA acceptance with priority review in March 2026, and robust clinical data supporting regulatory submissions.

• Positive results from the ReTRIACt withdrawal study and ongoing real-world evidence further support efficacy and safety.

Commercialization and market opportunity

• Q1 2026 revenue reached 13.4 MSEK, up 9% YoY, with momentum driven by increased patient identification and expanded access.

• Pricing and reimbursement processes are advancing in Germany, France, Spain, and Italy, with named patient sales in Poland and Turkey.

• US launch readiness includes >140 confirmed patients, established early access centers, and payer engagement for coverage and access.

• Annual treatment costs for comparable orphan drugs in the US range from $623K to $1.12M, with strong evidence supporting premium pricing for Emcitate.

• The orphan drug segment offers attractive returns, shorter development timelines, and higher success rates compared to non-orphan drugs.