Egetis Therapeutics (EGTX) Morgan Stanley 24th Annual Global Healthcare Conference summary

Company overview and strategy

• Focuses exclusively on rare diseases, with late-stage development and ambition to commercialize in Europe and the U.S., while expanding access globally through partnerships.

• Formed in late 2020, the company has grown to 60 employees and leverages a lean, targeted approach to market entry and expansion.

• Aims to build a sustainable rare disease platform, with plans for indication expansion and potential in-licensing or acquisition of additional assets.

Lead asset and clinical data

• Lead product targets MCT8 deficiency, an ultra-orphan, X-linked disorder affecting about 1 in 70,000 males, with severe neurocognitive and cardiovascular impacts.

• Emcitate (tiratricol) is the first and only approved treatment in the EU, with a U.S. PDUFA date set for September 28, 2026.

• Clinical data includes three prospective trials and three real-world studies, showing normalization of T3 levels and significant cardiovascular and survival benefits.

• A recent survival study with 612 patients demonstrated substantial survival benefit for treated patients.

Competitive landscape and patient access

• No other sponsor-driven drugs are in clinical development for MCT8 deficiency; only some investigator-initiated studies exist.

• Hundreds of patients have accessed the drug globally through managed access programs prior to approval.