Egetis Therapeutics (EGTX) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Company overview and strategy

• Focuses on late-stage development and commercialization of therapies for rare diseases, with a growing presence in the U.S. and Europe.

• Lead candidate is tiratricol (Emcitate) for MCT8 deficiency, an ultra-rare thyroid hormone disorder.

• Company has expanded to 60 employees, with a third based in the U.S., and is investing in U.S. market engagement.

• Partnerships established for Central Eastern Europe, Turkey, Gulf region, Australia, and New Zealand, with plans for further global expansion.

• U.S. market is the primary commercial focus, with ongoing regulatory submissions in other regions.

MCT8 deficiency: Market and patient identification

• Initially, only about 20 U.S. patients were identified; now over 100 are diagnosed, with 60 on an expanded access program (EAP).

• Patient identification initiatives include analytics, partnerships with genetic labs, registries, and advocacy groups.

• Diagnosis rates are expected to rise as awareness and access improve, as seen in Germany where diagnosed patients doubled post-launch.

• Transitioning EAP patients to commercial therapy is a top priority for rapid revenue realization.

• Estimated U.S. patient population is around 1,000, with significant opportunity for further identification.

Clinical data and regulatory progress

• Three prospective clinical trials and robust real-world evidence support tiratricol's efficacy and safety.

• Key clinical benefits include rapid and durable normalization of T3, improved cardiovascular outcomes, and potential survival benefits.

• Product launched in Germany in May 2022, with strong initial uptake and increased physician interest.

• U.S. NDA under Priority Review, with PDUFA date set for September 28; collaborative discussions ongoing with FDA.

• Patent protection extends to 2045, supplementing orphan drug exclusivity in the U.S. and Europe.