Applied Therapeutics (APLT) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Strategic overview and pipeline

• Transitioning from clinical development to commercial stage with a focus on rare diseases.

• Lead asset, govorestat, is under regulatory review for Classic galactosemia in the US and Europe.

• Positive late cycle FDA review; no advisory committee meeting required, with next FDA interaction in about a month.

• EMA review is in Day 120 clock stop, with CHMP opinion expected late 2024 or early 2025.

• SORD deficiency program is preparing for NDA submission in early Q1 2025.

Clinical data and efficacy

• Govorestat demonstrated rapid and sustained reduction of toxic metabolites in both galactosemia and SORD deficiency.

• Clinical trials showed stabilization or improvement in behavioral, cognitive, and motor symptoms in pediatric galactosemia patients.

• Phase III SORD deficiency study showed significant sorbitol reduction and correlation with improved clinical outcomes.

• Clean safety profile observed across studies, with long-term exposure data supporting favorable risk-benefit.

• Nearly 200 patients treated, with some on therapy for over three years.

Market opportunity and commercial readiness

• Galactosemia affects about 3,300 patients in the US, with mandatory newborn screening ensuring accurate patient identification.

• SORD deficiency impacts around 5,000 US patients, with numbers expected to grow as more mutations are identified.

• No approved treatments exist for either indication, positioning govorestat as a potential first-in-class therapy.

• Strong commercial and medical field teams in place, with high physician and patient advocacy engagement.

• Market research indicates high anticipated early adoption by physicians upon approval.