Applied Therapeutics (APLT) Baird's 2024 Global Healthcare Conference summary

Pipeline and regulatory progress

• Lead drug govorestat is under FDA and EMA review for galactosemia, with a PDUFA date of November 28 and a parallel submission in Europe.

• NDA for SORD deficiency, a neuromuscular rare disease, is planned for accelerated approval submission in Q1 2025.

• Both indications target rare diseases with no current treatments, addressing urgent unmet needs.

• Pediatric and adult studies show rapid, sustained, and statistically significant galactitol reduction, correlating with symptom improvement in children.

• EMA review is progressing in parallel, with positive rapporteur meetings and ongoing responses to regulatory comments.

Clinical data and efficacy

• Govorestat demonstrated 40–50% reduction in galactitol, leading to improvements in behavior, cognition, and motor skills in galactosemia children.

• Strong biomarker data in adults supports the application for approval, with plans for future clinical outcome studies.

• In SORD deficiency, govorestat reduced toxic sorbitol levels and showed significant effects on patient-reported outcomes and motor function.

• FDA alignment achieved for accelerated approval in SORD deficiency, based on biomarker and clinical data.

• Confirmatory study protocol for SORD deficiency is being finalized with the FDA.

Regulatory and advisory committee updates

• FDA has tentatively scheduled an advisory committee meeting for October 9 for galactosemia, with preparations underway.

• The new Genetic Metabolic Diseases Advisory Committee reflects increased regulatory flexibility for rare diseases.

• EMA review is proceeding smoothly, with conditional approval sought in Europe.

• Commercial launch preparations are underway, with heads of commercial functions hired and sales force hiring planned closer to approval.