Applied Therapeutics (APLT) UBS Global Healthcare Conference 2024 summary

Regulatory and commercial milestones

• Govorestat is nearing commercial launch for galactosemia, with a US PDUFA date of November 28 and a European CHMP opinion expected in Q1 2025.

• NDA submission for SORD deficiency is planned for Q1 2025, following FDA alignment on accelerated approval.

• Both indications have orphan and pediatric rare disease designations, with late-stage regulatory review ongoing.

• Commercial infrastructure and patient support programs are established and will be leveraged for both indications.

• Market research indicates strong anticipated uptake among physicians, caregivers, and payers.

Disease background and unmet need

• Galactosemia and SORD deficiency are rare, progressive diseases involving sugar metabolism, affecting about 7,000 and up to 5,000 patients in the US and Europe, respectively.

• Galactosemia is identified at birth via mandatory newborn screening, but no current treatments exist beyond dietary restriction.

• SORD deficiency, a subtype of Charcot-Marie-Tooth disease, leads to progressive neuromuscular impairment and is now recognized as more prevalent due to new genetic findings.

• Both diseases cause significant lifelong disability and burden for patients and families.

• Patient advocacy groups are actively engaged in awareness and support efforts.

Clinical development and efficacy

• Govorestat is an oral, once-daily small molecule inhibitor of aldose reductase, targeting toxic metabolite buildup in both diseases.

• In galactosemia, clinical studies show rapid and sustained reduction of galactitol, correlating with stabilization or improvement in neurological symptoms.

• Placebo groups in trials showed disease progression, highlighting the urgency of treatment.

• In SORD deficiency, govorestat demonstrated significant sorbitol reduction and correlation with improved clinical outcomes in a phase 3 trial.

• Both indications have shown strong safety and tolerability profiles over multiple years of use.