Aardvark Therapeutics (AARD) Morgan Stanley 23rd Annual Global Healthcare Conference summary

Lead program and disease focus

• Lead oral small molecule program, ARD-101, is in phase III for Prader-Willi syndrome, targeting hunger aggregation.

• Prader-Willi syndrome affects 10,000–20,000 in the US and 300,000–400,000 globally, with severe hunger, anxiety, inflammation, and constipation.

• ARD-101 activates gut taste receptors, stimulating hormones like CCK and GLP-1 to reduce hunger and improve glucose homeostasis.

• The drug is gut-restricted, minimizing systemic exposure and enhancing safety.

• Phase III HERO trial includes younger patients and aims for a robust label and broader patient benefit.

Clinical data and trial design

• Phase II data showed an 8-point reduction in the HQ-CT hunger score after 28 days, with rapid onset of hunger reduction and anecdotal improvements in anxiety and inflammation.

• Phase III HERO trial uses a 1:1 placebo-to-active ratio, 12-week primary endpoint, and expanded secondary endpoints including biomarkers.

• Dose escalation in phase II led to selection of 800 mg b.i.d. for phase III, with no new safety concerns at this dose.

• Enhanced trial controls address placebo effect and environmental factors, with more precise baseline assessments.

• Enrollment is strong, with data expected in Q3 next year.

Competitive landscape and differentiation

• Only one approved drug exists for Prader-Willi hyperphagia; ARD-101 offers a differentiated, complementary mechanism and favorable safety profile.

• Gut restriction avoids renal contraindications and systemic side effects, addressing unmet needs.

• Orphan drug status and late-stage development provide competitive positioning.