Aardvark Posts Exploratory HERO/OLE Data, Targets New Phase 3 After FDA Hold Lift

summarizeSummary

Aardvark disclosed exploratory HERO/OLE data showing a modest HQ-CT benefit for ARD-101 versus placebo, an additional ~6-point drop in placebo patients switched to active drug, and mostly mild-to-moderate safety. With the FDA hold lifted, the company plans a lower-dose restart and an FDA meeting to design a new Phase 3, targeting resumed PWS clinical activity in 2027.

check_boxKey Events · Product Development and Regulatory · AARD

• Exploratory HERO/OLE Efficacy Data Released

  HERO randomized 67 of 90 planned participants before early termination, so all efficacy data are exploratory. HQ-CT scores fell modestly versus placebo by Week 2 of dose escalation, and the ARD-101 group returned to near baseline after dosing stopped while placebo rebounded less.

• Placebo-to-ARD-101 Switch Adds ~6 Points

  In the OLE cohort, participants who switched from placebo to ARD-101 had an observed average reduction of 6 points in HQ-CT score by Day 28.

• One Grade 4 Cardiac Event at 800 mg BID

  A single serious adverse event of polymorphic ventricular tachycardia occurred at 800 mg BID in a participant also taking pitolisant, a CYP2D6 substrate; the FDA concurred it is a drug-drug interaction. Most other adverse events were mild to moderate.

• Lower-Dose Restart and FDA Meeting Planned

  Aardvark plans to resume dosing in PWS patients at a dose below the 800 mg BID studied and will request an FDA meeting to align on a new Phase 3 design, expecting PWS clinical activities to resume in 2027.

auto_awesomeAnalysis · AARD · Life Sciences

Aardvark has released the first look at efficacy and safety data from its terminated Phase 3 HERO trial and open-label extension. The numbers are explicitly exploratory — HERO randomized only 67 of 90 planned participants — but the direction is supportive: HQ-CT scores fell modestly versus placebo by Week 2 of dose escalation, symptoms rebounded toward baseline after ARD-101 stopped while placebo rebounded less, and placebo patients who switched to ARD-101 in the OLE saw an additional ~6-point HQ-CT drop. Safety was mostly mild-to-moderate, with the single Grade 4 cardiac event tied to a drug-drug interaction with pitolisant at the 800 mg BID dose. Combined with the FDA lifting the clinical hold on October 22, the company now has a stated path: resume PWS dosing at a lower dose and request an FDA meeting to design a new Phase 3, with clinical activity expected to resume in 2027. The stock trades near its 52-week low, so this is the first substantive evidence management can point to since the August trial termination — but the data are not powered to prove efficacy, and the next Phase 3 is at least a year away.