ProQR Therapeutics (PRQR) Q3 2026 earnings summary

Executive summary

• AX-0811 Phase 1 Cohorts 1 and 2 fully enrolled; initial target-engagement data expected in early Jan 2027, following positive AX-0810 Phase 1 target-engagement data reported in June 2026.

• €103.0m cash and cash equivalents at Sep. 30, 2026, with runway expected into mid-2028.

• Multiple pipeline milestones expected in H1 2027, including pediatric biliary atresia, AX-0422 in MPS I Hurler syndrome and AX-2911 for MASH.

Financial highlights

• Q3 revenue €5.155m vs €2.882m YoY; 9M revenue €15.947m vs €11.218m.

• Q3 R&D costs €14.323m vs €11.080m YoY; G&A costs €3.967m vs €3.119m.

• Q3 net loss €12.447m vs €11.006m; 9M net loss €34.558m vs €33.264m.

• Q3 net cash used in operating activities €13.586m vs €12.235m YoY; 9M cash used €37.001m vs €39.434m.

Outlook and guidance

• Full AX-0810 Phase 1 dataset, including Cohort 3 and 12-week follow-up, and new AX-0811 preclinical data expected in Dec. 2026.

• AX-0811 initial target-engagement data from healthy-volunteer Cohorts 1 and 2 expected in early Jan. 2027.

• Pediatric biliary atresia IIT in China targeted for initiation and initial data in H1 2027; a potentially registration-enabling Phase 2 program is expected to start mid-2027, subject to regulatory interactions, with interim data by mid-2028.

• AX-0422 CTA filing for a first-in-patient MPS I Hurler study anticipated in early 2027, with initial data in H1; AX-2911 MASH IIT in China expected in H1 2027.

• Lilly collaboration continues; potential data updates and milestone payments anticipated.