Status note Pivotal registrational Phase 3 under open FDA IND enrolling. FDA Fast Track (March 2026); Japan MHLW Orphan Drug Designation (June 2026). Compassionate use program in 8 pediatric patients.
QRX009
Preclinical · ActiveModality: topical formulation
Partnerships University College Cork (UCC): research agreement for novel topical rapamycin formulations
Indications
Pachyonychia Congenita(Preclinical)
Gorlin Syndrome(Preclinical)
Tuberous Sclerosis Complex(Preclinical)
Microcystic lymphatic malformations(Preclinical)
Venous malformations(Preclinical)
Angiofibromas(Preclinical)
Catalysts
— Q3 2026
Status note Topical rapamycin (sirolimus). Investigator-led clinical studies planned in Pachyonychia Congenita, Gorlin Syndrome, and Tuberous Sclerosis Complex.
QRX008
Preclinical · Active
Target VLA-4
Partnerships Queensland University of Technology (QUT): research agreement; option for a global license (Netherton Syndrome research with QUT discontinued July 2025)
Indications
Scleroderma(Preclinical)
Status note Preclinical research program; company has option for global license.
Per-asset detail extracted from the 10-Q filed 2026-08-14. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
No lead program on file for this company.
No active ClinicalTrials.gov trials matched to this company as sponsor or collaborator.
Institutional holders
Fund
Shares
Q/Q change
% of portfolio
Filing
Ikarian Capital
211,538
no change
0.17%
2026-08-14
Institutional holder positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.
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