uniQure's AMT-130 Shows 61% Slowing of Huntington's Progression at 48 Months, Supporting Pending BLA

uniQure reported new long-term data from its ongoing Phase I/II study of ifezuntirgene inilparvovec, known as AMT-130, showing continued slowing of Huntington's disease progression in 12 high-dose patients followed for 48 months. The primary endpoint of cUHDRS showed 44% slowing of disease progression versus an external control, while the Total Functional Capacity measure showed a nominally significant 61% slowing. The company noted the 48-month treatment effect is likely understated due to substantial missing data and survivor bias in the updated external control, with a post-hoc analysis using a prior external control showing 54% slowing on cUHDRS and 68% on TFC.

At the regulatory anchor timepoint of 36 months, an updated analysis of all 15 high-dose patients showed an 80% slowing of progression on cUHDRS and 67% on TFC, both nominally significant. Those figures support uniQure's submitted Biologics License Application, which the FDA confirmed in June 2026 could use the three-year analysis as its primary regulatory basis. Huntington's disease currently has no approved disease-modifying treatments.

In a separate oncology development, Cellectar Biosciences reported a Phase 2 CLOVER WaM subset analysis showing encouraging efficacy for iopofosine I 131 in Waldenstrom macroglobulinemia patients refractory or resistant to BTK inhibitor therapy. Earlier data from the same program had shown a 79.2% major response rate and a median duration of response exceeding 16 months in patients treated immediately after BTKi therapy, with a Phase 3 confirmatory trial site activation already underway and first patient dosing expected in late 2026 or early 2027.

Decoy Therapeutics also released preclinical pharmacokinetic data supporting once-weekly intranasal dosing of DCOY-CoV, its lead program targeting endemic human coronaviruses. Separately, Jaguar Health shares fell roughly 9% in premarket trading after the company announced that preliminary results from an open-label study of crofelemer in adult short bowel syndrome patients with intestinal failure were accepted for presentation at the annual American College of Gastroenterology meeting.