Uniqure, Inc.

FDA agrees that ongoing Phase 1/2 trials of AMT-130, compared with an external control group, may support accelerated approval for Huntington’s disease. Key measures include changes in cUHDRS and NfL levels. Accelerated approval pathway aims to speed delivery of potentially life-changing therapy, reducing time to potential licensure by approximately five years.

uniQure's stock surged due to FDA's willingness to review AMT-130, a Huntington's disease gene therapy. Despite challenges, AMT-130 shows potential, with strong evidence of slowing disease progression. The stock's modest market cap could increase significantly if AMT-130 is approved, making it a high-risk, high-reward investment.

AstraZeneca and BeiGene presented at ASH on leukemia treatments. uniQure advanced its Huntington’s therapy, while Q32 Bio and Keros faced setbacks in their trials.

Clene receives FDA guidance on accelerated approval for CNM-Au8 in ALS, planning biomarker analysis. uniQure aligns with FDA on accelerated approval pathway for AMT-130 in Huntington disease. AbbVie's tavapadon meets primary and secondary endpoints in early Parkinson disease phase 3 trial.

Clene's ALS drug CNM-Au8 and UniQure's Huntington's disease gene therapy aim for accelerated FDA approval using real-world evidence from compassionate use and natural history data.

FDA agrees to uniQure's accelerated approval pathway for AMT-130 gene therapy in Huntington disease, using phase 1/2 trial data and cUHDRS as intermediate clinical endpoint. High-dose AMT-130 showed significant slowing of disease progression and lowering of NfL in CSF.

uniQure's AMT-130, a gene therapy for Huntington's disease, aligns with FDA for accelerated approval, using cUHDRS and NfL metrics. Two ongoing trials (HD-GeneTRX-1 and HD-GeneTRX-2) aim to determine safety and potential effects on HD symptoms. The FDA's acceptance of cUHDRS and NfL as endpoints for accelerated approval sets a precedent for future HD treatments.

UniQure's discussions with the FDA have paved the way for accelerated approval of its gene therapy AMT-130 for Huntington's disease, potentially saving five years.

The FDA agreed with uniQure that the cUHDRS can serve as an intermediate clinical endpoint for AMT-130's BLA for HD, potentially accelerating approval. Reductions in CSF NfL levels may also support therapeutic benefit. AMT-130, a gene therapy, shows promise in slowing HD progression in ongoing trials.

uniQure's (QURE) stock surged 111% to $15.43 after announcing an FDA Accelerated Approval pathway agreement for its Huntington's disease gene therapy, AMT-130. The agreement leverages ongoing Phase I/II data, accelerating the BLA process and highlighting the robustness of uniQure's clinical data. Raymond James upgraded QURE to Strong Buy, reflecting investor confidence in the company's potential to bring a new therapy to market for Huntington's disease.