QURE - uniQure inks licensing deal for gene therapy candidate targeting ALS
- Dutch biotech uniQure N.V. ( NASDAQ: QURE ) traded higher pre-market Tuesday in reaction to a licensing agreement with Apic Bio for APB-102, a gene therapy targeted at a rare form of neurodegenerative disorder, amyotrophic lateral sclerosis (ALS).
- The FDA has already cleared an investigational new drug (IND) application for APB-102, a one-time gene therapy targeting ALS caused by mutations in SOD1. uniQure ( QURE ) expects to begin a Phase I/II clinical study for APB-102 in H2 2023.
- Per the terms, UniQure ( QURE ) will pay $10M upfront to Apic Bio in addition to milestone payments worth $45M subject to U.S. and EU regulatory approvals for APB-102 and the achievement of pre-specified annual net sales targets.
- The arrangement also provides tiered royalties ranging from mid-single digits to low double digits on net sales.
- The deal comes as Biogen ( BIIB ) and Ionis Pharma ( IONS ) await FDA approval for their SOD1-targeting ALS therapy tofersen in Q1 2023.
- In December, Seeking Alpha contributor Zach Bristow issued uniQure ( QURE ) a Buy rating and a $31.50 per share target.
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uniQure inks licensing deal for gene therapy candidate targeting ALS