UniQure In-Licenses Early-Stage Amyotrophic Lateral Sclerosis Candidate, Analyst Says Deal Compliments Its Pipeline

UniQure N.V. QURE and Apic Bio entered into a global licensing agreement for APB-102 to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).
Under the agreement, UniQure acquired global rights for developing and commercializing APB-102, adding to its pipeline of gene therapies to treat neurological disorders.
The FDA cleared the investigational new drug application for APB-102 and has granted Orphan Drug and Fast Track designations.
UniQure expects to initiate a Phase 1/2 clinical study of APB-102 in the second half of 2023.
Also See: FDA Approves UniQure's Hemgenix, First Hemophilia B Gene Therapy For Adults.
Under the terms of the agreement, UniQure will make an initial cash payment of $10 million. In addition, UniQure will pay Apic Bio up to $45 million in milestones upon achieving regulatory approvals in the U.S. and Europe, pre-specified annual net sales, and a tiered sales-based royalty.
Analyst Take: SVB Leerink says APB-102 is a good strategic fit for QURE's pipeline, particularly given their expertise with miRNA gene therapy development through their AMT-130 Huntington's disease program.
It also writes that APB-102 is complementary to QURE's internal AMT-161 gene therapy program for c9orf72 ALS and that it comes to QURE ready for the clinic to provide near-term catalysts.
While ALS is largely viewed as a high-risk/reward space for drug development, SVB appreciates that QURE is pursuing genetic-targeted approaches.
Price Action: QURE shares are up 3.51% at $21.37 on the last check Tuesday.

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