uniQure N.V. and Apic Bio announced that they have entered into a global licensing agreement for APB-102 to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS. Under the agreement, uniQure acquires global rights for the development and commercialization of APB-102, adding to its pipeline of gene therapies to treat neurological disorders. The U.S. Food and Drug Administration has cleared the investigational new drug (IND) application for APB-102 and has granted Orphan Drug and Fast Track designations.

APB-102 is designed to be a novel, one-time, intrathecally administered gene therapy for ALS caused by mutations in SOD1, a rapidly progressing, rare motor neuron disease that leads to loss of everyday functions and is uniformly fatal. Mutations in the SOD1 gene of ALS account for approximately one-fifth of all inherited forms of this fatal disease(1) . APB-102 is comprised of a recombinant AAVrh10 vector that expresses a micro ribonucleic acid (miRNA) designed to knock down the expression of SOD1 with the goal of slowing down or potentially reversing the progression of ALS in patients with SOD1 mutations.

The clinical development of APB-102 is based on nearly 30 years of research demonstrating the link between the SOD1 gene mutation and ALS. Preclinical studies in a SOD1-ALS mouse model demonstrated that APB-102 greatly enhanced survival in affected mice. Relevant SOD1 reduction in spinal cord motor neurons also was demonstrated in rodents, as well as in non-human primates at proposed clinical doses.

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 achievement of regulatory approvals in the U.S. and Europe and pre- specified annual net sales, and a tiered royalty on net sales ranging from the mid-single digits to low double digits.SVB Securities acted as exclusive financial advisor to uniQure in the transaction.Torreya Partners acted as exclusive financial advisor to Apic Bio in the transaction.