After snagging most expensive drug title, uniQure doles out $10M upfront for ALS prospect

Three months after scoring FDA approval for a hemophilia B therapy that is now the priciest drug in the world, uniQure is sealing a deal for its next big thing with $10 million upfront to Apic Bio for an amyotrophic lateral sclerosis (ALS) gene therapy. 

The deal, announced Tuesday, gives uniQure global development and commercialization rights to APB-102, a gene therapy for superoxide dismutase 1 ALS, a rarer form of the degenerative disease. In exchange, uniQure is paying $10 million in upfront cash plus $45 million in milestones. The deal thrusts uniQure into the race to treat the lesser-known version of ALS, which Ionis and Biogen are also targeting with tofersen. 

The Apic Bio pact is the first glimpse at uniQure’s larger business strategy since it nabbed FDA approval for the CSL Behring-partnered hemophilia B therapy Hemgenix in November 2022. The $3.5 million gene therapy is now the most expensive drug in the world. 

UniQure nabbed APB-102 for a fairly small price tag compared to other licensing deals from biopharmas in recent months but will have to shell out for the full clinical development process. The asset is currently in preclinical studies and received fast-track designation from the FDA in 2021. UniQure says preclinical models have found that APB-102 “greatly enhanced” survival in SOD1 ALS-affected mice, according to a release. The company did not specify when it intends to move the med into the clinic. 

Much will be gleaned about regulators' expectations for treating SOD1 ALS once Biogen and Ionis meet with FDA experts at the end of March to discuss tofersen. The FDA was originally slated to deliver a decision on approval by the end of this month but extended the review period after receiving more data from the sponsors. Tofersen failed to improve the functional status of SOD1 ALS patients in a phase 3 trial, but Biogen is hoping biomarker data from 12 months out and from an open-label extension trial will make a case for accelerated approval.