UniQure cashes out $10M upfront to get candidate for inherited ALS

Gene therapy player uniQure has signed a new agreement with Apic Bio for its ALS gene therapy.

The biotech said Tuesday morning that it now has a global licensing deal with Apic for APB-102, a gene therapy candidate for a rare form of the disease called superoxide dismutase 1 amyotrophic lateral sclerosis, or SOD1 ALS.

SOD1 ALS is the same disease subset Biogen’s drug tofersen is attempting to treat. Biogen submitted tofersen for accelerated approval last year.

As part of the agreement, uniQure now has global rights to both development and commercialization of the therapy.

The biotech said in its statement that it will be paying Apic Bio $10 million as an initial payment, and will pay Apic up to another $45 million in milestone payments, depending on regulatory approvals in Europe and the US and reaching pre-specified sales numbers — plus royalties.

Richard Porter

UniQure added that the therapy, currently given both orphan drug and fast track designation by the FDA, has been cleared to start clinical trials. UniQure CBO Richard Porter tells Endpoints News that the goal is to get the therapy in a Phase I/II trial by later this year.

“I think that all the building blocks are in place, we just need to fill in some of the gaps now,” Porter said, highlighting that there is some stuff to finish in terms of contracting with trial sites and other items to start the trial.

The therapy works by using a vector to express a microRNA, which is designed to knock down expression of SOD1 — and the hopeful result would be to either slow down or even reverse progression of ALS in these specific patients. Porter added that the modality of APB-102 is aligned with uniQure’s approach and strategy in other neurological disorders.

Mutations of SOD1, a gene that provides the body with instructions on how to make an enzyme called superoxide dismutase, are responsible for around 20% of all inherited forms of the disease, uniQure said in a statement. Around 5-10% of ALS is inherited.

The deal with Apic comes just two months after Big Pharma partner Bristol Myers Squibb scrapped its gene therapy partnership with uniQure. The focus of that collaboration was cardiovascular conditions, as the companies started working together back in 2015.

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