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UniQure Raises $10M Upfront to Get Hereditary ALS Candidate – Endpoints News

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 agreement 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 subset of diseases that Biogen’s tofersen drug is attempting to treat. Biogen submitted tofersen for accelerated approval last year.

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

Biotech said in its filing that it will pay Apic Bio $10 million as an upfront payment and pay Apic up to an additional $45 million in milestone payments, depending on regulatory approvals in Europe and the United States and hitting pre-agreed sales numbers. more royalties.

Richard Porter

UniQure added that the therapy, currently designated as both an orphan drug and fast track by the FDA, has been cleared to begin clinical trials. Richard Porter, CBO of UniQure, tells Endpoint news that the goal is to get the therapy in a Phase I/II trial later this year.

“I think all the building blocks are in place, we just need to fill some of the gaps now,” Porter said, noting that there are some things to finish in terms of test site contracts and other items to start the process.

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

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

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

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