Ricardo Dolmetsch, president of R&D, uniQure/courtesy of uniQure

uniQure inked a deal with Apic Bio Tuesday to gain development and commercialization rights for APB-102, a therapeutic intended to treat a rare, genetic form of ALS.

APB-102 is designed to be a one-time gene therapy for patients whose ALS is caused by mutations in the superoxide dismutase 1 (SOD1) gene. This represents about 20% of the familial ALS population and 2% of all ALS patients.

The therapy utilizes a recombinant AAVrh10 vector that expresses a microribonucleic acid (miRNA). This, in turn, reduces the expression of SOD1 to slow or reverse ALS progression.

The therapeutic is “strategically aligned” with uniQure’s current pipeline, said Ricardo Dolmetsch, Ph.D., president of research and development in a statement issued Tuesday. He emphasized that APB-102 is “highly complementary” to AMT-161, a gene therapy currently in preclinical development to treat ALS caused by mutations in the c9orf72 gene.

APB-102 will now be known as AMT-162, and Dolmetsch said uniQure expects to begin a Phase I/II trial studying the gene therapy in the latter half of 2023.

Targeting Familial ALS

AMT-161 could become the first therapy approved for a genetic cause of ALS - but only if Biogen and Ionis’ tofersen doesn’t get there first.

The FDA’s Peripheral and Central Nervous System Drugs advisory committee will meet on March 22nd to discuss tofersen, an experimental therapy being developed for SOD1-ALS.

As BioSpace previously reported, the adcomm will vote on the potential accelerated approval of the candidate.

Unlike uniQure’s newly acquired candidate, tofersen is an anti-sense therapeutic that aims to reduce the synthesis of SOD1 protein production. Biogen submitted for approval largely on the basis of neurofilament light chain (NfL) as a surrogate biomarker.

The journey to this point has been rocky, which could point to a similarly difficult path ahead for uniQure’s AMT-162.

In October 2021, Biogen released data that showed tofersen failed to meet the primary endpoint in the Phase III VALOR trial. Despite this, the FDA accepted the company’s New Drug Application and granted the drug priority review.

In an effort to defend the candidate’s potential efficacy, Biogen published a review in September 2022 that included additional data and analyses of the VALOR trial and open-label extension study. The longer-term data showed tofersen was effective in slowing the decline of the disease.

The PDUFA date for tofersen is set for April 25.

There are only three FDA-approved treatments for ALS - the most recent being Amylyx’s Relyvrio, greenlit in September 2022.