Japanese pharma company, Shionogi, is set to acquire IntraBio in a multibillion-dollar deal, adding neurological and movement disorder drug, Aqneursa (levacetylleucine), to its portfolio and marking its fortification in the rare disease space.

As per the transaction, Shionogi will pay $2bn to acquire all of IntraBio’s outstanding shares, which will see the company secure the worldwide IP and commercialisation rights to Aqneursa – a small molecule drug that acts by addressing metabolic dysfunction in the central nervous system (CNS).

Discover B2B Marketing That Performs

Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.

Find out more

IntraBio will become a wholly owned subsidiary of Shionogi Inc., a New Jersey-based subsidiary of Shionogi.

Aqneursa was first approved by US regulators to treat neurological manifestations of a rare lysosomal disorder called Niemann Pick disease type C (NPC), which is characterised by the gradual buildup of harmful lipids in the cells, in 2024. Since then, the drug has also secured the EU regulatory greenlight in NPC, as well as the US Food and Drug Administration (FDA) go-ahead in rare genetic movement disorder, ataxia-telangiectasia (A-T). EU regulators are currently reviewing an approval application for Aqneursa in A-T.

By adding Aqneursa to its portfolio of medicines, Shionogi would double down on its new-found intentions to break into the rare disease space. This build on its $2.5bn acquisition of Tanabe Pharma’s amyotrophic lateral sclerosis (ALS) medication, Radicava (edaravone), which came earlier this year.

The Japanese drugmaker also hopes to leverage IntraBio’s expertise in the rare disease space to fortify its current pipeline, which includes drugs for conditions with high unmet needs such as Fragile X syndrome, Pompe disease and Jordan’s syndrome, alongside its early-stage programmes for neurodegenerative diseases.

According to Nathan McCutcheon, President and CEO of Shionogi Inc, bringing Aqneursa to the company would help it to continue its expansion into the rare disease market across Europe, the US and further afield, building on the “meaningful foundation IntraBio has established with the NPC community,” while delivering the first and only therapy for A-T.

“In addition to Aqneursa, the capabilities and resources gained through this acquisition would help to accelerate the development of new treatment options. With this transaction, we deepen our commitment to serving rare disease communities today and delivering innovation tomorrow,” McCutcheon concluded.