Shionogi Expands Rare Disease Portfolio via $2 Billion IntraBio Purchase

Shionogi & Co. is advancing its growth strategy by acquiring IntraBio, a biotechnology firm that adds a drug approved for two rare conditions together with expertise that complements Shionogi’s pipeline of neurological therapies for rare diseases.

The primary asset in the transaction is levacetylleucine, marketed as Aqneursa. The FDA first approved Aqneursa in 2024 for the neurological symptoms of Niemann‑Pick disease type C, an inherited enzyme deficiency, and it remains one of only two FDA‑approved treatments for this condition. In the previous month, the agency extended the label to cover ataxia‑telangiectasia (A‑T), marking the first therapy approved for this rare neurodegenerative disorder.

Based in Osaka, Japan, Shionogi bolstered its rare‑disease portfolio earlier this year with a $2.5 billion deal securing worldwide rights to Radicava, an amyotrophic lateral sclerosis medication developed by Tanabe Pharma. Its additional rare‑disease candidates also originate from similar acquisitions.

S‑606001, presently in mid‑stage development for Pompe disease, a rare lysosomal storage disorder, was licensed from Maze Therapeutics in 2024. Zatolmilast was acquired through Shionogi’s 2020 purchase of Tetra Therapeutics. In May, Shionogi reported that this oral small molecule did not meet efficacy endpoints in two Phase 3 trials for Fragile X syndrome, a genetic condition associated with intellectual disability and developmental delays, and a subsequent mid‑stage trial continues to assess it for Jordan syndrome, another rare neurodevelopmental disorder.

IntraBio, a privately held company, was established in 2015. Its scientific founders, drawn from the University of Oxford and the University of Munich, discovered and developed small molecules that modulate lysosomal function and intracellular calcium signaling. The firm originated in Oxford, United Kingdom, before moving to Austin, Texas, in 2024.

Aqneursa is a modified form of L‑leucine, an essential amino acid obtained from the diet and required for muscle repair and cellular energy production. The drug’s formulation is engineered to facilitate passage across cell membranes, including the blood‑brain barrier. Its label indicates that the mechanism of action for treating Niemann‑Pick disease and ataxia‑telangiectasia is currently unknown. Nevertheless, the company believes the agent could be applicable to a broad spectrum of rare and common neurological conditions, and it reported $67.8 million in revenue for fiscal year 2025.

Shionogi’s primary revenue stream derives from royalties on HIV therapeutics marketed by ViiV Healthcare, a majority‑owned subsidiary of GSK. Earlier this year, the company raised its minority interest in ViiV from 10% to 21.7%. These HIV products constitute a component of Shionogi’s overarching strategic plan, which targets growth milestones by 2030 and includes expansion into the global rare‑disease market.

In addition to its FDA approvals, Aqneursa received European Commission authorization earlier this year for Niemann‑Pick disease type C, and a separate European review is currently underway for ataxia‑telangiectasia. In a prepared statement, Shionogi CEO Isao Teshirogi said the IntraBio acquisition underscores the company’s commitment to establishing a global rare‑disease business.

“Integrating Aqneursa into Shionogi following our acquisition of Radicava will deepen our commitment to rare‑disease communities, broaden our capabilities, and reinforce our portfolio as we pursue future innovations for patients with substantial unmet needs,” he said.

The transaction remains subject to regulatory approval; Shionogi anticipates closing the deal by the end of the year.

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