Japan-based Shionogi & Co. announced that an agreement has been reached to acquire Austin, Texas-based IntraBio Inc. for USD 2.0 billion upfront, targeting the approved rare-neurology therapy Aqneursa (levacetylleucine) to a growing US and European rare disease business.
Under the agreement signed October 5, 2026, Shionogi's US subsidiary will acquire all outstanding shares of IntraBio, with closing expected in November or December subject to regulatory clearances. The transaction gives Shionogi global intellectual property and commercialization rights to Aqneursa.
Aqneursa was approved by the US FDA in September 2024 for neurological manifestations of Niemann-Pick disease type C and authorized in the EU in January 2026. The FDA added a second indication on September 18, 2026, approving the drug for ataxia in adults and pediatric patients with ataxia-telangiectasia, making it the first approved treatment for ataxia in that rare inherited disorder. An EU review in ataxia-telangiectasia is ongoing.
Levacetylleucine is intended to improve cellular energy metabolism and mitochondrial function, although its specific molecular target in Niemann-Pick disease type C and ataxia-telangiectasia remains unknown. IntraBio is also preparing a pivotal Phase III study in CACNA1A-related disorders.