Rare Daily Staff

Astellas Pharma said it will buy Audentes Therapeutics for $3 billion in order to take a major position in gene therapies for rare diseases.  

The deal will give the Japanese biopharma a pipeline of experimental gene therapies for rare neuromuscular diseases including its lead candidate AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM), a serious, life-threatening, rare neuromuscular disease affecting infants and young boys that is characterized by extreme muscle weakness, respiratory failure, and early death.

“Recent scientific and technological advances in genetic medicine have advanced the potential to deliver unprecedented and sustained value to patients, and even to curing diseases with a single intervention,” said Kenji Yasukawa, president and CEO, Astellas. “By joining together with Audentes’ talented team, we are establishing a leading position in the field of gene therapy with the goal of addressing the unmet needs of patients living with serious, rare diseases.”

Audentes is leveraging AAV gene therapy technology platform and proprietary manufacturing expertise to develop programs across three modalities: gene replacement, vectorized exon skipping, and vectorized RNA knockdown. Its product candidates are showing promising therapeutic profiles in clinical and preclinical studies across a range of neuromuscular diseases.

Under the terms of their agreement, Astellas will acquire Audentes at a price of $60.00 per share in cash, which represents a premium of 110 percent to its closing stock price ahead of the announcement. The premium is less than what Roche offered for Spark Therapeutics almost a year ago, a deal that has been stymied by regulators and has yet to close. It also follows a spate of acquisitions in the gene therapy space over the past year as positive trial data continues to grow and large biopharmaceutical companies jockey to stake positions in the space.

Audentes’ experimental gene therapy XLMTM delivers a functional copy of the MTM1 gene that is dysregulated in the disease. In a study of 10 infants who treated with the gene therapy, all were able to sit, stand, and walk, and saw a reduction in breathing support after treatment. Astellas believes AT132 could be submitted for approval by mid-2020.

Astellas says the acquisition will form a foundation for a new research focus area centered on genetic regulation. Besides access to Audentes’ neuromuscular development expertise, a pipeline of preclinical candidates for rare disorders, such as Duchenne muscular dystrophy and Pompe disease, it also acquires an in-house large-scale manufacturing facility, creating the opportunity for additional gene therapy partnerships. Audentes will remain an independent subsidiary.

“With its focus on innovative science and a global network of research, development and commercialization resources, we believe that operating as part of the Astellas organization optimally positions us to advance our pipeline programs and serve our patients,” said Matthew Patterson, chairman and CEO of Audentes.

Photo: Matthew Patterson, CEO of Audentes

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