California-based Audentes is a clinical-stage company that focuses on developing adeno-associated virus (AAV)-based gene therapies for serious rare neuromuscular diseases. Astellas currently has four primary areas of focus: antigen-specific immunomodulation, immuno-oncology, mitochondrial function, and regenerative medicine. Therefore, the acquisition of Audentes will allow Astellas to expand into a fifth primary focus area: genetic regulation. The acquisition is expected to close in Q1 2020.
Audentes has one clinical-stage product in its pipeline, AT132, gene therapy for X-linked myotubular myopathy (XLMTM) that is currently in Phase II development. However, with strong trial results, fast track designation, and orphan drug designation in the US, as well as PRIME designation in the EU, Audentes has announced that it plans to file for approval in the US and the EU in 2020. The Phase I/II ASPIRO trial assessing AT132 has demonstrated its efficacy to increase neuromuscular function and reduce dependence on ventilators.
XLMTM is a life-threatening neuromuscular disease characterized by extreme muscle weakness, respiratory failure, and early death. Patients with XLMTM must rely on supportive care as there are no approved treatments for the indication. As such, the market entry of AT132 would fulfil a major unmet need. Despite this, AT132 is unlikely to be a large driver of sales growth for Astellas. XLMTM is a rare disease, affecting one in 40,000 to 50,000 live male births, so the commercial prospects for this gene therapy may be limited based on the small size of the patient population.
Astella’s key driver for this acquisition is the manufacturing capacity of Audentes, which will allow the quick advancement of various gene therapy programmes in the future, giving Astellas the opportunity to establish itself in the gene therapy field. Audentes also has several pre-clinical programs for a variety of neuromuscular diseases including Pompe disease and Duchenne muscular dystrophy. The manufacturing capacity of Audentes could also allow Astellas to expand its gene therapy research into other disease areas in the future.