Astellas spends $3bn on a bold move into gene therapy

The Japanese group has seen potential in Audentes' manufacturing capabilities as well as its pipeline.


Astellas’s move on Audentes is the fourth acquisition of a gene therapy player this year. Though the $3bn price comes at a 110% premium to Audentes’ closing price yesterday it is fairly modest compared with the sums paid for other gene therapy developers. 

The deal is good news for the biopharma sector as it heads into a year where unpredictability caused by the forthcoming presidential election is making investors nervous. It also points up the ongoing appetite for Japanese pharmas for M&A. 

Audentes’ lead asset, AT132, is in phase II trials for the rare neuromuscular condition X-linked myotubular myopathy. The extreme muscle weakness caused by this condition limits patients’ ability to move, eat and breathe, and mortality rates are around 50% in the first 18 months of life, and a further 25% of patients die by the age of 10. It occurs in one in 40,000 to 50,000 live male births.

Patients have a mutated form of the gene myotubularin 1; the protein this produces is necessary for normal development and function of skeletal muscle cells. AT132 delivers a functional copy of this gene using an AAV8 vector. 

Filings next year

Audentes has only released data in seven patients treated with AT132 so far, and only one that has received the higher dose that looks likely to be filed with regulators. The phase II Aspiro trial AT132 achieved “significant and durable” reductions in ventilator dependence, which is considered to correlate with morbidity and mortality in XLMTM patients, Audentes said in October. 

AT132 is currently being tested in a pivotal expansion cohort to confirm safety and efficacy at the higher dose of 3 x 1014vg/kg, and Audentes had intended to file a BLA in mid-2020 with a European submission in the second half.

Sales of AT132 will reach $381m in 2024, according to EvaluatePharma’s sellside consensus, though this figure will be highly risk adjusted.


This deal looks to be largely driven by Audentes lead asset, but it is some of Audentes’ other programmes that might pose a bit of a risk to the acquisition. 

Audentes has three preclinical-stage oligonucleotides for Duchenne’s muscular dystrophy that might overlap with Astellas’s Duchenne’s candidate, ASP0367 (also called MA-0211), an oral small molecule in phase I. Astellas is also developing reldesemtiv, also called CK-2127107, in phase II for spinal muscular atrophy, which could have some similarity to AT132.

With the Roche/Spark deal still yet to close owing to the FTC’s review, and Bristol-Myers Squibb only getting Celgene after the latter group sold off its psoriasis blockbuster Otezla, it is not inconceivable that Astellas might attract the attention of antitrust regulators.

Audentes’ pipeline, however, is probably only part of its appeal. Astellas is probably as interested in Audentes’ AAV-based technology platform and in-house large-scale gene therapy manufacturing capabilities. It was this kind of thinking that led Thermo Fisher Scientific to buy Brammer Bio earlier this year.

Investors clearly believe that more gene therapy-based deals are coming. Uniqure and Regenxbio are trading up 17% and 7%, respectively, so far today. 

Audentes' pipeline
        Annual WW sales ($m)
Status Product Indication Summary First WW launch 2020e 2022e 2024e
Phase II AT132 Muscular dystrophy (XLMTM*) 2020 2 171 381
  AT342 Crigler-Najjar syndrome  2022   1 3
Phase I AT982 Pompe's disease 2024     18
Preclinical AT702 Duchenne muscular dystrophy        
  AT845 Pompe's disease        
  AT307 Arrhythmia, ventricular        
  AT751 Duchenne muscular dystrophy        
  AT753 Duchenne muscular dystrophy        
  AT466 Myotonic dystrophy        
  AT720 Other neurological indications        
*XLMTM=X-linked myotubular myopathy. Source: EvaluatePharma.

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