BioPharma

Astellas dives into gene therapy with $3B acquisition of Audentes Therapeutics

Shares of Audentes rose more than 100% following news that the Japanese drugmaker would acquire it. The company's lead candidate is a gene therapy for a rare muscular weakness disorder, currently in a Phase I/II study for which it presented data in October.

A large Japanese drugmaker plans to buy up a U.S. company developing gene therapies for a genetic disease that causes muscle weakness.

Tokyo-based Astellas Pharma said Monday it would acquire San Francisco-based Audentes Therapeutics for about $3 billion, or $60 per share. Shares of the biotech company were up nearly 106% on the Nasdaq Tuesday morning following the announcement.

Under the deal, Audentes will operate as an independent subsidiary while having access to Astellas’ scientific and development resources.

“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,” Astellas CEO Kenji Yasukawa said in a statement.

Audentes’ lead product candidate is AT132, which is undergoing a Phase I/II study of 24 patients in X-linked myotubular myopathy, or XLMTM, a rare genetic disease that causes extreme muscle weakness, respiratory failure and death among 50% of patients in the first 18 months of life. The disease, which results from mutations in the MTM1 gene, affects about 1-in-40,000 to 50,000 newborn males, according to the company. AT132 is an adeno-associated viral vector therapy designed to insert into cells a functional copy of the MTM1 gene.

The company presented data from 12 patients in the Phase I/II study’s dose-escalation cohort in October at the World Muscle Society’s annual congress, while also stating that the pivotal cohort – stated in the ClinicalTrials.gov page to have a target enrollment of eight patients – is almost fully enrolled. Patients receiving the therapy had shown significant and durable reductions in dependence on ventilators, which is considered to be closely correlated with morbidity and mortality in XLMTM patients. The first seven patients treated – including six treated with 100 trillion vectors per kilogram of body weight and one treated at 300 trillion vectors – had achieved ventilator independence and the ability to stand up or walk.

Audentes is also developing therapies for Pompe disease, Duchenne muscular dystrophy (DMD) and myotonic dystrophy Type 1. An eight-patient Phase I/II clinical trial of AT845 in Pompe disease – a lysosomal storage disorder – was posted on the ClinicalTrials.gov database on Nov. 22, but hasn’t yet started recruiting patients. Sanofi’s Lumizyme (alglucosidase alfa) is an existing treatment for Pompe disease, while Spark Therapeutics has a gene therapy, SPK-3006, in Phase I/II development.

The Astellas-Audentes deal is one of several examples of large drugmakers acquiring gene therapy companies in recent years. Swiss drugmaker Roche said in February that it would acquire Spark – which markets the first FDA-approved gene therapy, Luxturna (voretigene neparvovec-rzyl), for a genetic form of blindness – for $4.8 billion, but that deal has hit regulatory snags. Novartis, also based in Switzerland, acquired AveXis for $8.7 billion in April 2018, winning approval earlier this year for the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy in May. In June, Vertex Pharmaceuticals spent $245 million to acquire Exonics Therapeutics, which is developing gene therapies for DMD. And in March, Biogen spent $800 million to acquire Nightstar Therapeutics, a company developing gene therapies in the ophthalmology space.

Photo: designer491, Getty Images

Shares1
Shares1