Pharmaceutical Business review

Roche to acquire gene therapy company Spark Therapeutics for $4.3bn

Image: Roche signs deal to acquire Spark Therapeutics. Photo: courtesy of F. Hoffmann-La Roche Ltd.

As per the terms of the merger agreement, Roche will buy each share of Spark Therapeutics at a price of $114.50.

Founded in 2013, Spark Therapeutics is engaged in discovery, development and delivery of gene therapies for genetic diseases such as blindness, haemophilia, neurodegenerative diseases and lysosomal storage disorders.

In December 2017, the company’s gene therapy Luxturna (voretigene neparvovec) was approved by the US Food and Drug Administration (FDA) for the treatment of biallelic RPE65 mutation-associated retinal dystrophy in children and adults.

Luxturna became the first gene therapy to be approved by the FDA for a genetic disease.

In less than a year after the FDA approval, the adeno-associated viral (AAV) vector gene therapy was approved by the European Commission for the same indication, which is a rare inherited form of vision loss that could result in blindness.

Currently the lead clinical asset of Spark Therapeutics is SPK-8011, which is being developed as a gene therapy for the treatment of haemophilia A, and is expected to be put through a phase 3 trial this year.

Another gene therapy in the making is SPK-8016, which is being evaluated by the company in a phase 1/2 trial in the haemophilia A inhibitor population.

Other clinical assets of the company are SPK-9001 as potential treatment of haemophilia B, which is in phase 3 development, and SPK-7001 for choroideremia, which is in phase ½ development.

The gene therapy company is also developing SPK-3006 for the treatment of Pompe disease and SPK-1001 for CLN2 disease, which is a type of Batten disease. Spark Therapeutics is expected to make both the gene therapies ready for clinical development this year apart from undertaking additional preclinical programs for Huntington’s disease and Stargardt disease.

Roche CEO Severin Schwan said: “Spark Therapeutics’ proven expertise in the entire gene therapy value chain may offer important new opportunities for the treatment of serious diseases.

“In particular, Spark Therapeutics’ haemophilia A programme could become a new therapeutic option for people living with this disease.”

The transaction is expected to be closed in the second quarter of this year, subject to meeting of certain customary conditions. Post-acquisition, Spark Therapeutics will continue its operations in Philadelphia as a standalone company within the Roche Group.

Spark Therapeutics CEO Jeffrey Marrazzo said: “With its worldwide reach and extensive resources, Roche will help us accelerate the development of more gene therapies for more patients for more diseases and further expedite our vision of a world where no life is limited by genetic disease.”