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Roche acquisition of Spark Therapeutics continues to build gene therapy market

Roche (SIX: RO, ROG; OTCQX: RHHBY) and Spark Therapeutics, Inc. (NASDAQ: ONCE) (“Spark”) recently announced the completion of the acquisition of the gene therapy company, following regulatory approval from government authorities. Roche’s $4.8 billion acquisition of Spark was a 64% increase from its initial $2.7 billion offer in what turned out to be a two-way bidding war for the gene therapy company. Spark Therapeutics will now continue its operations in Philadelphia as an independent company within Roche.

 

Spark Therapeutics was a spin-out of the Children’s Hospital of Philadelphia (CHOP), University of Philadelphia, which founded a gene therapy company to develop several drug candidates including Leber’s congenital amurosis (LCA), haemphelia and choroideremia.  Prior to the acquisition, the company submitted a Marketing Authorization Application (MAA) to the FDA and EMA (European Medicines Agency) for their investigational “LUXTURNA” (voretigene neparvovec), now approved.  The gene therapy treatment for LUXTURNA had three clinical trials comprising 41 patients in total, representing the first randomized, controlled Phase 3 trial of a gene therapy for a genetic disease.  Results of the clinical studies, previously reported in the medical journal, The Lancet, showed a statistically significant and clinically meaningful difference between intervention (n=21) and control participants (n=10) at one year and in the mean bilateral multi-luminance mobility testing (MLMT) change score (difference of 1.6; 95% CI, 0.72, 2.41; p=0.0013). According to the company, participants in the original Phase 3 intervention group maintained functional gains observed by the day-30 visit through at least two years, as measured by MLMT and full-field light sensitivity threshold (FST). Results from the study indicated a more than 100-fold (or > 2 log units) average improvement in FST testing observed in the original intervention group at one year which appear to be similarly maintained through at least two years.  The data were a key rationale to acquire the company.

 

Commenting on the acquisition, Severin Schwan, CEO of Roche, stated, “We are excited about this important milestone because we believe that together, Roche and Spark will be able to significantly improve the lives of patients through innovative gene therapies.  This acquisition supports our long-lasting commitment to bringing transformational therapies and innovative approaches to people around the world with serious diseases.”