Spark Therapeutics on track to file gene therapy in 2017
- Spark CEO Jeffrey Marrazzo expressed his excitement over the company's Phase 3 trial results during an investor's call this week, noting the pivotal part of this trial was the "first successful, randomized, controlled, Phase 3 trial ever completed for a gene therapy for a genetic disease."
- Spark Therapeutics announced its lead gene-therapy candidate voretigene neparvovec, in development for treatment of inherited retinal disease (IRD), was successful in a late-stage trial. Spark's development program for voretigene neparvovec has benefited from being designated by the Food and Drug Administration as a Breakthrough Therapy and an Orphan Drug.
- The success of voretigene neparvovec in improving retinal and visual function in patients with IRD bolstered Marrazzo's confidence that Spark could become the first company ever to submit a BLA to the Food and Drug Administration for a gene therapy aimed at treating an inherited disease.
Despite uneventful earnings, Marrazzo had a lot of positive news to bring to investors during the second quarter earnings call.
Not only did Spark's lead gene therapy hit its primary endpoint of bilateral mobility testing, but eight out of nine patients showed the "maximum improvement measurable" on the primary endpoint when measured after one year. The therapy hit two out of three secondary endpoints, but missed on visual acuity. The gene therapy is on track to be filed with the FDA in early 2017.
The company also had a positive turn for its SPK-FIX program, which it is working on with Pfizer.
Last month, the companies received a Breakthrough Therapy Designation for SPK-9001, which is being developed for treatment of hemophilia B. Spark's partnership with Pfizer dates back to late 2014, and includes milestone payments.
Spark's stock rose on the news, underscoring the market's positive reception to the news about voretigene neparvovec.
Spark is applying its proprietary gene therapy platform to a broad range of development programs, in addition to inherited retinal diseases. The company is also focused on liver-associated and neurodegenerative diseases, and is starting to see some traction with its preclinical hemophilia A program.
- Saprk Therapeutics Statement