- After a string of gene-therapy related failures in clinical trials, recent results from a pivotal trial evaluating SPK-RPE65 therapy from Spark Therapeutics in patients with inherited night blindness were positive.
- The study met its primary endpoints, which included ability to navigate a mobility course under a variety of light levels. The results, which were statistically significant, demonstrated true functional improvement.
- Spark Therapeutics floated at $161 million IPO in January. The stock was up 40% on the news on Monday.
It's been a long, tough ride for gene therapy in the U.S., and it's not over yet. The first gene therapy has yet to be approved by the FDA.
But at this point, it's safe to call Spark Therapeutics the comeback kid for this therapeutic space. For more than 10 years, researchers at the Children's Hospital of Philadelphia (CHOP) have been researching the use of gene therapy for various eye disorders. At one point, the CHOP researchers found that previously blind patients regained vision when they delivered gene therapy virally to the eyes, instructing the eyes to begin producing healthy copies of the RPE65 gene. However, three years later, they started to lose vision again.
This was just one failure of many, including a recent disappointment for Celladon's MYDICAR treatment for heart failure in April and Avalanche Biotechnologies' failure of a wet age-related macular degeneration (AMD) therapy in June. And although there had been a resurgence of market interest in companies with a stake in the gene therapy arena, each failure dampened enthusiam, contributing in part to a 20% decline in biotech stocks in the third quarter.
But, every day is a new day, and Spark is planning to continue its development program, with the hope of filing by the end of 2016. There are roughly 3,500 REP65 patients in the U.S. and the five major E.U. nations; however, Spark sees the potential for broadening the potential patient pool by also testing the treatment in other inherited retinal diseases.