|UPenn's Albert Maguire|
After repeatedly batting back doubts about its lead gene therapy, Spark Therapeutics says its pivotal study for SPK-RPE65, an FDA-designated "breakthrough" for sight-blighting inherited retinal dystrophies, came through with positive data. The biotech says that the treatment hit the primary and two of three secondary endpoints in the late-stage study, setting up a biologics license application at the FDA next year.
The biotech says that a group of patients in the small Phase III--which included a total of 31 patients who were growing blind--saw their vision improve after treatment with the gene therapy.
Spark's shares ($ONCE), which had been dented by doubts raised by other setbacks in the gene therapy, soared 60% on the news in pre-market trading. Spark was named a Fierce 15 company in 2014. But the lofty surge failed to stick for Spark. The rally lost much of its thrust by midmorning, with shares trading up 26%. Bluebird ($BLUE), another gene therapy stalwart, also picked up gains today as investors were encouraged to back other leaders in the gene therapy field.
"The majority of the subjects given SPK-RPE65 derived the maximum possible benefit that we could measure on the primary visual function test, and this impressive effect was confirmed by a parallel improvement in retinal sensitivity," noted Dr. Albert Maguire, principal investigator in the trial and professor of ophthalmology at the Perelman School of Medicine of the University of Pennsylvania.
Spark was spun out of Children's Hospital of Philadelphia (CHOP) with a pipeline of therapies developed by some notable researchers in the field, including Dr. Jean Bennett, at its Center for Cellular and Molecular Therapeutics. That instant rep helped inspire a $161 million IPO for the company as Spark took its place in the front ranks of a new wave of gene therapy companies.
Pfizer ($PFE) also stepped up to sign on for the first big partnership with Spark.
If Spark goes on to win approval of a pioneering application for a gene therapy, the company will help establish the reputations of a new group of upstarts that have set out to prove that a single treatment could possibly cure a variety of diseases. Spark would also provide a test case of how these therapies will be priced, and paid for, as analysts puzzle over the huge costs expected with gene therapy treatment. Many expect these treatments to be billed at $1 million, inspiring a discussion about an annual billing approach that could break up payments over a number of years.
These new treatments will also test just how long these so-called cures can last, with some evidence that early gains for patients can be lost over a period of years. As a result, there's new work in the clinic looking at technologies that could allow for repeat treatments.
- here's the release
Special Report: FierceBiotech's 2014 Fierce 15 - Spark Therapeutics