Spark Therapeutics beats by $2.24, beats on revs
- Reports Q2 (Jun) earnings of $2.07 per share, $2.24 better than the Capital IQ Consensus of ($0.17); revenues rose 1601.7% year/year to $25.19 mln vs the $14.65 mln Capital IQ Consensus.
- 4.3 million was net sales of LUXTURNA and $20.9 million was associated with agreements with Pfizer.
- Second quarter results were favorably impacted by the sale of PRV to Jazz Pharmaceuticals for $110 million.
Spark Therapeutics gene therapy reduces bleeding in hemophilia A trial
- Spark Therapeutics says its experimental treatment for hemophilia A reduced bleeds and the need for therapeutic infusions by 97 percent in a trial of 12 patients.
- It plans to start a late-stage clinical trial in the fourth quarter.
- Wall Street was looking for a level of at least 30 percent to consider the trial a success, according to Evercore.
Spark Therapeutics, the biotechnology company focused on gene therapy, said Tuesday its experimental treatment for hemophilia A reduced bleeds and the need for therapeutic infusions by 97 percent in a trial of 12 patients. It plans to start a late-stage clinical trial in the fourth quarter.
At the highest dose in the study, which the company plans to use in its phase 3 trial, five of seven patients saw levels of the protein important in hemophilia A restored to an average of 30 percent of normal, the company said when it reported second-quarter financial results. Two patients, though, had an immune response that caused the level of that protein to decline to less than 5 percent.
Hemophilia is a rare disorder in which the blood doesn't clot properly. It's currently treated with drugs that replace proteins — called Factor VIII, for hemophilia A, and Factor IX, for the less common hemophilia B — known as clotting factors. About 20,000 people in the U.S. are estimated to have hemophilia, according to the Centers for Disease Control and Prevention.
New approaches, from Spark and competitors like BioMarin, aim to deliver healthy copies of the gene that makes the proteins important in hemophilia. The goal of what's called gene therapy is to dramatically improve, or even cure, the disease with just one treatment (Spark's stock ticker, accordingly, is ONCE).
"The totality of the data we're really pleased with and encouraged by, and it's driving our decision and plans to move into a phase 3 trial starting in the fourth quarter," Spark Chief Executive Officer Jeff Marrazzo told CNBC in a telephone interview.
Wall Street was looking for a level of 30 percent expression of Factor VIII or more to consider the trial a success, Evercore ISI analyst Steve Breazzano wrote in a July 30 research note, "provided that the results are not highly variable."
Spark's stock has gained more than 50 percent this year, following a massive drop in December on an earlier look at four patients in this clinical trial. After a presentation at the American Society of Hematology Conference (ASH) in Atlanta, Spark's stock dropped 35 percent in a single day as investors worried about a lack of uniformity in the results, and were more encouraged by an update from BioMarin.
