sarepta excitement...from Endpoints19 Jun 2018 17:07
Sarepta $SRPT has leaped out into the front of the race to develop a once-and-done gene therapy for Duchenne muscular dystrophy.
With their R&D day in full swing Tuesday, company execs and the senior researcher on their groundbreaking human gene therapy study posted a first look at the promising results seen in three young patients who received treatments in a small study with no control which will involve a total of 12 boys. The data are very early, but the immediate response seen in the first few months has inspired the company to believe that it is on the right track to get a new therapy to these patients in as little as two years — provided all the stars align in its favor.
“If you were me, looking under the microscope, you would be so amazed you wouldn’t sleep at night,” says Jerry Mendell, the noted gene therapy expert at Nationwide Children’s Hospital in Columbus, OH who is leading the project.
CEO Doug Ingram called the results a “home run” during a preview of today’s presentation. Looking over the timeline, Ingram told me that it was conceivable that they would be ready to roll with a therapy in 24 months, but that would require a near perfect execution of the plan, with no nasty surprises.
That doesn’t happen very often in biotech. But they’re trying. Hard.
Baird’s Brian Skorney called it “amazing,” and the market agreed. The stock, which has been climbing north steadily over the past few months, rocketed up 56%.
The main goal of this first human study is safety, but investigators also tracked how each of the first three boys in a cohort of six boys aged 4 to 7 responded based on some well understood biomarkers. And while the numbers represent only three patients, small numbers and initial results like this are key in gene therapy — with the caveat that there’s a ton of additional work that needs to be done to prove that the benefits are life-changing for the boys and durable for years to come.
The first picture looks stellar, easily outstripping expectations.
Here’s what Mendell has been losing sleep over, for good reasons:
• Mean gene expression, as measured by percentage of micro-dystrophin positive fibers was 76.2% and the mean intensity of the fibers was 74.5% compared to normal control. Breaking that down on a patient-by-patient basis the percentages on micro-dystrophin expression in muscle fibers demonstrated some variability, ranging from a low of 59% to 83%, while the percentage of dystrophin-positive fibers fell in a more narrow band of 73.5% to 78%.
• Biopsies run by investigators showed a mean level of micro-dystrophin of at least 36.5%, ranging up using a different measure.
• Levels of creatine kinase in blood — an enzyme that leaks out of damaged muscles which is used to diagnose the disease — plunged by a mean of 87%
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