InVivo Therapeutics (OTC:NVIV) hopes to hear back from the FDA within four weeks about its application to run the first human trials of its biodegradable scaffold for treating traumatic spinal cord injuries, according to CEO Frank Reynolds.
"We’ve gotten back, we think, a really positive letter from the FDA. We’ve actually answered all of the questions in it now and we’re sending it back to FDA for the scheduling of our meeting, which we expect to take place hopefully in the next four weeks," Reynolds told analysts on a conference call today. "We could have that [approval] in the next six, seven, eight months – we’ll have to see what the FDA gives us as a timeline."
It was the Cambridge, Mass.-based firm’s first live update since it took itself public with a reverse merger in October 2010. InVivo filed the investigational device exemption application in July, seeking permission from the federal watchdog agency for a 10-patient trial of the device.
The scaffold is designed to protect and support spinal tissue and prevent secondary injury, including inflammation and glial scarring, following traumatic spinal cord injury. Trial subjects would join the study within a day or so of being injured, Reynolds said today.
The proposed study would follow patients for a year after implantation with the device at centers in Boston, Washington, D.C., and Pennsylvania, he said. The subjects would then be sent to Atlanta following the implantation procedure for rehabilitation.
The study’s main endpoint is safety data, according to InVivo medical director Dr. Jonathan Slotkin, who noted that secondary endpoints include restoration of bowel and bladder function and recovery of both sensory and motor function. The aim is to provide recoverry two vertebrae below the site of injury, Reynolds added.
InVivo is also preparing data from a primate trial of the bioscaffold, he said during the call, hoping for publication in a peer-reviewed journal after this month.
"Our team is the first team ever to get a monkey to benefit after being paralyzed," Reynolds claimed. "We proved that back in 2008."
That’s significant because the jump from proving the technology in one primate to proving it in another is less daunting than the jump from technology that works in rodents and in primates, he explained.
"If you look at the animal data, it’s all very impressive stuff," Zacks Investment Research analyst Jason Napodano told MassDevice. "I believe what they say when they say the leap is between rodents and primates, but things have a tendency to work in preclinicals that don’t always work in clinical studies."
The nature of the spinal cord injury in the primate trial is significantly different than th typical traumatic spinal cord injury, Napodano added. Human spinal cord injuries are usually crushing injuries, unlike the clean, surgical incisions of the spinal cord employed in the monkey trial.
"That’s what presents the risk in the human trial. The animal data looks fantastic and will be peer-review published hopefully later this year, and you’ll see how fantastic it looks," he said. "It’s the nature of the injury that creates the additional risk."
That said, it seems that the company has set reasonable goals in its trial proposal, Napodano said.
"They don’t have to take somebody who’s completely paralyzed and get them walking for it to be a success," he said. "Even if your legs are still paralyzed, if you can operate a wheelchair with your fingertips, your life is a whole lot better than before. So far it sounds like a well-designed trial. I don’t think this will be a huge hurdle they’re going after."
Should the FDA see fit to allow the trial to begin, Reynolds said during the call, the company plans to pursue a humanitarian device exemption that would essentially designate the scaffold as an "orphan" product.
"We’re hoping to have that designation soon," he said. "It gives market exclusivity for about seven years. We’ll be able to do about 4,000 patients a year while we continue to do a larger clinical study. That would lead to a full market, open market application of the technology."