Today Hawthorne, NY-based Acorda Therapeutics (NASDAQ: ACOR) announced that it licensed an experimental compound to treat spinal cord injury from medical-device giant Medtronic (NYSE: MDT). Acorda paid $3 million up front for worldwide rights to the compound, and pledged up to $32 million in regulatory and development milestone payments to Medtronic, which is based in Minneapolis. Medtronic will also receive a “single digit” percentage royalty if the product is commercialized, Acorda says.

The Medtronic deal is part of Acorda’s grand plan to build up its Phase 2 pipeline of drugs to address unmet medical needs in neurology. The fast-growing company already has two marketed products—dalfampridine (Ampyra) to treat multiple sclerosis, and tizanidine hydrochloride (Zanaflex) to relieve muscle spasms—plus one drug in Phase 1 clinical testing and two more on the way. “But we have nothing in the middle,” said CEO Ron Cohen in a phone interview this spring, when he hinted the company was on the hunt for molecules to acquire. “To diversify our risk and leverage our core expertise—neurological therapeutics—we feel we need to have middle-stage projects.”

The Medtronic compound, which Acorda will call AC105, is a modified form of the mineral magnesium. It has long been known that a loss of magnesium after spinal cord injuries creates a cascade of problems that lead to the loss of motor function that commonly results. “If you can restore magnesium to proper levels, you can protect the tissue” in the brain and spinal cord, says Acorda’s chief scientific officer, Andrew Blight.

Problem is, it’s difficult to give magnesium to patients in high enough levels without causing dangerous side effects, such as heart and brain issues. So Medtronic created a formulation of the mineral that includes polyethylene glycol. That improves the process of getting magnesium from the blood to the injured nervous system, but without the need for the large magnesium doses that raise the risk for serious side effects. In rodent models of spinal cord injury, the drug promoted recovery of some movement to paralyzed limbs and performed better than simple magnesium salt formulations that have been tried in the past.

There are about 12,000 incidents of spinal cord injuries in the U.S. every year. But Blight believes that AC105 may prove useful in other neurological applications, too, such as traumatic brain injury. “Anything that’s useful in spinal cord injury is likely to be useful in other conditions, as well,” he says. Medtronic has already done a Phase 1 safety study in healthy volunteers, so Acorda is picking up the project in time to plan the Phase 2 program in spinal cord injury.

Acorda is also moving forward on compounds in its early-stage pipeline—including one that may take the company beyond its neurology comfort zone. Glial Growth Factor 2 (GGF2) is a “neuregulin”—a molecule that stimulates tissue repair in the nervous system. Acorda licensed GGF2 from a company called CeNeS Pharmaceuticals (now owned by Paion) in 2002. Since then, Acorda has been examining the compound in animal models of multiple sclerosis, Parkinson’s disease, and stroke.

In the course of those studies, Acorda’s scientists recognized that GGF2 was showing promise in cardiology. “Neuregulins are potent stimulators of cardiomyocytes—the muscle cells in the heart,” says Anthony Caggiano, V.P. of research and development for Acorda. “What’s exciting for us is … Next Page »

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Biotech executives were on the prowl a couple weeks ago in San Francisco, many of them urgently seeking tens or hundreds of millions of dollars from Wall Street. But one of the fun, offbeat ideas I encountered during my trip was from a Kalamazoo, MI, entrepreneur seeking to nail down his first $5 million for a startup called Axonia Medical. This company is betting it can treat nerve damage in arms and legs without touching an embryonic stem cell.

Axonia’s president and founder, Harry Ledebur, told me about his dream for this regenerative medicine startup during a break between meetings with investors at the JP Morgan Healthcare Conference. He’s a molecular and cell biologist by training, so he knows the difference between an axon and neuron (more on that later). But he also has an unusual personal experience battling the problem his company is seeking to solve, as someone who has lost a leg because of a peripheral nerve injury.

If Axonia can raise the cash it seeks, the company will be off and running with a new approach to regenerative medicine. Several companies, like Menlo Park, CA-based Geron (NASDAQ: GERN) have made headlines with efforts to treat spinal cord injuries through embryonic stem cell-based approaches to regenerate new nerve cells. But these approaches are still controversial, because of the way embryonic stem cells are obtained. Axonia is seeking to help reconnect the wiring of the central nervous system a different way, essentially by using a laboratory device to grow and stretch out the key cells—known as axons—and then implanting them in an injured limb.

If these implants can take hold and spur growth of new axons in clinical trials—still a very big if—then Axonia could someday offer a new treatment strategy for the 50,000 to 100,000 people in the U.S. who undergo surgery for moderate to severe nerve damage in their arms and legs each year. Eventually, it could also be a part of combination treatment for spinal cord injury, traumatic brain injury, and stroke, Ledebur says. Axonia’s investor prospectus says the market opportunity for peripheral nerve injury alone—the most likely use of the technology at first—is $1.2 billion a year.

“This is one of the coolest technologies I’ve seen in a long time,” Ledebur says. “It’s a business opportunity that could be transformative.”

Harry Ledebur

Ledebur found the idea for Axonia while sifting through more than 2,000 different biotechnology proposals from universities and research centers, as part of his work as an executive in residence for Southwest Michigan First, a Kalamazoo-based economic development organization that seeks to recruit and build companies to the region. The Axonia technology, developed in the lab of Douglas Smith at the University of Pennsylvania, stood out for him.

The concept, as the name suggests, is all about axons. These are the long, thin cells that act as extensions of neurons, carrying electrical signals throughout the body. While humans might be able to naturally regenerate a lot of oxygen-carrying blood cells after they get knocked down by cancer chemotherapy, axons aren’t nearly so resilient, which isn’t news to anyone who has suffered a spinal cord injury or a nerve damage to an arm or leg, like combat veterans.

Scientists have long struggled to find ways to even grow these long unwieldy axons in a conventional cell culture in a lab. They can grow to be as long as 30 meters … Next Page »

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Cambridge, MA-based InVivo Therapeutics said today it has completed a reverse merger and is conducting a $10.5 million private placement financing in part to fund a human study of its implant to treat spinal cord injuries. The firm did the reverse merger with an entity called InVivo Therapeutics Holdings, and it will be quoted on the OTC market under the symbol “NVIV” starting October 28. Frank Reynolds, who founded the company with technology from the MIT lab of inventor Bob Langer in 2005, is continuing to serve as CEO of the company. Reynolds, who suffered a spinal cord injury himself before starting the company, shared InVivo’s story with Xconomy in December 2008.

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On March 2, I reported to Suffolk County Superior Court for jury duty, certain that I’d be let go after my day of service or excused, just like every other time. So it was a bit of a shock to find myself seated, by the end of the day, as Juror No. 14 on a medical malpractice trial that, according to the judge’s prediction, would take until March 23. (She was exactly right, as it turned out.)

I won’t dwell here on the irony of being forced to spend most of Mass Mobile Month immobile in the jury box, separated from my laptop and unable to use my cell phone. Being part of the 14-member jury on a three-week civil trial was no more of a hardship for me than it was for the other jurors, so I’m not going to complain. But I do want to share a few observations from the experience—some encouraging, some not. I’ll try to restrict myself mainly to talking about subjects related to technology and medicine, which were the big themes in the case.

The trial, in a nutshell, was about a health emergency that went undiagnosed far too long. The plaintiffs were an elderly church pastor from a Boston suburb and his wife. (I’m not going to use their names.) The pastor had a history of back trouble, but nothing incapacitating. The weekend before Thanksgiving in 2003, he began to experience unbearable back pain, and was taken to the ER of a local hospital (which I also will not name—if you want to go dig up the details, I’m sure there are public records).

Doctors there quickly determined that the pastor had a streptococcal infection in his bloodstream and started him on the appropriate antibiotics. They began a series of tests intended to locate the source of the infection and the pain. But the agony continued, and it wasn’t until five days later, after the pastor had been transferred to a prominent Boston hospital, that its true source was discovered.

An imaging study showed that the infection had taken root in the pastor’s spine in the form of a large epidural abscess, a pocket of pus inside the spinal canal between the bone and the dura, the outer lining of the spinal cord. As soon as the abscess was detected, surgeons operated to drain the pus. But by then it was too late. The abscess had pinched off the pastor’s spinal cord, causing permanent nerve damage. The pastor, now 75 years old, can’t walk on his own and suffers from a range of other disabilities.

The plaintiffs’ attorney was an outstanding Boston trial lawyer and medical malpractice specialist named Gregg Pasquale, of Keches Law Group. I’ve since learned that in the 1980s Pasquale was an assistant district attorney in Middlesex County, where he prosecuted murder cases. His fiery zeal was evident every day in our courtroom. Pasquale argued that the defendants in the case—a radiologist from the suburban hospital and two doctors from the Boston hospital—should have done more to diagnose the pastor’s problem.

One of the many difficulties in the pastor’s case was that the abscess didn’t appear on the standard MRI exam ordered by the ER doctor at the suburban hospital. But Pasquale argued that … Next Page »

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Frank Reynolds, the CEO of Cambridge, MA-based InVivo Therapeutics, says he still feels pain in his lower back when he steps off a curb. The pain stems from the spinal cord injury Reynolds suffered in an auto accident in 1992. It’s no coincidence that today he is running a medical technology firm with an implant that could prevent some cases of paralysis.

This week InVivo launched a second study to test its polymer-based device—implanted at the site of the spinal cord injury—in monkeys, Reynolds says. If successful, the study could provide enough evidence to get the “green light” from the FDA to begin human testing next year. Reynolds also aims to raise $15 million in a Series A financing round in the next few months to fund the company through human clinical trials and the cost of a factory to mass-produce the device. (Thus far, the company has raised nearly $3 million from family and friends.)

“The rest of the world is focused on regeneration.” Reynolds says. “We take a different path than the rest of the world.”

Initially developed in the lab of prolific MIT inventor Bob Langer—a scientific advisor of InVivo—the company’s fingertip-sized device is intended to be implanted within days of a spinal cord injury to limit tissue damage, or secondary injuries, that most often cause patients to become paralyzed. The device is made of biodegradable polymers engineered to dissolve in the body in weeks. Reynolds says the fabrication and use of the materials and not the materials themselves make the device unique.

Today’s limited options to treat spinal cord injuries include stabilizing patients’ bodies to prevent further damage, anti-inflammatory drugs, and, if needed, surgeries to decompress injured areas and to remove bone fragments.

With mixed results, many experimental treatments for spinal injuries have focused on treating damaged neurons. Hopkinton, MA-based Alseres Pharmaceuticals (NASDAQ:ALSE), for example, is developing a … Next Page »

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