‘Uncertainty in our minds’

Bio-tech company working on drug to reverse paralysis stymied by shutdown

NEW HAVEN — After years of scientific study, a New Haven company is on the verge of going to clinical trials to prove it has a drug that will reverse paralysis.

But that breakthrough is being stymied by a different type of paralysis at the White House and in the Congress during the struggle to find a way to reopen the government and allow this study and others to advance.

Erika R. Smith, CEO of ReNetX Bio, Inc., on Wednesday made her case to the state’s two U.S. Sens., Chris Murphy and Richard Blumenthal, who came to her office on Church Street.

Smith said the groundbreaking work of Dr. Stephen Strittmatter at the Yale School of Medicine has evolved to a new drug, Axer 204, that has shown it is possible to regrow the neural connection between the brain and the spinal cord.

She said the company needs the approval of the U.S. Food and Drug Administration to advance to clinical trials for humans after successfully showing nerve regrowth in animals in work that started in the early 2000s.

Smith said 41 percent of the FDA is shut down, including those offices that would rule on a new investigational drug. She said it was her understanding that projects in the pipeline would continue.

The normal turnaround to get approval to proceed is 30 days. Smith expected to submit their application in April and start the trials in May, but it was uncertain how the backlog due to the shutdown will push that forward.

She predicted that once they start, in two years they should be able to prove the safety and efficacy of Axer 204.

Smith told the senators that all these considerations are internal, but serving the patients suffering from paralysis, “is the broad piece we are concerned about.” She said the company hears from hundreds of patients asking for progress on the drug.

Smith said, if needed, the company would move the trials outside the country, although they haven’t starting planning for such a scenario.

“I am not going to let this technology not be successful. We have to figure out a way to make that happen, and if we can’t do it in the path we have set forward, then we are going to have to make other plans,” she told the senators.

Neither senator had high hopes that budget votes in the Senate on Wednesday to reopen the government would be successful.

Murphy said his worry is that if they give in to President Donald Trump “in this hostage taking, the president will just shut down the government over and over again just to get what he wants.”

The government shutdown, which has been going on for more than a month, started after Trump declined to sign a budget until he gets $5.7 billion toward a wall along the southern border with Mexico.

Murphy said he is concerned about the effect of an extended shutdown on the overall economy.

“If investors are all of a sudden faced with regular FDA shutdowns, I imagine they will look for other places to put their money. It is risky enough,” for investors to underwrite pharmaceutical advances, Murphy said.

Blumenthal said the shutdown is not just hurting the 800,000 federal workers furloughed or working without pay.

Like Murphy he feared the ripple effect.

“In Connecticut, biotech is part of our DNA. We don’t have gold or diamond mines. We have smart people who are inventing things,” Blumenthal said. “When life-saving drugs can’t come to patients, it’s unconscionable.”

An animated Murphy said, “What does this company have to do with building a wall?” He said it makes no sense to stop funding the FDA that approves drugs and inspects food safety.

Smith said the company, as of now, is facing practical issues. She said the material to be used in the trials has a shelf life; there are contracts with the clinic sites in place and as a small firm, they have to make sure they have the funds to continue to pay staff.

“Every day that this goes on, puts uncertainty in our minds,” she said.

There are around 300,000 people in the United States with chronic spinal cord injuries, Smith said, with about 17,000 new injuries occurring every year.

She said they tend to be very young men who are injured in accidents or playing sports. Smith said beyond the obvious personal cost to individuals, there is a broader cost of millions per patient in medical care and loss of jobs.

Smith said her company has “the utmost confidence” they will be successful if they can keep moving ahead.

She said, depending on the extent of the spinal injury, using the new drug could mean an individual will be able to use a muscle that was impossible before. That could be as fundamental as being able to dress or feed oneself.

“This is something that truly, on all levels, could be incredibly valuable,” Smith said. Now, only physical therapy is available to those who are injured, and that is not a cure.