‘Un­cer­tainty in our minds’

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

The News-Times - - NEWS - By Mary E. O’Leary mary.oleary@hearst­medi­act.com; 203-741-2577.

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.

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