About a year ago, Howard Berman was sit­ting in a wait­ing room at Hous­ton Methodist Hos­pi­tal with his fa­ther, who was show­ing cog­ni­tive loss. They were there to see Joseph Mas­deu, one of the lead­ing neu­rol­o­gists in the area.

Howard Berman

“You know what?” Berman re­calls Mas­deu say­ing. “Why don’t you meet with Dr. Stan Ap­pel, he’s work­ing on some very in­no­v­a­tive work in the field of neu­rode­gen­er­a­tive dis­ease.”

And so last spring, Berman found him­self in a pri­vate meet­ing with Ap­pel, the 86-year-old co-di­rec­tor of Hous­ton Methodist’s Neu­rol­o­gy In­sti­tute, who was don­ning his icon­ic bow tie.

Ap­pel pre­sent­ed on his reg­u­la­to­ry T cell (Treg) re­search — and by the third slide, Berman was hooked. Not long af­ter, he quit his job at Ab­b­Vie and launched Coya Ther­a­peu­tics — named af­ter an is­land off Cos­ta Ri­ca — to de­vel­op Ap­pel’s Treg ap­proach for ALS.

On Wednes­day, Coya un­veiled a $10 mil­lion Se­ries A to ad­vance that pro­gram and build out a pipeline.

“We want to do for ALS what peo­ple did for HIV and AIDS,” Berman said: ex­tend pa­tients’ lives. ALS pa­tients typ­i­cal­ly live an av­er­age of three years af­ter di­ag­no­sis, he added.

Stan­ley Ap­pel

Ap­pel and his team ob­served that many ALS pa­tients have low lev­els of Treg cells, or ones that don’t func­tion prop­er­ly. Im­prov­ing the num­ber and func­tion of the Treg cells, they hy­poth­e­sized, just might slow the pro­gres­sion of dis­ease.

Many com­pa­nies have eyed Treg cell ther­a­pies in the last decade, but the rar­i­ty and plas­tic­i­ty of en­doge­nous Treg cells pose a sig­nif­i­cant chal­lenge.

Coya’s process be­gins with leuka­phere­sis, or draw­ing a pa­tient’s blood and iso­lat­ing the Treg cells. Then sci­en­tists add cer­tain types of agents — like ra­pamycin or IL-2 — de­signed to sta­bi­lize the Tregs and help them grow and func­tion prop­er­ly. Coya then ex­pands the cells, con­vert­ing them from mil­lions to bil­lions of Tregs, which are frozen for fu­ture use.

“What we found out is you can in­fuse the cells, (and) it stops pro­gres­sion for about a month… dur­ing the course of in­fu­sion. But when you stop the in­fu­sions, what hap­pens is the pa­tients start to de­cline again,” Berman said. “It’s not a one-shot deal. You need to be able to give it month­ly,” he added lat­er.

Their longest cry­op­re­served cells have been frozen for a year and a half. And so far, they’re still vi­able, ac­cord­ing to Berman.

Coya has al­ready con­duct­ed a Phase I tri­al, which “suc­cess­ful­ly demon­strat­ed… the safe­ty and tol­er­a­bil­i­ty of au­tol­o­gous in­fu­sions of ex­pand­ed Tregs in ALS pa­tients, with the po­ten­tial of slow­ing or halt­ing dis­ease pro­gres­sion,” Ap­pel said in a state­ment.

An on­go­ing Phase II tri­al with eight pa­tients should pro­duce da­ta this June or Ju­ly, Berman added.

“We will be work­ing with the FDA to de­sign an­oth­er tri­al that will re­al­ly pro­vide, hope­ful­ly, com­pelling da­ta to bring us in­to an ap­proval process,” he said.

The com­pa­ny is al­so go­ing af­ter Parkin­son’s, Alzheimer’s, fron­totem­po­ral de­men­tia and oth­er au­toim­mune dis­eases, as well as non-cell based ex­o­somes and small mol­e­cules.

“We’re not just a cell ther­a­py com­pa­ny per sé,” Berman said.

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.