Sev­er­al months af­ter Bio­gen tout­ed the first proof-of-con­cept da­ta from its $800 mil­lion eye gene ther­a­py, MeiraGTx and J&J are out with their own ear­ly da­ta for a ri­val treat­ment.

In a small Phase I/II tri­al for their gene ther­a­py for the de­gen­er­a­tive eye dis­ease x-linked re­tini­tis pig­men­tosa, MeiraGTx found that the 7 pa­tients in the two low­est dose co­horts saw their vi­sion im­prove on mul­ti­ple mea­sures for 6 months. The study will pro­vide the ba­sis for the New York biotech to move in­to a piv­otal Phase III for the gene ther­a­py that J&J paid $100 mil­lion to get in on last year.

Jim List

“It’s re­al­ly ex­cit­ing da­ta,” Jim List, Janssen’s glob­al ther­a­peu­tic head for car­dio­vas­cu­lar and meta­bol­ic dis­ease, told End­points News. “This shows the po­ten­tial of the ther­a­py to have an im­prove­ment in vi­sion from this treat­ment, but al­so for this to slow or halt the pro­gres­sion of the dis­ease, and that would be amaz­ing.”

The re­tini­tis pro­gram is MeiraGTx’s lead pro­gram and one of two ma­jor gene ther­a­py plays for the dis­or­der, which is gen­er­al­ly di­ag­nosed in ado­les­cence and caus­es slow de­te­ri­o­ra­tion in vi­sion for years af­ter. Bio­gen spent $877 mil­lion last year to ac­quire Night­star Ther­a­peu­tics and their gene ther­a­py pro­gram.

In Jan­u­ary, Bio­gen pub­lished a sim­i­lar set of Phase I/II da­ta for Night­star’s treat­ment in Na­ture Med­i­cine, al­so show­ing that the drug was safe and that a sub­set of pa­tients saw im­prove­ment that was sus­tained over 3 to 6 months.

Like Bio­gen, MeiraGTx al­so found though that a sub­set of pa­tients ex­pe­ri­enced an in­flam­ma­to­ry re­sponse. Al­though that re­sponse was quick­ly re­solved with oral steroids, it ap­peared to in­ter­fere with the ef­fec­tive­ness of the drug. Pa­tients on the high­est dose of MeiraGTx’s tri­al did not see a strong im­prove­ment, and the biotech will con­duct their Phase III with the low and in­ter­me­di­ate dose.

The 4 pa­tients on the in­ter­me­di­ate dose arms saw a reti­nal sen­si­tiv­i­ty in­crease of 1.02 db, and an in­crease of cen­tral vi­su­al field pro­gres­sion rate at 1.26 db/year. Cen­tral vi­su­al field pro­gres­sion rate im­proved 1.10 db/year for the low dos­es. For the high dose, both of those num­bers were neg­a­tive.

Michel Michaelides

Michel Michaelides, lead in­ves­ti­ga­tor and MeiraGTx’s sci­en­tif­ic founder, said that Bio­gen did not look at some of the core mea­sures of vi­sion that they did. He al­so point­ed to the dif­fer­ence in im­prove­ments be­tween eyes. On­ly one of each pa­tients’ eyes re­ceived ther­a­py – but cau­tioned that cross-tri­al com­par­isons were in­her­ent­ly fraught.

“I’m not sure they have such a clear dis­par­i­ty be­tween treat­ed and un­treat­ed,” he said.

Michaelides al­so said pa­tients re­port­ed im­proved vi­sion, greater clar­i­ty, greater ease track­ing signs and read­ing on a com­put­er. There’s no place­bo-con­trol for those met­rics yet and that will have to wait for Phase II. Still, Michaelides said the ear­li­er tri­als have al­ready start­ed chang­ing sci­en­tists’ goals.

“It wasn’t long ago where the am­bi­tion for de­gen­er­a­tive dis­eases was to slow halt pro­gres­sion, and what we’re show­ing here is im­prove­ment,” he said. “That’s huge for pa­tients.”

Two weeks after joining Verastem Oncology as chief medical officer, Frank Neumann is leaving the company for another job.

Neumann had joined Verastem after leaving bluebird bio, which surprisingly split into two companies last week, one in oncology and one in rare diseases. It’s not yet clear to where Neumann is headed next, but he noted in a statement that Verastem’s data and strategy were “truly exciting.”

A month after filing the IND application for its human extracellular matrix designed to regenerate knee cartilage, Histogen has hit a roadblock.

The FDA on Tuesday verbally notified the San Diego-based biotech that it was placing a clinical hold on the planned Phase I/II clinical trial of HST-003 due to pending CMC information and additional questions needed to complete their review.

Histogen had planned to test the safety and efficacy of implanting hECM within microfracture interstices and related cartilage defects to regenerate that cartilage in conjunction with a microfracture procedure. The company said in a press release that it expects to receive written notice of the clinical hold from the FDA by Feb 12.

Gritstone Oncology has had a big week, and it’s only Wednesday.

On Tuesday, the biotech revealed plans to start clinical testing of an experimental Covid-19 vaccine — in tandem with NIAID — that can also target other coronaviruses, with the goal of preventing future pandemics should SARS-CoV-2 prove difficult to cure with current vaccines. Then, on Wednesday morning, Gritstone licensed lipid nanoparticle technology from Genevant Sciences to develop what it’s calling “self-amplifying RNA vaccines” against Covid-19.

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

The past two months have been tough for Bristol Myers Squibb and its checkpoint inhibitor Opdivo after setbacks in lung and brain cancers. But in the battle against Merck’s Keytruda, any success matters — and now Bristol could be looking at a quick approval for Opdivo in an unmatched indication.

The FDA will launch a speedy review of a combination of Bristol Myers Squibb’s Opdivo and chemotherapy to treat first-line patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer or esophageal adenocarcinoma, the drugmaker said Wednesday. The agency set an action date of May 25 for the application.