J&J-part­nered MeiraGTx aims at Bio­gen with new eye gene ther­a­py da­ta

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.”

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

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Anap­tys­Bio's etokimab pro­vides more dis­ap­point­ing re­sults, rais­ing ques­tions about com­pound's fu­ture

The lead program for AnaptysBio’s in-house pipeline has hit another setback.

Etokimab, an IL-33 inhibitor, did not achieve statistically significant improvement in a Phase II trial for patients suffering from chronic rhinosinusitis with nasal polyps. Researchers measured the individuals’ bilateral nasal polyps score and sino-nasal outcome test, finding that neither improved upon a placebo after both four- and eight-week time markers, though they did demonstrate improvement over baseline levels of the examinations.

Brian Stuglik, Verastem CEO

The du­velis­ib hot pota­to is tossed to a new own­er as Ve­rastem looks to re­or­ga­nize around the pipeline

When Infinity put up duvelisib for a no-money-down instant deal, the biotech was looking for a quick exit from a clinical disaster. AbbVie had walked away from their alliance after looking at how the data stacked up in a crowded field.

And while it was approvable, it wasn’t looking pretty to anyone who thought in commercial terms.

One Big Pharma’s trash, though, was seen as a biotech treasure as a deeply troubled Verastem stepped up to grab the PI3K-delta/gamma — promising to run it across the goal lines at the FDA. And they did just that, only with little to show for it.

Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Vi­da Ven­tures co-leads Dyne's $115M megaround for next-gen oli­go ther­a­pies aimed square­ly at mus­cles

Dyne Therapeutics started out last April with a modest $50 million to mine targeted muscle disease therapies from its in-house conjugate technology. The biotech has now convinced more investors that it’s got gems on its hands, closing $115 million in fresh financing to push its next-gen oligonucleotide drugs into the clinic.

Vida Ventures and Surveyor Capital led the round, joined by a group of other new backers including Wellington Management Company, Logos Capital and Franklin Templeton.

Eli Lil­ly teams with Pieris on HER2+ tu­mors; Op­di­vo + Yer­voy best chemo in mesothe­lioma

Despite the FDA putting a partial clinical hold on its lead program only a few weeks ago, Boston-based Pieris Pharmaceuticals is plowing forward with a new collaboration.

Pieris will work with Eli Lilly to further advance studies on PRS-343, a 4-1BB/HER2 bispecific for HER2-positive tumors, in combination with the latter’s ramucirumab and paclitaxel for the second-line treatment of patients with HER2-positive gastric cancer in a single-arm, Phase II study.

Gallia Levy, Spark CMO (Roche)

Spark Ther­a­peu­tics nabs new CMO from Genen­tech, fill­ing a ma­jor post-merg­er de­par­ture

Spark Therapeutics is getting a new CMO from their new owners.

The gene therapy company-turned-subsidiary has named Gallia Levy, who had been running rare blood disorders — including clinical development for their blockbuster-potential hemophilia antibody Hemlibra for Roche’s big biotech sub Genentech — to run medical affairs.

The appointment is a fitting one. Roche spent $4.8 billion to acquire Spark last year in large part to get their hands on their hemophilia gene therapy, SPK-8011, and expand the toe-hold Hemlibra gave them in an crowded hemophilia space.  It’s also a somewhat ironic appointment: The FTC held up the Spark acquistions for nearly a year, reportedly over concerns about the anti-trust implications of Roche owning both a top chronic treatment in Hemlibra and a top one-time treatment in Spark’s gene therapy.

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