In wake of Eli Lil­ly set­back, Mer­ck KGaA's lead­ing BTK drug for au­toim­mune dis­ease clears PhI­Ib hur­dle

What kind of fu­ture awaits Bru­ton’s ty­ro­sine ki­nase drugs in au­toim­mune con­di­tions?

A fa­mil­iar play­er in hema­tol­ogy, which at­tract­ed the first big round of in­vest­ment for BTK drugs like ibru­ti­nib that can hit back at ab­nor­mal B cells, re­searchers have been ex­plor­ing un­chart­ed av­enues of drug de­vel­op­ment for au­toim­mune dis­eases. And the po­ten­tial leader in the field, Mer­ck KGaA’s evo­bru­ti­nib, just cleared a big hur­dle in the clin­ic that just might lay the foun­da­tion for a piv­otal ef­fort around mul­ti­ple scle­ro­sis.

We just don’t know for sure ex­act­ly what hap­pened.

Lu­ciano Ros­set­ti, Mer­ck KGaA

Mer­ck KGaA put out one of the least de­tailed state­ments you’ll ever see for a Phase IIb study, say­ing the drug did hit the pri­ma­ry end­point. But even though their top ex­ecs are stay­ing mum about ex­act­ly what they found, they’re al­so clear­ly proud about what they have ac­com­plished so far.

“It’s our own drug, one of our ma­jor achieve­ments,” com­pa­ny R&D chief Lu­ciano Ros­set­ti tells me.

This is a first for the field, he adds, with clear da­ta to prove that a Bru­ton’s ty­ro­sine ki­nase in­hibitor can work in treat­ing MS. The drug clear­ly hit on two of three dos­es with sta­tis­ti­cal­ly sig­nif­i­cant da­ta, he says, demon­strat­ed by track­ing le­sions pe­ri­od­i­cal­ly up to week 24 for the pri­ma­ry end­point.

Just how big an achieve­ment it is, though, we don’t know. The com­pa­ny is keep­ing the da­ta un­der tight wraps for now, and there’s no au­to­mat­ic de­ci­sion in place to move on in­to a big, pricey piv­otal pro­gram.

BTK in­hibitors for au­toim­mune con­di­tions have at­tract­ed some deep in­ter­est among the top play­ers in the field. Sanofi struck an $805 mil­lion deal — with a $40 mil­lion up­front — to line up a pact with Prin­cip­ia on a pre­clin­i­cal pro­gram for PRN2246 last fall. But Eli Lil­ly just wrote off a mid-stage BTK drug for rheuma­toid arthri­tis that it in-li­censed from Han­mi. Cel­gene lost in­ter­est in CC-292 af­ter their BTK drug per­formed poor­ly in Phase IIa. Bris­tol-My­ers is push­ing ahead with BMS-986142, mean­while, while Ab­b­Vie has AB­BV-105 in ear­ly-stage de­vel­op­ment.

In ad­di­tion to the drug arm, Ros­set­ti says, re­searchers al­so tracked a place­bo group as well as a Tec­fidera arm. And in a com­pet­i­tive field like this, there are plen­ty of con­sid­er­a­tions that have to be re­viewed about the mar­ket wor­thi­ness of a drug like this.

“We wish to see the com­plete da­ta in­for­ma­tion,” says the R&D chief. And even though the pri­ma­ry end­point was set for 24 weeks, they’re al­so gath­er­ing 48 week da­ta as well. In ad­di­tion, the drug is al­so in tri­als for lu­pus and rheuma­toid arthri­tis, with read­outs ex­pect­ed lat­er for the oth­er two in­di­ca­tions.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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