Celtaxsys spins 'clin­i­cal­ly mean­ing­ful' da­ta on an­oth­er failed PhII cys­tic fi­bro­sis study

At first glance, you would think At­lanta-based Celtaxsys had plen­ty to be hap­py about in its re­lease about their Phase II study of an ex­per­i­men­tal drug for cys­tic fi­bro­sis called ace­bilu­s­tat. The an­ti-in­flam­ma­to­ry drug had pro­duced the first “clin­i­cal­ly mean­ing­ful” ev­i­dence of its po­ten­tial to re­duce pul­monary ex­ac­er­ba­tions in pa­tients.

That must be worth a cel­e­bra­tion, right?

But if you look at it close­ly, the da­ta — such as they are — leave a lot to be de­sired.

First, the study was a fail­ure. The pri­ma­ry ef­fi­ca­cy end­point in the 200-pa­tient study was a clas­sic mea­sure of FEV1 from base­line — forced ex­pi­ra­to­ry vol­ume in 1 sec­ond — as out­lined in clin­i­cal­tri­als.gov. If you dig in­to their state­ment, you’ll find there was no dif­fer­ence in lung func­tion be­tween the drug and the con­trol arm.

So what about those “clin­i­cal­ly mean­ing­ful” ben­e­fits for pa­tients on pul­monary ex­ac­er­ba­tions? 

The com­pa­ny said there was a “19% re­duc­tion in PEx and a 22% re­duced risk in pro­gress­ing to first PEx ver­sus place­bo.” And in sub­group analy­sis, some pa­tients did even bet­ter. The pul­monary ex­ac­er­ba­tion re­sults were based on a per pro­to­col ba­sis as op­posed to in­tent-to-treat. 

I asked a spokesper­son for the com­pa­ny what p-val­ues the com­pa­ny had nailed down for these sec­ondary end­points. She replied that da­ta were be­ing held back for a fu­ture con­fer­ence (at least the part that wasn’t in the re­lease), then not­ed that the sec­ondary end­points on pul­monary ex­ac­er­ba­tions were not pow­ered suf­fi­cient­ly to de­ter­mine sta­tis­ti­cal sig­nif­i­cance.

OK. I replied. But the com­pa­ny had those p-val­ues, right? So what were they and could it be trans­lat­ed to sig­nif­i­cance?

Here’s what I got:

The re­duc­tion in the rate of pul­monary ex­ac­er­ba­tions and in­creased time to first PEx re­port­ed are nu­mer­ic re­duc­tions for pa­tients treat­ed with ace­bilu­s­tat, not sta­tis­ti­cal­ly sig­nif­i­cant.

For con­text, the mag­ni­tude and con­sis­ten­cy of the ef­fects across our key, pre-spec­i­fied sub­groups (i.e. mild lung dis­ease, con­comi­tant CFTR mod­u­la­tor ther­a­py) has been char­ac­ter­ized by the CF Foun­da­tion, who have ex­pressed con­tin­ued sup­port of our P3 pro­gram, as clin­i­cal­ly mean­ing­ful.

So you have a failed study with no hard ev­i­dence of a clear drug ben­e­fit for pa­tients, now be­ing steered in­to Phase III. The Reuters re­port help­ful­ly not­ed that the com­pa­ny is look­ing to raise funds some­how, though com­pa­ny ex­ecs haven’t ex­act­ly de­ter­mined how.

There’s been con­sid­er­able at­ten­tion on cys­tic fi­bro­sis as Ver­tex con­tin­ues to dom­i­nate the space and move for­ward with promis­ing com­bo stud­ies. A host of ri­vals have ap­peared, in­clud­ing Gala­pa­gos, but no one has pro­vid­ed any re­cent da­ta to sup­port one of these oth­er con­tenders.

Celtaxsys didn’t change that pic­ture to­day, ei­ther.

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

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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.

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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.

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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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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.