Now with ac­tu­al da­ta in hand, Cel­gene touts an ear­ly re­sponse for Crohn’s drug mon­gersen

Scott Smith, Cel­gene

A month af­ter post­ing an un­der­whelm­ing top-line as­sess­ment of a Phase Ib study of its close­ly watched Crohn’s drug mon­gersen (GED-0301) for Crohn’s dis­ease, Cel­gene post­ed an up­date Sun­day evening that in­clud­ed a pos­i­tive snap­shot of the ac­tu­al da­ta.

In­ves­ti­ga­tors for the study say they were able to track a quick re­sponse to the drug, which Cel­gene paid a whop­ping $710 mil­lion in cash to in-li­cense the drug for Crohn’s. The re­searchers tracked the im­pact of 160 mg dos­es of the drug on en­do­scop­ic re­sponse and clin­i­cal re­mis­sion among 63 pa­tients with ac­tive Crohn’s dis­ease over short pe­ri­ods stretch­ing from 4 to 8 to 12 weeks. And they say the 12-week group has re­spond­ed with the most im­pres­sive re­sults — though some might ob­ject to what qual­i­fies as im­pres­sive at this stage of the game for such an ex­pen­sive ther­a­py.

The da­ta:

Clin­i­cal im­prove­ment was seen by week 2, and clin­i­cal re­sponse (CDAI de­crease ≥100) and re­mis­sion (CDAI <150) rates were high­est in the 12-week treat­ment group at 67 and 48 per­cent re­spec­tive­ly, at week 12. The mean CDAI re­duc­tion from base­line at week 12 in the 12-week treat­ment group was 133 points. Of the pa­tients with evalu­able en­do­scopies at week 12 (n=52), 37 per­cent had an en­do­scop­ic re­sponse (≥25 per­cent re­duc­tion in SES-CD score from base­line), with no mean­ing­ful dif­fer­ence across treat­ment groups. In ad­di­tion, of those pa­tients with greater en­do­scop­ic dis­ease ac­tiv­i­ty at base­line (SES-CD score of >12; n=16), 63 per­cent ex­hib­it­ed a re­duc­tion ≥25 per­cent in SES-CD score and 31 per­cent had a re­duc­tion of ≥50 per­cent.

The in­ves­ti­ga­tors tout­ed the re­sults be­ing post­ed at Unit­ed Eu­ro­pean Gas­troen­terol­o­gy Week in Vi­en­na as ev­i­dence of the kind of ef­fi­ca­cy that Cel­gene was look­ing for. An­a­lysts con­cen­trat­ed on that re­sponse rate and whether it qual­i­fies as the kind of re­sult they were look­ing for. And they can be a tough crowd.

Baird’s Bri­an Sko­r­ney gave a thumbs up to 48% re­mis­sion rate, but was unim­pressed by the 37% en­do­scop­ic re­sponse score. His bot­tom line:

This da­ta ap­pears to be more promis­ing than we ex­pect­ed but it is un­like­ly to dri­ve a ma­te­r­i­al in­crease in con­fi­dence. The en­doscopy re­sults fall short of what we have seen from oth­er stud­ies but the CDAI re­spons­es do ap­pear in line with oth­er agents.

Like­wise, Leerink’s Ge­of­frey Porges saw the po­ten­tial here for block­buster sales of $1.3 bil­lion in 2023 with the drug a like­ly win­ner on front­line ap­proval sta­tus. But there’s noth­ing here that sug­gests an out-of-the-park home run hit. And Cred­it Su­isse cau­tions that this is still ear­ly da­ta from a small study. Doc­tors don’t usu­al­ly do en­do­scopies at 12 weeks and in­ves­ti­ga­tors were re­al­ly look­ing for signs of ac­tiv­i­ty.

In­vestors liked the sounds of that, dri­ving up Cel­gene’s shares by 1.3% Mon­day af­ter­noon.

Add it all up and the drug still looks promis­ing though the ju­ry is def­i­nite­ly still out on what kind of mar­ket awaits GED-0301.

High­light­ed as a key pro­gram in its at­tempt to build a fran­chise for in­flam­ma­tion and im­munol­o­gy, GED-0301 is one of the com­pa­ny’s biggest ex­per­i­men­tal as­sets in the pipeline of the big biotech, which has struck a se­ries of bold deals to build its port­fo­lio of ex­per­i­men­tal ther­a­pies.  The drug is de­signed to slice Smad7 pro­tein lev­els. Spik­ing lev­els of Smad7 in­ter­fere with TGF-β1 an­ti-in­flam­ma­to­ry path­ways in the gut, spurring in­flam­ma­tion.

Cel­gene, though, was clear­ly hap­py to praise the re­sults.

“We are en­cour­aged that oral GED-0301 showed both mean­ing­ful en­do­scop­ic im­prove­ment and clin­i­cal re­mis­sion at an ear­ly time point in this study,” said Scott Smith, pres­i­dent of Cel­gene In­flam­ma­tion & Im­munol­o­gy, in a state­ment. “The fact that this study in­clud­ed near­ly 50 per­cent bi­o­log­ic-ex­pe­ri­enced pa­tients fur­ther re­flects the po­ten­tial of GED-0301 as a nov­el ap­proach for pa­tients with Crohn’s dis­ease search­ing for al­ter­na­tives.”

The big biotech al­so post­ed some up­dat­ed ozan­i­mod da­ta from a mid-stage pro­gram on ul­cer­a­tive col­i­tis, not­ing some fresh signs of ef­fi­ca­cy from the ex­ten­sion re­sults. The added da­ta were al­so re­leased in Vi­en­na.

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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