Un­der a short as­sault, Cor­bus coun­ter­at­tacks with pos­i­tive PhII da­ta for sys­temic scle­ro­sis drug

Yu­val Co­hen, Cor­bus CEO

Cor­bus Phar­ma­ceu­ti­cals says that its sole clin­i­cal-stage drug came through in a small Phase II study for sys­temic scle­ro­sis, an au­toim­mune dis­ease of con­nec­tive tis­sue, with the pa­tients tak­ing the treat­ment post­ing a sig­nif­i­cant re­sponse com­pared to the place­bo arm.

Us­ing a com­pos­ite score to gauge a re­sponse on a slate of key mea­sures like lung func­tion, the com­bined drug arm in­clud­ing pa­tients on a range of dos­es of re­sunab (JBT-101) post­ed a me­di­an 33% CRISS score af­ter 16 weeks of ther­a­py, com­pared to 0% in the place­bo group. And that marks a sig­nif­i­cant ben­e­fit for pa­tients, says the biotech, with a p-val­ue of 0.044 — not a great num­ber, but with­in the mar­gin need­ed to de­clare a win.

The tri­al list­ing on clin­i­cal­tri­als.gov, though, does list 12-week re­sults as the mark­er for the pri­ma­ry end­point, when the CRISS score was a low­er 27.5%. Al­so, one pa­tient had to drop out of the drug arm af­ter com­plain­ing of dizzi­ness. CEO Yu­val Co­hen tells me that any CRISS score over 20% would be “med­ical­ly mean­ing­ful.”

“The p=0.044 isn’t for week 16,” he adds in an email. “It is a com­par­i­son be­tween the drug and place­bo group across the en­tire study.”

Shares of Cor­bus rock­et­ed up 95% this morn­ing in pre-mar­ket trad­ing as in­vestors’ tem­pera­ments changed dra­mat­i­cal­ly on their first take of the da­ta.

Nor­wood, MA-based Cor­bus $CRBP had been on a roll in the lead up to the tri­al re­sults, with its shares ris­ing steadi­ly right up to the end of Oc­to­ber, when TheStreet’s Adam Feuer­stein ran a col­umn pre­sent­ing an anony­mous short at­tack that was in cir­cu­la­tion, spec­u­lat­ing the re­sunab was un­like­ly to demon­strate an ef­fect in Phase II. The drug has bounced around for years among dif­fer­ent com­pa­nies and the da­ta is “weak and in­con­sis­tent.” And sim­i­lar drugs that tar­get the CB2 re­cep­tor have failed as well.

That mes­sage res­onat­ed with in­vestors, and Cor­bus’ shares cratered in the days af­ter the Feuer­stein col­umn was post­ed, drop­ping 40%.

On Mon­day, in­ves­ti­ga­tors and com­pa­ny ex­ecs were push­ing back with a much dif­fer­ent mes­sage on the study, which in­clud­ed a to­tal of 42 pa­tients in both arms.

“This is the first dou­ble-blind, ran­dom­ized, place­bo-con­trolled tri­al in dif­fuse cu­ta­neous sys­temic scle­ro­sis to demon­strate a clin­i­cal ben­e­fit us­ing the CRISS as an end­point, with a drug that was safe and well tol­er­at­ed. These re­sults bring hope to pa­tients and their physi­cians that JBT-101 may be an ef­fec­tive drug for sys­temic scle­ro­sis where cur­rent­ly there are no proven treat­ments,” said Robert Spiera, the prin­ci­pal in­ves­ti­ga­tor and di­rec­tor of the Vas­culi­tis and Scle­ro­der­ma Pro­gram at the Hos­pi­tal for Spe­cial Surgery, Weill Cor­nell Med­ical Col­lege.

A one-year ex­ten­sion study is now un­der way to test safe­ty and long-term dura­bil­i­ty of the drug as the com­pa­ny looks at “next steps” with reg­u­la­tors.

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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