Rigel’s 2nd PhI­II for fos­ta­ma­tinib scut­tled by a sin­gle place­bo re­sponse, shares crater

Rigel Phar­ma­ceu­ti­cals con­tin­ues to be plagued by a low re­sponse rate in its big Phase III pro­gram for fos­ta­ma­tinib. But un­like its first late-stage da­ta drop, which scored a hit on the pri­ma­ry end­point, the biotech now has to ex­plain why its sec­ond con­fir­ma­to­ry study failed.

Raul Ro­driguez, Rigel CEO

It’s not an en­vi­able task. At the end of Au­gust the South San Fran­cis­co-based biotech’s share price $RIGL went on a roller coast­er ride with a pos­i­tive but unim­pres­sive 18% re­sponse rate for im­mune throm­bo­cy­tope­nia pa­tients who achieved a sta­ble platelet re­sponse. That was ex­act­ly the same rate seen in the sec­ond Phase III, on­ly this time one pa­tient on a place­bo al­so achieved a sta­ble platelet re­sponse, and that scut­tled any shot at hit­ting the pri­ma­ry end­point. For an­a­lysts who had been look­ing for a re­sponse rate at least in the low 20s with clear signs of ef­fi­ca­cy, this stum­ble will not play well.

In­vestors didn’t care for news, ei­ther. Rigel’s shares plunged 33% in ear­ly trad­ing Thurs­day.

Rigel CEO Raul Ro­driguez, though, says if you add all the da­ta from both Phase III stud­ies to­geth­er, you still come up with a pos­i­tive out­come. And that’s what they want to take to the FDA.

“We be­lieve that the to­tal­i­ty and con­sis­ten­cy of da­ta from the FIT Phase III pro­gram, which in­clud­ed two Phase III stud­ies and one long-term ex­ten­sion study, strong­ly sup­ports a clear treat­ment ef­fect, with a sus­tained clin­i­cal ben­e­fit of fos­ta­ma­tinib,” said Ro­driguez in a state­ment.  “We are en­cour­aged by these re­sults and be­lieve that the risk/ben­e­fit ra­tio for fos­ta­ma­tinib is pos­i­tive for pa­tients with chron­ic/per­sis­tent ITP, a pop­u­la­tion with a se­ri­ous un­met med­ical need.  As a re­sult, we will con­tin­ue to pur­sue this op­por­tu­ni­ty. Our next step is to seek feed­back from the FDA.”

The ther­a­py is a spleen ty­ro­sine ki­nase (Syk) in­hibitor, which should help stop the bleed­ing that oc­curs when some­one has a low platelet count. But in a small proof-of-con­cept study for throm­bo­cy­tope­nia, the ther­a­py scored a 50% re­sponse rate, which had whet­ted ap­petites for some­thing sol­id in Phase III.

There’s no turn­ing back now, though. Rigel laid off close to half of its staff last month as it cleared the decks for an FDA ap­pli­ca­tion and launch plans.

Back in 2013 fos­ta­ma­tinib flopped in a Phase III study of rheuma­toid arthri­tis, forc­ing As­traZeneca to wash its hands of their part­ner­ship and leav­ing Rigel to un­der­go an over­haul, shed­ding staff and re­or­ga­niz­ing around dry eye dis­eases (R348) and lu­pus (R333). They both quick­ly failed Phase II. An­oth­er drug, R118, had to be aban­doned af­ter in­ves­ti­ga­tors tracked some se­ri­ous side ef­fects.

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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