Blue­bird’s gene ther­a­py shows promise in keep­ing lethal Loren­zo’s Oil dis­ease in check

Blue­bird Bio CEO Nick Leschly Im­age: Get­ty

Af­ter an av­er­age of 29 months fol­low­ing a one-time gene ther­a­py in­fu­sion, a clear ma­jor­i­ty of boys suf­fer­ing from lethal cas­es of cere­bral adrenoleukody­s­tro­phy were alive and free of ma­jor com­pli­ca­tions from the dis­ease — the pri­ma­ry end­point in the Phase II/III study spon­sored by blue­bird bio $BLUE.

Pub­lish­ing in the New Eng­land Jour­nal of Med­i­cine, in­ves­ti­ga­tors at Dana-Far­ber, Boston Chil­dren’s Hos­pi­tal and Mass Gen­er­al re­cruit­ed 17 pa­tients with this dis­ease, mak­ing it one of the largest gene ther­a­py stud­ies now un­der­way. And they con­clud­ed that at this in­ter­im stage they have good ev­i­dence of dis­ease sta­bil­i­ty for this very vul­ner­a­ble group, with 88% still alive and com­pli­ca­tion free.

The pub­li­ca­tion am­pli­fies the ini­tial in­ter­im re­sults post­ed last sum­mer, with blue­bird not­ing that they’ll be adding more re­sults at an up­com­ing sci­en­tif­ic con­fer­ence.

One of the pa­tients died from the dis­ease — pop­u­lar­ly known as Loren­zo’s Oil dis­ease — and an­oth­er has suf­fered se­vere and rapid dis­ease pro­gres­sion.

That all still meets blue­bird’s pre­de­fined bench­mark for suc­cess, which was pegged at 76%.

There was one se­vere ad­verse event linked to the ther­a­py, vi­ral cys­ti­tis, but the in­ves­ti­ga­tors say that case was re­solved. And while ad­verse events were al­so at­trib­uted to the mye­loab­la­tive chemother­a­py used to prep pa­tients, there was no ev­i­dence of graft ver­sus host dis­ease or in­fec­tions from the ther­a­py.

Ac­cord­ing to the re­searchers, blue­bird ex­ecs are en­gaged in talks with reg­u­la­tors about get­ting the gene ther­a­py ap­proved for use.

Adri­an Thrash­er

“This da­ta we are see­ing from this study of gene ther­a­py is a re­al­ly ex­cit­ing de­vel­op­ment for boys with CALD. Not on­ly does the da­ta show that Lenti-D has the po­ten­tial to min­i­mize fur­ther pro­gres­sion of this dev­as­tat­ing dis­ease, but the tech­nique al­so avoids the need for a matched bone mar­row donor, which can be dif­fi­cult to find and can de­lay vi­tal treat­ment,” said Pro­fes­sor Adri­an Thrash­er, pe­di­atric im­mu­nol­o­gist at Great Or­mond Street Hos­pi­tal and co-in­ves­ti­ga­tor of the study. “The re­sults from this tri­al thus far are very en­cour­ag­ing and will pave the way for fur­ther de­vel­op­ment of state-of-the-art treat­ments for chil­dren with life-lim­it­ing con­di­tions.”

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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