Roche gets pri­or­i­ty re­view for Hem­li­bra's sec­ond in­di­ca­tion; Ora­sis gets $13M B round for pres­by­opia eye drop

Roche $RHH­BY is now one step clos­er to re­al­iz­ing its block­buster am­bi­tions for Hem­li­bra, se­cur­ing a pri­or­i­ty re­view for a sec­ond, huge­ly im­por­tant in­di­ca­tion — he­mo­phil­ia A pa­tients who haven’t de­vel­oped re­sis­tance to fac­tor re­place­ment ther­a­py, more com­mon­ly known as pa­tients with­out fac­tor VI­II in­hibitors or non-in­hibitor pa­tients. The an­nounce­ment comes days af­ter the phar­ma gi­ant’s Genen­tech group rolled out a slate of new Phase III da­ta that backed Hem­li­bra’s case for re­duc­ing treat­ed bleeds: a 97% re­duc­tion com­pared with those with no pro­phy­lax­is and 68% com­pared to pre­vi­ous fac­tor VI­II treat­ment. Hem­li­bra was orig­i­nal­ly ap­proved to treat pa­tients with in­hibitors. An ap­proval here would make Roche an of­fi­cial ri­val of Shire, which has built a fran­chise around fac­tor re­place­ment ther­a­py. The FDA — hav­ing al­ready grant­ed the drug break­through sta­tus — has promised to give a de­ci­sion by Oc­to­ber 4, 2018.

→ Is­rael’s Ora­sis Phar­ma­ceu­ti­cals has raised a $13 mil­lion B round to fu­el its work on an eye drop for the treat­ment of pres­by­opia symp­toms. The fi­nanc­ing was led by Vi­sion­ary Ven­tures, with par­tic­i­pa­tion from Se­quoia Cap­i­tal, SBI (Japan) In­no­va­tion Ven­tures, LifeSci Ven­ture Part­ners, and “oth­er pri­vate in­vestors.”

→ New York’s Uro­Gen Phar­ma $URGN is los­ing its CFO Gary Ti­tus, the com­pa­ny an­nounced Tues­day. Uro­Gen is putting its COO Stephen Mul­len­nix in an in­ter­im CFO role while the com­pa­ny hunts for his re­place­ment. Uro­Gen al­so an­nounced the ad­di­tion of Ja­son Pa­tel, the for­mer se­nior di­rec­tor of ac­count­ing and fi­nance at Kite, as the com­pa­ny’s new VP of fi­nance.

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?

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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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.