Pro­to­cols: Retrophin shares soar on promis­ing Phase II da­ta; FDA read­ies Keytru­da re­view for lung can­cer

Retrophin CEO Stephen Ase­lage

Shares of San Diego-based Retrophin $RTRX shot up 26% Wednes­day morn­ing af­ter the com­pa­ny un­veiled promis­ing Phase II da­ta for their lead drug. In­ves­ti­ga­tors say they tracked a mean re­duc­tion of 44.8% in pro­tein­uria among pa­tients tak­ing sparsen­tan, well over twice the rate of re­duc­tion tracked in the irbe­sar­tan group. CEO Stephen Ase­lage says the com­pa­ny will now meet with the FDA to map out the short­est pos­si­ble route to the mar­ket. Retrophin is best known for its for­mer CEO, the no­to­ri­ous Mar­tin Shkre­li, who was kicked out of the com­pa­ny amid fraud al­le­ga­tions and now faces fed­er­al charges re­lat­ed to his han­dling of the biotech’s fi­nances and a now de­funct hedge fund.

Just weeks af­ter Bris­tol-My­ers Squibb suf­fered a set­back in the de­vel­op­ment of its check­point drug Op­di­vo for lung can­cer, ri­val Mer­ck says that the FDA is ready to re­view the use of Keytru­da as a first-line ther­a­py for lung can­cer. Keytru­da is al­ready ap­proved as a sec­ond-line ther­a­py.

Cam­bridge, MA-based up­start Deci­bel Ther­a­peu­tics has re­cruit­ed Agios sci­en­tif­ic co-founder Michael Su to the com­pa­ny as its new CSO. The new ex­ec­u­tive crew now al­so in­cludes Paula K. Cobb, se­nior vice pres­i­dent of strat­e­gy, port­fo­lio and pro­grams; John J. Lee, se­nior vice pres­i­dent of phar­ma­ceu­ti­cal de­vel­op­ment; and Pe­te Cas­tri­chi­ni, vice pres­i­dent of hu­man re­sources. The com­pa­ny is helmed by ex-Bio­gen ex­ec Steve Holtz­man.

Japan’s Min­istry of For­eign Af­fairs has a stern warn­ing for the UK bio­phar­ma in­dus­try: If you lose the EMA, don’t ex­pect Japan­ese phar­ma com­pa­nies to keep their R&D op­er­a­tions in and around Lon­don. “Many Japan­ese phar­ma­ceu­ti­cal com­pa­nies are op­er­at­ing in Lon­don, due to the EMA’s lo­ca­tion in Lon­don,” reads the mes­sage. “If the EMA were to trans­fer to oth­er EU Mem­ber States, the ap­peal of Lon­don as an en­vi­ron­ment for the de­vel­op­ment of phar­ma­ceu­ti­cals would be lost, which could pos­si­bly lead to a shift in the flow of R&D [re­search and de­vel­op­ment] funds and per­son­nel to Con­ti­nen­tal Eu­rope. This could force Japan­ese com­pa­nies to re­con­sid­er their busi­ness ac­tiv­i­ties.”

Au­ri­gene Dis­cov­ery Tech­nolo­gies is swap­ping a batch of mile­stones for 10.2 mil­lion shares of Curis. They’re work­ing on an on­col­o­gy col­lab­o­ra­tion.

Take­da has vac­ci­nat­ed the first sub­ject in the Tetrava­lent Im­mu­niza­tion against Dengue Ef­fi­ca­cy Study (TIDES), a Phase III tri­al of its live-at­ten­u­at­ed tetrava­lent dengue vac­cine can­di­date (TAK-003).

The con­tract de­vel­op­ment and man­u­fac­tur­ing group Phar­ma­ceu­tics In­ter­na­tion­al has raised $93 mil­lion from a con­sor­tium of in­vestors led by Signet Health­care Part­ners and in­clud­ing Athyri­um Cap­i­tal Man­age­ment, Hil­dred Cap­i­tal Part­ners and Phar­ma­science. The mon­ey comes as debt and eq­ui­ty.

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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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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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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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.