With a big as­sist from the FDA, Sage plots a swift march through PhI­II for oral de­pres­sion drug -- mar­ket cap swells $1B-plus

The FDA is putting Sage Ther­a­peu­tics’ oral de­pres­sion drug SAGE-217 on their in­side track, bless­ing a fast-paced late-stage strat­e­gy that in­cludes des­ig­nat­ing their on­go­ing study for post-par­tum de­pres­sion a piv­otal ef­fort.

Jeff Jonas

Ac­cord­ing to Sage ex­ecs $SAGE, reg­u­la­tors will de­mand on­ly one more late-stage study, which will now get un­der­way in a mat­ter of months. Sage plans to eval­u­ate “two weeks of 20mg or 30mg SAGE-217 treat­ment com­pared to place­bo in 450 pa­tients with MDD, with four weeks of ad­di­tion­al fol­low-up.”

Re­searchers will con­tin­ue to fol­low hun­dreds of pa­tients for up to a year to bet­ter con­sid­er the drug’s safe­ty and how to han­dle the re­cur­rence of ma­jor de­pres­sion.

Sage is go­ing af­ter episod­ic cas­es of de­pres­sion af­ter not­ing in its most re­cent round of da­ta that the drug’s ef­fect dropped off in the 4- to 6-week stretch, los­ing its im­pact with pa­tients as re­mis­sion rates tailed off in­to in­signif­i­cance.

The news sound­ed up­beat to in­vestors, dri­ving shares up about 6% in pre-mar­ket trad­ing, then surg­ing to an 18% gain by ear­ly af­ter­noon. Sage start­ed the day with a whop­ping $6.8 bil­lion mar­ket cap which has now swelled past the $8 bil­lion mark.

The agency is sign­ing off on a plan that of­fers a dra­mat­ic shift from the usu­al long and drawn out late-stage test­ing of de­pres­sion drugs in big, mul­ti­ple stud­ies — mak­ing it one of the most dif­fi­cult fields in R&D plagued by high place­bo re­spons­es. The biotech is go­ing af­ter a new mech­a­nism — ex­tra-synap­tic GA­BA-mod­u­la­tion — while the cur­rent gen­er­a­tion of drugs share com­mon neu­ro­trans­mit­ter tar­gets like sero­tonin, dopamine and nor­ep­i­neph­rine.

The cur­rent ther­a­pies are prob­lem­at­ic at best, with pa­tients of­ten cy­cling through mul­ti­ple meds in search of one that can work, for awhile.

Scott Got­tlieb

The ac­cel­er­at­ed late-stage pro­gram un­der­scores the new man­age­ment that’s now at the FDA. Scott Got­tlieb promised to ac­cel­er­ate de­vel­op­ment un­der his tenure as com­mis­sion­er, and this open­ing for Sage is a clear in­di­ca­tion of his will­ing­ness to cham­pi­on speed over cau­tion. Oth­er biotechs are like­ly to pay very close at­ten­tion to what’s hap­pen­ing here, look­ing to mim­ic Sage’s rapid for­ward move­ment.

The move comes soon af­ter reg­u­la­tors gave Sage’s in­fused de­pres­sion drug brex­anolone a pri­or­i­ty re­view, lop­ping months off the process and pos­si­bly herald­ing the biotech’s first com­mer­cial launch — if every­thing goes ac­cord­ing to plan.

As al­ways, CEO Jeff Jonas is all in tout­ing their drug as a game chang­er. He said:

In this de­vel­op­ment pro­gram, we are ex­plor­ing the po­ten­tial for pa­tients with MDD to feel well with­in days, with just a 2-week course of treat­ment – sim­i­lar to how an­tibi­otics are used to­day – in­stead of en­dur­ing long-term chron­ic treat­ment. We be­lieve a med­i­cine with rapid on­set and ro­bust re­sponse could be tru­ly par­a­digm shift­ing. SAGE-217, if suc­cess­ful­ly de­vel­oped and ap­proved, may rewrite the text­book on how the tens of mil­lions of peo­ple suf­fer­ing from MDD are treat­ed, ul­ti­mate­ly turn­ing de­pres­sion in­to a dis­or­der, not an iden­ti­ty.

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.