Nabri­va goes 2 for 2 in Phase III an­tibi­ot­ic pro­gram, scor­ing on a key cat­a­lyst -- but shares plunge on safe­ty wor­ries

Nabri­va Ther­a­peu­tics $NBRV has scored a sec­ond, piv­otal Phase III win for its an­tibi­ot­ic lefa­mulin in com­mu­ni­ty ac­quired bac­te­r­i­al pneu­mo­nia — set­ting up a pitch to the FDA lat­er this year and quite like­ly clear­ing a path to its first com­mer­cial launch.

Their an­tibi­ot­ic achieved non-in­fe­ri­or­i­ty com­pared to mox­i­floxacin for ear­ly clin­i­cal re­sponse (ECR) — 72 to 120 hours fol­low­ing ini­ti­a­tion of ther­a­py in the in­tent to treat pa­tient pop­u­la­tion. In this tri­al, ECR rates were 90.8% for lefa­mulin and 90.8% for mox­i­floxacin. The tri­al al­so achieved Eu­ro­pean goals out­lined for an ap­proval.

Nabri­va’s shares rock­et­ed up 24% on the news, and then re­versed course as in­vestors wres­tled over the safe­ty da­ta, falling 17% by the end of trad­ing. Di­ar­rhea proved a more ex­pan­sive con­cern than the ef­fi­ca­cy da­ta, with the an­tibi­ot­ic arm do­ing worse than moxi. Tox­i­c­i­ty is­sues grabbed in­vestors by the throat, with plen­ty of fret­ting about po­ten­tial lim­its to its use.

A cru­cial cat­a­lyst for this biotech, lefa­mulin came out of the sec­ond late-stage study look­ing iden­ti­cal to moxi, fit­ting a pro­file reg­u­la­tors re­quire for a mar­ket­ing ap­proval. If every­thing goes ac­cord­ing to plan, and gets around the safe­ty wor­ries, that will set up a launch in 2019 as the biotech seeks to carve out a large seg­ment of a big mar­ket, and not just due to drug re­sis­tance.

Col­in Broom

“I do not see this as an an­tibi­ot­ic to keep in re­serve,” CEO Col­in Broom tells me in a pre­view of the news. “It’s a short course of ther­a­py (5 days for lefa­mulin com­pared to 7 days for moxi), high­ly ef­fec­tive, with com­plete cov­er­age of the pathogens we wor­ry about….It’s re­al­ly the on­ly an­tibi­ot­ic out there that has the op­por­tu­ni­ty to be used out of the gate.”

Mak­ing that ar­gu­ment stick with pay­ers will re­quire some mod­er­a­tion on the pric­ing, he adds, with­out spelling out the num­bers. An­a­lysts will like­ly stay mind­ful that pay­ers will keep fo­cused on price, al­ways re­quir­ing cheap gener­ics when­ev­er pos­si­ble.

The biotech al­so re­port­ed that one pa­tient in their study al­so de­vel­oped C diff dur­ing an ex­tend­ed hos­pi­tal stay.

Gary Sender

Nei­ther Broom nor CFO Gary Sender are of­fer­ing their own peak sales es­ti­mates, but the CFO notes that an­a­lysts cov­er­ing the com­pa­ny have pen­cilled in es­ti­mates rang­ing from $500 mil­lion to $700 mil­lion a year — and they ex­pect those an­a­lysts to do some re­cal­cu­la­tions in their fa­vor with the lat­est batch of piv­otal da­ta.

Even now Nabri­va has a sales force of 20 work­ing the pre-com­mer­cial­iza­tion mar­ket. That will like­ly ex­pand to the 30-to-60 range, says the CEO, and even­tu­al­ly up to around 100. Broom ex­pects to line up part­ners for the ex-US mar­ket.

In do­ing so, Nabri­va may soon find it­self go­ing up against Paratek’s an­tibi­ot­ic, which al­so suc­ceed­ed for CABP and was filed for an ap­proval in Feb­ru­ary. Broom shrugs that off, though, dis­count­ing the ri­val as a broad spec­trum al­ter­na­tive that will like­ly be held in re­serve, al­low­ing lefa­mulin to push ahead in­to a broad­er mar­ket.

It won’t hurt Nabri­va that the FDA height­ened its warn­ings against the use of flu­o­ro­quinolones like maxi a cou­ple of years ago, af­ter iden­ti­fy­ing new safe­ty is­sues that in­cludes dis­abling side ef­fects in­volv­ing ten­dons, mus­cles, joints and nerves. And he adds that the com­mon­ly used z packs have be­come lit­tle bet­ter than a place­bo for about half of all cas­es of bac­te­r­i­al pneu­mo­nia.

Still, for years now the de­vel­op­ment of new an­tibi­otics has been left to small biotechs like Nabri­va, af­ter Big Phar­ma’s large­ly bowed out of a field they iden­ti­fied with nar­row mar­gins — de­spite the grow­ing num­ber of alarms from mul­ti­tude of glob­al health agen­cies over a steadi­ly ris­ing tide of drug re­sis­tance. It’s not an easy field, as a slate of re­cent clin­i­cal mishaps un­der­score. And cheap gener­ics are typ­i­cal­ly thrown at cas­es as they arise.

Broom, though, like oth­er CEOs in the field, be­lieves the eco­nom­ics of an­tibi­otics will grad­u­al­ly im­prove as more cas­es of re­sis­tance rise up. And he plans to be there with one of the new breed when it does.

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.