Strike two: Au­ris shares crushed again as its sec­ond PhI­II pro­gram fails to de­liv­er

Au­ris Med­ical has lurched from a failed late-stage study of its ex­per­i­men­tal tin­ni­tus drug last year to a Phase III flop for its hear­ing loss ther­a­py. And once again, it’s claim­ing that a change­up in tri­al de­sign could fix every­thing.

The Swiss biotech $EARS test­ed two dos­es of AM-111 in pa­tients with se­vere to pro­found sud­den deaf­ness, and both failed to achieve sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments for the peo­ple who took the drug com­pared to a place­bo.

How­ev­er.

Thomas Mey­er

Au­ris im­me­di­ate­ly shift­ed to a post hoc analy­sis — not the most pop­u­lar route for de­vel­op­ers — of one group of pa­tients “with pro­found acute hear­ing loss,” and dis­cov­ered a pos­i­tive change in the low dose arm. The biotech, though, says it is scrap­ping an­oth­er Phase III that has a sim­i­lar de­sign with vir­tu­al­ly no chance of suc­cess.

Au­ris’ post hoc work wasn’t con­vinc­ing enough for share­hold­ers, who greet­ed the sec­ond round of bad news with an­oth­er slap at the stock price, now down 41% in pre-mar­ket trad­ing as Au­ris shares drill a deep­er hole in pen­ny stock ter­ri­to­ry.

The lat­est set­back fol­lows the Phase III fail­ure of Au­ris’ tin­ni­tus ther­a­py, which they al­so re­spond­ed to with a pitch that it could fix the da­ta with a change­up in the pri­ma­ry end­points.

Au­ris CEO Thomas Mey­er said that the lat­est tri­al was a “ma­jor mile­stone” for the com­pa­ny.

Al­though the tri­al did not meet our ex­pec­ta­tions for the pri­ma­ry ef­fi­ca­cy end­point in the over­all study pop­u­la­tion, we are very pleased to see the clin­i­cal­ly and sta­tis­ti­cal­ly sig­nif­i­cant treat­ment ef­fect in the pro­found hear­ing loss sub­pop­u­la­tion. Con­sid­er­ing the high un­met med­ical need, we look for­ward to dis­cussing with reg­u­la­to­ry agen­cies the next steps on our path to bring­ing AM-111 to pa­tients.

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.