An­oth­er cheap, old drug is be­ing fresh­ened up for brand pric­ing in the US — and shares soar, briefly

BioReg­num, the view from John Car­roll

When the ex­ecs at Fortress went about set­ting up their pain drug biotech Av­enue Ther­a­peu­tics $ATXI, they went to some ex­tremes to avoid R&D risk. In­stead of try­ing some­thing new, they tweaked a Eu­ro­pean IV for­mu­la­tion for tra­madol — nev­er ap­proved in the US, where oral tra­madol has been pre­scribed for 23 years — and took a shot at a piv­otal pro­gram.

To get start­ed, the com­pa­ny paid a grand to­tal of $3 mil­lion — in two stag­gered pay­ments — for the rights to an old, well un­der­stood and quite pow­er­ful gener­ic pain med, with mile­stones at­tached to their suc­cess.

That qual­i­fies as lunch mon­ey in the bio­phar­ma busi­ness.

Lucy Lu

More ex­pen­sive — but still rel­a­tive­ly cheap — was a piv­otal de­vel­op­ment pro­gram they reck­oned would cost $30 mil­lion when Av­enue pitched its S-1 to in­vestors. Rolled out at a pro­posed $10 a share, they took $6 at the pric­ing, raised $33 mil­lion for the re­search and saw the price surge to about $8 be­fore grad­u­al­ly de­scend­ing to a lit­tle more than $4 a share.

The mar­ket cap closed at a mi­cro lev­el of $44 mil­lion on Fri­day.

Which brings us to to­day. 

Av­enue is an­nounc­ing that the first piv­otal study, com­par­ing IV tra­madol to a place­bo, was a suc­cess, with a p-val­ue of 0.005 for the 50 mg dose. Three sec­ondary end­points al­so hit on the 50 mg. They’re hold­ing back the de­tails on the da­ta for a con­fer­ence and plan to start the sec­ond, con­fir­ma­to­ry Phase III lat­er this year.

Av­enue’s ex­ecs went in­to the study with the odds on their side. Tra­madol’s ef­fec­tive­ness is so well un­der­stood, it’s be­gun rais­ing con­cerns about be­com­ing the poor man’s opi­oid in oth­er coun­tries. The DEA des­ig­nat­ed the syn­thet­ic anal­gesic as a Sched­ule IV drug in 2015. So I asked Av­enue CEO Lucy Lu about that by email, and whether the com­pa­ny was sim­ply set­ting up a shot at charg­ing a pre­mi­um in the US for an­oth­er old gener­ic run by the reg­u­la­tors — al­beit af­ter a fair­ly sig­nif­i­cant de­vel­op­ment pro­gram.

Her re­sponse:

The FDA re­quires two place­bo-con­trolled stud­ies be­cause it’s not nec­es­sar­i­ly easy to beat place­bo in a pain study and on­ly tru­ly ef­fi­ca­cious drugs can do that. In our case, IV tra­madol beat place­bo not on­ly on the pri­ma­ry end­point, but al­so all of the key sec­ondary end­points, in­clud­ing pa­tient glob­al as­sess­ment, in a very con­sis­tent and dose de­pen­dent man­ner. In com­bi­na­tion with what we know about oral tra­madol, it points to a very ef­fec­tive IV drug.

We have not done a de­tailed pric­ing study. How­ev­er, all of the spon­sors in our space that have dis­cussed pric­ing of new drugs have men­tioned around $100 per day. I think that pro­vides a use­ful bench­mark.

Gener­ic tra­madol pills cost about $1.50 each.

In­vestors liked the sound of it all. Av­enue’s shares jumped 29% in pre-mar­ket trad­ing. But then peo­ple be­gan to think about it, and the shares end­ed down the day about 4%. It is, af­ter all, not the kind of sto­ry that peo­ple are at­tract­ed to.

There’s noth­ing new about grab­bing an old drug OK’d in Eu­rope and then bring­ing it in cheap years lat­er for the US mar­ket. Marathon did that with a steroid, de­vel­oped specif­i­cal­ly for Duchenne, which it then rolled out as an ex­pen­sive rare dis­ease drug — un­til the whole sit­u­a­tion blew up in­to a con­tro­ver­sy that forced the com­pa­ny to sell it to PTC. Marathon ex­ecs were Mar­tin Shkre­li’s role mod­el in drug de­vel­op­ment.

You can de­cide for your­self if Av­enue is gam­ing the sys­tem.

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.