Ul­tragenyx wins a block­buster OK for its lead­ing rare dis­ease drug buro­sum­ab -- priced at $200,000

Ul­tragenyx $RARE has won the big FDA ap­proval they were look­ing for.

The biotech — al­lied with Ky­owa Hakko Kirin — re­port­ed Tues­day af­ter­noon that the agency came through on buro­sum­ab, an in­her­it­ed form of rick­ets. This is its sec­ond OK and the one that the com­pa­ny be­lieves will put them on the map for com­mer­cial­iza­tion work.

Emil Kakkis, Ul­tragenyx

This drug is cen­tral to Ul­tragenyx’s nar­ra­tive. The biotech re­cent­ly scored an ap­proval for Mep­se­vii, a drug that treats rare cas­es of mu­copolysac­cha­ri­do­sis type VII . But it’s the buro­sum­ab launch that has every­one’s at­ten­tion on Wall Street. The biotech has been set­ting up a small, 30-per­son sales force as it be­gins to han­dle what Ul­tragenyx ex­ecs hope will be a sub­stan­tial port­fo­lio of rare dis­ease drugs.  And buro­sum­ab has peak sales fig­ures of $1 bil­lion tied to it.

The drug will be sold as Crysvi­ta for X-linked hy­pophos­phatemia (XLH) in adult and pe­di­atric pa­tients 1 year of age and old­er. This is the first drug ap­proved for XLH in the US, “and the on­ly treat­ment that tar­gets the un­der­ly­ing cause of the dis­ease.”

In a call with an­a­lysts, the com­pa­ny said it would set a re­al-world price of about $160,000 for chil­dren and $200,000 a year for adults, de­pend­ing on their weight.

The dis­ease trig­gers high lev­els of phos­pho­rous in the blood. In one study 94% of adults re­ceiv­ing Crysvi­ta once a month achieved nor­mal phos­pho­rus lev­els — com­pared to on­ly 8% of those re­ceiv­ing place­bo.

Ul­tragenyx al­so wins its sec­ond pri­or­i­ty re­view vouch­er with the ap­proval af­ter sell­ing the first for $130 mil­lion.

An­a­lysts like the win, but some are al­so of­fer­ing caveats. From Joseph Schwartz at Leerink:

We be­lieve that in­vestors will wel­come to­day’s up­date al­though there may re­main some con­tro­ver­sy about the at­trac­tive­ness of the clin­i­cal pro­file, par­tic­u­lar­ly in adults who did not ben­e­fit from a sig­nif­i­cant pain re­duc­tion in Phase 3.

“XLH dif­fers from oth­er forms of rick­ets in that vi­t­a­min D ther­a­py is not ef­fec­tive,” stat­ed Julie Beitz, di­rec­tor of the Of­fice of Drug Eval­u­a­tion III in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search. “This is the first FDA-ap­proved med­ica­tion for the treat­ment of XLH and a re­al break­through for those liv­ing with this se­ri­ous dis­ease.”

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.