Busy Sanofi takes a pass on one of Al­ny­lam’s rare dis­ease drugs, FDA of­fers ‘break­through’ sta­tus for PhI­II

A cou­ple of months af­ter Sanofi $SNY and Al­ny­lam $AL­NY re­tooled their RNAi col­lab­o­ra­tion so that the French phar­ma gi­ant could con­cen­trate on their he­mo­phil­ia ther­a­py while giv­ing up rights to the biotech’s patisir­an, the two part­ners are al­so go­ing their sep­a­rate ways on a late-stage drug for rare cas­es of pri­ma­ry hy­per­ox­aluria type 1.

John Maraganore

Sanofi is tak­ing a pass on an op­tion for lumasir­an (ALN-GO1), leav­ing Al­ny­lam CEO John Maraganore to take the drug in­to Phase III with a brand new break­through drug des­ig­na­tion from the FDA to speed things along.

Their drug is de­signed to “re­duce the he­pat­ic lev­els of the GO en­zyme, there­by de­plet­ing the sub­strate nec­es­sary for ox­alate pro­duc­tion,” which trig­gers se­vere ad­verse events of­ten lead­ing to kid­ney fail­ure. And Al­ny­lam is in com­pe­ti­tion with Dicer­na $DR­NA, a blood ri­val which is go­ing af­ter a dif­fer­ent tar­get in treat­ing the same dis­ease.

Sanofi may have all the R&D ac­tion it wants right now. Af­ter leav­ing the mar­ket hang­ing on promis­es of a ma­jor new deal to as­sist the strug­gling phar­ma gi­ant, Sanofi ac­quired Biover­a­tiv for $11.6 bil­lion and quick­ly fol­lowed with its ac­qui­si­tion of Abl­ynx. The Biover­a­tiv deal helped ex­plain Sanofi’s in­ter­est in gain­ing the world­wide rights to Al­ny­lam’s fi­tusir­an, giv­ing it a big­ger pipeline of he­mo­phil­ia ther­a­pies.

Yvonne Green­street

Al­ny­lam didn’t sound in any way ag­griev­ed to­day, hap­py to col­lect their third BTD from the agency and an­oth­er Phase III can­di­date as they prep what is wide­ly ex­pect­ed to be a near-term ap­proval for patisir­an, now un­der re­view.

“Based on Phase I/II clin­i­cal da­ta pre­sent­ed to date, we be­lieve lumasir­an could be a trans­for­ma­tive treat­ment for pa­tients with PH1, an ul­tra-rare dis­ease with no ap­proved ther­a­pies, in which ex­cess ox­alate pro­duc­tion leads to kid­ney fail­ure and sig­nif­i­cant mor­bid­i­ty and mor­tal­i­ty,” said Yvonne Green­street, the COO at Al­ny­lam. “We are al­so thrilled with FDA’s de­ci­sion to grant Break­through Ther­a­py Des­ig­na­tion to lumasir­an. We be­lieve this is a tes­ta­ment to the drug’s po­ten­tial to ad­dress the se­vere un­met med­ical need that PH1 rep­re­sents for pa­tients and their fam­i­lies. Al­ny­lam is now poised to ad­vance lumasir­an in­to Phase 3 in late 2018 and, as­sum­ing ap­proval, to com­mer­cial­ize lumasir­an glob­al­ly.”

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.