Pen­ny stock biotech Syn­thet­ic Bi­o­log­ics sees its 'break­through' sta­tus pulled as FDA pon­ders safe­ty is­sues

A lit­tle un­der a year ago the pen­ny stock of Syn­thet­ic Bi­o­log­ics $SYN gy­rat­ed high­er on the news that its drug SYN-004 (rib­axa­m­ase) — de­signed to pro­tect the gut mi­cro­bio­me from the hav­oc caused by some an­tibi­otics and guard against Clostrid­i­um dif­fi­cile in­fec­tion — had won the FDA’s not-so-rare break­through ther­a­py des­ig­na­tion.

Steven Shall­cross, Syn­thet­ic in­ter­im CEO

To­day, though, af­ter the biotech re­viewed plans for an up­com­ing Phase III pro­gram, they fol­lowed up with the rare news that the FDA is drop­ping the BTD af­ter reg­u­la­tors con­clud­ed that they couldn’t fig­ure out why the drug arm ex­pe­ri­enced a high­er rate of deaths in their Phase IIb tri­al.

From their state­ment:

Fol­low­ing FDA’s re­view of the ad­di­tion­al da­ta, it was de­ter­mined that the re­quire­ments for Break­through Ther­a­py Des­ig­na­tion were no longer met due to the nu­mer­i­cal im­bal­ance in fa­tal ad­verse events ob­served in the study which could not be ful­ly eval­u­at­ed due to the lim­it­ed safe­ty data­base, and the study’s method of sta­tis­ti­cal treat­ment of pa­tients who did not com­plete the study for any rea­son. 

The biotech’s stock slid 12% in pre-mar­ket trad­ing — falling to 30 cents a share.

You don’t of­ten hear about BTD’s be­ing with­drawn, but it hap­pens more of­ten than you might think. The Twit­ter buzz that met the news this morn­ing in­clud­ed a link to the FDA’s page on BTDs that showed 4 break­through ti­tles were re­scind­ed so far in fis­cal year 2018, which got start­ed Oc­to­ber 1.

The agency set up the BTD pro­gram in or­der to make a com­mit­ment to biotechs to help ac­cel­er­ate the re­view of drugs that war­rant­ed spe­cial at­ten­tion. And it’s been gain­ing steam un­der Com­mis­sion­er Scott Got­tlieb, who would like to see more reg­u­la­tors em­brace it the same way the on­col­o­gy di­vi­sion has.

The FDA did ex­press a will­ing­ness to keep help­ing, says Syn­thet­ic Bi­o­log­ics. And the biotech has worked out a plan with reg­u­la­tors on the Phase III de­sign that will keep a care­ful fo­cus on safe­ty, so they can keep a rein on risk.

They al­so agreed that the pri­ma­ry end­point should be “the re­duc­tion of the in­ci­dence of Clostrid­i­um dif­fi­cile in­fec­tion (CDI) in the rib­axa­m­ase treat­ment group rel­a­tive to place­bo.”

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.