Pro­teosta­sis shares whacked hard as CF drug falls short; Ad­vax­is ax­es tri­al plans in wake of a clin­i­cal hold

Pro­teosta­sis tried mighty hard to per­suade in­vestors that its pos­i­tive bio­mark­er da­ta from a study of its cys­tic fi­bro­sis drug PTI-801 un­der­scored the drug’s po­ten­tial. But with a fail on im­prov­ing lung func­tion, added to some caus­tic crit­i­cism from a promi­nent an­a­lyst, the stock got whacked. 

“While PTI’s cor­rec­tor binds to a dif­fer­ent site on the CFTR pro­tein than Ver­tex’s med­i­cines, the fail­ure to match the high ef­fi­ca­cy bar es­tab­lished by Ver­tex $VRTX is a tes­ta­ment to the dif­fi­cult de­vel­op­ment path for com­peti­tors with small mol­e­cule ther­a­pies for cys­tic fi­bro­sis,” not­ed Leerink’s Ge­of­frey Porges. 

Next up, notes Leerink, is Gala­pa­gos’ GLPG- 2737 $GLPG, which will al­so be mea­sured against Ver­tex’s dom­i­nant drug. Gala­pa­gos will have to get around Ver­tex’s loy­al le­gion of an­a­lysts, who are quick to at­tack ri­vals seek­ing to ap­proach the throne. But da­ta are da­ta.

Shares of the com­pa­ny $PTI plunged 27% in pre-mar­ket trad­ing Thurs­day.

→ With its lead drug on clin­i­cal hold fol­low­ing the death of a pa­tient in a study, Ad­vax­is $ADXS is re­work­ing its pipeline plans to slash its burn rate and guard cash. The biotech re­port­ed Thurs­day morn­ing that it will cut its in­vest­ment in ax­al­imo­gene filolis­bac and amp up work on oth­er ther­a­pies while carv­ing $30 mil­lion out of its an­nu­al bud­get. Ex­ecs are al­so look­ing for a part­ner, say­ing that if they can’t strike a pact fair­ly soon they will stop their “AIM2CERV tri­al in high-risk lo­cal­ly ad­vanced cer­vi­cal can­cer, and will not con­duct the AD­VANCE PD-1 com­bi­na­tion tri­al in metasta­t­ic cer­vi­cal can­cer, which has not yet been ini­ti­at­ed. Ad­vax­is has de­ter­mined to fo­cus fu­ture de­vel­op­ment ef­forts for AX­AL on HPV-pos­i­tive head-and-neck can­cer through cost-ef­fec­tive clin­i­cal stud­ies that are cur­rent­ly be­ing ex­plored.”

→ Mi­cro­cap biotech Oph­thotech $OPHT shot up 30% — and count­ing — Thurs­day morn­ing af­ter an­nounc­ing a gene ther­a­py col­lab­o­ra­tion with Uni­ver­si­ty of Penn­syl­va­nia and Uni­ver­si­ty of Flori­da. The New York-based com­pa­ny now has a li­cense to de­vel­op and com­mer­cial­ize an ade­no-as­so­ci­at­ed virus (AAV) gene ther­a­py that treats an or­phan mono­genic dis­ease called rhodopsin-me­di­at­ed au­to­so­mal dom­i­nant re­tini­tis pig­men­tosa (RHO-adRP). Af­fect­ing around 11,000 pa­tients in the US and five ma­jor Eu­ro­pean mar­kets, the dis­ease trig­gers se­vere vi­sion loss lead­ing to blind­ness. The pre­clin­i­cal prod­uct com­ing out of Penn and Flori­da, Oph­thotech says, is de­signed to si­mul­ta­ne­ous­ly knock down mu­tant rhodopsin pro­tein and de­liv­er a func­tion­al re­place­ment. This marks the sec­ond gene ther­a­py deal for Oph­thotech, which is al­so col­lab­o­rat­ing with the Uni­ver­si­ty of Mass­a­chu­setts Med­ical School. In ad­di­tion to work­ing to­ward an IND, the biotech al­so made a sep­a­rate spon­sored re­search pact with Penn to con­duct fur­ther pre­clin­i­cal stud­ies.

→ En­gulfed in a cu­ri­ous se­ries of reg­u­la­to­ry and fi­nan­cial woes, MiMedx $MDXG is let­ting its CFO Michael Senken go af­ter wrap­ping up an in­ter­nal in­ves­ti­ga­tion in­to its sales and dis­tri­b­u­tion prac­tices. Al­so shown the door: John Cranston, the cor­po­rate con­troller and trea­sur­er. An in­ter­im CFO, Ed­ward Borkows­ki, has been brought in to re­state fi­nan­cial state­ments from the past six years, as the com­pa­ny claims that in­for­ma­tion can no longer be re­lied up­on. That’s a con­clu­sion of the still on­go­ing in­ves­ti­ga­tion by its own au­dit com­mit­tee, which be­gan this Feb­ru­ary and de­layed the re­lease of 2017’s fi­nan­cial re­sults (the com­pa­ny still doesn’t have a time­line for fil­ing its 10-K). The in­ter­nal re­view was quick­ly fol­lowed by a re­port­ed pre­lim­i­nary probe by the US Jus­tice De­part­ment in the same mat­ter as well as whether the tis­sue graft mak­er over­charged the gov­ern­ment for its prod­ucts. Ma­ri­et­ta, Geor­gia-based MiMedx, which has a trou­bled his­to­ry with short sell­ers, saw its stock fall by close to 13%.

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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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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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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