Work­ing to get its pain R&D strat­e­gy back on track, Acel­Rx steps clos­er to an FDA pitch

Acel­Rx has de­liv­ered an­oth­er pos­i­tive piece of the Phase III puz­zle for ARX-04, its un­der-the-tongue ap­pli­ca­tion of a 30 mcg dose of the pow­er­ful opi­oid sufen­tanil de­signed to de­liv­er a jolt of fast pain re­lief.

Ex­pand­ing on an im­prove­ment in pain scores al­ready record­ed for the first co­hort of 40 in­jured pa­tients who turned up at the ER and re­ceived a sin­gle dose of ARX-04, the Red­wood City, CA-based biotech says that it record­ed a slight­ly bet­ter out­come for a small group of 36 more pa­tients who were green-light­ed for mul­ti­ple ap­pli­ca­tions.

The lat­est batch of da­ta leaves this treat­ment on track to a near-term NDA, com­pa­ny ex­ecs tell me, as Acel­Rx works to stay on track with this pro­gram af­ter be­ing de­railed on its lead ef­fort last year.

The biotech $ACRX re­port­ed last Feb­ru­ary that ARX-04 cut base­line pain scores by an av­er­age of 2.7 on a 10-point scale. In the sec­ond co­hort, the drop was 3.1 points from a base­line score of 8.1, hit­ting the pri­ma­ry end­point. Most of the pa­tients, 75%, got the pain re­lief they were look­ing for from a sin­gle dose un­der the tongue, while 7 re­quired two dos­es and two got a third. Three were al­so dosed with mor­phine. Ap­pro­pri­ate­ly, the re­sults were pre­sent­ed at the 7th World Con­gress of Moun­tain & Wilder­ness Med­i­cine.

Pam Palmer, CMO and Co-Founder of Acel­Rx

Those are small num­bers, but com­pa­ny co-founder and Chief Med­ical Of­fi­cer Pam Palmer says that the com­pa­ny has run 11 stud­ies look­ing at dif­fer­ent dos­es of sufen­tanil, in­clud­ing the 15 mcg dose. And all of that will be used to sup­port its pitch to the agency.

“We want­ed to go af­ter a broad­er pa­tient pop­u­la­tion than the post-op­er­a­tive set­ting,” she notes, not­ing that that is where you’ll find the bulk of the re­lat­ed painkillers used. Ac­cord­ing to the CDC, there are some 136 mil­lion vis­its to the emer­gency de­part­ment each year, ac­count­ing for 44 vis­its for every 100 Amer­i­cans. Acel­Rx says that close to 50 mil­lion of those vis­its re­quire at least a cou­ple of dos­es of opi­oids to con­trol pain. And that’s the mar­ket the com­pa­ny hopes to tap in on, once it gets past the FDA.

It’s al­so no easy task to re­cruit emer­gency room pa­tients for a clin­i­cal tri­al, she adds in ex­pla­na­tion of the num­bers used in the late-stage study, not­ing that most peo­ple rush­ing to the ER for help aren’t in the mood to be re­cruit­ed for a tri­al.

The U.S. Army Med­ical Re­search and Ma­teriel Com­mand has helped fund the work on ARX-04, look­ing for a treat­ment that can be used in bat­tle­field sit­u­a­tions. And the treat­ment has al­so demon­strat­ed its use­ful­ness with­out se­vere­ly af­flict­ing cog­ni­tion, notes the com­pa­ny – a use­ful fea­ture for a drug that could be used in com­bat.

Acel­Rx’s stock, though, still hasn’t re­cov­ered from a nasty shock back in the spring of 2015, when the biotech had to sud­den­ly and un­ex­pect­ed­ly re­nege on its promise to in­vestors than no new Phase III study would be re­quired for its top opi­oid drug Za­lvi­so.

Like ARX-04, Za­lvi­so of­fers a twist on dos­ing the pow­er­ful opi­oid sufen­tanil. It is sup­posed to al­low hos­pi­tal pa­tients to self-ad­min­is­ter mi­cro tablets of the drug un­der the tongue, re­plac­ing an IV, for light­ning fast pain re­lief. But reg­u­la­tors, who have been on high alert wher­ev­er opi­oids are in­volved, want to see da­ta from an­oth­er study — the fourth from Acel­Rx -to bet­ter eval­u­ate the risks of dis­pens­ing er­rors.

The set­back forced the com­pa­ny to re­struc­ture and re­con­fig­ure its com­mer­cial­iza­tion plans. Its CEO at the time left soon af­ter.

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.