Fu­eled by ear­ly suc­cess, Enan­ta paves way for mid-stage RSV study in adult pa­tients lat­er this year

RSV, a field lit­tered with fail­ure, has an­oth­er hope­ful.

On Fri­day, Enan­ta Phar­ma­ceu­ti­cals showed that its ex­per­i­men­tal treat­ment, EDP-938, con­ferred a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion in the res­pi­ra­to­ry syn­cy­tial virus (RSV) vi­ral load in healthy adults in­oc­u­lat­ed with the virus. Now comes the hard part.

Jay Lu­ly Enan­ta

“While we view to­day’s pos­i­tive re­sults from the Phase 2a hu­man chal­lenge study as en­cour­ag­ing, we an­tic­i­pate that re­sults from the Phase 2b will go a much longer way as to de­ter­min­ing whether EDP-938 is a vi­able treat­ment for RSV…We think the path to com­mer­cial­iza­tion here is par­tic­u­lar­ly dif­fi­cult to nav­i­gate, giv­en that RSV in­fec­tion re­solves fair­ly quick­ly with­out in­ter­ven­tion and the key will be show­ing a dif­fer­ence in terms of time to res­o­lu­tion,” Baird’s Bri­an Sko­r­ney wrote in a note.

Once 115 healthy adults were in­oc­u­lat­ed with the virus, pa­tients were ran­dom­ized to ei­ther get place­bo or ad­min­is­tered two dif­fer­ent EDP-938 reg­i­mens. The Enan­ta drug is an N-pro­tein in­hibitor, which the com­pa­ny be­lieves has an edge over fu­sion in­hibitors cur­rent­ly in de­vel­op­ment, as it tar­gets the virus’ repli­ca­tion ma­chin­ery. Com­pared to the place­bo, pa­tients giv­en ei­ther reg­i­men achieved a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion (p<0.001) in RSV vi­ral load and was well tol­er­at­ed, Enan­ta said.

“We think this study large­ly con­firms the an­ti-vi­ral ac­tiv­i­ty of this drug, but was wide­ly ex­pect­ed and the pro­gram is now mov­ing in­to the more cost­ly and dif­fi­cult pe­ri­od of clin­i­cal de­vel­op­ment…Re­call, RSV has been a tough space, with John­son and John­son dis­con­tin­u­ing de­vel­op­ment of lu­mic­itabine, even af­ter the med­ica­tion had pro­duced promis­ing Phase 2a re­sults from a sim­i­lar hu­man chal­lenge mod­el,” Sko­r­ney not­ed.

The com­plex mol­e­c­u­lar struc­ture of RSV has large­ly thwart­ed drug­mak­ers from de­vel­op­ing a vac­cine or a treat­ment for an in­fec­tion that af­flicts most Amer­i­can in­fants be­fore their sec­ond birth­day. For most old­er healthy chil­dren and adults, RSV caus­es lit­tle dis­com­fort be­yond a com­mon cold, but in high-risk groups with com­pro­mised im­mune sys­tems such as in­fants and the el­der­ly, it can lead to more se­ri­ous lung and air­way in­fec­tions.

But ef­forts to sti­fle the in­fec­tion have cul­mi­nat­ed in a litany of fail­ures. Most re­cent­ly, J&J $JNJ ter­mi­nat­ed Phase IIb tri­als of its RSV drug lu­mic­itabine, Re­gen­eron $REGN aban­doned its RSV drug af­ter a late-stage fail­ure and No­vavax’s $NVAX RSV vac­cine has failed a pair of Phase III tri­als (al­though it in­sists there’s a way for­ward). The on­ly sil­ver lin­ing is from As­traZeneca $AZN, the orig­i­nal mak­er of the on­ly pre­ven­ta­tive treat­ment, Synagis, for se­ri­ous low­er res­pi­ra­to­ry tract in­fec­tions (LR­TI) caused by RSV in in­fants and young chil­dren. In Feb­ru­ary, the British drug­mak­er was grant­ed ex­pe­dit­ed re­view from US and EU reg­u­la­tors for its long-act­ing RSV drug, ME­DI8897.

Buoyed by its ear­ly suc­cess, Enan­ta is plan­ning its Phase IIb study by the end of 2019 in adult out­pa­tients with con­firmed RSV in­fec­tions, com­pa­ny chief Jay Lu­ly said.

The com­pa­ny’s shares $EN­TA ini­tial­ly rose, but closed down about 12% at $85.81 on Fri­day. “I just don’t think RSV treat­ment is an in­di­ca­tion that in­vestors are ready to give cred­it to at this stage so it be­came a sell the news event,” Sko­r­ney told End­points News.

So­cial im­age: Shut­ter­stock

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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UP­DAT­ED: Stay tuned: Bio­gen’s num­bers are great — it’s their wor­ri­some fu­ture that leaves an­a­lysts skit­tish

Biogen came out with an upbeat assessment of their Q2 numbers today, discounting the arrival of a key rival for its blockbuster Spinraza franchise. But the top execs remain grimly determined to not say much anything new about the sore points that have dragged down its stock, including the future of its big investment in Alzheimer’s or how it plans to invest the considerable cash that the big biotech continues to reap.

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PACT Phar­ma says it's per­fect­ed the tech to se­lect neoanti­gens for per­son­al­ized ther­a­py — now on­to the clin­ic

At PACT Pharma, the lofty goal to unleash a “tsunami” of T cells personalized for each patient has hinged on the ability to correctly identify the neoantigens that form something of a fingerprint for each tumor, and extract the small group of T cells primed to attack the cancer. It still has a long way to go testing a treatment in humans, but the biotech says it has nailed that highly technical piece of the process.

UP­DAT­ED: My­ovan­t's uter­ine fi­broid drug looks com­pet­i­tive in PhI­II — but can they van­quish mighty Ab­b­Vie?

Vivek Ramaswamy’s Myovant $MYOV has closely matched its positive first round of Phase III data for their uterine fibroid drug relugolix, setting up a head-to-head rivalry with pharma giant AbbVie as the little biotech steers to the market with a planned filing in Q4.

Here’s how Myovant plans to prevail over the AbbVie $ABBV empire.

In the study, 71.2% of women receiving once-daily relugolix combination therapy achieved the clinical response they were looking for, compared to only 14.7% in the control arm. The data comfortably reflected the same outcomes in the first Phase III — 73.4% of women receiving once-daily oral relugolix combination therapy achieved the responder criteria compared with 18.9% of women receiving placebo — which will reassure regulators that they are getting the carefully randomized data that qualifies for the FDA’s gold standard for success.

Lit­tle Mar­i­nus sees its shares eclipsed as the Sage ri­val fails to com­pare on PPD in PhII

The executive team at Sage $SAGE have skirted another potential pitfall on its way to racking up a big future for its depression drug Zulresso.

Little Marinus Pharmaceuticals $MRNS had sought to challenge the Sage drug with an IV formulation — followed by an oral version — of ganaxolone for postpartum depression. But researchers say their Phase II study failed to positively differentiate itself from a placebo at 28 days — leaving them to hold up “clinically meaningful” data within the first day of administration compared to the control arm.

Roche cuts loose Tam­i­flu OTC rights, hand­ing Sanofi the keys as the phar­ma gi­ant dou­bles down on Xofluza

Roche set out to make a better flu medicine than Tamiflu as that franchise was headed to a generic showdown. Now they’ll see just how well Xofluza stacks up against the mainstay drug after handing off over-the-counter rights in the US to Sanofi.

Sanofi $SNY says it will now step in to negotiate a deal with the FDA to steer Tamiflu into the OTC market, a role that could well involve new studies to ease passage of the drug out of doctor’s hands and into the consumer end of the market. And the French pharma giant will have first dibs over “selected” OTC markets around the world as they push ahead.

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.