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

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine and serotonin, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs. It can also lead to multiple organ failure.

To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

Ather­sys tries to post-hoc-an­a­lyze its way out of an­oth­er tri­al fail for stroke stem cell ther­a­py

Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

While a greater percentage of patients in the treatment group reached the primary endpoint compared to placebo, that difference was not statistically significant.

Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

All Blue's $733M bid to ac­quire Zymeworks turns hos­tile as board bat­tles back — af­ter a biotech celebri­ty jumps in

Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts.

This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share.

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