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

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

When Link Fund Solutions announced that it is closing Woodford Equity Income Fund permanently and kicking out Neil Woodford, it was implied that investors probably won’t get back everything they entrusted to the fund manager. But nobody knew just how much they would lose.

An internal analysis commissioned by Link suggested that the collective loss could amount to £1 billion — out of a fund last valued at £3.1 billion — Citywire has revealed.

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.

Hold up your hand. Make a fist. Now open it. And again.

If you can do it fully and with ease, then the proteins in your hand are likely working properly. If you can’t then they may not be. In people with myotonic muscular dystrophy, something more atomic is going on.

In those folks, the problem is RNA. Certain base pairs repeat far beyond normal, up to 11,000 superfluous letters in some cases. The extended strands form “clumps.” Proteins misform and can’t function properly. They often allow one movement but not the reverse, a condition called myotonia that gives the dystrophy its name.