In an­oth­er set­back for the be­lea­guered an­tibi­otics field, Nabri­va Ther­a­peu­tics says the FDA re­ject­ed its fos­fomycin for com­pli­cat­ed uri­nary tract in­fec­tions.

Ted Schroed­er

Im­por­tant­ly, the biotech says, reg­u­la­tors are not ask­ing for any new clin­i­cal or safe­ty da­ta. The com­plete re­sponse let­ter cen­ters around “is­sues re­lat­ed to fa­cil­i­ty in­spec­tions and man­u­fac­tur­ing de­fi­cien­cies at one of Nabri­va’s con­tract man­u­fac­tur­ers,” ac­cord­ing to a brief state­ment.

Over the next few weeks Nabri­va plans to re­quest a “Type A” meet­ing to dis­cuss the FDA’s find­ings. Shares $NR­BV are down 24% pre-mar­ket.

Across the in­dus­try, an­tibi­otics de­vel­op­ers and back­ers are suf­fer­ing from a new low in morale af­ter Achao­gen filed for bank­rupt­cy de­spite notch­ing an FDA ap­proval of its cU­TI drug, due to dis­ap­point­ing sales. And that fol­lows rounds of lay­offs at both Achao­gen and oth­er an­tibi­otics com­pa­nies like Melin­ta, with Big Phar­ma large­ly out of the pic­ture al­to­geth­er.

No­body would de­ny the ur­gent need for an­tibi­otics — just two days ago the UN is­sued an­oth­er re­port warn­ing of a “silent tsuna­mi” of an­timi­cro­bial re­sis­tance that threat­ens to kill 10 mil­lion peo­ple an­nu­al­ly by 2050 — but it’s in­creas­ing­ly hard to make a case for the R&D in­vest­ment when the prod­ucts are too niche to turn a prof­it in a world dom­i­nat­ed by gener­ics.

Nabri­va’s Con­tepo (IV fos­fomycin) is an old drug that’s been used out­side the US for over 45 years. Ac­cord­ing to the com­pa­ny, it can treat a num­ber of Gram-neg­a­tive and Gram-pos­i­tive bac­te­ria in­fec­tions, ac­tive against many com­mon mul­ti-drug re­sis­tant strains.

It bagged the drug via an ac­qui­si­tion of Za­vante, whose CEO Ted Schroed­er then took the helm of the new en­ti­ty. Nabri­va’s in-house lead pro­gram for com­mu­ni­ty-ac­quired bac­te­r­i­al pneu­mo­nia, lefa­mulin, is al­so un­der re­view with a PDU­FA date in Au­gust.

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

In­vestors are in no mood to hear biotechs tout the suc­cess of a “key” sec­ondary end­point when the piv­otal Phase III flunks the pri­ma­ry goal. Just ask Savara. 

The Texas biotech $SVRA went look­ing for a sil­ver lin­ing as com­pa­ny ex­ecs blunt­ly con­ced­ed that Mol­gradex, an in­haled for­mu­la­tion of re­com­bi­nant hu­man gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor (GM-CSF), failed to spur sig­nif­i­cant­ly im­proved treat­ment out­comes for pa­tients with a rare res­pi­ra­to­ry dis­ease called au­toim­mune pul­monary alve­o­lar pro­teinosis, or aPAP.

Late-stage da­ta pre­sent­ed at the Amer­i­can Di­a­betes As­so­ci­a­tion an­nu­al meet­ing in 2010 pushed Eli Lil­ly to put a crimp on teplizum­ab as the phar­ma gi­ant found it un­able to re­set the clock on new­ly di­ag­nosed type 1 di­a­betes. At the same con­fer­ence but in dif­fer­ent hands nine years lat­er, the drug is mak­ing a crit­i­cal come­back by scor­ing suc­cess in an­oth­er niche: de­lay­ing the on­set of the dis­ease.

In a Phase II tri­al with 76 high-risk in­di­vid­u­als — rel­a­tives of pa­tients with type 1 di­a­betes who have di­a­betes-re­lat­ed au­toan­ti­bod­ies in their bod­ies — teplizum­ab al­most dou­bled the me­di­an time of di­ag­no­sis com­pared to place­bo (48.4 months ver­sus 24.4 months). The haz­ard ra­tio for di­ag­no­sis was 0.41 (p=0.006).