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.

The p-val­ue was an aw­ful 0.17.

Re­searchers blamed a high place­bo ef­fect, teas­ing out the pos­i­tive as­pects of an im­proved nu­mer­i­cal out­come for sec­on­daries on the 6-minute walk test and a need for whole lung lavage — a pro­ce­dure used to clean the lungs of pa­tients. Those sec­on­daries al­so failed the sta­tis­ti­cal analy­sis.

Bruce Trap­nell Cincin­nati Chil­dren’s

But that didn’t stop Savara from sug­gest­ing that a suc­cess­ful score on a pa­tient-re­port­ed qual­i­ty of life as­sess­ment/out­comes test could help set the stage for an ap­proval. 

“Tak­en to­geth­er, the trends in re­duc­tion of A-aDO2, as well as the im­prove­ment in the dif­fu­sion ca­pac­i­ty, are very con­sis­tent with re­duc­tion of the sur­fac­tant bur­den that caus­es the clin­i­cal symp­toms of aPAP,” said Bruce Trap­nell, the lead in­ves­ti­ga­tor. “Most im­por­tant­ly, the im­pres­sive im­prove­ment in qual­i­ty of life, as mea­sured by the SGRQ, sug­gest that Mol­gradex not on­ly im­proved ob­jec­tive mea­sures of oxy­gena­tion, but al­so had a clin­i­cal­ly mean­ing­ful ther­a­peu­tic ef­fect. I be­lieve these da­ta demon­strate that Mol­gradex can be­come an im­por­tant phar­ma­co­log­i­cal treat­ment op­tion for pa­tients with aPAP.”

In­vestors weren’t buy­ing it, though. The stock cratered on the num­bers, plung­ing more than 70% af­ter the bell on Wednes­day.

Im­age: Shut­ter­stock

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.

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).

A snap­shot of neg­a­tive da­ta from an on­go­ing 52-week mid-stage NASH study eval­u­at­ing CymaBay Ther­a­peu­tics’ lead drug has trig­gered alarm, af­ter the ex­per­i­men­tal liv­er drug, se­ladel­par, per­formed worse than a place­bo at a three-month read­out.

Sur­prised and aghast, in­vestors of the San Fran­cis­co-based biotech wast­ed lit­tle time in reg­is­ter­ing their dis­ap­point­ment. The com­pa­ny’s shares $CBAY plum­met­ed about 44.5% to $6.16 in ear­ly Tues­day trad­ing.