Mount­ing a pre­lim­i­nary ef­fort to catch up with oth­er phar­ma gi­ants in the on­colyt­ic virus space, As­traZeneca has found a part­ner in France’s Trans­gene.

In the deal, the biotech — which is al­ready al­lied with Bris­tol-My­ers Squibb and Pfiz­er/Mer­ck KGaA on com­bi­na­tion pro­grams with its in-house ther­a­peu­tic vac­cines — will come up with five new armed on­colyt­ic vac­cinia virus can­di­dates. As­traZeneca is start­ing small with $10 mil­lion up­front and just $3 mil­lion in pre­clin­i­cal suc­cess mile­stones, but promis­es an undis­closed li­cense fee for each clin­i­cal can­di­date they ul­ti­mate­ly de­cide to co-de­vel­op.

Jean-Charles So­ria

The phar­ma gi­ant $AZN will al­so be able to choose the trans­genes to be en­cod­ed with­in the virus, a key ad­di­tion that pro­vides added fire­pow­er to the im­mune sys­tem against the tu­mor.

Long rec­og­nized as a tool to di­rect­ly kill can­cer cells, on­colyt­ic virus­es gained trac­tion in re­cent years af­ter re­search sug­gests that by burst­ing can­cer cells and re­leas­ing anti­gens with­in, they can al­so trig­ger an im­mune re­sponse. That makes them a po­tent im­munother­a­pies on its own as well as an ide­al pair­ing agent for the next gen­er­a­tion of I/O com­bos.

As­traZeneca first got its hands on an on­colyt­ic virus in 2015 when it inked a li­cens­ing agree­ment with Om­nis Phar­ma­ceu­ti­cals (now Vyr­i­ad), but it told Bio­Cen­tu­ry that “there are cur­rent­ly no on­go­ing AZ-spon­sored clin­i­cal tri­als of the can­di­date.”

Jean-Charles So­ria, SVP of As­traZeneca’s new­ly named on­col­o­gy unit, added they have “an ex­cit­ing port­fo­lio of mol­e­cules that we be­lieve may aug­ment on­colyt­ic virus ac­tiv­i­ty.”

Oth­er big play­ers such as Mer­ck, J&J, Pfiz­er and Boehringer have each bagged or seed­ed their own on­colyt­ic virus projects on the foot­steps of Am­gen’s T Vec, along­side biotech play­ers like PsiOxus, un­der­scor­ing the mo­men­tum of the next wave.

Mean­while, Trans­gene will con­tin­ue plough­ing on its in-house pipeline and promis­es to send a few more drugs to the clin­ic in 2020.

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).