Ele­phants can't jump? IDEA's Mike Rea says some Big Phar­ma play­ers are crush­ing old be­liefs

This is an in­dus­try that typ­i­cal­ly says ‘in­no­va­tion’ when what it means is ‘in­ven­tion.’ In­no­va­tion, as most purists will ar­gue, is about re­turn on in­ven­tion, an abil­i­ty to de­rive val­ue from pipeline. That is what our Phar­ma­ceu­ti­cal In­no­va­tion In­dex mea­sures — a com­pa­ny’s abil­i­ty to launch suc­cess­ful­ly, to add more val­ue to pipeline mol­e­cules than an­oth­er com­pa­ny would. It is ob­jec­tive, and about how well the past 5 years have gone, re­gard­less of how well the sto­ry has been spun.

This year took us by sur­prise. The idea that ‘ele­phants can’t jump’ may well be true bi­o­log­i­cal­ly, but it has been mis­ap­plied to large phar­ma, it seems. Where the trend had been to­wards small­er, more ‘ag­ile’ com­pa­nies in pre­vi­ous in­dices, this year the three fastest ris­ers were As­traZeneca, No­var­tis and Pfiz­er. This should give us all pause — if the nat­ur­al dis­rup­tion’ of the di­nosaurs was self-ev­i­dent, we’d be see­ing it. In­stead, they are fight­ing back, us­ing their scale, com­bined with wit, to take on the more buzz­wor­thy new guys.

The PII ranks com­pa­nies by their abil­i­ty to bring prod­ucts from Phase I/II to mar­ket and com­mer­cial­ize them suc­cess­ful­ly, and uti­lizes a range of clin­i­cal, reg­u­la­to­ry and com­mer­cial met­rics to do this, rang­ing from the cor­po­rate lev­el down to in­di­vid­ual prod­ucts.

The Pro­duc­tive In­no­va­tion In­dex (PII), now in its eighth year, pro­vides a sys­tem­at­ic and ob­jec­tive as­sess­ment of how well the top 30 com­pa­nies per­form in suc­cess­ful­ly bring­ing new med­i­cines to mar­ket and com­mer­cial­is­ing them.

The biggest shak­er this year is As­traZeneca, com­ing from the mid­dle of the pack in 2017 to take the 2018 PII Crown. Gilead al­so con­tin­ued their PII as­cent, be­com­ing the run­ner-up in 2018, ad­vanc­ing from 2017’s 3rd. John­son and John­son, hav­ing ced­ed the top spot last year af­ter a 4 year run on top, was able to re­gain some of the lost ground in 2018, land­ing in a joint 3rd spot with a rapid­ly-im­prov­ing No­var­tis.

1 As­traZeneca

How does a com­pa­ny cat­a­pult from the 15th spot in 2017 all the way to the top seat on the Phar­ma­ceu­ti­cal In­no­va­tion In­dex? A se­ries of strate­gic and suc­cess­ful de­ci­sions that helped gen­er­ate a sub­stan­tial amount of ro­bust pos­i­tive da­ta for their pipeline and be­gan to stem the com­pa­ny’s down­ward trend since the loss of sev­er­al prod­uct ex­clu­siv­i­ties be­tween 2011 and 2017. As­traZeneca man­aged to launch five sig­nif­i­cant new med­i­cines in 2017, across their pri­ma­ry ther­a­py ar­eas, in­clud­ing Imfinzi in ad­vanced blad­der can­cer, Calquence for r/r MCL, Qtern in T2D, Bevespi Aeros­phere in COPD, and fi­nal­ly Fasen­ra for se­vere asth­ma. Sig­nif­i­cant­ly, 2 of these 5 launch­es re­ceived ac­cel­er­at­ed ap­proval. In ad­di­tion to these new drug ap­provals, As­traZeneca ac­cu­mu­lat­ed 14 oth­er reg­u­la­to­ry ap­provals across in­di­ca­tion ex­pan­sion and LCM projects in ma­jor mar­kets. Over the past year AZ has al­so racked up 3 Break­through Ther­a­py Des­ig­na­tions (in­clud­ing 2 Or­phan Drug Des­ig­na­tions) and 6 ad­di­tion­al Pri­or­i­ty Re­view Des­ig­na­tions.

These reg­u­la­to­ry suc­cess­es are mir­rored strong­ly with a myr­i­ad of clin­i­cal tri­al suc­cess and ad­vance­ment. Based on key in­ter­nal guid­ance, the com­pa­ny has nar­rowed their R&D fo­cus to pro­vide deep­er re­sults from their pri­or­i­tized ther­a­peu­tic ar­eas, most no­tably on­col­o­gy. Short­ly af­ter Imfinzi re­ceived ac­cel­er­at­ed ap­proval in ad­vanced blad­der can­cer, AZ an­nounced pos­i­tive top-line re­sults for the Phase III PA­CIF­IC tri­al with Imfinzi demon­strat­ing su­pe­ri­or PFS in pa­tients with lo­cal­ly-ad­vanced, un­re­sectable NSCLC. In ad­di­tion to these ma­jor da­ta read­outs, AZ was able to bol­ster their pipeline with the ad­vance­ment of 14 phase II tri­als in 2017.

2017 wasn’t with­out its chal­lenges how­ev­er, as the MYS­TIC tri­al did not meet its pri­ma­ry end­point of im­prov­ing PFS com­pared to stan­dard of care (SoC) in PD-L1 >25% in pa­tients with 1st line NSCLC. How­ev­er, the MYS­TIC tri­al con­tin­ues as planned to as­sess the ad­di­tion­al pri­ma­ry end­point of over­all sur­vival – a da­ta read­out that is ac­cu­mu­lat­ed sig­nif­i­cant in­vestor in­ter­est. In fact, by the end of year EP Van­tage re­port­ed a 27% in­crease in AZ share price- demon­strat­ing both con­fi­dence in MYS­TIC’s OS out­comes and the bright fu­ture of As­traZeneca’s pipeline.


2 Gilead

Gilead saw an ex­cit­ing con­tin­u­a­tion of last years in­no­v­a­tive suc­cess, climb­ing to the 2nd spot on the PII for 2018. De­spite crum­bling rev­enues from block­buster Har­voni (a drop off of more than half, from $9.08 bil­lion in 2016 to $4.37 bil­lion in 2017) Gilead is re­port­ing net prod­uct sales be­tween $24.5 bil­lion and 25.5 bil­lion in 2017, up from a range of $22.5 bil­lion to $24.5 bil­lion. This in­crease in rev­enue comes from new prod­ucts, such as Vo­se­vi, as well as ex­ter­nal ac­qui­si­tions which drove in­creas­ing mar­ket share from their core strate­gic ther­a­peu­tic ar­eas.

Gilead has made a habit of en­ter­ing mar­kets with the goal of gen­er­at­ing step-wise change, ex­em­pli­fied by both Tru­va­da in HIV  and So­val­di in he­pati­tis C. When Gilead pur­chased Kite to join the CAR-T race, it’s clear their as­pi­ra­tion for game-chang­ing ther­a­peu­tics was a key dri­ver. In Oc­to­ber, Gilead was re­ward­ed for their jump in­to the cell ther­a­py mar­ket with the ap­proval of Yescar­ta for 3rd line treat­ment in large B-cell lym­phoma. Gilead has al­so con­tin­ued to back their CAR-T bet with ad­di­tion­al da­ta read­outs fea­tur­ing com­pelling long term da­ta in non-Hodgkin’s Lym­phoma and ag­gres­sive ex­pan­sion of treat­ment cen­ter ed­u­ca­tion and fa­cil­i­ta­tion, hop­ing to reach 80% of cen­ters over the next few years.

The huge in­vest­ment in Kite’s CAR-T was a bold and am­bi­tious move by Gilead. Some crit­ics ar­gue that the po­ten­tial is­sues with safe­ty, man­u­fac­tur­ing and oth­er re­al world ob­sta­cles to treat­ment made the $11 bil­lion dol­lar price tag too steep. Gilead hopes to over­come these crit­i­cisms by ex­pand­ing the la­bel through a wide breadth of hema­to­log­i­cal in­di­ca­tions and be­com­ing the hema­tol­o­gist’s first choice in CAR-T tech­nol­o­gy.

In 2017, Gilead’s Selon­sert­ib al­so con­tin­ued to ad­vance, ini­ti­at­ing a pair of phase III tri­als. Non-al­co­holic steato­hep­ati­tis (NASH) could rep­re­sent an enor­mous op­por­tu­ni­ty by 2020, with an ex­pect­ed an­nu­al mar­ket size pro­ject­ed be­tween $20 and $35 bil­lion. Da­ta from the late phase tri­als should be avail­able ear­ly in 2019.

Gilead’s de­ci­sion to con­tin­ue build­ing and de­vel­op­ing their HIV port­fo­lio af­ter the ac­qui­si­tion of Tri­an­gle Phar­ma­ceu­ti­cals (and Tru­va­da) led to con­tin­ued suc­cess when they could have oth­er­wise rest­ed on the lau­rels of their ini­tial suc­cess. Af­ter watch­ing its U.S. HIV mar­ket share grad­u­al­ly de­cline to just over 70% in Q3 2015, Gilead has seen a resur­gence to 78% mar­ket share in Q3 2017. These im­pact­ful strate­gic de­ci­sions have paved the way for Gilead’s suc­cess in 2017.

3 John­son & John­son

Al­so notch­ing a sin­gle rung high­er for the 2018 PII is John­son & John­son. Over the course of 2017, J&J had a strong show­ing of reg­u­la­to­ry mile­stones, clin­i­cal da­ta read outs and drug spe­cif­ic rev­enues. Strong growth in new prod­ucts in­clude Darza­lex, for the treat­ment of pa­tients with mul­ti­ple myelo­ma, Im­bru­vi­ca, for use in treat­ing cer­tain B-cell ma­lig­nan­cies, and Trem­fya, for the treat­ment of adults liv­ing with mod­er­ate to se­vere plaque pso­ri­a­sis.

Among the biggest stars of 2017 for John­son & John­son was Im­bru­vi­ca, which notched an enor­mous win with the first ever ap­proval in graft-ver­sus-host by the FDA. The suc­cess­es con­tin­ued to mount as suc­cess­ful long term phase 3 da­ta read­outs helped so­lid­i­fy the Im­bru­vi­ca mar­ket po­si­tion in Man­tle Cell Lym­phoma (MCL). Short­ly af­ter the da­ta was an­nounced in De­cem­ber, J&J re­ceived fur­ther good news when NICE re­versed their pre­vi­ous neg­a­tive opin­ion of Im­bru­vi­ca in MCL.

Ad­di­tion­al­ly, Darza­lex scored mas­sive phase 3 da­ta in front line myelo­ma, demon­strat­ing a 50% re­duc­tion in dis­ease pro­gres­sion or death in new­ly di­ag­nosed myelo­ma pa­tients. This mon­u­men­tal read­out, cou­pled with the ap­proval of Darza­lex (in com­bi­na­tion with dex­am­etha­sone and Cel­gene’s Po­m­a­lyst) for late line mul­ti­ple myelo­ma pa­tients, high­light a suc­cess­ful year for the ther­a­py, de­spite re­ceiv­ing a neg­a­tive opin­ion from NICE in March. 2017 end­ed – and 2018 be­gan – pos­i­tive­ly for the Darzelex brand team how­ev­er, with the FDA ac­cept­ing J&J’s sub­mis­sion and sub­se­quent­ly pri­or­i­tiz­ing the sBLA for their ther­a­peu­tic (in com­bo) for the treat­ment of new­ly di­ag­nosed mul­ti­ple myelo­ma pa­tients.

In ad­di­tion to growth from new prod­ucts, J&J man­aged to pre­serve Rem­i­cade rev­enues, no easy feat in a ever-ris­ing tide of biosim­i­lars. Over the last year, rev­enues de­clined to $6.32 bil­lion, a 9.3% de­cline from the pre­vi­ous year, but fared be­yond ex­pec­ta­tions with the launch of in­flix­imab biosim­i­lars en­ter­ing the US mar­ket. Ex-US has been a dif­fer­ent tale al­to­geth­er, with Rem­i­cade’s mar­ket share falling to 50% in the Eu­ro­pean mar­ket, which rais­es the ques­tion: how will the ad­vance of biosim­i­lars in the US in 2018 and be­yond af­fect rev­enues?

Al­though 2017 demon­strat­ed some ter­rif­ic suc­cess, the year was marred by the IL-6/IL-3 pro­gram fail­ures. The FDA re­jec­tion and fail­ure of the once-hope­ful block­buster can­di­date sirukum­ab and the dis­con­tin­u­a­tion of the phase 3 tri­al of ta­la­co­tuzum­ab were a low point of the 2017 year for John­son and John­son. To com­bat this pair of fail­ures, John­son & John­son need­ed a con­sid­er­able amount of help from the re­main­der of their es­tab­lished brands and pipeline to dig out – luck­i­ly for the big Phar­ma, many of their oth­er ther­a­peu­tic ar­eas were able to de­liv­er. Sales from John­son & John­son’s ‘World­wide Phar­ma­ceu­ti­cal’ unit tal­lied $36.3 bil­lion for the full-year 2017 – an in­crease of 8.3% ver­sus the pri­or year.


3 No­var­tis

Dur­ing the last year, No­var­tis jumped a con­sid­er­able 9 places and holds the third spot, ty­ing with John­son & John­son in the 2018 PII. Ma­jor dri­vers for this up­ward move­ment in­clude im­por­tant new prod­uct launch­es and ground­break­ing ad­vance­ments in per­son­al­ized can­cer ther­a­py.

No­var­tis reached three sig­nif­i­cant on­col­o­gy mile­stones in 2017, per­haps the most im­por­tant the sig­nif­i­cant mile­stone of achiev­ing the first FDA ap­proved chimeric anti­gen re­cep­tor T-cell (CAR-T) ther­a­py. Kym­ri­ah, an in­no­v­a­tive CAR-T ther­a­py was ap­proved in Au­gust of last year to treat chil­dren and young adults up to 25 years of age with acute lym­phoblas­tic leukemia (ALL). The com­pa­ny is now in the process of seek­ing ap­proval to ex­tend the im­muno­cel­lu­lar ther­a­py to treat adults with non-Hodgkin’s lym­phoma. CAR-T ther­a­py is an in­no­v­a­tive ap­proach which uti­lizes a pa­tient’s en­gi­neered healthy T-cells to tar­get and kill can­cer cells. Ad­di­tion­al­ly, Kisqali re­ceived FDA ap­proval as first-line treat­ment for (HR+/HER2-) ad­vanced or metasta­t­ic breast can­cer, and Ry­dapt was ap­proved in the US for treat­ment of FLT3- mu­tat­ed acute myeloid leukemia and three types of sys­temic mas­to­cy­to­sis.

Al­though No­var­tis faced gener­ic com­pe­ti­tion from patent ex­pi­ra­tion of its can­cer drug Gleevec/Glivec, the com­pa­ny’s com­mit­ment to strength­en­ing its on­col­o­gy port­fo­lio is ev­i­dent with their re­cent de­vel­op­ments and launch­es in this space. Ad­di­tion­al­ly, in an ef­fort to de­fend its pso­ri­a­sis drug Cosen­tryx from new en­trants, No­var­tis pub­lished phase 3 da­ta in­di­cat­ing that 44% of pa­tients had com­plete­ly clear skin af­ter one year of treat­ment and 41% main­tained clear skin in­to the 5th year. No­var­tis de­liv­ered a sol­id fi­nan­cial per­for­mance in 2017 with net sales of $49.1 bil­lion (up 2% in con­stant cur­ren­cies (cc)) and pro­gressed its pipeline by de­liv­er­ing 16 ma­jor ap­provals, 6 FDA break­through ther­a­py des­ig­na­tions, and 16 ma­jor sub­mis­sions.


5 Ab­b­Vie

Ab­b­Vie, mak­er of 2017’s glob­al top sell­er Hu­mi­ra, holds 5th place on the 2018 PII. In 2017, Ab­b­Vie’s de­liv­ered an ad­mirable fi­nan­cial per­for­mance with net rev­enues of $28.2 bil­lion, a jump from $25.6 in 2016. Hu­mi­ra brought in $18.4 Bil­lion in sales, an in­crease of 14.4 % on an op­er­a­tional ba­sis from 2016. Its run­ner up, Im­bru­vi­ca, for which Ab­b­Vie shares rights with J&J, brought in net rev­enues of $2.5 bil­lion, an in­crease of 40.5% from the pri­or year.

Ab­b­Vie’s mega block­buster Hu­mi­ra is ex­pect­ed to hold on to the num­ber one spot in sales for 2018 and is on track for reach­ing fore­cast­ed peak sales fig­ures of $21 bil­lion in 2020. This is large­ly thanks to last year’s set­tle­ment that was reached to stave off com­pe­ti­tion in the US from Am­gen’s FDA ap­proved Am­je­vi­ta un­til Jan 31st 2023. How­ev­er, Am­gen is ex­pect­ed to launch its biosim­i­lar in Eu­rope lat­er in 2018, where it may be­gin to steal away mar­ket share from Hu­mi­ra. Fol­low­ing its set­tle­ment with Am­gen, Ab­b­Vie filled a suit for patent in­fringe­ment against Boehringer In­gel­heim for its FDA ap­proved biosim­i­lar.

The com­pa­ny is ag­gres­sive­ly work­ing to­wards grow­ing its an­ti-in­flam­ma­to­ry port­fo­lio in prepa­ra­tion for fu­ture com­pe­ti­tion to Hu­mi­ra, which los­es ex­clu­siv­i­ty in 2023. They an­nounced top-line re­sults from a phase 3 study in­ves­ti­gat­ing a JAK1 se­lec­tive in­hibitor upadac­i­tinib (ABT-494) as a monother­a­py for the treat­ment of pa­tients with mod­er­ate to se­vere RA. In the study, 45% of pa­tients in the 15 mg and 53% in the 30 mg group had low dis­ease ac­tiv­i­ty by week 14 of treat­ment. Ad­di­tion­al­ly, the com­pa­ny re­port­ed pos­i­tive re­sults from a phase 3 study eval­u­at­ing IL-23 in­hibitor Risankizum­ab for treat­ment of pa­tients with mod­er­ate to se­vere plaque pso­ri­a­sis. Risankizum­ab achieved 84 to 88 per­cent re­spose rates of clear to al­most clear skin in pa­tients at week 16; a re­sponse rate sig­nif­i­cant­ly greater com­pared to STE­LARA (ustek­inum­ab) and HU­MI­RA (adal­i­mum­ab).

In par­al­lel, Ab­b­Vie is so­lid­i­fy­ing its pres­ence in on­col­o­gy with Im­bru­vi­ca, a drug orig­i­nal­ly de­vel­oped by Phar­ma­cyclics, which was bought by Ab­b­Vie in 2015 for $21 bil­lion. The drug re­ceived ex­pand­ed in­di­ca­tion out­side blood can­cer in 2017, when it land­ed FDA ap­proval for chron­ic graft-ver­sus-host dis­ease (cGVHD). This nov­el ap­proval made Im­bru­vi­ca the first drug to ever be in­di­cat­ed in this space. Ad­di­tion­al­ly, in on­col­o­gy, Ab­b­Vie’s phase 3 MU­RA­NO study eval­u­at­ing Ven­clex­ta/Ven­clyx­to (vene­to­clax) in com­bi­na­tion with Roche’s Rit­ux­an in pa­tients with re­lapsed or re­frac­to­ry chron­ic lym­pho­cyt­ic leukemia (CLL) met its pri­ma­ry end­point. Ven­clex­ta is FDA ap­proved for pa­tients with CLL with 17p dele­tion. How­ev­er, Ab­b­Vie has com­mit­ted ef­forts to­wards ex­pand­ing the role of Ven­clex­ta, seek­ing to be­come the go to ther­a­py  for pa­tients who de­vel­op re­sis­tance to first line treat­ment with Im­bru­vi­ca.


6 BMS

BMS holds the 6th place in the 2018 Phar­ma­ceu­ti­cal In­no­va­tion In­dex (PII). The com­pa­ny had a 7% growth in rev­enues from 2016, bring­ing in a to­tal of $20.8 bil­lion in 2017. Sales for Op­di­vo, its star im­muno-on­col­o­gy drug, in­creased by 31% with sales of $4.9 bil­lion in 2017. A flur­ry of ac­tiv­i­ty sur­round­ed Op­di­vo in 2017 as BMS tried to max­i­mize reach across glob­al mar­kets and ex­pand use of the ther­a­py across mul­ti­ple can­cer in­di­ca­tions, both as a monother­a­py and in com­bi­na­tion with Yer­voy.

Cur­rent­ly, Op­di­vo is ap­proved for treat­ment in 9 tu­mor types and 15 in­di­ca­tions. In ad­di­tion to this im­pres­sive la­bel, Bris­tol-My­ers Squibb has re­cent­ly re­leased da­ta that could lead to yet an­oth­er ap­proval in kid­ney can­cer, demon­strat­ing a 37% re­duc­tion in risk of death in pa­tients treat­ed with com­bi­na­tion of Op­di­vo plus Yer­voy. How­ev­er, the biggest win for Op­di­vo alone or in com­bi­na­tion (with Yer­voy) would be the high­ly sought FDA ap­proval in front-line treat­ment in non-small cell lung can­cer. In in­ter­na­tion­al mar­kets, the com­pa­ny filed a BLA for Op­di­vo to the Chi­na Food and Drug Ad­min­is­tra­tion and hopes to achieve this ap­proval in the Chi­nese mar­ket where lung can­cer is the most com­mon­ly di­ag­nosed can­cer.

In the car­dio­vas­cu­lar space, Eliquis (the an­ti­co­ag­u­lant drug that BMS shares rights to with Pfiz­er) has seen tremen­dous growth in 2017. Sales of the drug in­creased by 46%, reach­ing $4.9 bil­lion in 2017. The next gen­er­a­tion an­ti­co­ag­u­lant is on the way to dom­i­nat­ing this mar­ket by steadi­ly steal­ing away share from com­pe­ti­tion. Their rheuma­toid arthri­tis drug, Oren­cia, had the third high­est sales in 2017, fol­lowed by on­col­o­gy drug Spry­cel.

There is a lot to look for­ward to in 2018 for BMS. In on­col­o­gy, BMS con­tin­ues to ad­vance its port­fo­lio with nu­mer­ous stud­ies in phase 3. In im­muno­science, the com­pa­ny is quick­ly ad­vanc­ing its TYK2 in­hibitor in pso­ri­a­sis. Fi­nal­ly, in fi­brot­ic dis­ease their FGF21 for the po­ten­tial treat­ment of non-al­co­holic steato­hep­ti­tis (NASH).


7 Mer­ck

For Mer­ck, Keytru­da un­doubt­ed­ly has stolen the spot­light in 2017. Hav­ing land­ed three ap­provals last May, for use in front-line com­bi­na­tion ther­a­py with chemo for pa­tients with non-small cell lung can­cer (NSCLC), first and sec­ond-line ther­a­py in blad­der can­cer, as well as treat­ment of chil­dren and adults with mi­crosatel­lite in­sta­bil­i­ty-high (MSI-H) or a mis­match re­pair de­fi­cient (dMMR) sol­id tu­mors, Keytru­da’s sales re­flect­ed its ma­jor reg­u­la­to­ry suc­cess­es. In 2017 the PD-L1’s sales grew an im­pres­sive 172% from the pre­vi­ous year, bring­ing in $3.8 bil­lion in sales in 2017. Mer­ck re­port­ed end year sales of $40.1 bil­lion, over­all on­ly a 1% change from pri­or year.

Not all news was good news for Keytru­da in 2017 how­ev­er, in Ju­ly, Mer­ck an­nounced the Im­muno-On­col­o­gy drug fell short of de­liv­er­ing a sta­tis­ti­cal­ly sig­nif­i­cant ad­van­tage in over­all sur­vival over the cur­rent stan­dard treat­ment in a phase 3 study in head and neck can­cer. Lat­er in the year, the com­pa­ny’s am­bi­tious at­tempt to move Keytru­da in­to sec­ond line treat­ment for stom­ach-can­cer was shot down by the FDA af­ter the drug failed to meet its pri­ma­ry end­point in a phase 3 tri­al. In ad­di­tion, pa­tient deaths in two Keytru­da + chemo myelo­ma tri­als led the com­pa­ny to halt the tri­als and raised con­cerns in the med­ical and reg­u­la­to­ry com­mu­ni­ty.

In the vac­cines space Mer­ck con­tin­ues to strength­en its Gar­dasil fran­chise; a Mer­ck-fund­ed study found that the Quadri­va­lent HPV vac­cine Gar­dasil could pro­vide pro­tec­tion against vac­cine spe­cif­ic HPV types 6, 11, 16, and 18 for 10 years. Ad­di­tion­al­ly, in a large phase 3 tri­al the new­er vac­cine Gar­dasil 9 de­liv­ered pro­tec­tion in sub­jects for at least 6 years. On the oth­er hand, Mer­ck’s Zoster­vax was not viewed fa­vor­ably by a CDC ad­vi­so­ry com­mit­tee on im­mu­niza­tion prac­tices. The group vot­ed 8 to 7 in fa­vor of GSK’s Shin­grix over Zoster­vax, and fur­ther ex­pand­ed the rec­om­men­da­tion in peo­ple 50 years and old­er. In 2018, the com­pa­ny is ex­pect­ed to file for ap­proval for its Ebo­la vac­cine. Last Sep­tem­ber, the com­pa­ny an­nounced the ter­mi­na­tion of their He­pati­tis C treat­ment de­vel­op­ment ef­forts, but with nu­mer­ous pro­grams in phase 3, we’ll con­tin­ue to watch close­ly as they progress in 2018.


8 Roche

Roche had a steady flow of pos­i­tive news in 2017, dri­ving the Swiss drug­mak­er up 3 spots in this years PII rank­ings, to num­ber 8. Their up­ward trend can be at­trib­uted to a com­bi­na­tion of fac­tors, in­clud­ing: a sig­nif­i­cant num­ber of pos­i­tive tri­al read­outs, an im­pres­sive reg­u­la­to­ry per­for­mance and sol­id rev­enue growth.

Roche’s to­tal sales rose 5% in 2017 to $56.32 bil­lion. Both the phar­ma­ceu­ti­cal and di­ag­nos­tics di­vi­sions in­creased rev­enue by 5% from 2016. Key dri­vers of the phar­ma­ceu­ti­cal di­vi­sons growth came from new­er drugs, main­ly Ocre­vus, Tecen­triq, Per­je­ta and Ale­cen­sa, con­tribut­ing $1.48 bil­lion in new sales.

In 2017 Roche re­ceived a num­ber of key ap­provals, in­clud­ing two new med­i­cines, name­ly Ocre­vus for the treat­ment of re­laps­ing and pri­ma­ry pro­gres­sive mul­ti­ple scle­ro­sis, and Hem­li­bra for haemophlia A.  Roche al­so re­ceived a num­ber of ad­di­tion­al ap­provals for ex­ist­ing prod­ucts. These ad­di­tion­al ap­provals in­clude: Per­je­ta for ad­ju­vant, as well as for neoad­ju­vant, use in HER2-pos­i­tive breast can­cer, Ale­cen­sa for 1st line treat­ment in ALK-pos­i­tive NSCLC, Zelb­o­raf in Erd­heim-Chester dis­ease, Gazy­va for un­treat­ed ad­vanced fol­lic­u­lar lym­phoma, Lu­cen­tis for the treat­ment of all forms of di­a­bet­ic retinopa­thy, and Avastin for Glioblas­toma in adult pa­tients whose can­cer has pro­gressed.

Roche al­so re­ceived ap­proval in 1st line metasta­t­ic bad­der can­cer for its IO ther­a­py, Tecen­triq. How­ev­er, in a twist of fate, it lat­er went on to fail a Phase III tri­al in its 2nd line tri­al in UC.  Al­though the fail­ure has yet to re­sult in the re­moval of Tecen­triq, it did have dra­mat­ic neg­a­tive im­pacts on Tecen­triq’s UC mar­ket share. Some fear could this de­vel­op­men­tal mis­step could lead to fu­ture neg­a­tive im­pacts on the as­sets per­for­mance in oth­er large in­di­ca­tions, such as NSCLC, where there is lit­tle to clear­ly dif­fer­en­ti­ate Tecen­triq from the oth­er IO ther­a­pies.

Roche al­so had a num­ber of pos­i­tive clin­i­cal read outs in key ar­eas in 2017. Of par­tic­u­lar note were Roche’s pos­i­tive phase III IM­pow­er150 and IM­mo­tion151 stud­ies, which both met their pri­ma­ry end­point of PFS. The IM­pow­er150 study demon­strat­ed that the com­bi­na­tion of Tecen­triq and Avastin plus chemother­a­py sig­nif­i­cant­ly im­proved PFS com­pared to Avastin plus chemother­a­py in the first-line treat­ment of peo­ple with ad­vanced non-squa­mous non-small cell lung can­cer. The IM­mo­tion151 study demon­strat­ed that the com­bi­na­tion of Tecen­triq and Avastin pro­vid­ed a sig­nif­i­cant im­prove­ment in PFS in peo­ple who ex­pressed PD-L1 com­pared with suni­tinib for first-line metasta­t­ic re­nal cell car­ci­no­ma.  Both were sig­nif­i­cant as they help to bol­ster Roche’s newest po­ten­tial block­buster, Tecen­triq, while re­new­ing the util­i­ty of one of its his­tor­i­cal block­busters, Avastin, cre­at­ing a very val­u­ble can­cer-fight­ing dy­nam­ic duo. Oth­er pos­i­tive da­ta read outs for Roche in­clud­ed: pos­tive da­ta for Hem­li­bra, which de­liv­ered “clin­i­cal­ly mean­ing­ful” re­duc­tions in the num­ber of bleeds for he­mo­phil­ia A pa­tients, pos­i­tive da­ta for po­latuzum­ab ve­dotin in com­bi­na­tion with ben­damus­tine plus Rit­ux­an (BR) in r/r dif­fuse large B-cell lym­phoma and pos­titve da­ta for Ven­clex­ta plus Rit­ux­an for r/r chron­ic lym­pho­cyt­ic leukaemia (CLL).

De­spite a sol­id year of pos­i­tive news, Roche did face some chal­lenges in 2017. One such con­cern came from de­clin­ing sales of Tarce­va and Avastin. Avastin sales de­clined for the first time in the US, large­ly due to in­creas­ing use of new­er im­munother­a­py agents. Rit­ux­an/MabThera al­so suf­fered a 11% rev­enue de­crease in the EU in 2017, due pri­mar­i­ly to the en­try of biosim­i­lars.

Over­all, Roche de­liv­ered an im­pres­sive 2017 per­for­mance, with a re­mark­able set of new med­i­cines and exa­pan­sions in­to new in­di­ca­tions where true un­met needs ex­ist. As Roche looks to the fu­ture, they will con­tin­ue to try and re­ju­ve­nate their on­col­o­gy port­fo­lio with new, in­no­v­a­tive med­i­cines that can help take the place of for­mer block­busters.  Their cur­rent pipeline of on­col­o­gy prod­ucts is di­verse and should help in their ef­forts to dri­ve their con­tin­ued suc­cess in the com­ing years.


9 Pfiz­er

Pfiz­er had quite a pro­duc­tive 2017, jump­ing up 10 spots from their 2017 rank­ing (19), land­ing at num­ber 9 on this years PII. Their sol­id per­for­mance was the re­sult of sev­er­al fac­tors, in­clud­ing: sig­nif­i­cant progress on sev­er­al pipeline pro­grams, a decades best, ten ap­provals from the FDA, and con­tin­ued strength from sev­er­al core brands, in­clud­ing Ibrance, Eliquis and Xel­janz. No­tably, glob­al rev­enue for Eliquis in­creased by 43%, while glob­al Xel­janz rev­enue grew by 47% from 2016. Against these must be bal­anced in­creased com­pe­ti­tion for the im­munol­o­gy block­buster En­brel and a luke­warm per­for­mance by the com­pa­ny’s best-sell­ing prod­uct, pneu­mo­coc­cal vac­cine Pre­vnar 13. Over­all, to­tal rev­enue for Pfiz­er in­creased by 1%, with rev­enue from their In­no­v­a­tive Health Busi­ness up by 5% from 2016.

2017 was a par­tic­u­lar­ly strong reg­u­la­to­ry year for Pfiz­er, with a to­tal of 10 new ap­provals. Par­tic­u­lar­ly note­wor­thy was the ap­proval of their an­ti- PD-L1 im­muo-on­col­o­gy agent, avelum­ab, re­ceiv­ing ap­proval for the treat­ment of urotheial car­ci­no­ma (UC) and Merkel cell car­ci­no­ma (MCC), both com­ing with­in a span of two months. While Pfiz­er will face stiff com­pe­ti­tion as the 5th IO to mar­ket in UC, avelum­ab is the first and on­ly IO in MCC, mak­ing it a tru­ly in­no­v­a­tive ap­proval. Some of Pfiz­er’s oth­er key ap­proval this year in­clud­ed: Bo­sulif (bo­su­tinib), Steglatro (er­tugliflozin), a new in­di­ca­tion for Xel­janz (to­fac­i­tinib) in PsA, and a new in­di­ca­tion for Su­tent (suni­tinib malate) as an ad­ju­vant treat­ment in RCC.

Al­so of note, was Pfiz­er’s growth in emerg­ing mar­kets, with to­tal rev­enue growth at $1.1 bil­lion, or 11% from 2016. Pfiz­er showed a con­tin­ued com­mit­ment to emerg­ing growth mar­kets with deals like the one it made with Basilea Phar­ma­ceu­ti­cals. In the agree­ment, Pfiz­er was giv­en ex­clu­sive de­vel­op­ment and com­mer­cial­iza­tion rights for Cre­sem­ba (isavu­cona­zole), a nov­el an­ti­fun­gal med­i­cine in Chi­na and sev­er­al coun­tries in the Asia Pa­cif­ic re­gion.

Some of Pfiz­er’s biggest set­backs in 2017 came from loss­es of ex­clu­siv­i­ty, which neg­a­tive­ly im­pact­ed 2017 rev­enues by $2.1 bil­lion. This in­clud­ed loss of ex­clu­siv­i­ty for En­brel in Eu­rope, Pris­tiq and Vi­a­gra in the U.S., and Lyri­ca and Vfend in Eu­rope. A sec­ond note­wor­thy set­back came with Pfiz­er’s failed at­tempt to bol­ster the Lyri­ca fran­chise, when the FDA re­ject­ed Lyri­ca CR in the treat­ment of fi­bromyal­gia.

Pfiz­er’s biggest clin­i­cal set­back came with the fail­ure of avelum­ab in its phase 3 tri­al in ad­vanced stom­ach can­cer. Two of its reg­u­la­to­ry fail­ures in 2017 in­clud­ed the re­jec­tion of its Epogen biosim­i­lar and Xtan­di in breast can­cer.

Al­so of note in late 2017 was Pfiz­er’s de­ci­sion to end its in­ter­nal neu­ro­science dis­cov­ery and ear­ly de­vel­op­ment pro­gram. Like­ly a re­sult of decades of fail­ures, Pfiz­er de­cid­ed to shut down the pro­gram in or­der to re-al­lo­cate fund­ing to oth­er ar­eas where the com­pa­ny has stronger sci­en­tif­ic lead­er­ship.

Over­all, Pfiz­er had a pro­duc­tive 2017, with a decades-high 10 new ap­provals that should pro­vide sol­id ad­di­tion­al rev­enue for years to come. Look­ing ahead to 2018, Pfiz­er is hop­ing for more pos­i­tive out­comes in a se­ries of up­com­ing reg­u­la­to­ry de­ci­sions and clin­i­cal da­ta read­outs across their pipeline as they con­tin­ue to builds to­ward its next wave of in­no­v­a­tive treat­ments.


10 Bio­gen

De­spite an ap­pre­cia­ble year of rev­enue growth (up 7% from 2016), Bio­gen dropped from 1st in 2017, to 10th on this years PII. While still 2nd on the fresh­ness in­dex (per­cent of sales from drugs launched in the pre­vi­ous 5 years), a reg­u­la­to­ry set back in their core ther­a­peu­tic area (MS), a lack of pos­i­tive news, and strong per­for­mances from oth­er com­pa­nies led to Bio­gen’s drop on this year’s in­dex.

Bio­gen achieved 4% rev­enue growth in 2017, with over 90% of its rev­enue com­ing from its core MS busi­ness, de­spite an in­creas­ing­ly com­pet­i­tive en­vi­ron­ment in MS, lead pri­mar­i­ly by Tec­fidera.

A sig­nif­i­cant por­tion of this year’s growth al­so came from Bio­gen’s new­ly launched Spin­raza (ap­proved in De­cem­ber, 2016), which gen­er­at­ed over $880 mil­lion in rev­enue. The launch rep­re­sents one of the most suc­cess­ful rare dis­ease launch­es of all time, for which Bio­gen re­ceived the 2017 Prix Galien USA Award for Best Biotech­nol­o­gy Prod­uct.

2017 was al­so a year of re­new­ing fo­cus for Bio­gen. In Feb­ru­ary, Bio­gen com­plet­ed the spin-off of its he­mo­phil­ia busi­ness, Biover­a­tiv Inc., as an in­de­pen­dent com­pa­ny. In Oc­to­ber, Bio­gen ap­proved a cor­po­rate re­struc­tur­ing, in­tend­ed to cre­ate a lean­er and sim­pler op­er­at­ing mod­el. Both ef­forts were in­tend­ed to al­low Bio­gen to strate­gi­cal­ly re­al­lo­cate re­sources to­wards it’s core com­pe­ten­cies and emerg­ing growth ar­eas (all in neu­ro­science).

Bio­gen al­so made sig­nif­i­cant progress with its emerg­ing biosim­i­lar busi­ness in 2017. Benepali, an etan­er­cept biosim­i­lar, and Flix­abi, an in­flix­imab biosim­i­lar, both showed wide ac­cep­tance and adop­tion in the EU. In Au­gust, 2017 the EC al­so grant­ed mar­ket­ing au­tho­riza­tion for Im­ral­di, an adal­i­mum­ab biosim­i­lar.

Un­for­tu­nate­ly Bio­gen’s lat­est MS as­set, Zin­bry­ta (da­clizum­ab), failed to live up to its ex­pec­ta­tion of be­com­ing the next best fol­low-up ther­a­py for pa­tients who stop Tysabri (na­tal­izum­ab). The EMA de­cid­ed to sig­nif­i­cant­ly re­strict its use in pa­tients just one year af­ter its ap­proval due to safe­ty con­cerns (in March, 2018,  Bio­gen an­nounced a vol­un­tary world­wide with­draw­al of the prod­uct, though this was not fac­tored in­to this years PII). Though the as­set was not ex­pect­ed to be a ma­jor block­buster, this rep­re­sents a sig­nif­i­cant set­back for Bio­gen in it’s core ther­a­peu­tic area (MS).

Al­though Bio­gen faced some stag­na­tion in 2017, it is still well placed for fu­ture per­for­mance in the com­ing years. In 2017, Bio­gen added sev­en clin­i­cal pro­grams to its neu­ro­science pipeline, in­di­cat­ing con­tin­ued com­mit­ment to the fu­ture of its core busi­ness. Bio­gen’s most promis­ing pipeline as­set, ad­u­canum­ab, is cur­rent­ly in phase 3 clin­i­cal tri­als for pro­dro­mal AD. A pos­i­tive read­out as a DMT in AD would pro­pel Bio­gen clos­er to its stat­ed goal of be­com­ing the fastest grow­ing large cap biotech. Thus­ly, the next 12-18 months will see piv­otal da­ta read­outs that will have ma­jor im­pli­ca­tions on Bio­gen’s fu­ture suc­cess.


Fi­nal thoughts on PII per­for­mance…

Of the com­pa­nies out­side this year’s top 10 rank­ings, most had rel­a­tive­ly mod­est move­ments be­tween 2017 and 2018’s PII (with the ex­cep­tion of one). This like­ly speaks to the in­dus­try’s lack of dy­nam­ic risk tak­ing as a whole in 2017, with lit­tle in terms of dra­mat­ic cat­a­stro­phes or ma­jor par­a­digm shifts.

Take­da is the ex­cep­tion to this, hav­ing a pre­cip­i­tous fall from 5th in 2017 to 31st in this year’s PII. CEO Christophe We­ber warned of this po­ten­tial de­cline in the com­pa­ny’s mid-year state­ment when he stat­ed “we ex­pect head­winds in the sec­ond half of the year”. While a cou­ple of break­through des­ig­na­tions were seen, Take­da scored near the bot­tom on all key PII met­rics, in­clud­ing a dis­ap­point­ing year in terms of R&D, reg­u­la­to­ry ap­provals, key da­ta read­outs, HTA fail­ures and key fi­nan­cial mea­sures. Over­all, Take­da is one of the least ‘fresh’ com­pa­nies in terms of rev­enue from re­cent­ly launched drugs.

Al­ler­gan and Valeant were no­table new en­trants to this years PII, com­ing in at 16 and 18, re­spec­tive­ly. Al­ler­gan had a sol­id and steady year, with mid­dle-of-the-pack per­for­mances across all key PII met­rics. Valeant had a more mixed set of PII met­rics, with a very strong reg­u­la­to­ry per­for­mance (in the top 5), but a poor fresh­ness in­dex (one of the worst), which tak­en in com­bi­na­tion, pre­vent­ed it from mov­ing any high­er than 18th in its de­but on the PII.

The largest movers out­side the top 10 were Te­va, up 7 spots to 23rd, and Eli Lil­ly, up 8 places to 13th. Lil­ly was punch­ing above its weight in terms of ap­provals, R&D and reg­u­la­to­ry but with a low Fresh­ness In­dex and poor key fi­nan­cial met­rics. Te­va, by con­trast punched slight­ly above its weight in all met­rics bar NME ap­provals, where it was in the top 10.

In terms of the over­all PII, a key shift from 2016 to 2017 was the re­claimed su­prema­cy of big phar­ma in the top 10 rank­ings, push­ing out small­er com­pa­nies.

An­oth­er key trend of 2017 was the near record num­ber of NMEs ap­proved by the FDA in rare dis­eases and on­col­o­gy. While these in­no­v­a­tive med­i­cines are pro­vid­ing true clin­i­cal val­ue to pa­tients, they al­so come with an ex­or­bi­tant price tag and con­tin­ue to put ex­treme pres­sure on pay­er or­ga­ni­za­tions. A key ex­am­ple of this came in 2017 with the launch of Lux­tur­na, a sin­gle in­jec­tion for a rare oph­thalmic con­di­tion at the price of $425,000. While some can ar­gue that the price is jus­ti­fi­able giv­en that the in­ter­ven­tion quite lit­er­al­ly can cure blind­ness, it could be the next source of back­lash from both com­mer­cial pay­ers, gov­ern­men­tal sys­tems and the gen­er­al pub­lic. On­ly time will tell.


For more de­tails, vis­it http://ideaphar­ma.com/pii. Im­age: SHUT­TER­STOCK

UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

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J&J's block­buster Ste­lara wins US ap­proval for ul­cer­a­tive col­i­tis

J&J’s Stelara, which is set to be in the top ten list of blockbusters come 2025, is now cleared by the FDA for use in ulcerative colitis (UC), an inflammatory disease of the large intestine.

The biologic targets interleukin (IL)-12 and IL-23 cytokines, which are known to play a key role in inflammatory and immune responses. Stelara, which generated about $4.7 billion in the first nine months of 2019, is a key player in the crowded marketplace of drugs to treat autoimmune disorders such as psoriasis, rheumatoid arthritis and Crohn’s disease. AbbVie’s star therapy, Humira, continues to dominate, despite its looming patent cliff in the United States, while others including J&J’s $JNJ own anti-IL23 Tremfya, Lilly’s $LLY anti-IL-17 Taltz and AbbVie’s $ABBV recently approved anti-IL-23 antibody Skyrizi carve out a slice of market share.

Drug com­pa­nies reach $260M set­tle­ment just ahead of opi­oid tri­al; Oys­ter Point set terms for $85M IPO

→ Hours before the first federal opioid trial was set to begin, three drug distributors and an opioid manufacturer agreed to a $260 million agreement settlement, the Wall Street Journal was the first to report. The deal — which will see McKesson, Cardinal Health and AmerisourceBergen pay $215 million to Summit and Cuyahoga counties, and Teva deal out $35 million in cash and addiction treatments — does not resolve the pending, nationwide litigation that may result in a settlement worth upwards of $40 billion. Negotiators in that case, brought by 2,300 tribes, counties and cities nationwide and led by several states’ attorneys general, worked through much of Friday without success. Josh Stein, the attorney general for North Carolina, said they were trying to put together a $48 billion deal.

GSK of­floads two vac­cines in $1.1B deal as it works to re­vive the pipeline

GlaxoSmithKline is leaving the deep dark woods and its viruses behind.

GSK has agreed to divest its vaccines for rabies, RabAvert, and tick-born encephalitis vaccine, Encepur, to Bavarian Nordic, part of the company’s broader efforts to narrow its pipeline and focus on oncology and immunology.

The deal is worth up to nearly $1.1 billion, with a $336 million upfront payment. GSK acquired the vaccines from Novartis as part of an exchange for their late-stage oncology programs in 2015 under former chief Sir Andrew Witty.

Pfiz­er gets some en­cour­ag­ing PhI­II news on a fran­chise sav­ior, but is a dos­ing ad­van­tage worth the $295M up­front?

Close to 3 years after Opko tried to defend itself as shares tumbled on the news that its long-acting growth hormone had failed to outperform a placebo, the Pfizer partner $PFE is back. And this time they’re pitching Phase III data that demonstrate their drug is non-inferior — or maybe a tad better — than their well-known but fading standard in the field.
The comparator drug here is Genotropin, which earned a marginal $142 million for Pfizer last year — down 9% from the year before. Approved 24 years ago, biosimilars are now in development that Pfizer would like to stay out in front of. The market leader here is Norditropin, a growth hormone from Novo Nordisk that uses the same basic ingredient as Genotropin, which the Danish company sells with a kid-friendly self-injectable pen. That would also present some big competition if the new therapy from Opko/Pfizer makes it to the market.
The new data, says researchers, underscore that a weekly injection of somatrogon performed as well or slightly better than Genotropin (somatropin) in young children with growth hormone deficiency. Investigators tracked height velocity at 10.12 cm/year, edging out the older drug’s 9.78 cm/year. That 0.33 difference may not prove compelling to payers, though, who have been known to overlook dosing advantages in favor of lower costs.
That message may have weighed on the stock reaction this morning, with a 30%-plus hike $OPK giving way to more marginal gains.
Back in late 2016, Opko had to defend itself against a devastating Phase III setback as their initial late-stage trial failed against a sugar pill. Opko later blamed that setback on outliers in the study, though it wasn’t able to expunge the failure.

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IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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As­traZeneca's Farx­i­ga scores FDA nod to cut risk of hos­pi­tal­iza­tion for heart fail­ure in di­a­bet­ics

While the FDA recently spurned an application to allow AstraZeneca’s blockbuster drug Farxiga for type 1 diabetes that cannot be controlled by insulin, citing safety concerns — the US regulator has endorsed the use of the SGLT2 treatment to reduce the risk of hospitalisation for heart failure in patients with type-2 diabetes and established cardiovascular disease or multiple CV risk factors.

Sofinno­va-backed Abi­vax touts longer term mid-stage da­ta in ul­cer­a­tive col­i­tis

Two months after Abivax convinced Sofinnova to bankroll several mid-stage studies of its lead drug — ABX464 — with a €12 million stock purchase, the Paris-based biotech has rolled out more data on the anti-inflammatory molecule for all investors to see.

In a Phase IIa maintenance study involving 22 patients with moderate to severe ulcerative colitis who have been failed by previous treatments, 12 achieved clinical remission as assessed by endoscopy. But since only 19 completed the full one-year trial, 16 of whom had an endoscopy, investigators scored the remission rate at 75%. Although there’s no comparator arm, execs were pleased with improvements over an initial two-month, placebo-controlled induction phase by a number of measures ranging from remission to Mayo score and a fecal biomarker.

Alex­ion clinch­es aHUS ap­proval for Ul­tomiris as the clock ticks on Soliris con­ver­sion

Alexion has racked up a second approval for Ultomiris, the successor therapy to Soliris, as its mainstay blockbuster therapy faces a patent review process that could drastically shorten its patent exclusivity.

The FDA OK for atypical hemolytic uremic syndrome (aHUS) on Friday was widely expected after Alexion posted a full slate of positive Phase III data in January. But regulators also flagged concerns about serious meningococcal infections, slapping a black box warning on the label and mandating a REMS.