Glob­al fund­ing for ne­glect­ed dis­ease R&D hit record high in 2017 — with meaty con­tri­bu­tions by UK, EC, In­dia and Ger­many

About a decade ago, in the throes of the glob­al fi­nan­cial cri­sis when most coun­tries fo­cused their re­sources on stim­u­lat­ing their do­mes­tic economies, the Unit­ed States’ NIH over­whelm­ing­ly con­tributed to glob­al fund­ing for ne­glect­ed dis­ease R&D. But oth­er na­tions are now catch­ing up. In 2017, glob­al in­vest­ment for ne­glect­ed dis­eases hit record lev­els at $3.5 bil­lion — the largest in­crease since 2008 — dri­ven large­ly by the UK and the Eu­ro­pean Com­mis­sion (EC), along with In­dia and Ger­many, ac­cord­ing to a key sur­vey fund­ed by the Bill and Melin­da Gates Foun­da­tion.

The G-FIND­ER sur­vey is con­duct­ed by Pol­i­cy Cures Re­search, which came out with its first re­port in 2008, track­ing glob­al pub­lic, pri­vate, and phil­an­thropic in­vest­ment in­to prod­uct R&D for ne­glect­ed dis­eases, which chiefly im­pact de­vel­op­ing coun­tries and are not giv­en sig­nif­i­cant R&D at­ten­tion. For the 2017 re­port, 197 or­ga­ni­za­tions com­plet­ed the sur­vey, which en­com­passed 33 ne­glect­ed dis­eases and all rel­e­vant prod­uct types: drugs, vac­cines, di­ag­nos­tics, mi­cro­bi­cides and vec­tor con­trol prod­ucts from ba­sic re­search to post-ap­proval stud­ies.

Mil­lions of lives are plagued by in­fec­tious dis­eases like HIV/AIDS, tu­ber­cu­lo­sis, and malar­ia or oth­er par­a­sitic dis­eases, which dis­pro­por­tion­ate­ly af­fect low and mid­dle in­come coun­tries — where ac­cess to health care, drink­ing wa­ter and hy­gien­ic liv­ing con­di­tions is not typ­i­cal­ly guar­an­teed. In­vest­ment to di­min­ish the bur­den of pover­ty-re­lat­ed and ne­glect­ed dis­eases, and their as­so­ci­at­ed mor­bid­i­ty and mor­tal­i­ty, is im­per­a­tive to ex­tri­cate suf­fer­ers from the vi­cious cy­cle of pover­ty and dis­ease. Ac­cord­ing to the END fund, ne­glect­ed trop­i­cal dis­eases af­fect more than 1.5 bil­lion of the world’s most im­pov­er­ished peo­ple, in­clud­ing 836 mil­lion chil­dren, and kill more than 170,000 peo­ple each year.

Da­ta from the G-FIND­ER re­port showed that glob­al in­vest­ment in­to ne­glect­ed dis­ease R&D jumped 7% ($232 mil­lion) over 2016, and in­di­cat­ed for the first time since 2009 that there has been two con­sec­u­tive years of growth in glob­al fund­ing for ne­glect­ed dis­ease R&D.

Back in 2009, the NIH pro­vid­ed near­ly 98% of the net over­all rise in spend­ing and most of those re­sources were di­rect­ed to­ward aca­d­e­m­ic in­sti­tu­tions that typ­i­cal­ly fo­cus on ba­sic re­search, as well as small US-based bio­phar­ma com­pa­nies. In 2017, the in­jec­tion dri­ven by in­vest­ments from the UK, EC, In­dia and Ger­many al­so came from the pub­lic sec­tor, but that fund­ing was chiefly di­rect­ed at or­ga­ni­za­tions that fo­cus on clin­i­cal tri­als and prod­uct de­vel­op­ment.

In­vest­ments from emerg­ing fun­ders, such as Uni­taid, Médecins Sans Fron­tières, Gavi, and the gov­ern­ments of Japan, In­dia and Brazil were un­der­scored for 2016, and each of these en­ti­ty’s raised their fund­ing in 2017 with the ex­cep­tion of Brazil, where a cap was im­posed on pub­lic sec­tor fund­ing. In­dia sharply raised its fund­ing by 38% ($21 mil­lion) — and ac­count­ed for the fourth largest pub­lic fun­der over­all. Mean­while South Africa al­so upped its con­tri­bu­tion by 24% ($2.7 mil­lion) — the largest ever in­vest­ment as a slice of GDP pro­vid­ed by a low- and mid­dle-in­come coun­try.

In terms of high in­come coun­tries, siz­able in­creas­es by the UK (up 89% or $87 mil­lion) and EC (up 50% or $40 mil­lion) nar­rowed the gap be­tween sec­ond and third high­est con­trib­u­tors and the biggest con­trib­u­tor of all — the US — which al­so lift­ed its fund­ing mar­gin­al­ly by 1.5% ($23 mil­lion).

De­spite hit­ting record highs in fund­ing in 2017, not a sin­gle gov­ern­ment met the WHO rec­om­men­da­tion that mem­ber states ded­i­cate at least 0.01% of their GDP to re­search in­to the health needs of de­vel­op­ing coun­tries, the re­port high­light­ed. “On­ly two coun­tries – the US with 0.0082% and the UK with 0.0071% – were even close, with no oth­er coun­try even reach­ing half the tar­get lev­el. In fact, over the 11 year his­to­ry of the G-FIND­ER re­port, on­ly the US has ever met this tar­get (which it did be­tween 2007 and 2012),” the re­port not­ed.

Fund­ing by the bio­phar­ma in­dus­try inched low­er in 2017 for both big phar­ma and small drug and de­vice de­vel­op­ers, fol­low­ing five con­sec­u­tive years of growth. But this may be due to de­vel­op­ments in in­dus­try pipelines — for ex­am­ple the no­table rise and fall in malar­ia drug fund­ing was dri­ven by the late-stage de­vel­op­ment and even­tu­al ap­proval of GSK’s malar­ia vac­cine tafeno­quine, now sold as Krintafel.

HIV/AIDS, malar­ia and tu­ber­cu­lo­sis to­geth­er in­spired more than two-thirds (70% or $2,496 mil­lion) of 2017 fund­ing, in line with pre­ced­ing years. With a pletho­ra of treat­ments akin to a cure now avail­able for hep C, the drop in fund­ing rel­e­gat­ed the dis­ease to the group of ail­ments that get less than 0.5% of glob­al fund­ing each year.


Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

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.


Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

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.

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.

Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.

Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.

Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

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Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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Savara shares are crushed as PhI­II tri­al flunks pri­ma­ry, key sec­on­daries — but they can’t stop be­liev­ing

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.

As an­oth­er an­tibi­otics biotech sinks in­to a cri­sis, warn­ings of a sec­tor ‘col­lapse’

Another antibiotics company is scrambling to survive today, forcing the company’s founding CEO to exit in a reorganization that eliminates its research capabilities as the survivors look to improve on minuscule sales of their newly approved treatment. And the news — on top of an alarming series of failures — spurred at least one figure in the field to warn of a looming collapse of the antimicrobial resistance research field.

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'We kept at it': Jef­frey Blue­stone plots late-stage come­back af­ter teplizum­ab shown to de­lay type 1 di­a­betes

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).