Pox­el, part­ner Sum­it­o­mo Dainip­pon taste suc­cess in first piv­otal test for oral di­a­betes drug

The French meta­bol­ic ex­perts at Pox­el and their Asian part­ners at Sum­it­o­mo Dainip­pon Phar­ma have hit a home run with the first late-stage Japan­ese test of their oral di­a­betes drug imeglim­in, which bodes well for the pair of Phase III tri­als ex­pect­ed to read­out lat­er this year and fu­els the block­buster ex­pec­ta­tions tagged on the mi­to­chon­dr­i­al ther­a­py.

The drug be­longs to a new chem­i­cal class of oral agents called ‘glim­ins’ that si­mul­ta­ne­ous­ly tar­get all three key or­gans in­volved in di­a­betes: the pan­creas, the liv­er and the mus­cles. Imeglim­in is de­signed to work on the two main de­fects seen in pa­tients with type II di­a­betes: by in­creas­ing in­sulin se­cre­tion in the pan­creas, in a glu­cose-de­pen­dent man­ner; and by de­creas­ing the ex­cess pro­duc­tion of glu­cose by the liv­er, while en­hanc­ing ‘in­sulin sen­si­tiv­i­ty’ in the mus­cles.

Thomas Kuhn, Pox­el

The TIMES 1 tri­al test­ed a 1000 mg dose of imeglim­in against a place­bo in 213 Japan­ese pa­tients with type 2 di­a­betes. The drug-in­duced a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion (p<0.0001) in HbA1c — av­er­age blood sug­ar lev­els over a pe­ri­od of weeks/months — at week 24, meet­ing the main goal. The place­bo-cor­rect­ed mean change in HbA1c from base­line was – 0.87%, the com­pa­nies said.

The drug al­so met the main sec­ondary goal of re­duc­ing fast­ing plas­ma glu­cose, and analy­ses of oth­er sec­ondary end­points are on­go­ing.

With an ag­ing pop­u­la­tion and grow­ing rates of obe­si­ty, di­a­betes in Japan has bal­looned, with the num­ber of sus­pect­ed di­a­bet­ics hit­ting a record 10 mil­lion for the first time in 2016, ac­cord­ing to gov­ern­ment es­ti­mates cit­ed by Nikkei.

“The TIMES 1 re­sults con­firm the ro­bust ef­fi­ca­cy com­bined with fa­vor­able safe­ty ob­served in the Phase 2b tri­al in Japan and the po­ten­tial ben­e­fits that Imeglim­in can bring to type 2 di­a­betes pa­tients glob­al­ly,” said Pox­el chief Thomas Kuhn. “The TIMES 1 da­ta is the first ma­jor step to­wards fil­ing the Japan­ese New Drug Ap­pli­ca­tion in 2020. Japan rep­re­sents the sec­ond largest sin­gle mar­ket for type 2 di­a­betes and, Asia, in broad­er terms, is con­sid­ered the most im­por­tant ge­o­graph­ic lo­ca­tion with re­gards to treat­ing the di­a­betes pan­dem­ic in the fu­ture.

In 2016, Pox­el re­port­ed that a Phase IIb tri­al in Japan reg­is­tered he­mo­glo­bin A1c re­duc­tions of 0.52%, 0.94% and 1.00% for the 500 mg, 1000 mg and 1500 mg dose twice-dai­ly. In 2017, the Mer­ck Serono spin­out forked over the Asia rights to the treat­ment to Sum­it­o­mo Dainip­pon Phar­ma in a deal worth up to $300 mil­lion.

Da­ta from TIMES-2 and TIMES-3 are ex­pect­ed lat­er this year. Pox­el (EPA: $POX­EL) out-li­censed the rights to imeglim­in in the Unit­ed States and Eu­rope to Vivek Ra­maswamy’s Roivant Sci­ences last year, with this part­ner fund­ing the ma­jor­i­ty of the cost­ly Phase III pro­gram ex­pect­ed to com­mence this year.

“Imeglim­in has a nov­el mech­a­nism of ac­tion as a mi­to­chon­dr­i­al bioen­er­get­ics en­hancer, po­ten­tial­ly pro­vid­ing a unique po­si­tion in the com­pet­i­tive type II di­a­betes treat­ment land­scape,” Jef­feries an­a­lysts wrote in a note last month. They mod­eled a 60% prob­a­bil­i­ty of com­mer­cial suc­cess for imeglim­in in the US/EU and Asia, with an es­ti­mate of $3.1 bil­lion in glob­al peak sales, in­clud­ing $600 mil­lion in Japan.


Im­age: Shut­ter­stock

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.

Ted Love. HAVERFORD COLLEGE

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

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.

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.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

NASH con­tender CymaBay runs in­to trou­ble as mid-stage da­ta dis­ap­point

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.