Months away from com­plet­ing a key sick­le cell tri­al, up­start Imara scores $63M in round co-led by Ar­ix, Or­bimed

A Cam­bridge, Mass­a­chu­setts-based sick­le cell dis­ease start­up that launched about three years ago has sparked the in­ter­est of UK life sci­ences com­pa­ny Ar­ix, which has agreed to co-lead a $63 mil­lion round of fi­nanc­ing for the drug de­vel­op­er Imara a few months ahead of the com­ple­tion of a key Phase II study.

Rahul Bal­lal

Imara burst in­to the biotech zeit­geist in June 2016 — as a col­lab­o­ra­tion be­tween or­phan drug ac­cel­er­a­tor Cy­dan De­vel­op­ment and Lund­beck — with $31 mil­lion in Se­ries A fund­ing from a slew of in­vestors, in­clud­ing New En­ter­prise As­so­ci­ates and Pfiz­er Ven­ture In­vest­ments. Since then it has en­list­ed the ser­vices of Rahul Bal­lal — who is tap­ping his busi­ness de­vel­op­ment ex­pe­ri­ence at North­ern Bi­o­log­ics and Flex­ion Ther­a­peu­tics and his VC chops from Ver­sant Ven­tures and No­var­tis Ven­ture Fund — in his role as CEO.

Imara is fo­cused on its sole ex­per­i­men­tal drug, IMR-687, for sick­le cell dis­ease (SCD) — a group of in­her­it­ed red blood cell dis­or­ders that large­ly af­flict those of African an­ces­try. SCD pa­tients have atyp­i­cal he­mo­glo­bin mol­e­cules, which can dis­tort red blood cells in­to a sick­le, or cres­cent, shape. Symp­toms such as ane­mia, re­peat­ed in­fec­tions and pe­ri­od­ic episodes of sear­ing pain be­gin to ap­pear in ear­ly child­hood. These episodes de­prive the body of oxy­gen-rich blood, which can cul­mi­nate in wide­spread tis­sue and or­gan dam­age, par­tic­u­lar­ly in the lungs, kid­neys, spleen, heart and brain, and dras­ti­cal­ly di­min­ish life ex­pectan­cy.

IMR-687 — an in­hibitor of phos­pho­di­esterase-9 (PDE9i) in blood cells — is de­signed to to treat the un­der­ly­ing caus­es of SCD by tar­get­ing the same bio­chem­i­cal path­way as stan­dard sick­le cell treat­ment (and chemother­a­peu­tic agent) hy­drox­yurea, but sans its safe­ty is­sues. Hy­drox­yurea makes the red blood cells big­ger, help­ing them stay rounder and more flex­i­ble — and makes them less like­ly to turn in­to a sick­le shape. In an­i­mal mod­els, IMR-687 has been shown to in­crease fe­tal glo­bin, sub­vert­ing the poly­mer­iza­tion of the sick­led he­mo­glo­bin — there­by re­duc­ing red blood cell sick­ling, red blood cell death and the oc­clu­sion of blood ves­sels. PDE9 in­hi­bi­tion al­so di­min­ish­es white blood cell “stick­i­ness” which fur­ther lessens the block­age of blood ves­sels, Imara said.

The drug is cur­rent­ly be­ing test­ed in a mid-stage study, which is ex­pect­ed to be con­clud­ed by June. Ini­tial da­ta is ex­pect­ed by the sec­ond half of this year, and the full read­out in the first quar­ter of 2020, an Ar­ix spokesper­son told End­points News.

Oth­er than hy­drox­yurea — which has been used for decades for sick­le cell — Em­maus Med­ical’s En­dari was ap­proved in 2017 for adult and pe­di­atric pa­tients to re­duce the acute com­pli­ca­tions of sick­le cell dis­ease. Oth­er de­vel­op­ers, in­clud­ing No­var­tis $NVS, Glob­al Blood Ther­a­peu­tics $GBT, CRISPR Ther­a­peu­tics $CR­SP/Ver­tex $VRTX and blue­bird bio $BLUE (whose gene ther­a­py-in-de­vel­op­ment gar­nered a flur­ry of at­ten­tion in a re­cent 60 min­utes episode) are al­so de­vel­op­ing drugs for the or­phan dis­ease that af­fects an es­ti­mat­ed 100,000 Amer­i­cans.

Mark Chin

The Se­ries B round for Imara was co-led by new in­vestors Ar­ix and Or­bimed Ad­vi­sors, and in­clud­ed the par­tic­i­pa­tion of RA Cap­i­tal and Rock Springs Cap­i­tal, as well as ex­ist­ing in­vestors NEA, Pfiz­er Ven­tures, Bay City Cap­i­tal, Lund­beck­fonden Ven­tures and Alexan­dria Ven­ture In­vest­ments. The in­jec­tion will be used for clin­i­cal de­vel­op­ment for the sick­le cell pro­gram, as well as broad­er ap­pli­ca­tions in be­ta tha­lassemia and oth­er haema­to­log­i­cal con­di­tions.

As part of the fi­nanc­ing, Ar­ix has com­mit­ted to in­vest $15 mil­lion for a 10% stake in Imara and Ar­ix’s in­vest­ment di­rec­tor Mark Chin will join Imara’s board, it said on Mon­day.

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.

Bain’s biotech team has cre­at­ed a $1B-plus fund — with an eye to more Big Phar­ma spin­outs

One of the biggest investors to burst onto the biotech scene in recent years has re-upped with more than a billion dollars flowing into its second fund. And this next wave of bets will likely include more of the Big Pharma spinouts that highlighted their first 3 years in action.

Adam Koppel and Jeff Schwartz got the new life sciences fund at Bain Capital into gear in the spring of 2016, as they were putting together a $720 million fund with $600 million flowing in from external investors and the rest drawn from the Bain side of the equation. This time the external investors chipped in $900 million, with Bain coming in for roughly $180 million more.

They’re not done with Fund I, with plans to add a couple more deals to the 15 they’ve already posted. And once again, they’re estimating another 15 to 20 investments over a 3- to 5-year time horizon for Fund II.

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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.


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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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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Adding mar­quee in­vestors, Black­Thorn bags $76M to back an AI-dri­ven strat­e­gy for pre­ci­sion neu­ro med­i­cine

As ar­ti­fi­cial in­tel­li­gence and ma­chine learn­ing loom ever larg­er in drug dis­cov­ery and de­vel­op­ment, a biotech op­er­at­ing at the “nexus” of tech­nol­o­gy and neu­ro­sciences has cashed in with $76 mil­lion in fresh fi­nanc­ing.

The big idea at Black­Thorn Ther­a­peu­tics is to do for neu­robe­hav­ioral dis­or­ders what ge­net­i­cal­ly tar­get­ed ther­a­py has done for on­col­o­gy: Re­de­fine pa­tient pop­u­la­tions by the un­der­ly­ing bi­ol­o­gy — dys­reg­u­lat­ed brain cir­cuits, or neu­rotypes — in­stead of symp­toms, there­by find­ing the pa­tients who are most like­ly to ben­e­fit at en­roll­ment phase.

Fol­low­ing CAR-T pi­o­neer­s' foot­steps, Tes­sa launch­es Chi­na JV in $120M deal

These days just about every biotech se­ri­ous about glob­al de­vel­op­ment — and not just com­mer­cial­iza­tion — has a Chi­na strat­e­gy. Tes­sa Ther­a­peu­tics, a Bay­lor as­so­ci­at­ed out­fit based out of Sin­ga­pore, is no ex­cep­tion.

Tak­ing a page out of the CAR-T pi­o­neers’ play­book, Tes­sa is es­tab­lish­ing a joint ven­ture with Chi­na-Sin­ga­pore Guangzhou Knowl­edge City, which is ini­tial­ly putting down $40 mil­lion for a 13% stake with $40 mil­lion more to come in a sec­ond stage. The biotech, which now re­tains an 87% con­trol, is al­so rolling out its own con­tri­bu­tions in two phas­es, start­ing with $20 mil­lion and all its tech­nol­o­gy li­cense rights for Chi­na.

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.

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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