De­liv­er­ing CNS-fo­cused pa­tient in­no­va­tions

End­points Stu­dio Q&A with Kei­th Kendall, CEO of Aque­s­tive Ther­a­peu­tics

The route to be­com­ing the spe­cial­ty phar­ma com­pa­ny Aque­s­tive is to­day — with a mix of com­mer­cial­ized prod­ucts and in­ves­ti­ga­tion­al med­i­cines in the clin­ic — start­ed with a tech­nol­o­gy called Pharm­Film®. End­points pub­lish­er Ar­salan Arif spoke with CEO Kei­th Kendall about the com­pa­ny’s tech­nol­o­gy, its evo­lu­tion in­to a spe­cial­ty phar­ma­ceu­ti­cal com­pa­ny, and its ap­proach to bring­ing val­ue to pa­tients and in­dus­try part­ners.

End­points Stu­dio

Could you give read­ers a brief in­tro to Aque­s­tive?

Kei­th Kendall, Aque­s­tive

We use our tech­nol­o­gy to solve prob­lems that pa­tients have in­ter­act­ing with their pre­scribed med­ica­tions. We work to im­prove dose ac­cu­ra­cy, med­ica­tion ab­sorp­tion, and ad­dress med­ica­tion de­sign chal­lenges that fac­tor in­to poor ad­her­ence. By in­no­vat­ing treat­ment for­mu­la­tions, we try to im­prove the treat­ment ex­pe­ri­ence and out­comes for pa­tients.

End­points Stu­dio

Why the fo­cus on CNS?

Kei­th Kendall, Aque­s­tive

Al­though our Pharm­Film® tech­nol­o­gy is broad­ly ap­plic­a­ble, and we have projects out­side of CNS, we chose to fo­cus our ini­tial pipeline de­vel­op­ment ef­forts on this space for a cou­ple of rea­sons.

First, CNS pop­u­la­tions of­ten have dif­fi­cul­ty ad­her­ing to treat­ment and get­ting the op­ti­mal dose of a med­ica­tion. These pa­tients have a high­er in­ci­dence of dys­pha­gia — or dif­fi­cul­ty swal­low­ing. Some con­di­tions al­so lead to cog­ni­tive im­pair­ment, which can make pa­tients re­sis­tant to be­ing dosed. These are is­sues for care­givers and ul­ti­mate­ly for the pa­tient as well. Our tech­nol­o­gy and know-how can add a great deal of val­ue by pro­vid­ing med­ica­tions in high­ly dose-able forms that are suit­able for use in dif­fer­ent care set­tings.

Ad­di­tion­al­ly, as we trans­form our com­pa­ny in­to a spe­cial­ty phar­ma com­pa­ny that com­mer­cial­izes its own prod­ucts, the CNS space rep­re­sents a man­age­able call point for our field team. We can be more ef­fi­cient and build stronger com­mu­ni­ty re­la­tion­ships with a fo­cused sales force that car­ries mul­ti­ple prod­ucts with rel­e­vance for their cus­tomers. For ex­am­ple, our cur­rent prod­uct in the mar­ket, Sym­pa­zan, and what we hope to be one of our next prod­ucts, Liber­vant [a buc­cal film for­mu­la­tion of di­azepam that is still un­der in­ves­ti­ga­tion] are both treat­ments for epilep­sy – and have around a 90% over­lap in pre­scribers.

End­points Stu­dio

You men­tioned the trans­for­ma­tion of Aque­s­tive in­to a spe­cial­ty phar­ma­ceu­ti­cal com­pa­ny. Could you share some of the chal­lenges and op­por­tu­ni­ties be­hind that move?

Kei­th Kendall, Aque­s­tive

Aque­s­tive was orig­i­nal­ly formed back in the ear­ly 2000s on the the­sis that its tech­nol­o­gy could be li­censed and used to en­hance nu­mer­ous prod­ucts in the mar­ket. While part­ner­ing re­mains im­por­tant to us, we’ve al­so worked to evolve our busi­ness mod­el to dri­ve more growth and op­por­tu­ni­ty.

Af­ter some ex­am­i­na­tion, we rec­og­nized that bio­phar­ma com­pa­nies are re­ward­ed for three things: clin­i­cal, reg­u­la­to­ry, and com­mer­cial suc­cess. It’s dif­fi­cult for a con­tract man­u­fac­tur­er to con­trol these fac­tors. So, with the twin goals of cre­at­ing max­i­mum val­ue for our share­hold­ers and ful­ly ex­ploit­ing the strength of our tech­nol­o­gy and its ap­plic­a­bil­i­ty for pa­tients, we saw an op­por­tu­ni­ty to mi­grate in­to a spe­cial­ty phar­ma com­pa­ny.

Our plan fo­cused strate­gi­cal­ly on three things: One, de­vel­op­ing our own prod­ucts. Two, com­mer­cial­iz­ing those prod­ucts to max­i­mize the eco­nom­ic re­turn for the de­vel­op­ment work we do. And three, con­tin­u­ing to in­vest in the core tech­nolo­gies that make up our prod­ucts and en­hance their us­abil­i­ty for pa­tients.

In 2014, we em­barked on that path. And we see this strat­e­gy be­gin­ning to yield the re­sults we want­ed.

We’re very proud of our first com­mer­cial prod­uct and the ac­cep­tance it’s got­ten in the Lennox-Gas­taut syn­drome (LGS) com­mu­ni­ty. And, we’re look­ing for­ward to the po­ten­tial Liber­vant may of­fer pa­tients with clus­ter or break­through seizures, if ap­proved by the FDA.

The Epilep­sy Foun­da­tion es­ti­mates that as many as 3.4 mil­lion peo­ple in the U.S. have epilep­sy. A re­cent study in 300 adult pa­tients with ac­tive epilep­sy in­di­cat­ed that, when fol­lowed for a year, near­ly half had seizure clus­ters. Res­cue med­ica­tion was un­der­uti­lized in this pop­u­la­tion, but when used, it was as­so­ci­at­ed with few­er in­juries and emer­gency de­part­ment vis­its. The pri­ma­ry di­azepam prod­uct that’s avail­able to them now is a rec­tal gel, which many pa­tients sim­ply will not use. We’re in­ves­ti­gat­ing a nov­el, oral di­azepam treat­ment that’s de­signed for res­cue sit­u­a­tions. We’re very proud of the work that’s gone in­to re­search­ing this po­ten­tial ad­vance.

All of this re­flects our strat­e­gy com­ing to fruition.

End­points Stu­dio

Could you talk about your per­spec­tives on part­ner­ing? As you ap­proach in­dus­try and med­ical con­fer­ences, what kind of op­por­tu­ni­ties are you look­ing for?

Kei­th Kendall, Aque­s­tive

Aque­s­tive is built on a fun­da­men­tal prin­ci­ple around col­lab­o­ra­tion and part­ner­ing – work­ing to­geth­er to solve prob­lems and bring mean­ing­ful new med­i­cines to the mar­ket. And if you look at the prod­ucts in our port­fo­lio, you can see good ex­am­ples of that.

We be­lieve we have knowl­edge, tech­nol­o­gy, and in­tel­lec­tu­al prop­er­ty that are broad­ly ap­plic­a­ble – and may im­prove how med­ica­tions are used by pa­tients. But we’ll nev­er be able to in­vest in, and do jus­tice to, every op­por­tu­ni­ty on our own. As such, we seek part­ners that have a chal­lenge de­sign­ing pa­tient-friend­ly med­ica­tions or op­ti­miz­ing med­ica­tion ad­min­is­tra­tion. We be­lieve our ex­per­tise and tech­nol­o­gy can help ad­vance their med­ica­tions.

Sub­ox­one® is a great ex­am­ple of what our Pharm­Film tech­nol­o­gy can do for in­dus­try part­ners – cre­at­ing a strong fran­chise that ad­dress­es re­al needs in the mar­ket. In spite of both brand­ed and new gener­ic com­pe­ti­tion, that prod­uct con­tin­ues to be very suc­cess­ful. We’re very pleased that we cre­at­ed a prod­uct that has that much util­i­ty and that earned strong pref­er­ence among providers and pa­tients.

End­points Stu­dio

Switch­ing gears a bit, can you tell me a lit­tle bit about your­self and your back­ground? Can you tell me about how you got in­to the in­dus­try? I un­der­stand you’re not orig­i­nal­ly “from” bio­phar­ma?

Kei­th Kendall, Aque­s­tive

Yes, I’m not a “na­tive” bio­phar­ma ex­ec­u­tive. I spent the ear­ly part of my ca­reer in fi­nan­cial ser­vices. I sup­pose that the orig­i­nal in­vestors in this com­pa­ny rec­og­nized that there were two kinds of skills they need­ed at the ex­ec­u­tive lev­el. The first is sci­en­tif­ic ex­per­tise to de­vel­op prod­ucts the right way. The oth­er is busi­ness ex­pe­ri­ence — some­one fa­mil­iar with build­ing or­ga­ni­za­tions, rais­ing mon­ey and deal­ing with share­hold­ers.

This is the fifth start­up or turn­around com­pa­ny that I’ve led. So I un­der­stand how to build an or­ga­ni­za­tion that can ma­ture and grow, by tak­ing on more com­plex work — like Aque­s­tive.

I’m par­tic­u­lar­ly proud of the cul­ture we’ve cre­at­ed at Aque­s­tive. As our name im­plies, we are pas­sion­ate about “the quest” to cre­ate in­no­v­a­tive treat­ments that solve prob­lems pa­tients have tak­ing med­ica­tions in their cur­rent form. That work is com­plex. But we know that by work­ing to­geth­er, we can iden­ti­fy for­mu­la­tion ad­vances and de­sign med­i­cines that re­al­ly fit in­to pa­tients’ lives. And those are mean­ing­ful con­tri­bu­tions to their care.

Learn more about Aque­s­tive here.

Pa­tients should speak to their health­care provider to de­ter­mine what treat­ments are best for them. For more in­for­ma­tion, in­clud­ing im­por­tant safe­ty in­for­ma­tion, peo­ple should vis­it: https://www.sym­pa­

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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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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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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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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Ab­b­Vie touts new da­ta for Hu­mi­ra suc­ces­sor; Gilead inks dis­cov­ery deal

→ Ab­b­Vie is tout­ing new pos­i­tive da­ta com­par­ing their ag­ing block­buster Hu­mi­ra with their hoped-for block­buster upadac­i­tinib. Over 48 weeks a larg­er pro­por­tion of pa­tients tak­ing the ex­per­i­men­tal drug ex­pe­ri­enced clin­i­cal re­mis­sion than in the con­trol arm with Hu­mi­ra. Their drug brought in $20 bil­lion last year, top­ping the scales in the num­ber 1 slot.

→ Gilead has turned to Van­cou­ver-based Ab­Cellera for its lat­est dis­cov­ery deal. Ab­Cellera will use its know-how in “sin­gle-cell screen­ing of nat­ur­al im­mune sources” to find an­ti­body can­di­dates for Gilead to pur­sue in the in­fec­tious dis­ease field. The deal in­cludes an up­front and mile­stones.

Turns out, Rudy Tanzi did­n't see much of a sto­ry about a hid­den link be­tween En­brel and Alzheimer's ei­ther

The Wash­ing­ton Post man­aged to whip up the quick­est in­dus­try con­sen­sus I’ve ever seen that one of its re­porters was pur­vey­ing overblown non­sense with a sto­ry that Pfiz­er was sit­ting on da­ta sug­gest­ing that En­brel could be an ef­fec­tive treat­ment for Alzheimer’s. 

In cov­er­ing that bit of an­ti-Big Phar­ma fan­ta­sy — there are lots of rea­sons to go af­ter phar­ma, but this piece was lu­di­crous — I not­ed com­ments in the sto­ry from some promi­nent peo­ple in the field crit­i­ciz­ing Pfiz­er for not pub­lish­ing the da­ta. I sin­gled out Rudy Tanzi at Har­vard and then ap­plied some added crit­i­cism for the things he’s done to hype — in my opin­ion — high­ly ques­tion­able as­sump­tions. You can see it in the link.