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

UP­DAT­ED: In sur­prise switch, Bris­tol-My­ers is sell­ing off block­buster Ote­zla, promis­ing to com­plete Cel­gene ac­qui­si­tion — just lat­er

Apart from revealing its checkpoint inhibitor Opdivo blew a big liver cancer study on Monday, Bristol-Myers Squibb said its plans to swallow Celgene will require the sale of blockbuster psoriasis treatment Otezla to keep the Federal Trade Commission (FTC) at bay.

The announcement — which has potentially delayed the completion of the takeover to early 2020 — irked investors, triggering the New York-based drugmaker’s shares to tumble Monday morning in premarket trading.

Celgene’s Otezla, approved in 2014 for psoriasis and psoriatic arthritis, is a rising star. It generated global sales of $1.6 billion last year, up from the nearly $1.3 billion in 2017. Apart from the partial overlap of Bristol-Myers injectable Orencia, the company’s rival oral TYK2 psoriasis drug is in late-stage development, after the firm posted encouraging mid-stage data on the drug, BMS-986165, last fall. With Monday’s decision, it appears Bristol-Myers is favoring its experimental drug, and discounting Otezla’s future.

The move blindsided some analysts. Credit Suisse’s Vamil Divan noted just days ago:

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Suf­fer­ing No­var­tis part­ner Cona­tus is pack­ing it in on NASH af­ter a se­ries of un­for­tu­nate tri­al events

The NASH par­ty is over at No­var­tis-backed Cona­tus. And this time they’re turn­ing off the lights.

More than 2 years af­ter No­var­tis sur­prised the biotech in­vest­ment com­mu­ni­ty with its $50 mil­lion up­front and promise of R&D sup­port to part­ner with the lit­tle biotech on NASH — ig­nit­ing a light­ning strike for the share price — Cona­tus $CNAT is back with the lat­est bit­ter tale to tell about em­ri­c­as­an, which once in­spired con­fi­dence at the phar­ma gi­ant.

Dean Hum. Nasdaq via YouTube

Gen­fit goes to Chi­na with a deal worth up to $228M for NASH drug

Fresh off the high of its Nas­daq IPO de­but, and the low of com­par­isons to Cymabay — whose NASH drug re­cent­ly stum­bled — Gen­fit on Mon­day un­veiled an up to $228 mil­lion deal with transpa­cif­ic biotech Terns Phar­ma­ceu­ti­cals to de­vel­op its flag­ship ex­per­i­men­tal liv­er drug — elafi­bra­nor — in Greater Chi­na.

The deal comes more than a week af­ter Gen­fit $GN­FT is­sued a fiery de­fense of its dual PPAR ag­o­nist elafi­bra­nor, when com­peti­tor Cymabay’s PPARδ ag­o­nist, se­ladel­par, fiz­zled in a snap­shot of da­ta from an on­go­ing mid-stage tri­al. The main goal at the end of 12 weeks was for se­ladel­par to in­duce a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in liv­er fat con­tent, but da­ta showed that pa­tients on the place­bo ac­tu­al­ly per­formed bet­ter.

Fol­low­ing news of job cuts in Eu­ro­pean R&D ops, Sanofi con­firms it’s of­fer­ing US work­ers an 'ear­ly ex­it'

Ear­li­er in the week we learned that Sanofi was bring­ing out the bud­get ax to trim 466 R&D jobs in Eu­rope, re­tool­ing its ap­proach to car­dio as re­search chief John Reed beefed up their work in can­cer and gene ther­a­pies. And we’re end­ing the week with news that the phar­ma gi­ant has al­so been qui­et­ly re­duc­ing staff in the US, tar­get­ing hun­dreds of jobs as the com­pa­ny push­es vol­un­tary buy­outs with a fo­cus on R&D sup­port ser­vices.

Alex­ion wins pri­or­i­ty re­view for Ul­tomiris' aHUS in­di­ca­tion; FDA ex­pands ap­proval of Ver­tex's Symdeko

→ Alex­ion $ALXN has scored a speedy re­view for Ul­tomiris for pa­tients with atyp­i­cal he­molyt­ic ure­mic syn­drome (aHUS) af­ter post­ing pos­i­tive da­ta from a piv­otal study in Jan­u­ary. The drug is the rare dis­ease com­pa­ny’s shot at pro­tect­ing its block­buster blood dis­or­der fran­chise that is cur­rent­ly cen­tered around its flag­ship drug, Soliris, which is a com­ple­ment in­hibitor typ­i­cal­ly ad­min­is­tered every two weeks. Ul­tomiris has a sim­i­lar mech­a­nism of ac­tion but re­quires less-fre­quent dos­ing — every eight weeks. The de­ci­sion date has been set to Oc­to­ber 19. Late last year, Ul­tomiris se­cured ap­proval for noc­tur­nal he­mo­glo­bin­uria (PNH) pa­tients.

Bet­ter than Am­bi­en? Min­er­va soars on PhI­Ib up­date on sel­torex­ant for in­som­nia

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.

Gene ther­a­py biotech sees its stock rock­et high­er on promis­ing re­sults for rare cas­es of but­ter­fly dis­ease

Shares of Krys­tal Biotech took off this morn­ing $KRYS af­ter the lit­tle biotech re­port­ed promis­ing re­sults from its gene ther­a­py to treat a rare skin dis­ease called epi­der­mol­y­sis bul­losa.

Fo­cus­ing on an up­date with 4 new pa­tients, re­searchers spot­light­ed the suc­cess of KB103 in clos­ing some stub­born wounds. Krys­tal says that of 4 re­cur­ring and 2 chron­ic skin wounds treat­ed with the gene ther­a­py, the KB103 group saw the clo­sure of 5. The 6th — a chron­ic wound, de­fined as a wound that had re­mained open for more than 12 weeks — was par­tial­ly closed. That brings the to­tal so far to 8 treat­ed wounds, with 7 clo­sures.

Ab­b­Vie gets a green light to re­sume re­cruit­ing pa­tients for one myelo­ma study — but Ven­clex­ta re­mains un­der a cloud

Three months af­ter reg­u­la­tors at the FDA forced Ab­b­Vie to halt en­rolling pa­tients in its tri­als of a com­bi­na­tion us­ing Ven­clex­ta (vene­to­clax) to treat drug-re­sis­tant cas­es of mul­ti­ple myelo­ma, the agency has green-light­ed the re­sump­tion of one of those stud­ies, while keep­ing the rest on the side­lines.

The CANO­VA (M13-494) study can now get back in busi­ness re­cruit­ing pa­tients to test the drug for a pop­u­la­tion that shares a par­tic­u­lar ge­net­ic bio­mark­er. To get that per­mis­sion, Ab­b­Vie — which is part­nered with Roche on this pro­gram — was forced to re­vise the pro­to­col, mak­ing un­spec­i­fied changes in­volv­ing risk mit­i­ga­tion mea­sures, pro­to­col-spec­i­fied guide­lines and an up­dat­ed fu­til­i­ty cri­te­ria.