Part­nered with No­var­tis, tiny Ca­dent jumps out of stealth with $40M round for its CNS pipeline

A small Cam­bridge, Mass­a­chu­setts-based com­pa­ny is hop­ing its bet on CNS drugs us­ing a well es­tab­lished phar­ma­ceu­ti­cal ap­proach — al­losteric mod­u­la­tors — will ce­ment its place as a biotech pow­er­house in the years to come. Ca­dent Ther­a­peu­tics — cur­rent­ly a 9-per­son team — has al­ready found a part­ner in No­var­tis $NVS.

The com­pa­ny is work­ing on treat­ments for move­ment and cog­ni­tive dis­or­ders us­ing al­losteric mod­u­la­tors, from which some of the biggest neu­ro­science prod­ucts, such as Xanax and Am­bi­en, have been de­vel­oped. On Thurs­day, Ca­dent said it had emerged “out of stealth mode” by an­nounc­ing a $40 mil­lion Se­ries B round of fund­ing, led by Cowen Health­care In­vest­ments and At­las Ven­ture. In­vestors such as Qim­ing Ven­ture Part­ners and No­var­tis In­sti­tutes for Bio­med­ical Re­search al­so par­tic­i­pat­ed in the round, which will be used to shep­herd two of the com­pa­ny’s ex­per­i­men­tal drugs in­to three Phase II tri­als.

Ca­dent is us­ing al­losteric mod­u­la­tors – which func­tion some­what like dim­mer switch­es – to tar­get ion chan­nels that are es­sen­tial to neu­ronal ac­tiv­i­ty. The biotech’s move­ment dis­or­ders pro­gram is fo­cused on the SK chan­nel – which reg­u­lates the amount of potas­si­um that en­ters a cell –  for the treat­ment of es­sen­tial tremor (ET) and spin­ocere­bel­lar atax­ia (SCA). Mean­while, Ca­dent’s sec­ond pro­gram is cen­tered on cog­ni­tive dis­or­ders. It em­ploys the use of al­losteric mod­u­la­tors to reg­u­late the the NM­DA re­cep­tor, which has been shown to play a crit­i­cal role in cog­ni­tion and de­pres­sion.

Bob Dagher

“We have both fla­vors of the NM­DA re­cep­tor, we can turn it up in the case of cog­ni­tive im­pair­ment, and we can turn it down, in the case of de­pres­sion,” chief Michael Cur­tis told End­points News. 

Ca­dent’s de­pres­sion pro­gram is part­nered with No­var­tis $NVS, so the Swiss drug­mak­er is tasked with com­pet­ing with Sage $SAGE, Al­ler­gan $AGN, John­son & John­son $JNJ and oth­ers in the de­pres­sion space, he added. With­out delv­ing in­to specifics, Cur­tis said the No­var­tis deal gen­er­ates non-di­lu­tive fund­ing for Ca­dent.

In the case of pos­i­tive al­losteric mod­u­la­tors for the NM­DA re­cep­tor, Ca­dent is tar­get­ing cog­ni­tive im­pair­ment as­so­ci­at­ed with schiz­o­phre­nia. What sets the com­pa­ny apart, ac­cord­ing to Cur­tis, is the use of an EEG (elec­troen­cephalo­gram) — that mea­sures brain elec­tri­cal ac­tiv­i­ty — as a bio­mark­er. An EEG deficit is be­lieved to be di­rect­ly re­lat­ed to NM­DA re­cep­tor hy­po-ac­tiv­i­ty, but us­ing this mea­sure as a bio­mark­er was pre­vi­ous­ly not a prac­ti­cal propo­si­tion be­cause sci­en­tists re­quired the use of a spe­cial­ized fa­cil­i­ty to con­duct the test in a clin­i­cal tri­al set­ting. Ca­dent has solved this prob­lem by part­ner­ing with a com­pa­ny called Cog­ni­sion that has de­vel­oped a portable EEG sys­tem, Cur­tis said.

“The the­o­ry is that if we can turn up the NM­DA re­cep­tor us­ing al­losteric mod­u­la­tors — we can re­store the EEG sig­na­ture, which is con­sid­ered to be a pre­cur­sor in im­prov­ing cog­ni­tion. That gives us a rapid way in a Phase II to look at tar­get en­gage­ment in the pa­tient pop­u­la­tion that we’re go­ing af­ter be­fore we ever have to ask the cog­ni­tion ques­tion.”

Tim An­der­son

“We have sev­er­al shots on goal in in­di­ca­tions that don’t re­quire huge cap­i­tal in­vest­ments – we’re talk­ing about a 2-week phase II in ET, a 12-week phase II in SCA. Maybe a hun­dred pa­tients tops in these tri­als, so for rel­a­tive­ly small cap­i­tal we can get the val­ue in­flec­tive points,” Cur­tis said.

De­pend­ing on the re­sults of these mid-stage stud­ies, which the com­pa­ny ex­pects to read­out over the next 12-18 months, Ca­dent is open to all pos­si­bil­i­ties, such as fur­ther cap­i­tal rais­es, an IPO, a part­ner­ship and a takeover, he added.

Ca­dent al­so an­nounced it had hired in­dus­try vet­er­an Bob Dhager, who has worked with Co­v­ance, Sanofi $SNY and Glax­o­SmithK­line $GSK, as their chief med­ical of­fi­cer and that Cowen’s Tim An­der­son, among oth­ers, had joined the com­pa­ny’s board.

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.

Evotec CEO Werner Lanthaler, File Photo

Ox­ford, Evotec ramp up LAB10x with AI ex­perts at Sen­syne — fo­cused on biotech spin­outs

Ox­ford is al­ly­ing it­self with Evotec and ar­ti­fi­cial in­tel­li­gence out­fit Sen­syne Health to ramp up some new biotech spin­outs while look­ing to “ac­cel­er­ate da­ta-dri­ven drug dis­cov­ery and de­vel­op­ment.”

The big idea here is that Ox­ford sci­en­tists — some of the best drug hunters in the world — can uti­lize Sen­syne’s AI plat­form for their work, re­ly­ing on the chemists and hands-on de­vel­op­ers at Evotec to push ahead to a crit­i­cal proof of con­cept mo­ment. And they’ll do it through a project leader called LAB10x, which gets £5 mil­lion over the next three years to fund the work.

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.

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.

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.

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.