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.

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

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Game on: Re­gen­eron's BC­MA bis­pe­cif­ic makes clin­i­cal da­ta de­but, kick­ing off mul­ti­ple myelo­ma matchup with Bris­tol-My­ers

As J&J attempts to jostle past Bristol-Myers Squibb and bluebird for a landmark approval of its anti-BCMA CAR-T — and while GlaxoSmithKline maps a quick path to the FDA riding on its own BCMA-targeting antibody-drug conjugates — the bispecifics are arriving on the scene to stake a claim for a market that could cross $10 billion per year.

The main rivalry in multiple myeloma is shaping up to be one between Regeneron and Bristol-Myers, which picked up a bispecific antibody to BCMA through its recently closed $74 billion takeover of Celgene. Both presented promising first-in-human data at the ASH 2019 meeting.

FDA lifts hold on Abeon­a's but­ter­fly dis­ease ther­a­py, paving way for piv­otal study

It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.

Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

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Roche faces an­oth­er de­lay in strug­gle to nav­i­gate Spark deal past reg­u­la­tors — but this one is very short

Roche today issued the latest in a long string of delays of its $4.3 billion buyout of Philadelphia-based Spark Therapeutics. The delay comes as little surprise — it is their 10th in as many months — as their most recent delay was scheduled to expire before a key regulatory deadline.

But it is notable for its length: 6 days.

Previous extensions had moved the goalposts by about 3 weeks to a month, with the latest on November 22 expiring tomorrow. The new delay sets a deadline for next Monday, December 16, the same day by which the UK Competition and Markets Authority has to give its initial ruling on the deal. And they already reportedly have lined up an OK from the FTC staff – although that’s only one level of a multi-step process.

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KalVis­ta's di­a­bet­ic mac­u­lar ede­ma da­ta falls short — will Mer­ck walk away?

Merck’s 2017 bet on KalVista Pharmaceuticals may have soured, after the UK/US-based biotech’s lead drug failed a mid-stage study in patients with diabetic macular edema (DME).

Two doses of the intravitreal injection, KVD001, were tested against a placebo in a 129-patient trial. Patients who continued to experience significant inflammation and diminished visual acuity, despite anti-VEGF therapy, were recruited to the trial. Typically patients with DME — the most frequent cause of vision loss related to diabetes — are treated with anti-VEGF therapies such as Regeneron’s flagship Eylea or Roche’s Avastin and Lucentis.

UP­DAT­ED: Ob­sE­va makes case for best-in-class hor­mone sup­pres­sive ther­a­py in pos­i­tive uter­ine fi­broid study

About a month after the Swiss biotech disclosed a failed late-stage study in its IVF program, ObsEva on Monday unveiled positive pivotal data on its experimental treatment for heavy menstrual bleeding triggered by uterine fibroids.

ObsEva in-licensed the drug, linzagolix, from Japan’s Kissei Pharmaceutical in 2015. Two doses of the drug (100 mg and 200 mg) were tested against a placebo in the 535-patient Phase III study, dubbed PRIMROSE 2, in patients who were both on and off hormonal add-back therapy (ABT).

Ear­ly-stage can­cer biotech nails $85M C round; Flem­ming Orn­skov's Gal­der­ma scores 'break­through' sta­tus

→ Zentalis Pharmaceuticals just nabbed an $85 million round from a syndicate that includes Matrix Capital, Viking Global Investors, Redmile Group, Farallon Capital, Perceptive Advisors, Surveyor Capital and Eventide Asset Management. Their lead drug is ZN-c5, which is currently in Phase I/II trials. The biotech describes that drug as a “potential best-in-class oral Selective Estrogen Receptor Degrader for estrogen receptor-positive, HER2-negative (ER+/ HER2-) breast cancer.”