Aptinyx's lead drug makes cut in small fi­bromyal­gia tri­al, set­ting the stage for larg­er chron­ic pain study

Four months ago, its lead drug fell short of hit­ting sta­tis­ti­cal sig­nif­i­cance in a di­a­bet­ic pe­riph­er­al neu­ropa­thy (DPN) study. On Mon­day, the new­ly-pub­lic Aptinyx re­vealed the ex­per­i­men­tal treat­ment has scored in a small mid-stage fi­bromyal­gia tri­al, paving the way for a larg­er study to be­gin in the sec­ond half of this year.

The drug — dubbed NYX-2925 — was de­vel­oped by the Evanston, IL-based biotech $AP­TX that went pub­lic last Ju­ly bank­ing on its ap­proach to mod­u­late NM­DA re­cep­tors, which are cru­cial to brain and ner­vous sys­tem func­tion.

Fi­bromyal­gia pa­tients suf­fer wide­spread mus­cu­loskele­tal pain ac­com­pa­nied by fa­tigue, sleep, mem­o­ry and mood is­sues — and re­searchers sus­pect that the dis­or­der am­pli­fies painful sen­sa­tions by af­fect­ing the way the brain process­es pain sig­nals. Fi­bromyal­gia is al­so con­sid­ered to be as­so­ci­at­ed with in­creased over­all lev­els of glu­ta­mate/glu­t­a­mine (Glx) in cer­tain brain re­gions, which is un­der­stood to cor­re­spond with en­hanced pain sever­i­ty.

NYX-2925 was test­ed in a 23-pa­tient sin­gle-blind study. In a se­quen­tial man­ner, but blind­ed to the pa­tient, all pa­tients re­ceived dai­ly dos­es of place­bo, 20 mg dose of the drug and 200 mg NYX-2925 for two weeks each. Over the course of the tri­al, pa­tients were test­ed for key brain ac­tiv­i­ty and neu­ro­chem­istry bio­mark­ers known to be as­so­ci­at­ed with per­cep­tion and pro­cess­ing of cen­tral­ized chron­ic pain.

Com­pared to place­bo, ad­min­is­tra­tion of NYX-2925 re­sult­ed in sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tions of Glx lev­els in these key pain-reg­u­lat­ing brain re­gions, and the use of the drug al­so re­sult­ed in re­duced con­nec­tiv­i­ty be­tween brain re­gions that are known to be as­so­ci­at­ed with the pro­cess­ing of cen­tral­ized chron­ic pain, Aptinyx said, adding that the pa­tient-re­port­ed sec­ondary end­points were al­so met. 

Daniel Clauw

“The re­sults with NYX-2925…com­pare very fa­vor­ably with the ef­fects of ap­proved fi­bromyal­gia drug treat­ments we pre­vi­ous­ly eval­u­at­ed in sep­a­rate and sim­i­lar stud­ies,” said Daniel Clauw, pro­fes­sor of med­i­cine at the Uni­ver­si­ty of Michi­gan and an in­ves­ti­ga­tor in the study. “(I)t is no­table that, de­spite the small num­ber of pa­tients in the study, the ef­fects on these clin­i­cal as­sess­ments of pain and oth­er symp­toms were al­so sta­tis­ti­cal­ly sig­nif­i­cant.”

Even though the DPN study did not meet its pri­ma­ry end­point, the com­pa­ny claims cer­tain dos­es of the drug did con­fer a nu­mer­i­cal im­prove­ment. Aptinyx says it plans to be­gin fol­low-up stud­ies in DPN and fi­bromyal­gia lat­er this year.

Exterior of the 1 million square foot Discovery Labs in Upper Merion, PA (PR Newswire)

Philadel­pha cham­pi­ons life sci­ences 'co-work­ing,' re­viv­ing for­mer GSK cam­pus in $500M makeover

In a boost to Philadel­phia’s thriv­ing life sci­ences scene, a for­mer Glax­o­SmithK­line cam­pus and a near­by site is get­ting turned in­to what its de­vel­op­er calls “the largest cowork­ing ecosys­tem” for health­care com­pa­nies in the coun­try.

The Dis­cov­ery Labs, a com­pa­ny spawned by MLP Ven­tures, has se­lect­ed two lo­ca­tions in the King of Prus­sia area as the $500 mil­lion test case for its strat­e­gy of ac­quir­ing and con­vert­ing old phar­ma­ceu­ti­cal R&D fa­cil­i­ties world­wide. The sites add up to 1.64 mil­lion square feet.

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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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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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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Savara shares are crushed as PhI­II tri­al flunks pri­ma­ry, key sec­on­daries — but they can’t stop be­liev­ing

In­vestors are in no mood to hear biotechs tout the suc­cess of a “key” sec­ondary end­point when the piv­otal Phase III flunks the pri­ma­ry goal. Just ask Savara. 

The Texas biotech $SVRA went look­ing for a sil­ver lin­ing as com­pa­ny ex­ecs blunt­ly con­ced­ed that Mol­gradex, an in­haled for­mu­la­tion of re­com­bi­nant hu­man gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor (GM-CSF), failed to spur sig­nif­i­cant­ly im­proved treat­ment out­comes for pa­tients with a rare res­pi­ra­to­ry dis­ease called au­toim­mune pul­monary alve­o­lar pro­teinosis, or aPAP.

Fu­eled by ear­ly suc­cess, Enan­ta paves way for mid-stage RSV study in adult pa­tients lat­er this year

RSV, a field lit­tered with fail­ure, has an­oth­er hope­ful.

On Fri­day, Enan­ta Phar­ma­ceu­ti­cals showed that its ex­per­i­men­tal treat­ment, EDP-938, con­ferred a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion in the res­pi­ra­to­ry syn­cy­tial virus (RSV) vi­ral load in healthy adults in­oc­u­lat­ed with the virus. Now comes the hard part.

“While we view to­day’s pos­i­tive re­sults from the Phase 2a hu­man chal­lenge study as en­cour­ag­ing, we an­tic­i­pate that re­sults from the Phase 2b will go a much longer way as to de­ter­min­ing whether EDP-938 is a vi­able treat­ment for RSV…We think the path to com­mer­cial­iza­tion here is par­tic­u­lar­ly dif­fi­cult to nav­i­gate, giv­en that RSV in­fec­tion re­solves fair­ly quick­ly with­out in­ter­ven­tion and the key will be show­ing a dif­fer­ence in terms of time to res­o­lu­tion,” Baird’s Bri­an Sko­r­ney wrote in a note.

'We kept at it': Jef­frey Blue­stone plots late-stage come­back af­ter teplizum­ab shown to de­lay type 1 di­a­betes

Late-stage da­ta pre­sent­ed at the Amer­i­can Di­a­betes As­so­ci­a­tion an­nu­al meet­ing in 2010 pushed Eli Lil­ly to put a crimp on teplizum­ab as the phar­ma gi­ant found it un­able to re­set the clock on new­ly di­ag­nosed type 1 di­a­betes. At the same con­fer­ence but in dif­fer­ent hands nine years lat­er, the drug is mak­ing a crit­i­cal come­back by scor­ing suc­cess in an­oth­er niche: de­lay­ing the on­set of the dis­ease.

In a Phase II tri­al with 76 high-risk in­di­vid­u­als — rel­a­tives of pa­tients with type 1 di­a­betes who have di­a­betes-re­lat­ed au­toan­ti­bod­ies in their bod­ies — teplizum­ab al­most dou­bled the me­di­an time of di­ag­no­sis com­pared to place­bo (48.4 months ver­sus 24.4 months). The haz­ard ra­tio for di­ag­no­sis was 0.41 (p=0.006).