Min­er­va claims 'pos­i­tive' PhII da­ta on de­pres­sion drug while skep­tics push stock down

It might have come in a bit of an un­ortho­dox shape, but Min­er­va Neu­ro­sciences says it has a Phase II win in its hands.

When mea­sured against the bar of a 2-sided type I er­ror lev­el of 0.1, the biotech said, one of three test­ed dos­es of its de­pres­sion drug sel­torex­ant pass­es muster for a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment over place­bo. In their words:

The least squares mean (LS mean) dif­fer­ence from place­bo of the change in [Mont­gomery-As­berg De­pres­sion Rat­ing Scale] to­tal score at the end of week 6 was 3.1 for the 20 mg dose of sel­torex­ant, and the 2-sided p-val­ue was 0.083, which is be­low the pre-spec­i­fied 2-sided type I er­ror lev­el of 0.1.

Re­my Luthringer

The 20mg dose is the on­ly arm that Min­er­va was pre­pared to re­port on. In­ves­ti­ga­tors halt­ed en­roll­ment in the 40 mg group ear­li­er, while the 10 mg arm had too few pa­tients to be in­for­ma­tive.

That sounds like cher­ry pick­ing for some in­vestors, send­ing shares $NERV down 7% to $6.42.

Jef­feries an­a­lysts, on the oth­er hand, en­dorsed the rosy view: “We think con­fu­sion on ef­fi­ca­cy da­ta caused the ini­tial stock re­ac­tion, how­ev­er, the da­ta sug­gests a path for­ward w/ PI­II start­ing ear­ly ’20.”

In the tri­al, the oral pill was giv­en to adult pa­tients with ma­jor de­pres­sive dis­or­der along­side stan­dard an­ti­de­pres­sant ther­a­pies, in­clud­ing sero­tonin re­up­take in­hibitors (SS­RIs) and sero­tonin-nor­ep­i­neph­rine re­up­take in­hibitors (SNRIs), to which they have not re­spond­ed ad­e­quate­ly.

Min­er­va want­ed to boost the re­sponse rates (on­ly 30% to 40% by its count) by tin­ker­ing with the orex­in sys­tem, which is as­so­ci­at­ed with feed­ing, home­osta­sis, arousal, mod­u­la­tion of sleep-wake cy­cles and mo­ti­va­tion. Janssen signed on as a part­ner to sel­torex­ant, an ORX2 in­hibitor, on two in­di­ca­tions in 2014, but re­turned the rights for in­som­nia three years lat­er.

The amend­ed deal left Min­er­va sole­ly in charge of de­vel­op­ing sel­torex­ant as an in­som­nia treat­ment. It took the op­por­tu­ni­ty to high­light the con­nec­tion be­tween the two con­di­tions by sin­gling out one of 19 sec­ondary analy­ses: a sub­group with clin­i­cal­ly sig­nif­i­cant in­som­nia. Those pa­tients, who strug­gle to sleep, saw an LS mean dif­fer­ence ver­sus place­bo of 4.9 on the MADRS score with a 2-sided p-val­ue of 0.050.

Ac­cord­ing to the Jef­feries note, around 105 pa­tients be­tween the drug and place­bo arms be­longed in that group.

“While the 3.1 point dif­fer­ence in the over­all pop­u­la­tion does not ap­pear to dif­fer­en­ti­ate from oth­er an­ti­de­pres­sants ap­proved for ad­junc­tive use (2.8 point dif­fer­ence from pbo ob­served on avg), the 4.9 point dif­fer­ence in the pts with sig­nif­i­cant in­som­nia ap­pears clin­i­cal­ly mean­ing­ful,” the an­a­lysts wrote.

A sep­a­rate Phase IIb tri­al for in­som­nia has com­plet­ed en­roll­ment, CEO Re­my Luthringer added with topline re­sults ex­pect­ed lat­er this quar­ter.

Im­age: Shut­ter­stock

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.

As an­oth­er an­tibi­otics biotech sinks in­to a cri­sis, warn­ings of a sec­tor ‘col­lapse’

Another antibiotics company is scrambling to survive today, forcing the company’s founding CEO to exit in a reorganization that eliminates its research capabilities as the survivors look to improve on minuscule sales of their newly approved treatment. And the news — on top of an alarming series of failures — spurred at least one figure in the field to warn of a looming collapse of the antimicrobial resistance research field.

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'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).

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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