Plot­ting to go toe-to-toe with heavy­weight Pfiz­er, lit­tle Ei­dos plans quick score in PhI­II AT­TR-CM study

Neil Ku­mar at an End­points event. (Jeff Ru­mans for End­points News)

So how does a small cap biotech go toe-to-toe with the likes of Pfiz­er, when the phar­ma gi­ant is lin­ing up for one of the most care­ful­ly watched FDA de­ci­sions in bio­phar­ma?

Ei­dos Ther­a­peu­tics $EI­DX thinks it has the an­swer as they play David to Pfiz­er’s Go­liath.

The biotech to­day out­lined its piv­otal strat­e­gy for AG10, an oral small mol­e­cule de­signed to treat transthyretin amy­loid car­diomy­opa­thy, AT­TR-CM. Work­ing with the FDA, the biotech came up with a Phase III tri­al de­sign that the ex­ecs in charge say gives them two po­ten­tial piv­otal dead­lines. The first ar­rives af­ter 12 months of treat­ment, as pa­tients are giv­en a 6-minute walk test. The next comes af­ter 30 months, as re­searchers eval­u­ate pa­tient da­ta on all-cause mor­tal­i­ty and fre­quen­cy of car­dio­vas­cu­lar-re­lat­ed hos­pi­tal­iza­tions.

That tri­al starts to­day. And Pfiz­er gets its FDA de­ci­sion — a like­ly yes — in Ju­ly. That puts them right up against Al­ny­lam, which got a pi­o­neer­ing OK for its RNAi ther­a­py last year.

Tri­al launch­es by and large don’t get a lot of cov­er­age. In this heat­ed de­vel­op­ment era we’re in, it’s hard to gain much at­ten­tion for your piv­otal time­line — es­pe­cial­ly if you’re play­ing catch-up. But Ei­dos hopes to pre­pare a case­book ex­am­ple of how to carve out a time­line that can give you a shot at com­pet­ing rel­a­tive­ly quick­ly with the heavy­weights of the world. And it’s worth a look as we be­gin to track their late-stage progress.

“If you are com­ing from be­hind with a bet­ter drug, you should get it to the mar­ket­place,” says Neil Ku­mar, who runs Bridge­Bio, the um­brel­la or­ga­ni­za­tion that con­trols Ei­dos and a slate of oth­er star­tups.

“It’s a pret­ty cool path,” says Ku­mar, “a 12-month place­bo-con­trolled tri­al, which is a great op­tion for pa­tients with no ac­cess to any small mol­e­cule sta­bi­liz­er.”

Ku­mar doesn’t want to talk price right now — no one does in this busi­ness ahead of an FDA ap­proval. But you can pret­ty much bet that if it can get out ear­ly with 12-month da­ta, pric­ing will be key if it wants to carve away at the fran­chise Pfiz­er will have al­ready built.

The phar­ma gi­ant has been un­der the gun on pric­ing, and just about every­one ex­pects they’ll come up with an ag­gres­sive 6-fig­ure price tag on their drug, once it’s ap­proved — par­tic­u­lar­ly if they can’t keep rais­ing the price af­ter it hits the mar­ket.

In ad­di­tion, a lot of these pa­tients will be on Medicare, and could be fac­ing a big out-of-pock­et ex­pense as long as the donut hole re­mains. If Ei­dos comes up with a much bet­ter price, they stand a bet­ter chance at win­ning over mar­ket share. And they can move in af­ter Pfiz­er helps iden­ti­fy the pa­tient pop­u­la­tion — which Ku­mar says is in the hun­dreds of thou­sands — through im­proved di­ag­no­sis.

“It’s al­most like Alzheimer’s of the heart, one of the biggest ge­net­ic mar­kets out there,” he adds.

Ei­dos shares got a 10% boost on the plan, but the biotech is play­ing a tough game, against tough odds. 

Pfiz­er’s mar­ket­ing ma­chine is one of the biggest and best in the busi­ness. Any new launch like this will be giv­en a top pri­or­i­ty. And they’ll have an ear­ly mover ad­van­tage, which in this mar­ket­place comes with a ton of built-in ad­van­tages.

Ei­dos al­so is hop­ing to score ear­ly with 12-month da­ta against the more sub­stan­tial 30-month out­come da­ta that Pfiz­er al­ready has on hand. Ku­mar coun­ters, though, that his team will be able to demon­strate im­proved ef­fi­ca­cy where it counts with the spe­cial­ists who mat­ter the most. Then they can fol­low up with ri­val 30-month da­ta to seal the deal.

There are, of course, no guar­an­tees that he’s right.

We’ll see how this plays out.

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.

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.


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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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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