Vivek Ra­maswamy’s Urovant claims a big win in PhI­II — but ques­tions linger about a cheap gener­ic and their brand­ed ri­val

Af­ter the great Ax­o­vant im­plo­sion that swept away its high­ly tout­ed Alzheimer’s drug, Roivant chief Vivek Ra­maswamy need­ed one of the biotechs in his fast-grow­ing league of drug de­vel­op­ers to win a big Phase III tri­al. Based on to­day’s top-line re­lease for Urovant’s Phase III uri­nary in­con­ti­nence drug vibegron, the team is claim­ing that vic­to­ry and prep­ping an ap­pli­ca­tion for the FDA.

But there are some lin­ger­ing ques­tions about the cheap gener­ic that was used as an ac­tive com­para­tor in the study, and it’s dri­ving the biotech’s stock in­to the red Tues­day morn­ing.

Urovant’s drug hit sta­tis­ti­cal sig­nif­i­cance for both co-pri­ma­ry end­points and all 7 sec­on­daries when com­pared against a place­bo. But the drug was al­so test­ed along­side an old gener­ic drug called toltero­dine. And there the re­search showed that vibegron “achieved nu­mer­i­cal­ly bet­ter ef­fi­ca­cy that toltero­dine.”

In the slide show pre­sen­ta­tion this morn­ing, the com­pa­ny went on to il­lus­trate the specifics of the com­para­tor arms ver­sus place­bo, but re­searchers did not run a p val­ue for the Urovant drug against the gener­ic. But they were rel­a­tive­ly close, as you can see here:

Source: Urovant

Urovant’s drug cut in­stances of in­con­ti­nence by 2 from a base­line of 3.43, achiev­ing a clear win over the 1.4 drop in the place­bo arm. Toltero­dine, though, came much clos­er at 1.8. Both drugs beat the place­bo with sta­tis­ti­cal sig­nif­i­cance.

For uri­na­tion fre­quen­cy, there was a sim­i­lar spread. The place­bo arm hit a drop of 1.3, with a neg­a­tive 1.8 for vibegron and -1.6 for toltero­dine. Vibegron hit sta­tis­ti­cal dif­fer­ence ver­sus place­bo, toltero­dine did not. But there was no di­rect com­par­i­son of the ex­per­i­men­tal drug and the cheap place­bo.

In their an­nounce­ment back in 2012, Astel­las post­ed these re­spons­es for Myr­be­triq.

Myr­be­triq 25 mg, in­con­ti­nence episodes were re­duced by 1.36 episodes from a base­line of 2.65, a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion of 0.40 vs. place­bo in 12 weeks. The num­ber of uri­na­tions was re­duced by 1.65 uri­na­tions from a base­line of 11.68, a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion of 0.47 vs. place­bo in 12 weeks.

Myr­be­triq 50 mg, in­con­ti­nence episodes were re­duced by 1.49 episodes from a base­line of 2.71, a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion of 0.40 vs. place­bo in 12 weeks.  Num­ber of uri­na­tions was re­duced by 1.75 uri­na­tions from a base­line of 11.70, a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion of 0.55 vs. place­bo in 12 weeks.

You can be sure that an­a­lysts will al­so be fill­ing in the blanks by ex­trap­o­lat­ing the head-to-head com­par­isons them­selves.

Urovant’s pitch is built around demon­strat­ing a bet­ter safe­ty and ef­fi­ca­cy pro­file than the an­ti­cholin­er­gics — like toltero­dine, where there’s been a link with de­men­tia — and the block­buster drug Myr­be­triq (mirabegron), which was launched by Astel­las back in 2012. Vibegron is a be­ta-3 drug, like Myr­be­triq, which is backed by patents that ex­pire in 2023 — about 3 years af­ter Urovant could ex­pect an ap­proval.

Giv­en that this drug is in­tend­ed for a mass mar­ket with mil­lions of pa­tients, teas­ing out the specifics of how a new, brand­ed drug com­pares to well es­tab­lished mar­ket­ed drugs would be cru­cial to its longterm suc­cess or fail­ure on the mar­ket. 

Biren Amin

Urovant, which has a mar­ket cap of $442 mil­lion, is let­ting it all ride on this Phase III. Ra­maswamy nabbed the drug from Mer­ck with a small, $25 mil­lion up­front — one of a slate of deals he struck ear­ly on as he grabbed ther­a­pies that had been side­lined at the ma­jors and aimed them for quick cat­a­lysts.

Big gam­ble? Jef­feries’ Biren Amin not­ed that a clear win here would be worth a 100% spike in the stock, with a neg­a­tive out­come elim­i­nat­ing up to half of its mar­ket cap.

That’s not what hap­pened, though. Af­ter pop­ping up 20% be­fore the bell, the stock turned south, plung­ing to a mi­nus 17% as in­vestors tried to sort out the re­sults for them­selves.


Im­age: Vivek Ra­maswamy at the US-Chi­na Bio­phar­ma In­no­va­tion & In­vest­ment Sum­mit, 2018. PHARM­CUBE, END­POINTS

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.

Ted Love. HAVERFORD COLLEGE

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

'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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.